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TRANSCRIPT
The Official Newsletter of Hemophilia of South Carolina
Upcoming Events
Year-End State Meeting and Holiday Celebration (Columbia) – December 3, 2016
YES Holiday Support Gather-ing (Greenville) – December 10, 2016
HSC 2017 State Advocacy Days (Columbia) – February 28—March 1, 2017
Featured Articles:
A Rocking Walk/Run Fundraiser HSC’s 5K Turkey Trot November 5, 2016 ~ Columbia
The weather was perfect; it was a brisk fall
morning, which made its way to a warm
sunshine filled day at Saluda Shoals Park for
the 5th Annual 5K Walk/Run for Bleeding
Disorders. The excitement was mounting the
last few days before the event, when the
Chapter reached its fundraising goal of
$40,000.00 and participant goal of over 300.
When all was said and done, $43,799.00
dollars was raised for the South Carolina
community with a donation from the
proceeds being provided to the NHF Judith
Graham Pool Postdoctoral Research Fund.
As kids and families gathered before the race
began, early morning snacks and beverages,
hot coffee, donated by Starbucks, and
yummy treats helped assure no one went
hungry before they would take off to make
the ½ mile short course or 3.1 mile official
5K.
The morning was spirited with the sounds of
a rocking DJ and Dreher High School’s
Marching Band, which was part of the Team
Spidermen/VBOA. The band entertained the
participants during the event and played the
national
anthem
just
before
the walk
was set
in
motion.
With Big Time Entertainment giving the
official go, runners quickly began the 5k,
with walkers, kids in strollers, dogs and the
band, pulling in the rear. Medals were
provided to all
participants
crossing the
finish line.
Finishing in
First Place was
Zack Davis;
Second Place
went to
William
Cannon with
the Third-Place medal given to Zy Miller.
Awards followed shortly after all returned,
and were provided as follows: The “Oldest
Walker”, Mrs. Mary Whitmire and the
youngest participant, just 14 months old.
The “Largest Team Walking” runner up went
to Team Fleming, and the “Top Walking
Team” was awarded to Team Spidermen/
VBOA. The “Largest Fundraiser Team” was
Team Fleming, and Team Free Bleeders was
a close second. The “Most Creative and
Original Individual Costume” was awarded
A Rocking Walk/Run Fund-raiser, page 1, 5 Featured Family: Meet the Hughes Family , page 10 PROB-ing Patients with He-mophilia , page 17
Volume 7, Issue 1 Fall 2016
Continued on page 5
Person to Person ■ Parent to Parent ■ Peer to Peer
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Person to Person Parent to Parent Peer to Peer
ᴥ About Hemophilia of South Carolina and The News Infusion ᴥ
Lead Article: HSC Turkey Trot
1, 5
About Us 2
Board’s Blog 3-4
Letter from the Executive Director
3
Community News
6-8
Community Voices
10-12
Have You Heard? 13-15
National and World News
17
Advocacy 18-19
Research 20-21
Resources and Contact Information
22
Calendar of Events 23
Inside this issue:
Executive Director Sue Martin
President Lisa Bordelon
Vice President Mike Walden
Secretary Felicia Alexander
Treasurer Patricia Tucker
Members Crystal Day Bonnie Phifer Edna Rabb Anita Rice Shawn Sperry
HSC MISSION
The purpose of this organization shall be to promote awareness of bleeding disorders to the general public; provide education and support to persons with bleeding disorders, as well as their families; and to support the research of the National Hemophilia Foundation.
HSC VISION
Hemophilia of South Carolina is to be recognized as a leading organization providing valued services; as ambassadors of public outreach to enlighten and foster an understanding of what matters most to those affected by hemophilia and bleeding disorders; and to be our community’s first choice in partnership to achieve their highest potential through empower-ment, connection to their community and being part of the solutions that affect them the most, until a cure is achieved.
About This Publication
The News Infusion’s mission is to provide communication, connections, education and advocacy awareness. It is published quar-terly by Hemophilia of South Carolina as an informational service to its members, friends, and affiliates of South Carolina’s bleeding disorders community. The News Infusion does not endorse any provider, com-pany or product, and further recommends that its readers always consult with their physicians and health care providers. The News Infusion is for educational and commu-nication purposes only and makes no claim to its accuracy. The News Infusion is en-dorsed by the South Carolina Hemophilia Treatment Center and the National Hemo-philia Foundation.
We encourage your feedback and communi-cations as a forum for exchange of infor-mation, ideas, and opinions. We welcome your letters, articles, questions and pictures. All articles may be published or distributed in print, electronic, online or in other forms. All submissions will be verified and subject to editing. Please send to the editor-in-chief at: [email protected]. Mail to: 439 Congaree Road, Suite #5, SC 29607, At-tention: Editor-in-Chief, The News Infusion. We look forward to hearing from you soon!
Hemophilia of South Carolina 439 Congaree Road, Suite #5
Greenville, SC 29607
Phone: 864-350-9941
Fax: 864-244-8287
www.hemophiliasc.org
Editor-in-Chief & Contributing Writer Sue Martin
Publisher/Editor Felicia Alexander
Contributing Writers Sue Martin, Hannah Gross, Jacob Sperry,
Tina and Cole Dean, Brandon Gross & Paige Hughes
Printing Services
A Chapter of the National Hemophilia Foundation
A Chapter Member of the Hemophilia Federation of America
A Registered Greenville County Charitable Organization
A Proud Member of the South Carolina Associations of Non Profit Organizations (SCANPO)
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From the Executive Director... It’s hard to believe we are coming to the end of the year. Where did it go? I remember the excitement I had waiting on New Year’s Eve at midnight (yes, I am that silly) for the launch of our new website on New Year’s Day, and how proud the Chapter was for all its resources and communications capabilities. I am sure you can imagine how exciting it was for us to receive the National Hemophilia Foundations award of Distinction in Communication for our new website at the 2016 National Hemophilia Foundation Annual Meeting and World Congress. I wish to thank Hieu Ngo, our staff webmaster, for working with us in our shared visions of communication. There is so much more that we want to add, so please log on often at www.hemophiliasc.org!
We have had a very busy year, and I will be proud to share a recap of our 2016 accomplishments at our annual year-end gathering, as well as present our visions for 2017. We have accomplished a lot this year
and have grown our membership immensely. We still have many things we envision for the future of HSC, and I am so hon-ored to work for such a wonderful community, YOU!---South Carolina. Equally, I am honored to work with the dedicated Board of Directors who share my vision of who we can become if we continue to work together. I feel a little giddy as I wait now for the new promo video we are currently producing with our new friends in partnership, Bryton Entertainment, LLC. Many of you may have been interviewed by them at the walk, or witnessed them recording. We will be excited to feature so many of our wonderful community members. My goal for the video is to be ready by January. Maybe a launch on New Year’s Day!!! (Oh the pressure is building up).
I wish to personally thank on behalf of myself and the Board of Directors, each and every community member, their friends, colleagues, businesses and local and national corporations for your support to the Chapter in fundraising and financial com-mitments. You make our job so much easier when we know we have the funds to support our community, especially when the calls come in. We are so proud to offer so many programs and services and look forward to our continuing, because of all of you. Thank YOU!
With the elections now over, I am grateful we have been preparing our community members on the importance of advocacy over the past several years. I know you will be ready, if the call is made to help us assure our access to quality care and treat-ment therapies continue. We have all learned that our voices and collective community strength is without boundaries in what we can achieve together, with hard work, and we will continue to work just as hard to keep what we currently have achieved. Yes, changes will ultimately come, as with life, there is always change. I ask you to walk with the Chapter, alongside our national partners in advocacy, get involved and take ownership of your healthcare. We will be working a great deal on advocacy in 2017, as we continue to be a leading voice for our South Carolina community. May this New Year be YOUR year, and may the sun shine brightly on you and your families.
With the warmest admiration for a wonderful community, Happy Holidays!
Sue
Additional ways to help HSC Go to www.hemophiliasc.org and click “Make a Donation” to learn more about how you can support HSC!
Register with Amazon Smiles. HSC receives 5% of the sale! Go to https://smile.amazon.com/
Donate through United Way! Go to http://www.unitedway.org/
Use Goodsearch.com for all your shopping needs. Go to http://www.goodsearch.com/nonprofit/hemophilia-of-south-carolina.aspx
Shop, sell, and donate for a good cause! Go to http://ebaygivingworks.com/
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Person to Person Parent to Parent Peer to Peer
2016 Donations, Grants & SponsorsA Speciality Advocate Pharmacy Gerald Lake Metromont
Accredo Express Scripts Greg Gibson, Gibson Retirement, LLC. Michael Ravan
Adam Choate Grifols Michelin North America
Amin and Aguilla Gaines Heather Daniels Midway Presbyterian Church
Anita Rice Hemophilia Alliance Foundation Miguelina Huerta
Anne Troy Hemophilia Federation of America Mike and Kim Walden
Anthony and Jamella Malcolm Houston Federal Credit Union Mr and Mrs Lucian R. Lee, JR.
Aptevo In Touch Pharmacy Mr and Mrs. R.Clifford Sperry / In honor of their son - Shawn Sperry
Baxalta, Inc. Jakeya M. Brown Mrs. Esters Mitch
Bayer HealthCare James and Lori Beaty Nancy Bassire
BDI Pharma, Inc. James Mancke National Hemophilia Foundation
Biogen James Whitmire Nettie Gass
BioRX Jane Beck Novo Nordisk
Blanche Rayfield Jason and Sammie Garris Octapharma
Bob Bushong, Bushong Industrials, Inc. Jean Dangelewicz Page Wiggins
Boeing Company Gift Match Program Donation /Mark Crisp Jeffrey Harper Paige Hughes
Bonnie and Tim Hill Jessi Rice Patricia Tucker
Bonnie Phifer Jessica Gonzalez Paula and Darrell Shewsbury
Brenda Garrett Jessico Ernst Peter and Patricia Korn
Brian Whitlock/ Automotive Exteriors Joey Merrell-Tires Unlimited Greer Pfizer Hemophilia
Caliber Collision John Tiller Rachelle Perkins
Calvin Carrion Johnnie Brasley Ric and Sue Martin
Camilo Rodas Johnston Orthodontic, LLC- Michael S, Johnston, DMD,MS Richard and Linda Ashmore
Carol Appelbaum Jon Gmitter Rimica Brown
Carolina Dental Group / Dr. Kevin and Scott Stoller Justin Feasel Rodney Hawley
Century Concrete of the Carolinia's, Inc. Kathy Brasley Seymore Myers, Jr.
Christopher Dietz Kedrion BioPharma Shannon King
Chuck Harvey/ SR Grading Keith Simmons Shari Young
CK Colburn Keenan Foundation, Inc Kennethy Myers Shawn Sperry
Connie Graham Kevin Lucas/The Bohemian Café Shire
Cristal Day Keystone Constructions St Lukes Lutheran Church
Crystal Bessette Kristen Callahan Stanley Clinkscale
Crystal Day Lawrence and Barbara Winkler Stephen and Lea Sorensen
CSL Behring Leesa Powers Synergy Homecare
CSL Behring Charitable Grant Lindsey Bartz Tamatha Deam
CVS Caremark Lindsey Stadler The Boeing Company Gift Match/Mark Crisp
Daniel Richter Lisa Bordelon The Durant Family in honor of Lucas Crisp
Danny Turpin Lois Geer The Wright Choice Associates, LLC
Diana Garrett Longs Drugs Tiful Mc Laughlin
Donation in Honor Of Jukavius Watkins Maddie Watkins Tim and Cheryl Belay
Drew Scott/All State Insurance Mahogany Lenz United Way of Anderson/Campaigns
DrugCo Healthcare Marilynn Bordelon United Way of Peidmont, Inc./Campaigns/Richard Lawson
Earle Walden Mark Crisp Viviana Kesel
Emergent BioSolutions Mark Elchelberger Wendy Dean
Evelyn Ivory Mark Henderson Wendy LeGrand
Felicia Alexander Market Place USA Federal Credit Union WG Yates
GA West and Company, Inc./ Mobile AL. Martix HealthCare William Ashley
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ᴥ Lead Article ᴥ
Thanks to our Sponsors & Volunteers!!! Our Title Sponsor: CSL Behring
Gold Sponsors: Aptevo, Bayer Healthcare, Biogen, Grifols, InTouch Pharmacy, Novo Nordisk, Octapharma, Pfizer Hemophilia
Our Bronze Sponsors: Accredo, CVS Caremark, Houston Federal credit Union, Market USA Federal Credit Union, Specialty Advocacy Pharmacy, Shire
Mile Sponsor: BioRX
In-Kind Donations: Carolina Rogers-Catchy Colours, Coca-Cola, Starbucks, Belk, BILO Groceries, Frito-Lay, Walgreens, Dana Pfeifer, LMT, Midland Biz and Bryton Entertainment, LLC
Special Thanks to our Walk Committee:
Sue Martin (Event Manager), Lisa Bordelon, Shawn Sperry, Patricia Tucker, Felicia Alexander, Bonnie Phifer, Cristal Day, Anita Rice, James Whitmire, Renita Johnson.
Thanks to the assistance of our walk day volunteers:
Suzanne Nash, April Baird and family, Ginger, Dillon and Justen Snead, Martha Breitwieser, Ms. Sims, Edna Rabb, Carolyn Tyler and Amauri, Jordan Smith, Jacob Sperry, Brandy Sperry, Kevin Bordelon, Matt Bordelon, Ashley Bordelon, Corbin Martin, Bryce and Jordan Martin.
HSC’s 5K Turkey Trot (continued from page 1)
to Cole Dean of Team
Factorbusters, and the team
costume award for the “Most
Original and Creative Team”
went to Team Hemoji’s. We
were excited to have over
twenty-five teams participating
in the competition fun, and a full
listing can be found on our
website walk
page.
The event
provided sponsor
tents, craft fun
and face painting
for the kids, t-
shirts, swag bags
and even a freshly baked piece of holiday pie. The walk
was a blast for all and we thank everyone who had a hand
in its success. We couldn’t do it without our sponsors, our
donors and everyone who supported their team’s efforts.
A very special thanks to the walk committee for their
dedication, and to our walk day volunteers. We wish to
send our sincere appreciation to Caroline Rogers, our
volunteer photographer, for capturing our memories with
walk day photos, now available for viewing on our
Facebook 2016 Walk/Run Album. Check them out!
Save the date for next year! November 4, 2017!
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The coastlines of South
Carolina are beautiful,
and a wonderful venue to
have a huge family
gathering. As we like to
think of our members as
one big family, the setting
was again perfect for this year’s educational family camp.
The weather held out with sunshine, and the Ocean Creek
Plantation Resort and Conference Center provided the
Villas for our 3 day, 2 night educational family camp
experience. Members throughout the state all gathered
for a weekend of learning, connecting, sharing and
empowering each other in a way that is just not possible
in a regular meeting setting. By the time the weekend was
over, most community members had met someone new,
learned something
different, and came
home with a feeling
that we are “Building,
One Strong
Community”!
Education was
provided by several national organizations. Hemophilia
Federation of America sponsored “Are you Smarter Than a
5th Grader” and “There’s No Place Like Home”, a
wonderful infusion clinic provided by Sue Geraghty, RN.
The National Hemophilia Foundation presented “To
Reveal or Conceal: Navigating Discloser” to our teens and
parents. Several sponsors also provided education; Shire
with “Staying Active” and “Understanding von Willebrand
Disease” with Nancy Roy, RN, and Aptevo, presented
“How not to Marry your Mother”, an active presentation
for our teens in transition.
Novo Nordisk and CSL Behring provided presentations on
School Communications and the “Gettin’ in the Game”
Junior Championships. We are always grateful to have
Inalex Communications with us, and James Stroker was
fabulous presenting “Creating your Goals and Living your
Vision”. Even the children received some education on
bleeding disorders provided by CVS Caremark and Ana
Garcia. Arts and Crafts were available with the painting of
HSC logo’s for teens, while children painted on canvasses,
enjoyed clay fun and more. The multitude of variety of
board games was a huge favorite for all ages. The famous
sand castle contest is always creative, with the competition
judged by the teens. A Saturday night party complete with
dancing, karaoke, ice cream, and popcorn ended the
educational day experience. Group breakouts and the
Turkey Trot walk Kick-off finished out the weekend on
Sunday, with a late checkout and good-byes.
With each family camp experience, our community grows
stronger. We look forward to next year’s event. Save the
Date for September 22-24, 2017 to join us again at the
Ocean Creek Resort and Conference Center in Myrtle Beach.
We wish to thank our sponsors, volunteers and speakers for
their support and assistance. We simply couldn’t present
this event to our membership without their support.
Moby Disk Sponsors:
Aptevo, Bayer
HealthCare, Biogen,
CSL Behring, Novo
Nordisk,
Octapharma, Shire
Great White Sharks:
Accredo, BioRX, CVS
Caremark, Matrix
Healthcare, Pfizer
Entertainment
Sponsor: Colburn-
Keenan Foundation
ᴥ Community News ᴥ
Keeping it Coastal Family Camp “Building One Strong Community”
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ᴥ Community News ᴥ
Spotlight on Unaffected Siblings
Ever wonder how hemophilia or bleeding disorders may impact unaffected brothers and sisters? In late August in Greenville,
early September in Columbia and mid-November in Fort Mill, HSC and Biogen partnered to provide an educational dinner event
to discuss raising multiple kids, especially if one child has a chronic condition like hemophilia. Balancing the needs of the child
with hemophilia can be a challenge on a family. The program was interactive and participants shared their experiences, made
suggestions, and all came home with some ideas on how to handle the needs of all children in the
family. We‘d like to thank Biogen for this opportunity to bring our community together in education and
support. We look forward to beginning again in 2017 to bring education to our member’s home towns.
Community Advocacy Training Sharing our Stories
Advocacy is in all of us. We just
don’t know it. When we speak up
for our children or loved ones’
needs in schools, physicians’
offices or in the work place, we are
advocating. On October 17th, in
Columbia, HSC partnered with
Shire to bring the community
together for a pre-advocacy
training to prepare for our March
1, 2017 State Legislative Days in
Columbia, with an educational
HELLO dinner, “Sharing our
Stories”, presented by Marvin
Pool, US Field Advocacy
Communications & Public Affairs
with Shire. Everyone has a story to
tell, and it is important to be able to share a strong story with those beyond the bleeding disorders community. This will help
build self-confidence and will increase awareness of bleeding disorders in general, and the need for access to high quality
healthcare. Marvin discussed the critical elements of a compelling story and helped attendees develop their own story through
effective communication skills. Sixty-four (64) community members were in attendance for the advocacy dinner, and we thank
Shire and Marvin for this wonderful dinner training! We hope to see everyone at our advocacy days in March!
Ladies, Let’s Talk About Hemophilia
We wish to thank Shire for our year-ending, “On the Road” educational dinner event held on November 15th in Columbia, to
discuss hemophilia in women. “Am I a symptomatic carrier or do I have hemophilia?”, The HELLO Talk provided an educational
format with a special emphasis on the experiences of women with hemophilia, and it addressed the unique set of challenges
women face. This conversation of a proper diagnosis for some women has brought about a deeper understanding of women
with hemophilia and the conversations and questions were many. We thank Nancy Roy, RN for her continued educational
presentations which help our community understand all aspects of hemophilia care and treatment,
both in men and women!
HSC’s takes Education “On The Road”
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ᴥ Community News ᴥ
Ladies Night Out October 18th, Joe’s Crab Shack
The fall season brought about the perfect background for a creative activity for 21 women and girls, all joined by a common
bond. They gathered together in Greenville to participate for dinner, some conversations of support and empowerment, and to
test their creative artistry. This is a wonderful way to de-stress after the busy summer activities. CSL Behring provided an
educational Common Factors Program which included an informative, interactive presentation from Lori Kunkel. The group
shared questions and supportive conversations and then hit the canvasses of wood. It was a fun night for all and something we
would love to do again. We send a special thanks and appreciation to Burlap and Ribbons and CSL Behring!
Sponsored in partnership with
Da BOYZ Night Out AMF Star Bowling Lanes, Greenville November 11th 6:00-10:00pm
“Da Boyz” Night Out Support Group activity in Greenville was a blast. Beginning with an Italian Buffett dinner, the activity
supported men and boys whose lives are affected by bleeding disorders. They gathered for conversations and support and then
they bowled. It is important for men and boys to also learn from one another and provide some ideas, thoughts, share
experience and provide support as they build relationships within the Chapter. Thanks to Grifols and Jeff for beginning the
conversations by sharing his story and for the rest of the group for their participation. Three hours of unlimited bowling was
enjoyed by the young and old.
Sponsored in Partnership with
YES Holiday Event December 10th, 11:00-3:00pm Children’s Museum of the Up-State
The YES Program Support Group comes to the Up-
State for a Holiday Season closing activity and you
are invited! Join Hemophilia of South Carolina and
members of the community for Empowerment,
Support and Friendships. Learn from others in the community. Share your support and experiences. Become engaged, and
meet a new friend. Provide your young children ages 0-12 years with the friendship of other young individuals affected by a
bleeding disorder in their family. Space is limited, so register early.
Lunch and tickets to the Children’s Museum for the rest of the afternoon will be provided to all participants. RSVP to the
Chapter at 864.350.9941 or [email protected] by December 5th.
Sponsored in Partnership with
HSC’s Support Group Activities
Yes They Can, Yes They Do, and
Yes They Will …
Up-State Support Group Activity
for Families with Young Chil-
dren and the newly diagnosed
WHERE: The Big Idea Conference Room
The Children’s Museum of the Upstate
300 College Street Greenville, SC 29601
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Annual Statewide Year-End Meeting and Holiday Celebration “Make it YOUR Year”
December 3, 5:30-10:00pm Embassy Suites / Greystone, Columbia
We hope you will be joining us for our final event of the year, our annual year-end gathering of the membership. The meeting will take a stroll back through 2016 and
celebrate our accomplishments, while hearing our visions for 2017 as we look forward to the New Year ahead!
Enjoy our Keynote presentation from Myles Ganley, a man affected by hemophilia who uses “Fitness to Fight Back”. Search for “Jingles”, our Elf on the Shelf, visit with
our sponsors, kids will enjoy holiday crafts, have fun with JAY- our balloon artist, and enjoy a visit from Santa. Story time fun, raffles and fellowship will provide something
for everyone. We have had a great year, let’s celebrate!
With Appreciation to our Contributing Sponsors: Speaker and Elf Sponsor ~ Aptevo
Craft and Elf Sponsor ~ CVS CareMark Elf Sponsors ~ Accredo, Bayer HealthCare, Biogen,
Cottrill’s Pharmacy, CSL Behring, Matrix Health, Novo Nordisk, Pfizer Hemophilia, Shire
Holiday Support ~ BioRX
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Our Featured Family: Meet the Hughes!
Last year, my then 6-month old daughter Annabelle was diagnosed with type 3 Von Willebrand Disease. We were stunned and had no idea where to begin! Annabelle was the first one in our families to be diagnosed with a bleeding disorder.
Our doctors gave us what information they had but even they had exceedingly limited experience with Type 3 VWD. We felt like no one really knew how to help us. In my search for more information I called many hospitals and experts. As Type 3 is relatively rare, we had no trouble finding people in the field who wanted to talk to us and /or meet us but they couldn’t really offer what we needed. We needed to know what it meant to live with a bleeding disorder. We went up to Columbia and it was the Social Worker there that suggested we contact Hemophilia of South Carolina. I’m so glad that we did!
Hemophilia of South Carolina really is like a family. Every meeting we’ve had the pleasure of meeting more families like us. We get to learn from their experiences so that we can give Annabelle the best life possible. We have learned so much. More than anything, we have come to realize that it’s not all that bad. She can have a normal life and do most anything she wants!
At the Family Camp at Myrtle Beach we learned about how much self-infusion can improve her quality of life. We learned how important it is for as many people as possible that are close to her, to know how to give her infusions. I’m excited to share that my husband and I have both given her infusions at the hospital and we are now teaching Annabelle’s grandmother, my mother in law, how to do it too! In the next few weeks we will have our own supply of medicine at home so we can treat her on our own time. It’s liberating to be able to spend a lot less time at the hospital! I would highly recommend learning how to infuse!
We look forward to getting to know even more of you and welcome you to share in our journey, as we hope to help in yours. God Bless Hemophilia of South Carolina and all of you!
The Hughes Family
Paige, Alexander,
George, Annabelle,
and Nikolas
Hello… My name is Hannah Gross, and I have a brother,
cousin, and grandfather who all have hemophilia. I have
known what it is my whole life. When people ask about
my life, I tell them about hemophilia, but they don’t know
what it is. I think that more people should know about
hemophilia and other bleeding disorders. We are trying to
spread the knowledge, but we are not reaching out to
enough people. Most Chapters have a 5K run/walk that a
lot of people go to. We raise a generous amount of
money to find a cure. There are also family camps, teen
retreats, dinners, and other get togethers. I try to attend
every gathering possible. I tell everyone I can about how
fun these are. I try to invite my friends, but can’t get
enough of them to come. I encourage other friends, co-
workers, and even distant family members.
Once a year, the North and South Carolina Chapters get
together and have a teen retreat at Camp Canaan. People
with bleeding disorders and their siblings are allowed to
attend, and they can also bring a friend. I have gone to
the retreat for two years and plan on going again for
years to come. We do many different activities that are
fun for everyone. We have ping-pong, Ga-Ga ball, archery
tag, bubble soccer, swimming, basketball, zip-lining, and
more. It is a fast and easy way to make new friends. I will
definitely miss this retreat but have to live in the
moment. There will be many more opportunities in the
future to make more friends and spread the word about
hemophilia.
Hannah Gross, High School Student
ᴥ Community Voices “Features” ᴥ
Our Teens: Meet Hannah
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Our Teens: Meet Jacob
My name Is Jacob
Sperry. I live in
Greenville, SC. I
am 15 years old
and my father has
hemophilia. I am a
sophomore at
Wade Hampton
High School. I am a member of the marching band and I
play the trumpet.
I have been playing the trumpet for five years now. In
band, I play only trumpet. I am a second chair player
which means I either play the higher part of the music or I
help the lower parts with sound quality. My father helps
with the marching band as well making props and
dragging trailers from competition to competition. Over
the summer, I started writing a book, I helped my dad
with yard work and I also went to the HSC teen camp at
Camp Canaan for the first time. The teen camp this
summer was very fun.
My dad stopped working about a year and a half ago
because his doctors and his muscles said he couldn’t do it
anymore. He worked construction for what was at the
time Ashmore Brothers. His uncle was a partial owner of
the company which made leaving even harder. Driving
dozers and heavy equipment was hurting him too much. It
was hard for him to get out of bed every morning.
I wish more people understood what hemophiliacs went
through every day. With muscle bleeds and thigh pain
that can be excruciating, people just don’t understand
what that is like. Some days it’s hard to even look at my
dad without tearing up, seeing him barely able to walk.
Pushing the accelerator in his truck hurts but the brake
hurts worse. I see it in his eyes. Every day I wake up with
the goal to try my best to make him feel better. I help him
by taking out the trash, bending down to get something
for him, or anything that may hurt him in the long run. My
dad and every other hemophiliac are fighters. They do not
and will not surrender and give up on trying to have
somewhat of a fun time.
Jacob Sperry, High School Student
ᴥ Community Voices “Features” ᴥ
Congratulations to Dr. James A. French,
Medical Director
at the South
Carolina
Hemophilia
Treatment Center,
Columbia, for his
participation in
the 2016 Turkey
Trot Walk. The
Treatment Center
was presented
with the Health
and Fitness
Hospital Cup
Award. Dr. French has participated in our past three
walks and we appreciate his supportive partnership. We
encourage all our healthcare partners in bleeding
disorders care to join us next year!
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MY EXPERIENCE AT THE JNC By: Tim and Cole Dean
I was chosen to represent Hemophilia
of South Carolina and compete in the
CSL Behring, 2016 “Gettin’ in the Game
Junior National Championship” in
Phoenix, Arizona. It was one of the
greatest experiences I have ever had.
They had three sports to compete in;
baseball, golf and swimming. I chose
baseball! On the first day we went to
the Mountain Vista baseball complex, and they had a
coaching clinic set up where we worked on the
fundamentals of baseball and proper techniques. The
coaches told us we always need to stretch before physical
activity and how staying active is very important.
The next day we went to the Big League Dreams sports
park in Gilbert, Arizona. It was here where we would
compete to raise money for our local chapter. It was so
cool to receive a bunch of high fives from everyone as we
got off the bus. I have never before played at such an
amazing ballpark! The fields were replicated after real
major league ballparks. I felt like a real big leaguer as I
stepped onto the field. They had music playing, and
people everywhere were cheering! It felt great! I was
coached up by two former baseball players in Jesse
Schrader and Pete Dyson! They were great! They gave me
some great tips and showed me how to position my feet
correctly at the plate and how to pitch properly.
Saturday night we had an awards celebration after dinner.
The dinner was delicious. I didn’t win a trophy for getting
1st place, but I was shocked to hear my name being called
as I received a medal for participating in the event. After
the awards were handed out they had ice cream and
music with a dance floor. I danced away the rest of the
night with all the friends I met at the JNC!
Aside from the competition, I really enjoyed swimming in
the pool at the Wild Horse Pass Resort that we were
staying in. The pool had a water slide, which was
awesome! I also really liked sitting in the hot tub at night
before going back to the room for the night. Also, nearby
the resort, my Dad took me to a place called “Rawhide.”
It is a small western town and nothing but cowboys and
cowgirls live there! It was one of my favorite things we
did! They had a gun show, a train, rides, and we went
panning for gold, and they had a jail house where they
will make you an official sheriff’s deputy for the day. I had
such an awesome time at the JNC and I will never forget
it! Thank you, Hemophilia of South Carolina, for choosing
me for this wonderful experience.
Brandon Gross:
I was lucky enough to go
to JNC for a second year
in a row for baseball.
This year I met two
major league pitchers:
Chris Capuano, he’s my
favorite pitcher, and
Kameron Loe. Both
pitchers played for the
Milwaukee Brewers, which is where we are originally
from. Kameron Loe pitched to us for the batting
competition. I also won the competition for my age
group! In addition to meeting families from all over the
United States who also deal with hemophilia on a daily
basis, I was able to spend time with my cousin, who also
has hemophilia, and my aunt as
they were selected to represent the
Wisconsin Chapter. While I would
love to go to JNC many more times,
I encourage other kids to apply next
year for this amazing experience.
ᴥ Community Voices “Features” ᴥ
Voices of the Junior National Championships
Page 13, The News Infusion Volume 6, Issue 4
Person to Person Parent to Parent Peer to Peer
Thinking of switching factor brands or switching to a
new prolonged half-life clotting factor? Having
breakthrough bleeds on your current prophylaxis
schedule? Planning on having surgery? Noticing your
factor doesn’t protect as long as it should? Dealing
with inhibitors?
If you answered yes to any of these questions, then
you should probably know a little something about
PK—and probably have PK testing done.
What is PK?
PK is the abbreviation for the word pharmacokinetics.
Derived from the Greek, it means “movement of
drugs.” PK is a series of different kinds of
measurements designed to show us what happens to
a drug in the body over a period of time after it’s
ingested or infused. The measurements—or
parameters—used to determine PK include half-life,
peak factor concentration, and clearance (how
quickly your body removes factor from your blood).
All of us who deal with a bleeding disorder have seen
PK measurements. Every box of clotting factor has a
product insert (PI) that includes a PK table showing
the average PK measurements for that drug. If you’ve
seen ads for the new prolonged half-life clotting
factors now on the market, then you’ve also probably
seen PK measurements and a graph of those
measurements.
You may not have spent much time looking at PK
measurements because they are often listed in PK
tables using abbreviations, and they appear in
unfamiliar units (like IU*hr/dL), making the whole
thing look kind of scary.1 But with a little effort, you
can understand a few of the most common PK
parameters, and this will help you ask your
ᴥ Have You Heard? ᴥ
And now, a few words about PK!
Paul Clement
hematologist more informed questions about product
choice or your prophylaxis schedule.
How is PK testing done?
PK testing requires multiple blood draws over a period of
time: sometimes one before a factor infusion and then
several after factor is infused. Depending on your HTC and
the type of factor product (factor VIII, IX, or VII; standard
or prolonged half-life), you may be asked to provide
anywhere from 3 to 11 blood samples. For example, in a
clinical trial of a prolonged half-life factor VIII product, 8
blood draws were recommended: within 30 minutes
before infusion; 5–30 minutes post-infusion; then 3, 8, 24,
48, 72, and 96 hours post-infusion. Obviously, requiring a
patient to stay near the HTC for days to complete this
testing is inconvenient, especially for children, and for
people who must travel long distances. This is the major
reason that PK studies haven’t been done more often in
the past.
A newer way to determine PK uses a statistical analysis
method called Bayesian analysis, which involves using
“population PK” data (results of PK studies on a large
patient sample using the same product). The Bayesian
analysis method requires only three blood draws,
recommended at 4, 24, and 48 hours post-infusion for a
factor VIII analysis—much more convenient for patients
and HTC staff! 2 Unfortunately, this method isn’t available
at all HTCs because the computer software needed is not
commercially available. Also, Bayesian analysis can’t be
used for the new prolonged half-life products because the
population PK data for those products is not yet available.
It’s hoped that the Bayesian analysis method will become
more common in future, as the software and necessary
population PK data becomes available and HTC staff
become more familiar with its use.
How many different parameters are measured when PK
tests are performed? There is no set number of tests done
when performing PK. In some cases, a physician may only
be interested in one parameter, such as half-life; but a
pharmaceutical company conducting a clinical trial may
test more than a dozen parameters to gain a thorough
understanding of how the drug is processed in the body.
Typically, the results of about seven different parameters
Continued on page 14
Page 14, The News Infusion Volume 6, Issue 4
Person to Person Parent to Parent Peer to Peer
are reported in most PK tables found in PIs. Here, we’ll
look at one PK parameter you may already know
something about: half-life. 3
Half-life (t1/2)
In PK tests of clotting factor, half-life is the most
commonly reported PK parameter—and, for you,
probably one of the most important ones. Half-life is also
the most easily understood PK parameter because it’s
measured in a familiar unit: hours. There are several
different types of half-life, but most PK reports list what’s
known as terminal or elimination half-life (t1/2). That’s the
amount of time for the concentration of factor in plasma
to drop by one-half. PIs usually list the average half-life of
factor for adults aged 16 and older. For standard factor
VIII concentrates, the half-life is about 12 hours. For
standard factor IX concentrates, the half-life is 18 to 24
hours.
If you’re contemplating switching products, perhaps to a
prolonged half-life product, then the half-life of the
product as shown in a product insert PK table allows you
to compare the half-life of various products. This may help
you choose a product. Otherwise, the average half-life of
factor listed in a PK table is not much use to you. Why?
Because you’re not average! There is wide variation in
how each of us processes a drug—so, for instance, the
half-life of factor in your body will rarely match the
average. More important is your individual PK, or how
your body processes a drug over time. Your individual PK is
not a single measurement or parameter; it’s a series of
measurements of several parameters. And these
parameters, taken as a whole, describe how your body
processes a drug. It’s all about YOU: no two individual PKs
are the same. For example, in a child, it’s likely that none
of the PK measurements will match those in a PK table of
average adult PK measurements. Here are a few
considerations that can affect the half-life of factor in your
body:
• Inhibitors have a dramatic effect on half-life. If you
have an active high-titer inhibitor, the half-life of factor
may be measured in minutes. But also know that even
after you have been tolerized—your immune system
desensitized to the presence of factor—you may still
have very low-level inhibitors
that can shorten the half-life of
factor.
• Age significantly affects half
-life of factor. The younger you
are, the faster you clear or
remove factor from the
bloodstream, and the shorter
the half-life of factor in your
system. For factor VIII, babies
may have a factor half-life only
50% or less than that of adults.
The half-life of factor slowly increases as children get
older, and at age 16 they’re often approaching the
average half-life listed in PK tables. The half-life of
factor slowly continues to increase until at age 60,
some people may have a factor VIII half-life of 20 hours
or more.
• Liver disease, such as chronic hepatitis C infection,
reduces clearance of factor, resulting in an increased
half-life.
• Blood type influences half-life. People with blood type
O have lower levels of von Willebrand factor (VWF), a
protein that helps protect factor VIII. With less VWF,
type O people clear factor faster, resulting in a shorter
half-life. In people with blood type AB, factor tends to
have a longer half-life.
Your individual PK, and in particular, the half-life of factor
in your system, is important to know: to make sure you’re
being dosed correctly, and when planning a prophylaxis
schedule with proper factor coverage. Yet most people
with hemophilia have not had PK testing done, and many
don’t know the average half-life of their factor brand as
noted in its PI. When you don’t know your individual PK
measurements, you’re being dosed by guesswork—based
on your weight and the average half-life of your factor
product. As already noted, for children, the half-life of
factor is likely to be significantly shorter than the average
for adults, requiring children to have higher doses and/or
more frequent infusions to maintain the target factor
level.
Continued on page 15
ᴥ Have You Heard? ᴥ
And now, a few words about PK! (continued from page 13)
Page 15, The News Infusion Volume 6, Issue 4
Person to Person Parent to Parent Peer to Peer
Research has also shown that there is no direct
relationship between weight and factor dose. Obese
people have a smaller blood volume than non-obese
people who are the same weight—and because most
infused factor stays in the blood, this means that when
factor is dosed by body weight, an obese patient will
probably receive more factor than is needed to achieve
the same factor level as a non-obese patient. Dosing
according to your individual PK would eliminate these
problems and could potentially reduce factor use while
reducing breakthrough bleeds.
Did you answer yes to any of the questions at the
beginning of this article? If so, have you had a PK test
done? Talk to your hematologist about whether PK
testing is right for you. And if you’re thinking about
switching to a prolonged half-life product, talk to your
hematologist early. Discussing your intention to switch to
a prolonged half-life factor product will benefit you, and
And now, a few words about PK! (continued from page 14)
ᴥ Have You Heard? ᴥ
possibly other people with hemophilia. Why? Because we
need more data about prolonged half-life factor products.
Many physicians see the transition from a standard half-
life product to a prolonged half-life product as an optimal
time to do PK testing. Performing PK around the time of
your switch will allow you and your doctor to see how
your body processed your old factor, and how well the
new prolonged half-life factor is working—vital
information needed to design a prophylaxis schedule
around your individual PK.
Some pharmaceutical companies have become more consumer-friendly, and are now using words instead of symbols in PK tables.
S. Bjorkman and P. Collins, “Measurement of Factor VIII Pharmacokinetics in Routine Clinical Practice,” Journal of Thrombosis and Haemostasis 11 (2013): 180–82.
Other PK parameters will be discussed in future issues of PEN.
© 2016 LA Kelley Communications, Inc. Reprinted with
permission. All rights reserved.
Page 16, The News Infusion Volume 6, Issue 4
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Page 17, The News Infusion Volume 6, Issue 4
Person to Person Parent to Parent Peer to Peer
Hemophilia of South Carolina and three other US Chapters are participating in the PROBE research project and many of our community members have participated in Phase I. Now it is time for Phase 2. If you have not taken the Probe Survey, please consid-er doing so. The Chapter will send you upon request the paper version and we will even send it in for you. You can also take the survey online below! Thanks so much for your participation in furthering ad-vancement in treatment care.
On-line Probe Access Here. https://plus.mcmaster.ca/PROBE
PROBE-ıng Patients with Hemophilia Global survey aims to im-prove quality of life By Sarah M. Aldridge, MS | 07.15.2016 Originally Published July 2016
“Life is good,” says the wheel cover on the Jeep next to your car in the parking lot. “Good?” you think cynical-ly. Try getting in a car with a bum knee. Or pushing a grocery cart
after an elbow bleed. Hang in there, because life with a bleeding disorder can be good, even great.
The Patient Reported Outcomes, Burdens and Experi-ences (PROBE) study is a global survey of the quality of life for people with hemophilia A and B, and their family members. The goal of the study is to collect useful data that can improve lives. “We want to be able to have enough information to advocate for better care for people with bleeding disorders,” says Neil Frick, MS, National Hemophilia Foundation (NHF) vice president for research and medical information.
“PROBE gives a direct understanding of what living with hemophilia means to an individual patient,” says
ᴥ National and World News ᴥ
What is PROBE?
Mark Skinner, JD, the lead author of the study. “It will al-low us to provide a population profile within a country.” He is former president of the World Federation of Hemo-philia (WFH) and of NHF. Skinner has severe hemophilia A.
Now in its second phase, PROBE has already unearthed some interesting findings.
Nuts and Bolts
NHF is the administrator of the study. “We receive all of the funding, pay the investigators and make sure that it’s moving forward,” Frick says.
The first phase included national member organizations of WFH that had the interest and ability to conduct the sur-vey. A total of 704 people filled out the paper question-naire; two-thirds had hemophilia A or B and one-third were family members. They represented patient organiza-tions in 17 countries, from Argentina to Australia, and from Ireland to Italy. Most respondents received the sur-vey at their organization’s annual meeting.
One of the unique aspects of the study is the use of un-affected family members as the control group. “In many countries there is no comparator data available,” Skinner says. By including parents and siblings, the survey canvass-es people of the same socioeconomic background to see what difference having hemophilia makes. “If everybody in a particular society has a high school education, but people with hemophilia only have a sixth-grade education, we can begin to interpret that there is an impact on edu-cational opportunity,” says Skinner.
Phase I tested the strength of the survey. It was patterned after the EQ-5D-5L™, a standardized questionnaire devel-oped by the EuroQol group of international researchers that measures health outcomes. Investigators needed to ensure that the survey was validated, meaning it accurate-ly measured what it set out to measure. They also wanted it to be feasible for the societies to administer, low cost and easy to implement. The PROBE questionnaire gathers information via open text questions, such as “please de-scribe…,” and check-off boxes. Most respondents com-plete it within 15 minutes.
The data are then grouped to find patterns or trends. But individuals’ data are not identified and will not be pub-lished. “Their anonymity is protected throughout the pro-cess,” Skinner says.
(Excerpt from HemAware—07.15.2016)
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Person to Person Parent to Parent Peer to Peer
Hemophilia of South Carolina is a proud recipient of the NHF Advocacy & Public Policy Chapter of Excellence Award in recognition of our commitment to advocacy and education initiatives both at the state and local level, aimed at improving the lives of those in the bleeding disorders community.
The Coalition Mission: To Empower the Hemophilia and Bleeding Disorders Community in South Carolina to speak to public and private decision-makers about improving Access to Quality and Affordable Health Care and Treatment.
The Advocacy Coalition met in September, for the first of what will be many meetings for the next three years. Coming from around the nation and our state, they worked to address the survey results that were provided by our South Carolinian community and focused on access to quality care, treatment and improving educational needs and assistance. The
survey concerns have now been intergraded into a three year strategic plan which has been developed and is just beginning to be implemented. The survey results are available now for reading on our website advocacy page at http://hemophiliasc.org/advocacy/. Please know the results are in the raw format from the survey, however, comments have been
edited to protect the participants and other organizations.
Some highly mentioned concerns with access to care and treatment were the time needed away from work to access treatment and care and the need for additional bleeding disorders education in our emergencies rooms. This is of national concern and several ongoing conversations have been discussed to see how this can be address nationally and locally here at home. Would Saturday appointments at the treatment center be possible? We asked the HTC in Columbia this question and in a candid conversation
ᴥ Advocacy ᴥ
Meet the Advocacy Coalition of Hemophilia of South Carolina By Sue Martin /Executive Director, Advocacy Coalition Member
with Dr. French, the HTC Medical Director, we also learned a few things.
We all understand that things come up in our daily lives which cause us to have to change scheduled appointments we make. However, too many appointments are being canceled the day of the HTC clinic appointments and there are also too many “no-shows, no call”. We must be diligent to call in a timely manner to cancel any appointments which are made for us; provided twice per month on Wednesdays and one Friday morning each month. The Friday appointment time was added to help meet the needed additional appointment requests. When appointments are canceled; clinic physicians, nurses, researchers, social workers, and physical therapists are waiting around with no one to see. The rescheduling of appointments have to be made, which makes a new patient wanting to access the HTC wait an unacceptable time for a new patient comprehensive appointment. Let’s all work to do our best to keep our annual HTC appointments made on our behalf to assure our access to care and treatment.
The idea of Saturday appointments is wonderful but probably not practical at this time. Can it be done in the future? We will have to see. Staffing overtime would have to be addressed, approved, and funded; the clinic offices and examining rooms would have to be opened, available and secured; labs and staff for blood draws would have to be available, funded and secured. We will continue to look at ways to help limit work time off needed to make clinic appointments but until then--- grin and bear it and make an annual comprehensive visit, and keep it!
Meet Our Advocacy Coalition Team
Shawn Sperry- Coalition Chairman, Greenville County Cristal Day- Coalition Vice-Chair, Pickens County Bonnie Phifer- HFA/HSC Board Advocacy Chair, Richland County Sue Martin-HSC Executive Director, Greenville County Lisa Bordelon- HSC President, Sumter County Shelly Crisp, Berkeley County Wendy Le Grand, Dorchester County Steve Nash, Lexington County Aquilla Gaines, Richland County
Continued on page 19
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Person to Person Parent to Parent Peer to Peer
Advocacy Coalition (continued from page 18) emergency or in need of services for you or your loved one.
To Contact the HTC in Columbia:
For all HTC, new and previously scheduled appointments, factor refills, school needs, general questions or general concerns:
Contact Robin Jones, MSN, MHA, RN, CPN, CPHON Children's Cancer and Blood Disorders Nurse Navigator/ SC Hemophilia Treatment Center Nurse Coordinator at
1-803-434-1028 or email @ [email protected]. You may leave a message and your call will be returned as soon as possible. Robin may be away from her desk or with a patient in the clinic.
For TRUE Emergencies if Robin is not at her desk when you call into the HTC- Call 803-434-3533, (Cancer and Blood Disorders Center staff and after hour’s emergency services) and Robin or Dr. French will be immediately contacted for you.
What is a True Emergency? a. You or your child/loved one has an active bleed and you need assistance b. You or your child/loved one has been in or had an accident c. You or your child/loved one needs an emergency surgery, or you are in an emergency room and the physician needs to speak to your healthcare provider for information d. You or your child/loved one needs factor treatment and you have no factor but you are actively treating a bleed
Do you need to call the HTC Social Services Social Worker- Caitlin Sullivan? Please contact her at 803-434-3522 or [email protected]
Ex-Officio Members and Support Advocacy Members: Director of Government Relations/PSI - James Romano NHF Government Specialist/Public Policy - Brendan Hayes Director, Policy & Government Relations- Katie Verb Senior Manager/US Patient Advocacy/CSL- Karla White US Field Advocacy Communications & Public Affairs/Shire- Marvin Poole
Each member of the team has identified a specific area of interest that they will be working to address. On behalf of the Chapter and our community, we thank these dedicated individuals for helping us to empower the Hemophilia and Bleeding Disorders Community in South Carolina to speak to public and private decision-makers about improving Access to Quality and Affordable Health Care and Treatment.
Advocacy Coalition Action Announcements!!!
Each newsletter, we will address a new “Advocacy Action Highlight” to assure Access to Care and Treatment for our South Carolina Community with Bleeding Disorders. Become your best advocate!
New contact information for the federally funded South Carolina Hemophilia Treatment Center at Palmetto Health Richland in Columbia.
Check it out and be sure you are covered in the event of an
Page 20, The News Infusion Volume 6, Issue 4
Person to Person Parent to Parent Peer to Peer
Personalizing Patient-Reported Outcomes for Hemophilia
ᴥ Research ᴥ
patient works with his or her physician to select important
personal goals that are linked to the clinical or functional
impacts of the patient’s condition. An assessment of actu-
al goal attainment is made after a predetermined amount
of time. According to the authors, GAS has been applied
successfully in both clinical practice and research in pa-
tients with a broad range of other chronic conditions, in-
cluding dementia, diabetes, acquired brain injury and vari-
ous types of physical disabilities. One primary advantage
of GAS is that it allows for the discovery of seemingly mi-
nor, though clinically and practically significant, changes,
with real-world implications for a unique individual’s qual-
ity of life.
“We believe that this approach most directly addresses
the need for personalization of outcome measurement in
hemophilia,” concluded the authors. “Adoption of this or
a similarly qualified patient-centric outcome measure will
provide clinicians and researchers with an important inno-
vation that addresses the contemporary challenge of
measuring incremental but clinically meaningful improve-
ment in hemophilia patients.”
Source: Hemophilia News Today, September 12, 2016
New Research Sheds Light on Dis-
ease Suppression
Researchers from the University of North Carolina (UNC)
at Chapel Hill School of Medicine have published a new
study suggesting that genetic material known as hetero-
chromatin performs a key role in disease suppression. The
study, “Direct Interrogation of the Role of H3K9 in Meta-
zoan Heterochromatin Function,” was published August
2016, in the journal Genes & Development. The new find-
ings could have future therapeutic implications for condi-
tions such as cancer and hemophilia.
Heterochromatin represents one of two distinct groupings
of DNA found within cells known as chromatin. Loosely-
packed euchromatin carries out several important func-
tions, including the efficient packaging of DNA so that it
fits into the nucleus of a cell. In contrast, heterochromatin
is densely packed and relatively inactive. Both are found in
chromosomes, the thread-like structures located inside
the nucleus of all animal and plant cells. DNA, which when
In a recent paper, a group of experienced hematologists
reviewed two tools used for assessing patient-reported
outcomes (PROs). While standard modes of outcome
measurement, such as annualized bleeding rate and joint
health score, are relatively effective in evaluating larger
groups of bleeding disorders patients, they are not de-
signed to capture the unique healthcare needs, perspec-
tives and goals of each individual. The need for a person-
alized approach for people with hemophilia is even great-
er in light of dramatic changes in the clinical landscape
over the last two decades, including the proliferation of
progressively safer and more effective therapies, and the
availability of preventive treatment regimens like prophy-
laxis.
The article, “Recognizing the Need for Personalization of
Haemophilia Patient-Reported Outcomes in the Prophy-
laxis Era,” was published August 31, 2016, in the journal
Haemophilia. The lead author of the study was Michael
Recht, MD, a pediatric hematologist-oncologist at the He-
mophilia Center at the Oregon Health & Science Universi-
ty in Portland. Recht and his colleagues assessed a pair of
tools: the patient-reported outcomes measurement infor-
mation system (PROMIS®) and the goal attainment scaling
(GAS).
First launched in 2004 by the US National Institutes of
Health (NIH), PROMIS takes the traditional questionnaire-
based approach and employs modern measurement theo-
ry to develop new and enhanced PRO tools. The system
improves on the traditional PRO techniques in part
through the use of a psychometric method, which
measures knowledge, abilities, attitudes and personality
traits among individuals to produce scores associated with
probable answers to questions.
These scores can then be used by computerized adaptive
testing (CAT) to pinpoint the most informative follow-up
question to an initial question from a predetermined bank
of questions. According to investigators, PROMIS allows
clinicians and researchers access to “efficient, precise,
valid and responsive adult- and child-reported measures”
in the physical, mental and social areas. These can then be
applied across a broad range of chronic conditions, includ-
ing hemophilia.
GAS involves a truly collaborative approach whereby a
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Person to Person Parent to Parent Peer to Peer
New Research Sheds Light on Disease Suppression (continued from page 20)
The researchers subsequently observed a 98% mortality
rate amongst the flies, which had their working H3 genes
replaced with a mutant form, which allowed the transpos-
ons to infiltrate important genes. The finding is significant
as it suggests that by removing working H3 genes, investi-
gators also eliminated the flies’ ability to produce hetero-
chromatin and its critical jumping gene suppressing func-
tion.
“It seems that the major role for the methylation of his-
tone H3 that triggers this type of heterochromatin is to
keep transposons from jumping around and screwing up
the genome,” explained Duronio.
The UNC team plans to conduct additional research to
further their understanding of the biological relationship
between transposons, heterochromatins and the genome.
“During embryonic and fetal development, there is nor-
mally a high-fidelity replication of the genome, and that is
a significant mechanism for repressing cancer and other
diseases,” concluded Duronio. “With studies like these,
we’re understanding how heterochromatin does its job in
that respect.”
Source: Science Daily, September 1, 2016
passed from parents to offspring, carries with it the spe-
cific genetic instructions that account for an individual’s
unique hereditary traits.
While scientists have generally assumed that heterochro-
matin is responsible for controlling gene expression and
cell proliferation, UNC investigators may have assigned it
some newfound relevance. Using Drosophila fruit flies
they demonstrated that heterochromatin actually works
to suppress the activity of transposons, or “jumping
genes,” a notable finding as these genes have the capacity
to replicate and paste copies throughout the genome. By
inserting themselves into certain key genes, transposons
can cause adverse cellular changes such as disrupting tu-
mor-suppressing genes or fragmenting DNA to destabilize
the chromosome. These and other molecular level chang-
es have been associated with several conditions, such as
cancer and hemophilia.
Lead investigator Robert Duronio, PhD, and his colleagues
also discovered that heterochromatin form when a pro-
tein called histone H3 is chemically modified or
“methylated,” a theory tested in the Drosophila fruit flies.
Page 22, The News Infusion Volume 6, Issue 4
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ᴥ Resources and Contact Information ᴥ
The Hemophilia Treatment Center of South Carolina Children's Cancer & Blood Disorders Clinic at Palmetto Health Richland Hospital 7 Richland Medical Park Rd., Suite 7215 Columbia, SC 29203-6872 Phone: 803-434-3533 For all new and previously scheduled appointments, factor refills, school needs, general questions or general concerns: Contact Robin Jones, MSN, MHA, RN, CPN, CPHON Children's Cancer and Blood Disorders Nurse Navigator/ SC Hemophilia Treatment Center Nurse Coordinator at 803-434-1028 or email @ [email protected]. You may leave a message and your call will be returned as soon as possible. Robin may be away from her desk or with a patient in the clinic. The Children's Hospital of the Greenville Health System MAIN CAMPUS: BI-LO Charities Children's Cancer Center Serving Hematology/Oncology Patients 900 W. Faris Road Greenville, SC 29605 Phone: 864-455-8898 Fax: 864-455-5164 Hours: Mon, Wed., Thurs. 8:00-4:30; Tues & Fri. 8:00-12:00 SATELLITE OFFICE: Spartanburg- Children’s Hospital Outpatient Specialties 1700 Skylyn Drive, Suite 200 Spartanburg, SC 29307 Phone: 864-716-6490 Fax: 864-596-5164 Hours: Wednesday - alternating mornings and afternoons SATELLITE OFFICE: Anderson- Pediatric Specialties of the Upstate 2000 E. Greenville Street, Suite 3500 Anderson, S.C. 29621 Phone: 864-716-6490 Fax: 864-716-6492 Hours: Mondays - afternoons Medical University of South Carolina Department of Pediatric Hematology/Oncology 135 Rutledge Avenue, MSC 558 Charleston, South Carolina 29425 Shayla Bergmann, MD, Assistant Professor Director, Pediatric Hemophilia Clinic Phone: 843-876-1980 Mobile: 843-812-5682 Fax: 843-792-7562
Medical Facilities
Bleeding Disorders Legal Hotline Phone: 800-520-6154 Centers for Disease Control & Prevention Phone: 1-800-311-3435 Website: www.cdc.gov Committee of Ten Thousand (COTT) Phone: 800-488-2688 Website: www.cott1.org The Factor Foundation of America Phone: 866-843-3362 Website: www.factorfoundation.org Inalex Communications Phone: 201-493-1399 Website: www.inalex.com HIV / HCV Newsletter Hemophilia Association of the Capital Area Website: www.hacacares.org LA Kelley Communications, Inc. Phone: 978-352-7657 Website: www.kelleycom.com Patient Notification System The Patient Notification System is a free, confidential, 24-hour communication system providing information on plasma-derived and recombinant analog therapy withdrawals and recalls. Phone: 888-UPDATE U (873-2838) Website: www.patientnotificationsystem.org Patient Services Incorporated (PSI) Assists persons with chronic medical illnesses in accessing health insurance and pharmacy co-payment assistance. Phone: 800-366-7741 Website: https://www.patientservicesinc.org/ Medic Alert Foundation 2323 Colorado Avenue Turlock, CA 95382 Phone: 800-432-5378 Website: www.medicalert.org
Contact Numbers Resource Information (cont.)
Hemophilia of South Carolina 439 Congaree Road, Suite #5 Greenville, SC 29607 Phone: 1-864-350-9941 Fax: 864-244-8287 Email: [email protected] Web site: www.hemophiliasc.org National Hemophilia Foundation 7 Penn Plaza, Suite 1204 New York, NY 10001 Phone: 212-328-3700 Fax: 212-328-3777 Phone: 800- 42-HANDI (4-2634) Fax: 212- 328-3799 Email: [email protected] Website: http://www.hemophilia.org/
Hemophilia Federation of America 210 7th St. SE, Suite 200 B Washington, DC 20003 Phone: 800-230-9797 Email: [email protected] Website: www.hemophiliafed.org
World Federation of Hemophilia 1425, boul. René-Lévesque O. Bureau 1010 Montréal, Québec H3G 1T7 Canada Phone: +1 (514) 875-7944 Fax: +1 (514) 875-8916 Email: [email protected] Website: www.wfh.org
Resource Information
S.C Dept. of Health & Environmental Control (DHEC) Jessica A. Drennan, MSW Program Director, Children with Special Health Care Needs Hemophilia Program 2100 Bull Street, Columbia, SC 29201 Phone: 803-898-0789 American Pain Foundation (APF) Phone: 1-888-615-PAIN (7246) Hemophilia Chronic Pain Support Group Website: http://painaid.painfoundation.org American Society of Pediatric Hematology/Oncology Phone: 847-275-4716 Website: www.aspho.ord The Coalition for Hemophilia B, Inc. Phone: 212-520-8272 E-Mail- [email protected] Website: www.coalitionforhemophiliab.org
Page 23, The News Infusion Volume 6, Issue 4
Person to Person Parent to Parent Peer to Peer
To see the most up-to-date information on events and happenings, please, visit the Chapter’s website Calendar each week at: http://hemophiliasc.org/programs-and-events/calendar/
DECEMBER Winter Holiday Meeting December 3, 2016 5:30 to 10:00pm Embassy Suites Greystone, Columbia
YES Support Group Activity Children’s Museum of the Upstate December 10, 2016, 11:00-3:00pm Greenville, SC
FEBRUARY Advocacy Days February 28—March 1, 2017 Columbia, SC
MARCH NHF Washington Days March 8-10, 2017 Washington, DC
APRIL HFA Symposium - Providence, RI April 6-9, 2017
Par for the Clot Golf Fundraiser April 21, 2017 Greenville, SC
JUNE
HSC Annual State Meeting June 2-4, 2017 Greenville, SC Camp Burnt Gin Summer Camp Bleeding Disorders Week Monday June 26-Saturday July 1, 2017 Applications will be available January 2nd Wedgefield, SC
JULY
Teen Retreat - Camp Canaan July 6-9, 2017 Rock Hill, SC
AUGUST
69th National Hemophilia Foundation Annual Meeting August 24-26, 2017 Chicago, IL
NOVEMBER
Turkey Trot November 4, 2017 Saluda Shoals Park Columbia, SC
ᴥ Upcoming HSC Community Activities/Events ᴥ
Hemophilia of South Carolina
439 Congaree Road, Suite #5 Greenville, SC 29607 Website: www.hemophiliasc.org
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