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Fabry Outcome SurveyAnnual Report 2017
This report has been prepared by Shire Outcome Surveys, on behalf of the FOS Steering Committee
Reporting Period: 17 April 2001 to 5 January 2018
Date of preparation: August 2018 Item code: VV-RIR-0005
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FOS Annual Report 2017
Contents
Executive summary 2
Background to the FOS registry 3
FOS Scientific Boards 4
Update on demographic data as reported in FOS 6
FOS publications and congress activities 13
FOS publication archive (2001–2017) 14
Appendix: useful contacts 18
Data in this report are from the 5 January 2018 datacut.
Patients should direct any treatment-related questions to their treating physician.
FOS management, administration and infrastructure are funded by Shire.Data collection and analysis in FOS are also supported by Shire.
Data review and analysis are conducted by Shire under the direction of the clinical expert members of the FOS Steering Committee and Task Forces. Meetings of the FOS Steering Committee and Task Forces are organized and supported by Shire.
All publications containing FOS data are prepared in compliance with the applicable Shire policies and procedures.
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FOS Annual Report 2017
Outcome SurveyAnnual Report 2017
Executive summary
Welcome to the 2017 Fabry Outcome Survey (FOS) Annual Report, which provides an overview of FOS as of 5 January 2018. This report includes details of the FOS Steering Committee as well as a summary of patient demographics and the publications that have been developed using the data collected in the registry.
FOS (ClinicalTrials.gov identifier: NCT03289065) is a large, global, multicentre, observational registry, sponsored by Shire, for patients with Fabry disease. The registry was established in 2001 with the aim of collecting real-world data on the long-term safety and effectiveness of enzyme replacement therapy (ERT) with agalsidase alfa and the natural history of the disease. FOS became a disease registry during 2017 and is now enrolling any patient with Fabry disease, including those who have received an approved treatment for Fabry disease other than agalsidase alfa.
Figure 1. Map highlighting (in blue) the countries in which patients with Fabry disease are enrolled in FOS.
Key highlights from FOS, as of January 2018
As of January 2018, there were 3515 patients enrolled in FOS, which represents an increase of 11% compared with January 2017. In total, data have been collected by 137 centres in 25 countries.
Similarly to previous years, 57% of enrolled patients are female and 12% are children (defined as < 18 years old at FOS entry).
Overall, 2171 patients (62%) have received one or more doses of approved treatment for Fabry disease: agalsidase alfa (n = 2112), agalsidase beta (n = 63) or migalastat (n = 34); 38 patients have received more than one approved treatment. The relative proportions of patients who have received agalsidase beta or migalastat are lower than would be expected based on worldwide use of Fabry treatments because enrolment of patients receiving approved treatment for Fabry disease other than agalsidase alfa began in 2017.
Four manuscripts based on FOS data were published during 2017, so that there was a total of 54 FOS publications as of January 2018. In addition, three posters based on FOS data were presented at two meetings in North America and South America.
Shire would like to take this opportunity to thank all of the patients and their families, and the physicians and their staff, who have participated in FOS and contributed data to the registry.
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FOS Annual Report 2017
Background to the FOS registry
Registry design
FOS is designed to collect information on various aspects of Fabry disease, based on data obtained during routine patient visits and assessments. The registry is open to individuals with confirmed diagnosis of Fabry disease. At its initiation, FOS was set up as a drug registry (for patients currently untreated or treated with agalsidase alfa, or previously treated with any ERT), but at the start of 2017 it became a disease registry and it is open to all patients with Fabry disease irrespective of their treatment in countries where Protocol Amendment 4 has been approved by institutional review boards.
Registry objectives
A broad range of disease- and treatment-related information is captured in the registry with the overall aim of collecting and disseminating information about the long-term course of Fabry disease. The objectives of the registry are to: monitor the long-term safety and effectiveness of agalsidase alfa; characterize the population of patients with Fabry disease and provide data on the clinical course of the disease in untreated patients and those receiving approved therapies; and enhance the understanding of the natural history of the disease, including intra- and inter-familial variation. Patients can also complete questionnaires on issues such as health-related quality of life. It is hoped that the data collected will help to form a basis for further development of clinical management recommendations for the disease.
Achievements of FOS
The data recorded in FOS can be used to research specific topics of interest within the field of Fabry disease, such as the effect of treatment on particular organ systems or in specific populations such as children. Since the registry’s initiation more than 15 years ago, publications based on FOS data have helped to establish disease severity scores, describe disease manifestations in different patient populations, and report the long-term effects of ERT with agalsidase alfa on kidney and heart function, mortality and morbidity (see pages 14–17). As well as becoming a disease registry during 2017, another significant activity for FOS was the analysis of genetic data. As of March 2018, there were 1592 patients in the registry who had consented to collection of genetic data in FOS confirming Fabry diagnosis and genetic data have been entered into the database for 1405 of these patients. It is hoped that additional insights in the natural history and treatment outcomes of Fabry disease will become available with the collection of data on mutations and treatment outcomes for all approved treatments for Fabry disease.
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FOS Annual Report 2017
The FOS registry is committed to increasing the knowledge of Fabry disease and its treatment. With this in mind, FOS scientific boards enable the smooth operation of the registry and ensure the scientific accuracy and appropriateness of all research concepts and proposals for data analyses and publications. The scientific boards comprise the FOS Steering Committee (SC), the Shire FOS team and the FOS Task Forces.
The FOS SC, consisting of 10 FOS experts in the area of Fabry disease, plus representatives of Shire, provides overall direction for the FOS registry and represents FOS at scientific meetings. As well as providing input on operational changes, members of the SC review data reports, endorse proposed data analyses and topics for publication, and oversee the functioning and activities of the Task Forces.
The SC met twice in 2017, and its most recent meeting was in February 2018 at the Lysosomal Disease Network (LDN) 14th Annual WORLD Symposium (WORLD 2018) in San Diego, CA, USA. Currently, the SC members are reviewing the FOS case report form. They are also categorizing the mutation data according to Fabry phenotype, with the aim of investigating potential associations with disease severity and treatment outcome.
FOS Scientific Boards
Roberto Giugliani (Chair) (Porto Alegre, Brazil) Medical genetics
Michael Beck (Mainz, Germany) Paediatrics
Kathy Nicholls (Melbourne, Australia) Nephrology
Derralynn Hughes (London, UK) Haematology
Christoph Kampmann (Mainz, Germany) Paediatric cardiology
Guillem Pintos-Morell (Badalona, Spain) Paediatric nephrology
Uma Ramaswami (London, UK) Inherited metabolic disorders
Ricardo Reisin (Buenos Aires, Argentina) Neurology
Dau-Ming Niu (Taipei, Taiwan) Medical genetics
Michael West (Halifax, Canada) Nephrology
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FOS Annual Report 2017
The Shire FOS team
Nataliya Kemenyash Clinical Programs Lead – Real-World, Late-Phase Studies (Zug, Switzerland)
Andrey GurevichGlobal Medical Lead Fabry and FOS Medical Monitor(Zug, Switzerland)
Colin McKinnonPublications Lead Fabry Disease(Zug, Switzerland)
Jörn SchenkGlobal Medical TeamLead, LSD(Zug, Switzerland)
Jaco BothaBiostatistics Project Lead(Zug, Switzerland)
Vicky Kalampoki FOS Lead Biostatistician(Zug, Switzerland)
FOS Task Forces
There are currently five FOS Task Forces, each relating to an organ system manifestation or patient subgroup: Cardiac, Female, Neurology, Paediatric and Renal. Task Force members are physicians or researchers with particular expertise and interest in the relevant area, and they are responsible for all activities related to conducting and publishing in-depth analyses of FOS data in their field of interest.
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FOS Annual Report 2017
Update on demographic data as reported in FOS
Patient enrolment
As of January 2018, 3515 patients were enrolled in FOS (Figure 2). This represents an increase in the rate of enrolment compared with recent previous years, from 6% in the years 2015 and 2016 each to 11% during 2017, and reflects the inclusion of patients with Fabry disease regardless of treatment during 2017 (see page 10).
As in previous years, the proportion of females enrolled in FOS (57%, n = 2005) was greater than the proportion of males, and 12% (n = 413) of patients were children (defined as < 18 years old at FOS entry). As of January 2018, 3350 (95%) patients were recorded as being alive (1396 males and 1954 females);* no children had died. Overall, the median (Q1–Q3) current age of these patients was 45.8 (32.7–58.6) years: the median current age of patients aged ≥ 18 years and < 18 years was 47.1 (35.3–59.5) years (n = 3145) and 13.5 (10.1–15.9) years (n = 205), respectively.
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Figure 2. Cumulative number of patients in FOS since 2001 (n = 3515). Men and women are defined as patients ≥ 18 years old at FOS entry; boys and girls are defined as patients
< 18 years old at FOS entry.
*Of the 3350 patients who did not have a death record as of January 2018, 1115 have discontinued their participation in FOS, and follow-up information on whether they have since died is not available. The current age of discontinued patients is reported for age at last visit.
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FOS Annual Report 2017
Global reach of FOS
As of January 2018, patients were enrolled at 137 FOS centres in 25 countries (Figure 3), and a site opened for the first time in South Korea in the past year. Similarly to previous years, most patients have been enrolled in Europe, Asia and North America (62%, 22% and 9%, respectively), with large proportions of the overall patient population having been enrolled at sites in Germany, the UK, Japan, Canada and Taiwan (18%, 14%, 13%, 9% and 9%, respectively) (Figure 4). FOS is no longer being conducted in Japan, the Netherlands, Norway, Sweden and the USA.
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Figure 3. Cumulative number of centres (n = 137) and countries (n = 25) in which patients have been enrolled in FOS since 2001.
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FOS Annual Report 2017
Germany638 (18%)
UK490 (14%)
Japan461 (13%)
Canada308 (9%)
Italy217 (6%)
Taiwan307 (9%)
France142 (4%)
Netherlands137 (4%)
Spain145 (4%)
Australia133 (4%)
Switzerland 78 (2%)
Czech Republic 64 (2%)Finland 69 (2%)
Austria 52 (1%)Brazil 47 (1%)
Slovenia 31 (< 1%)
Sweden 23 (< 1%)Belgium 25 (< 1%)
Hungary 18 (< 1%)Portugal 17 (< 1%)
USA 14 (< 1%)Russia 13 (< 1%)
Israel 5 (< 1%)South Korea 1 (< 1%)
Argentina 80 (2%)
Figure 4. Geographical distribution of patients enrolled in FOS (n = 3515). The number and proportion (%) of patients enrolled are given for each country.
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FOS Annual Report 2017
Symptom onset and diagnosis
As in previous years, onset of signs or symptoms of Fabry disease were reported in males earlier than in females (median [Q1–Q3] age: 11.0 [7.0–27.0] years vs 20.0 [10.0–38.0] years, respectively) (Figure 5). The age at diagnosis in males was also earlier than in females (30.0 [16.0–45.0] years vs 37.0 [23.0–51.0] years, respectively). In patients with data available for age at symptom onset and at diagnosis, the delay between onset of symptoms and diagnosis in males and females was similar (6.0 [0.0–19.0] years and 6.0 [0.0–20.0] years, respectively).
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Figure 5. Median age at symptom onset and at diagnosis, and median delay between onset of symptoms and diagnosis for male and female patients enrolled in FOS.
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FOS Annual Report 2017
Patients who have received therapy
FOS became a disease registry during 2017 and started to collect data from patients with Fabry disease regardless of treatment. Of the 3515 patients in FOS, 2171 patients (62%) have received one or more doses of approved treatment for Fabry disease. Of these treated patients, 2112 patients have received agalsidase alfa,* 63 patients have received agalsidase beta† and 34 patients have received migalastat,‡ either as a sole treatment or after a switch from another approved Fabry-specific therapy. The relative proportions of patients receiving agalsidase beta or migalastat are lower than would be expected based on worldwide use of Fabry treatments because enrolment of patients receiving approved treatment for Fabry disease other than agalsidase alfa began in 2017. The numbers of patients who have received each treatment are shown in Figure 6.
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Figure 6. Number of patients enrolled in FOS who have received at least one dose of an approved treatment for Fabry disease (n = 2171). The sum of the numbers of patients who have received each treatment is greater than the number of patients treated because some
patients have switched treatment. Boys and girls are defined as patients < 18 years old at the start of the respective treatment.
*EU Summary of Product Characteristics for agalsidase alfa is available here: http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Product_Information/human/000369/WC500053612.pdf. Accessed March 2018.†EU Summary of Product Characteristics for agalsidase beta is available here: http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Product_Information/human/000370/WC500020547.pdf. Accessed March 2018.‡EU Summary of Product Characteristics for migalastat is available here: http://www.ema.europa.eu/docs/en_GB/document_library/ EPAR_-_Product_Information/human/004059/WC500208434.pdf. Accessed March 2018.
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FOS Annual Report 2017
Of the 2171 patients who have received approved treatment for Fabry disease, 2075 patients have received treatment with agalsidase alfa only, 43 and 15 patients have received agalsidase beta only or migalastat only, respectively, and 38 patients have switched from one treatment for Fabry disease to another (Figure 7).
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Figure 7. Number of treated patients enrolled in FOS (n = 2171) who have or have not switched from one treatment for Fabry disease to another.
For all treated patients, the median (Q1–Q3) duration of treatment was 7.7 (5.9–11.9) years. A higher proportion of males were currently receiving an approved treatment for Fabry disease as compared with females, in both the adult population (men, 80%; women, 51%) and the paediatric population (boys, 73%; girls, 32%).
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FOS Annual Report 2017
Home therapy with agalsidase alfa
Of the 2112 patients treated with agalsidase alfa, 44% of patients (n = 919) had received home therapy, with median (Q1–Q3) duration of treatment of 8.7 (6.4–13.2) years. Most patients receiving home therapy (78%) were from Europe (Figure 8). The UK, Germany and Canada had the greatest numbers of patients receiving home therapy (n = 248, 204 and 135, respectively).
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Figure 8. Proportion of patients who have received home therapy with agalsidase alfa in FOS in recent years. Patients were considered to have received home therapy if at least one
infusion was recorded as ‘home infusion’ in the FOS database; no patients from Asia (Japan and Taiwan) have received home therapy with agalsidase alfa. The numbers of patients who have received therapy with agalsidase alfa as of January 2012, 2014, 2016 and 2018 were
1530, 1738, 1828 and 2112, respectively.
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FOS Annual Report 2017
The FOS registry disseminates the results of data analyses in peer-reviewed journals and at congresses. Since the initiation of the registry in 2001, FOS publications have covered various topics of interest in Fabry disease, including measures of disease severity, signs and symptoms in specific populations and the long-term effects of ERT on kidney and heart function, mortality and morbidity. A full list of completed FOS publications (2001–2017) can be found on pages 14–17. These articles have been cited more than 3600 times in total, providing an indication of the value of the data collected in FOS.
During 2017, four original articles were published in peer-reviewed journals, bringing the total number of FOS publications to 54. Two articles assessed neurological and cardiac findings in patients with classic disease compared with the late-onset cardiac variant IVS4+919G>A mutation. A third article investigated cerebrovascular, cardiac and renal outcomes in treated patients. The fourth manuscript compared FOS data from two time periods to assess changes in time to diagnosis and treatment in recent years; it is hoped that this will contribute to an increasing awareness of the need for timely diagnosis. Furthermore, three posters were presented at two meetings in North America and South America during 2017.
Currently, the FOS Task Forces are developing four new FOS manuscripts which will present data on cardiovascular and renal involvement in Fabry disease, the implications of prompt treatment initiation, and treatment outcomes in young males. In addition, with the collection of genetic data from a large number of patients in FOS, it may soon become possible to analyse data relating to the influence of Fabry mutations on disease manifestations and outcomes. It is hoped that new insights into the natural history of Fabry disease and its treatment will be gained from these data analyses.
FOS publications and congress activities
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FOS Annual Report 2017
Author Title Reference
Beck M et al. Long-term outcomes with agalsidase alfa enzyme replacement therapy: analysis using deconstructed composite events
Mol Genet Metab Rep 2018; 14: 31–5 (online 9 November 2017)
Lee H-J et al. A comparison of central nervous system involvement in patients with classical Fabry disease or the later-onset subtype with the IVS4+919G>A mutation
BMC Neurol 2017; 17: 25
Reisin R et al. Time delays in the diagnosis and treatment of Fabry disease
Int J Clin Pract 2017; 71: e12914
Hsu T-R et al. Correlations between endomyocardial biopsies and cardiac manifestations in Taiwanese patients with the Chinese hotspot IVS4+919G>A mutation: data from the Fabry Outcome Survey
Int J Mol Sci 2017; 18: 119
Barba-Romero MÁ and Pintos-Morell G
Gender differences in the application of Spanish criteria for initiation of enzyme replacement therapy for Fabry disease in the Fabry Outcome Survey
Int J Mol Sci 2016; 17: 1965
Kalkum G et al. Paediatric Fabry disease: prognostic significance of ocular changes for disease severity
BMC Ophthalmol 2016; 16: 202–8
Giugliani R et al. A 15-year perspective of the Fabry Outcome Survey
J Inborn Errors Metab Screen 2016; 4: 1–12
Lidove O et al. Fabry in the older patient: clinical consequences and possibilities for treatment
Mol Genet Metab 2016; 118: 319–25
Kampmann C et al. Effectiveness of agalsidase alfa enzyme replacement in Fabry disease: cardiac outcomes after 10 years’ treatment
Orphanet J Rare Dis 2015; 10: 125
Pitz S et al. Ocular signs correlate well with disease severity and genotype in Fabry disease
PLoS One 2015; 10: e0120814
Liu H-C et al. Age at first cardiac symptoms in Fabry disease: association with a Chinese hotspot Fabry mutation (IVS4+919G>A), classical Fabry mutations, and sex in a Taiwanese population from the Fabry Outcome Survey (FOS)
JIMD Rep 2015; 22: 107–13
Beck M et al. Long-term effectiveness of agalsidase alfa enzyme replacement in Fabry disease: a Fabry Outcome Survey analysis
Mol Genet Metab Rep 2015; 3: 21–7
Terryn W et al. Questioning the pathogenic role of the GLA p.Ala143Thr “mutation” in Fabry disease: implications for screening studies and ERT
JIMD Rep 2013; 8: 101–8.
Barba-Romero MÁ et al.
Comparison of patients from a Spanish registry of Fabry disease in two periods
Med Clin (Barc) 2012; 139: 379–84 (in Spanish)
FOS publication archive (2001–2017)
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FOS Annual Report 2017
Author Title Reference
Ramaswami U et al. Measuring patient experiences in Fabry disease: validation of the Fabry-specific Paediatric Health and Pain Questionnaire (FPHPQ)
Health Qual Life Outcomes 2012; 10: 116
Ramaswami U et al. Fabry disease in children and response to enzyme replacement therapy: results from the Fabry Outcome Survey
Clin Genet 2012; 81: 485–90
Hughes DA et al. Fabry International Prognostic Index: a predictive severity score for Anderson–Fabry disease
J Med Genet 2012; 49: 212–20
Feriozzi S et al. The effectiveness of long-term agalsidase alfa therapy in the treatment of Fabry nephropathy
Clin J Am Soc Nephrol 2012; 7: 60–9
Barba-Romero MÁ et al.
Fabry disease in Spain: description of Spanish patients and a comparison with other European countries using data from the Fabry Outcome Survey (FOS)
Int J Clin Pract 2011; 65: 903–10
Clarke JTR et al. Impact of measures to enhance the value of observational surveys in rare diseases: the Fabry Outcome Survey (FOS)
Value Health 2011; 14: 862–6
Hughes DA et al. Response of women with Fabry disease to enzyme replacement therapy: comparison with men, using data from FOS – the Fabry Outcome Survey
Mol Genet Metab 2011; 103: 207–14
Ramaswami U et al. Safety of agalsidase alfa in Fabry disease patients under 7 years
Acta Paediatr 2011; 100: 605–11
Hughes DA et al. Age adjusting severity scores for Anderson–Fabry disease
Mol Genet Metab 2010; 101: 219–27
Barba-Romero MÁ et al.
Does geographical location influence the phenotype of Fabry disease in women in Europe?
Clin Genet 2010; 77: 131–40
Mehta A et al. Enzyme replacement therapy with agalsidase alfa in patients with Fabry’s disease: an analysis of registry data
Lancet 2009; 374: 1986–96. Erratum Lancet 2010; 375: 200
Keilmann A et al. Ear symptoms in children with Fabry disease: data from the Fabry Outcome Survey
J Inherit Metab Dis 2009; 32: 739–44
Mehta A et al. Natural course of Fabry disease: changing pattern of causes of death in FOS – Fabry Outcome Survey
J Med Genet 2009; 46: 548–52
Malmenäs M et al. Analysis of effectiveness in patient registry data ISPOR Connections 2009; 15: 9–10
Feriozzi S et al. Agalsidase alfa slows the decline in renal function in patients with Fabry disease
Am J Nephrol 2009; 29: 353–61
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FOS Annual Report 2017
Author Title Reference
Cybulla M et al. Kidney transplantation in patients with Fabry disease
Transplant Int 2009; 22: 475–81
Sodi A et al. Ocular manifestations of Fabry’s disease: data from the Fabry Outcome Survey
Br J Ophthalmol 2007; 91: 210–14
Cybulla M et al. Fabry disease: demographic data since introduction of enzyme replacement therapy
Dtsch Med Wochenschr 2007; 132: 1505–9 (in German)
Orteu CH et al. Fabry disease and the skin: data from FOS, the Fabry Outcome Survey
Br J Dermatol 2007; 157: 331–7
Hoffmann B et al. Gastrointestinal symptoms in 342 patients with Fabry disease: prevalence and response to enzyme replacement therapy
Clin Gastroenterol Hepatol 2007; 5: 1447–53
Linhart A et al. Cardiac manifestations of Anderson–Fabry disease: results from the international Fabry Outcome Survey
Eur Heart J 2007; 28: 1228–35
Hoffmann B et al. Nature and prevalence of pain in Fabry disease and its response to enzyme replacement therapy – a retrospective analysis from the Fabry Outcome Survey
Clin J Pain 2007; 23: 535–42
Hoffmann B and Keshav S
Gastrointestinal symptoms in Fabry disease: everything is possible, including treatment
Acta Paediatr Suppl 2007; 96: 84–6
Schwarting A et al. Enzyme replacement therapy and renal function in 201 patients with Fabry disease
Clin Nephrol 2006; 66: 77–84
Rivera-Gallego A et al.
Fabry disease in Spain: first analysis of the response to enzyme replacement therapy
Med Clin (Barc) 2006; 127: 481–4 (in Spanish)
Lidove O et al. Hyperhidrosis: a new and often early symptom in Fabry disease. International experience and data from the Fabry Outcome Survey
Int J Clin Pract 2006; 60: 1053–9
Hajioff D et al. Agalsidase alfa and hearing in Fabry disease: data from the Fabry Outcome Survey
Eur J Clin Invest 2006; 36: 663–7. Erratum Eur J Clin Invest 2007; 37: 828
Hegemann S et al. Hearing loss in Fabry disease: data from the Fabry Outcome Survey
Eur J Clin Invest 2006; 36: 654–62. Erratum Eur J Clin Invest 2007; 37: 828
Kleinert J et al. Prevalence of uncontrolled hypertension in patients with Fabry disease
Am J Hypertens 2006; 19: 782–7
Ginsberg L et al. Magnetic resonance imaging changes in Fabry disease
Acta Paediatr Suppl 2006; 95: 57–62
Deegan PB et al. Natural history of Fabry disease in females in the Fabry Outcome Survey
J Med Genet 2006; 43: 347–52
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FOS Annual Report 2017
Author Title Reference
Ramaswami U et al. Clinical manifestations of Fabry disease in children: data from the Fabry Outcome Survey
Acta Paediatr 2006; 95: 86–92
Kleinert J et al. Anaemia is a new complication in Fabry disease: data from the Fabry Outcome Survey
Kidney Int 2005; 67: 1955–60
Schaefer E et al. Genotype and phenotype in Fabry disease: analysis of the Fabry Outcome Survey
Acta Paediatr Suppl 2005; 94: 87–92
Mehta A and Ginsberg L
Natural history of the cerebrovascular complications of Fabry disease
Acta Paediatr Suppl 2005; 94: 24–7
Hoffmann B et al. Effects of enzyme replacement therapy on pain and health related quality of life in patients with Fabry disease: data from FOS (Fabry Outcome Survey)
J Med Genet 2005; 42: 247–52
Beck M et al. Fabry disease: overall effects of agalsidase alfa treatment
Eur J Clin Invest 2004; 34: 838–44
Barba-Romero M-Á et al.
Fabry’s disease in Spain. Study of 24 cases Med Clin (Barc) 2004; 123: 57–60 (in Spanish)
Mehta A et al. Fabry disease defined: baseline clinical manifestations of 366 patients in the Fabry Outcome Survey
Eur J Clin Invest 2004; 34: 236–42
Dehout F et al. Effects of enzyme replacement therapy with agalsidase alfa on glomerular filtration rate in patients with Fabry disease: preliminary data
Acta Paediatr Suppl 2003; 92: 14–15
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FOS Annual Report 2017
Appendix: useful contacts
Nataliya KemenyashClinical Programs Lead – Real-World, Late-Phase StudiesZug, [email protected]
Fintan LeonardInternational Patient AdvocacyZug, [email protected]
Jörn SchenkGlobal Medical Team Lead, LSDZug, [email protected]
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Outcome Surveys
Date of preparation: August 2018 Item code: VV-RIR-0005