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International Patient Survey of MPS I Disease Management

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  1. International Patient Survey ofMPS I Disease Management A collaboration of Genzyme and national MPS societies Presented to the 10th International Symposium of Mucopolysaccharide and Related Diseases, Vancouver, June 2008 ©Genzyme Corporation 2008

  2. Objectives • Learn from individuals with MPS I (and parents/guardians) about current health, both physical and emotional • Examine their experience of transplantation and enzyme replacement therapy with laronidase (Aldurazyme), and how well these therapies live up to expectations in managing symptoms and improving quality of life • Obtain input on support services provided by Genzyme and national MPS societies in order to plan programs that encourage optimal treatment across the spectrum of MPS I

  3. Methodology • Survey design • 8-page questionnaire designed by Genzyme in collaboration with national MPS societies; translated into local languages • Survey distributed & collected by national MPS societies • Mail: Japan, Australia, Canada, US, Netherlands, Germany • Society meeting: Poland, Brazil • Telephone: UK • Survey period • September-October 2006 (US) • June-November 2007 (all other participating countries) • Survey participation • 45% response rate • 238 completed questionnaires: Japan (9), Australia (9), Canada (17), US (61), UK (83), Netherlands (10), Germany (14), Poland (14), Brazil (21)

  4. Self-assessment of most MPS I patients’ health is positive on overall physical and emotional measures Overall Health Assessment Emotional Health Physical Health Higher favorable scores for Hurler (45% Ex/VG), <1 year old at transplant (80%) Higher unfavorable scores for Hurler-Scheie (29% Fair/Poor), Scheie (27%) Higher unfavorable scores for Hurler-Scheie (20% Fair/Poor) Q4. On most days, how would you rate your . . . Physical health? (N=237). . . Emotional health? (N=235)

  5. 84% of recipients rate transplantation performance highly favorably (8,9,10 ratings), including majority rating “10” 1=Extremely Poor 10=Extremely Well No “1,2,3” ratings received Mean Score: 8.95 Q14. Based on your experience to date, how would you rate the overall performance of transplant therapy as a treatment for MPS I? (N=103)

  6. Transplantation patients report “a lot” or “some” improvement on many parameters (including mental development); less on musculoskeletal Q15. How much improvement, if any, do you believe transplant therapy has had on your health and symptoms in each of these areas? (N=103)

  7. The earlier that patients receive transplant, the more likely they are to experience improvement on several health parameters Age at transplant Musculoskeletal Respir Cranio Vision Abdomin Sleep Cardiac Mental QoL Perceived Improvement Q25. How much improvement, if any, do you believe transplant therapy has had on your health and symptoms in each of these areas? (N=10 <1 year old at transplant [caution, small sample size]; N=93 age 1+ at transplant)

  8. A 63% majority rate Enzyme Replacement Therapy performance highly favorably (8,9,10 ratings) 1=Extremely Poor 10=Extremely Well Mean Score: 8.14 Q24. Based on your experience to date, how would you rate the overall performance of ERT as a treatment for MPS I? (N=135)

  9. Widespread reports of improvements from ERT on quality of life and physical symptoms, including musculoskeletal • The most improvements across parameters reported: • Hurler and Hurler-Scheie • Age <6 • Currently on ERT 3+ yrs • The least improvements across parameters reported: • Scheie • Age 6-17 • Currently on ERT <3 yrs Q25. How much improvement, if any, do you believe ERT has had on your health and symptoms in each of these areas? (N=133)

  10. The longer that patients receive ERT, the better their reported outcomes on musculoskeletal, respiratory, abdominal, cardiac and quality of life Time on ERT Musculoskeletal Respir Cranio Vision Abdomin Sleep Cardiac Mental QoL Perceived Improvement Q25. How much improvement, if any, do you believe ERT has had on your health and symptoms in each of these areas? (N=47 currently on ERT <3 yr; N=63 currently on ERT 3+ yr)

  11. ERT adherence rate is high; however, 2 in 10 patients report missing four or more consecutive infusions Biggest gaps in adherence reported among Hurler-Scheie (25%), Poland (57%), Canada (33%), Brazil (30%) Highest adherence rates among patients on home infusion (100%), Scheie (93%), US (97%), Australia (100%), Japan (100%) • The main reasons reported for missing infusions are: • health related, i.e., hospitalization, surgery, feeling too sick, pregnancy • scheduling problems, i.e., too busy, traveling, moving, vacationing • Drug not available and/or financial/insurance issues especially in Poland, Brazil Q20. Have/had you ever missed 4 or more consecutive infusions? (N=134) Q21. Which of the following items, if any, describe the main reasons you missed or stopped ERT? (N=134)

  12. In addition to those already receiving home infusion, many other respondents express high interest Home infusion experience reported in US (40%), Netherlands (56%), Germany and Canada Greatest additional interest in Canada (88%, 7 out of 8 patients extremely interested) and UK 62% Q32. How interested would you be in each of the following types of ERT administration and infusion methods? Home infusion:A nurse or clinician comes to your home to prepare the solution and administer the infusion. (N=89 currently on ERT)

  13. Conclusions • MPS I patients receiving therapy give overall positive reports on physical and emotional health and high satisfaction rates for both transplantation and ERT. • Among transplanted patients, 60% reported complications and 26% repeat procedures; however, fully 84% rated transplantation performance highly favorably (mean score 8.95 on a 1-10 scale) and 75% reported some/a lot of improvement on mental development. • Overall, the earlier the transplant, the greater the satisfaction. Lowest rates of satisfaction were with cardiac, visual, and musculoskeletal symptoms. • Among patients who received ERT, 63% rated performance highly favorably (mean score 8.14), with highest ratings for quality of life, gastrointestinal, sleep, and musculoskeletal symptoms and the lowest ratings for mental development, cardiac, craniofacial, and visual symptoms. • The longer the patients were on ERT, the greater their satisfaction with treatment.