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Comparative Efficacy of Risdiplam and Nusinersen in Type 2 and 3 Spinal Muscular Atrophy Patients: A Cohort Study Using Real-World Data Publisher Pubmed



Ashrafi MR1, 2 ; Babaee M3 ; Hashemi Nazari SS4 ; Barzegar M5 ; Ghazavi M6 ; Beiraghi Toosi M7 ; Nafissi S8 ; Inaloo S9 ; Zamani Ghaletaki G1 ; Fatehi F8 ; Heshmat R10 ; Ghahvechi Akbari M11 ; Abdi A12 ; Bakhtiary H13 Show All Authors
Authors
  1. Ashrafi MR1, 2
  2. Babaee M3
  3. Hashemi Nazari SS4
  4. Barzegar M5
  5. Ghazavi M6
  6. Beiraghi Toosi M7
  7. Nafissi S8
  8. Inaloo S9
  9. Zamani Ghaletaki G1
  10. Fatehi F8
  11. Heshmat R10
  12. Ghahvechi Akbari M11
  13. Abdi A12
  14. Bakhtiary H13
  15. Montazerlotfelahi H14
  16. Abbaskhanian A15
  17. Hosseini SA16
  18. Farshadmoghadam H17
  19. Hosseiny SMM18
  20. Shariatmadari F19
  21. Ziaadini B20
  22. Babaei M21
  23. Tavasoli A22
  24. Nikbakht S23
  25. Momen A24
  26. Khajeh A25
  27. Aminzadeh V26
  28. Mollamohammadi M27
  29. Taghdiri MM28
  30. Nasehi MM28
  31. Memarian S1
  32. Badv RS1
  33. Heidari M1
  34. Jafari N28
Show Affiliations
Authors Affiliations
  1. 1. Department of Pediatrics, Division of Pediatric Neurology, Children's Medical Center, Pediatrics Center of Excellence, Tehran University of Medical Sciences, Tehran, Iran
  2. 2. Pediatric Cell and Gene Therapy Research Center, Cell & Tissue Research Institute, Tehran University of Medical Sciences, Gene, Tehran, Iran
  3. 3. Physical Medicine and Rehabilitation Research Center, Shahid Beheshti University of Medical Sciences, Tehran, Iran
  4. 4. Prevention of Cardiovascular Disease Research Center, Department of Epidemiology, School of Public Health and Safety, Shahid Beheshti University of Medical Sciences, Tehran, Iran
  5. 5. Pediatric Health Research Center, Mardani Azari Children Hospital, Tabrizi University of Medical Sciences, Tabriz, Iran
  6. 6. Pediatric Neurology department, Imam Hossein Children's Hospital, Isfahan University of Medical Sciences, Iran
  7. 7. Pediatric ward, Faculty of Medicine, Mashhad University of Medical Sciences, Mashhad, Iran
  8. 8. Iranian Neuromuscular Research Center (INMRC), Tehran Medical University of Medical Sciences, Tehran, Iran
  9. 9. Neonatal Research Center Shiraz University of Medical Sciences, Shiraz, Iran
  10. 10. Chronic Diseases Research Center, Endocrinology and Metabolism Population Sciences Institute, Tehran University of Medical Sciences, Tehran, Iran
  11. 11. Physical Medicine and Rehabilitation Department, Children's Medical Center, Tehran University of Medical Sciences, Tehran, Iran
  12. 12. Department of pediatrics, Urmia University of Medical Sciences, Iran
  13. 13. Moosavi Hospital, Zanjan University of Medical Sciences, Zanjan, Iran
  14. 14. Imam Ali Hospital, Faculty of Medicine, Alborz University of Medical Sciences, Karaj, Iran
  15. 15. Clinical Research Development Unit of Bu-Ali Sina Hospital, Mazandaran University of Medical Science, Sari, Iran
  16. 16. Neonatal and children's health research center, Taleghani Medicine Educational Center, Golestan University of Medical Sciences, Gorgan, Iran
  17. 17. Department of Pediatrics, Children Growth Research Centre, Research Institute for Prevention of Non Communicable Disease, Qazvin University of Medical Science, Qazvin, Iran
  18. 18. Department of Pediatrics, School of Medicine, Hamadan University of Medical Sciences, Hamadan, Iran
  19. 19. Department of Pediatrics, Arak Medical University, Arak, Iran
  20. 20. Neurology Research Center, Kerman University of Medical Sciences, Kerman, Iran
  21. 21. Department of Pediatrics, North Khorasan University of Medical Sciences, Bojnurd, Iran
  22. 22. Department of Pediatric Neurology, Iran University of Medical Sciences, Tehran, Iran
  23. 23. Lorestan University of Medical Sciences, Khorramabad, Iran
  24. 24. Pediatric Department, Ahvaz Jundishapur University of Medical Sciences, Ahvaz, Iran
  25. 25. Children and Adolescents Health Research Center, Zahedan University of Medical Science, Zahedan, Iran
  26. 26. Pediatric Diseases Research Center, Guilan University of Medical Sciences, Rasht, Iran
  27. 27. Qom University of Medical Sciences, Qom, Iran
  28. 28. Pediatric Neurology Research Center, Research Institute for Children's Health, Shahid Beheshti University of Medical Sciences, Tehran, Iran

Source: Journal of neuromuscular diseases Published:2024


Abstract

BACKGROUND: Three medications have been approved for spinal muscular atrophy (SMA) treatment. No head-to-head clinical trials have directly compared the efficacy of nusinersen and risdiplam. We compare the efficacy of them in Type 2 and 3 SMA patients, with 6 months of follow-up. METHODS: A multicenter cohort study was conducted. Demographic, genetic and clinical findings containing Hammersmith Functional Motor Scale Expanded (HFMSE) and revised upper limb module (RULM) scales were gathered. An increase of at least 3 points in HFMSE and RULM scores was considered a positive response. RESULTS: 73 (58.4%) children received risdiplam, and 52 (41.6%) received nusinersen non-randomly, based on clinical decision. The difference in HFMSE and RULM scores compared to the baseline was significant in both groups (P-value <0.001). However, there was no significant difference between mean difference changes in HFMSE and RULM scores between the two groups. 80.4% of patients in the risdiplam group and 72% in the nusinersen group achieved the 3-point cutoff after 6 months, and there is no significant difference between the two groups (P-Value:0.33). CONCLUSIONS: This study showed that both medications significantly changed the HFSME and RULM after 3 and 6 months of follow-up. However, there was no significant difference between the two drugs according to the HFSME.