Transitioning From Nusinersen to Risdiplam for Spinal Muscular Atrophy in Clinical Practice: A Single-Center Experience.
| Title: | Transitioning From Nusinersen to Risdiplam for Spinal Muscular Atrophy in Clinical Practice: A Single-Center Experience. |
|---|---|
| Authors: | Bekircan-Kurt CE; Department of Neurology, School of Medicine, Hacettepe University, Ankara, Turkey.; Subramanian S; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, Ohio, USA.; Chagat S; Department of Neurology, Neuromuscular Division, Nationwide Children's Hospital, Columbus, Ohio, USA.; Mackenzie SJ; Department of Neurology, Neuromuscular Division, University of Rochester Medical Center, Rochester, New York, USA.; Iammarino M; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, Ohio, USA.; Reash N; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, Ohio, USA.; Richardson C; Biostatistics Research Core, Ohio State University, Rochester, Ohio, USA.; Tsao CY; Department of Neurology, Neuromuscular Division, Nationwide Children's Hospital, Columbus, Ohio, USA.; Department of Pediatrics, Ohio State University, Wexner Medical Center, Columbus, Ohio, USA.; Noritz G; Department of Pediatrics, Ohio State University, Wexner Medical Center, Columbus, Ohio, USA.; Gushue C; Department of Pediatrics, Ohio State University, Wexner Medical Center, Columbus, Ohio, USA.; Department of Pulmonary Medicine, Nationwide Children's Hospital, Columbus, Ohio, USA.; Kotha K; Department of Pediatrics, Ohio State University, Wexner Medical Center, Columbus, Ohio, USA.; Department of Pulmonary Medicine, Nationwide Children's Hospital, Columbus, Ohio, USA.; Paul G; Department of Pediatrics, Ohio State University, Wexner Medical Center, Columbus, Ohio, USA.; Department of Pulmonary Medicine, Nationwide Children's Hospital, Columbus, Ohio, USA.; Shell R; Department of Pediatrics, Ohio State University, Wexner Medical Center, Columbus, Ohio, USA.; Department of Pulmonary Medicine, Nationwide Children's Hospital, Columbus, Ohio, USA.; Alfano LN; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, Ohio, USA.; Department of Pediatrics, Ohio State University, Wexner Medical Center, Columbus, Ohio, USA.; Lowes LP; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, Ohio, USA.; Department of Pediatrics, Ohio State University, Wexner Medical Center, Columbus, Ohio, USA.; Connolly AM; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, Ohio, USA.; Department of Neurology, Neuromuscular Division, Nationwide Children's Hospital, Columbus, Ohio, USA.; Department of Pediatrics, Ohio State University, Wexner Medical Center, Columbus, Ohio, USA.; Waldrop MA; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, Ohio, USA.; Department of Neurology, Neuromuscular Division, Nationwide Children's Hospital, Columbus, Ohio, USA.; Department of Pediatrics, Ohio State University, Wexner Medical Center, Columbus, Ohio, USA. |
| Source: | Muscle & nerve [Muscle Nerve] 2025 Mar; Vol. 71 (3), pp. 414-421. Date of Electronic Publication: 2025 Jan 02. |
| Publication Type: | Journal Article |
| Language: | English |
| Journal Info: | Publisher: John Wiley & Sons Country of Publication: United States NLM ID: 7803146 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1097-4598 (Electronic) Linking ISSN: 0148639X NLM ISO Abbreviation: Muscle Nerve Subsets: MEDLINE |
| Imprint Name(s): | Publication: : Hoboken, NJ : John Wiley & Sons; Original Publication: New York, NY : John Wiley & Sons |
| MeSH Terms: | Oligonucleotides*/therapeutic use ; Muscular Atrophy, Spinal*/drug therapy ; Muscular Atrophy, Spinal*/physiopathology ; Spinal Muscular Atrophies of Childhood*/drug therapy; Humans ; Male ; Female ; Retrospective Studies ; Child, Preschool ; Infant ; Treatment Outcome ; Child ; Drug Combinations ; Azo Compounds ; Pyrimidines |
| Abstract: | Background: Nusinersen and risdiplam are U.S. Food and Drug Administration (FDA)-approved treatments for spinal muscular atrophy (SMA). No head-to-head clinical trials to assess efficacy exist. Observational studies are needed to determine if transitioning to risdiplam is safe and efficacious.; Methods: This retrospective study at Nationwide Children's Hospital included individuals with SMA treated with nusinersen who switched to risdiplam. Motor, pulmonary and bulbar function were assessed before and 2 years after nusinersen and risdiplam initiation.; Results: Forty-four individuals were included: 11 with SMA type 1, 25 with SMA type 2 and 8 with SMA type 3. Motor function improved after initiation of nusinersen treatment with the most significant improvements seen in the first year. After transition to risdiplam, motor function remained largely stable. Need for noninvasive ventilation (NIV) overnight occurred in both groups. Cough peak flow significantly improved in the risdiplam group. Hospitalizations were the same in both groups. One individual in the nusinersen group gained the ability to take some food by mouth; two individuals in the risdiplam group achieved some oral feeding and two became exclusively orally fed.; Conclusions: As expected, motor function was most improved in treatment naïve individuals in the first year after nusinersen initiation. Over half of our study population had posterior spinal fusion surgery (57%) which significantly impacted motor and respiratory outcomes, though slightly less so in the risdiplam group. Overall, our data demonstrates that transitioning from nusinersen to risdiplam is associated with a favorable safety profile and stable motor outcomes.; (© 2025 The Author(s). Muscle & Nerve published by Wiley Periodicals LLC.) |
| References: | N Engl J Med. 2018 Feb 15;378(7):625-635. (PMID: 29443664); J Child Neurol. 2011 Dec;26(12):1499-507. (PMID: 21940700); Pediatr Neurol. 2021 Sep;122:21-26. (PMID: 34271497); J Neuromuscul Dis. 2024;11(2):361-368. (PMID: 38189761); Neuromuscul Disord. 2018 Mar;28(3):197-207. (PMID: 29305137); J Pers Med. 2024 Feb 24;14(3):. (PMID: 38540986); Neurosci Insights. 2020 Nov 23;15:2633105520973985. (PMID: 33283185); Neuromuscul Disord. 2018 Feb;28(2):103-115. (PMID: 29290580); Orphanet J Rare Dis. 2011 Nov 02;6:71. (PMID: 22047105); J Neuromuscul Dis. 2020;7(2):97-100. (PMID: 32007960); Neuromuscul Disord. 2019 Nov;29(11):842-856. (PMID: 31704158); J Coll Physicians Surg Pak. 2024 Aug;34(8):948-955. (PMID: 39113515); N Engl J Med. 2017 Nov 2;377(18):1713-1722. (PMID: 29091557); Pediatr Pulmonol. 2021 Apr;56(4):700-709. (PMID: 32720756); Pediatr Pulmonol. 2023 Jul;58(7):1866-1874. (PMID: 37144867); Neurol Ther. 2023 Apr;12(2):543-557. (PMID: 36780114); N Engl J Med. 2017 Nov 2;377(18):1723-1732. (PMID: 29091570); N Engl J Med. 2021 Jul 29;385(5):427-435. (PMID: 34320287); Neurology. 2019 May 21;92(21):e2492-e2506. (PMID: 31019106); J Comp Eff Res. 2022 Apr;11(5):347-370. (PMID: 35040693); Chest. 1997 Oct;112(4):1024-8. (PMID: 9377912); Muscle Nerve. 2024 Feb;69(2):179-184. (PMID: 38040488); Muscle Nerve. 2024 Nov;70(5):1095-1098. (PMID: 39136364); Neuromuscul Disord. 2010 Mar;20(3):155-61. (PMID: 20074952); J Neurol. 2024 Aug;271(8):4871-4884. (PMID: 38733387); Muscle Nerve. 2017 Jun;55(6):869-874. (PMID: 27701745); J Neuromuscul Dis. 2018;5(2):145-158. (PMID: 29614695); Eur J Neurol. 2023 Jul;30(7):1945-1956. (PMID: 35837793); Neurol Clin Pract. 2024 Aug;14(4):e200310. (PMID: 38915908) |
| Grant Information: | K12 HS026393 United States HS AHRQ HHS; 5K12HS026393-03 PEDSnet Scholars Training Program; Agency for Healthcare Research and Quality |
| Contributed Indexing: | Keywords: CHOP; HFMSE; NIV; nusinersen; risdiplam; spinal muscular atrophy |
| Substance Nomenclature: | 5Z9SP3X666 (nusinersen); 0 (Oligonucleotides); 76RS4S2ET1 (Risdiplam); 0 (Drug Combinations); 0 (Azo Compounds); 0 (Pyrimidines) |
| Entry Date(s): | Date Created: 20250102 Date Completed: 20250429 Latest Revision: 20260127 |
| Update Code: | 20260130 |
| PubMed Central ID: | PMC11799403 |
| DOI: | 10.1002/mus.28329 |
| PMID: | 39744899 |
| Database: | MEDLINE |
Journal Article