Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015-2022): 2022 interim analysis

Imagem de Miniatura
Arquivos
art_MERCURI_Safety_and_effectiveness_of_ataluren_in_patients_with_2023.PDF (860.94 KB)
Citações na Scopus
8
Tipo de produção
article
Data de publicação
2023
DOI
Scopus
Web of Science
PubMed
Título da Revista
ISSN da Revista
Título do Volume
Editora
SPRINGER HEIDELBERG
Autores
MERCURI, Eugenio
OSORIO, Andres Nascimento
MUNTONI, Francesco
BUCCELLA, Filippo
DESGUERRE, Isabelle
KIRSCHNER, Janbernd
TULINIUS, Mar
MORGENROTH, Lauren P.
GORDISH-DRESSMAN, Heather
Citação
JOURNAL OF NEUROLOGY, v.270, n.8, p.3896-3913, 2023
Projetos de Pesquisa
Unidades Organizacionais
Fascículo
Resumo
ObjectiveStrategic Targeting of Registries and International Database of Excellence (STRIDE) is an ongoing, international, multicenter registry of real-world ataluren use in individuals with nonsense mutation Duchenne muscular dystrophy (nmDMD) in clinical practice. This updated interim report (data cut-off: January 31, 2022), describes STRIDE patient characteristics and ataluren safety data, as well as the effectiveness of ataluren plus standard of care (SoC) in STRIDE versus SoC alone in the Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS).MethodsPatients are followed up from enrollment for at least 5 years or until study withdrawal. Propensity score matching was performed to identify STRIDE and CINRG DNHS patients who were comparable in established predictors of disease progression.ResultsAs of January 31, 2022, 307 patients were enrolled from 14 countries. Mean (standard deviation [SD]) ages at first symptoms and at genetic diagnosis were 2.9 (1.7) years and 4.5 (3.7) years, respectively. Mean (SD) duration of ataluren exposure was 1671 (56.8) days. Ataluren had a favorable safety profile; most treatment-emergent adverse events were mild or moderate and unrelated to ataluren. Kaplan-Meier analyses demonstrated that ataluren plus SoC significantly delayed age at loss of ambulation by 4 years (p < 0.0001) and age at decline to %-predicted forced vital capacity of < 60% and < 50% by 1.8 years (p = 0.0021) and 2.3 years (p = 0.0207), respectively, compared with SoC alone.ConclusionLong-term, real-world treatment with ataluren plus SoC delays several disease progression milestones in individuals with nmDMD. NCT02369731; registration date: February 24, 2015.
Palavras-chave
Ataluren, Effectiveness, Nonsense mutation Duchenne muscular dystrophy, Safety, STRIDE Registry
URI
https://observatorio.fm.usp.br/handle/OPI/54942
Referências
  1. Andrews JG, 2019, SAGE OPEN MED, V7, DOI 10.1177/2050312119840518
  2. Bello Luca, 2016, Acta Myol, V35, P122
  3. Bello L, 2016, NEUROLOGY, V87, P401, DOI 10.1212/WNL.0000000000002891
  4. Birnkrant DJ, 2018, LANCET NEUROL, V17, P347, DOI 10.1016/S1474-4422(18)30025-5
  5. Birnkrant DJ, 2018, LANCET NEUROL, V17, P251, DOI 10.1016/S1474-4422(18)30024-3
  6. Bushby K, 2014, MUSCLE NERVE, V50, P477, DOI 10.1002/mus.24332
  7. Bushby K, 2010, LANCET NEUROL, V9, P177, DOI 10.1016/S1474-4422(09)70272-8
  8. Chesshyre M, 2022, J CACHEXIA SARCOPENI, V13, P1360, DOI 10.1002/jcsm.12914
  9. D'Amico A, 2017, NEUROMUSCULAR DISORD, V27, P447, DOI 10.1016/j.nmd.2017.02.006
  10. Ellis JA, 2013, NEUROMUSCULAR DISORD, V23, P682, DOI 10.1016/j.nmd.2013.05.008
  11. European Medicines Agency, TRANSL SUMM PROD CHA
  12. Guglieri M, 2022, JAMA-J AM MED ASSOC, V327, P1456, DOI 10.1001/jama.2022.4315
  13. Humbertclaude V, 2012, EUR J PAEDIATR NEURO, V16, P149, DOI 10.1016/j.ejpn.2011.07.001
  14. Inacio MCS, 2015, CLIN ORTHOP RELAT R, V473, P2722, DOI 10.1007/s11999-015-4239-4
  15. Landfeldt E, 2020, EUR J EPIDEMIOL, V35, P643, DOI 10.1007/s10654-020-00613-8
  16. Mayer OH, 2017, US NEUROL, V13, P35, DOI 10.17925/USN.2017.13.01.35
  17. Mazzone ES, 2013, PLOS ONE, V8, DOI 10.1371/journal.pone.0052512
  18. McDonald CM, 2021, J COMP EFFECT RES, V11, P139, DOI 10.2217/cer-2021-0196
  19. McDonald CM, 2018, LANCET, V391, P451, DOI 10.1016/S0140-6736(17)32160-8
  20. McDonald CM, 2017, LANCET, V390, P1489, DOI 10.1016/S0140-6736(17)31611-2
  21. McDonald CM, 2013, MUSCLE NERVE, V48, P32, DOI 10.1002/mus.23807
  22. Mercuri E., 2022, VIDEO J BIOMED, DOI [10.2217/vjbm-2022-0014, DOI 10.2217/VJBM-2022-0014]
  23. Mercuri E, 2020, J COMP EFFECT RES, V9, P341, DOI 10.2217/cer-2019-0171
  24. Muntoni F, 2023, NEUROLOGY, V100, pE1540, DOI 10.1212/WNL.0000000000201626
  25. Muntoni F, 2019, J COMP EFFECT RES, V8, P1187, DOI 10.2217/cer-2019-0086
  26. Peltz SW, 2013, ANNU REV MED, V64, P407, DOI 10.1146/annurev-med-120611-144851
  27. Phillips MF, 2001, AM J RESP CRIT CARE, V164, P2191, DOI 10.1164/ajrccm.164.12.2103052
  28. Ricotti V, 2016, J NEUROL NEUROSUR PS, V87, P149, DOI 10.1136/jnnp-2014-309405
  29. Roy B, 2016, P NATL ACAD SCI USA, V113, P12508, DOI 10.1073/pnas.1605336113
  30. Ryder S, 2017, ORPHANET J RARE DIS, V12, DOI 10.1186/s13023-017-0631-3
  31. Trucco F, 2022, MUSCLE NERVE, V65, P67, DOI 10.1002/mus.27427
  32. Vry Julia, 2016, J Neuromuscul Dis, V3, P517
  33. Wang L, 2017, FRONT NEUROL, V8, DOI 10.3389/fneur.2017.00196

Coleções
Artigos e Materiais de Revistas Científicas - HC/ICHC
Artigos e Materiais de Revistas Científicas - ODS/03