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AlRuthia Y, Almuaythir GS, H Alrasheed H, et al. Proxy-Reported Quality of Life and Access to Nusinersen Among Patients with Spinal Muscular Atrophy in Saudi Arabia. Patient Prefer Adherence. 2021;15:729-739doi: 10.2147/PPA.S305849.
AlRuthia, Y., Almuaythir, G. S., H Alrasheed, H., Alsharif, W. R., Temsah, M. H., Alsohime, F., Sales, I., Alwhaibi, M., & Bashiri, F. A. (2021). Proxy-Reported Quality of Life and Access to Nusinersen Among Patients with Spinal Muscular Atrophy in Saudi Arabia. Patient preference and adherence, 15729-739. https://doi.org/10.2147/PPA.S305849
AlRuthia, Yazed, et al. "Proxy-Reported Quality of Life and Access to Nusinersen Among Patients with Spinal Muscular Atrophy in Saudi Arabia." Patient preference and adherence vol. 15 (2021): 729-739. doi: https://doi.org/10.2147/PPA.S305849
AlRuthia Y, Almuaythir GS, H Alrasheed H, Alsharif WR, Temsah MH, Alsohime F, Sales I, Alwhaibi M, Bashiri FA. Proxy-Reported Quality of Life and Access to Nusinersen Among Patients with Spinal Muscular Atrophy in Saudi Arabia. Patient Prefer Adherence. 2021 Apr 13;15:729-739. doi: 10.2147/PPA.S305849. eCollection 2021. PMID: 33880016; PMCID: PMC8053517.
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Patient Prefer Adherence. 2021 Apr 13;15:729-739. doi: 10.2147/PPA.S305849. eCollection 2021.

Proxy-Reported Quality of Life and Access to Nusinersen Among Patients with Spinal Muscular Atrophy in Saudi Arabia.

Patient preference and adherence

Yazed AlRuthia, Ghadah S Almuaythir, Hala H Alrasheed, Wejdan R Alsharif, Mohamad-Hani Temsah, Fahad Alsohime, Ibrahim Sales, Monira Alwhaibi, Fahad A Bashiri

Affiliations

  1. Department of Clinical Pharmacy, College of Pharmacy, King Saud University, Riyadh, Saudi Arabia.
  2. Pharmacoeconomics Research Unit, College of Pharmacy, King Saud University, Riyadh, Saudi Arabia.
  3. College of Medicine, King Saud University, Riyadh, Saudi Arabia.
  4. Pediatric Intensive Care Unit, Pediatric Department, King Saud University Medical City, Riyadh, Saudi Arabia.
  5. Division of Neurology, Department of Pediatrics, King Saud University Medical City, Riyadh, Saudi Arabia.

PMID: 33880016 PMCID: PMC8053517 DOI: 10.2147/PPA.S305849

Abstract

BACKGROUND: The recent approval of innovative therapies for spinal muscular atrophy (SMA), such as nusinersen, has brought hope to patients and their families.

OBJECTIVE: The aims of this study were to compare the characteristics and HRQoL of SMA patients treated with nusinersen and those treated with the standard of care.

METHODS: This was a cross-sectional, interviewer-administered telephone questionnaire, which used a purposive sampling of SMA patients through a social support network. EuroQol five-dimensions-3-level (EQ-5D-3L) and the visual analog scale (VAS) have been used to assess the HRQoL. Different descriptive and inferential tests have been performed to compare the characteristics, EQ-5D responses, and mean scores of EQ-VAS between patients on nusinersen and the standard of care.

RESULTS: Eleven out of 36 SMA patients (30.55%) have been treated with nusinersen. Patients with type I SMA represented 54% of those treated with nusinersen (

CONCLUSION: The results of this study highlight the uncertainty about the impact of nusinersen on SMA patients' HRQoL. Therefore, the impact of nusinersen on HRQoL should be examined using more robust study designs.

© 2021 AlRuthia et al.

Keywords: Saudi Arabia; health services accessibility; health-related quality of life; nusinersen; spinal muscular atrophy

Conflict of interest statement

The authors report no conflicts of interest in this work.

References

  1. Orphanet J Rare Dis. 2017 Jul 4;12(1):124 - PubMed
  2. Genet Med. 2002 Jan-Feb;4(1):20-6 - PubMed
  3. Lancet. 2008 Jun 21;371(9630):2120-33 - PubMed
  4. Pharmacoeconomics. 2019 Jun;37(6):845-865 - PubMed
  5. Health Qual Life Outcomes. 2008 Nov 07;6:94 - PubMed
  6. Ther Adv Neurol Disord. 2020 Mar 5;13:1756286420907803 - PubMed
  7. J Clin Med. 2020 Jul 13;9(7): - PubMed
  8. J Neuromuscul Dis. 2018;5(2):135-143 - PubMed
  9. N Engl J Med. 2018 Feb 15;378(7):625-635 - PubMed
  10. BMJ Paediatr Open. 2019 Dec 5;3(1):e000572 - PubMed
  11. Lancet. 2017 Dec 17;388(10063):3017-3026 - PubMed
  12. Cell. 1995 Jan 13;80(1):155-65 - PubMed
  13. Arch Neurol. 2011 Aug;68(8):979-84 - PubMed
  14. Eur J Paediatr Neurol. 2019 May;23(3):347-356 - PubMed
  15. Cost Eff Resour Alloc. 2020 Oct 6;18:41 - PubMed
  16. N Engl J Med. 2017 Nov 2;377(18):1723-1732 - PubMed
  17. JAMA. 2013 Nov 27;310(20):2191-4 - PubMed
  18. Ann Saudi Med. 2009 Jul-Aug;29(4):304-8 - PubMed
  19. Neurology. 2018 Oct 2;91(14):e1312-e1318 - PubMed
  20. Orphanet J Rare Dis. 2020 Jun 12;15(1):148 - PubMed
  21. J Neurosci. 2015 Jun 10;35(23):8691-700 - PubMed
  22. Ann R Coll Surg Engl. 2013 Jan;95(1):5-7 - PubMed
  23. Gene Ther. 2017 Sep;24(9):520-526 - PubMed
  24. J Public Health (Oxf). 2005 Sep;27(3):281-91 - PubMed
  25. Orphanet J Rare Dis. 2020 Aug 24;15(1):217 - PubMed
  26. Ann Neurol. 2019 Sep;86(3):443-451 - PubMed

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