Wild, C. (2020): Update 12 months follow-up: Nusinersen in spinal muscular atrophy ("late onset") in children and adolescents ≥ 6 years. AIHTA Policy Brief 001.
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Background: In June 2017, Nusinersen (Spinraza®) was approved broadly by the European Medicines Agency (EMA) for the treatment of chromosome 5q13 (5q)-associated SMA - for all SMA typologies (despite the lack of evidence). It is currently the only disease-modifying (containment of disease progression) treatment approved in Europe. Another therapy for presymptomatic children has been approved in the USA (Zolgensma®, Onasemnogen abeparvovec), but not in Europe due to the lack of preclinical data. Further therapy developments for SMA 1-3 patients are taking place and will be approved in the next few years. The question of the update presented here is which scientific evidence from clinical intervention or observational studies for the medium (or long-term) benefit of Nusinersen (Spinraza®) in children and adolescents ≥ 6 years with SMA "late onset" (SMA 2 + 3) is available.
Method: For this purpose, a hand search was carried out on March 17, 2020 in one database (Medline) restricted to the years 2018-2020 and to data on medium-term effects (≥ 12 months) in the patient groups children and adolescents ≥ 6 years with SMA "late onset" (SMA 2 + 3).
Results: Three publications on the question of the medium-term benefit (follow-up period ≥ 12 months) of Nusinersen (Spinraza®) in children and adolescents ≥ 6 years (SMA 2 + 3 with a total of 40 patients) were identified. The available evidence for medium-term (> 12 months) follow-up data comes from a single prospective sponsor-funded ISIS CS12 study, as well as from a small retrospective study (of poor quality). Moderate HFSME improvements beyond the thresholds of clinical relevance in SMA2 and hardly any improvements (stabilisation) in SMA 3 are reported. While the baseline data are reported in detail, there is no data-based - only narrative - reporting of the follow-up data. No information about the many patients who start the study, but are not reported, is given.
Conclusion: Even after more than two years after the approval of Nusinersen, the evidence for this patient population is of extremely low quality, lacking validity.
|Item Type:||Rapid Assessment LBI-HTA|
|Keywords:||Nusinersen, Spinraza, spinal muscular atrophy|
|Subjects:||WB Practice of medicine > WB 300-962 Therapeutics|
WE Musculoskeletal system > WE 500-600 Muscles and tendons
|Series Name:||AIHTA Policy Brief 001|
|Deposited on:||27 Mar 2020 13:28|
|Last Modified:||15 Apr 2020 16:59|
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