scholarly journals Comparison of Nusinersen and Evrysdi in the Treatment of Spinal Muscular Atrophy

2021 ◽  
Vol 271 ◽  
pp. 03035
Author(s):  
Xiaoying Zhu
Keyword(s):  
Quality Of Life ◽  
Spinal Muscular Atrophy ◽  
Positive Impact ◽  
Muscular Atrophy ◽  
Indirect Comparison ◽  
Permanent Disability ◽  
Real World Evidence ◽  
Adjusted Indirect Comparison ◽  
Matching Adjustment

Spinal Muscular Atrophy (SMA) is a genetic neuromuscular disease that commonly affects children, and usually worsens with age that often leads to permanent disability and death for many of the SMA patients. Recently, two drugs are developed to improving the quality of life of SMA sufferers: Evrysdi and Nusinersen. This study is identified by a systematic literature review to compare two treatments. The comparison attempts to focus on mechanism, administration and clinical trials. The trials include the ENDEAR study for Nusinersen, and the FIREFISH study for Evrysdi. Due to the different baselines of two trials, matching-adjusted indirect comparison (MAIC) is used to “weighted” baseline characteristics to match each other across all the studies. Each of the trials highlighted the effectiveness for comparison. Both Nusinersen and Evrysdi have had a major and positive impact on improving the quality of life of SMA, and both therapies have been shown to be highly effective. Moreover, the indirect comparison with Matching Adjustment Indirect Comparison shows that Risdiplam is more effective as compared to Nusinersen. Nonetheless, the comparison is still inaccurate due to lack of real-world evidence from patients.

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

2021 ◽  
Vol Volume 15 ◽  
pp. 729-739
Author(s):  
Yazed AlRuthia ◽  
Ghadah S Almuaythir ◽  
Hala H Alrasheed ◽  
Wejdan R Alsharif ◽  
Mohamad-Hani Temsah ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Saudi Arabia ◽  
Spinal Muscular Atrophy ◽  
Muscular Atrophy

Prenusinersen economic and health-related quality of life burden of spinal muscular atrophy

Neurology ◽  
2020 ◽  
Vol 95 (1) ◽  
pp. e1-e10 ◽  
Author(s):  
Georgina M. Chambers ◽  
Stella Nalukwago Settumba ◽  
Kate A. Carey ◽  
Anita Cairns ◽  
Manoj P. Menezes ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Spinal Muscular Atrophy ◽  
Purchasing Power Parity ◽  
Muscular Atrophy ◽  
Total Annual Cost ◽  
Health Related Quality ◽  
Power Parity ◽  
Related Quality ◽  
Health Related

ObjectiveTo quantify the economic and health-related quality of life (HRQoL) burden incurred by households with a child affected by spinal muscular atrophy (SMA).MethodsHospital records, insurance claims, and detailed resource use questionnaires completed by caregivers were used to capture the direct and indirect costs to households of 40 children affected by SMA I, II, and III in Australia between 2016 and 2017. Prevalence costing methods were used and reported in 2017 US dollar (USD) purchasing power parity (PPP). The HRQoL for patients and primary caregivers was quantified with the youth version of the EQ-5D and CareQoL multiattribute utility instruments and Australian utility weights.ResultsThe average total annual cost of SMA per household was $143,705 USD PPP for all SMA types (SMA I $229,346, SMA II $150,909, SMA III $94,948). Direct costs accounted for 56% of total costs. The average total indirect health care costs for all SMA types were $63,145 per annum and were highest in families affected by SMA II. Loss of income and unpaid informal care made up 24.2% and 19.8% respectively, of annual SMA costs. Three of 4 (78%) caregivers stated that they experienced financial problems because of care tasks. The loss in HRQoL of children affected by SMA and caregivers was substantial, with average caregiver and patient scores of 0.708 and 0.115, respectively (reference range 0 = death and 1 = full health).ConclusionOur results demonstrate the substantial and far-ranging economic and quality of life burden on households and society of SMA and are essential to fully understanding the health benefits and cost-effectiveness associated with emerging disease-modifying therapies for SMA.

Correlates of health related quality of life in adult patients with spinal muscular atrophy

2016 ◽  
Vol 54 (5) ◽  
pp. 850-855 ◽  
Author(s):  
Esther Th Kruitwagen-Van Reenen ◽  
Renske I Wadman ◽  
Johanna Ma Visser-Meily ◽  
Leonard H. van den Berg ◽  
Carin Schröder ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Spinal Muscular Atrophy ◽  
Muscular Atrophy ◽  
Adult Patients ◽  
Health Related Quality ◽  
Related Quality ◽  
Health Related

A Prospective, Crossover Survey Study of Child- and Proxy-Reported Quality of Life According to Spinal Muscular Atrophy Type and Medical Interventions

2020 ◽  
Vol 35 (5) ◽  
pp. 322-330
Author(s):  
Meaghann S. Weaver ◽  
Rewais Hanna ◽  
Scott Hetzel ◽  
Karen Patterson ◽  
Alice Yuroff ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Spinal Muscular Atrophy ◽  
Muscular Atrophy ◽  
Survey Study ◽  
Type I ◽  
Family Impact ◽  
Proxy Report ◽  
Spinal Muscular Atrophy Type ◽  
Medical Interventions

Background: Spinal muscular atrophy is an autosomal-recessive, progressive neuromuscular disease associated with extensive morbidity. Children with spinal muscular atrophy have potentially increased life spans due to improved nutrition, respiratory support, and novel pharmaceuticals. Objectives: To report on the quality of life and family experience for children with spinal muscular atrophy with attentiveness to patient- and proxy-concordance and to stratify quality of life reports by spinal muscular atrophy type and medical interventions. Methods: A prospective, crossover survey study inclusive of 58 children (26 spinal muscular atrophy type I, 23 type II, 9 type III) and their family caregivers at a free-standing Midwestern children’s hospital. Twenty-eight families completed the 25-item PedsQL 3.0 Neuromuscular Module. Forty-four participants completed the 36-item PedsQL Family Impact Module and 47 completed the Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD) questionnaire. Results: The PedsQL Family Impact Module demonstrated significant differences between spinal muscular atrophy types I and II in functioning domains including physical, emotional, social, and family relations ( P < .03). Child self-report and proxy report surveys demonstrated significant differences between spinal muscular atrophy types in the communication domains ( P < .003). Children self-reported their quality of life higher than proxy report of child quality of life. Gastrostomy tube ( P = .001) and ventilation support ( P = .029) impacted proxy-reported quality of life perspectives, whereas nusinersen use did not. Spinal surgery was associated with improved parental quality of life and family impact ( P < .03). Conclusions: The measurement and monitoring of quality of life for children with spinal muscular atrophy and their families represents an implementable priority for care teams.

Mobility and quality of life among adults with 5q-spinal muscular atrophy: the influence of individual history

2022 ◽  
Vol 65 (2) ◽  
pp. 101552
Author(s):  
Tymothée Poitou ◽  
François Constant Boyer ◽  
Charlène Benoit ◽  
Jean-Baptiste Ferté ◽  
Charlotte Pineau ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Spinal Muscular Atrophy ◽  
Muscular Atrophy

Quality of life of patients with spinal muscular atrophy: A systematic review

2019 ◽  
Vol 23 (3) ◽  
pp. 347-356 ◽  
Author(s):  
Erik Landfeldt ◽  
Josefin Edström ◽  
Thomas Sejersen ◽  
Már Tulinius ◽  
Hanns Lochmüller ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Systematic Review ◽  
Spinal Muscular Atrophy ◽  
Muscular Atrophy
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