Nusinersen (Spinraza)

CADTH,

doi:10.51731/cjht.2022.496

cjht

2022

DOI: 10.51731/cjht.2022.496

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Nusinersen (Spinraza)

CADTH CADTH¹

Abstract: CADTH reimbursement reviews are comprehensive assessments of the clinical effectiveness and cost-effectiveness, as well as patient and clinician perspectives, of a drug or drug class. The assessments inform non-binding recommendations that help guide the reimbursement decisions of Canada's federal, provincial, and territorial governments, with the exception of Quebec. This review assesses nusinersen (Spinraza), 4 mg/mL solution for intrathecal injection. Indication: For the treatment o… Show more

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2023

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Cited by 2 publications

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Patient and Caregiver Outcomes After Onasemnogene Abeparvovec Treatment: Findings from the Cure SMA 2021 Membership Survey

Toro,

Yang,

Georgieva

et al. 2023

Adv Ther

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Introduction Onasemnogene abeparvovec (OA) is the only gene replacement therapy currently approved for spinal muscular atrophy (SMA) treatment. We sought to assess real-world patient and caregiver outcomes after OA treatment for SMA. Methods Patients who received OA were identified from the 2021 Cure SMA Membership Survey. Those treated at 6–23 months of age were matched to non–patients treated with OA on the basis of age at the time of survey and survival motor neuron 2 gene copy number. Patient characteristics, motor milestones, and resource and supportive care use, as well as caregiver proxy-reported health-related quality of life (HRQOL), were described. Caregiver unmet needs and HRQOL were also assessed. Results Of the 614 patients in the survey, 64 received OA, and 17 were matched with 28 non–OA-treated patients. In general, a greater percentage of OA-treated patients achieved various motor milestones, including 100% sitting without support and 58.8% walking with assistance. OA-treated patients also had numerically lower rates of hospitalization and surgery. None required tracheostomy with a ventilator. The rate of using oxygen or a breathing machine for more than 16 h was also lower for OA-treated patients. OA-treated patients had less frequent trouble swallowing. HRQOL was reported to be similar to non–OA-treated patients. Caregivers of OA-treated patients reported better patient mobility scores and less work impairment. Conclusions The study suggests that treatment with OA is associated with greater rates of motor milestone achievements and less resource and supportive care use for patients with SMA treated at 6–23 months of age in the real world. For caregivers, it may also potentially reduce unmet needs, improve HRQOL, and reduce work impairment. Supplementary Information The online version contains supplementary material available at 10.1007/s12325-023-02685-w.

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Patient and Caregiver Outcomes After Onasemnogene Abeparvovec Treatment: Findings from the Cure SMA 2021 Membership Survey

Toro,

Yang,

Georgieva

et al. 2023

Adv Ther

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Time-Efficacy in SMA Type 1 and 2 Cases Treated with Nusinersen

Axente¹,

Sporea

Mirea

et al. 2023

Balneo and PRM Research Journal

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Spinal muscular atrophy is a neuromuscular degenerative disorder characterized by progressive apoptosis of motor neurons, with severe weakness and bulbar dysfunction. The aim of the study was to analyze the correlations between the moment of initiation of treatment (nusinersen) and clinical evolution, and also the change of electrophysiological parameters and motor scales, fol-lowed up for 2 years. This study was carried out between 2018 and 2022 on 60 SMA children (29 girls, 31 boys), (29 type 1 and 31 type 2; 29 with 2 copies of SMN2, 29 with 3 copies, and 2 with 4 copies), aged between 3 weeks and 196 months, divided into 2 groups according type of SMA. For both types of SMA, statistically significant negative correlations were found between the elapsed interval from the onset of the disease to the initiation of treatment and upper motor acquisitions (type 1: p < 0.0001, r = -0.713, type 2: p<0.001, r = -0.560) and between age at the beginning of treatment and improvement in motor function (type 1: p <0.0001, r =-0.726, type 2: p<0.001, r=-0.553). For patients with type 2 SMA, a negative correlation was also identified between age at the time of onset and motor evolution (p<0.05, r = -0.378). Electrophysiological parameters were strongly positive correlated with motor improvement (p<0.0001, r=0.600). Our study established the necessity of early SMA diagnosis and therapy beginning, and demonstrated that Compound Motor Action Potential can be a predictive factor in the disease's progression. Keywords: Compound Motor Action Potential; motor scales; Spinal Muscular Atrophy

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Nusinersen (Spinraza)

Cited by 2 publications

References 23 publications

Patient and Caregiver Outcomes After Onasemnogene Abeparvovec Treatment: Findings from the Cure SMA 2021 Membership Survey

Patient and Caregiver Outcomes After Onasemnogene Abeparvovec Treatment: Findings from the Cure SMA 2021 Membership Survey

Time-Efficacy in SMA Type 1 and 2 Cases Treated with Nusinersen

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