Adherence and Persistence to Nusinersen for Spinal Muscular Atrophy: A US Claims-Based Analysis
Year: 2022
Type: article
Study design: Observational Study
Abstract: Spinal muscular atrophy (SMA) is a genetic, neuromuscular disease caused by deletions and/or mutations in the survival of motor neuron 1 (SMN1) gene leading to reduced SMN protein levels. Nusinersen, an intrathecally administered antisense oligonucleotide therapy that increases SMN protein levels, is approved for use in adult and pediatric patients with SMA. Data to inform real-world patient adherence and persistence to nusinersen are limited, with disparities in the population with SMA, study design, and results. The objective of this study is to characterize real-world nusinersen adherence and persistence in patients with SMA. This retrospective study examined nusinersen adherence and persistence over a 2-year period in patients with SMA in the USA from the IQVIA PharMetrics Plus claims database. Patients were followed from the date of first evidence of nusinersen treatment (occurring after 1 July 2017) until the end of the study period (31 December 2019) or end of continuous pharmacy and medical benefit enrollment, whichever came first. Subgroup analyses for nusinersen adherence and persistence were performed on the basis of age and presence or absence of spinal complications. The final cohort consisted of 179 patients with SMA treated with nusinersen. Adherence to nusinersen treatment was 41% at 56 weeks and 39% at 104 weeks. In the base-case persistence analysis, there was a decrease in persistence before 6 months (67%) and further decline at 1 (57%) and 2 years (55%). Patients with spinal complication versus without had numerically higher persistence with nusinersen. The findings suggest that adherence and persistence to nusinersen treatment appear low. Demographic (age ≥ 18 years) and clinical factors (no spinal complications) may contribute to nusinersen treatment discontinuation. Future research should explore possible reasons for low adherence and persistence to nusinersen treatment, such as clinical or logistical factors, patient preferences, and payer restrictions. Spinal muscular atrophy (SMA) is a rare, genetic disease that causes patients to lose motor neurons over time. This makes tasks that involve movement control like walking and talking more difficult. SMA can be treated, but it is important that patients receive their scheduled doses of medicine as prescribed and stay on treatment. Nusinersen (SPINRAZA®) is a treatment for SMA that is given as an intrathecal injection into the cerebrospinal fluid of the spine. Patients receive six doses of nusinersen in the first year. After the first year, patients receive three doses every year for life. This study looked at whether patients received their scheduled doses, also called adherence, and how many patients remained on treatment, or persistence, over two years. This study involved 179 nusinersen-treated patients with SMA and used data from US health insurance plans. After 56 weeks of treatment, 41% of patients were adherent. After 104 weeks, 39% were adherent. After 6 months, 67% of patients were still on treatment. After 1 year, 57% were still on treatment. After 2 years, 55% of patients were still on treatment. The study showed that a low number of patients with SMA, particularly those older than 18 years with no spinal problems, remained on nusinersen for the intended time and received the treatment as prescribed. Future studies will look at possible reasons for low adherence and persistence to nusinersen, which may include difficulties traveling to a clinic or scheduling a visit, patient preference, or insurance restrictions.
Source: Advances in Therapy
Language: English
Subfield: Genetics
Field: Medicine
Domain: Health Sciences
Open Access status: hybrid
Estimated APC paid: $4,190
Funders: F. Hoffmann-La Roche, Genentech
OpenAlex ID: W4311930024