Nusinersen for Spinal Muscular Atrophy in the United States: Findings From a Retrospective Claims Database Analysis

Gauthier‐Loiselle, Marjolaine; Cloutier, Martin; Toro, Walter; Patel, Anish; Shi, Sherry; Davidson, M.; Bischof, Matthias; LaMarca, Nicole; Dabbous, O.

doi:10.1007/s12325-021-01938-w

Cited by 14 publications

(33 citation statements)

References 25 publications

Supporting

Mentioning

Contrasting

Unclassified

Order By: Relevance

“…In addition, identifying the time of actual treatment initiation using claims or administrative data has challenges, especially for newly introduced medications. In a recent study of nusinersen adherence based on US claims [ 17 ], the authors reported that their study was limited to patients who completed their loading phase (≥ 4 doses). However, their analysis showed that ∼20% of patients were classified as having discontinued after the first month of treatment, which is inconsistent with the loading phase of nusinersen (requiring 2 months to complete).…”

Section: Discussionmentioning

confidence: 99%

Real-world Adherence to Nusinersen in Adults with Spinal Muscular Atrophy in the US: A Multi-site Chart Review Study

Elman

Youn

Proud

et al. 2022

Journal of Neuromuscular Diseases

View full text Add to dashboard Cite

Limited evidence exists on real-world adherence to nusinersen for the treatment of spinal muscular atrophy (SMA). Data are presented from a multi-site retrospective chart review of 86 adults with SMA initiating nusinersen at nine US centers between January 2017 and February 2019. Seventy-nine (92%) adults remained on nusinersen during the study; 454 (92%) of 493 total nusinersen doses were received on time. Fifty-eight (67%) adults received all nusinersen doses on time. The majority of patients with at least one nonadherent dose resumed nusinersen on time. Most patients followed the dosing schedule across the loading and maintenance dose periods.

show abstract

Section: Discussionmentioning

confidence: 99%

Real-world Adherence to Nusinersen in Adults with Spinal Muscular Atrophy in the US: A Multi-site Chart Review Study

Elman

Youn

Proud

et al. 2022

Journal of Neuromuscular Diseases

View full text Add to dashboard Cite

show abstract

“…*Per product label and grace period (7 days for loading doses, 28 days for maintenance doses). All patients represents the total number of patients with the corresponding follow-up time [24,25]. In this base-case persistence analysis, a decrease in persistence before 6 months (67%) and further decline at 1 (57%) and 2 years (55%) was observed.…”

Section: Discussionmentioning

confidence: 88%

“…Continued nusinersen treatment is contingent on reapproval and may represent a barrier to adherence and persistence for patients and their families. Outcome measurement at 104 weeks (2 years) aligns with the follow-up period used in the study by Gauthier-Loiselle et al [25].…”

Section: Outcome Measures and Statisticalmentioning

confidence: 81%

“…*Discontinuation definition: two missed doses and grace period (7 days for loading doses, 28 days for maintenance doses) reapproval for nusinersen coverage at that time. Gauthier-Loiselle et al reported data from all payment types, including commercial insurance plans, Medicare Part D, Medicaid, and other assistance programs, but the breakdown of insurance plans is not publicly available [25].…”

Section: Discussionmentioning

confidence: 99%

See 1 more Smart Citation

Adherence and Persistence to Nusinersen for Spinal Muscular Atrophy: A US Claims-Based Analysis

Fox¹,

To²,

Seetasith³

et al. 2022

Adv Ther

View full text Add to dashboard Cite

Introduction: 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. Methods: 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

show abstract

“…In fact, most commonly reported adverse events during treatment with nusinersen were consistent with symptoms of SMA and lumbar puncture (Stolte et al, 2021;Wataya et al, 2021). Notably, retrospective claims database analysis of more than 300 SMA patients for years 2016-2019 indicated that fewer than 50% of patients who completed the loading phase of nusinersen treatment remained in treatment 24 months after start, likely due to logistical and medical challenges of the IT administration route, which highlights the importance of a non-invasive route of administration for wide acceptance of NBTs (Gauthier-Loiselle et al, 2021).…”

Section: Clinical Experience With Rnatargeting Therapiesmentioning

confidence: 94%

Natural antisense transcripts as drug targets

Khorkova

Stahl

Joji

et al. 2022

Front. Mol. Biosci.

View full text Add to dashboard Cite

The recent discovery of vast non-coding RNA-based regulatory networks that can be easily modulated by nucleic acid-based drugs has opened numerous new therapeutic possibilities. Long non-coding RNA, and natural antisense transcripts (NATs) in particular, play a significant role in networks that involve a wide variety of disease-relevant biological mechanisms such as transcription, splicing, translation, mRNA degradation and others. Currently, significant efforts are dedicated to harnessing these newly emerging NAT-mediated biological mechanisms for therapeutic purposes. This review will highlight the recent clinical and pre-clinical developments in this field and survey the advances in nucleic acid-based drug technologies that make these developments possible.

show abstract

Nusinersen for Spinal Muscular Atrophy in the United States: Findings From a Retrospective Claims Database Analysis

Cited by 14 publications

References 25 publications

Real-world Adherence to Nusinersen in Adults with Spinal Muscular Atrophy in the US: A Multi-site Chart Review Study

Real-world Adherence to Nusinersen in Adults with Spinal Muscular Atrophy in the US: A Multi-site Chart Review Study

Adherence and Persistence to Nusinersen for Spinal Muscular Atrophy: A US Claims-Based Analysis

Natural antisense transcripts as drug targets

Contact Info

Product

Resources

About