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

被引：8

作者：

Elman, Lauren ^{[1
]}

Youn, Bora ^{[2
]}

Proud, Crystal M. ^{[3
]}

Frey, Margaret R. ^{[4
]}

Ajroud-Driss, Senda ^{[5
]}

McCormick, M. Eileen ^{[6
]}

Michelson, David ^{[7
]}

Cartwright, Michael S. ^{[8
]}

Heiman-Patterson, Terry ^{[9
]}

Choi, Joseph M. ^{[10
]}

Chandak, Aastha ^{[11
]}

Khachatryan, Artak ^{[12
]}

Martinez, Marta ^{[12
]}

Paradis, Angela D. ^{[2
]}

机构：

[1] Univ Penn, Philadelphia, PA 19104 USA

[2] Biogen, Cambridge, MA USA

[3] Childrens Hosp Kings Daughters, Norfolk, VA USA

[4] Mem Healthcare Inst Neurosci, Owosso, MI USA

[5] Northwestern Univ, Feinberg Sch Med, Chicago, IL 60611 USA

[6] Oakland Univ, William Beaumont Sch Med, Royal Oak, MI USA

[7] Loma Linda Univ Hlth, Loma Linda, CA USA

[8] Wake Forest Sch Med, Winston Salem, NC 27101 USA

[9] Temple Univ, Lewis Katz Sch Med, Philadelphia, PA 19122 USA

[10] Georgetown Univ Hosp, Washington, DC 20007 USA

[11] Certara, New York, NY USA

[12] Certara, London, England

来源：

JOURNAL OF NEUROMUSCULAR DISEASES | 2022年 / 9卷 / 05期

关键词：

Medication adherence; spinal muscular atrophy; antisense oligonucleotides; neuromuscular diseases; retrospective studies; adults; SHAM CONTROL;

D O I：

10.3233/JND-210768

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

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.

引用

页码：655 / 660

页数：6

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