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

共 24 条

[1] Quantification of missing prescriptions in commercial claims databases: results of a cohort study
Cepeda, Maria Soledad
Fife, Daniel
Denarie, Michel
Bradford, Dan
Roy, Stephanie
Yuan, Yingli
[J]. PHARMACOEPIDEMIOLOGY AND DRUG SAFETY, 2017, 26 (04) : 386 - 392
[2] Chen E, 2020, J MANAG CARE SPEC PH, V26, pS39
[3] Age related treatment effect in type II Spinal Muscular Atrophy pediatric patients treated with nusinersen
Coratti, Giorgia
Pane, Marika
Lucibello, Simona
Pera, Maria Carmela
Pasternak, Amy
Montes, Jacqueline
Sansone, Valeria A.
Duong, Tina
Young, Sally Dunaway
Messina, Sonia
D'Amico, Adele
Civitello, Matthew
Glanzman, Allan M.
Bruno, Claudio
Salmin, Francesca
Tacchetti, Paola
Carnicella, Sara
Sframeli, Maria
Antonaci, Laura
Frongia, Anna Lia
De Vivo, Darryl C.
Darras, Basil T.
Day, John
Bertini, Enrico
Muntoni, Francesco
Finkel, Richard
Mercuri, Eugenio
[J]. NEUROMUSCULAR DISORDERS, 2021, 31 (07) : 596 - 602
[4] An Integrated Safety Analysis of Infants and Children with Symptomatic Spinal Muscular Atrophy (SMA) Treated with Nusinersen in Seven Clinical Trials
Darras, Basil T.
Farrar, Michelle A.
Mercuri, Eugenio
Finker, Richard S.
Foster, Richard
Hughes, Steven G.
Bhan, Ishir
Farwell, Wildon
Gheuens, Sarah
[J]. CNS DRUGS, 2019, 33 (09) : 919 - 932
[5] Nusinersen in later-onset spinalmuscular atrophy Long-term results from the phase 1/2 studies
Darras, Basil T.
Chiriboga, Claudia A.
Iannaccone, Susan T.
Swoboda, Kathryn J.
Montes, Jacqueline
Mignon, Laurence
Xia, Shuting
Bennett, C. Frank
Bishop, Kathie M.
Shefner, Jeremy M.
Green, Allison M.
Sun, Peng
Bhan, Ishir
Gheuens, Sarah
Schneider, Eugene
Farwell, Wildon
De Vivo, Darryl C.
Castro, Diana
Cowie, Margaret
Gilbreath, Heather
Johnson, Shanda
Kauk, Melissa
Khan, Muna
McElroy, Ebra
Kneirbein, Erin Neil
Nelson, Leslie
Smith, Lauren
Trest, Stephanie
Aziz-Zaman, Sonya
Cappell, Joshua
Constantinescu, Andrei
Cruz, Rosangel
Dastgir, Jahannaz
Dunaway, Sally
Holuba, Nicole
Khandji, Alexander G.
Kramer, Samantha
Marra, Jonathan
Ortiz-Miller, Courtney
Popolizio, Molly
Salazar, Rachel
Sanabria, Luz
Sproule, Douglas M.
Weimer, Louis
Berde, Charles
Binalsheikh, Ibrahim
Graham, Robert
Kang, Peter
Koka, Anjali
Landrigan-, Mary
[J]. NEUROLOGY, 2019, 92 (21) : E2492 - E2506
[6] Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study
De Vivo, Darryl C.
Bertini, Enrico
Swoboda, Kathryn J.
Hwu, Wuh-Liang
Crawford, Thomas O.
Finkel, Richard S.
Kirschner, Janbernd
Kuntz, Nancy L.
Parsons, Julie A.
Ryan, Monique M.
Butterfield, Russell J.
Topaloglu, Haluk
Ben-Omran, Tawfeg
Sansone, Valeria A.
Jong, Yuh-Jyh
Shu, Francy
Staropoli, John F.
Kerr, Douglas
Sandrock, Alfred W.
Stebbins, Christopher
Petrillo, Marco
Braley, Gabriel
Johnson, Kristina
Foster, Richard
Gheuens, Sarah
Bhan, Ishir
Reyna, Sandra P.
Fradette, Stephanie
Farwell, Wildon
[J]. NEUROMUSCULAR DISORDERS, 2019, 29 (11) : 842 - 856
[7] Nusinersen Treatment in Adults With Spinal Muscular Atrophy
Duong, Tina
Wolford, Connie
McDermott, Michael P.
Macpherson, Chelsea E.
Pasternak, Amy
Glanzman, Allan M.
Martens, William B.
Kichula, Elizabeth
Darras, Basil T.
De Vivo, Darryl C.
Zolkipli-Cunningham, Zarazuela
Finkel, Richard S.
Zeineh, Michael
Wintermark, Max
Sampson, Jacinda
Hagerman, Katharine A.
Young, Sally Dunaway
Day, John W.
[J]. NEUROLOGY-CLINICAL PRACTICE, 2021, 11 (03) : E317 - E327
[8] Safety, Tolerability, and Effect of Nusinersen Treatment in Ambulatory Adults With 5q-SMA
Elsheikh, Bakri
Severyn, Steven
Zhao, Songzhu
Kline, David
Linsenmayer, Matthew
Kelly, Kristina
Tellez, Marco
Bartlett, Amy
Heintzman, Sarah
Reynolds, Jerry
Sterling, Gary
Weaver, Tristan
Rajneesh, Kiran
Kolb, Stephen J.
Arnold, W. David
[J]. FRONTIERS IN NEUROLOGY, 2021, 12
[9] Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy
Finkel, R. S.
Mercuri, E.
Darras, B. T.
Connolly, A. M.
Kuntz, N. L.
Kirschner, J.
Chiriboga, C. A.
Saito, K.
Servais, L.
Tizzano, E.
Topaloglu, H.
Tulinius, M.
Montes, J.
Glanzman, A. M.
Bishop, K.
Zhong, Z. J.
Gheuens, S.
Bennett, C. F.
Schneider, E.
Farwell, W.
De Vivo, D. C.
[J]. NEW ENGLAND JOURNAL OF MEDICINE, 2017, 377 (18) : 1723 - 1732
[10] 209th ENMC International Workshop: Outcome Measures and Clinical Trial Readiness in Spinal Muscular Atrophy 7-9 November 2014, Heemskerk, The Netherlands
Finkel, Richard
Bertini, Enrico
Muntoni, Francesco
Mercuri, Eugenio
[J]. NEUROMUSCULAR DISORDERS, 2015, 25 (07) : 593 - 602

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