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

被引：9

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 [J].

Cepeda, Maria Soledad ;

Fife, Daniel ;

Denarie, Michel ;

Bradford, Dan ;

Roy, Stephanie ;

Yuan, Yingli .

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 [J].

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 .

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 [J].

Darras, Basil T. ;

Farrar, Michelle A. ;

Mercuri, Eugenio ;

Finker, Richard S. ;

Foster, Richard ;

Hughes, Steven G. ;

Bhan, Ishir ;

Farwell, Wildon ;

Gheuens, Sarah .

CNS DRUGS, 2019, 33 (09) :919-932

[5] Nusinersen in later-onset spinalmuscular atrophy Long-term results from the phase 1/2 studies [J].

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 .

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 [J].

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 .

NEUROMUSCULAR DISORDERS, 2019, 29 (11) :842-856

[7] Nusinersen Treatment in Adults With Spinal Muscular Atrophy [J].

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. .

NEUROLOGY-CLINICAL PRACTICE, 2021, 11 (03) :E317-E327

[8] Safety, Tolerability, and Effect of Nusinersen Treatment in Ambulatory Adults With 5q-SMA [J].

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 .

FRONTIERS IN NEUROLOGY, 2021, 12

[9] Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy [J].

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. .

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 [J].

Finkel, Richard ;

Bertini, Enrico ;

Muntoni, Francesco ;

Mercuri, Eugenio .

NEUROMUSCULAR DISORDERS, 2015, 25 (07) :593-602

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