Medical management of muscle weakness in Duchenne muscular dystrophy

Sarah R. Rivera; Sumit K. Jhamb; Hoda Z. Abdel-Hamid; Gyula Acsadi; John Brandsema; Emma Ciafaloni; Basil T. Darras; Susan T. Iannaccone; Chamindra G. Konersman; Nancy L. Kuntz; Craig M. McDonald; Julie A. Parsons; Carolina Tesi Rocha; Craig M. Zaidman; Russell J. Butterfield; Anne M. Connolly; Katherine D. Mathews

doi:10.1371/journal.pone.0240687

Medical management of muscle weakness in Duchenne muscular dystrophy

Sarah R. Rivera, Sumit K. Jhamb, Hoda Z. Abdel-Hamid, Gyula Acsadi, John Brandsema, Emma Ciafaloni, Basil T. Darras, Susan T. Iannaccone, Chamindra G. Konersman, Nancy L. Kuntz, Craig M. McDonald, Julie A. Parsons, Carolina Tesi Rocha, Craig M. Zaidman, Russell J. Butterfield, Anne M. Connolly, Katherine D. Mathews

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Abstract

Introduction Duchenne muscular dystrophy (DMD) is a childhood onset muscular dystrophy leading to shortened life expectancy. There are gaps in published DMD care guidelines regarding recently approved DMD medications and alternative steroid dosing regimens. Methods A list of statements about use of currently available therapies for DMD in the United States was developed based on a systematic literature review and expert panel feedback. Panelists’ responses were collected using a modified Delphi approach. Results Among corticosteroid regimens, either deflazacort or prednisone weekend dosing was preferred when payer requirements do not dictate choice. Most patients with exon 51 skip-amenable mutations should be offered eteplirsen, before or with a corticosteroid. Discussion The options available for medical management of the motor symptoms of DMD are expanding rapidly. The choice of medical therapies should balance expected benefit with side effects.

Original language	English
Article number	e0240687
Journal	PloS one
Volume	15
Issue number	10 October
DOIs	https://doi.org/10.1371/journal.pone.0240687
State	Published - Oct 2020

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Rivera, S. R., Jhamb, S. K., Abdel-Hamid, H. Z., Acsadi, G., Brandsema, J., Ciafaloni, E., Darras, B. T., Iannaccone, S. T., Konersman, C. G., Kuntz, N. L., McDonald, C. M., Parsons, J. A., Rocha, C. T., Zaidman, C. M., Butterfield, R. J., Connolly, A. M., & Mathews, K. D. (2020). Medical management of muscle weakness in Duchenne muscular dystrophy. PloS one, 15(10 October), Article e0240687. https://doi.org/10.1371/journal.pone.0240687

@article{b57a7fba61794a2098c19ea2892525aa,

title = "Medical management of muscle weakness in Duchenne muscular dystrophy",

abstract = "Introduction Duchenne muscular dystrophy (DMD) is a childhood onset muscular dystrophy leading to shortened life expectancy. There are gaps in published DMD care guidelines regarding recently approved DMD medications and alternative steroid dosing regimens. Methods A list of statements about use of currently available therapies for DMD in the United States was developed based on a systematic literature review and expert panel feedback. Panelists{\textquoteright} responses were collected using a modified Delphi approach. Results Among corticosteroid regimens, either deflazacort or prednisone weekend dosing was preferred when payer requirements do not dictate choice. Most patients with exon 51 skip-amenable mutations should be offered eteplirsen, before or with a corticosteroid. Discussion The options available for medical management of the motor symptoms of DMD are expanding rapidly. The choice of medical therapies should balance expected benefit with side effects.",

author = "Rivera, {Sarah R.} and Jhamb, {Sumit K.} and Abdel-Hamid, {Hoda Z.} and Gyula Acsadi and John Brandsema and Emma Ciafaloni and Darras, {Basil T.} and Iannaccone, {Susan T.} and Konersman, {Chamindra G.} and Kuntz, {Nancy L.} and McDonald, {Craig M.} and Parsons, {Julie A.} and Rocha, {Carolina Tesi} and Zaidman, {Craig M.} and Butterfield, {Russell J.} and Connolly, {Anne M.} and Mathews, {Katherine D.}",

note = "Publisher Copyright: Copyright: {\textcopyright} 2020 Rivera et al. This is an open access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.",

year = "2020",

month = oct,

doi = "10.1371/journal.pone.0240687",

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Rivera, SR, Jhamb, SK, Abdel-Hamid, HZ, Acsadi, G, Brandsema, J, Ciafaloni, E, Darras, BT, Iannaccone, ST, Konersman, CG, Kuntz, NL, McDonald, CM, Parsons, JA, Rocha, CT, Zaidman, CM, Butterfield, RJ, Connolly, AM & Mathews, KD 2020, 'Medical management of muscle weakness in Duchenne muscular dystrophy', PloS one, vol. 15, no. 10 October, e0240687. https://doi.org/10.1371/journal.pone.0240687

TY - JOUR

T1 - Medical management of muscle weakness in Duchenne muscular dystrophy

AU - Rivera, Sarah R.

AU - Jhamb, Sumit K.

AU - Abdel-Hamid, Hoda Z.

AU - Acsadi, Gyula

AU - Brandsema, John

AU - Ciafaloni, Emma

AU - Darras, Basil T.

AU - Iannaccone, Susan T.

AU - Konersman, Chamindra G.

AU - Kuntz, Nancy L.

AU - McDonald, Craig M.

AU - Parsons, Julie A.

AU - Rocha, Carolina Tesi

AU - Zaidman, Craig M.

AU - Butterfield, Russell J.

AU - Connolly, Anne M.

AU - Mathews, Katherine D.

N1 - Publisher Copyright: Copyright: © 2020 Rivera et al. This is an open access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.

PY - 2020/10

Y1 - 2020/10

N2 - Introduction Duchenne muscular dystrophy (DMD) is a childhood onset muscular dystrophy leading to shortened life expectancy. There are gaps in published DMD care guidelines regarding recently approved DMD medications and alternative steroid dosing regimens. Methods A list of statements about use of currently available therapies for DMD in the United States was developed based on a systematic literature review and expert panel feedback. Panelists’ responses were collected using a modified Delphi approach. Results Among corticosteroid regimens, either deflazacort or prednisone weekend dosing was preferred when payer requirements do not dictate choice. Most patients with exon 51 skip-amenable mutations should be offered eteplirsen, before or with a corticosteroid. Discussion The options available for medical management of the motor symptoms of DMD are expanding rapidly. The choice of medical therapies should balance expected benefit with side effects.

AB - Introduction Duchenne muscular dystrophy (DMD) is a childhood onset muscular dystrophy leading to shortened life expectancy. There are gaps in published DMD care guidelines regarding recently approved DMD medications and alternative steroid dosing regimens. Methods A list of statements about use of currently available therapies for DMD in the United States was developed based on a systematic literature review and expert panel feedback. Panelists’ responses were collected using a modified Delphi approach. Results Among corticosteroid regimens, either deflazacort or prednisone weekend dosing was preferred when payer requirements do not dictate choice. Most patients with exon 51 skip-amenable mutations should be offered eteplirsen, before or with a corticosteroid. Discussion The options available for medical management of the motor symptoms of DMD are expanding rapidly. The choice of medical therapies should balance expected benefit with side effects.

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VL - 15

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JF - PloS one

IS - 10 October

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Medical management of muscle weakness in Duchenne muscular dystrophy

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