Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy

Perry B. Shieh; Gary Elfring; Panayiota Trifillis; Claudio Santos; Stuart W. Peltz; Julie A. Parsons; Susan Apkon; Basil T. Darras; Craig Campbell; Craig M. McDonald; Richard J. Barohn; Enrico Bertini; Kate Bushby; Brigitte Chabrol; Emma Ciafaloni; Jaume Columer; Giacomi Pietro Comi; Anne Connolly; Richard S. Finkel; Kevin M. Flanigan; Nathalie Goemans; Michela Guglieri; Susan T. Iannaccone; Kristi J. Jones; Petra Kaufmann; Janbernd Kirschner; Jean K. Mah; Katherine Mathews; Eugenio Mercuri; Francesco Muntoni; Yoram Nevo; Andrés Nascimento Osorio; Yann Péréon; Rosaline Quinlivan; J. Ben Renfroe; Barry Russman; Monique Ryan; Jacinda Sampson; Ulrike Schara; Kathryn Selby; Thomas Sejersen; Douglas M. Sproule; H. Lee Sweeney; Már Tulinius; Juan J. Vilchez; Giuseppe Vita; Thomas Voit; Stephanie Burns-Wechsler; Brenda Wong; Ted Abresch; Erik K. Henricson; Kim Coleman; Michelle Eagle; Julaine Florence; Ed Gappmaier; Hoda Z. Abdel-Hamid; Richard J. Barohn; Clemens Bloetzer; Kate Bushby; Russell J. Butterfield; Brigitte Chabrol; Jong Hee Chae; Giacomi Pietro Comi; Jahannaz Dastgir; Isabelle Desguerre; Raul G. Escobar; Erika Finanger; Richard S. Finkel; Nathalie Goemans; Michela Guglieri; Peter Heydemann; Imelda Hughes; Susan T. Iannaccone; Kristi J. Jones; Anna Kaminska; Peter Karachunski; Janbernd Kirschner; Martin Kudr; Timothy Lotze; Katherine Mathews; Francesco Muntoni; Yoram Nevo; Andrés Nascimento Osorio; Yann Péréon; Alexandra Prufer De Queiroz Campos Araujo; J. Ben Renfroe; Maria Bernadete Dutra De Resende; Monique Ryan; Jacinda Sampson; Ulrike Schara; Kathryn Selby; Thomas Sejersen; H. Lee Sweeney; Gihan Tennekoon; Haluk Topaloglu; Ricardo Erazo Torricelli; Már Tulinius; Giuseppe Vita; Thomas Voit; Brenda Wong; Lindsay N. Alfano; Michelle Eagle; Meredith K. James; Linda Lowes; Anna Mayhew; Elena S. Mazzone; Leslie Nelson; Kristy J. Rose

doi:10.2217/cer-2021-0018

Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy

Perry B. Shieh, Gary Elfring, Panayiota Trifillis, Claudio Santos, Stuart W. Peltz, Julie A. Parsons, Susan Apkon, Basil T. Darras, Craig Campbell, Craig M. McDonald, Richard J. Barohn, Enrico Bertini, Kate Bushby, Brigitte Chabrol, Emma Ciafaloni, Jaume Columer, Giacomi Pietro Comi, Anne Connolly, Richard S. Finkel, Kevin M. FlaniganNathalie Goemans, Michela Guglieri, Susan T. Iannaccone, Kristi J. Jones, Petra Kaufmann, Janbernd Kirschner, Jean K. Mah, Katherine Mathews, Eugenio Mercuri, Francesco Muntoni, Yoram Nevo, Andrés Nascimento Osorio, Yann Péréon, Rosaline Quinlivan, J. Ben Renfroe, Barry Russman, Monique Ryan, Jacinda Sampson, Ulrike Schara, Kathryn Selby, Thomas Sejersen, Douglas M. Sproule, H. Lee Sweeney, Már Tulinius, Juan J. Vilchez, Giuseppe Vita, Thomas Voit, Stephanie Burns-Wechsler, Brenda Wong, Ted Abresch, Erik K. Henricson, Kim Coleman, Michelle Eagle, Julaine Florence, Ed Gappmaier, Hoda Z. Abdel-Hamid, Richard J. Barohn, Clemens Bloetzer, Kate Bushby, Russell J. Butterfield, Brigitte Chabrol, Jong Hee Chae, Giacomi Pietro Comi, Jahannaz Dastgir, Isabelle Desguerre, Raul G. Escobar, Erika Finanger, Richard S. Finkel, Nathalie Goemans, Michela Guglieri, Peter Heydemann, Imelda Hughes, Susan T. Iannaccone, Kristi J. Jones, Anna Kaminska, Peter Karachunski, Janbernd Kirschner, Martin Kudr, Timothy Lotze, Katherine Mathews, Francesco Muntoni, Yoram Nevo, Andrés Nascimento Osorio, Yann Péréon, Alexandra Prufer De Queiroz Campos Araujo, J. Ben Renfroe, Maria Bernadete Dutra De Resende, Monique Ryan, Jacinda Sampson, Ulrike Schara, Kathryn Selby, Thomas Sejersen, H. Lee Sweeney, Gihan Tennekoon, Haluk Topaloglu, Ricardo Erazo Torricelli, Már Tulinius, Giuseppe Vita, Thomas Voit, Brenda Wong, Lindsay N. Alfano, Michelle Eagle, Meredith K. James, Linda Lowes, Anna Mayhew, Elena S. Mazzone, Leslie Nelson, Kristy J. Rose

Research output: Contribution to journal › Article › peer-review

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Abstract

Aim: Compare efficacies of deflazacort and prednisone/prednisolone in providing clinically meaningful delays in loss of physical milestones in patients with nonsense mutation Duchenne muscular dystrophy. Materials & methods: Placebo data from Phase IIb (ClinicalTrials.gov Identifier: NCT00592553) and ACT DMD (ClinicalTrials.gov Identifier: NCT01826487) ataluren nonsense mutation Duchenne muscular dystrophy clinical trials were retrospectively combined in meta-analyses (intent-to-treat population; for change from baseline to week 48 in 6-min walk distance [6MWD] and timed function tests). Results: Significant improvements in change in 6-min walk distance with deflazacort versus prednisone/prednisolone (least-squares mean difference 39.54 m [95% CI: 13.799, 65.286; p = 0.0026]). Significant and clinically meaningful improvements in 4-stair climb and 4-stair descend for deflazacort versus prednisone/prednisolone. Conclusion: Deflazacort provides clinically meaningful delays in loss of physical milestones over 48 weeks compared with prednisone/prednisolone for patients with nonsense mutation Duchenne muscular dystrophy.

Original language	English
Pages (from-to)	1337-1347
Number of pages	11
Journal	Journal of Comparative Effectiveness Research
Volume	10
Issue number	18
DOIs	https://doi.org/10.2217/cer-2021-0018
State	Published - 1 Dec 2021

Bibliographical note

Publisher Copyright:
© 2021 Shieh.

Keywords

Duchenne muscular dystrophy
deflazacort
meta-analysis
nonsense mutation Duchenne muscular dystrophy
prednisolone
prednisone

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

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10.2217/cer-2021-0018

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Shieh, P. B., Elfring, G., Trifillis, P., Santos, C., Peltz, S. W., Parsons, J. A., Apkon, S., Darras, B. T., Campbell, C., McDonald, C. M., Barohn, R. J., Bertini, E., Bushby, K., Chabrol, B., Ciafaloni, E., Columer, J., Comi, G. P., Connolly, A., Finkel, R. S., ... Rose, K. J. (2021). Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy. Journal of Comparative Effectiveness Research, 10(18), 1337-1347. https://doi.org/10.2217/cer-2021-0018

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title = "Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy",

abstract = "Aim: Compare efficacies of deflazacort and prednisone/prednisolone in providing clinically meaningful delays in loss of physical milestones in patients with nonsense mutation Duchenne muscular dystrophy. Materials & methods: Placebo data from Phase IIb (ClinicalTrials.gov Identifier: NCT00592553) and ACT DMD (ClinicalTrials.gov Identifier: NCT01826487) ataluren nonsense mutation Duchenne muscular dystrophy clinical trials were retrospectively combined in meta-analyses (intent-to-treat population; for change from baseline to week 48 in 6-min walk distance [6MWD] and timed function tests). Results: Significant improvements in change in 6-min walk distance with deflazacort versus prednisone/prednisolone (least-squares mean difference 39.54 m [95% CI: 13.799, 65.286; p = 0.0026]). Significant and clinically meaningful improvements in 4-stair climb and 4-stair descend for deflazacort versus prednisone/prednisolone. Conclusion: Deflazacort provides clinically meaningful delays in loss of physical milestones over 48 weeks compared with prednisone/prednisolone for patients with nonsense mutation Duchenne muscular dystrophy.",

keywords = "Duchenne muscular dystrophy, deflazacort, meta-analysis, nonsense mutation Duchenne muscular dystrophy, prednisolone, prednisone",

author = "Shieh, {Perry B.} and Gary Elfring and Panayiota Trifillis and Claudio Santos and Peltz, {Stuart W.} and Parsons, {Julie A.} and Susan Apkon and Darras, {Basil T.} and Craig Campbell and McDonald, {Craig M.} and Barohn, {Richard J.} and Enrico Bertini and Kate Bushby and Brigitte Chabrol and Emma Ciafaloni and Jaume Columer and Comi, {Giacomi Pietro} and Anne Connolly and Finkel, {Richard S.} and Flanigan, {Kevin M.} and Nathalie Goemans and Michela Guglieri and Iannaccone, {Susan T.} and Jones, {Kristi J.} and Petra Kaufmann and Janbernd Kirschner and Mah, {Jean K.} and Katherine Mathews and Eugenio Mercuri and Francesco Muntoni and Yoram Nevo and Osorio, {Andr{\'e}s Nascimento} and Yann P{\'e}r{\'e}on and Rosaline Quinlivan and Renfroe, {J. Ben} and Barry Russman and Monique Ryan and Jacinda Sampson and Ulrike Schara and Kathryn Selby and Thomas Sejersen and Sproule, {Douglas M.} and Sweeney, {H. Lee} and M{\'a}r Tulinius and Vilchez, {Juan J.} and Giuseppe Vita and Thomas Voit and Stephanie Burns-Wechsler and Brenda Wong and Ted Abresch and Henricson, {Erik K.} and Kim Coleman and Michelle Eagle and Julaine Florence and Ed Gappmaier and Abdel-Hamid, {Hoda Z.} and Barohn, {Richard J.} and Clemens Bloetzer and Kate Bushby and Butterfield, {Russell J.} and Brigitte Chabrol and Chae, {Jong Hee} and Comi, {Giacomi Pietro} and Jahannaz Dastgir and Isabelle Desguerre and Escobar, {Raul G.} and Erika Finanger and Finkel, {Richard S.} and Nathalie Goemans and Michela Guglieri and Peter Heydemann and Imelda Hughes and Iannaccone, {Susan T.} and Jones, {Kristi J.} and Anna Kaminska and Peter Karachunski and Janbernd Kirschner and Martin Kudr and Timothy Lotze and Katherine Mathews and Francesco Muntoni and Yoram Nevo and Osorio, {Andr{\'e}s Nascimento} and Yann P{\'e}r{\'e}on and {De Queiroz Campos Araujo}, {Alexandra Prufer} and Renfroe, {J. Ben} and {Dutra De Resende}, {Maria Bernadete} and Monique Ryan and Jacinda Sampson and Ulrike Schara and Kathryn Selby and Thomas Sejersen and Sweeney, {H. Lee} and Gihan Tennekoon and Haluk Topaloglu and Torricelli, {Ricardo Erazo} and M{\'a}r Tulinius and Giuseppe Vita and Thomas Voit and Brenda Wong and Alfano, {Lindsay N.} and Michelle Eagle and James, {Meredith K.} and Linda Lowes and Anna Mayhew and Mazzone, {Elena S.} and Leslie Nelson and Rose, {Kristy J.}",

note = "Publisher Copyright: {\textcopyright} 2021 Shieh.",

year = "2021",

month = dec,

day = "1",

doi = "10.2217/cer-2021-0018",

language = "English",

volume = "10",

pages = "1337--1347",

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Shieh, PB, Elfring, G, Trifillis, P, Santos, C, Peltz, SW, Parsons, JA, Apkon, S, Darras, BT, Campbell, C, McDonald, CM, Barohn, RJ, Bertini, E, Bushby, K, Chabrol, B, Ciafaloni, E, Columer, J, Comi, GP, Connolly, A, Finkel, RS, Flanigan, KM, Goemans, N, Guglieri, M, Iannaccone, ST, Jones, KJ, Kaufmann, P, Kirschner, J, Mah, JK, Mathews, K, Mercuri, E, Muntoni, F, Nevo, Y, Osorio, AN, Péréon, Y, Quinlivan, R, Renfroe, JB, Russman, B, Ryan, M, Sampson, J, Schara, U, Selby, K, Sejersen, T, Sproule, DM, Sweeney, HL, Tulinius, M, Vilchez, JJ, Vita, G, Voit, T, Burns-Wechsler, S, Wong, B, Abresch, T, Henricson, EK, Coleman, K, Eagle, M, Florence, J, Gappmaier, E, Abdel-Hamid, HZ, Barohn, RJ, Bloetzer, C, Bushby, K, Butterfield, RJ, Chabrol, B, Chae, JH, Comi, GP, Dastgir, J, Desguerre, I, Escobar, RG, Finanger, E, Finkel, RS, Goemans, N, Guglieri, M, Heydemann, P, Hughes, I, Iannaccone, ST, Jones, KJ, Kaminska, A, Karachunski, P, Kirschner, J, Kudr, M, Lotze, T, Mathews, K, Muntoni, F, Nevo, Y, Osorio, AN, Péréon, Y, De Queiroz Campos Araujo, AP, Renfroe, JB, Dutra De Resende, MB, Ryan, M, Sampson, J, Schara, U, Selby, K, Sejersen, T, Sweeney, HL, Tennekoon, G, Topaloglu, H, Torricelli, RE, Tulinius, M, Vita, G, Voit, T, Wong, B, Alfano, LN, Eagle, M, James, MK, Lowes, L, Mayhew, A, Mazzone, ES, Nelson, L & Rose, KJ 2021, 'Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy', Journal of Comparative Effectiveness Research, vol. 10, no. 18, pp. 1337-1347. https://doi.org/10.2217/cer-2021-0018

TY - JOUR

T1 - Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy

AU - Shieh, Perry B.

AU - Elfring, Gary

AU - Trifillis, Panayiota

AU - Santos, Claudio

AU - Peltz, Stuart W.

AU - Parsons, Julie A.

AU - Apkon, Susan

AU - Darras, Basil T.

AU - Campbell, Craig

AU - McDonald, Craig M.

AU - Barohn, Richard J.

AU - Bertini, Enrico

AU - Bushby, Kate

AU - Chabrol, Brigitte

AU - Ciafaloni, Emma

AU - Columer, Jaume

AU - Comi, Giacomi Pietro

AU - Connolly, Anne

AU - Finkel, Richard S.

AU - Flanigan, Kevin M.

AU - Goemans, Nathalie

AU - Guglieri, Michela

AU - Iannaccone, Susan T.

AU - Jones, Kristi J.

AU - Kaufmann, Petra

AU - Kirschner, Janbernd

AU - Mah, Jean K.

AU - Mathews, Katherine

AU - Mercuri, Eugenio

AU - Muntoni, Francesco

AU - Nevo, Yoram

AU - Osorio, Andrés Nascimento

AU - Péréon, Yann

AU - Quinlivan, Rosaline

AU - Renfroe, J. Ben

AU - Russman, Barry

AU - Ryan, Monique

AU - Sampson, Jacinda

AU - Schara, Ulrike

AU - Selby, Kathryn

AU - Sejersen, Thomas

AU - Sproule, Douglas M.

AU - Sweeney, H. Lee

AU - Tulinius, Már

AU - Vilchez, Juan J.

AU - Vita, Giuseppe

AU - Voit, Thomas

AU - Burns-Wechsler, Stephanie

AU - Wong, Brenda

AU - Abresch, Ted

AU - Henricson, Erik K.

AU - Coleman, Kim

AU - Eagle, Michelle

AU - Florence, Julaine

AU - Gappmaier, Ed

AU - Abdel-Hamid, Hoda Z.

AU - Barohn, Richard J.

AU - Bloetzer, Clemens

AU - Bushby, Kate

AU - Butterfield, Russell J.

AU - Chabrol, Brigitte

AU - Chae, Jong Hee

AU - Comi, Giacomi Pietro

AU - Dastgir, Jahannaz

AU - Desguerre, Isabelle

AU - Escobar, Raul G.

AU - Finanger, Erika

AU - Finkel, Richard S.

AU - Goemans, Nathalie

AU - Guglieri, Michela

AU - Heydemann, Peter

AU - Hughes, Imelda

AU - Iannaccone, Susan T.

AU - Jones, Kristi J.

AU - Kaminska, Anna

AU - Karachunski, Peter

AU - Kirschner, Janbernd

AU - Kudr, Martin

AU - Lotze, Timothy

AU - Mathews, Katherine

AU - Muntoni, Francesco

AU - Nevo, Yoram

AU - Osorio, Andrés Nascimento

AU - Péréon, Yann

AU - De Queiroz Campos Araujo, Alexandra Prufer

AU - Renfroe, J. Ben

AU - Dutra De Resende, Maria Bernadete

AU - Ryan, Monique

AU - Sampson, Jacinda

AU - Schara, Ulrike

AU - Selby, Kathryn

AU - Sejersen, Thomas

AU - Sweeney, H. Lee

AU - Tennekoon, Gihan

AU - Topaloglu, Haluk

AU - Torricelli, Ricardo Erazo

AU - Tulinius, Már

AU - Vita, Giuseppe

AU - Voit, Thomas

AU - Wong, Brenda

AU - Alfano, Lindsay N.

AU - Eagle, Michelle

AU - James, Meredith K.

AU - Lowes, Linda

AU - Mayhew, Anna

AU - Mazzone, Elena S.

AU - Nelson, Leslie

AU - Rose, Kristy J.

PY - 2021/12/1

Y1 - 2021/12/1

N2 - Aim: Compare efficacies of deflazacort and prednisone/prednisolone in providing clinically meaningful delays in loss of physical milestones in patients with nonsense mutation Duchenne muscular dystrophy. Materials & methods: Placebo data from Phase IIb (ClinicalTrials.gov Identifier: NCT00592553) and ACT DMD (ClinicalTrials.gov Identifier: NCT01826487) ataluren nonsense mutation Duchenne muscular dystrophy clinical trials were retrospectively combined in meta-analyses (intent-to-treat population; for change from baseline to week 48 in 6-min walk distance [6MWD] and timed function tests). Results: Significant improvements in change in 6-min walk distance with deflazacort versus prednisone/prednisolone (least-squares mean difference 39.54 m [95% CI: 13.799, 65.286; p = 0.0026]). Significant and clinically meaningful improvements in 4-stair climb and 4-stair descend for deflazacort versus prednisone/prednisolone. Conclusion: Deflazacort provides clinically meaningful delays in loss of physical milestones over 48 weeks compared with prednisone/prednisolone for patients with nonsense mutation Duchenne muscular dystrophy.

AB - Aim: Compare efficacies of deflazacort and prednisone/prednisolone in providing clinically meaningful delays in loss of physical milestones in patients with nonsense mutation Duchenne muscular dystrophy. Materials & methods: Placebo data from Phase IIb (ClinicalTrials.gov Identifier: NCT00592553) and ACT DMD (ClinicalTrials.gov Identifier: NCT01826487) ataluren nonsense mutation Duchenne muscular dystrophy clinical trials were retrospectively combined in meta-analyses (intent-to-treat population; for change from baseline to week 48 in 6-min walk distance [6MWD] and timed function tests). Results: Significant improvements in change in 6-min walk distance with deflazacort versus prednisone/prednisolone (least-squares mean difference 39.54 m [95% CI: 13.799, 65.286; p = 0.0026]). Significant and clinically meaningful improvements in 4-stair climb and 4-stair descend for deflazacort versus prednisone/prednisolone. Conclusion: Deflazacort provides clinically meaningful delays in loss of physical milestones over 48 weeks compared with prednisone/prednisolone for patients with nonsense mutation Duchenne muscular dystrophy.

KW - Duchenne muscular dystrophy

KW - deflazacort

KW - meta-analysis

KW - nonsense mutation Duchenne muscular dystrophy

KW - prednisolone

KW - prednisone

UR - http://www.scopus.com/inward/record.url?scp=85120708840&partnerID=8YFLogxK

U2 - 10.2217/cer-2021-0018

DO - 10.2217/cer-2021-0018

M3 - Article

C2 - 34693725

AN - SCOPUS:85120708840

SN - 2042-6305

VL - 10

SP - 1337

EP - 1347

JO - Journal of Comparative Effectiveness Research

JF - Journal of Comparative Effectiveness Research

IS - 18

ER -

Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy

Abstract

Bibliographical note

Keywords

UN SDGs

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