Efficacy of prednisone for the treatment of ocular myasthenia (EPITOME)

A randomized, controlled trial

MUSCLE STUDY GROUP (MSG)

Research output: Contribution to journalArticle

38 Citations (Scopus)

Abstract

Introduction: In this study we evaluated the safety, tolerability, and efficacy of prednisone in patients with ocular myasthenia gravis (OMG) concurrently treated with pyridostigmine. Methods: This investigation was a randomized, double-blind, placebo-controlled trial. Participants whose symptoms failed to remit on pyridostigmine were randomized to receive placebo or prednisone, initiated at 10 mg every other day, and titrated to a maximum of 40 mg/day over 16 weeks. The primary outcome measure was treatment failure. Results: Fewer subjects were randomized than the 88 planned. Of the 11 randomized, 9 completed 16 weeks of double-blind therapy. Treatment failure incidence was 100% (95% CI 48%-100%) in the placebo group (n=5) vs. 17% (95% CI 0%-64%) in the prednisone group, P = 0.02 (n=6). Median time to sustained minimal manifestation status (MMS) was 14 weeks, requiring an average prednisone dose of 15 mg/day. Adverse events were infrequent and generally mild in both groups. Conclusions: A strategy of low-dose prednisone with gradual escalation appears to be safe, well-tolerated, and effective in treating OMG.

Original languageEnglish (US)
Pages (from-to)363-369
Number of pages7
JournalMuscle and Nerve
Volume53
Issue number3
DOIs
StatePublished - Mar 1 2016

Fingerprint

Prednisone
Randomized Controlled Trials
Pyridostigmine Bromide
Myasthenia Gravis
Placebos
Treatment Failure
Outcome Assessment (Health Care)
Safety
Incidence
Therapeutics

Keywords

  • Clinical trial
  • Neuromuscular
  • Ocular myasthenia
  • Prednisone
  • Steroids

ASJC Scopus subject areas

  • Clinical Neurology
  • Cellular and Molecular Neuroscience
  • Physiology (medical)
  • Physiology

Cite this

Efficacy of prednisone for the treatment of ocular myasthenia (EPITOME) : A randomized, controlled trial. / MUSCLE STUDY GROUP (MSG).

In: Muscle and Nerve, Vol. 53, No. 3, 01.03.2016, p. 363-369.

Research output: Contribution to journalArticle

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abstract = "Introduction: In this study we evaluated the safety, tolerability, and efficacy of prednisone in patients with ocular myasthenia gravis (OMG) concurrently treated with pyridostigmine. Methods: This investigation was a randomized, double-blind, placebo-controlled trial. Participants whose symptoms failed to remit on pyridostigmine were randomized to receive placebo or prednisone, initiated at 10 mg every other day, and titrated to a maximum of 40 mg/day over 16 weeks. The primary outcome measure was treatment failure. Results: Fewer subjects were randomized than the 88 planned. Of the 11 randomized, 9 completed 16 weeks of double-blind therapy. Treatment failure incidence was 100{\%} (95{\%} CI 48{\%}-100{\%}) in the placebo group (n=5) vs. 17{\%} (95{\%} CI 0{\%}-64{\%}) in the prednisone group, P = 0.02 (n=6). Median time to sustained minimal manifestation status (MMS) was 14 weeks, requiring an average prednisone dose of 15 mg/day. Adverse events were infrequent and generally mild in both groups. Conclusions: A strategy of low-dose prednisone with gradual escalation appears to be safe, well-tolerated, and effective in treating OMG.",
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author = "{MUSCLE STUDY GROUP (MSG)} and Benatar, {Michael G} and Mcdermott, {Michael P.} and Sanders, {Donald B.} and Wolfe, {Gil I.} and Barohn, {Richard J.} and Nowak, {Richard J.} and Michael Hehir and Vern Juel and Hans Katzberg and Rabi Tawil and Alexandre Waltz and Michon, {Sara Claude} and Alexis Smirnow and Farheen Hussain and Smith, {Patricia C.} and Nuran Dilek and McDermott, {Michael P.} and Joanne Janciuras and Cornelia Kamp and Tim Hackett and Pat Bolger and Joan Woodcook and McVey, {April L.} and Dimachkie, {Mazen M.} and Mamatha Pasnoor and Whittaker, {Thomas J.} and Laura Herbelin and Gabrielle Rico and Waqar Waheed and Shannon Lucy and Dicapua, {Daniel B.} and Goldstein, {Jonathan M.} and Benison Keung and Joan Nye and Vera Bril and Tapia, {Carolina Barnett} and Ari Breiner and Seint Kokokyi and Ted Burns and Guillermo Solorzano and Amruta Joshi and Silvestri, {Nicholas J.} and Kara Patrick and Srikanth Muppidi and Sharon Nations and Steve Hopkins and Juel, {Vern C.} and Guptill, {Jeffrey T.} and Hobson, {Lisa D.} and Massey, {Janice M.}",
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AU - Sanders, Donald B.

AU - Wolfe, Gil I.

AU - Barohn, Richard J.

AU - Nowak, Richard J.

AU - Hehir, Michael

AU - Juel, Vern

AU - Katzberg, Hans

AU - Tawil, Rabi

AU - Waltz, Alexandre

AU - Michon, Sara Claude

AU - Smirnow, Alexis

AU - Hussain, Farheen

AU - Smith, Patricia C.

AU - Dilek, Nuran

AU - McDermott, Michael P.

AU - Janciuras, Joanne

AU - Kamp, Cornelia

AU - Hackett, Tim

AU - Bolger, Pat

AU - Woodcook, Joan

AU - McVey, April L.

AU - Dimachkie, Mazen M.

AU - Pasnoor, Mamatha

AU - Whittaker, Thomas J.

AU - Herbelin, Laura

AU - Rico, Gabrielle

AU - Waheed, Waqar

AU - Lucy, Shannon

AU - Dicapua, Daniel B.

AU - Goldstein, Jonathan M.

AU - Keung, Benison

AU - Nye, Joan

AU - Bril, Vera

AU - Tapia, Carolina Barnett

AU - Breiner, Ari

AU - Kokokyi, Seint

AU - Burns, Ted

AU - Solorzano, Guillermo

AU - Joshi, Amruta

AU - Silvestri, Nicholas J.

AU - Patrick, Kara

AU - Muppidi, Srikanth

AU - Nations, Sharon

AU - Hopkins, Steve

AU - Juel, Vern C.

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N2 - Introduction: In this study we evaluated the safety, tolerability, and efficacy of prednisone in patients with ocular myasthenia gravis (OMG) concurrently treated with pyridostigmine. Methods: This investigation was a randomized, double-blind, placebo-controlled trial. Participants whose symptoms failed to remit on pyridostigmine were randomized to receive placebo or prednisone, initiated at 10 mg every other day, and titrated to a maximum of 40 mg/day over 16 weeks. The primary outcome measure was treatment failure. Results: Fewer subjects were randomized than the 88 planned. Of the 11 randomized, 9 completed 16 weeks of double-blind therapy. Treatment failure incidence was 100% (95% CI 48%-100%) in the placebo group (n=5) vs. 17% (95% CI 0%-64%) in the prednisone group, P = 0.02 (n=6). Median time to sustained minimal manifestation status (MMS) was 14 weeks, requiring an average prednisone dose of 15 mg/day. Adverse events were infrequent and generally mild in both groups. Conclusions: A strategy of low-dose prednisone with gradual escalation appears to be safe, well-tolerated, and effective in treating OMG.

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KW - Neuromuscular

KW - Ocular myasthenia

KW - Prednisone

KW - Steroids

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