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01-05-2019 | Leuprolide | Original Communication

Efficacy and safety of leuprorelin acetate for subjects with spinal and bulbar muscular atrophy: pooled analyses of two randomized-controlled trials

Authors: Atsushi Hashizume, Masahisa Katsuno, Keisuke Suzuki, Haruhiko Banno, Yu Takeuchi, Motoshi Kawashima, Noriaki Suga, Tomoo Mano, Amane Araki, Yasuhiro Hijikata, Akihiro Hirakawa, Gen Sobue, JASMITT study group

Published in: Journal of Neurology | Issue 5/2019

Abstract

Background

Spinal and bulbar muscular atrophy (SBMA) is an adult-onset, hereditary neuromuscular disease characterized by muscle atrophy, weakness, contraction fasciculation, and bulbar involvement. Although the causative gene, androgen receptor, has been identified, the development of novel therapeutics for SBMA is incomplete. In this study, the efficacy and safety of leuprorelin acetate administration for patients with SBMA, using the pooled data of two randomized-controlled trials, was studied.

Methods

Two randomized double-blinded studies (JASMITT-06DB and JASMITT-11DB) were done as multicentric, investigator-initiated clinical trials in Japan. In both studies, eligible patients were randomly assigned 1:1 to receive leuprorelin acetate administration once per 12 weeks for 48 weeks. The primary endpoint was the longitudinal change of pharyngeal barium residues from the baseline data measured with videofluorographic swallowing analyses. The pooled analysis plan was decided upon after the 06B study was finished and before the 11DB study began.

Results

The primary endpoint difference between the leuprorelin group and the placebo group was pharyngeal barium residue after initial swallowing, − 4.12% (95% CI, − 8.40–0.15; p = 0.058). The primary endpoint of this study does not reach significant results, although inter-group differences of pharyngeal barium residues after the initial swallowing indicated that leuprorelin acetate may be effective at each assessment point in both study groups.

Conclusions

The efficacy of leuprorelin acetate for patients with SBMA was statistically similar in two randomized-controlled trials, and suggested that leuprorelin acetate may be effective and safe. Further investigations are needed to clarify the promising efficacy of the drug.

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Title: Efficacy and safety of leuprorelin acetate for subjects with spinal and bulbar muscular atrophy: pooled analyses of two randomized-controlled trials
Authors: Atsushi Hashizume
Masahisa Katsuno
Keisuke Suzuki
Haruhiko Banno
Yu Takeuchi
Motoshi Kawashima
Noriaki Suga
Tomoo Mano
Amane Araki
Yasuhiro Hijikata
Akihiro Hirakawa
Gen Sobue
JASMITT study group
Publication date: 01-05-2019
Publisher: Springer Berlin Heidelberg
Keyword: Leuprolide
Published in: Journal of Neurology / Issue 5/2019
Print ISSN: 0340-5354
Electronic ISSN: 1432-1459
DOI: https://doi.org/10.1007/s00415-019-09251-x

Keynote webinar | Spotlight on medication adherence

Springer Medicine

Efficacy and safety of leuprorelin acetate for subjects with spinal and bulbar muscular atrophy: pooled analyses of two randomized-controlled trials

Abstract

Background

Methods

Results

Conclusions

Keynote webinar | Spotlight on medication adherence

Springer Medicine

Abstract

Background

Methods

Results

Conclusions

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