球脊髄性筋萎縮症に対するleuprorelinの有用性と安全性


球脊髄性筋萎縮症に対するleuprorelinの有用性と安全性

 

名古屋大学祖父江元教授らの長年にわたる球脊髄性筋萎縮症に対する治療の治験の結果がLancet Neurologyという超一流誌に発表された。この病気を診療している一人として、早く、この治療薬が認可されることを切望していた。

全体としては、有意差が見られなかったが、罹病期間が10年以内の患者で、実薬投与群がplacebo投与群と比較して、最初の嚥下後のバリウム残渣が有意に減少していた。

発症10年以内という制限が設けられると、僕の患者では使用できない。この薬が早く認可されることを希望するが、さらに治験が必要であろう。

 

http://www.thelancet.com/journals/laneur/article/PIIS1474-4422(10)70182-4/fulltext

The Lancet Neurology, Volume 9, Issue 9, Pages 875 – 884, September 2010

Efficacy and safety of leuprorelin in patients with spinal and bulbar muscular atrophy (JASMITT study): a multicentre, randomised, double-blind, placebo-controlled trial

 

Masahisa Katsuno MD a c *, Haruhiko Banno MD a c *, Keisuke Suzuki MD a *, Yu Takeuchi MD a, Motoshi Kawashima MD a, Ichiro Yabe MD d, Prof Hidenao Sasaki MD d, Masashi Aoki MD e, Mitsuya Morita MD f, Prof Imaharu Nakano MD f, Kazuaki Kanai MD g, Shoichi Ito MD g, Kinya Ishikawa MD h, Prof Hidehiro Mizusawa MD h, Tomotaka Yamamoto MD i, Prof Shoji Tsuji MD i, Kazuko Hasegawa MD j, Takayoshi Shimohata MD k, Prof Masatoyo Nishizawa MD k, Hiroaki Miyajima MD l, Prof Fumio Kanda MD m, Yasuhiro Watanabe MD n, Prof Kenji Nakashima MD n, Akira Tsujino MD o, Taro Yamashita MD p, Prof Makoto Uchino MD p, Yasushi Fujimoto MD b, Fumiaki Tanaka MD a, Prof Gen Sobue MD a , for the Japan SBMA Interventional Trial for TAP-144-SR (JASMITT) study group†

 

Summary

Background

Spinal and bulbar muscular atrophy is a hereditary motor neuron disease caused by the expansion of a polyglutamine tract in the androgen receptor. At present there are no treatments for spinal and bulbar muscular atrophy, although leuprorelin suppressed the accumulation of pathogenic androgen receptors in a phase 2 trial. We aimed to assess the efficacy and safety of leuprorelin for spinal and bulbar muscular atrophy.

Methods

The Japan SBMA Interventional Trial for TAP-144-SR (JASMITT) was a 48-week, randomised, double-blind, placebo-controlled trial done at 14 hospitals between August, 2006, and March, 2008. Patients with spinal and bulbar muscular atrophy were randomly assigned (1:1) by minimisation to subcutaneous 11·25 mg leuprorelin or identical placebo every 12 weeks. Patients and investigators were masked to treatment allocation. The primary endpoint was pharyngeal barium residue, which indicates incomplete bolus clearance, measured at week 48 by videofluorography. All patients who were randomly assigned and who were assessed with videofluorography at least once were included in the analyses. This study is registered with the JMACCT clinical trials registry, number JMA-IIA00009, and the UMIN clinical trials registry, number UMIN000000465.

Findings

204 patients were randomly assigned and 199 started treatment: 100 with leuprorelin and 99 with placebo. At week 48, the pharyngeal barium residue after initial swallowing had changed by −5·1% (SD 21·0) in the leuprorelin group and by 0·2% (18·2) in the placebo group (difference between groups −5·3%; 95% CI −10·8 to 0·3; p=0·063). The mean difference in pharyngeal barium residue after piecemeal deglutition at week 48 was −3·2% (−6·4 to 0·0; p=0·049), but there was no significant difference between the groups after covariate adjustment for the baseline data (−4·1 to 1·6; p=0·392). In a predefined subgroup analysis, leuprorelin treatment was associated with a greater reduction in barium residue after initial swallowing than was placebo in patients with a disease duration less than 10 years (difference between groups −9·8, −17·1 to −2·5; p=0·009). There were no significant differences in the number of drug-related adverse events between groups (57 of 100 in the leuprorelin group and 54 of 99 in the placebo group; p=0·727).

Interpretation

48 weeks of treatment with leuprorelin did not show significant effects on swallowing function in patients with spinal and bulbar muscular atrophy, although it was well tolerated. Disease duration might influence the efficacy of leuprorelin and thus further clinical trials with sensitive outcome measures should be done in subpopulations of patients.

 

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marugametorao について

神経内科専門医 neurologist
カテゴリー: 神経学 パーマリンク

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