Long-term treatment with leuprorelin for spinal and bulbar muscular atrophy: natural history-controlled study.

Abstract

OBJECTIVE:

To evaluate the prognosis and progression of spinal and bulbar muscular atrophy (SBMA), a rare X-linked motor neuron disorder caused by trinucleotide repeat expansion in the AR (androgen receptor) gene, after long-term androgen suppression with leuprorelin acetate treatment.

METHODS:

In the present natural history-controlled study, 36 patients with SBMA treated with leuprorelin acetate for up to 84 months (leuprorelin acetate-treated group; LT group) and 29 patients with SBMA with no specific treatment (non-treated group; NT group) were analysed. Disease progression was evaluated by longitudinal quantitative assessment of motor functioning using the revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R), and the modified Norris score. In addition, we selected two major clinical endpoint events, namely the occurrence of pneumonia requiring hospitalisation and death, to evaluate disease prognosis following long-term leuprorelin acetate treatment.

RESULTS:

In our analysis of the longitudinal disease progression using the random slope model, we observed a significant difference in the ALSFRS-R total score, the Limb Norris Score, and the Norris Bulbar Score (p=0.005, 0.026 and 0.020, respectively), with the LT group exhibiting a slower per-12-months decline compared with the NT group. As for the event analysis, the prognosis of the LT group was better in comparison to the NT group as for the event-free survival period (p=0.021).

CONCLUSION:

Long-term treatment with leuprorelin acetate appears to delay the functional decline and suppress the incidence of pneumonia and death in subjects with SBMA.

• Authors: Atsushi Hashizume, Masahisa Katsuno, Keisuke Suzuki, Akihiro Hirakawa, Yasuhiro Hijikata, Shinichiro Yamada, Tomonori Inagaki, Haruhiko Banno, Gen Sobue
• Journal: Journal of neurology, neurosurgery, and psychiatry (J Neurol Neurosurg Psychiatry) Vol. 88 Issue 12 Pg. 1026-1032 (12 2017) ISSN: 1468-330X [Electronic] England
• PMID: 28780536 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't)
• Copyright: © Article author(s) (or their employer(s) unless otherwise stated in the text of the article) 2017. All rights reserved. No commercial use is permitted unless otherwise expressly granted.

Chemical References

• Antineoplastic Agents, Hormonal
• Leuprolide

Topics

• Adult
• Aged
• Antineoplastic Agents, Hormonal (adverse effects, therapeutic use)
• Disease Progression
• Disease-Free Survival
• Endpoint Determination
• Female
• Humans
• Kaplan-Meier Estimate
• Leuprolide (adverse effects, therapeutic use)
• Long-Term Care
• Male
• Middle Aged
• Muscular Atrophy, Spinal (complications, drug therapy, genetics)
• Pneumonia (complications, prevention & control)
• Prognosis