Long-term leuprorelin treatment may curb incidence of pneumonia, death in SBMA
Use of leuprorelin acetate for the long-term treatment of spinal and bulbar muscular atrophy (SBMA), a rare X-linked motor neuron disorder caused by trinucleotide repeat expansion in the androgen receptor gene, may delay the functional decline of SBMA patients, as well as suppress the incidence of pneumonia and death, according to a recent study.
In this present natural history-controlled study, researchers assessed the prognosis and progression of SBMA following long-term androgen suppression with leuprorelin acetate treatment. They analysed a total of 36 SBMA patients treated with leuprorelin acetate for up to 84 months (leuprorelin acetate-treated group; LT group) and 29 patients with no specific treatment (nontreated group; NT group).
Using the revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) and the modified Norris score, disease progression was evaluated by longitudinal quantitative assessment of motor functioning. There were two major clinical endpoints, namely the occurrence of pneumonia requiring hospitalization and death.
Analysis of the longitudinal disease progression using the random slope model revealed a significant difference in the ALSFRS-R total score (p=0.005), the Limb Norris Score (p=0.026) and the Norris Bulbar Score (p=0.020), with the LT group showing a slower per-12-months decline compared with the NT group.
Based on the event analysis, the LT group exhibited better prognosis than the NT group as for the event-free survival period (p=0.021).
In a 2010 study that assessed the safety and efficacy of leuprorelin, the results revealed that 48 weeks of treatment with leuprorelin was well tolerated but did not show significant effects on swallowing function in SBMA patients. Researchers said that disease duration might influence the efficacy of leuprorelin. [Lancet Neurol 2010;9:875-84]