Long-term treatment with leuprorelin for spinal and bulbar muscular atrophy: natural history-controlled study
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 theALSFRS-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.
选择了36位肯尼迪病人接受了84个月（7年）的亮丙瑞林注射，同时选择了29个肯尼迪病人不给予亮丙瑞林的注射，以进行病情进展的对比。发现，每年都能观察到注射组比未注射组病情发展减慢。经过84个月的注射后，利用随机斜率法进行了注射组与不注射组的对比。对比采用了ALSFRS-R总分数,Limb Norris分数, 和 Norris Bulbar 分数 (p=0.005, 0.026 and 0.020）对比的方法。（我们不是专业人员，不必关心这种分数）。不仅观察到注射组病情发展的减慢，而且观察到亮丙瑞林的注射能抑制肯尼迪病人肺炎和死亡的发生率。