Zatz M, Rapaport D, Vainzof M, Pavanello R de C, Rocha J M, Betti R T, Otto P A
Departamento de Biologia, Universidade de São Paulo, Brazil.
Am J Med Genet. 1988 Dec;31(4):821-33. doi: 10.1002/ajmg.1320310415.
Human growth hormone (HGH) inhibition may be beneficial in Duchenne muscular dystrophy (DMD) and slow down the rate of progression of the disease. The purposes of the present investigation were: 1) to assess, through pharmacological stimuli (L-dopa test), the HGH response in untreated DMD patients, and 2) to evaluate the inhibitory effect of mazindol on HGH levels as a potential treatment for DMD. In 55 DMD patients, HGH levels were measured through the L-dopa test, and 40 received mazindol. After 1 year, there was wide variability in the individual response to mazindol. An apparent diminution in the mean HGH level was observed in the whole group of patients; this was statistically significant after 3 and 6 months but not after 9 and 12 months of treatment. The results suggest that this drug is not effective for arresting growth or inhibiting HGH secretion for a prolonged period of time.
抑制人体生长激素(HGH)可能对杜氏肌营养不良症(DMD)有益,并减缓疾病的进展速度。本研究的目的是:1)通过药理学刺激(左旋多巴试验)评估未经治疗的DMD患者的HGH反应,以及2)评估马吲哚对HGH水平的抑制作用,作为DMD的一种潜在治疗方法。在55名DMD患者中,通过左旋多巴试验测量HGH水平,40名患者接受了马吲哚治疗。1年后,个体对马吲哚的反应存在很大差异。在整个患者组中观察到平均HGH水平明显下降;在治疗3个月和6个月后具有统计学意义,但在治疗9个月和12个月后无统计学意义。结果表明,这种药物在长期抑制生长或抑制HGH分泌方面无效。