Mallette L E
Division of Endocrinology and Metabolism, Baylor College of Medicine, Houston, Tex.
Arch Intern Med. 1989 Dec;149(12):2765-7.
Several agents are available for treatment of Paget's disease of bone, but their long-term use may be limited by resistance or adverse effects. I have treated two patients with exceptionally severe polyostotic Paget's disease (serum alkaline phosphatase values 25- to 30-fold above normal), in whom salmon calcitonin, sodium etidronate, and plicamycin each had become ineffective or could not be used. Pamidronate (3-amino-1-hydroxypropylidene-1,1-bisphosphonate) decreased serum alkaline phosphatase to normal or near-normal values and almost completely relieved symptoms, without recognizable adverse effects, except for transient mild hypocalcemia. The symptomatic and biochemical responses were maintained through 2 years of treatment and for more than 6 months after the treatment was discontinued. Thus, pamidronate can be very effective in severe, resistant cases of Paget's disease. Further study of this potent agent is needed to define the optimum regimen for maximum effectiveness and minimal long-term toxicity.
有几种药物可用于治疗骨Paget病，但其长期使用可能会受到耐药性或不良反应的限制。我治疗了两名患有极其严重的多骨型Paget病的患者（血清碱性磷酸酶值比正常水平高25至30倍），在他们身上，鲑鱼降钙素、依替膦酸钠和普卡霉素均已失效或无法使用。帕米膦酸盐（3-氨基-1-羟基亚丙基-1,1-双膦酸盐）可使血清碱性磷酸酶降至正常或接近正常水平，并几乎完全缓解症状，除了短暂的轻度低钙血症外，没有明显的不良反应。在2年的治疗期间以及停药后6个多月，症状和生化反应均得以维持。因此，帕米膦酸盐在严重的、耐药的Paget病病例中可能非常有效。需要对这种强效药物进行进一步研究，以确定能实现最大疗效和最小长期毒性的最佳治疗方案。
