Naveh Y, Alon U, Kaftori J K, Berant M
Pediatrics. 1985 Feb;75(2):321-3.
Progressive diaphyseal dysplasia is characterized clinically by crippling leg pain, fatigue, headache, poor appetite, muscle weakness, and waddling gait. Twelve affected patients, aged 2 years 4 months to 40 years, were treated with intermittent courses of low doses of prednisone given in a single dose on alternate mornings for periods ranging from 6 months to 10 years. The average initial dose of prednisone was 0.6 mg/kg/d, and average maintenance dose was 0.3 mg/kg/d. Relief of all crippling symptoms was achieved in all patients. No untoward serious side effects have been observed, and the growth of children was not slowed. However, corticosteroid therapy should be restricted to patients suffering from crippling pain. The mechanism through which steroids act remains undefined.
进行性骨干发育异常的临床特征为严重的腿痛、疲劳、头痛、食欲不振、肌肉无力和蹒跚步态。12例患者年龄在2岁4个月至40岁之间，接受了间歇性低剂量泼尼松治疗，隔日早晨单次给药，疗程为6个月至10年。泼尼松的平均初始剂量为0.6mg/kg/d，平均维持剂量为0.3mg/kg/d。所有患者的严重症状均得到缓解。未观察到严重的不良反应，儿童生长也未减缓。然而，皮质类固醇治疗应仅限于有严重疼痛的患者。类固醇的作用机制尚不清楚。
