帕米膦酸二钠治疗畸形性骨炎的前瞻性观察

目的　了解帕米膦酸二钠对畸形性骨炎的治疗效果。方法　畸形性骨炎患者 5例 ,男性 2例 ,女性 3例 ,年龄 2 7～ 74岁 ,病程 4～ 4 8年。均为多骨性损害 ,骨痛分级 2例为 4级 ,一直卧床 ;3例为 3级 ,翻身和行走困难。每次静脉滴注帕米膦酸二钠 30～ 6 0mg ,2～ 3周内应用 90～ 2 70mg。治疗前、疗程结束时和治疗后 4、12、2 4、4 8周 ,对患者进行骨痛分级和骨转换生化指标血碱性磷酸酶(ALP)、Ⅰ型胶原羧基末端前肽 (PICP)、Ⅰ型胶原交联羧基末端肽 (ICTP)、2 4h尿羟脯氨酸 (HOP)的检测 ,并观察药物不良反应。结果　疗程结束时 5例患者骨痛均明显减轻 ,骨痛分级 2例从 4级降到 2级 ,3例从 3级降到 1级 ;治疗后 12周骨痛均完全消失 ,活动功能明显改善 ,能行走 ;治疗后 4 8周仍无骨痛。疗程结束时ALP、PICP、ICTP和HOP水平即明显下降 ;治疗后 12、2 4周各指标平均下降率均≥5 0 % ;治疗后 4 8周各指标分别从治疗前 398u/L(中位数 )、818ng/L(中位数 )、(2 9± 14 )ng/L和 (71±16 )mg/ 2 4h尿 ,降到 15 9u/L、12 9ng/L、(12± 4 )ng/L和 (34± 7)mg/ 2 4h尿 ,差异均有显著意义 (均P<0 0 5 )。仅出现低热、皮疹、瘙痒和一过性血天冬氨酸氨基转移酶稍升高等轻微不良反应。结论　静脉滴注帕米膦酸二钠治疗畸形

Prospective study of pamidronate disodium in treatment of Paget's disease of bone

OBJECTIVE: To determine the efficacy of pamidronate disodium (pamidronate) in treatment of Paget's disease of bone. METHODS: Intravenous drip of pamidronate was given at the dose of 30 - 60 mg per day for 2 - 3 weeks with a total dosage of 90 - 270 (168 +/- 84) mg to 5 patients with Paget's disease of bone, 2 males and 3 females, aged 27 - 74 (61 on average) with the course of disease of 4 - 48 years (24 years on average), all of them having multiple bone lesions, 2 being in grade 4 of pain assessment and bedridden and 3 being in grade 3 with impaired mobility. Pain assessment, biochemical markers of bone turnover, including serum alkaline phosphatase (ALP), carboxy-terminal propeptide of type I collagen (PICP), carboxy-terminal cross-linked telopeptide of type I collagen (ICTP) and urinary hydroxyproline (HOP), and side effects of pamidronate were observed before treatment, by the end of treatment, and 4, 12, 24, and 48 weeks after treatment. RESULTS: The Pagetic bone pain was significantly reduced by the end of treatment in all patients. The degree of pain assessment declined from grade 4 to grade 2 in 2 patients, and from grade 3 to grade 1 in 3 patients. The mobility was markedly improved without bone pain 12 weeks after treatment and relief of pain lasted for 1 year in all patients. Serum ALP, PICP, ICTP and urinary HOP concentrations were significant decrease by the end of treatment. Moreover, the mean percentage decreases for above biochemical markers at 12 and 24 weeks after treatment were 50% or over. The values of serum ALP, PICP, ICTP and urinary HOP decreased from 398 u/L (median), 818 ng/L (median), (29 +/- 14) ng/L and (71 +/- 16) mg/24 h urine respectively to 159 u/L, 129 ng/L, (12 +/- 4) ng/L and (34 +/- 7) mg/24 h urine respectively 48 weeks after treatment (all P < 0.05). Side effects, including a transient increase in body temperature, skin eruption and pruritus, and a transient increase of serum aspartate aminotransferase concentration, were negligible. CONCLUSION: Intravenous infusion of pamidronate is effective on Paget's disease of bone. Pamidronate of the dose of 90 - 270 mg administered within 2 - 3 weeks produces a year-long remission. Side effects of pamidronate are slight and transient.