Long-term effects of intravenous pamidronate in fibrous dysplasia of bone
Long-term effects of intravenous pamidronate in fibrous dysplasia of bone
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DOI：
10.1359/jbmr.1997.12.10.1746
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发表时间：
1997-10-01
影响因子：
6.2
通讯作者：
Meunier, PJ
中科院分区：
文献类型：
--
作者：
Chapurlat, RD;Delmas, PD;Meunier, PJ
骨纤维发育不良（Fibrous dysplasia of bone，FD）是一种罕见的疾病，其特征在于骨髓中的纤维组织增生，导致溶骨性病变，其引起骨痛和骨折。迄今为止，唯一的治疗是骨科，FD和佩吉特骨疾病之间的组织学和生物化学相似性与骨吸收增加有关，这使得我们建议用双膦酸盐帕米膦酸盐治疗。本研究的目的是评估静脉注射帕米膦酸二钠治疗FD的长期疗效。（男性11名，女性9名;平均年龄31岁）接受每6个月静脉注射180 mg帕米膦酸盐的疗程（60 mg/天，3天内输注），平均随访时间为39个月（范围18-64），骨痛的严重程度，每位患者疼痛骨骼部位的数量，所有受累区域的X线检查，血清碱性磷酸酶，每6个月评估一次空腹尿羟脯氨酸和尿I型胶原C端肽，骨痛的严重程度和疼痛部位的数量似乎显着减少，骨重建的所有生化标志物均显着降低，我们观察到放射学反应9名溶骨病变再填充的患者，1例经骨活检证实为矿化缺损，4例患者持续骨应力线，但无骨折发生。我们认为静脉注射帕米膦酸盐可以减轻骨痛，降低生化指标评估的骨转换率，并改善FD的放射学病变。观察到很少的副作用。
Fibrous dysplasia of bone (FD) is a rare disorder characterized by proliferation of fibrous tissue in bone marrow leading to osteolytic lesions, It causes bone pain and fractures, To date the only treatment is orthopedic, Histological and biochemical similarities between FD and Paget's bone disease related to increased osteoclastic resorption led us to propose treatment with the bisphosphonate pamidronate. The aim of the study,vas to assess the long-term effects of intravenous pamidronate in FD, In this open label phase III study, 20 patients with FD (11 males and 9 females; mean age 31 years) received courses of 180 mg of intravenous pamidronate every 6 months (60 mg/day during 3 days by infusion), The mean duration of follow-up was 39 months (range 18-64), Severity of bone pain, number of painful skeletal sites per patient, X-rays of all involved areas, serum alkaline phosphatase, fasting urinary hydroxyproline, and urinary type I collagen C-telopeptide were assessed every 6 months, The severity of bone pain and the number of painful sites appeared to be significantly reduced, All biochemical markers of bone remodeling were substantially lowered, We observed a radiographic response in nine patients with refilling of osteolytic lesions, A mineralization defect proven by bone biopsy was observed in one case, Four patients sustained bone stress lines, but no fracture occurred. We suggest that intravenous pamidronate alleviates bone pain, reduces the rate of bone turnover assessed by biochemical markers, and improves radiological lesions of FD. Few side effects were observed.