University of Verona, Rheumatology Unit, Valeggio, Verona, Italy.
J Bone Miner Res. 2010 Jun;25(6):1460-2. doi: 10.1359/jbmr.091113.
Fibrodysplasia ossificans progressiva (FOP) is a rare genetic condition characterized by progressive heterotopic ossification, increasing disability, and cumulative immobility. Thiazolidinediones, introduced in 1999 for the treatment of diabetes, enhance bone marrow adipogenesis at the expense of new bone formation, and this might be exploited for the treatment of FOP. A 48-year-old woman with severe FOP characterized by continuous flares that she was partially controlling only with high prednisone doses was given rosiglitazone (initially 4 mg and then 8 mg daily) for 14 months. No new flare-ups were observed during rosiglitazone therapy as compared to the five episodes observed during the previous year while on 20 to 25 mg prednisone daily. The steroid dose could be lowered progressively to 5 mg/day, the skin became softer, and the articular mobility improved impressively. This case report seems to suggest that rosiglitazone therapy, possibly in association with small doses of prednisone, is associated with important clinical improvements in patients with FOP.
进行性骨化性纤维发育不良（FOP）是一种罕见的遗传性疾病，其特征为进行性异位骨化、残疾程度增加和逐渐丧失活动能力。噻唑烷二酮类药物于 1999 年被引入，用于治疗糖尿病，但其会牺牲新骨形成来促进骨髓脂肪生成，这可能被用于治疗 FOP。一位 48 岁的女性患有严重的 FOP，其特征为持续发作，仅靠高剂量泼尼松才能部分控制，她接受了罗格列酮（最初每天 4 毫克，然后每天 8 毫克）治疗 14 个月。与前一年每天服用 20 至 25 毫克泼尼松时观察到的五次发作相比，在罗格列酮治疗期间未观察到新的发作。泼尼松剂量可逐渐降至 5 毫克/天，皮肤变软，关节活动度显著改善。这个病例报告似乎表明，罗格列酮治疗可能与小剂量泼尼松联合使用，可使 FOP 患者获得显著的临床改善。
