OXANDROLONE FOR TREATMENT OF BONE MARROW APLASIA

该子项目是利用NIH/NCRR资助的中心赠款提供的资源的许多研究子项目之一。子项目和研究者（PI）可能从另一个NIH来源获得了主要资金，因此可以在其他CRISP条目中表示。所列机构为中心机构，不一定是研究者机构。这项研究将主要评估药物氧雄龙在范可尼贫血（FA）患者中的安全性，其次确定这种药物是否有助于治疗这些患者的骨髓衰竭。人们希望氧甲氢龙的副作用比氧甲氢龙更少，氧甲氢龙是FA患者骨髓衰竭短期治疗中最常使用的雄激素。受试者将入组约18 - 30个月（12 - 24个月治疗和6个月额外监测）。氧雄龙的起始剂量为0.04mg/kg/天。研究监测包括每周一次的全血细胞计数、每月一次的血清化学实验室检查、每季度一次的体格检查，包括男性化检查和肝脏超声检查。每半年进行一次手部X光片检查，以了解骨成熟情况，并进行行为评估，以检测任何攻击性行为或情绪变化。如果在治疗4个月后未观察到受试者的血细胞计数改善，则剂量将增加至0.08 mg/kg/天，再持续4个月。如果没有改善后，注意到总共八个月，氧雄龙将停止。如果血细胞计数显示改善，则药物将持续至少12个月。如果受试者的血细胞计数出现应答且无不可接受的副作用，则受试者可继续参加研究并接受总计24个月的治疗。治疗后监测包括每三个月进行一次血液检查和超声检查，以及六个月时的手部X光片。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. This research study will primarily evaluate the safety of the drug oxandrolone in patients with Fanconi anemia (FA), and secondarily determine if this drug can help in the treatment of bone marrow failure in these patients. It is hoped that oxandrolone will have less side effects than oxymetholone, the androgen used most frequently in the short-term treatment of bone marrow failure in FA patients. Subjects will be enrolled for approximately 18 to 30 months (12 - 24 months of treatment and 6 months additional monitoring). The oxandrolone starting dose is 0.04mg/kg/day. Study monitoring includes weekly complete blood counts, monthly serum chemistry labs, quarterly physical examinations including virilization exams and liver ultrasounds. Semi-annually, hand radiographs are obtained for bone maturation and behavioral assessments are conducted to detect any aggressive behavior or mood changes. If no improvement n the subject's blood counts are noted after 4 months of therapy, the dose will be increased to 0.08mg/kg/day for a period of 4 more months. If no improvement is noted after a total of eight months, oxandrolone will be discontinued. If the blood counts show improvement, then the drug will continue for a minimum of twelve months. Subjects may remain on study and receive a total of 24 months of therapy if they have a response in their blood counts without unacceptable side effects. Post-treatment monitoring includes blood work and ultrasound every three months, and hand radiograph at six months.