OXANDROLONE FOR TREATMENT OF BONE MARROW APLASIA

这个子项目是利用由NIH/NCRR资助的中心拨款提供的资源的许多研究子项目之一。子项目和调查员(PI)可能从另一个NIH来源获得了主要资金，因此可能会出现在其他CRISE条目中。列出的机构是针对中心的，而不一定是针对调查员的机构。这项研究将主要评估药物奥曲酮在范可尼贫血(FA)患者中的安全性，并再次确定该药物是否有助于治疗这些患者的骨髓衰竭。希望氧雄酮的副作用比羟甲醇酮少，后者是在FA患者骨髓衰竭的短期治疗中最常用的雄激素。受试者将登记约18至30个月(12至24个月的治疗和6个月的额外监测)。奥曲酮起始量为0.04 mg/kg/d。研究监测包括每周完整的血细胞计数，每月的血清化学实验室，每季度的体检，包括男性化检查和肝脏超声。每半年获取一次手部X光片，以了解骨骼成熟情况，并进行行为评估，以发现任何攻击性行为或情绪变化。如果在治疗4个月后，受试者的血细胞计数没有改善，剂量将增加到每天0.08 mg/kg，持续4个月。如果在总共八个月后仍然没有改善，奥昔洛酮将停止使用。如果血细胞计数有改善，那么这种药物将持续至少12个月。如果受试者的血细胞计数有反应而没有不可接受的副作用，他们可以继续研究并接受总共24个月的治疗。治疗后监测包括每3个月一次的血常规和超声检查，每6个月做一次手部X线片。

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.