OXANDROLONE FOR TREATMENT OF BONE MARROW APLASIA

氧雄龙治疗骨髓再生障碍

• 批准号: 7374525
• 负责人: Franklin O. Smith
• 金额: $ 1.71万
• 依托单位: CINCINNATI CHILDRENS HOSP MED CTR
• 依托单位国家: 美国
• 财政年份: 2005
• 资助国家: 美国
• 起止时间: 2005-12-01 至 2006-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7374525
• 关键词: OXANDROLONE; TREATMENT; BONE; MARROW; APLASIA

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.

该子项目是利用 NIH/NCRR 资助的中心拨款提供的资源的众多研究子项目之一。子项目和研究者 (PI) 可能已从另一个 NIH 来源获得主要资金，因此可以在其他 CRISP 条目中得到体现。列出的机构是中心的机构，不一定是研究者的机构。这项研究将首先评估氧雄龙药物在范可尼贫血（FA）患者中的安全性，其次确定该药物是否有助于治疗这些患者的骨髓衰竭。人们希望氧雄龙比羟甲龙具有更少的副作用，羟甲龙是 FA 患者骨髓衰竭短期治疗中最常用的雄激素。受试者将入组大约 18 至 30 个月（12 - 24 个月的治疗和 6 个月的额外监测）。氧雄龙起始剂量为0.04mg/kg/天。研究监测包括每周全血细胞计数、每月血清化学实验室、每季度体检（包括男性化检查和肝脏超声检查）。每半年进行一次手部X光照片以了解骨骼成熟情况，并进行行为评估以检测任何攻击性行为或情绪变化。如果治疗4个月后受试者的血细胞计数没有改善，则剂量将增加至0.08mg/kg/天，持续4个月以上。如果八个月后仍未见改善，则将停用氧雄龙。如果血细胞计数显示改善，则药物将持续至少十二个月。如果受试者的血细胞计数有反应且没有不可接受的副作用，则他们可以继续研究并接受总共 24 个月的治疗。治疗后监测包括每三个月进行一次血液检查和超声检查，以及六个月时进行手部X光检查。