OXANDROLONE FOR TREATMENT OF BONE MARROW APLASIA

氧雄龙治疗骨髓再生障碍

• 批准号: 7607749
• 负责人: Franklin O. Smith
• 金额: $ 1.17万
• 依托单位: CINCINNATI CHILDRENS HOSP MED CTR
• 依托单位国家: 美国
• 财政年份: 2007
• 资助国家: 美国
• 起止时间: 2007-02-15 至 2007-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7607749
• 关键词: Adverse effects; Aggressive behavior; Androgens; Behavior assessment; Blood; Bone Marrow; Chemistry; Complete Blood Count; Computer Retrieval of Information on Scientific Projects Database; Count; Dose; Enrollment; Fanconi' s Anemia; Funding; Grant; Hand; Institution; Liver; Monitor; Moods; Oxandrolone; Oxymetholone; Pancytopenia; Patients; Pharmaceutical Preparations; Physical Examination; Research; Research Personnel; Resources; Safety; Serum; Source; Ultrasonography; United States National Institutes of Health; Virilism; Work; bone; day; research study; response

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.

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