CLINICAL RESEARCH IN PEDIATRIC SICKLE CELL DISEASE

儿科镰状细胞病的临床研究

• 批准号: 6798682
• 负责人: BRIGITTA U MUELLER
• 金额: $ 12.57万
• 依托单位: BAYLOR COLLEGE OF MEDICINE
• 依托单位国家: 美国
• 财政年份: 2000
• 资助国家: 美国
• 起止时间: 2000-09-01 至 2006-02-28
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6798682
• 关键词: analgesics; blood chemistry; child (0-11); clinical research; clinical trials; data collection methodology /evaluation; genotype; human subject; human therapy evaluation; magnesium; medical complication; pediatric pharmacology; pediatrics; sickle cell anemia; sickle cell crisis; sickling inhibitor; statistics /biometry; urinalysis

Clinical research in cell disease (SCD) has reached a new developmental milestone. Ironically, the recent success of hydroxyurea and other new treatments makes the work of clinical investigation harder. In order to move the field forward, we now need to develop clinical investigators who excel in the arenas of clinical investigation and sickle cell disease. Dr. Brigitta U. Mueller is an ideal person to grow into this role. She is a pediatric hematologist/oncologist who early on focused on clinical trials related to pediatric HIV disease at the National Cancer Institute's (NCI) intramural program. We have recruited her to Children's Hospital so that she can shift her attention to sickle cell disease while expanding her training and experience in clinical investigation. The goal of our five-year development plan is for her to become an independent investigator - lead sickle cell clinical research at our institution, and direct multi-institutional studies. Dr. Mueller will pursue her career development under the mentorship of Dr. Orah S. Platt, an experienced investigator in sickle cell disease, and Dr. Carlo Brugnara, an expert in design of innovative therapies in sickle cell disease. She will focus her efforts in four major areas: 1) Throughout the 5 years she will see and discuss patients with Dr. Platt, and meet with Dr. Brugnara to discuss research ideas and review data. 2) She will conduct and analyze the proposed clinical trial - evaluating the effect of Mg, a known inhibitor of K-Cl cotransport, in patients with Hb SC disease. Our hypothesis is that oral Mg will block K- Cl cotransport, prevent cell dehydration, and reduce polymerization-induced vasoocclusive complications. 3) She will design and implement a research infrastructure that will be used as a resource for future proposals. 4) She will do course work at the Harvard School of Public Health and obtain the MPH degree, concentrating on research design, implementation; and analysis. The trial of Mg for the prevention of pain crises in patients with Hb SC disease that we propose, will serve as a template for bringing a variety of treatments designed in basic laboratories to clinical trial.

细胞疾病（SCD）的临床研究已达到一个新的发展里程碑。 具有讽刺意味的是，羟基脲和其他新疗法的最新成功使临床研究的工作更加严重。 为了向前发展，我们现在需要开发在临床研究和镰状细胞疾病领域表现出色的临床研究人员。 Brigitta U. Mueller博士是成长为这一角色的理想人选。 她是一名儿科血液学家/肿瘤学家，早期，她在国家癌症研究所（NCI）的壁内计划上重点关注与儿科HIV疾病有关的临床试验。 我们已将她招募到儿童医院，以便她可以将注意力转移到镰状细胞疾病上，同时扩大她在临床研究中的培训和经验。 我们五年发展计划的目的是让她成为我们机构的独立研究者 - 镰状细胞临床研究，并直接进行多机构研究。 Mueller博士将在镰状细胞病经验丰富的研究人员Orah S. Platt博士和Carlo Brugnara博士的指导下追求自己的职业发展，他是镰状细胞疾病创新疗法设计专家Carlo Brugnara。 她将把自己的努力集中在四个主要领域：1）在整个5年中，她将与Platt博士见面并讨论患者，并与Brugnara博士会面，讨论研究思想和审查数据。 2）她将进行和分析提出的临床试验 - 评估Mg（K -CL共同竞争的已知抑制剂）对HB SC疾病患者的影响。 我们的假设是口服MG将阻止K-CL共转移，防止细胞脱水，并减少聚合引起的血管隔离并发症。 3）她将设计和实施研究基础设施，该研究基础设施将被用作未来建议的资源。 4）她将在哈佛大学公共卫生学院进行课程工作，并获得MPH学位，专注于研究设计，实施；和分析。我们提出的HB SC疾病患者中MG预防疼痛危机的试验将作为将基本实验室中设计的各种治疗方法带入临床试验的模板。