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）的临床研究已经达到了一个新的发展里程碑。 具有讽刺意味的是，最近的成功，羟基脲和其他新的治疗方法，使临床研究的工作更加困难。 为了推动这一领域的发展，我们现在需要培养在临床研究和镰状细胞病领域表现出色的临床研究者。 Brigitta U博士穆勒是成长为这个角色的理想人选。 她是一名儿科血液学家/肿瘤学家，早期专注于国家癌症研究所（NCI）内部计划中与儿科HIV疾病相关的临床试验。 我们招募她到儿童医院，这样她就可以将注意力转移到镰状细胞病，同时扩大她在临床研究方面的培训和经验。 我们的五年发展计划的目标是让她成为一名独立的研究者-领导我们机构的镰状细胞临床研究，并指导多机构研究。Mueller博士将在Orah S博士的指导下追求她的职业发展。Platt博士是一位经验丰富的镰状细胞病研究者，Carlo Brugnara博士是镰状细胞病创新疗法设计专家。 她将集中精力在四个主要领域：1）在整个5年中，她将看到和讨论与普拉特博士的病人，并会见了博士。 2)她将进行和分析拟议的临床试验-评估Mg（一种已知的K-Cl共转运抑制剂）在Hb SC疾病患者中的作用。 我们的假设是，口服镁将阻止钾-氯共转运，防止细胞脱水，并减少聚合诱导的血管闭塞并发症。 3)她将设计和实施一个研究基础设施，将被用作未来提案的资源。 4)她将在哈佛公共卫生学院学习课程，并获得公共卫生硕士学位，专注于研究设计，实施和分析。我们提出的预防Hb SC疾病患者疼痛危象的Mg试验将作为将基础实验室设计的各种治疗方法引入临床试验的模板。