Severe Chronic Neutropenia International Registry

描述（由申请人提供）：严重慢性中性粒细胞减少症国际登记研究（登记研究）于1994年启动，旨在研究粒细胞集落刺激因子（G-CSF）治疗先天性、周期性和特发性中性粒细胞减少症患者的长期安全性和有效性。登记研究最初评价了血液学参数、生长发育、妊娠后果和其他重要临床事件。书记官处现在是一个设在华盛顿大学的国际研究组织。在这笔赠款中，我们要求提供资金，以继续书记官处的工作。简而言之，具体目标是：（1）扩大严重慢性中性粒细胞减少症患者的纵向临床数据库，（2）扩大生物材料库，以支持遗传和分子研究，并研究有发生骨髓增生异常和白血病风险的患者的演变，（3）作为支持临床试验的资源，以改善治疗，（4）作为患者、家属、医生和其他护理人员的教育资源。（5）与研究其他造血功能衰竭疾病的研究人员合作。所有这些努力都是为了提高对严重慢性中性粒细胞减少症患者护理的理解。公共卫生相关性：登记处通过向患者提供有关SCN自然史及其治疗方案的最新信息，使患者及其家属和治疗他们的医生受益，从而改善医疗保健。它也用于进一步研究，以确定中性粒细胞减少症的原因。

DESCRIPTION (provided by applicant): The Severe Chronic Neutropenia International Registry (the Registry) opened in 1994 to study the long-term safety and efficacy of granulocyte-colony stimulating factor (G-CSF) treatment for patients with congenital, cyclic and idiopathic neutropenia. The Registry initially evaluated hematological parameters, growth and development, consequence of pregnancy, and other important clinical events. The Registry is now an international research organization based at the University of Washington. In this grant, we request funding to continue the work of the Registry. In brief, the specific aims are: (1) To expand the longitudinal clinical database on patients with severe chronic neutropenia, (2) To expand a bank of biological materials to support genetic and molecular research and study the evolution of patients at risk to develop myelodysplasia and leukemia, (3) To serve as a resource to support clinical trials to improve therapy, (4) To serve as a educational resource for patients, families, physicians and other care providers, (5) To cooperate with researchers studying the other hematopoietic failure disorders. All of these efforts are directed toward improving understanding of the care of patients with severe chronic neutropenia. PUBLIC HEALTH RELEVANCE: The Registry benefits patients, their families and the physicians who treat them by providing the most up to date information to them on the natural history of SCN and its treatment options, leading to improved medical care. It is also used to further research to determine the causes of neutropenia.