Severe Chronic Neutropenia International Registry

描述(申请人提供)：严重慢性中性粒细胞减少症国际注册中心(注册中心)成立于1994年，目的是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。该注册中心最初评估了血液学参数、生长发育、妊娠后果和其他重要的临床事件。登记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，我们请求提供资金以继续书记官处的工作。简而言之，其具体目标是：(1)扩大关于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，并研究有可能发展为骨髓发育不良和白血病的患者的演变，(3)作为支持临床试验的资源，以改进治疗，(4)作为患者、家庭、医生和其他护理提供者的教育资源，(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。

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.