Matched and Haploidentical transplantation for Adults with Sickle Cell Anemia

镰状细胞性贫血成人匹配和单倍体移植

• 批准号: 7856761
• 负责人: NEAL FLOMENBERG
• 金额: $ 99.29万
• 依托单位: THOMAS JEFFERSON UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2009
• 资助国家: 美国
• 起止时间: 2009-09-30 至 2011-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7856761
• 关键词: Address; Adult; Adverse effects; Affect; African American; Aggressive Clinical Course; Allogenic; Blood; Caring; Cell Count; Characteristics; Chimerism; Clinical; Communities; Counseling; Diarrhea; Disease; Donor person; Economics; Ethnic group; Evaluation; Event; Exanthema; Fever; Financial Support; Genetic; Grant; Health; Hematological Disease; Hematopoietic Stem Cell Transplantation; Homologous Transplantation; Immune; Infertility; Infusion procedures; Institution; International; Intervention; Laboratories; Left; Life; Lymphocyte; Marrow; Medical; Medical Care Team; Methods; Modeling; Monitor; Morbidity - disease rate; Patient Participation; Patients; Perception; Phenotype; Population; Preventive; Process; Protocols documentation; Publishing; Registries; Research; Risk; Screening procedure; Siblings; Sickle Cell; Sickle Cell Anemia; Sickle Hemoglobin; Social support; Source; Stem cells; Surveys; Syphilis; Technology; Testing; Therapeutic; Time; Toxic effect; Transplantation; Trust; Underrepresented Minority; abstracting; base; cell type; chronic graft versus host disease; clinical application; follow-up; improved; innovation; leukemia/lymphoma; mortality; patient population; pre-clinical; programs; racial and ethnic; reconstitution; research study; social; socioeconomics; tool; young adult

DESCRIPTION (provided by applicant): Abstract: Sickle Cell Anemia (SCA) has been successfully treated by allogeneic transplantation of normal blood forming stem cells to replace the patient's own marrow. Obstacles to the broader application of this treatment have included problems in finding an appropriately matched donor for the transplant and transplant related toxicity, as well as a number of socioeconomic obstacles. Reduced intensity approaches to allogeneic hematopoietic stem cell transplantation (HSCT) have been recognized as a tool for reducing morbidity and mortality to safe and acceptable levels. However, application of reduced intensity approaches is still limited by the fact that well less than half of appropriate patients have an approriately matched sibling donor. Haploidentical HSCT, if more consistently successful, would make the largest impact on transplant availability for all racial and ethnic groups. We have developed a safe and effective transplant approach for both matched sibling and haploidentical related transplants which addresses the problem of donor availability in patients with leukemia, lymphoma, and related disorders. This approach has been particularly useful for African American patients with these disorders who have not had available matched sibling donors. African Americans constitute the majority of patients with SCA at our institution and worldwide. We propose to now test this innovative approach to HSCT in patients with SCA as a means of making transplant available to the vast majority of otherwise appropriate transplant candidate patients with this illness. Beyond donor availability, socioeconomic barriers also limit access of SCA patients to transplantation. We will address known issues and patient needs and identify new ones through use of a preventative health based approach using decision based counseling sessions to identify barriers to care. We will endeavor to provide a seamless approach to care with few disruptions or changes from what is already a comfortable and trusted care team by coordinating the efforts of the Sickle Cell Program and the transplant team. We will provide a patient navigator to assist the patients during the peri-transplant period. Throughout the process, the Sickle Cell Program and BMT team will participate together in an iterative process of evaluation and reassessment. The significant reduction of both donor availability issues and socioeconomic barriers to transplantation for SCA should help to address a substantial national and international health issue for severely affected patients with this illness. We believe we have the appropriate patient population, technology, and health care team in place to substantially improve these issues and help this population of patients. (End of Abstract)

描述（由申请人提供）： 摘要：镰状细胞贫血（SCA）已通过正常血液形成干细胞的同种异体移植成功治疗，以取代患者自己的骨髓。更广泛应用这种处理的障碍包括在寻找适当匹配的移植和移植相关毒性的捐助者方面的问题，以及许多社会经济障碍。 降低的同种异体造血干细胞移植（HSCT）的强度方法已被认为是将发病率和死亡率降低到安全可接受的水平的工具。但是，降低强度方法的应用仍然受到以下事实的限制：不到一半的适当患者具有适当匹配的兄弟姐妹供体。单倍型HSCT，即使更始终如一的成功，将对所有种族和族裔群体的移植供应产生最大的影响。 我们已经为匹配的同胞和单倍性相关移植提供了一种安全有效的移植方法，该方法解决了白血病，淋巴瘤和相关疾病患者的供体可用性问题。这种方法对于这些尚未有匹配兄弟姐妹捐助者的非裔美国人患者特别有用。非裔美国人在我们机构和全球范围内构成大多数SCA患者。我们现在建议在SCA患者中测试这种创新的HSCT方法，以此作为使大多数原本适当的移植候选患者可用于移植的手段。 除了捐助者的可用性之外，社会经济障碍还限制了SCA患者进入移植。我们将通过使用基于决策的咨询会议来识别护理障碍，通过使用基于预防健康的方法来解决已知问题和患者需求。我们将努力通过协调Sickle Cell计划和移植团队的努力来提供一种无缝护理的方法，或者几乎没有舒适和值得信赖的护理团队的变化。我们将在移植期期间提供患者导航器来协助患者。在整个过程中，镰状细胞计划和BMT团队将共同参与评估和重新评估的迭代过程。 SCA移植的捐助者可用性问题和社会经济障碍的大幅度降低应有助于解决严重影响患有这种疾病的患者的实质性国家和国际健康问题。我们认为，我们拥有适当的患者人群，技术和医疗团队，可以实质上改善这些问题并帮助这些患者。 （抽象的结尾）