Matched and Haploidentical transplantation for Adults with Sickle Cell Anemia

Thomas Jefferson University, Philadelphia PA

Investigators

Abstract

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)