Allogeneic Hematopoietic Transplantation for Primary Immunodeficiency Diseases

Division Of Basic Sciences - Nci

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Abstract

Background: With the availability of whole exome sequencing (WES) and whole genome sequencing (WGS) for patients with suspected immune deficiency, the number of recognized PID has increased in recent years to over 400 distinct immune defects. Allogeneic hematopoietic stem cell transplantation represents a potentially curative therapy for many hematologic diseases. Hematopoietic stem cell transplant is now an accepted standard or an appropriate experimental approach for treatment of an increasing number of PID The use of conditioning regimens containing high doses of alkylating agents, often in combination with total body irradiation, are considered myeloablative and are essential and for suppression of the host-versus-graft response to the donor stem cells (i.e. rejection) We propose to evaluate the efficacy and safety of allogeneic hematopoietic stem cell transplantation (HSCT) using selected conditioning regimens and selected donor sources in reconstituting normal hematopoiesis and immune function and reversing the disease phenotype in patients with primary immunodeficiency diseases.