Outcomes after Haploidentical Stem Cell Transplantation with Post-Transplantation Cyclophosphamide in Patients with Primary Immunodeficiency Diseases

Primary immunodeficiency diseases (PID) are a heterogeneous group of monogenic disorders (majority) affecting the immune system. To date, more than 430 different genes causing PID are identified.1 Since the first report in 1968 showing that allogeneic hematopoietic cell transplantation (HCT) could correct the immune function of a patient with severe combined immunodeficiency (SCID)2, it has become the treatment of choice for a significant number of PIDs. An HLA identical sibling is considered the ideal donor for HCT, but less than 25% of patients will have an available unaffected matched sibling donor (MSD).

Source: Biology of Blood and Marrow Transplantation - July 8, 2020 Category: Hematology Authors: Juliana Folloni Fernandes, Samantha Nichele, Leonardo Javier Arcuri, Lisandro Ribeiro, Gabriele Zamperlini Netto, Gisele Loth, Ana Luiza Melo Rodrigues, Cilmara Kuwahara, Adriana Koliski, Joanna Trennepohl, Julia Lopes Garcia, Liane Esteves Daudt, Adriana Source Type: research