Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Phase II prospective trial to assess the rates of donor engraftment using reduced intensity conditioning (RIC) hematopoietic stem cell transplant (HSCT) and post-transplant cyclophosphamide (PTCy) for patients with primary immune deficiencies (PID), immune dysregulatory syndromes (IDS), inherited bone marrow failure syndromes (IBMFS), short telomere syndromes, Fanconi anemia, and non-Fanconi DNA double-strand break (DNA-dsb) repair disorder.
Allogeneic blood and marrow transplantation (alloBMT) is the only curative therapy for many primary immune deficiencies (PID), immune dysregulatory syndromes (IDS), and inherited bone marrow failure syndromes (IBMFS). The best reported outcomes are with human leukocyte antigen (HLA)- matched sibling donors, however, only 30% of children in need of a BMT have an HLA-matched sibling. Use of alternative donors has been limited by unacceptably high rates of transplant-related mortality (TRM), graft rejection, infection, and graft-versus-host disease (GVHD). Reduced intensity conditioning (RIC) reg…
Inclusion Criteria Cohort A: Primary Immune Deficiencies with indication for HCT: * Chronic granulomatous disease (CGD) * Wiskott-Aldrich syndrome (WAS) * Hyper-IgM syndrome * Common variable immunodeficiency (CVID) * Leukocyte adhesion deficiency-1 (LAD-1) * Severe Combined Immunodeficiency (SCID) * CTLA-4 deficiency * CARD9 deficiency * DOCK8 deficiency Immune Dysregulatory Syndromes: * Immunodysregulation polyendocrinopathy enteropathy X-linked (IPEX) syndrome * Hemophagocytic lymphohistiocytosis (HLH) or related disorder with indication for transplant * CAEBV: Patients with chronic EB…
Preparative regimen
Preparative regimen
Preparative regimen
Preparative regimen
GVHD prophylaxis
GVHD prophylaxis
GVHD prophylaxis
Arctuva estimate
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