A phase II trial of a reduced intensity conditioned (RIC) allogeneic hematopoietic cell transplant (HCT) with post-transplant cyclophosphamide (PTCy) for idiopathic severe aplastic anemia (SAA), paroxysmal nocturnal hemoglobinuria (PNH), acquired pure red cell aplasia (aPRCA), or acquired amegakaryocytic thrombocytopenia (aAT) utilizing population pharmacokinetic (popPK)-guided individual dosing of pre-transplant conditioning and differential dosing of low dose total body irradiation based on age, presence of myelodysplasia and/or clonal hematopoiesis.
Platelets <20,000/uL or transfusion dependent
Absolute neutrophil count <500/uL without hematopoietic growth factor support
Absolute reticulocyte count <60,000/uL AND bone marrow cellularity <50% (with < 30% residual hematopoietic cells)
Early myelodysplastic features (bone marrow (BM) blasts <5%), without history of MDS/AML pre-treatment.
Pregnant, breastfeeding or intending to become pregnant during the study. Persons of childbearing potential must have a negative pregnancy test (serum or urine) within 7 days of the start of treatment
Uncontrolled infection
Evidence of moderate or severe portal fibrosis or cirrhosis on biopsy
Known allergy to any of the study components
Masonic Cancer Center, University of Minnesota
Lead sponsor