Long-Term Follow-Up of the Baltimore Experience with Hematopoietic Cell Transplantation for Severe Aplastic Anemia Using Post-Transplantation Cyclophosphamide

Severe aplastic anemia aplastic anemia: (ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most… (SAA) is a marrow failure disorder with high morbidity and mortality. It is increasingly treated with hematopoietic cell transplantation (HCT) upfront or at relapse after immunosuppressive therapy immunosuppressive therapy: Immunosuppressive drug therapy lowers your body's immune response. This prevents your immune system from attacking your bone marrow, allowing bone marrow stem cells to grow, which raises blood counts. For older patients with acquired aplastic anemia, immunosuppressive drug therapy is the… with alternative donor options. We report the outcomes of pediatric and adult recipients of HCT using reduced-intensity conditioning regimens, related HLA HLA: See human leukocyte antigen. -haploidentical or unrelated donors, and post-transplantation cyclophosphamide cyclophosphamide: Cyclophosphamide is in a class of medications called alkylating agents. When used to treat cancer, it works by slowing or stopping the growth of cancer cells in your body. When cyclophosphamide is used to treat bone marrow failure, it works by suppressing your body's immune system. -based graft-versus-host disease (GVHD) graft-versus-host disease (GVHD): Also called GVHD, it is a common complication of bone marrow/stem cell transplantation. It is caused when the donor's immune cells, now in the patient, begin to see the the patient's body as foreign and mount an immune response. GVHD most commonly effects the recipient's skin, intestines, or liver… prophylaxis. One hundred eleven transplantation-eligible patients with treatment-naïve or relapsed/refractory SAA were treated uniformly at our center in Baltimore, Maryland. These 111 patients included 65 males (59%), and the median age was 27 years (range, 3 to 71 years; interquartile range [IQR], 19 to 53 years). The median age of the predominantly haploidentical (68%) donors was 32 years (range, 13 to 56 years; IQR, 24 to 42 years). The overall median follow-up for these SAA patients was 61.3 months (95% confidence interval [CI], 47.5 to 76.6 months). Overall survival for all patients at 3 years was 92% (95% CI, 86% to 97%). Graft failure-free survival was 80% (95% CI, 73% to 88%), 79% (95% CI, 72% to 87%) at 2 years, and 79% (95% CI, 72% to 87 %) at 3 years. Rates of acute and chronic graft-versus-host disease (GVHD) were low, and the majority of patients stopped all immunosuppressive therapy for GVHD by 6 months. Surviving patients also returned to premorbid functional status, indicative of the meaningful benefit of this curative approach.