Towards graft- versus-host disease-free alternative donor transplant platforms for patients with acquired aplastic anemia

Hematopoietic stem cell transplantation is a well-established treatment option for acquired 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… . Historically, upfront hematopoietic stem cell transplantation (HCT) with HLA HLA: See human leukocyte antigen. -matched sibling donors is used in young patients and 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… (IST) is used for all others. Over time, innovations in the transplant platform have decreased unwanted complications such as graft versus host disease (GVHD), failure to engraft, and infections thereby expanding the use of alternative donors. This has led to an evolution towards HCT and away from IST despite increased intensity of treatment due to improved event free survival. The ideal conditioning regimen for HCT in aplastic anemia results in sustained engraftment engraftment: Refers to how well a graft (donor cells) is accepted by the host (the patient) after a bone marrow or stem cell transplant. Several factors contribute to better engraftment - physical condition of the patient, how severe the disease is, type of donor available, age of patient. Successful… , minimal toxicity, lack of GVHD and is not limited by donor availability. Two transplant platforms have been refined to meet these needs. TCR αβ+ T cell T cell: see lymphocyte depletion has excellent outcomes with minimal graft-rejection and GVHD in the matched and mismatched unrelated donor unrelated donor: A donor that is not a sibling or other familial relation of the patient (recipient). setting. Haploidentical grafts however still require further optimization. Bone marrow Bone marrow: The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow. grafts given with ATG and post-transplant 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. have similarly excellent results with extremely low rates of GVHD. Peripheral mobilized grafts need further optimization in this setting. This review provides an overview of the current perspective on regimens that minimize GVHD in aplastic anemia and how they can be further refined so that all patients with aplastic anemia have curative therapy available to them.