Pretransplantation: Indications and timing

Abstract

Hematopoietic stem cell transplantation (HSCT) is now indicated in over 100 disease types and subtypes. While autologous stem cell transplantation is mainly reserved for high-risk malignancy, allogeneic stem cell transplantation can be used in both malignant and nonmalignant disorders. The most common types of malignant disorders that undergo treatment with transplantation include leukemia, lymphoma, brain tumors, and neuroblastoma. The most common types of nonmalignant diseases that are treated with transplantation include severe aplastic anemia, inherited bone marrow failure syndromes, immunodeficiencies, hemoglobinopathies, and inherited metabolic disorders. For nearly all indications, chemotherapy is given prior to the transplant either as primary treatment for the disease and/or to allow space for the new stem cells through myeloablation. Toxicity from both the chemotherapy as well as infectious risks should be balanced with the existing clinical status of the patient prior to moving ahead with any transplant in order to choose the best approach. Because the number of pediatric patients undergoing transplantation is relatively small, it is strongly recommended that when available, patients be treated in the context of a clinical trial. Recommendations on whom and when to transplant are frequently being updated, so it is important that transplant physicians and referring physicians carefully review therapeutic options for each patient.