TRANS.00029 Hematopoietic Stem Cell Transplantation for Genetic Diseases and Aplastic Anemias

This policy addresses hematopoietic stem cell transplantation (HSCT), specifically allogeneic (ablative and non‑myeloablative) HSCT for genetic diseases and aplastic anemias. Allogeneic HSCT is medically necessary for acquired aplastic anemia and select heritable bone marrow failure syndromes (congenital amegakaryocytic thrombocytopenia, Diamond‑Blackfan anemia, dyskeratosis congenita, Fanconi anemia, Schwachman‑Diamond syndrome) and for paroxysmal nocturnal hemoglobinuria; for sickle cell disease and thalassemia, coverage applies only when disease‑specific criteria are met. Autologous HSCT, allogeneic HSCT for genetic diseases or acquired anemias not listed (e.g., cystic fibrosis), planned tandem HSCT, and second/repeat allogeneic HSCT for persistent/progressive/relapsed disease are considered investigational and not medically necessary.

"Allogeneic (ablative and non-myeloablative) hematopoietic stem cell transplantationis consideredmedically necessaryfor individuals with the following disorders. In addition, individuals with Sickle..."