[Hematopoietic cell transplantation indications for genetic disorders]

Allogeneic hematopoietic cell transplantation (HCT) is a curative therapy for various genetic disorders, including inherited bone marrow failure syndromes (IBMFS), primary immunodeficiencies (PID), and inherited metabolic diseases (IMD). However, the therapeutic strategies and clinical challenges for these conditions differ significantly from those for malignant diseases, influencing the development of disease-specific transplantation strategies. For IBMFS, HCT indications are determined by the severity classification of acquired aplastic anemia and evidence of malignant progression in patients presenting with pancytopenia, and by disease severity, treatment dependency, and the clinical impact of symptoms in those presenting with single-lineage cytopenia. While allogeneic HCT is curative for many PIDs, the indications vary by disease. For IMDs, although allogeneic HCT is indicated as a curative treatment for some diseases, the indications differ by disease type, and patients with advanced disease are often ineligible.