Table 1. Allogeneic HCT for nonmalignant... | American Society of Hematology

Table 1.

Allogeneic HCT for nonmalignant diseases: clinical considerations

Disease categories	Specific examples	NMD HCT: clinical considerations	NMD HCT: goals to improve outcomes
Bone marrow failure syndromes	Severe aplastic anemia Fanconi anemia Congenital amegakaryocytic thrombocytopenia Dyskeratosis congenita Diamond-Blackfan anemia Shwachman-Diamond syndrome Congenital sideroblastic anemia GATA2-associated marrow failure SAMD9 or SMDL9 disorder Paroxysmal nocturnal hemoglobinuria	1) Increased risk of graft failure 2) No benefit of any GVHD as no role of GVL 3) Full donor chimerism not necessary for long-term disease control 4) Increased risk for prolonged IMCs 5) Increased risk of atypical infections in certain PIDs	1) Limit graft failure 2) Limit GVHD 3) Limit IMCs
Hemoglobinopathies	Sickle cell disease Thalassemia
Primary immunodeficiency diseases	SCID Non-SCID combined immunodeficiencies T-cell immunodeficiency, SCID variants Wiskott-Aldrich syndrome Hemophagocytic disorders Severe congenital neutropenia Chronic granulomatous disease Other phagocytic cell disorders X-linked hyper-IgM syndrome IPEX syndrome IFN-γ deficiency
Inherited metabolic disorders	Mucopolysaccharidoses I (severe; Hurler syndrome) Other mucopolysaccharidoses (II, III, VI) Other lysosomal metabolic diseases Globoid cell leukodystrophy Metachromatic leukodystrophy Cerebral X-linked adrenoleukodystrophy
Other disorders	Juvenile rheumatoid arthritis Osteopetrosis Systemic sclerosis

Disease categories	Specific examples	NMD HCT: clinical considerations	NMD HCT: goals to improve outcomes
Bone marrow failure syndromes	Severe aplastic anemia Fanconi anemia Congenital amegakaryocytic thrombocytopenia Dyskeratosis congenita Diamond-Blackfan anemia Shwachman-Diamond syndrome Congenital sideroblastic anemia GATA2-associated marrow failure SAMD9 or SMDL9 disorder Paroxysmal nocturnal hemoglobinuria	1) Increased risk of graft failure 2) No benefit of any GVHD as no role of GVL 3) Full donor chimerism not necessary for long-term disease control 4) Increased risk for prolonged IMCs 5) Increased risk of atypical infections in certain PIDs	1) Limit graft failure 2) Limit GVHD 3) Limit IMCs
Hemoglobinopathies	Sickle cell disease Thalassemia
Primary immunodeficiency diseases	SCID Non-SCID combined immunodeficiencies T-cell immunodeficiency, SCID variants Wiskott-Aldrich syndrome Hemophagocytic disorders Severe congenital neutropenia Chronic granulomatous disease Other phagocytic cell disorders X-linked hyper-IgM syndrome IPEX syndrome IFN-γ deficiency
Inherited metabolic disorders	Mucopolysaccharidoses I (severe; Hurler syndrome) Other mucopolysaccharidoses (II, III, VI) Other lysosomal metabolic diseases Globoid cell leukodystrophy Metachromatic leukodystrophy Cerebral X-linked adrenoleukodystrophy
Other disorders	Juvenile rheumatoid arthritis Osteopetrosis Systemic sclerosis

IFN, interferon; Ig, immunoglobulin; IPEX, immunodysregulation polyendocrinopathy enteropathy X-lined; PID, primary immunodeficiency diseases.