Table 2.
Indications for HCT in pediatric patients (generally age <18 years)
| Indication and Disease Status | Allogeneic HCT | Autologous HCT |
|---|---|---|
| Acute myeloid leukemia | ||
| CR1, low risk | N | N |
| CR1, intermediate risk | C | N |
| CR1, high risk | S | N |
| CR2+ | S | N |
| Not in remission | C | N |
| Acute promyelocytic leukemia, relapse | R | R |
| Acute lymphoblastic leukemia | ||
| CR1, standard risk | N | N |
| CR1, high risk | S | N |
| CR2 | S | N |
| CR3+ | C | N |
| Not in remission | C | N |
| Chronic myeloid leukemia | ||
| Chronic phase | C | N |
| Accelerated phase | C | N |
| Blast phase | C | N |
| Myelodysplastic syndromes | ||
| Low risk | C | N |
| High risk | S | N |
| Juvenile myelomonocytic leukemia | S | N |
| Therapy related | S | N |
| T-cell non-Hodgkin lymphoma | ||
| CR1, standard risk | N | N |
| CR1, high risk | S | N |
| CR2 | S | N |
| CR3+ | C | N |
| Not in remission | C | N |
| Lymphoblastic B-cell non-Hodgkin lymphoma (non-Burkitt) | ||
| CR1, standard risk | N | N |
| CR1, high risk | S | N |
| CR2 | S | N |
| CR3+ | C | N |
| Not in remission | C | N |
| Burkitt's lymphoma | ||
| First remission | C | C |
| First or greater relapse, sensitive | C | C |
| First or greater relapse, resistant | C | N |
| Hodgkin lymphoma | ||
| CR1 | N | N |
| Primary refractory, sensitive | C | C |
| Primary refractory, resistant | C | N |
| First relapse, sensitive | C | C |
| First relapse, resistant | C | N |
| Second or greater relapse | C | C |
| Anaplastic large cell lymphoma | ||
| CR1 | N | N |
| Primary refractory, sensitive | C | C |
| Primary refractory, resistant | C | N |
| First relapse, sensitive | C | C |
| First relapse, resistant | C | N |
| Second or greater relapse | C | C |
| Solid tumors | ||
| Germ cell tumor, relapse | D | C |
| Germ cell tumor, refractory | D | C |
| Ewing's sarcoma, high risk or relapse | D | S |
| Soft tissue sarcoma, high risk or relapse | D | D |
| Neuroblastoma, high risk or relapse | D | S |
| Wilm's tumor, relapse | N | C |
| Osteosarcoma, high risk | N | C |
| Medulloblastoma, high risk | N | C |
| Other malignant brain tumors | N | C |
| Non-malignant diseases | ||
| Severe aplastic anemia, new diagnosis | S | N |
| Severe aplastic anemia, relapse/refractory | S | N |
| Fanconi's anemia | R | N |
| Dyskeratosis congenita | R | N |
| Blackfan-Diamond anemia | R | N |
| Sickle cell disease | C | N |
| Thalassemia | S | N |
| Congenital amegakaryocytic thrombocytopenia | R | N |
| Severe combined immunodeficiency | R | N |
| T cell immunodeficiency, SCID variants | R | N |
| Wiskott-Aldrich syndrome | R | N |
| Hemophagocytic disorders | R | N |
| Lymphoproliferative disorders | R | N |
| Severe congenital neutropenia | R | N |
| Chronic granulomatous disease | R | N |
| Other phagocytic cell disorders | R | N |
| IPEX syndrome | R | N |
| Juvenile rheumatoid arthritis | D | R |
| Systemic sclerosis | D | R |
| Other autoimmune and immune dysregulation disorders | R | N |
| Mucopolysaccharoidoses (MPS-I and MPS-VI) | R | N |
| Other metabolic diseases | R | N |
| Osteopetrosis | R | N |
| Globoid cell leukodystrophy (Krabbe) | R | N |
| Metachromatic leukodystrophy | R | N |
| Cerebral X-linked Adrenoleukodystrophy | R | N |
Recommendation categories (see text for definition): Standard of care (S); Standard of care, clinical evidence available (C); Standard of care, rare indication (R); Developmental (D); Not generally recommended (N)
Rather than provide a long and evolving list of unique rare diseases, the indications table shows a concise categorical list together with selected unique diagnoses for which transplant is most frequently offered