Indications for Autologous and Allogeneic Hematopoietic Cell Transplantation

Publication Date: August 7, 2015
Last Updated: March 14, 2022

Indications for HCT in pediatric patients (generally age <18 years)

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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

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

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)

Indications for hematopoietic cell transplantation in adults (generally age ≥18 years)

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Indication and Disease Status Allogeneic HCT Autologous HCT
Acute myeloid leukemia
CR1, low risk N C
CR1, intermediate risk S C
CR1, high risk S C
CR2 S C
CR3+ C C
Not in remission C N
Acute promyelocyte leukemia
CR1 N N
CR2, molecular remission C S
CR2, not in molecular remission S N
CR3+ C N
Not in remission C N
Relapse after autologous transplant C N
Acute lymphoblastic leukemia
CR1, standard risk S C
CR1, high risk S N
CR2 S C
CR3+ C N
Not in remission C N
Chronic myeloid leukemia
Chronic phase 1, TKI intolerant C N
Chronic phase 1, TKI refractory C N
Chronic phase 2+ S N
Accelerated phase S N
Blast phase S N
Myelodysplastic syndromes
Low/intermeditate-1 risk C N
Intermediate-2/high risk S N
Therapy related AML/MDS
CR1 S N
Myelofibrosis & myeloproliferative diseases
Primary, low risk C N
Primary, intermediate/high risk C N
Secondary C N
Hypereosinophilic syndromes, refractory R N
Plasma cell disorders
Myeloma, initial response D S
Myeloma, sensitive relapse C S
Myeloma, refractory C C
Plasma cell leukemia C C
Primary amyloidosis N C
POEMS syndrome N R
Relapse after autologous transplant C C
Hodgkin lymphoma
CR1 (PET negative) N N
CR1 (PET positive) N C
Primary refractory, sensitive C S
Primary refractory, resistant C N
First relapse, sensitive S S
First relapse, resistant C N
Second or greater relapse C S
Relapse after autologous transplant C N
Diffuse large B-cell lymphoma
CR1 (PET negative) N N
CR1 (PET positive) N C
Primary refractory, sensitive C S
Primary refractory, resistant C N
First relapse, sensitive C S
First relapse, resistant C N
Second or greater relapse C S
Relapse after autologous transplant C N
Follicular lymphoma
CR1 N C
Primary refractory, sensitive S S
Primary refractory, resistant S N
First relapse, sensitive S S
First relapse, resistant S N
Second or greater relapse S S
Transformation to high grade lymphoma C S
Relapse after autologous transplant C N
Mantle cell lymphoma
CR1/PR1 C S
Primary refractory, sensitive S S
Primary refractory, resistant C N
First relapse, sensitive S S
First relapse, resistant C N
Second or greater relapse C S
Relapse after autologous transplant C N
T-cell lymphoma
CR1 C C
Primary refractory, sensitive C S
Primary refractory, resistant C N
First relapse, sensitive C S
First relapse, resistant C N
Second or greater relapse C C
Relapse after autologous transplant C N
Lymphoplasmacytic lymphoma
CR1 N N
Primary refractory, sensitive N C
Primary refractory, resistant R N
First or greater relapse, sensitive R C
First or greater relapse, resistant R N
Relapse after autologous transplant C N
Burkitt's lymphoma
First remission C C
First or greater relapse, sensitive C C
First or greater relapse, resistant C N
Relapse after autologous transplant C N
Cutaneous T-cell lymphoma
Relapse C C
Relapse after autologous transplant C N
Plasmablastic lymphoma
CR1 R R
Relapse R R
Chronic lymphocytic leukemia
High risk, first or greater remission C N
T-cell prolymphocytic leukemia R R
B-cell, prolymphocytic leukemia R R
Transformation to high grade lymphoma C C
Solid tumors
Germ cell tumor, relapse N C
Germ cell tumor, refractory N C
Ewing's sarcoma, high risk N C
Breast cancer, adjuvant high risk N D
Breast cancer, metastatic D D
Renal cancer, metastatic D N
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
Sickle cell disease C N
Thalassemia D N
Hemophagocytic syndromes, refractory R N
Mast cell diseases R N
Common variable immunodeficiency R N
Wiskott-Aldrich syndrome R N
Chronic granulomatous disease R N
Multiple sclerosis N D
Systemic sclerosis N D
Rheumatoid arthritis N D
Systemic lupus erythematosus N D
Crohn's disease N D
Polymyositis-dermatomyositis N D

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

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)

Overview

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Indications for Autologous and Allogeneic Hematopoietic Cell Transplantation

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