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)

Recommendation Grading

Overview

Title

Indications for Autologous and Allogeneic Hematopoietic Cell Transplantation

Authoring Organization

Publication Month/Year

August 7, 2015

Last Updated Month/Year

January 10, 2024

Document Type

Guideline

External Publication Status

Published

Country of Publication

US

Inclusion Criteria

Female, Male, Adolescent, Adult, Child, Infant, Older adult

Health Care Settings

Ambulatory, Hospital, Operating and recovery room, Outpatient

Intended Users

Clinical researcher, nurse, nurse practitioner, physician, physician assistant

Scope

Management, Treatment

Diseases/Conditions (MeSH)

D018380 - Hematopoietic Stem Cell Transplantation, D015470 - Leukemia, Myeloid, Acute, D054198 - Precursor Cell Lymphoblastic Leukemia-Lymphoma

Keywords

clinical trials, Hematopoietic cell transplantation, Autologous transplantation, Allogeneic transplantation

Source Citation

Majhail, N. S., Farnia, S. H., Carpenter, P. A., Champlin, R. E., Crawford, S., Marks, D. I., Omel, J. L., Orchard, P. J., Palmer, J., Saber, W., Savani, B. N., Veys, P. A., Bredeson, C. N., Giralt, S. A., & LeMaistre, C. F. (2015). Indications for Autologous and Allogeneic Hematopoietic Cell Transplantation: Guidelines from the American Society for Blood and Marrow Transplantation. Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 21(11), 1863–1869. https://doi.org/10.1016/j.bbmt.2015.07.032

Supplemental Methodology Resources

Data Supplement