What is treated with a transplant
Every condition here can be treated, in at least some people, with a bone marrow or blood stem cell transplant. For each one: what it is, what causes it, what it does to a person, how it is treated, and what people go through.
Each page also says plainly what part a donor actually plays — including the conditions where the answer is none, because the transplant uses the person’s own cells. That is true far more often than most people expect, and we would rather say so than let you assume otherwise.
94 conditions
Told one of these? It covers more than one condition
These are names people are given that doctors treat as several different diseases — and the answer about whether a donor is needed is not the same for all of them.
Leukemias
Cancers that begin in the blood-forming cells of the bone marrow, crowding out the healthy blood a body needs.
Acute myeloid leukemiaFull guide
AML · acute myeloid leukemia · acute myelogenous leukemia
A stranger can be the matchAcute promyelocytic leukemia with PML::RARA fusionFull guide
APL · AML-M3 · PML-RARA AML
Transplant is not the usual treatmentAdult T-cell leukemia/lymphomaFull guide
ATLL · ATL · adult T-cell leukemia/lymphoma
A stranger can be the matchB-cell acute lymphoblastic leukemia (B-ALL)Full guide
B-ALL/LBL NOS · B-ALL NOS · B-LBL NOS
A stranger can be the matchChronic lymphocytic leukemia/small lymphocytic lymphomaFull guide
CLL/SLL · CLL · SLL
Transplant is not the usual treatmentChronic myeloid leukemiaFull guide
CML · Ph+ CML · BCR::ABL1-positive CML
Transplant is not the usual treatmentMixed-phenotype acute leukemia, B/myeloid
MPAL B/myeloid · biphenotypic acute leukemia
A stranger can be the matchPhiladelphia-positive B-cell acute lymphoblastic leukemia
Ph+ B-ALL · BCR-ABL1 B-ALL · Ph-positive ALL
A stranger can be the matchSézary syndromeFull guide
SS · leukemic cutaneous T-cell lymphoma · cutaneous T-cell lymphoma
A stranger can be the matchT-cell acute lymphoblastic leukemia (T-ALL)Full guide
T-ALL/LBL NOS · T-ALL · T-LBL
A stranger can be the matchT-cell prolymphocytic leukemia
T-PLL · T-cell PLL
A stranger can be the match
Lymphomas
Cancers of the lymphatic system — the network of vessels and nodes that carries immune cells around the body.
ALK-negative anaplastic large cell lymphomaFull guide
ALK-negative ALCL · ALK− ALCL · systemic ALCL, ALK negative
Usually the person’s own cellsALK-positive anaplastic large cell lymphomaFull guide
ALK+ ALCL · ALCL, ALK-positive
Usually the person’s own cellsAngioimmunoblastic T-cell lymphomaFull guide
nTFHL-AI · nodal TFH lymphoma, angioimmunoblastic type · AITL
Usually the person’s own cellsBurkitt lymphomaFull guide
BL · Burkitt leukemia · L3 ALL (historical)
Transplant is not the usual treatmentClassic Hodgkin lymphomaFull guide
cHL · classic HL · Hodgkin disease (historical)
Usually the person’s own cellsDiffuse large B-cell lymphoma, not otherwise specifiedFull guide
DLBCL NOS · DLBCL · non-Hodgkin lymphoma
Gene therapy is now an option tooExtranodal NK/T-cell lymphoma, nasal typeFull guide
ENKTL · NKTCL · extranodal NK/TCL
A stranger can be the matchFollicular lymphomaFull guide
FL · follicle-centre lymphoma (older umbrella term) · non-Hodgkin lymphoma
Gene therapy is now an option tooHepatosplenic T-cell lymphomaFull guide
HSTCL · HSTL · hepatosplenic γδ T-cell lymphoma
A stranger can be the matchMantle cell lymphomaFull guide
MCL · mantle-zone lymphoma (historical) · non-Hodgkin lymphoma
Gene therapy is now an option tooMycosis fungoidesFull guide
MF · cutaneous T-cell lymphoma, mycosis-fungoides type · cutaneous T-cell lymphoma
Transplant is not the usual treatmentPeripheral T-cell lymphoma, not otherwise specifiedFull guide
PTCL-NOS · PTCL NOS · non-Hodgkin lymphoma
Usually the person’s own cellsPrimary CNS lymphomaFull guide
IP-LBCL · CNS IP-LBCL · PCNSL
Usually the person’s own cellsPrimary mediastinal large B-cell lymphomaFull guide
PMBCL · PMLBCL
Gene therapy is now an option tooWaldenström macroglobulinemiaFull guide
LPL · IgM-LPL/WM · WM
Transplant is not the usual treatment
Myelodysplastic neoplasms
The marrow is busy, but the blood cells it makes do not work properly and too few of them survive.
Chronic myelomonocytic leukemiaFull guide
CMML · MDS/MPN-CMML
A stranger can be the matchMyelodysplastic neoplasm with biallelic TP53 inactivationFull guide
MDS-biTP53 · MDS with multi-hit TP53 · myelodysplastic syndrome
A stranger can be the matchMyelodysplastic neoplasm with increased blastsFull guide
MDS-IB · MDS-EB · RAEB (historical)
A stranger can be the matchMyelodysplastic neoplasm with low blasts and isolated 5q deletionFull guide
MDS-del(5q) · del5q MDS · 5q− syndrome
Transplant is not the usual treatmentMyelodysplastic neoplasm with low blasts and SF3B1 mutationFull guide
MDS-SF3B1 · MDS-RS (older) · myelodysplastic syndrome
Transplant is not the usual treatment
Myeloproliferative neoplasms
The marrow makes too many of one kind of blood cell, and over years that can scar the marrow itself.
Chronic neutrophilic leukemia
CNL · CSF3R-mutated CNL · myeloproliferative neoplasm
A stranger can be the matchJuvenile myelomonocytic leukemiaFull guide
JMML · JCML (historical)
A stranger can be the matchPost-essential thrombocythemia myelofibrosis
post-ET MF · PET-MF · post-ET myelofibrosis
A stranger can be the matchPost-polycythemia vera myelofibrosis
post-PV MF · PPV-MF · post-PV myelofibrosis
A stranger can be the matchPrimary myelofibrosis
PMF · MF · CIMF (historical)
A stranger can be the match
Plasma cell disorders
Conditions of the antibody-making cells in the marrow. Most transplants here use the person’s own cells.
Immunoglobulin-related amyloidosis (AL amyloidosis)Full guide
AL amyloidosis · light-chain amyloidosis · amyloidosis
Usually the person’s own cellsMultiple myelomaFull guide
PCM · MM · myeloma
Gene therapy is now an option tooPOEMS syndromeFull guide
POEMS · polyneuropathy, organomegaly, endocrinopathy, monoclonal plasma-cell disorder, skin changes
Usually the person’s own cellsPrimary plasma cell leukemiaFull guide
pPCL · primary PCL · plasma-cell-leukemia presentation
Usually the person’s own cells
Bone marrow failure
The marrow stops making enough blood. Some of these are inherited and some are acquired, and the difference changes everything.
Acquired severe aplastic anemiaFull guide
SAA · VSAA · AA
A stranger can be the matchCongenital amegakaryocytic thrombocytopeniaFull guide
CAMT · MPL-related congenital thrombocytopenia · thrombopoietin-receptor deficiency
A stranger can be the matchDiamond-Blackfan anemiaFull guide
DBA · DBA syndrome · inherited erythroblastopenia
Usually a family donorDyskeratosis congenitaFull guide
DC · TBD (broader umbrella) · telomeropathy (broader search term)
Usually a family donorFanconi anemiaFull guide
FA · FANC-associated bone marrow failure · inherited chromosomal-instability syndrome
Usually a family donorParoxysmal nocturnal hemoglobinuriaFull guide
PNH · PIGA-mutant clonal complement-mediated hemolysis · AA/PNH overlap
Transplant is not the usual treatmentSevere congenital neutropeniaFull guide
SCN · congenital neutropenia · ELANE-associated neutropenia
Transplant is not the usual treatmentShwachman-Diamond syndromeFull guide
SDS · SBDS-associated marrow failure · ribosomopathy
Transplant is not the usual treatment
Inherited red blood cell disorders
Conditions carried in the genes that change how red blood cells are shaped or how long they last.
Pyruvate kinase deficiencyFull guide
PK deficiency · PKD · PKLR deficiency
Transplant is not the usual treatmentSickle cell diseaseFull guide
SCD · HbSS disease · HbSβ0-thalassemia
Gene therapy is now an option tooTransfusion-dependent beta-thalassemiaFull guide
TDT · β-thalassemia major · BT major
Gene therapy is now an option too
Inherited immune disorders
Children born with part of the immune system missing or not working. Each has its own gene and its own path.
Adenosine deaminase-deficient severe combined immunodeficiency
ADA-SCID · ADA deficiency · ADA-deficient SCID
Gene therapy is now an option tooCD40 ligand deficiency
CD40LG deficiency · CD40L deficiency · HIGM1
A stranger can be the matchChronic granulomatous disease
CGD · NADPH oxidase deficiency · primary immunodeficiency
A stranger can be the matchCombined immunodeficiency due to DOCK8 deficiency
DOCK8 deficiency · DOCK8-CID · HIES2
A stranger can be the matchDCLRE1C-associated Artemis-deficient severe combined immunodeficiency
ART-SCID · DCLRE1C-SCID · T-B-NK+ SCID
A stranger can be the matchGATA2 deficiency syndrome
GATA2 deficiency · MonoMAC · DCML deficiency
A stranger can be the matchIL2RG-associated X-linked severe combined immunodeficiency
X-SCID · SCID-X1 · IL2RG deficiency
A stranger can be the matchIL7R-deficient severe combined immunodeficiency
IL7R-SCID · IL7RA deficiency · CD127 deficiency
A stranger can be the matchImmune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome
IPEX · IPEX syndrome
A stranger can be the matchJAK3-deficient severe combined immunodeficiency
JAK3-SCID · JAK3 deficiency · T-B+NK- SCID
A stranger can be the matchLeukocyte adhesion deficiency type I
LAD-I · LAD1 · ITGB2 deficiency
Gene therapy is now an option tooMHC class II deficiency
BLS II · BLS2 · MHC II deficiency
A stranger can be the matchPurine nucleoside phosphorylase deficiency
PNP deficiency · PNP-SCID · PNP-def
A stranger can be the matchRAG1 deficiency
RAG1-SCID · RAG1 deficiency · T-B-NK+ SCID
A stranger can be the matchRAG2 deficiency
RAG2-SCID · RAG2 deficiency · T-B-NK+ SCID
A stranger can be the matchWiskott-Aldrich syndrome
WAS · WAS gene deficiency · primary immunodeficiency
Gene therapy is now an option tooX-linked lymphoproliferative disease type 1 due to SH2D1A deficiency
XLP1 · XLP-1 · SH2D1A deficiency
A stranger can be the matchX-linked lymphoproliferative disease type 2 due to XIAP deficiency
XLP2 · XLP-2 · BIRC4 deficiency
A stranger can be the match
Inherited metabolic disorders
A missing enzyme lets a substance build up in the body. A transplant can supply cells that make what is missing.
Adult-onset metachromatic leukodystrophy
Adult MLD · AO-MLD · ARSA deficiency
Transplant is not the usual treatmentAlpha-mannosidosis
MAN2B1 deficiency · alpha-mannosidase deficiency
Transplant is not the usual treatmentAutosomal recessive malignant osteopetrosis
ARO · IMO · malignant infantile osteopetrosis
A stranger can be the matchEarly-juvenile metachromatic leukodystrophy
EJ-MLD · early-juvenile MLD · ARSA deficiency
Gene therapy is now an option tooFucosidosis
alpha-fucosidase deficiency · alpha-L-fucosidase deficiency
Transplant is not the usual treatmentGaucher disease type 3
GD3 · GD III · type 3 Gaucher disease
Transplant is not the usual treatmentInfantile Krabbe disease
IKD · EIKD · infantile GLD
A stranger can be the matchLate-infantile and juvenile Krabbe disease
LOKD · later-onset GLD · GALC deficiency
A stranger can be the matchLate-infantile metachromatic leukodystrophy
LI-MLD · late-infantile MLD · ARSA deficiency
Gene therapy is now an option tooLate-juvenile metachromatic leukodystrophy
LJ-MLD · late-juvenile MLD · ARSA deficiency
A stranger can be the matchMucolipidosis II alpha/beta
ML II · MLII · GNPTAB-related mucolipidosis II
Transplant is not the usual treatmentMucopolysaccharidosis type I, Hurler syndrome
MPS IH · MPS I-H · Hurler syndrome
A stranger can be the matchNiemann-Pick disease type C2
NPC2 · NPC type C2 · NPC2 deficiency
Transplant is not the usual treatmentX-linked cerebral adrenoleukodystrophy
cALD · CALD · X-CALD
A stranger can be the match
Histiocytic disorders
Conditions of the immune cells that normally clear away debris and infection, when too many are made or they behave wrongly.
Blastic plasmacytoid dendritic cell neoplasm
BPDCN · CD4+ CD56+ hematodermic neoplasm (historical)
A stranger can be the matchFamilial hemophagocytic lymphohistiocytosis
FHL · FHLH · primary HLH
A stranger can be the matchLangerhans cell histiocytosis
LCH · Langerhans-cell histiocytosis · Langerhans cell histiocytosis
Transplant is not the usual treatment
Autoimmune conditions
The immune system attacks the body it belongs to. A transplant is a rare, last-resort option in a few severe forms.
Chronic inflammatory demyelinating polyradiculoneuropathy
CIDP · chronic immune demyelinating neuropathy · immune-mediated polyradiculoneuropathy
Transplant is not the usual treatmentCrohn disease
CD · Crohn ileitis · Crohn colitis
Transplant is not the usual treatmentEarly diffuse cutaneous systemic sclerosis
SSc · dcSSc · early dcSSc
Usually the person’s own cellsHighly active relapsing multiple sclerosis
MS · RRMS · highly active MS
Usually the person’s own cellsMyasthenia gravis
MG · gMG · AChR-positive MG
Transplant is not the usual treatmentNeuromyelitis optica spectrum disorder
NMOSD · NMO · AQP4-IgG-positive NMOSD
Transplant is not the usual treatmentStiff-person spectrum disorder
SPSD · SPS · PERM
Transplant is not the usual treatmentSystemic lupus erythematosus
SLE · lupus · systemic LE
Transplant is not the usual treatment