Diseases for Which Standard Treatments Are Currently Being Used*

Written by Andrew Ripps. Posted in Diseases Treated With Stem Cells


Acute leukemia
Acute lymphoblastic leukemia – ALL
Acute myelogenous leukemia – AML
Acute biphenotypic leukemia
Acute undifferentiated leukemia
Chronic leukemia
Chronic myelogenous leukemia - CML
Chronic lymphocytic leukemia - CLL
Juvenile chronic myelogenous leukemia - JCML
Juvenile myelomonocytic leukemia - JMML
Myelodysplastic syndromes
Refractory anemia - RA
Refractory anemia with ringed sideroblasts - RARS
Refractory anemia with excess blasts - RAEB
Refractory anemia with excess blasts in transformation - RAEB-T
Chronic myelomonocytic leukemia - CMML


Non-Hodgkin's lymphoma
Hodgkin's lymphoma

Other disorders of blood cell proliferation

Aplastic anemia
Congenital dyserythropoietic anemia
Fanconi anemia*
Paroxysmal nocturnal hemoglobinuria – PNH
Pure red cell aplasia

* The first cord blood transplantation in 1988 was for this disease.

Inherited red cell (erythrocyte) abnormalities
Beta thalassemia major – Cooley’s anemia
Blackfan-Diamond anemia
Pure red cell aplasia
Sickle cell disease

Inherited platelet abnormalities
Amegakaryocytosis / Congenital thrombocytopenia
Glanzmann thrombasthenia

Myeloproliferative disorders
Acute myelofibrosis
Agnogenic myeloid metaplasia – myelofibrosis
Polycythemia vera
Essential thrombocythemia

Inherited immune system disorders
Severe combined immunodeficiency – SCID
SCID with absence of T cells, normal B cells
SCID with adenosine deaminase deficiency – ADA-SCID
SCID with absence of T & B cells
Omenn syndrome
SCID which is X-linked

Inherited immune system disorders – neutropenias
Kostmann syndrome

Other inherited immune system disorders
Leukocyte adhesion deficiency
DiGeorge syndrome
Bare lymphocyte syndrome
Common variable immunodeficiency
Wiskott-Aldrich syndrome
Lymphoproliferative disorders LPD
Lymphoproliferative disorder, X-linked – aka Epstein-Barr virus susceptibility
Hemophagocytic lymphohistiocytosis
Wiskott-Aldrich syndrome

Bone marrow cancers
Multiple myeloma
Plasma cell leukemia
Waldenstrom's macroglobulinemia

Other cancers – Not originating in the blood system


Other inherited immune system disorders

Cartilage-hair hypoplasia
Gunther's disease erythropoietic porphyria
Hermansky-Pudlak syndrome
Pearson's syndrome
Shwachman-Diamond syndrome
Systemic mastocytosis

Transplants for Inherited Metabolic Disorders

Mucopolysaccharidoses – MPS
Hurler's syndrome - MPS-IH
Scheie syndrome - MPS-IS
Hunter's syndrome - MPS-II
Sanfilippo syndrome - MPS-III
Morquio syndrome - MPS-IV
Maroteaux-Lamy syndrome MPS-VI
Sly syndrome, beta-glucuronidase deficiency MPS-VII
Mucolipidosis II - I-cell disease
Leukodystrophy disorders
Adrenoleukodystrophy - ALD
Adrenomyeloneuropathy - AMN
Krabbe disease - globoid cell leukodystrophy
Metachromatic leukodystrophy
Pelizaeus-Merzbacher disease
Lysosomal storage diseases
Niemann-Pick disease
Sandhoff disease
Wolman disease
Other inherited metabolic disorders
Lesch-Nyhan syndrome (case report)


* Last updated November 2012