A bone marrow or cord blood transplant may be the best treatment option or the only potential for a cure for patients with leukemia, lymphoma, sickle cell anemia and many other diseases. As the science of transplant continues to advance, new diseases are being treated with transplant.
Leukemias and lymphomas, including:
- Acute myelogeneous leukemia (AML)—The most common type of acute leukemia with nearly 15,000 new cases found in the United States each year. AML can affect people of any age, but is most common in adults.
- Acute lymphoblastic leukemia (ALL)—There are about 6,000 new cases of ALL in the United States each year. It can affect people of any age, but is the most common type of leukemia in children under 15.
- Chronic lymphocytic leukemia (CLL)—Primarily an adult disease, CLL is very rare in children and young adults. About 15,000 people are diagnosed each year in the United States.
- Chronic myelogeneous leukemia (CML)—A relatively common form of leukemia, it affects more than 20,000 people in the United States with 6,000 new cases each year. Most cases of CML appear in adults.
- Juvenile myelomonocytic leukemia
- Hodgkin lymphoma
- Non-Hodgkin lymphoma (NHL)—Each year about 69,000 people are diagnosed with NHL in the United States. Most of these people are older than 60.
Bone marrow diseases and other diseases when bone marrow fails to work, including:
- Severe aplastic anemia—A rare disease with 600-900 people diagnosed each year. The disease appears more often in eastern Asian countries. It can affect people of any age, but is most common in young adults.
- Fanconi anemia
- Paroxysmal nocturnal hemoglobinuria (PNH)
- Pure red cell aplasia
- Amegakaryocytosis/congenital thrombocytopenia
Inherited immune system disorders, including:
- Severe combined immunodeficiency (SCID, all types)—SCID is a group of inherited immune system disorders that are present at birth. They can become life-threatening within the first year of life if left untreated. In the United States, about 1 in 100,000 babies are born with SCID.
- Wiskott-Aldrich syndrome (WAS)—WAS is an inherited immune system disorder that is present at birth. It affects mostly boys. In the U.S. about 1 in 100,000 boys are born with it.
Hemoglobinopathies (diseases with poorly functioning red blood cells), including:
- Beta thalassemia major
- Sickle cell disease (SCD)—Sickle cell disease is an inherited disease of the red blood cells. In the United States, it affects about 70,000-100,000 people and is most common among African Americans and Hispanics
Inherited metabolic disorders, including:
- Krabbe disease (GLD)—GLD is very rare. Only about 40 children are diagnosed in the United States each year. Most often it appears in the first months of life (early-onset). There is also another form of the disease that doesn’t show until later in childhood or even into teenage years (late-onset).
- Hurler syndrome (MPS-IH)—Occurs in about 1 of every 100,000 babies born. If not treated, children born with this disease usually die by 5 to 10 years of age.
- Adrenoleukodystrophy (ALD)—Both boys and girls can be born with ALD, but boys are more likely to have symptoms. One out of 3 boys born with ALD has the most severe form of it, cerebral X-linked ALD. The form is rare, affecting about 1 in 20,000 individuals worldwide.
- Metachromatic leukodystrophy (MLD)—MLD appears most often in babies and young toddlers, but it also occurs in older children and adults. MLD is rare. It occurs in about 1 in 40,000 to 160,000 individuals worldwide.
Myelodysplastic syndromes and myeloproliferative disorders. MDS are a group of diseases that affect the bone marrow and blood. About 19,000 people are diagnosed with MDS in the United States each year.
Multiple myeloma and other plasma cell disorders
Familial erythrophagocytic lymphohistiocytosis and other histiocytic disorders
Certain other malignancies or cancers