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Bone Marrow/Stem Cell Transplant Frequently Asked Questions
Helpful information for patients, caregivers and families
(Bilingual Spanish/English)

1. What diseases are typically treated with bone marrow/stem cell transplant?

Bone marrow/stem cell transplants are generally used to treat diseases of the blood, the immune system, or genetic disorders. A transplant can replace unhealthy bloodforming cells with healthy ones, or it can replenish blood-forming cells if chemotherapy and radiation destroy them. The diseases and disorders treated with bone marrow, peripheral blood, or cord blood transplantation include:

Leukemias

• Acute myelogenous leukemia (AML), acute lymphoblastic leukemia (ALL), chronic myelogenous leukemia (CML), chronic lymphocytic leukemia (CLL), juvenile myelomonocytic leukemia (JMML)

Lymphomas

• Hodgkin and non-Hodgkin lymphoma

Multiple myeloma and other plasma cell disorders

Severe aplastic anemia and other marrow failure states including:

• Severe aplastic anemia, Fanconi anemia, paroxysmal nocturnal hemoglobinuria (PNH), pure red cell aplasia, and amegakaryocytosis / congenital thrombocytopenia

Inherited immune system disorders including:

• Severe combined immunodeficiency (SCID) and Wiskott-Aldrich syndrome

Hemoglobinopathies including:

• Beta thalassemia major and sickle cell disease

Inherited metabolic disorders including:

• Hurler's syndrome (MPS-IH), adrenoleukodystrophy, and metachromatic leukodystrophy

Myelodysplastic and myeloproliferative disorders including:

• Refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, refractory cytopenias with multilineage dysplasia, polycythemia vera with fibrosis, essential thrombocythemia with fibrosis, chronic myelomonocytic leukemia, and agnogenic myeloid metaplasia (myelofibrosis)

Familial erythrophagocytic lymphohistiocytosis and other histiocytic disorders

Other conditions are also treated with bone marrow/ stem cell transplant, often through clinical trials. (See Question 18)

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