Conditions Treated with Blood and Marrow Transplantation

Malignant conditions treated with Blood and Marrow Transplant

Acute Lymphoblastic Leukemia (ALL)

Acute lymphocytic leukemia (ALL), also called acute lymphoblastic leukemia, is the most common cancer and the most common leukemia affecting children. It is a cancer that affects the bone marrow and subsequently, the blood.

Acute Myeloid Leukemia (AML)

Acute myeloid leukemia is a leukemia that starts in the bone marrow (the tissue inside the bone where blood is made) and results in the creation of immature white blood cells.

Aplastic Anemia (AA)

When the bone marrow doesn’t produce enough blood cells, this condition is known as aplastic anemia.

Brain Tumors

The term brain tumor refers to an unusual growth of tissue seen in the brain irrespective of the nature and cause of the growth.

Chronic Myelogenous Leukemia (CML)

Chronic myelogenous leukemia is a rare cancer of the blood in children where over time, too many immature white blood cells form because of an abnormality in the normal maturation process of the cell.

Juvenile Myelomonocytic Leukemia

JMML is a rare, serious chronic form of cancer of the blood in children (frequently boys), aged less than 4 years.


Lymphomas are a type of cancer involving lymphocytes, which are cells within the immune system that help the body fight off infections.


Neuroblastoma is a form of cancer that primarily affects children. Among infants, it ranks as the most common form of cancer. It usually occurs in the adrenal glands above the kidneys, but can also start anyway along the nerves in the back.

Non-malignant conditions treated with Blood and Marrow Transplant

Acquired Aplastic Anemia

Please see Aplastic Anemia for further information.

Chronic Granulomatous Disease

CGD is the disorder that develops when phagocytes (one of the body's blood cells) doen't function properly.

Congenital Amegakaryocytic Thrombocytopenia

CAT is a rare disorder found in infants where there are very few megakaryocytes and platelets in bone marrow.

Congenital Anemia

Congenital anemia, including Fanconi anemia, is a group of inherited diseases that affect the bone marrow, decreasing production of all types of blood cells.

Diamond-Blackfan Anemia

Diamond-Blackman anemia is a rare disorder of the bone marrow which fails to produce enough new red blood cells.

Dyskeratosis congenita

Dyskeratosis congenita is a rare form of bone marrow failure, with associated skin/nail abnormalities, and thickened white patches in the mouth.

Fanconi Anemia

Fanconi anemia is a rare inherited disorder that involves a wide spectrum of abnormalities involving bone, ears, kidneys, other physical abnormalities. Some of the patients may develop bone marrow failure or leukemia.

Hemophagocytic Lymphohistiocytosis

The disease is an extreme example of immune system dysfunction, where the body’s own immune system begins to attack important tissues and organs such as the brain, liver and bone marrow.


Leukodystrophy refers to a group of rare progressive diseases that affect the white matter of the brain (myelin), causing a slowing or blockage in brain signals.

Metabolic Storage Diseases

Metabolic storage diseases are a fairly large group of rare genetic inherited disorders in which specific enzymes of a child's cell are insufficient. Enzymes help break down certain materials within the cell for proper cell function.

Myelodysplastic Syndrome

Myelodysplastic syndrome (MDS) is referred to a group of different types of cancer. But they all impact the bone marrow and prevent it from developing mature, healthy blood cells.

Paroxysmal Nocturnal Hemoglobinuria

Paroxysmal nocturnal hemoglobinuria is a rare, dangerous, acquired blood disorder during which unpredictable episodes of blood red cell breakdown occurs, the bone marrow stops making new blood cells and blood clots.


Polycythemia is a rare and serious blood disease that causes the bone marrow to produce too many red blood cells to circulate in the blood stream.

Severe Combined Immune Deficiency

Severe combined immune deficiency is a rare genetic birth defect that doesn’t allow the body to develop an immune system, which fights all types of infections.

Shwachman-Diamond Syndrome

Shwachman-Diamond syndrome is a rare complex disorder that affects a child's bone marrow, pancreas, bones, and sometimes other parts of the body.

Sickle Cell Disease

Sickle cell disease is a group of familial red blood cell disorders. Sickle cell disease causes the red blood cells to be oddly shaped, and have difficulty flowing through the blood vessels properly which causes them to break up easily resulting in anemia and damage to the organs.