Conditions Treated by Pediatric Stem Cell Transplantation

Stem cell transplantation and gene therapy can help children with a wide range of conditions—many of which have few other treatments. Our doctors and researchers are working to better understand these conditions in the lab and apply what they learn to patient care, leading to better outcomes for your child.


Many pediatric cancers can be treated with stem cell transplantation, including:

Blood diseases

Many types of blood diseases can be treated with stem cell transplantation or gene therapy, including:

Bone marrow failure disorders

Since bone marrow failure disorders are caused by problems in the bone marrow, a bone marrow or stem cell transplant may be a good treatment option for conditions like the following:

  • Aplastic anemia.
  • Fanconi anemia.
  • Diamond Blackfan anemia (DBA).
  • Severe congenital neutropenia, or Kostmann syndrome—a rare genetic condition that increases your child’s risk of having frequent infections.
  • Paroxysmal nocturnal hemoglobinuria—a rare disorder in which red blood cells break apart.

White blood cell disorders

  • Chronic granulomatous disease.
  • Leukocyte adhesion deficiency—a rare disorder in which the immune system doesn’t work properly, resulting in frequent infections.
  • Chediak-Higashi syndrome—a rare genetic disease that affects many parts of the body and can cause symptoms like frequent infections, easy bruising, and albinism.

Immunological disorders

  • Autoimmune focal segmental glomerulosclerosis (FSGS).
  • Severe combined immunodeficiency (SCID).
  • Wiskott Aldrich syndrome.
  • IPEX syndrome.
  • Hyper IgM syndrome.
  • Adenosine deaminase deficiency (ADA)—an inherited condition that can damage the immune system, leading to other conditions, such as severe combined immunodeficiency (SCID).
  • Bare lymphocyte syndrome—a genetic immune disease that leads to frequent infections in the lungs.
  • Omenn syndrome—an inherited disorder characterized by red and peeling skin and frequent infections.
  • CD40 ligand deficiency, or X-linked Hyper IgM syndrome—an immune disease that causes frequent infections.
  • DOCK8 deficiency—a genetic immune disorder that can cause eczema and lung infections.
  • Hyper IgE syndrome—a rare immune disease characterized by eczema and frequent skin and lung infections.
  • X-linked lymphoproliferative (XLP) syndrome—also called Duncan’s syndrome, this rare genetic disorder causes the immune system to overreact to infections.
  • Hereditary lymphohistiocytosis—also called familial hemophagocytic lymphohistiocytosis (HLH), this condition occurs when the body makes too many activated immune cells.
  • STAT1 gain-of-function (GOF)—an immune disease that causes frequent skin and lung infections.
  • Autoinflammatory syndromes—a group of conditions in which the immune system overreacts, causing fevers and other symptoms.
  • 22q11.2 deletion syndrome (DiGeorge syndrome).

Genetic diseases

  • Adrenoleukodystrophy.
  • Mucopolysaccharidosis type I (Hurler syndrome)—a rare condition in which the body cannot break down certain molecules, leading to various health issues.
  • Metachromatic leukodystrophy—a rare disease in which the body cannot break down certain fatty substances (lipids), leading to problems with the brain and nervous system.
  • Gaucher disease—a buildup of fatty substances in the spleen, liver, and bone tissue.
  • Mucopolysaccharidosis type VI (Maroteaux-Lamy syndrome)—when the body lacks an enzyme to help it break down certain complex carbohydrates, leading to a buildup across the body.
  • Pyruvate kinase deficiency.
  • Schimke immuno-osseous dysplasia (SIOD).