Other Pediatric Conditions We Treat

The pediatric specialists at Dana-Farber/Boston Children's Cancer and Blood Disorders Center also participate in the care of children and teens with a variety of conditions that do not come under the umbrella of cancers or blood disorders. These conditions include genetic immunodeficiencies, metabolic disorders, vascular anomalies, and cardiovascular diseases such as pulmonary hypertension and pulmonary vein stenosis.

Our doctors work closely with other specialists to develop a multidisciplinary approach tailored to the needs of each child. This sometimes includes treatments originally designed for treating cancers and blood disorders—including chemotherapy and anti-angiogenic therapies—as appropriate.

We also offer advanced treatment strategies—including stem cell transplant and gene therapy—for select conditions. Some of these treatments have been groundbreaking for children and teens with cancers and blood disorders, but are being shown to be effective for other diseases as well..

Other Pediatric Conditions Treated through Stem Cell Transplantation

A stem cell transplant (also called bone marrow transplant) is the infusion of healthy stem cells into the body to stimulate new bone marrow growth.

While often used to treat cancers and blood disorders of children and teens, stem cell transplants are becoming an attractive treatment option for a growing range of conditions arising from problems with cells from the bone marrow, such as:

• Genetic immunodeficiencies
• Chronic granulomatous disease (CGD)
• Combined variable immunodeficiency (CVID)
• Hemophagocytic lymphohistiocytosis (HLH)
• X-linked lymphoproliferative disease (XLP)
• X-linked severe combined immunodeficiency 1 (SCID-X1)
• Wiskott Aldrich Syndrome (WAS)
• Metabolic diseases
• Adrenoleukodystrophy (ALD)
• Globoid cell leukodystrophy (Krabbe disease)
• Hunter syndrome
• Hurler syndrome
• Metachromatic leukodystrophy
• Morquio syndrome
• Sanfillipo syndrome
• Osteopetrosis

Other Pediatric Conditions Treated through Gene Therapy

Our genes, which hold the code for all of our body's functions, are made of DNA. Damage to DNA, such as a mutation, is an underlying cause of the genetic defects that lead to cancers, blood disorders, and other conditions. Gene therapy delivers DNA into a patient’s cells to replace faulty or missing genes, or add new genes.

Doctors at Dana-Farber/Boston Children's offer gene therapy clinical trials to treat:

• Adrenoleukodystrophy (ALD)
• X-linked severe combined immunodeficiency 1 (SCID-X1)
• Wiskott Aldrich Syndrome (WAS)

And hope to extend gene therapy to additional conditions in the future.