• 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:

    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:

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

  • Contact Us

    Our specialized new patient coordinators can answer your questions about treatment options and becoming a patient.
  • Stem Cell Transplant

    Dana-Farber/Boston Children’s has one of the largest and most experienced pediatric stem cell transplant programs in the U.S. Stem cell transplants can provide treatment for cancers, blood disorders, and other conditions.
  • Innovative Treatments

    Gene Therapy Callout2 

    Through clinical trials and research, Dana-Farber/Boston Children's is at the forefront of innovative treatments, including gene therapy.