Outcome of hematopoietic stem cell transplant in children with congenital heart disease.

CHD is the most commonly occurring birth defect in the United States. Improvements in supportive care for CHD result in increasing numbers of survivors who may develop benign or malignant conditions for which HSCT is indicated. However, the ability of individuals with CHD to tolerate HSCT is unknown. Retrospective medical record review of 1031 patients who underwent HSCT at Children's Hospital Boston between 1989 and 2007 identified those with CHD. Ten patients with CHD that required repair or palliation before or after HSCT, or with CHD that would have required repair had they survived HSCT, were identified. These patients tolerated chemotherapy and/or radiation therapy uneventfully. Although half experienced febrile neutropenia and two had documented bacteremia, no endocarditis was observed. During the first 100 days post-HSCT, combined rates of grade 3, 4, and 5 cardiac, renal, and pulmonary toxicity for these patients were 10%, 0%, and 10%, respectively. In children with underlying CHD, there was no clinical evidence of impaired ability to tolerate febrile neutropenia, volume challenge, or other regimen-related toxicities that might require significant cardiac reserve. CHD alone should not be considered an absolute contraindication for indicated HSCT.