Bone marrow transplant is beneficial for some rare pediatric conditions
Research Activities, March 2012, No. 379
Hematopoietic Stem-Cell Transplant (HSCT, also known as bone marrow transplant) for pediatric conditions, can be an effective treatment for Wolman's disease and Faber's disease Type 2/3, both rare fatal inherited metabolic disorders. That's the conclusion of a new research review from the Effective Health Care Program of the Agency for Healthcare Research and Quality (AHRQ). There is very limited evidence to suggest that autologous HSCT was associated with extended periods of drug- free clinical remission in a number of autoimmune diseases, including newly diagnosed type 1 juvenile diabetes and severe refractory juvenile idiopathic arthritis, systemic lupus erythematosus, systemic sclerosis, and Crohn's disease. However, the evidence is insufficient to determine the overall long-term benefits or harms of HSCT for these pediatric diseases. For the vast majority of rare pediatric diseases there is insufficient evidence to draw any conclusions about the benefit or harm of HSCT versus other approaches.
These findings are summarized in the research review, Hematopoietic Stem-Cell Transplantation in the Pediatric Population, and the future research needs are identified in an accompanying report, Future Research Needs for Hematopoietic Stem-Cell Transplantation in the Pediatric Population.
These materials and many others that explore the effectiveness and risks of treatment options for various conditions are available on AHRQ's Effective Health Care Program Web site.