Bone marrow transplantation for acute leukemia and Down syndrome: report of a successful case and results of a national survey.

We report a child with Down syndrome (DS) and acute lymphoblastic leukemia who is a healthy survivor 38 months after bone marrow transplantation (BMT). Psychometric evaluations performed before and after BMT indicate no demonstrable therapy-related change in intellectual function. A survey of BMT centers in the United States indicated that 16 leukemic DS children have had transplants at 10 BMT centers. Seven of these children survive at 11, 14, 17, 18, 22, 38, and 47 months, respectively, after BMT. Although these results are comparable to those for non-DS children, the number of DS children having transplants is only 20% to 25% of that predicted. We conclude that there is no justification for denial of BMT to otherwise appropriate candidates with DS and leukemia.