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What if we could just “fix” sickle cell disease? We have kidney transplants for congenital renal disease, liver transplants for biliary atresia, lung transplants for cystic fibrosis—it seems obvious to consider bone marrow transplantation for congenital abnormalities of hemoglobin. Yet, this therapy is brand new and still in its developing stages; there have only been a few hundred transplants so far in the United States in a population of nearly 100,000 with some sort of congenital hemoglobinopathy. The exciting news is that the huge majority of these patients have done very well, offering new hope to people with hemoglobinopathies and to the 1000 families each year who have a new baby with sickle cell disease and other hemoglobinopathies.

In this month’s issue of Current Problems in Pediatric and Adolescent Health Care, Dr. Jakub Tolar and his colleagues discuss the many aspects of this topic, including a parent’s perspective on this new therapeutic approach. The cutting edge of all this is consideration not just of standard transplant approaches, but of correcting the abnormality on the level of the gene. This is even further from “ready for prime time” than standard bone marrow transplant, but how exciting to consider where this could go! And just in case we think this is only medical news, bone marrow transplantation for sickle cell disease made the popular press last fall, when Carlos Boozer, All-Star forward for the Utah Jazz basketball team, sat out the start of the season as his one year old son received a bone marrow transplant. At this writing, the baby is doing well, and his father is back to work on the basketball court–just the happy ending we all hope for!