BMT Consult

We met with Dr. Weinberg and Dr. Dvorak. This was the first time we met Dr. Weinberg. He is the new BMT Head Doctor from Los Angeles Children's Hospital. He is very experienced with SCIDS babies and Sickle Cell Anemia, cases much worse than David's Wiskott-Aldrich Syndrome. He is a nice doctor and showed attention to David, while providing good medical information. It was a good meeting.

They came up with an alternative therapy for David. They are concerned about two things: (1) his previous brain hemorrhage (2) the numerous platelet transfusions. The traditional conditioning regimen consists of Busulfan (chemotherapy), Cyclophosphamide (chemotherapy) and anti-thymocyte globulin or ATG (immunosuppressive). The doses of these drugs can lead to complications such as severe mouth sores, liver, or lung complications. In patients with a history of several blood transfusions before transplant, the complications can be particularly serious. In order to decrease the complications they proposed a modified treatment.

The treatment David will receive includes Busulfan, Fludarabine and Campath, with lower doses of Cyclophosphamide. Basically, they are not using ATG, rather replacing it with Campath. This was an answered prayer! ATG is rabbit or horse serum. Rabbits or horses are injected with human blood cells and develop antibodies. The rabbits and horses are taken to the blood banks to give blood, the serum is given back to humans. There have been great results from ATG, however, one drawback is that ATG binds to platelets and destroys them. This needs to be avoided in David's case, to prevent another brain hemorrhage. I was so happy about this decision! Elated! Jumping for joy! Supposedly there are less toxic side effects using this alternative therapy regimen.