I wrote down a few things he said that were not on his slides…… just thought I’d share for those interested.
There are 7-8 million people in registry (US) and there are more in Europe. (This is up from the stats in 2006!!! Interesting)
He has a bit on the slides about the regimen…but not with numbers—he said they used this regimen on 50 patients in Cinci (I believe they were all Fanconi’s patients who have the same toxicity issues as SDSers) and they have used it on the 7 SDSers he reports on the slides.
The aplastic anemia patients did better than the MDS and Leukemia patients. The one adult with Leukemia is currently going through transplant again there in Cinci.
The regimen in Cinci begins 3 weeks before transplant—they start the Campath then. (this is on the slides)
For information on Shwachman-Diamond Syndrome check out Shwachman-Diamond America
