Results of Myeloablative Allogenic Stem Cell Transplant (SCT) for Severe Sickle Cell Disease in France
Reviewer: Julie Draznin Maltzman, MD
Last Modified: December 8, 2002
Presenter: Francoise Bernaudin
Presenter's Affiliation: SFGM-TC, France
Type of Session: Plenary
- Sickle cell disease effects the black population and is responsible for many hospitalizations and a high consumption of red blood cell transfusions.
- The only definitive cure remains a bone marrow transplant.
- However, the risks of a allogenic transplant must be wieghed by the incremental benefit that it will offer.
- This is a multi country and multi center trial looking at allo-transplants in sickle cell patients.
Materials and Methods
- 69 patients with sickle cell disease were taken to transplant.
- Pt age varied between 9 and 22 years.
- 38 of these pts had previous vascular events due to thier disease. 6 had severe transfusion requiring anemias, 25 had more than 3 crisis per year, and 7 pts were previously treated on Hydroxyurea.
- 65 pts had identical matched transplant whereas 7 had a cord blood transplant.
- three different conditioning treatments were used: Busulfan with Cytoxan, Busulfan, Cytoxan and TBI, and Busulfan, Cytoxan and ATG.
- 68 of the 69 pt had engraftment. One did not engraft.
- 7.2% of the pts experienced graft rejection. However no pts who received ATG had graft rejection.
- There were 6 deaths: one due to sepsis, 1 to a cerberal hemorrhage and 4 to GVHD.
- Event free survival at 54 months was 85%.
- Overall patients had long lasting improvements in transfusion requiremenets, veno occlusive crisis, and cerbral events.
- Allo transplants offer a viable choice for sickle cell pts.
- The main complications are those related to GVHD so techniques that decrease the incidence and severity of GVHD should be investigated in these pts.
- Sickle cell disease can be very debilitating causing both severe medical and emotional consequences. This study shows that allo transplant is a viable option for those willing to take the inital risks of transplant for a life long cure from Sickle Cell anemia.
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