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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