Langzeitverlauf nach Stammzelltransplantation bei Thalassaemia major
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Ressourcen- / MedientypDissertation, Text
Datum der Freischaltung2016-04-04
Beta-thalassemia is an autosomal genetic disorder that disrupts normal erythropoiesis and leads to profound anemia. As a result patients depend on life-long transfusion of red blood cells. The regular transfusion regime leads to secondary iron overload and, without adequate chelation therapy, to patient death due to cardiomyopathy before their third decade. Despite significant risks, the allogeneic stem cell transplantation remains the only causative cure to this day. We aimed to review the long-term outcome of 32 ex-thalassemic pediatric patients who received an allogeneic stem cell transplantation at Ulm University between 1995 and 2005. Special interest was given to hematological reconstitution, chimerism, iron overload and life quality. The majority of patients belonged to the pesaro risk class 1 and was under 16 years of age at the time of transplant. All received stem cells from HLA-identical matched donors. The GvHD rate was low. The overall survival was 100 %, the event-free survival was 97 %. One patient had a late graft failure with autologous reconstitution. This patient was successfully retransplanted. One third of patients had a split chimerism between nucleated and red blood cells (mean follow-up: 6 years). The vast majority of patients had elevated serum ferritin levels post transplantation, one third had clinical signs of iron overload. Life quality was comparable with that of healthy children. In keeping with results from other centers we can recommend that patients who have an HLA-identical matched donor should be transplanted early, before any significant iron overload develops.
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Stem cell transplantation