Therapeutic decision making in BMT/SCT for hemoglobinopathies

β-Thalassemia syndromes page 215 Complications of sickle cell disease 216 Results with conventional therapy for thalassemia 216 Results with conventional therapy for sickle cell disease 218 Results with allogeneic transplant for thalassemia major 219 Results with allogeneic transplant for sickle cell disease 222 Indications for transplant 223 Contraindications to transplant 223 Pretransplant workup 223 Myeloablative or reduced-intensity conditioning regimen for hemoglobinopathy transplants 224 Disease specific precautions (sickle cell disease) during transplant 225 Monitoring posttransplant 225 Further reading 226 β-Thalassemia syndromes Complications of sickle cell disease Results with conventional therapy for thalassemia The two main therapeutic options for patients with thalassemia major are transfusion/chelation therapy or allogeneic transplant, if a suitable donor is available. Results with conventional therapy for sickle cell disease Results with allogeneic transplant for thalassemia major Results with allogeneic transplant for sickle cell disease These data were recently extended by a publication from the CIBMTR (Panepinto et al., 2007). Sixty-four of 67 patients were alive with a 5-year probability of disease-free and overall survival of 85% and 97%. These patients were also mainly children (age median 10 years). Only three patients were older than 21 years. The conditioning used was busulfan and cyclophosphamide in 63 patients (94%). Indications for transplant If an HLA-identical sibling donor is available: Thalassemia major (transplant preferably in childhood, cord blood transplant feasible) Sickle cell disease (severe cases with impending vital organ damage or poor quality of life – transplant preferably in childhood before irreversible organ dysfunction) 2. If no HLA-identical sibling donor is available: