Il trapianto di midollo nelle anemie

Among the anemias, aplastic anemia, thalassemia and Fanconi's disease are the most common indications for bone marrow transplantation (BMT). However, BMT carriers an unavoidable risk of procedure-related mortality; furthermore, improvements in conventional treatments have significantly prolonged the life expectancy of anemic patients. This makes it necessary to define reliable and disease-specific prognostic factors in order to restrict the use of BMT to patients with a favourable risk/benefit ratio. The most widely accepted predictive factors are age and hepatic dysfunction. The optimal conditioning regimens should ensure myeloablative efficacy while minimizing toxicities; cyclophosphamide, alone or in combination with limited field irradiation, is the preferred option. The rate of long term event-free survival after BMT generally exceeds 60%, but these figures should be cautiously compared with those achieved with conventional therapy. Patients lacking an HLA-identical sibling donor may be suitable for BMT from a non- conventional donor (matched unrelated, unmatched related or cord blood). In the case of patients with anemia, this approach can be proposed only for very young patients (less than 20 years) or as second-line treatment; actually, the results seem to be less encouraging than those reported in neoplastic hematological diseases.