Marrow transplantation for thalassemia

Despite modern therapeutic regimens, thalassemia major remains a severe disease with an uncertain ultimate prognosis. Alternative and more definitive forms of treatment are actively sought; bone marrow transplantation is one of these. As of May 1984, data were available on 51 thalassemic children who had undergone the procedure: 29 of them are free of thalassemia 2 to 30 months after transplantation; 13 have died; 9 are alive with thalassemia after autologous reconstitution. Future developments in the field of transplantation should make the procedure safer and also render it available to the vast population of multiply transfused and hemosiderotic thalassemic patients who will not benefit from the improvement of conventional therapy.