MARROW TRANSPLANTATION FOR PAROXYSMAL-NOCTURNAL HEMOGLOBINURIA

Between 1971 and 1990, nine patients ranging in age from 14-38 years r eceived marrow transplants for paroxysmal nocturnal hemoglobinuria (PN H). Six were transplanted for aplastic complications of PNH. Four of t hese were from HLA-identical siblings, and the patients were condition ed with cyclophosphamide. One graft was from a syngeneic twin without conditioning, and one from a two HLA-antigen nonidentical father after conditioning with cyclophosphamide and total body irradiation. Three of the four recipients of allogeneic marrow developed acute and two ch ronic graft-versus-host disease (GVHD). Five of six transplanted for s evere aplastic anemia are long-term survivors with follow-up ranging f rom more than 6.2 to more than 19.1 years. The HLA nonidentical transp lant recipient experienced graft rejection and died of a pulmonary hem orrhage, Three patients were transplanted for nonaplastic complication s of PNH consisting of life threatening recurrent thromboses or refrac tory hemolysis. Two of these patients received marrow grafts from HLA- identical siblings after conditioning with busulfan and cyclophosphami de. They are surviving with normal hemograms > 2.2 and > 2.5 years and had mild chronic GVHD which resolved, although one has biochemical ev idence of PNH in 15% of the red cells. One received a syngeneic marrow graft without conditioning but reverted to PNH. He is alive > 8.6 yea rs after transplantation. Marrow transplantation for aplastic complica tions of PNH is successful, well tolerated, and compatible with long-t erm survival when an HLA-identical sibling or a syngeneic donor is ava ilable. For patients without aplasia, one must weigh the complications of transplantation with the life threatening nature of thrombotic epi sodes and hemolysis.