Allogeneic marrow transplantation for primary myelofibrosis and myelofibrosis secondary to polycythaemia vera or essential thrombocytosis

• Published in: British journal of haematology 1997-09, Vol.98 (4), p.1010-1016
• Main Authors: Anderson, Jeanne E., Sale, George, Appelbaum, Frederick R., Chauncey, Thomas R., Storb, Rainer
• Format: Article
• Language: English
• Subjects: Adolescent; Adult; agnogenic myeloid metaplasia; allogeneic transplantation; Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy; Biological and medical sciences; Bone Marrow Transplantation - adverse effects; Bone Marrow Transplantation - methods; Bone marrow, stem cells transplantation. Graft versus host reaction; essential thrombocytosis; Female; Humans; Male; Medical sciences; Middle Aged; myelofibrosis; polycythaemia vera; Polycythemia Vera - complications; Primary Myelofibrosis - etiology; Primary Myelofibrosis - therapy; Recurrence; Survival Analysis; Survival Rate; Thrombocythemia, Essential - complications; Transfusions. Complications. Transfusion reactions. Cell and gene therapy; Transplantation, Homologous
• ISSN: 0007-1048; 1365-2141
• DOI: 10.1046/j.1365-2141.1997.3083125.x

Primary myelofibrosis is a clonal haemopoietic disorder, incurable with conventional therapy, and associated with a median survival of 4–5 years. Patients with polycythaemia vera and essential thrombocytosis who progress into a myelofibrotic picture also have a poor prognosis. Between 1980 and 1996, 13 patients with myelofibrosis due to one of these three myeloproliferative disorders (primary myelofibrosis [n=8], essential thrombocytosis [n=3], polycythaemia vera [n=2]) underwent allogeneic marrow transplantation in Seattle. The median age was 40 years (range 18–49). The median time from myeloproliferative diagnosis to transplantation was 39 months (range 5–192). Three patients received preparative regimens containing total body irradiation and 10 received busulphan–cyclophosphamide regimens. Nine patients received marrow from HLA‐matched related donors, one from a one antigen mismatched related donor, and three from HLA‐matched unrelated donors. The median time to both granulocyte and platelet engraftment was 21 d. Nine patients survive between 1.2 and 7.1 years post‐transplant. Two patients relapsed at 1 year post‐transplant, both of whom survive in a chronic myeloproliferative state. Four patients died of transplant‐related complications between 43 d and 2.2 years post‐transplant. At 1 year post‐transplant the majority of the disease‐free survivors have normal peripheral blood counts and none‐to‐minimal marrow fibrosis. These preliminary results are encouraging, and suggest that stem cell transplantation can be curative therapy for selected patients with myelofibrosis.