title: Myeloablative conditioning in myelofibrosis using i.v. treosulfan and autologous peripheral blood progenitor cell transplantation with high doses of CD34+ cells results in hematologic responses - follow-up of three patients.
abstract: Autologous transplantation after myeloablation for myelofibrosis with myeloid metaplasia provides a palliative therapy with a long term relief of symptoms. We have transplanted three patients with more than 5 x 10(6) CD34+ cells/kg body weight after myeloablation with treosulfan (total dose 42 g/m(2)) with a 18 months follow-up. Two of the patients had symptomatic splenomegaly and severe anemia. One patient had symptomatic splenomegaly and thrombocytopenia (< 100x10(9)/L). Granulocyte colony-stimulating factor-supported peripheral blood progenitor cell mobilization and collection was not associated with increased toxicity. Following transplantation we observed a prolonged reconstitution period of 28-38 days without fever or severe mucositis. All patients became free of erythrocyte transfusions or recovered to normal thrombocyte counts. There was a significant reduction of max. spleen size in one patient. We conclude that myeloablation with treosulfan and autologous PBPCT in these three patients with myelofibrosis was safe and useful.
authors.affiliation: Department of Internal Medicine V, University of Heidelberg, Im Neuenheimer Feld 410, 69120 Heidelberg, Germany. stefan_fruehauf@med.uni-heidelberg.de
authors.forename: Stefan
authors.initials: S
authors.lastname: Fruehauf
authors.forename: Eike C
authors.initials: EC
authors.lastname: Buss
authors.forename: Julian
authors.initials: J
authors.lastname: Topaly
authors.forename: H H
authors.initials: HH
authors.lastname: Kreipe
authors.forename: A D
authors.initials: AD
authors.lastname: Ho
mesh_terms: D018952:Antigens, CD34 / Q000096:biosynthesis*; D002066:Busulfan / Q000008:administration & dosage / Q000031:analogs & derivatives* / Q000627:therapeutic use; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007262:Infusions, Intravenous; D008875:Middle Aged; D019653:Myeloablative Agonists / Q000627:therapeutic use; D036102:Peripheral Blood Stem Cell Transplantation / Q000379:methods*; D055728:Primary Myelofibrosis / Q000628:therapy*; D019172:Transplantation Conditioning / Q000379:methods*; D016896:Treatment Outcome
chemical_list: D018952:Antigens, CD34; D019653:Myeloablative Agonists; C018404:treosulfan; D002066:Busulfan