title: Myeloablation and autologous peripheral blood stem cell rescue results in hematologic and clinical responses in patients with myeloid metaplasia with myelofibrosis.
abstract: Current therapeutic options for myeloid metaplasia with myelofibrosis (MMM) are limited. A pilot study was conducted of autologous peripheral blood stem cell (PBSC) collection in 27, followed by transplantation in 21 patients with MMM. The median age was 59 (range 45-75) years. PBSCs were mobilized at steady state (n = 2), after granulocyte colony-stimulating factor (G-CSF) alone (n = 17), or after anthracycline-cytarabine induction plus G-CSF (n = 8). A median of 11.6 x 10(6) (range 0 to 410 x 10(6)) CD34(+) cells per kilogram were collected. Twenty-one patients then underwent myeloablation with oral busulfan (16 mg/kg) and PBSC transplantation. The median times to neutrophil and platelet recovery after transplantation were 21 (range 10-96) and 21 (range, 13 to > or = 246) days, respectively. Five patients received back-up PBSC infusion because of delayed neutrophil or platelet recovery. The median follow-up is 390 (range 70-1623) days after transplantation, and the 2-year actuarial survival is 61%. After transplantion, 6 patients died: 3 of nonrelapse causes (1 within 100 days of PBSC infusion) and 3 of disease progression. Erythroid response (hemoglobin > or = 100 g/L [10 gm/dL] without transfusion for > or = 8 weeks) occurred in 10 of 17 anemic patients. Four of 8 patients with a platelet count less than 100 x 10(9)/L (100 000/microL) responded with a durable platelet count more than 100 x 10(9)/L (100 000/microL). Symptomatic splenomegaly improved in 7 of 10 patients. It is concluded that (1) PBSC collection was feasible and stable engraftment occurred after transplantation in most patients with MMM, (2) myeloablation with busulfan was associated with acceptable toxicity, (3) a significant proportion of patients derived clinical benefit after treatment, and (4) further investigation of this novel approach is warranted. (Blood. 2001;98:586-593)
authors.affiliation: Division of Hematology, Department of Medicine, University of Texas Health Science Center at San Antonio, TX, USA.
authors.forename: J E
authors.initials: JE
authors.lastname: Anderson
authors.forename: A
authors.initials: A
authors.lastname: Tefferi
authors.forename: F
authors.initials: F
authors.lastname: Craig
authors.forename: L
authors.initials: L
authors.lastname: Holmberg
authors.forename: T
authors.initials: T
authors.lastname: Chauncey
authors.forename: F R
authors.initials: FR
authors.lastname: Appelbaum
authors.forename: P
authors.initials: P
authors.lastname: Guardiola
authors.forename: N
authors.initials: N
authors.lastname: Callander
authors.forename: C
authors.initials: C
authors.lastname: Freytes
authors.forename: Y
authors.initials: Y
authors.lastname: Gazitt
authors.forename: B
authors.initials: B
authors.lastname: Razvillas
authors.forename: H J
authors.initials: HJ
authors.lastname: Deeg
mesh_terms: D000368:Aged; D002066:Busulfan / Q000008:administration & dosage / Q000633:toxicity; D005500:Follow-Up Studies; D006085:Graft Survival; D016179:Granulocyte Colony-Stimulating Factor / Q000008:administration & dosage / Q000633:toxicity; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods / Q000401:mortality / Q000592:standards*; D006801:Humans; D008875:Middle Aged; D010865:Pilot Projects; D055728:Primary Myelofibrosis / Q000150:complications / Q000188:drug therapy / Q000401:mortality / Q000473:pathology / Q000628:therapy*; D013163:Splenomegaly / Q000139:chemically induced / Q000517:prevention & control; D015996:Survival Rate; D019172:Transplantation Conditioning / Q000379:methods / Q000592:standards*; D014182:Transplantation, Autologous / Q000379:methods / Q000592:standards; D016896:Treatment Outcome
chemical_list: D016179:Granulocyte Colony-Stimulating Factor; D002066:Busulfan