title: Allogeneic hematopoietic cell transplantation for advanced polycythemia vera and essential thrombocythemia.
abstract: Allogeneic hematopoietic cell transplantation (HCT) is curative for selected patients with advanced essential thrombocythemia (ET) or polycythemia vera (PV). From 1990 to 2007, 75 patients with ET (median age 49 years) and 42 patients with PV (median age 53 years) underwent transplantations at the Fred Hutchinson Cancer Research Center (FHCRC; n = 43) or at other Center for International Blood and Marrow Transplant Research (CIBMTR) centers (n = 74). Thirty-eight percent of the patients had splenomegaly and 28% had a prior splenectomy. Most patients (69% for ET and 67% for PV) received a myeloablative (MA) conditioning regimen. Cumulative incidence of neutrophil engraftment at 28 days was 88% for ET patients and 90% for PV patients. Acute graft-versus-host disease (aGVHD) grades II to IV occurred in 57% and 50% of ET and PV patients, respectively. The 1-year treatment-related mortality (TRM) was 27% for ET and 22% for PV. The 5-year cumulative incidence of relapse was 13% for ET and 30% for PV. Five-year survival/progression-free survival (PFS) was 55%/47% and 71%/48% for ET and PV, respectively. Patients without splenomegaly had faster neutrophil and platelet engraftment, but there were no differences in TRM, survival, or PFS. Presence of myelofibrosis (MF) did not affect engraftment or TRM. Over 45% of the patients who undergo transplantations for ET and PV experience long-term PFS.
authors.affiliation: Division of Hematology/Oncology, Massachusetts General Hospital, Boston, Massachusetts 02214, USA. kballen@partners.org
authors.forename: Karen K
authors.initials: KK
authors.lastname: Ballen
authors.forename: Ann E
authors.initials: AE
authors.lastname: Woolfrey
authors.forename: Xiaochun
authors.initials: X
authors.lastname: Zhu
authors.forename: Kwang Woo
authors.initials: KW
authors.lastname: Ahn
authors.forename: Baldeep
authors.initials: B
authors.lastname: Wirk
authors.forename: Mukta
authors.initials: M
authors.lastname: Arora
authors.forename: Biju
authors.initials: B
authors.lastname: George
authors.forename: Bipin N
authors.initials: BN
authors.lastname: Savani
authors.forename: Brian
authors.initials: B
authors.lastname: Bolwell
authors.forename: David L
authors.initials: DL
authors.lastname: Porter
authors.forename: Ed
authors.initials: E
authors.lastname: Copelan
authors.forename: Gregory
authors.initials: G
authors.lastname: Hale
authors.forename: Harry C
authors.initials: HC
authors.lastname: Schouten
authors.forename: Ian
authors.initials: I
authors.lastname: Lewis
authors.forename: Jean Yves
authors.initials: JY
authors.lastname: Cahn
authors.forename: Joerg
authors.initials: J
authors.lastname: Halter
authors.forename: Jorge
authors.initials: J
authors.lastname: Cortes
authors.forename: Matt E
authors.initials: ME
authors.lastname: Kalaycio
authors.forename: Joseph
authors.initials: J
authors.lastname: Antin
authors.forename: Mahmoud D
authors.initials: MD
authors.lastname: Aljurf
authors.forename: Matthew H
authors.initials: MH
authors.lastname: Carabasi
authors.forename: Mehdi
authors.initials: M
authors.lastname: Hamadani
authors.forename: Philip
authors.initials: P
authors.lastname: McCarthy
authors.forename: Steven
authors.initials: S
authors.lastname: Pavletic
authors.forename: Vikas
authors.initials: V
authors.lastname: Gupta
authors.forename: H Joachim
authors.initials: HJ
authors.lastname: Deeg
authors.forename: Richard T
authors.initials: RT
authors.lastname: Maziarz
authors.forename: Mary M
authors.initials: MM
authors.lastname: Horowitz
authors.forename: Wael
authors.initials: W
authors.lastname: Saber
mesh_terms: D000208:Acute Disease; D000328:Adult; D005260:Female; D006086:Graft vs Host Disease / Q000276:immunology / Q000517:prevention & control*; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D008137:Longitudinal Studies; D008297:Male; D008875:Middle Aged; D019653:Myeloablative Agonists / Q000627:therapeutic use; D009504:Neutrophils / Q000276:immunology; D011087:Polycythemia Vera / Q000150:complications / Q000276:immunology / Q000401:mortality / Q000628:therapy*; D012008:Recurrence; D013156:Splenectomy; D013163:Splenomegaly / Q000150:complications / Q000276:immunology / Q000401:mortality / Q000628:therapy*; D016019:Survival Analysis; D013920:Thrombocythemia, Essential / Q000150:complications / Q000276:immunology / Q000401:mortality / Q000628:therapy*; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous
chemical_list: D019653:Myeloablative Agonists