title: Allogeneic hematopoietic stem cell transplantation in patients with polycythemia vera or essential thrombocythemia transformed to myelofibrosis or acute myeloid leukemia: a report from the MPN Subcommittee of the Chronic Malignancies Working Party of the European Group for Blood and Marrow Transplantation.
abstract: The clinical course of polycythemia vera and essential thrombocythemia is potentially associated with long-term severe complications, such as evolution to myelofibrosis or acute myeloid leukemia. Allogeneic stem cell transplantation is currently the only potentially curative treatment for advanced polycythemia vera or essential thrombocythemia. We analyzed 250 consecutive patients with an initial diagnosis of polycythemia vera (n=120) or essential thrombocythemia (n=130), who underwent transplantation due to progression to myelofibrosis (n=193) or acute myeloid leukemia (n=57) and who were reported to the European Group for Blood and Marrow Transplantation registry between 1994 and 2010. Their median age was 56 years (range, 22-75) and in 52% of cases the interval between diagnosis and transplantation was 10 years or more. With a median follow-up from transplantation of 13 months, the 3-year overall survival rate and relapse incidence were 55% and 32%, respectively. In univariate analysis, the main parameters that negatively affected post-transplantation outcomes were older age (>55 years), a diagnosis at transplant of acute myeloid leukemia and the use of an unrelated donor. The overall 3-year cumulative incidence of non-relapse mortality was 28%, but was significantly higher in older patients than in younger ones (>55 years, 35% versus 20%, P=0.032), in those transplanted from an unrelated donor rather than a related donor (34% versus 18%, P=0.034) and in patients with a diagnosis of acute myeloid leukemia compared to myelofibrosis (29% versus 27%, P=0.045). This large retrospective study confirms that transplantation is potentially curative for patients with end-stage polycythemia vera/essential thrombocythemia progressing to myelofibrosis or acute myeloid leukemia. Relapse and non-relapse mortality remain unsolved problems for which innovative treatment approaches need to be assessed.
authors.affiliation: arambaldi@hpg23.it.
authors.forename: Federico
authors.initials: F
authors.lastname: Lussana
authors.forename: Alessandro
authors.initials: A
authors.lastname: Rambaldi
authors.forename: Maria Chiara
authors.initials: MC
authors.lastname: Finazzi
authors.forename: Anja
authors.initials: A
authors.lastname: van Biezen
authors.forename: Marijke
authors.initials: M
authors.lastname: Scholten
authors.forename: Elena
authors.initials: E
authors.lastname: Oldani
authors.forename: Alessandra
authors.initials: A
authors.lastname: Carobbio
authors.forename: Simona
authors.initials: S
authors.lastname: Iacobelli
authors.forename: Jurgen
authors.initials: J
authors.lastname: Finke
authors.forename: Arnon
authors.initials: A
authors.lastname: Nagler
authors.forename: Liisa
authors.initials: L
authors.lastname: Volin
authors.forename: Thierry
authors.initials: T
authors.lastname: Lamy
authors.forename: Renate
authors.initials: R
authors.lastname: Arnold
authors.forename: Mohamad
authors.initials: M
authors.lastname: Mohty
authors.forename: Mauricette
authors.initials: M
authors.lastname: Michallet
authors.forename: Theo
authors.initials: T
authors.lastname: de Witte
authors.forename: Eduardo
authors.initials: E
authors.lastname: Olavarria
authors.forename: Nicolaus
authors.initials: N
authors.lastname: Kröger
mesh_terms: D000328:Adult; D000368:Aged; D002423:Cause of Death; D005260:Female; D005500:Follow-Up Studies; D006086:Graft vs Host Disease / Q000209:etiology; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006801:Humans; D015470:Leukemia, Myeloid, Acute / Q000175:diagnosis / Q000209:etiology* / Q000401:mortality / Q000628:therapy*; D008297:Male; D008875:Middle Aged; D011087:Polycythemia Vera / Q000150:complications*; D055728:Primary Myelofibrosis / Q000175:diagnosis / Q000209:etiology* / Q000401:mortality / Q000628:therapy*; D012008:Recurrence; D013920:Thrombocythemia, Essential / Q000150:complications*; D014019:Tissue Donors; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D055815:Young Adult