Hematopoietic cell transplantation as curative therapy for idiopathic myelofibrosis, advanced polycythemia vera, and essential thrombocythemia.
A total of 104 patients, aged 18 to 70 years, with a diagnosis of chronic idiopathic myelofibrosis (CIMF), polycythemia vera (PV), or essential thrombocythemia (ET) with marrow fibrosis were transplanted from allogeneic (56 related and 45 unrelated) or syngeneic (n = 3) donors. Busulfan (BU) or total body irradiation (TBI)-based myeloablative conditioning regimens were used in 95 patients, and a nonmyeloablative regimen of fludarabine plus TBI was used in 9 patients. The source of stem cells was bone marrow in 43 patients and peripheral blood in 61 patients. A total of 63 patients were alive at a follow-up of 1.3-15.2 years (median, 5.3 years), for an estimated 7-year actuarial survival rate of 61%. Eleven patients had recurrent/persistent disease, of whom 8 died. Nonrelapse mortality was 34% at 5 years. Patients conditioned with targeted BU (plasma levels 800-900 ng/mL) plus cyclophosphamide (tBUCY) had a higher probability of survival (68%) than other patients. Dupriez score, platelet count, patient age, and comorbidity score were statistically significantly associated with mortality in univariate models. In a multivariable regression model, use of tBUCY (P = .03), high platelet count at transplantation (P = .01 for PV/ET; P = .39 for other diagnoses), younger patient age (P = .04), and decreased comorbidity score (P = .03) remained statistically significant for improved survival. Our findings show that hematopoietic cell transplantation offers potentially curative treatment for patients with ICMF, PV, or ET.
Kerbauy
Daniella M B
DM
Fred Hutchinson Cancer Research Center and the University of Washington School of Medicine, Seattle, Washington 98109-1024, USA.
Gooley
Theodore A
TA
Sale
George E
GE
Flowers
Mary E D
ME
Doney
Kristine C
KC
Georges
George E
GE
Greene
Joanne E
JE
Linenberger
Michael
M
Petersdorf
Effie
E
Sandmaier
Brenda M
BM
Scott
Bart L
BL
Sorror
Mohamed
M
Stirewalt
Derek L
DL
Stewart
F Marc
FM
Witherspoon
Robert P
RP
Storb
Rainer
R
Appelbaum
Frederick R
FR
Deeg
H Joachim
HJ
D000293:Adolescent; D000328:Adult; D000368:Aged; D016026:Bone Marrow Transplantation; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods* / Q000401:mortality; D006801:Humans; D008297:Male; D008875:Middle Aged; D015233:Models, Statistical; D036102:Peripheral Blood Stem Cell Transplantation; D011087:Polycythemia Vera / Q000401:mortality / Q000628:therapy*; D055728:Primary Myelofibrosis / Q000401:mortality / Q000628:therapy*; D011379:Prognosis; D015996:Survival Rate; D013920:Thrombocythemia, Essential / Q000401:mortality / Q000628:therapy*; D019172:Transplantation Conditioning / Q000379:methods; D014184:Transplantation, Homologous; D014185:Transplantation, Isogeneic