Outcome of Allogeneic Hematopoietic Stem Cell Transplantation for Patients with Chronic and Advanced Phase Myelofibrosis.
Myelofibrosis (MF) is a chronic progressive hematologic malignancy with a median overall survival (OS) of approximately 6 years. Allogeneic hematopoietic stem cell transplantation (HSCT) is the sole treatment approach that offers curative potential. The use of reduced-intensity conditioning regimens has expanded the application of HSCT to patients with MF up to age 70 years. Recent retrospective and prospective reports have suggested worse HSCT outcomes for patients with MF receiving an unrelated donor graft compared with those receiving a related donor graft. To identify patient- and HSCT-specific variables influencing outcomes, we conducted a retrospective analysis of 42 patients with chronic and advanced-phase MF who underwent HSCT at our institution. For this cohort, at a median follow-up of 43 months, progression-free survival (PFS) was 15 months and OS was 25 months. In multivariable analysis, the sole clinical variable that negatively influenced outcome was the use of an unrelated donor, with a median PFS and OS both of 11 months versus not yet reached in patients receiving a related donor graft. At 2 years, OS was 38% (95% confidence interval [CI], 20%-56%) and nonrelapse mortality (NRM) was 53% (95% CI, 36%-78%) in the unrelated donor graft group, compared with 75% (95% CI, 46%-90%) and 21% (95% CI, 9%-47%) in the related donor graft group. There was no difference in the rates of grade III-IV acute graft-versus-host disease between the unrelated and related donor groups (38% versus 38%). Despite a more aggressive disease state, 2-year PFS and OS were both 42% (95% CI, 15%-67%) in patients with myeloproliferative neoplasm-blast phase undergoing HSCT. Graft failure rate was higher in patients receiving a mismatched donor graft compared with those receiving a matched donor graft (60% versus 13%; P = .0398). Retransplantation of patients with graft failure resulted in long-term survival. Baseline splenomegaly did not affect transplantation outcomes. Given the particularly poor outcomes seen in the unrelated donor cohort here and elsewhere, a formal exploration of alternative hematopoietic stem cell sources is warranted.
Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, New York.
Alla
A
Keyzner
Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, New York.
Sarah
S
Han
Department of Medicine, Icahn School of Medicine at Mount Sinai, New York, New York.
Samantha
S
Shapiro
Department of Population Health Science and Policy, TCI Biostatistics Shared Resource Facility, Icahn School of Medicine, New York, New York.
Erin
E
Moshier
Department of Medicine, Icahn School of Medicine at Mount Sinai, New York, New York.
Emily
E
Schorr
Department of Pathology, Icahn School of Medicine at Mount Sinai, New York, New York.
Bruce
B
Petersen
Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, New York; Department of Pathology, Icahn School of Medicine at Mount Sinai, New York, New York.
Vesna
V
Najfeld
Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, New York.
Marina
M
Kremyanskaya
Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, New York.
Luis
L
Isola
Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, New York.
Ronald
R
Hoffman
Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, New York. Electronic address: john.mascarenhas@mssm.edu.
John
J
Mascarenhas
D000328:Adult; D000368:Aged; D002908:Chronic Disease; D005260:Female; D006086:Graft vs Host Disease / Q000401:mortality; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods* / Q000401:mortality; D006648:Histocompatibility; D006801:Humans; D008297:Male; D008875:Middle Aged; D055728:Primary Myelofibrosis / Q000401:mortality / Q000628:therapy*; D019233:Retreatment / Q000401:mortality; D012189:Retrospective Studies; D016019:Survival Analysis; D019172:Transplantation Conditioning / Q000379:methods; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D061349:Unrelated Donors
Blast phase; Myelofibrosis; Splenomegaly; Transplantation