title: Allogeneic Transplantation for Patients With Advanced Myelofibrosis: Splenomegaly and High Serum LDH are Adverse Risk Factors for Successful Engraftment.
abstract: BACKGROUND
  Thirty consecutive patients underwent hematopoietic stem cell transplantation for myelofibrosis (MF) at our institution. The median age at the time of transplant was 49 (range, 18-68) years, 74% of patients had advanced Dynamic International Prognostic Scoring System (DIPSS) scores, and 83% received reduced-intensity conditioning.
  
  
  METHODS
  With a long follow-up of our patients, we analyzed disease and transplant variables that contributed to engraftment and outcomes.
  
  
  RESULTS
  Neutrophil engraftment was achieved in 27 patients (90%) at a median time of 15 (range, 10-44) days, and 19 patients (63%) achieved platelet recovery at a median time of 18 (range, 8-100) days. Splenomegaly was associated with poor neutrophil engraftment (subdistributional hazard ratio [SHR], 0.42; 95% confidence interval [CI], 0.21-0.83; P = .01) and platelet engraftment (SHR, 0.18; 95% CI, 0.07-0.48; P < .001). Increased levels of lactate dehydrogenase (LDH) was associated with poor platelet engraftment (SHR, 0.39; 95% CI, 0.16-0.94; P = .04). The median follow-up for surviving patients was 49 (range, 3-155) months. The 1-year cumulative incidence of nonrelapse mortality (NRM) and relapse were respectively, 57% (95% CI, 29%-76%) and 25% (95% CI, 7%-48%). Increased levels of LDH was associated with high NRM (SHR, 2.82; 95% CI, 1.08-7.35; P = .03). The 4-year overall survival (OS) and relapse-free survival (RFS) were 44% (95% CI, 29%-67%) and 37% (95% CI, 23%-61%), respectively. In the multivariable model, splenomegaly and Eastern Cooperative Oncology Group (ECOG) performance status (PS) > 1 were associated with worse OS (hazard ratio [HR], 5.40; 95% CI, 1.19-24.56); P = .03) and RFS (HR, 3.78; 95% CI, 1.01-14.06; P < .05), respectively. ECOG PS > 1 was also associated with worse RFS (HR, 5.00; 95% CI, 1.31-19.14; P = .02). In this patient group with advanced disease, DIPPS score, Lille score, Janus-Associated Kinase V617F (JAK2 V617F) mutation status, and donor type did not predict transplant outcome.
  
  
  CONCLUSIONS
  We confirm curative potential, but high NRM of allogeneic transplant for advanced MF.
authors.affiliation: Division of Hematology and Medical Oncology, Weill Cornell Medical College, New York, NY; New York-Presbyterian Hospital, Weill Cornell Medical Center, New York, NY. Electronic address: usg2001@med.cornell.edu.
authors.forename: Usama
authors.initials: U
authors.lastname: Gergis
authors.affiliation: Division of Hematology and Medical Oncology, Weill Cornell Medical College, New York, NY; New York-Presbyterian Hospital, Weill Cornell Medical Center, New York, NY.
authors.forename: Emil
authors.initials: E
authors.lastname: Kuriakose
authors.affiliation: Division of Hematology and Medical Oncology, Weill Cornell Medical College, New York, NY; New York-Presbyterian Hospital, Weill Cornell Medical Center, New York, NY.
authors.forename: Tsiporah
authors.initials: T
authors.lastname: Shore
authors.affiliation: Division of Hematology and Medical Oncology, Weill Cornell Medical College, New York, NY; New York-Presbyterian Hospital, Weill Cornell Medical Center, New York, NY.
authors.forename: Sebastian
authors.initials: S
authors.lastname: Mayer
authors.affiliation: Division of Hematology and Medical Oncology, Weill Cornell Medical College, New York, NY; New York-Presbyterian Hospital, Weill Cornell Medical Center, New York, NY.
authors.forename: Tomer
authors.initials: T
authors.lastname: Mark
authors.affiliation: Division of Hematology and Medical Oncology, Weill Cornell Medical College, New York, NY; New York-Presbyterian Hospital, Weill Cornell Medical Center, New York, NY.
authors.forename: Roger
authors.initials: R
authors.lastname: Pearse
authors.affiliation: Division of Hematology and Medical Oncology, Weill Cornell Medical College, New York, NY; New York-Presbyterian Hospital, Weill Cornell Medical Center, New York, NY.
authors.forename: Michael
authors.initials: M
authors.lastname: Schuster
authors.affiliation: Division of Hematology and Medical Oncology, Weill Cornell Medical College, New York, NY; New York-Presbyterian Hospital, Weill Cornell Medical Center, New York, NY.
authors.forename: Eric
authors.initials: E
authors.lastname: Feldman
authors.affiliation: Division of Hematology and Medical Oncology, Weill Cornell Medical College, New York, NY; New York-Presbyterian Hospital, Weill Cornell Medical Center, New York, NY.
authors.forename: Gail
authors.initials: G
authors.lastname: Roboz
authors.affiliation: Division of Hematology and Medical Oncology, Weill Cornell Medical College, New York, NY; New York-Presbyterian Hospital, Weill Cornell Medical Center, New York, NY.
authors.forename: Ellen
authors.initials: E
authors.lastname: Ritchie
authors.affiliation: Division of Hematology and Medical Oncology, Weill Cornell Medical College, New York, NY; New York-Presbyterian Hospital, Weill Cornell Medical Center, New York, NY.
authors.forename: Joseph
authors.initials: J
authors.lastname: Scandura
authors.affiliation: New York-Presbyterian Hospital, Weill Cornell Medical Center, New York, NY; Division of Biostatistics, Department of Healthcare Policy and Research, Weill Cornell Medical College, New York, NY.
authors.forename: Hanhan
authors.initials: H
authors.lastname: Wang
authors.affiliation: New York-Presbyterian Hospital, Weill Cornell Medical Center, New York, NY; Division of Biostatistics, Department of Healthcare Policy and Research, Weill Cornell Medical College, New York, NY.
authors.forename: Xi Kathy
authors.initials: XK
authors.lastname: Zhou
authors.affiliation: Division of Hematology and Medical Oncology, Weill Cornell Medical College, New York, NY; New York-Presbyterian Hospital, Weill Cornell Medical Center, New York, NY.
authors.forename: Richard T
authors.initials: RT
authors.lastname: Silver
authors.affiliation: Division of Hematology and Medical Oncology, Weill Cornell Medical College, New York, NY; New York-Presbyterian Hospital, Weill Cornell Medical Center, New York, NY.
authors.forename: Koen
authors.initials: K
authors.lastname: van Besien
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D015415:Biomarkers; D005260:Female; D005500:Follow-Up Studies; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000209:etiology; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006801:Humans; D053614:Janus Kinase 2 / Q000235:genetics; D045022:Lactate Dehydrogenases / Q000097:blood; D008297:Male; D008875:Middle Aged; D009026:Mortality; D055728:Primary Myelofibrosis / Q000175:diagnosis* / Q000401:mortality / Q000628:therapy*; D011379:Prognosis; D012307:Risk Factors; D013163:Splenomegaly; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D015415:Biomarkers; D045022:Lactate Dehydrogenases; D053614:Janus Kinase 2
keywords: BMT for myelofibrosis; LDH in myelofibrosis; Myelofibrosis; Splenomegaly in myelofibrosis