Usama Gergis1, Emil Kuriakose2, Tsiporah Shore2, Sebastian Mayer2, Tomer Mark2, Roger Pearse2, Michael Schuster2, Eric Feldman2, Gail Roboz2, Ellen Ritchie2, Joseph Scandura2, Hanhan Wang3, Xi Kathy Zhou3, Richard T Silver2, Koen van Besien2. 1. 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. 2. Division of Hematology and Medical Oncology, Weill Cornell Medical College, New York, NY; New York-Presbyterian Hospital, Weill Cornell Medical Center, New York, NY. 3. 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.
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. PATIENTS AND 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. CONCLUSION: We confirm curative potential, but high NRM of allogeneic transplant for advanced MF.
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. PATIENTS AND 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. CONCLUSION: We confirm curative potential, but high NRM of allogeneic transplant for advanced MF.
Authors: Uday Popat; Rohtesh S Mehta; Roland Bassett; Piyanuch Kongtim; Julianne Chen; Amin M Alousi; Paolo Anderlini; Stefan Ciurea; Chitra Hosing; Roy Jones; Partow Kebriaei; Issa Khouri; Richard Lindsay; Yago Nieto; Amanda Olson; Betul Oran; Muzaffar H Qazilbash; Gabriela Rondon; Elizabeth J Shpall; Srdan Verstovsek; Borje S Andersson; Richard E Champlin Journal: Biol Blood Marrow Transplant Date: 2020-05-11 Impact factor: 5.742