Despite numerous studies assessing potential improvements in treating myelofibrosis, allogeneic hematopoietic cell transplantation (HCT) remains the only treatment option with the potential for a cure. However, HCT brings with it risks of morbidity and mortality, explains Tania Jain, MBBS. “It would be helpful to understand which patients are at an increased risk of relapse or death,” she adds. For a study published in the Blood Cancer Journal, Jain and colleagues sought to determine the factors that influence post-transplantation outcomes to identify patients, early in the course of the HCT, who are at high risk for morbidity and mortality.
The researchers studied factors at day +100 post-transplantation that had a potential of predicting worse outcomes. The team conducted a retrospective chart review to gather baseline data on patients, disease- and HCT-related data, day 100 characteristics, and post-HCT outcomes. Information collected included molecular mutation status, bone marrow fibrosis, spleen size, donor chimerism, presence of any grade of acute graft versus host disease (GVHD), and red blood cell (RBC) and platelet transfusion dependence. Data were then used to understand differences in survival by calculating individuals’ risk factor counts. RBC transfusion dependence (HR, 9.02), platelet transfusion dependence (HR, 8.17), 100% donor chimerism in CD33+ cells (HR, 0.21), unfavorable molecular status (HR, 4.41), normal spleen size (HR, 0.42), grade 2 or greater bone marrow fibrosis (vs. grade ≤ 1; HR, 2.7) and poor graft function (HR 2.6) at day 100 were statistically significantly associated with relapse-free survival. RBC transfusion dependence and unfavorable molecular status (defined as less than 100% donor chimerism in CD33+ cells or presence of detectable driver mutation on next generation sequencing testing) at day +100 were statistically significantly associated with inferior relapse-free survival, in the multivariate analysis. “Interestingly, the degree of marrow fibrosis at day +100 was not significantly correlated with outcomes,” adds Dr. Jain.
“This is only a first step that has helped us identify high-risk patients,” notes Dr. Jain. “Additional studies analyzing post-transplantation strategies could help address this risk in patients with HCT so that we can eventually improve outcomes of patients undergoing allogeneic stem cell transplantation for myelofibrosis.”