Contributions
Abstract: EP1256
Type: E-Poster Presentation
Session title: Stem cell transplantation - Clinical
Background
Allogeneic hematopoietic cell transplantation (alloHCT) remains the only curative option of myelofibrosis. Despite intensified efforts to broaden alloHCT indications with better patient stratification, treating physicians are reluctant to refer patients for alloHCT in the era of JAK2 inihibitors.
Aims
We investigated the long-term safety and efficacy of alloHCT in patients with myelofibrosis
Methods
We retrospectively enrolled consecutive adult alloHCT recipients transplanted for myelofibrosis over the last two decades (2000-2020) at our JACIE-accredited center. We excluded patients transplanted with acute myeloid leukemia progression post myelofibrosis. Factors included in the analysis were: age, type of myelofibrosis (primary or secondary due to polycythemia vera/essential thrombocytosis), dynamic IPSS score, donor (sibling/unrelated), HLA matching, graft failure, acute (grade II-III) and chronic (according to NIH classification) GVHD, relapse, second alloHCT, bacterial, viral or fungal infections, spleen size and ruxolitinib administration before/after alloHCT.
Results
We studied 12 alloHCT recipients aged 53 (31-66) years, after a median of 42.8 (range 4.1-421) months from alloHCT. Median DIPSS was 3 (range 2-4). Seven patients suffered from primary myelofibrosis, while five from secondary. Eight were transplanted from a sibling donor, and 4 from an unrelated (2 matched and 2 mis-matched donors). All patients received peripheral blood stem cells graft, after myeloablative (5) or reduced intensity (7) conditioning regimens. Neutrophil engraftment was achieved in all patients (median 13, range 9-23 days), while platelet engraftment in 11/12 (median 16, range 10-146 days). Two patients developed graft failure and received second alloHCT with reduced intensity regimens.
Splenectomy was performed before alloHCT in 2 patients, and 7 months post-transplant in 1. Incidence of acute GVHD (grade 2-3) was 33% patients, while cumulative incidence of moderate chronic GVHD reached 58%. Two-year cumulative incidence of treatment-related mortality (TRM) was 34.6% Ruxolitinib was administered in patients transplanted after 2015: before alloHCT in 6/6 patients, and after alloHCT in 4/6 (2 for relapsed disease, 1 for splenomegaly and thrombocytopenia despite involved field radiation therapy, and 1 for moderate chronic GVHD). Patients that received ruxolitinib post-transplant did not suffer from TRM. Additionally, 2-year disease-free survival was 75% for patients that received ruxolitinib post-transplant, compared to 14.3% for those that did not (p=0.137, Figure). Importantly, 2-year overall survival was significantly improved in patients with post-transplant ruxolitinib (100% compared to 13.1%, p=0.014, Figure).
Conclusion
Our real-world cohort indicates that the use of ruxolitinib has optimized outcomes of alloHCT. Especially after alloHCT, our results suggest a significant benefit of a personalized approach in ruxolitinib administration. Given the safety of alloHCT, further studies are needed to guide this personalized approach.
Keyword(s): Allogeneic hematopoietic stem cell transplant, Myelofibrosis, Ruxolitinib
Abstract: EP1256
Type: E-Poster Presentation
Session title: Stem cell transplantation - Clinical
Background
Allogeneic hematopoietic cell transplantation (alloHCT) remains the only curative option of myelofibrosis. Despite intensified efforts to broaden alloHCT indications with better patient stratification, treating physicians are reluctant to refer patients for alloHCT in the era of JAK2 inihibitors.
Aims
We investigated the long-term safety and efficacy of alloHCT in patients with myelofibrosis
Methods
We retrospectively enrolled consecutive adult alloHCT recipients transplanted for myelofibrosis over the last two decades (2000-2020) at our JACIE-accredited center. We excluded patients transplanted with acute myeloid leukemia progression post myelofibrosis. Factors included in the analysis were: age, type of myelofibrosis (primary or secondary due to polycythemia vera/essential thrombocytosis), dynamic IPSS score, donor (sibling/unrelated), HLA matching, graft failure, acute (grade II-III) and chronic (according to NIH classification) GVHD, relapse, second alloHCT, bacterial, viral or fungal infections, spleen size and ruxolitinib administration before/after alloHCT.
Results
We studied 12 alloHCT recipients aged 53 (31-66) years, after a median of 42.8 (range 4.1-421) months from alloHCT. Median DIPSS was 3 (range 2-4). Seven patients suffered from primary myelofibrosis, while five from secondary. Eight were transplanted from a sibling donor, and 4 from an unrelated (2 matched and 2 mis-matched donors). All patients received peripheral blood stem cells graft, after myeloablative (5) or reduced intensity (7) conditioning regimens. Neutrophil engraftment was achieved in all patients (median 13, range 9-23 days), while platelet engraftment in 11/12 (median 16, range 10-146 days). Two patients developed graft failure and received second alloHCT with reduced intensity regimens.
Splenectomy was performed before alloHCT in 2 patients, and 7 months post-transplant in 1. Incidence of acute GVHD (grade 2-3) was 33% patients, while cumulative incidence of moderate chronic GVHD reached 58%. Two-year cumulative incidence of treatment-related mortality (TRM) was 34.6% Ruxolitinib was administered in patients transplanted after 2015: before alloHCT in 6/6 patients, and after alloHCT in 4/6 (2 for relapsed disease, 1 for splenomegaly and thrombocytopenia despite involved field radiation therapy, and 1 for moderate chronic GVHD). Patients that received ruxolitinib post-transplant did not suffer from TRM. Additionally, 2-year disease-free survival was 75% for patients that received ruxolitinib post-transplant, compared to 14.3% for those that did not (p=0.137, Figure). Importantly, 2-year overall survival was significantly improved in patients with post-transplant ruxolitinib (100% compared to 13.1%, p=0.014, Figure).
Conclusion
Our real-world cohort indicates that the use of ruxolitinib has optimized outcomes of alloHCT. Especially after alloHCT, our results suggest a significant benefit of a personalized approach in ruxolitinib administration. Given the safety of alloHCT, further studies are needed to guide this personalized approach.
Keyword(s): Allogeneic hematopoietic stem cell transplant, Myelofibrosis, Ruxolitinib