Contributions
Abstract: PB1704
Type: Publication Only
Session title: Myeloproliferative neoplasms - Clinical
Background
Despite ruxolitinib is considered the standard of care for patients with myelofibrosis (PMF and post-ET/post-PV MF) in not allogeneic stem-cell transplant candidates with Intermediate or High-risk IPSS, is necessary to confirm in real live the clinical benefit, in spleen, symptomatic responses and overall survival (OS) outcomes, achieved in COMFORT-I and -II trials. In the other hand, nearly half of the patients develop grade 3/4 anemia or thrombocytopenia, that usually lead to a dose reduction, that can have a negative impact on its efficacy.
Aims
We conducted this study to evaluate the differences on clinical benefit of alternative rusolitinib dosing regime in real live.
Methods
6-year observational retrospective study of 53 patients with myelofibrosis (years 2013 -2019), 41 of them, not allogeneic stem-cell transplant candidates on ruxolitinib therapy, stratified by IPSS, DIPSS-PLUS and MIPSS 70+ v.2.0, and divided into 2 arms according to the maximum dose reached of ruxolitinib (A ≥ 15 mg BID and B <15 mg BID). We evaluated the rate of response according to IWG-MRT-2013 criteria, and we perform a survival analysis.
Results
41 not candidates for transplantation on Ruxolitinib therapy (median age 62 year; 11 PMF and 30 Post-ET and Post-PV). Group A ≥ 15 mg BID (N=19) and B <15 mg BID (N=22). With a median follow-up of 19 months (9,5-45), we observe a similar duration of treatment, IPSS and DIPSS-Plus score in both arms, although the MIPSS70+ high score was more frequent in B arm (66,7% vs. 15,4%; p=0.026). We did not observe statistically significant differences in spleen response (A 52,9% vs. B 25,0%; p=0.081), symptomatic responses (A 84,2% vs. B 63,6%; p=0.138) or 5-years OS (A 89.5% vs. B 75.1%; P=0.300), although the hematological improvement is more frequent in arm A (63.5% vs. 22.7%; p=0.009).
Conclusion
Despite the observed trend of lower spleen and symptomatic responses in arm B (ruxolitinib <15 mg BID), these are not statistically significant, and these do not translate into differences in OS in real live. High-risk MIPSS 70+ more frequently need dose reduction of Ruxolitinib, due to their baseline cytopenias, and probably this factor is the most important for evaluation of clinical benefit of Ruxolitinib in real live.
Keyword(s): Myelofibrosis, Ruxolitinib
Abstract: PB1704
Type: Publication Only
Session title: Myeloproliferative neoplasms - Clinical
Background
Despite ruxolitinib is considered the standard of care for patients with myelofibrosis (PMF and post-ET/post-PV MF) in not allogeneic stem-cell transplant candidates with Intermediate or High-risk IPSS, is necessary to confirm in real live the clinical benefit, in spleen, symptomatic responses and overall survival (OS) outcomes, achieved in COMFORT-I and -II trials. In the other hand, nearly half of the patients develop grade 3/4 anemia or thrombocytopenia, that usually lead to a dose reduction, that can have a negative impact on its efficacy.
Aims
We conducted this study to evaluate the differences on clinical benefit of alternative rusolitinib dosing regime in real live.
Methods
6-year observational retrospective study of 53 patients with myelofibrosis (years 2013 -2019), 41 of them, not allogeneic stem-cell transplant candidates on ruxolitinib therapy, stratified by IPSS, DIPSS-PLUS and MIPSS 70+ v.2.0, and divided into 2 arms according to the maximum dose reached of ruxolitinib (A ≥ 15 mg BID and B <15 mg BID). We evaluated the rate of response according to IWG-MRT-2013 criteria, and we perform a survival analysis.
Results
41 not candidates for transplantation on Ruxolitinib therapy (median age 62 year; 11 PMF and 30 Post-ET and Post-PV). Group A ≥ 15 mg BID (N=19) and B <15 mg BID (N=22). With a median follow-up of 19 months (9,5-45), we observe a similar duration of treatment, IPSS and DIPSS-Plus score in both arms, although the MIPSS70+ high score was more frequent in B arm (66,7% vs. 15,4%; p=0.026). We did not observe statistically significant differences in spleen response (A 52,9% vs. B 25,0%; p=0.081), symptomatic responses (A 84,2% vs. B 63,6%; p=0.138) or 5-years OS (A 89.5% vs. B 75.1%; P=0.300), although the hematological improvement is more frequent in arm A (63.5% vs. 22.7%; p=0.009).
Conclusion
Despite the observed trend of lower spleen and symptomatic responses in arm B (ruxolitinib <15 mg BID), these are not statistically significant, and these do not translate into differences in OS in real live. High-risk MIPSS 70+ more frequently need dose reduction of Ruxolitinib, due to their baseline cytopenias, and probably this factor is the most important for evaluation of clinical benefit of Ruxolitinib in real live.
Keyword(s): Myelofibrosis, Ruxolitinib