Contributions
Abstract: EP1121
Type: E-Poster Presentation
Session title: Myeloproliferative neoplasms - Clinical
Background
Ruxolitinib (Rx) is a Janus kinase (JAK) 1/JAK2 inhibitor with established efficacy in improving primary myelofibrosis (PMF)-related symptoms and survival. However, while aging is known to correlate with worse prognosis in PMF, real-life studies become essential to demonstrate the efficacy and safety of Rx in older populations.
Aims
To compare survival of elderly patients treated (Rx-t) versus non-treated (Rx-nt) with Rx.
Methods
Retrospective and unicentric analysis of records from elderly patients (age ≥65 years) diagnosed with PMF (WHO 2016) between January 2010 and December 2019.The Cox regression model and Kaplan-Meier method were used for the analysis of progression free survival (PFS) and overall survival (OS).
Results
We analysed a cohort of 70 patients, 70% males and with a median age of 74 years old (range, 65-92 years old). Thirteen patients (16.6%) had post-polycythemia vera (PV)/post-essential thrombocythemia (TE) MF. JAK2V617F mutation was identified in 71,4% of the patients, while 7,1% and 14.3% harboured a mutation in the MPL and CALR genes, respectively. Five patients (7.1%) were triple negative for those mutations.
About 87% of the patients required treatment in the course of the disease, 20% with ≥2 lines of therapy. Seventeen-percent were Rx-t. Rx-t patients were younger (69 versus 75 years old; p=0,011) and showed a higher incidence of MF post-PV/TE (50 versus 12%, p=0,002), compared to Rx-nt. The frequency of JAK2V617F mutation was equivalent in both groups (83 versus 69%; p=NS), however, the allele burden was higher in Rx-t patients (VAF 73 versus 33%; p=0,029). All Rx-t patients were at overt fibrotic stage (p=0,006). Blood tests at diagnosis demonstrated that the majority of the Rx-t presented with anaemia (Hb<12g/dL; p=0,034), however no significant differences were observed in leucocyte counts, LDH, circulating blasts or constitutional symptoms (CS), compared to Rx-nt. With no surprise, most patients selected for treatment with Rx presented with splenomegaly (92 versus 50%; p=0,008). There was no significant difference in the distribution through International Prognostic Scoring System (IPSS), dynamic IPSS (DIPSS) or DIPSS-plus, between Rx-t versus Rx-nt patients. PFS and OS in our cohort was estimated in 62,7 and 101,8 months, respectively.
In the univariate analysis, the variables age, anaemia, CS, circulating blasts, splenomegaly, IPSS and DIPSS intermediate-2/high-risk, were significantly associated with PFS; the same variables in addition to fibrosis grade ≥2, were related to OS. After Cox multivariate analysis and after adjusting for variables with impact on PFS and OS in the univariate analysis, we found that treatment with Rx improves PFS (HR 0,28, p=NS, IC95% 0,05-1,52) and OS (HR 0,05, p=0,009; IC95% 0,01-0,48).
Conclusion
In our cohort, Rx showed to have an independent beneficial impact in mortality of elderly patients with PMF, including patients with high risk IPSS and DIPSS score, therefore demonstrating that advanced age does not stand as a limitation for treatment with Rx.
Keyword(s): Elderly, Myelofibrosis, Myeloproliferative disorder, Ruxolitinib
Abstract: EP1121
Type: E-Poster Presentation
Session title: Myeloproliferative neoplasms - Clinical
Background
Ruxolitinib (Rx) is a Janus kinase (JAK) 1/JAK2 inhibitor with established efficacy in improving primary myelofibrosis (PMF)-related symptoms and survival. However, while aging is known to correlate with worse prognosis in PMF, real-life studies become essential to demonstrate the efficacy and safety of Rx in older populations.
Aims
To compare survival of elderly patients treated (Rx-t) versus non-treated (Rx-nt) with Rx.
Methods
Retrospective and unicentric analysis of records from elderly patients (age ≥65 years) diagnosed with PMF (WHO 2016) between January 2010 and December 2019.The Cox regression model and Kaplan-Meier method were used for the analysis of progression free survival (PFS) and overall survival (OS).
Results
We analysed a cohort of 70 patients, 70% males and with a median age of 74 years old (range, 65-92 years old). Thirteen patients (16.6%) had post-polycythemia vera (PV)/post-essential thrombocythemia (TE) MF. JAK2V617F mutation was identified in 71,4% of the patients, while 7,1% and 14.3% harboured a mutation in the MPL and CALR genes, respectively. Five patients (7.1%) were triple negative for those mutations.
About 87% of the patients required treatment in the course of the disease, 20% with ≥2 lines of therapy. Seventeen-percent were Rx-t. Rx-t patients were younger (69 versus 75 years old; p=0,011) and showed a higher incidence of MF post-PV/TE (50 versus 12%, p=0,002), compared to Rx-nt. The frequency of JAK2V617F mutation was equivalent in both groups (83 versus 69%; p=NS), however, the allele burden was higher in Rx-t patients (VAF 73 versus 33%; p=0,029). All Rx-t patients were at overt fibrotic stage (p=0,006). Blood tests at diagnosis demonstrated that the majority of the Rx-t presented with anaemia (Hb<12g/dL; p=0,034), however no significant differences were observed in leucocyte counts, LDH, circulating blasts or constitutional symptoms (CS), compared to Rx-nt. With no surprise, most patients selected for treatment with Rx presented with splenomegaly (92 versus 50%; p=0,008). There was no significant difference in the distribution through International Prognostic Scoring System (IPSS), dynamic IPSS (DIPSS) or DIPSS-plus, between Rx-t versus Rx-nt patients. PFS and OS in our cohort was estimated in 62,7 and 101,8 months, respectively.
In the univariate analysis, the variables age, anaemia, CS, circulating blasts, splenomegaly, IPSS and DIPSS intermediate-2/high-risk, were significantly associated with PFS; the same variables in addition to fibrosis grade ≥2, were related to OS. After Cox multivariate analysis and after adjusting for variables with impact on PFS and OS in the univariate analysis, we found that treatment with Rx improves PFS (HR 0,28, p=NS, IC95% 0,05-1,52) and OS (HR 0,05, p=0,009; IC95% 0,01-0,48).
Conclusion
In our cohort, Rx showed to have an independent beneficial impact in mortality of elderly patients with PMF, including patients with high risk IPSS and DIPSS score, therefore demonstrating that advanced age does not stand as a limitation for treatment with Rx.
Keyword(s): Elderly, Myelofibrosis, Myeloproliferative disorder, Ruxolitinib