Contributions
Abstract: PB2280
Type: Publication Only
Background
The main causes of morbidity and mortality in patients with myeloproliferative neoplasms are thrombohemorrhagic events in polycycthemia vera (PV) and essential thrombocythemia (ET) and transformation to acute leukemia (most frequent in primary myelofibrosis – PMF) (Frederiksen et al. Blood 2011;118:6515). The introduction of tyrosine kinase inhibitors (TKIs) has changed the clinical course of chronic myeloid leukemia (CML) from a slowly progressive disease into a chronic one with near-normal life expectancy (Bower et al. JCO 2016;34:2851). Moreover the cardiovascular complications of TKIs have raised concerns about their effect on hemostasis (Moslehi et al. JCO 2015;33:4210).
Aims
The aim of the study is to assess the incidence of the main causes of morbidity and mortality collectively in patients with MPNs.
Methods
A retrospective study was conducted in patients with MPNs in a tertiary center. Mortality rates were calculated, as well as incidence rates for thrombotic and hemorrhagic events, transformation to acute leukemia and occurrence of solid tumors.
Results
A total of 224 patients, with median age at diagnosis 64.5 years (range 17-92) were included in the analysis. The initial diagnosis was CML (13%), ET (51%), PV (28%), PMF (5%) and myeloproliferative/myelodysplastic syndrom in 2%. Mean duration of follow-up was 8 years. In 20% of patients the presenting symptom was a thrombotic episode (arterial, venous or splenic infarct).
During the follow-up, 83 events of interest were recorded. In the whole cohort, the incidence rate (in events per 100 patients per 10 years) of thrombosis was 13.42, hemorrhage 1.68, transformation to another hematologic malignancy 11.74, occurrence of solid tumor 5.59 and other events 13.98. Other events for which patients received medical attention included atypical chest pain, joint pain, arrythmias, pleural effusion and infections. Incidence of major events per diagnosis are presented in table 1. Cumulative incidence of thrombotic complications is illustrated in figure 1.
CML | ET | PV | PMF | |
Thrombosis | 2.39 (0.9-6.3) | 1.27 (0.75-2.13) | 1.29 (0.58-2.85) | 0 |
Hemorrhage | 0 | 0.27 (0.09-0.84) | 0 | 0 |
Hematological malignancy | 0.6 (0.08-4.2) | 0.82 (0.43-1.56) | 1.07 (0.45-2.57) | 2.21 (0.32-15.37) |
Solid tumor | 0.6 (0.08-4.2) | 0.36 (0.14-0.96) | 1.07 (0.45-2.57) | 0 |
Table 1. Incidence rate (per 100 patient-years) and 95% confidence interval (CI) for the major events during follow up for different myeloproliferative neoplasms.
All-cause mortality rates in deaths per 100 patient-years (95% CI) were for CML 1.8 (0.6-5.5), ET 0.9 (0.5-1.7), PV 1.9 (1-3.7), PMF 6.6 (2.2-19.8). Transformation to acute leukemia accounted for 46.7% of all deaths, solid tumors for 16.7% and other causes (cardiac death, sepsis) for 33.3% of deaths. All patients with atypical CML or other myeloproliferative/ myelodysplastic syndrom died at a median of 16.4 months from diagnosis, while their disease had transformed to AML at a median of 14 months from initial diagnosis.
Conclusion
Thrombotic complications account for the main burden of morbidity not only in patients with ET and PV, but also for CML patients on TKIs. Transformation to acute leukemia is the leading cause of death. It is crucial that future studies incorporate cardiovascular risk factors and estimate the effect of therapeutic interventions in both the thrombotic risk and the progression free survival.
Session topic: 16. Myeloproliferative neoplasms - Clinical
Keyword(s): Chronic myeloid leukemia, Mortality, Myeloproliferative disorder, Thrombosis
Abstract: PB2280
Type: Publication Only
Background
The main causes of morbidity and mortality in patients with myeloproliferative neoplasms are thrombohemorrhagic events in polycycthemia vera (PV) and essential thrombocythemia (ET) and transformation to acute leukemia (most frequent in primary myelofibrosis – PMF) (Frederiksen et al. Blood 2011;118:6515). The introduction of tyrosine kinase inhibitors (TKIs) has changed the clinical course of chronic myeloid leukemia (CML) from a slowly progressive disease into a chronic one with near-normal life expectancy (Bower et al. JCO 2016;34:2851). Moreover the cardiovascular complications of TKIs have raised concerns about their effect on hemostasis (Moslehi et al. JCO 2015;33:4210).
Aims
The aim of the study is to assess the incidence of the main causes of morbidity and mortality collectively in patients with MPNs.
Methods
A retrospective study was conducted in patients with MPNs in a tertiary center. Mortality rates were calculated, as well as incidence rates for thrombotic and hemorrhagic events, transformation to acute leukemia and occurrence of solid tumors.
Results
A total of 224 patients, with median age at diagnosis 64.5 years (range 17-92) were included in the analysis. The initial diagnosis was CML (13%), ET (51%), PV (28%), PMF (5%) and myeloproliferative/myelodysplastic syndrom in 2%. Mean duration of follow-up was 8 years. In 20% of patients the presenting symptom was a thrombotic episode (arterial, venous or splenic infarct).
During the follow-up, 83 events of interest were recorded. In the whole cohort, the incidence rate (in events per 100 patients per 10 years) of thrombosis was 13.42, hemorrhage 1.68, transformation to another hematologic malignancy 11.74, occurrence of solid tumor 5.59 and other events 13.98. Other events for which patients received medical attention included atypical chest pain, joint pain, arrythmias, pleural effusion and infections. Incidence of major events per diagnosis are presented in table 1. Cumulative incidence of thrombotic complications is illustrated in figure 1.
CML | ET | PV | PMF | |
Thrombosis | 2.39 (0.9-6.3) | 1.27 (0.75-2.13) | 1.29 (0.58-2.85) | 0 |
Hemorrhage | 0 | 0.27 (0.09-0.84) | 0 | 0 |
Hematological malignancy | 0.6 (0.08-4.2) | 0.82 (0.43-1.56) | 1.07 (0.45-2.57) | 2.21 (0.32-15.37) |
Solid tumor | 0.6 (0.08-4.2) | 0.36 (0.14-0.96) | 1.07 (0.45-2.57) | 0 |
Table 1. Incidence rate (per 100 patient-years) and 95% confidence interval (CI) for the major events during follow up for different myeloproliferative neoplasms.
All-cause mortality rates in deaths per 100 patient-years (95% CI) were for CML 1.8 (0.6-5.5), ET 0.9 (0.5-1.7), PV 1.9 (1-3.7), PMF 6.6 (2.2-19.8). Transformation to acute leukemia accounted for 46.7% of all deaths, solid tumors for 16.7% and other causes (cardiac death, sepsis) for 33.3% of deaths. All patients with atypical CML or other myeloproliferative/ myelodysplastic syndrom died at a median of 16.4 months from diagnosis, while their disease had transformed to AML at a median of 14 months from initial diagnosis.
Conclusion
Thrombotic complications account for the main burden of morbidity not only in patients with ET and PV, but also for CML patients on TKIs. Transformation to acute leukemia is the leading cause of death. It is crucial that future studies incorporate cardiovascular risk factors and estimate the effect of therapeutic interventions in both the thrombotic risk and the progression free survival.
Session topic: 16. Myeloproliferative neoplasms - Clinical
Keyword(s): Chronic myeloid leukemia, Mortality, Myeloproliferative disorder, Thrombosis