BASELINE CHARACTERISTICS AND RISK FACTORS FOR LEUKEMIA-FREE AND OVERALL SURVIVAL IN BULGARIAN PATIENTS WITH PRIMARY MYELOFIBROSIS ? A NATIONAL COHORT STUDY
(Abstract release date: 05/19/16)
EHA Library. Guenova M. 06/09/16; 134918; PB2018
Prof. Dr. Margarita Guenova
Contributions
Contributions
Abstract
Abstract: PB2018
Type: Publication Only
Background
Primary myelofibrosis (MF) is a rare form of BCR-ABL1(-) myeloproliferative neoplasm (MPN). Comprehensive research on the characteristic of this life-threatening condition has not been done in Bulgaria yet. Therefore, the Working Group of MPN of the Bulgarian Society of Hematology initiated a nationwide study.
Aims
To determine the prevalence of MF in the country, and to provide data on the baseline clinical and laboratory parameters and factors for leukemic transformation and overall survival (OS) of a national cohort of patients (pts) with the disease.
Methods
Data on 284 MF pts diagnosed and/or treated in 2014-2015 in 10 clinical centers covering the territory of the country with a population of 5242000>25-yrs of age were analysed. Major demographic, clinical and laboratory parameters, risk profiles according to the international prognostic system (IPSS) and the version for dynamic evaluation of risk (DIPSS and DIPSS-PLUS), and therapeutic approaches were evaluated at baseline as well as in regard to leukemia-free (LFS) and OS in 250 pts.
Results
A male/female ratio of 1.6:1 was determined. The mean age at diagnosis was 64.24 yrs (range 25-86 yrs). In 250 (88%) pts primary MF was diagnosed and in the other 34 (12%) secondary MF developed after another MPN. The most common symptoms were enlarged spleen (78%), anemia (58%), ≥1 constitutional symptoms (41%), thrombocytopenia (21%) and leukocytosis (20%). In 252 (91.2%) pts the diagnosis was confirmed with bone marrow histology and the severity of fibrosis was determined with silver stain in 77% of the cases. Molecular analysis was conducted in 179 (63%) pts and 113 (63.1%) were positive for JAK2 V617F mutation, including as expected 84% of secondary vs 60% of primary MF (p=.014). In primary MF, analysis revealed a significant association only between JAK2 mutation and platelet counts <100x109/L (p=.018) and transfusion dependence (p=.043). In 20 (7.4%) pts blast transformation was registered regardless of the primary or secondary nature of MF. Univariate analysis of parameters at the time of diagnosis revealed a significant association between inferior LFS and peripheral blood blasts >1% (p<.0001), leukocytosis >20x109/L (p<.0001), platelet counts <100x109/L (p=.001), hypercatabolic symptoms (p=.01), splenomegaly (p=.01), and treatment history with hydroxyurea (p=.03). Median OS was 6.2 yrs and as expected, it was significantly shorter in pts with leukaemic transformation (3.6 yrs vs 7.3 yrs, log rank test p=.002). According to the IPSS, DIPSS and DIPSS-PLUS pts were categorized predominantly in intermediate-high (IHR) and high risk (HR) groups: 68%>79%>87%, respectively. A significant association was confirmed between the OS and risk profiles (p<.0001). In total, 71% of pts required treatment (e.g. hydroxyurea, erythropoetin, androgen, interferon, corticosteroids, ruxolitinib). Splenectomy was performed in 4% of the pts, while RBC transfusions were indicated in 59%. Interestingly, therapy of any kind was significantly associated with better survival in all DIPSS-PLUS risk categories except for HR group who might be the candidates for novel therapies (p=.009 in low risk vs p=.002 in IHR groups vs p=.127 in HR).
Conclusion
The study provides, for the first time, nationwide data on the 5.4/100000 prevalence and basic features of Bulgarian pts with MF in regard to clinical and laboratory presentation as well as to prognostic parameters and OS.Contributions: LG, MG, GM, GB designed the study and contributed equally to this work. MG, YT conducted data analysis. DT, DT, GD, GT, LS, MZ, NP, SD, TD, VG, VS, YP performed research and contributed equally to this work.
Session topic: E-poster
Keyword(s): Myelofibrosis, Prevalence, Survival, Transformation
Type: Publication Only
Background
Primary myelofibrosis (MF) is a rare form of BCR-ABL1(-) myeloproliferative neoplasm (MPN). Comprehensive research on the characteristic of this life-threatening condition has not been done in Bulgaria yet. Therefore, the Working Group of MPN of the Bulgarian Society of Hematology initiated a nationwide study.
Aims
To determine the prevalence of MF in the country, and to provide data on the baseline clinical and laboratory parameters and factors for leukemic transformation and overall survival (OS) of a national cohort of patients (pts) with the disease.
Methods
Data on 284 MF pts diagnosed and/or treated in 2014-2015 in 10 clinical centers covering the territory of the country with a population of 5242000>25-yrs of age were analysed. Major demographic, clinical and laboratory parameters, risk profiles according to the international prognostic system (IPSS) and the version for dynamic evaluation of risk (DIPSS and DIPSS-PLUS), and therapeutic approaches were evaluated at baseline as well as in regard to leukemia-free (LFS) and OS in 250 pts.
Results
A male/female ratio of 1.6:1 was determined. The mean age at diagnosis was 64.24 yrs (range 25-86 yrs). In 250 (88%) pts primary MF was diagnosed and in the other 34 (12%) secondary MF developed after another MPN. The most common symptoms were enlarged spleen (78%), anemia (58%), ≥1 constitutional symptoms (41%), thrombocytopenia (21%) and leukocytosis (20%). In 252 (91.2%) pts the diagnosis was confirmed with bone marrow histology and the severity of fibrosis was determined with silver stain in 77% of the cases. Molecular analysis was conducted in 179 (63%) pts and 113 (63.1%) were positive for JAK2 V617F mutation, including as expected 84% of secondary vs 60% of primary MF (p=.014). In primary MF, analysis revealed a significant association only between JAK2 mutation and platelet counts <100x109/L (p=.018) and transfusion dependence (p=.043). In 20 (7.4%) pts blast transformation was registered regardless of the primary or secondary nature of MF. Univariate analysis of parameters at the time of diagnosis revealed a significant association between inferior LFS and peripheral blood blasts >1% (p<.0001), leukocytosis >20x109/L (p<.0001), platelet counts <100x109/L (p=.001), hypercatabolic symptoms (p=.01), splenomegaly (p=.01), and treatment history with hydroxyurea (p=.03). Median OS was 6.2 yrs and as expected, it was significantly shorter in pts with leukaemic transformation (3.6 yrs vs 7.3 yrs, log rank test p=.002). According to the IPSS, DIPSS and DIPSS-PLUS pts were categorized predominantly in intermediate-high (IHR) and high risk (HR) groups: 68%>79%>87%, respectively. A significant association was confirmed between the OS and risk profiles (p<.0001). In total, 71% of pts required treatment (e.g. hydroxyurea, erythropoetin, androgen, interferon, corticosteroids, ruxolitinib). Splenectomy was performed in 4% of the pts, while RBC transfusions were indicated in 59%. Interestingly, therapy of any kind was significantly associated with better survival in all DIPSS-PLUS risk categories except for HR group who might be the candidates for novel therapies (p=.009 in low risk vs p=.002 in IHR groups vs p=.127 in HR).
Conclusion
The study provides, for the first time, nationwide data on the 5.4/100000 prevalence and basic features of Bulgarian pts with MF in regard to clinical and laboratory presentation as well as to prognostic parameters and OS.Contributions: LG, MG, GM, GB designed the study and contributed equally to this work. MG, YT conducted data analysis. DT, DT, GD, GT, LS, MZ, NP, SD, TD, VG, VS, YP performed research and contributed equally to this work.
Session topic: E-poster
Keyword(s): Myelofibrosis, Prevalence, Survival, Transformation
Abstract: PB2018
Type: Publication Only
Background
Primary myelofibrosis (MF) is a rare form of BCR-ABL1(-) myeloproliferative neoplasm (MPN). Comprehensive research on the characteristic of this life-threatening condition has not been done in Bulgaria yet. Therefore, the Working Group of MPN of the Bulgarian Society of Hematology initiated a nationwide study.
Aims
To determine the prevalence of MF in the country, and to provide data on the baseline clinical and laboratory parameters and factors for leukemic transformation and overall survival (OS) of a national cohort of patients (pts) with the disease.
Methods
Data on 284 MF pts diagnosed and/or treated in 2014-2015 in 10 clinical centers covering the territory of the country with a population of 5242000>25-yrs of age were analysed. Major demographic, clinical and laboratory parameters, risk profiles according to the international prognostic system (IPSS) and the version for dynamic evaluation of risk (DIPSS and DIPSS-PLUS), and therapeutic approaches were evaluated at baseline as well as in regard to leukemia-free (LFS) and OS in 250 pts.
Results
A male/female ratio of 1.6:1 was determined. The mean age at diagnosis was 64.24 yrs (range 25-86 yrs). In 250 (88%) pts primary MF was diagnosed and in the other 34 (12%) secondary MF developed after another MPN. The most common symptoms were enlarged spleen (78%), anemia (58%), ≥1 constitutional symptoms (41%), thrombocytopenia (21%) and leukocytosis (20%). In 252 (91.2%) pts the diagnosis was confirmed with bone marrow histology and the severity of fibrosis was determined with silver stain in 77% of the cases. Molecular analysis was conducted in 179 (63%) pts and 113 (63.1%) were positive for JAK2 V617F mutation, including as expected 84% of secondary vs 60% of primary MF (p=.014). In primary MF, analysis revealed a significant association only between JAK2 mutation and platelet counts <100x109/L (p=.018) and transfusion dependence (p=.043). In 20 (7.4%) pts blast transformation was registered regardless of the primary or secondary nature of MF. Univariate analysis of parameters at the time of diagnosis revealed a significant association between inferior LFS and peripheral blood blasts >1% (p<.0001), leukocytosis >20x109/L (p<.0001), platelet counts <100x109/L (p=.001), hypercatabolic symptoms (p=.01), splenomegaly (p=.01), and treatment history with hydroxyurea (p=.03). Median OS was 6.2 yrs and as expected, it was significantly shorter in pts with leukaemic transformation (3.6 yrs vs 7.3 yrs, log rank test p=.002). According to the IPSS, DIPSS and DIPSS-PLUS pts were categorized predominantly in intermediate-high (IHR) and high risk (HR) groups: 68%>79%>87%, respectively. A significant association was confirmed between the OS and risk profiles (p<.0001). In total, 71% of pts required treatment (e.g. hydroxyurea, erythropoetin, androgen, interferon, corticosteroids, ruxolitinib). Splenectomy was performed in 4% of the pts, while RBC transfusions were indicated in 59%. Interestingly, therapy of any kind was significantly associated with better survival in all DIPSS-PLUS risk categories except for HR group who might be the candidates for novel therapies (p=.009 in low risk vs p=.002 in IHR groups vs p=.127 in HR).
Conclusion
The study provides, for the first time, nationwide data on the 5.4/100000 prevalence and basic features of Bulgarian pts with MF in regard to clinical and laboratory presentation as well as to prognostic parameters and OS.Contributions: LG, MG, GM, GB designed the study and contributed equally to this work. MG, YT conducted data analysis. DT, DT, GD, GT, LS, MZ, NP, SD, TD, VG, VS, YP performed research and contributed equally to this work.
Session topic: E-poster
Keyword(s): Myelofibrosis, Prevalence, Survival, Transformation
Type: Publication Only
Background
Primary myelofibrosis (MF) is a rare form of BCR-ABL1(-) myeloproliferative neoplasm (MPN). Comprehensive research on the characteristic of this life-threatening condition has not been done in Bulgaria yet. Therefore, the Working Group of MPN of the Bulgarian Society of Hematology initiated a nationwide study.
Aims
To determine the prevalence of MF in the country, and to provide data on the baseline clinical and laboratory parameters and factors for leukemic transformation and overall survival (OS) of a national cohort of patients (pts) with the disease.
Methods
Data on 284 MF pts diagnosed and/or treated in 2014-2015 in 10 clinical centers covering the territory of the country with a population of 5242000>25-yrs of age were analysed. Major demographic, clinical and laboratory parameters, risk profiles according to the international prognostic system (IPSS) and the version for dynamic evaluation of risk (DIPSS and DIPSS-PLUS), and therapeutic approaches were evaluated at baseline as well as in regard to leukemia-free (LFS) and OS in 250 pts.
Results
A male/female ratio of 1.6:1 was determined. The mean age at diagnosis was 64.24 yrs (range 25-86 yrs). In 250 (88%) pts primary MF was diagnosed and in the other 34 (12%) secondary MF developed after another MPN. The most common symptoms were enlarged spleen (78%), anemia (58%), ≥1 constitutional symptoms (41%), thrombocytopenia (21%) and leukocytosis (20%). In 252 (91.2%) pts the diagnosis was confirmed with bone marrow histology and the severity of fibrosis was determined with silver stain in 77% of the cases. Molecular analysis was conducted in 179 (63%) pts and 113 (63.1%) were positive for JAK2 V617F mutation, including as expected 84% of secondary vs 60% of primary MF (p=.014). In primary MF, analysis revealed a significant association only between JAK2 mutation and platelet counts <100x109/L (p=.018) and transfusion dependence (p=.043). In 20 (7.4%) pts blast transformation was registered regardless of the primary or secondary nature of MF. Univariate analysis of parameters at the time of diagnosis revealed a significant association between inferior LFS and peripheral blood blasts >1% (p<.0001), leukocytosis >20x109/L (p<.0001), platelet counts <100x109/L (p=.001), hypercatabolic symptoms (p=.01), splenomegaly (p=.01), and treatment history with hydroxyurea (p=.03). Median OS was 6.2 yrs and as expected, it was significantly shorter in pts with leukaemic transformation (3.6 yrs vs 7.3 yrs, log rank test p=.002). According to the IPSS, DIPSS and DIPSS-PLUS pts were categorized predominantly in intermediate-high (IHR) and high risk (HR) groups: 68%>79%>87%, respectively. A significant association was confirmed between the OS and risk profiles (p<.0001). In total, 71% of pts required treatment (e.g. hydroxyurea, erythropoetin, androgen, interferon, corticosteroids, ruxolitinib). Splenectomy was performed in 4% of the pts, while RBC transfusions were indicated in 59%. Interestingly, therapy of any kind was significantly associated with better survival in all DIPSS-PLUS risk categories except for HR group who might be the candidates for novel therapies (p=.009 in low risk vs p=.002 in IHR groups vs p=.127 in HR).
Conclusion
The study provides, for the first time, nationwide data on the 5.4/100000 prevalence and basic features of Bulgarian pts with MF in regard to clinical and laboratory presentation as well as to prognostic parameters and OS.Contributions: LG, MG, GM, GB designed the study and contributed equally to this work. MG, YT conducted data analysis. DT, DT, GD, GT, LS, MZ, NP, SD, TD, VG, VS, YP performed research and contributed equally to this work.
Session topic: E-poster
Keyword(s): Myelofibrosis, Prevalence, Survival, Transformation
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