Contributions
Abstract: EP919
Type: E-Poster Presentation
Session title: Myelodysplastic syndromes - Clinical
Background
Ordinarily, patients with MDS present with anemia and other cytopenias. Rarely, MDS may present with thrombocytopenia as an isolated abnormality (MDS-IT). There have been few systematic studies on MDS-IT and data regarding its prognosis are conflicting. Previous studies have defined MDS-IT based on the IPSS i.e. hemoglobin ≥10 g/dL, neutrophil count (ANC) ≥1.8×109/L, and platelet count (PLT) <100×109/L. However, these thresholds were developed for prognostic—not diagnostic—purposes which means that mild anemia and/or neutropenia might be present concomitantly with “isolated” thrombocytopenia.
Aims
We investigated the characteristics, overall survival (OS), and leukemia-free survival (LFS) of patients with MDS-IT. In this study, thrombocytopenia was defined as PLT<150×109/L.
Methods
We identified patients who had PLT<150×109/L, Hb>13 g/dL (men) or >12 g/dL (women), ANC≥1.8×109/L, and monocytes <1.0×109/L, registered in the Hellenic National Registry of Myelodysplastic and Hypoplastic Syndromes which includes 2792 patients diagnosed with MDS (analysis cut-off date; July 7, 2016). Patients were stratified into 4 subgroups: subgroup 1 had PLT 149-100×109/L; subgroup 2, 99-50×109/L; subgroup 3, <50×109/L; and subgroup 4, <25×109/L.
Results
A total of 77 patients (45 men; 32 women) with MDS-IT were identified (2.9% of total MDS cohort). Median PLT was 87×109/L (range, 12-145×109/L). Twenty-two patients (28.6%) were classified in subgroup 1; 38 (49.4%) in subgroup 2; 11 (14.3%) in subgroup 3; and 6 (7.8%) in subgroup 4. Median hemoglobin was 13.6 g/dL, and white-cell count 4.6×109/L. The proportion of bone-marrow blasts ranged from 0-9% (median, 2%). Median follow-up was 51.0 months (range, 41.6-60.4), during which 15 (19.5%) patients died. AML developed in 9 patients with MDS-IT (11.7%). IPSS-R score was ≤3.5 in 73.5% and 83.1% had favorable cytogenetics. Morphological classification was consistent with refractory cytopenia with multilineage dysplasia in 70.7%. Unilineage dysplasia was seen in only 10.7%. On subgroup comparisons, we noted no significant differences in age, sex, IPSS-R, blast percentage, cytogenetic pattern, and histological features (WHO type; cellularity; myelofibrosis). Median OS was 109 months (95% CI 103-115) and LFS 108 months (101-115). On subgroup comparisons, we noted no significant between-group difference in OS (P=0.891) and LFS (P=0.871). As compared with the total MDS cohort, patients with MDS-IT presented at younger age (64.7 vs. 72.4 years, P<0.001). Importantly, MDS-IT was characterized by superior survival rates as compared with the total MDS cohort, even after adjusting for age, cytogenetics, IPSS-R, and platelets (P=0.013 for OS and P=0.017 for LFS). There were, however, no differences in the top causes of death: infection was the commonest cause followed by disease progression and cardiovascular disease. Major bleeding comprised 10.3% of deaths in MDS-IT vs. 12.7% in total MDS registry (P=0.217).
Conclusion
In this large cohort study, one of the world’s largest cohorts of MDS-IT, we show that MDS-IT is associated with multilineage dysplasia and lower-risk IPSS-R score. Interestingly, the degree of thrombocytopenia did not seem to correlate with mortality in MDS-IT. The finding that MDS-IT is associated with better survival outcomes as compared with other MDS is important and has major implications for the management of this disorder.
Keyword(s): Myelodysplasia, Platelet, Thrombocytopenia
Abstract: EP919
Type: E-Poster Presentation
Session title: Myelodysplastic syndromes - Clinical
Background
Ordinarily, patients with MDS present with anemia and other cytopenias. Rarely, MDS may present with thrombocytopenia as an isolated abnormality (MDS-IT). There have been few systematic studies on MDS-IT and data regarding its prognosis are conflicting. Previous studies have defined MDS-IT based on the IPSS i.e. hemoglobin ≥10 g/dL, neutrophil count (ANC) ≥1.8×109/L, and platelet count (PLT) <100×109/L. However, these thresholds were developed for prognostic—not diagnostic—purposes which means that mild anemia and/or neutropenia might be present concomitantly with “isolated” thrombocytopenia.
Aims
We investigated the characteristics, overall survival (OS), and leukemia-free survival (LFS) of patients with MDS-IT. In this study, thrombocytopenia was defined as PLT<150×109/L.
Methods
We identified patients who had PLT<150×109/L, Hb>13 g/dL (men) or >12 g/dL (women), ANC≥1.8×109/L, and monocytes <1.0×109/L, registered in the Hellenic National Registry of Myelodysplastic and Hypoplastic Syndromes which includes 2792 patients diagnosed with MDS (analysis cut-off date; July 7, 2016). Patients were stratified into 4 subgroups: subgroup 1 had PLT 149-100×109/L; subgroup 2, 99-50×109/L; subgroup 3, <50×109/L; and subgroup 4, <25×109/L.
Results
A total of 77 patients (45 men; 32 women) with MDS-IT were identified (2.9% of total MDS cohort). Median PLT was 87×109/L (range, 12-145×109/L). Twenty-two patients (28.6%) were classified in subgroup 1; 38 (49.4%) in subgroup 2; 11 (14.3%) in subgroup 3; and 6 (7.8%) in subgroup 4. Median hemoglobin was 13.6 g/dL, and white-cell count 4.6×109/L. The proportion of bone-marrow blasts ranged from 0-9% (median, 2%). Median follow-up was 51.0 months (range, 41.6-60.4), during which 15 (19.5%) patients died. AML developed in 9 patients with MDS-IT (11.7%). IPSS-R score was ≤3.5 in 73.5% and 83.1% had favorable cytogenetics. Morphological classification was consistent with refractory cytopenia with multilineage dysplasia in 70.7%. Unilineage dysplasia was seen in only 10.7%. On subgroup comparisons, we noted no significant differences in age, sex, IPSS-R, blast percentage, cytogenetic pattern, and histological features (WHO type; cellularity; myelofibrosis). Median OS was 109 months (95% CI 103-115) and LFS 108 months (101-115). On subgroup comparisons, we noted no significant between-group difference in OS (P=0.891) and LFS (P=0.871). As compared with the total MDS cohort, patients with MDS-IT presented at younger age (64.7 vs. 72.4 years, P<0.001). Importantly, MDS-IT was characterized by superior survival rates as compared with the total MDS cohort, even after adjusting for age, cytogenetics, IPSS-R, and platelets (P=0.013 for OS and P=0.017 for LFS). There were, however, no differences in the top causes of death: infection was the commonest cause followed by disease progression and cardiovascular disease. Major bleeding comprised 10.3% of deaths in MDS-IT vs. 12.7% in total MDS registry (P=0.217).
Conclusion
In this large cohort study, one of the world’s largest cohorts of MDS-IT, we show that MDS-IT is associated with multilineage dysplasia and lower-risk IPSS-R score. Interestingly, the degree of thrombocytopenia did not seem to correlate with mortality in MDS-IT. The finding that MDS-IT is associated with better survival outcomes as compared with other MDS is important and has major implications for the management of this disorder.
Keyword(s): Myelodysplasia, Platelet, Thrombocytopenia