Distinctive Attributes of Indian Patients With Classical BCR::ABL1 Negative Myeloproliferative Neoplasms: Unified Clinical and Laboratory Data.

INTRODUCTION: We report one of the largest single center data from a mixed referral setting in India describing baseline characteristics and outcomes of patients with classical BCR::ABL1 negative myeloproliferative neoplasms (MPNs). MATERIALS AND METHODS: Patients diagnosed from June 2019 to 2022 were included. Workup and treatment was as per current guidelines. RESULTS: Diagnosis comprised polycythemia vera (PV) in 51(49%), ET in 33(31.7%) and prefibrotic primary myelofibrosis (MF) pre fibrotic myelofibrosis (prePMF) and myelofibrosis in 10(9.6%) patients each. Median age at diagnosis was 52 years for PV and ET, 65.5 for MF and 79 years for prePMF. Diagnosis was incidental in 63(56.7%) and after thrombosis in 8(7.2%) patients. Baseline next generation sequencing (NGS) was available for 63(60.5%) patients. Driver mutations in PV: JAK2 in 80.3%; in ET: JAK2 in 41%, CALR in 26%, MPL in 2.9%; in prePMF JAK2 in 70%, CALR in 20%, MPL in 10%, and in MF: JAK2 in 10%, MPL in 30% and CALR in 40%. Seven novel mutations were detected of which 5 were potentially pathogenic on computational analysis. After median follow up of 30 months, 2 patients had disease transformation and none had new episodes of thrombosis. Ten patients died, most commonly with cardiovascular events(n = 5,50%). Median overall survival was not reached. Mean OS time was 10.19 years(95%CI, 8.6 to 11.74) and mean time to transformation was 12.2 years(95% CI,11.8 to 12.6). CONCLUSION: Our data indicates comparatively indolent presentation of MPNs in India with younger age and lower risk of thrombosis. Further follow up will enable correlation with molecular data and guide modification of age based risk stratification models.

A cross-sectional study on thrombopoietin levels in immune thrombocytopenia and its correlation with platelet count, megakaryocytes, and treatment response.

INTRODUCTION: Thrombopoietin (TPO) being the major regulator of megakaryopoiesis is expected to show a compensatory increase in immune thrombocytopenia (ITP), however, it is not so observed. This study was undertaken to measure TPO levels in ITP and assesses its association with platelet count, megakaryocytes, and response to steroid therapy. MATERIALS AND METHODS: A total of 41 cases of ITP and twenty controls with normal platelet count were enrolled in this prospective study. Complete blood count, bone marrow examination, and ELISA for serum TPO were measured. Response to steroid therapy was evaluated for thirty cases. RESULTS: The TPO levels were increased in 80.5% of patients in comparison to the controls. The degree of rise, however, was variable. On analyzing low, normal, and high TPO levels with reference to platelet and megakaryocyte count no statistically significant difference was observed. Raised TPO levels were seen with significant lowering of functional megakaryocytes. The mean TPO levels in nonresponders were higher than responders but highly variable and statistically nonsignificant. CONCLUSION: Quantitative alterations in TPO are in a way regulated by qualitative efficacy of megakaryocytes rather than platelet or megakaryocyte count. Nonresponders with markedly increased TPO levels (due to qualitative megakaryocyte injury) are less likely to respond to TPO receptor agonist.