Background: Immune thrombocytopenia (ITP) is an autoimmune disorder characterized by increased platelet destruction and impaired platelet production. Over the past decade, use of thrombopoietin receptor agonists (TPO-RA) is steadily rising in ITP. Aims: In this study, we explore the clinical profile of ITP patients on TPO-RA therapy, response rates to TPO-RA therapy, and analyze various factors that can predict outcomes in one of the largest public-sector teaching hospitals in India. Methods: This retrospective observational study included consecutive patients diagnosed with ITP and initiated on TPO-RA therapy (Romiplostim and Eltrombopag) in our clinic from January 1, 2016, to December 31, 2021. Response was defined according to standard international criteria. Results: Over the past six years, 166 patients were initiated on TPO-RA therapy and had a total of 183 treatments (92 eltrombopag, 74 romiplostim, and 17 trailed on both). The median age in our cohort was 28.5 years (range, 1.5-70 years). Pediatric cohort (age < 18 years) included 57 patients. Almost half (N=93; 56%) of the patients had chronic ITP prior to TPO-RA initiation. Baseline characteristics were shown in Table 1. Response rates were significantly higher in the romiplostim group when compared to the eltrombopag group (91% vs. 60%; p<0.001) in the pediatric cohort, which was not observed in the adult cohort (77% vs. 84%; p=0.28). Response rates were significantly higher in the pediatric cohort when compared to the adult cohort in the romiplostim group (91% vs. 77%; p = 0.008); however, adults responded better in the eltrombopag group (84% vs. 60%; p<0.001). There was a significant reduction in bleeding manifestations at 1 month when compared with baseline (20% vs. 61%; p<0.001). On univariate analysis, prior splenectomy was a predictor of poor response to TPO-RA therapy. However, on multivariate analysis, age, sex, ITP duration, prior splenectomy, prior lines of therapy were not predictive of response. Adverse events: Thrombocytosis was noted in 12 (7%) patients with no evidence of thrombosis, and 2 (1%) patients progressed to myelodysplastic syndrome. Of the 17 patients who were trailed on both therapies, 6 (3.5%) patients failed both TPO-RA therapies. Among the 54 (31%) patients who discontinued therapy, 11 (6.6%) patients achieved durable response off treatment. The median follow-up duration was 13 months (range, 0.8-79 months). Image:Summary/Conclusion: Response rates to TPO-RA therapy in our cohort are consistent with those reported from the West. We observed that response rates were significantly higher in our pediatric cohort when compared to our adult cohort.
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