Abstract Number: PB0238
Meeting: ISTH 2022 Congress
Background: Treatment approach to chronic immune thrombopenia (ITPc) in pediatric patients has changed during the last decade thanks to the development of thrombopoietin receptor agonist (ar-TPO). During the last decade, series in adult patients have described that up to 30% of patients with ITPc on ar-TPO treatment may have a sustained platelet response after drug withdrawal, but there is still scarce information in children.
Aims: To describe tapering of ar-TPO in ITPc pediatric patients treated in Spanish hospitals.
Methods: A multicenter retrospective observational study was performed in children with chronic immune thrombopenia (ITPc) to describe ar-TPO tapering and withdrawal in patients who had achieved a sustained complete response to ar-TPO.
Results: Ten pediatric patients with ITPc were included. Table 1 resumes patients characteristics and information regarding ar-TPO tapering. None of them required relevant dose increasing during this process. One case had an asymptomatic relevant thrombopenia ( < 30x109/L), but platelet count increased progressively without the need of dose escalation. After a median time of 24 months (SD 8.7), two patients relapsed (figure 1). One of them achieved a new complete response with ar-TPO. The other was refractory to ar-TPO and immunosuppressants but responded to rituximab. Three patients had a transient decrease of total platelet count (34-72x109/L) that improved without treatment administration.
Conclusion(s): This series shows that tapering and withdrawal of ar-TPO treatment can be considered in pediatric patients with ITPc who have achieved a sustained complete response. Our experience suggests that discontinuation should be progressive and slow.
To cite this abstract in AMA style:Berrueco R, Solsona M, Sebastián E, Cervera A, Sastre A, Astigarraga I, Argiles B, Dasí M, Dapena J, Monteagudo E. Tapering of trombopoietin receptor agonist in pediatric patients with chronic ITP. Is it posible? [abstract]. https://abstracts.isth.org/abstract/tapering-of-trombopoietin-receptor-agonist-in-pediatric-patients-with-chronic-itp-is-it-posible/. Accessed February 20, 2024.
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