Background: Thrombopoietin receptor-agonists (TPO-RAs), eltrombopag and romiplostim, are effective in immune thrombocytopenia (ITP), resulting in 70-80% response rates if TPO-RAs are administered continuously. However, recent studies suggest that some patients can sustain their platelet response when treatment is withdrawn.

Aims: How many patients could maintain remission after withdrawal of thrombopoietin receptor-agonists in immune thrombocytopenia.

Methods: We retrospectively evaluated 39 adult primary ITP patients (24 women and 15 men, median age; 71 years) treated with TPO-RAs (28 with eltrombopag and 11 with romiplostim).

Results: Among the 27 patients who achieved complete remission (platelet count >10×10⁴/mL), TPO-RAs was withdrawn in 19 patients. Of these 19 patients, 11 patients showed sustained complete remission after TPO-RAs withdrawal (6 with eltrombopag and 5 with romiplostim), with a median follow-up of 17 (range; 2-67) months. Age, gender, duration of ITP, previous therapies, platelet count, immature platelet fraction, serum TPO and the positive rates of anti-platelet glycoprotein antibodies before starting TPO-RAs did not significantly differ between the patient groups with and without sustained response after withdrawal of TPO-RAs. Doses of TPO-RAs, duration of TPO-RAs treatment, duration of complete remission and platelet count before TPO-RAs withdrawal did not also significantly differ between the two groups.

Conclusions: Eventually, no factors predicting sustained response after TPO-RAs withdrawal were identified. Nonetheless this study provides observational evidence that 28．2% of adult ITP patients treated with TPO-RAs had sustained complete remission after withdrawal of TPO-RAs, which may be a strategy for cure of ITP.

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ISTH Congress Abstracts - https://abstracts.isth.org/abstract/sustained-remission-after-withdrawal-of-thrombopoietin-receptor-agonists-in-immune-thrombocytopenia/