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Objective: To analyze the long-term efficacy and safety of ATR in adult patients with primary resistant ITP in real-world clinical practice. Materials and methods.The article contains long-term results analysis of ATR application under real clinical practice conditions in 138 patients (40 men and 98 women) whose median age at the beginning of therapy was 59 (18-86) years. Two ATR medicines-romiplostim (100 patients) and eltrombopag (38 patients) were used. Results. During the first month of therapy, the median platelet count in the romiplostim group increased from 17·109 / L to 60·109 / L (9-600·109 / L), and the elethrombopag from 16.109 / L to 56.109 / L (9-400·109 / L). The minimal response (reaching platelet counts over 30·109 / L) was achieved in 92% of cases in both groups. Partial response (achievement of platelet count more than 50·109 / L) was achieved in 91 and 84% of patients in the rhombostim and eltrombopag groups, respectively. The frequency of complete response (an increase in platelet counts above 100·109 / L) was noted somewhat more often in the rhyploistim group-69% compared to 47% in the eltrombopag group (P = NS). Most patients demonstrated a long-term stable effect in the form of an increase in blood platelet count to a safe level during months and years of ATR treatment. The achievement of at least partial remission for 3 months or more was 70 and 71% in romiplostim and elthrombopag groups, respectively. Patients who started ATR- therapy are currently continuing treatment: 51% - in romiplostim group and in eltrombopag group-39%. The main reason of discontinuation the initially effective therapy were the loss of platelet response, toxicity, withdrawal from treatment (withdrawal with preservation of remission) and patients death. The tolerability of drugs with long-term admission was satisfactory. The most common AE were headache, bone pain, thrombosis, increased blood pressure and petechial hemorrhagic eruptions. The overall incidence of complications did not differ significantly between the romiplostim and eltrombopag groups -15.6 and 15.8%, respectively. Conclusion. Long-term ATR-therapy using in patients with resistant chronic ITP is an effective and largely safe treatment option.

Background:For a number of years, it has been shown that TPO‐RAs are a highly effective therapy of ITP. Still, there are few published guidelines/recommendations addressing the management of ITP patients (pts) on TPO‐RAs after they achieve continuous remission.Aims:To evaluate the TPO‐RAs «stop‐therapy» in ITP pts with complete response (CR) to TPO‐RAs.Methods:We analyzed data of TPO‐RA «stop‐therapy» in patients with CR. Primary endpoints of the study were the duration and type of response (CR, partial – PR, minor – MR, and loss of response) after TPO‐RAs had been stopped. Secondary endpoints included types of response (CR, PR, MR) after TPO‐RAs had been resumed in those pts who relapsed.Results:194 ITP pts treated at Moscow City Hematology Center (Botkin Hospital) in 2014–2018 were administered TPO‐ARs: (romiplostim (R): n = 123; eltrombopag (E): n = 71). 106 pts (55%) achieved CR (platelets >100х109/l)(R: n = 79 (64%); E: n = 27 (38%).28 pts in continuous CR (≥3 months (mo) had TPO‐RA stopped: (R: n = 19; E: n = 9). Group R (GR) comprised of 4 males and 15 females, with median age (Mе) age at the time of diagnosis of 51 (range 1 ‐ 69) years, and Me follow‐up time from the time of diagnosis was 108 (9 ‐ 483) mo. Group E (GE) included 1 male and 8 females with Me age of 54 (range 12 ‐ 75) years and Me follow‐up time from the time of diagnosis of 74 (27 ‐ 228) mo.Me stop‐therapy length was 69 (range 20 ‐ 276) weeks in GR, and 38 (range 13 ‐ 131) weeks in GE.At the time of analysis, CR was maintained in 5 (26%) GR pts, and in 1 (11%) GE pt. PR was seen in 7 (37%) GR pts and in 2 (23%) GE pts; and MR in 5 (26%) GR pts and in 1 (11%) GE pt. 2 (11%) GR pts and 5 (55%) GE pts did not respond to TPO‐RAs.TPO‐RAs were resumed in 2 GR and 2 GE pts who relapsed (7 pts have relapsed altogether), and 1 PR and 1 MR in each group were achieved.Summary/Conclusion:Our data adds to the growing body of evidence that ITP pts maintain remission after TPO‐RAs are stopped. This may possibly represent a change in therapeutic approach to these patients who usually require continuous maintenance therapy. Longer follow‐up and larger number of pts enrolled in the study are required to fully assess benefits of such approach.

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