PF701 ASSESSMENT OF ELTROMBOPAG IN PATIENTS WITH CHRONIC IMMUNE THROMBOCYTOPENIA UNDER ROUTINE CLINICAL PRACTICE IN THE MIDDLE EAST, TURKEY, ASIA, AND AUSTRALIA
Authors: R. Wong, I. Yavaşoğlu, M.A. D. Yassin, P. Tarkun, S.‐S. Yoon, M. Abdelmooti Samra, P. Angchaisuksiri, V. Pilipovic, J.H. Jang
Journal: HemaSphere (2019)

Background: Eltrombopag (EPAG), an oral thrombopoietin receptor agonist, is approved in the Middle East Turkey, Asia and Australia for the treatment of thrombocytopenia in adults with chronic immune thrombocytopenia (cITP) with insufficient response to prior therapy, such as corticosteroids and immunoglobulins. To date, real‐world data on EPAG use in patients (pts) with cITP have not been collected in a systematic manner in most countries outside the US and Europe. CITE is a prospective non‐interventional study (targeted to recruit ∼300 pts from June 2017 to May 2019) aimed to assess the use of EPAG in adult cITP under routine clinical practice in these countries. Aims: This first interim analysis of the study (as of 3 rd September 2018) evaluated the utility, efficacy, and safety of EPAG, including patients’ adherence to dosing. Methods: Adult pts aged ≥18 years (yrs) with cITP for whom the physician has prescribed EPAG treatment and who started EPAG treatment up to 12 weeks prior to enrollment were included. Dosage of EPAG was determined based on respective locally approved label and at the investigator's discretion. The maximum observational period for pt on treatment is 18 months (mos). Visits were documented approximately at baseline, and 1, 3, 6, 12, and 18 mos. Results: Overall, 122 pts with cITP were analyzed: median (range) age, 48 (18–88) yrs; 64% were females; and 30% had prior splenectomy. Pts received corticosteroids (68%), immunoglobulins (28%), EPAG (11%), rituximab (6%) and romiplostim (2%) as prior ITP treatment. Forty‐one, 32 and 22 pts had 1, 2 and 3 prior ITP medications, respectively. Median (range) time since first diagnosis of cITP was 3 (0–31.6) yrs. Most common reasons for initiating EPAG therapy were lack of efficacy of prior treatment (55%) and loss of response to prior treatment (18%). Overall, median (range) daily dose of EPAG was 50 mg (13.1–73.4) and median (range) duration of EPAG treatment at the time of this interim analysis was 148.5 (1–493) days. Fifteen pts discontinued EPAG treatment mainly due to lack of efficacy (n = 5) and sustained response (n = 4). The median (range) platelet count (10 3 /μL; figure) at baseline (n = 110) was 18 (0.03–405), at mo 2 after starting EPAG (n = 65) was 90 (2–715), at mo 3 after starting EPAG (n = 53) was 103 (2–690), and at mo 6 (n = 21) was 121 (20–595). The platelet response (defined as at least one platelet count of ≥50,000/μL in the absence of rescue therapy) was 49/65 (75%) and 16/21 (76%) at mos 2 and 6 after the start of EPAG, respectively. Adherence to EPAG dosing instructions was (median [range] days) 28 (0–46) at mo 1 and 57.5 (5–120) at mo 3. In total, 27 pts required rescue therapy during the study (platelet transfusions, 8%; new concomitant ITP medication, 16%). Three pts had grade 3 and 1 pt had grade 4 bleeding events. The FACIT‐Fatigue median (range) score was 37 (3–52) at baseline (n = 118) and 41 (2–52) at mo 3 (n = 69). Adverse events (AEs) were reported in 49 pts; 12 and 3 pts had grade 3 and 4 AEs, respectively. One pt each had cerebral venous thrombosis (grade 4) and abnormal hepatic function (grade 3), not leading to treatment discontinuation. Most commonly reported AEs (n%) with EPAG: anemia (3.3%), upper respiratory tract infection, and cough (4.1% each). One on‐treatment death occurred in the study due to urinary tract infection and sepsis, and was considered not related to study treatment. Summary/Conclusion: The efficacy outcomes and the safety profile of EPAG reported with this interim analysis of this observational study are consistent with prior EPAG trials in cITP. image