PMID- 32175766 OWN - NLM STAT- MEDLINE DCOM- 20210316 LR - 20210316 IS - 2042-6313 (Electronic) IS - 2042-6305 (Linking) VI - 9 IP - 7 DP - 2020 May TI - Real-world evidence on clinical outcomes in immune thrombocytopenia treated with thrombopoietin receptor agonists. PG - 447-457 LID - 10.2217/cer-2019-0177 [doi] AB - Aim: Eltrombopag and romiplostim are US FDA approved for treatment of immune thrombocytopenia in patients with insufficient response to other treatments. Clinical or real-world data comparing outcomes of the two drugs are limited. Methods: This retrospective cross-sectional study sought information on bleeding-related episodes (BREs), adverse events (AEs) and other outcomes of eltrombopag or romiplostim treatment in immune thrombocytopenia. Results: Patients receiving eltrombopag experienced significantly reduced BREs, severe BREs, rescue medication use and platelet transfusions. Diarrhea and headache were significantly less frequent in patients receiving eltrombopag; other AEs occurred equally in both groups. Conclusion: There may be a potential advantage for the use of eltrombopag versus romiplostim in the practice settings studied, based on rates of BREs and AEs and rescue medication utilization. FAU - Forsythe, Anna AU - Forsythe A AD - Evidence Generation, Purple Squirrel Economics, New York, NY 10010, USA. FAU - Schneider, John AU - Schneider J AD - Avalon Health Economics, Morristown, NJ 07960, USA. FAU - Pham, Timothy AU - Pham T AD - Health Economics and Outcomes Research, Novartis, East Hanover, NJ 07936, USA. FAU - Bhor, Menaka AU - Bhor M AD - Health Economics and Outcomes Research, Novartis, East Hanover, NJ 07936, USA. FAU - Said, Qayyim AU - Said Q AD - Health Economics and Outcomes Research, Novartis, East Hanover, NJ 07936, USA. FAU - Allepuz, Alejandro AU - Allepuz A AD - Clinical Development, Novartis Pharma AG, Basel, Switzerland. FAU - Socorro O Portella, Maria do AU - Socorro O Portella MD AD - Oncology Clinical Development, Novartis, East Hanover, NJ 07936, USA. FAU - Kwon, Christina S AU - Kwon CS AD - Evidence Generation, Purple Squirrel Economics, New York, NY 10010, USA. FAU - Roy, Anuja Nidumolu AU - Roy AN AD - Health Economics and Outcomes Research, Novartis, East Hanover, NJ 07936, USA. LA - eng PT - Journal Article PT - Observational Study PT - Research Support, Non-U.S. Gov't DEP - 20200316 PL - England TA - J Comp Eff Res JT - Journal of comparative effectiveness research JID - 101577308 RN - 0 (Benzoates) RN - 0 (Hydrazines) RN - 0 (Pyrazoles) RN - 0 (Receptors, Fc) RN - 0 (Receptors, Thrombopoietin) RN - 0 (Recombinant Fusion Proteins) RN - 9014-42-0 (Thrombopoietin) RN - GN5XU2DXKV (romiplostim) RN - S56D65XJ9G (eltrombopag) SB - IM MH - Adult MH - Aged MH - Benzoates/adverse effects/*therapeutic use MH - Cross-Sectional Studies MH - Female MH - Humans MH - Hydrazines/adverse effects/*therapeutic use MH - Male MH - Middle Aged MH - Platelet Count MH - Purpura, Thrombocytopenic, Idiopathic/*drug therapy MH - Pyrazoles/adverse effects/*therapeutic use MH - Receptors, Fc/*therapeutic use MH - Receptors, Thrombopoietin/*agonists MH - Recombinant Fusion Proteins/adverse effects/*therapeutic use MH - Retrospective Studies MH - Thrombopoietin/adverse effects/*therapeutic use OTO - NOTNLM OT - adverse events OT - bleeding episodes OT - eltrombopag OT - immune thrombocytopenia OT - romiplostim OT - treatment outcomes EDAT- 2020/03/17 06:00 MHDA- 2021/03/17 06:00 CRDT- 2020/03/17 06:00 PHST- 2020/03/17 06:00 [pubmed] PHST- 2021/03/17 06:00 [medline] PHST- 2020/03/17 06:00 [entrez] AID - 10.2217/cer-2019-0177 [doi] PST - ppublish SO - J Comp Eff Res. 2020 May;9(7):447-457. doi: 10.2217/cer-2019-0177. Epub 2020 Mar 16.