Effectiveness and Safety of Romiplostim Among Patients with Newly Diagnosed, Persistent and Chronic Immune Thrombocytopenia in European Clinical Practice.

Autor: Snell Taylor SJ; NoviSci, Inc., 201 W Main St, Ste 101, Durham, NC, 27701, USA. staylor@novisci.com., Nielson CM; Amgen Inc., Thousand Oaks, USA., Breskin A; NoviSci, Inc., 201 W Main St, Ste 101, Durham, NC, 27701, USA.; Department of Epidemiology, University of North Carolina at Chapel Hill, Chapel Hill, USA., Saul B; NoviSci, Inc., 201 W Main St, Ste 101, Durham, NC, 27701, USA., Yu Y; NoviSci, Inc., 201 W Main St, Ste 101, Durham, NC, 27701, USA., Alam N; Amgen Ltd, Uxbridge, UK., Eisen M; Amgen Inc., Thousand Oaks, USA., Hippenmeyer J; Amgen Inc., Rotkreuz, Switzerland., Janssens A; University Hospitals Leuven, Leuven, Belgium., Kozak T; Charles University, Prague, Czech Republic., Papadaki HA; University Hospital of Heraklion, Heraklion, Greece., Selleslag D; Algemeen Ziekenhuis Sint-Jan, Bruge, Belgium., Viallard JF; Hospital du Haut Leveque, Pessac, France., Feistritzer C; Universitätsklinik für Innere Medizin V Innsbruck, Innsbruck, Austria., Kaiafa G; AHEPA University Hospital, Aristotle University of Thessaloniki, Thessaloniki, Greece., Kelsh M; Amgen Inc., Thousand Oaks, USA., Kilpatrick K; Amgen Inc., Thousand Oaks, USA., Brookhart MA; NoviSci, Inc., 201 W Main St, Ste 101, Durham, NC, 27701, USA.; Department of Population Health Sciences, Duke University, Durham, UK., McGrath LJ; NoviSci, Inc., 201 W Main St, Ste 101, Durham, NC, 27701, USA.
Jazyk: angličtina
Zdroj: Advances in therapy [Adv Ther] 2021 May; Vol. 38 (5), pp. 2673-2688. Date of Electronic Publication: 2021 Apr 18.
DOI: 10.1007/s12325-021-01727-5
Abstrakt: Introduction: Romiplostim has been approved in Europe since 2009 to treat patients with chronic primary immune thrombocytopenia (ITP). Using real-world data from seven European countries, we measured the effectiveness and safety outcomes within 24 weeks following romiplostim initiation by duration of ITP: less than 3 months ("newly diagnosed"), 3-12 months ("persistent"), and more than 12 months ("chronic").
Methods: Adults with ITP and ≥ 1 romiplostim administration between 2009 and 2012 were included. Endpoints included durable platelet response, median platelet count, rescue therapy, bleeding and adverse events. We used inverse probability of censoring weighted estimators to estimate cumulative risk of each outcome. There were 64 newly diagnosed, 50 persistent, and 226 chronic ITP patients at romiplostim initiation.
Results: Durable platelet response at 24 weeks ranged from 32% [confidence interval (CI): 18-46%] in newly diagnosed patients to 53% (CI 37-68%) in persistent patients. Median platelet count during follow-up ranged from 88 (CI 80-96) × 10 9 /L in chronic patients to 131 (CI 102-160) × 10 9 /L in newly diagnosed patients.
Conclusion: Regardless of ITP duration, over half of patients discontinued concomitant ITP medications. Few adverse events were observed. Although only approved for chronic patients, estimates of the romiplostim treatment effect were similar across patients being managed in European clinical practice, regardless of ITP duration at romiplostim initiation.
Databáze: MEDLINE
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