Safety and efficacy of long-term treatment of chronic/persistent ITP with eltrombopag: final results of the EXTEND study
- PMID: 29042367
- DOI: 10.1182/blood-2017-04-748707
Safety and efficacy of long-term treatment of chronic/persistent ITP with eltrombopag: final results of the EXTEND study
Erratum in
-
Wong RSM, Saleh MN, Khelif A, et al. Safety and efficacy of long-term treatment of chronic/persistent ITP with eltrombopag: final results of the EXTEND study. Blood. 2017;130(23):2527-2536.Blood. 2018 Feb 8;131(6):709. doi: 10.1182/blood-2017-12-823252. Blood. 2018. PMID: 29438973 No abstract available.
Abstract
In phase 2/3 trials, eltrombopag treatment of 6 months or less in patients with chronic/persistent immune thrombocytopenia (ITP) increased platelet counts and reduced bleeding. The open-label EXTEND study evaluated long-term safety and efficacy of eltrombopag in adults with ITP who had completed a previous eltrombopag study. For the 302 patients enrolled, median duration of eltrombopag treatment was 2.37 years (2 days-8.76 years). Median platelet counts increased to 50 × 109/L or more by week 2 and were sustained throughout the treatment period. Overall, 259 patients (85.8%) achieved a response (platelet count ≥50 × 109/L at least once in the absence of rescue), and 133 (52%) of 257 patients achieved a continuous response of 25 weeks or longer. Responses in patients with platelet counts lower than 15 × 109/L, more previous therapies, and/or splenectomy were somewhat lower. Thirty-four (34%) of 101 patients receiving concomitant ITP medication discontinued 1 or more medication. In patients with assessments, bleeding symptoms (World Health Organization grades 1-4) decreased from 57% at baseline to 16% at 1 year. Forty-one patients (14%) withdrew because of adverse events. Hepatobiliary adverse events (n = 7), cataracts (n = 4), deep vein thrombosis (n = 3), cerebral infarction (n = 2), headache (n = 2), and myelofibrosis (n = 2) occurred in more than 1 patient; the remaining adverse events occurred only once. Rates of thromboembolic events (6%) and hepatobiliary adverse events (15%) did not increase with treatment duration past 1 year. EXTEND demonstrated that long-term use of eltrombopag was effective in maintaining platelet counts of 50 × 109/L or more and reducing bleeding in most patients with ITP of more than 6 months' duration. Important adverse events (eg, thrombosis, hepatobiliary, and bone marrow fibrosis) were infrequent. (ClinicalTrials.gov:NCT00351468).
© 2017 by The American Society of Hematology.
Similar articles
-
Eltrombopag for children with chronic immune thrombocytopenia (PETIT2): a randomised, multicentre, placebo-controlled trial.Lancet. 2015 Oct 24;386(10004):1649-58. doi: 10.1016/S0140-6736(15)61107-2. Epub 2015 Jul 28. Lancet. 2015. PMID: 26231455 Clinical Trial.
-
Safety and efficacy of eltrombopag for treatment of chronic immune thrombocytopenia: results of the long-term, open-label EXTEND study.Blood. 2013 Jan 17;121(3):537-45. doi: 10.1182/blood-2012-04-425512. Epub 2012 Nov 20. Blood. 2013. PMID: 23169778 Clinical Trial.
-
Effect of eltrombopag on platelet counts and bleeding during treatment of chronic idiopathic thrombocytopenic purpura: a randomised, double-blind, placebo-controlled trial.Lancet. 2009 Feb 21;373(9664):641-8. doi: 10.1016/S0140-6736(09)60402-5. Lancet. 2009. PMID: 19231632 Clinical Trial.
-
Spotlight on eltrombopag in treatment-refractory chronic primary immune thrombocytopenia.BioDrugs. 2011 Dec 1;25(6):401-4. doi: 10.2165/11207620-000000000-00000. BioDrugs. 2011. PMID: 22050343 Review.
-
Eltrombopag: a review of its use in treatment-refractory chronic primary immune thrombocytopenia.Drugs. 2011 Jul 9;71(10):1333-53. doi: 10.2165/11207390-000000000-00000. Drugs. 2011. PMID: 21770480 Review.
Cited by
-
Romiplostim for the Treatment of Immune Thrombocytopenia: Spotlight on Patient Acceptability and Ease of Use.Patient Prefer Adherence. 2020 Jul 22;14:1237-1250. doi: 10.2147/PPA.S192481. eCollection 2020. Patient Prefer Adherence. 2020. PMID: 32801654 Free PMC article. Review.
-
Changes in health-related quality of life with long-term eltrombopag treatment in adults with persistent/chronic immune thrombocytopenia: Findings from the EXTEND study.Am J Hematol. 2019 Feb;94(2):200-208. doi: 10.1002/ajh.25348. Epub 2018 Nov 29. Am J Hematol. 2019. PMID: 30417939 Free PMC article.
-
Refractory primary immune thrombocytopenia (ITP): current clinical challenges and therapeutic perspectives.Ann Hematol. 2022 May;101(5):963-978. doi: 10.1007/s00277-022-04786-y. Epub 2022 Feb 24. Ann Hematol. 2022. PMID: 35201417 Free PMC article. Review.
-
Relationship between response to lusutrombopag and splenic volume.World J Gastroenterol. 2018 Dec 14;24(46):5271-5279. doi: 10.3748/wjg.v24.i46.5271. World J Gastroenterol. 2018. PMID: 30581275 Free PMC article.
-
Comparison of Splenectomy and Eltrombopag Treatment in the Second-Line Treatment of Immune Thrombocytopenic Purpura.Turk J Haematol. 2021 Aug 25;38(3):181-187. doi: 10.4274/tjh.galenos.2021.2021.0216. Epub 2021 Jun 24. Turk J Haematol. 2021. PMID: 34162173 Free PMC article.
Publication types
MeSH terms
Substances
Associated data
LinkOut - more resources
Full Text Sources
Other Literature Sources
Medical
Miscellaneous