{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,14]],"date-time":"2026-05-14T00:10:04Z","timestamp":1778717404485,"version":"3.51.4"},"reference-count":34,"publisher":"Georg Thieme Verlag KG","issue":"CP","license":[{"start":{"date-parts":[[2026,5,4]],"date-time":"2026-05-04T00:00:00Z","timestamp":1777852800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["TH Open"],"abstract":"<jats:p>Immune thrombocytopenia (ITP) is an autoimmune disorder characterized by platelet counts\u2009&lt;\u2009100 \u00d7109\/L and variable risk of bleeding. Many adults develop chronic disease requiring second-line therapy. Avatrombopag, an oral thrombopoietin receptor agonist (TPO-RA), has demonstrated high efficacy and favorable safety in clinical trials, but real-world data from Poland are lacking.<\/jats:p>\n                  <jats:p>To evaluate the real-world efficacy, durability, treatment-free remission (TFR), and safety of avatrombopag in Polish adults with primary ITP, and to explore predictors of treatment response.<\/jats:p>\n                  <jats:p>We conducted a retrospective multicenter study including adults with primary ITP treated with avatrombopag across eight hematology centers in Poland. Responses were defined according to International Working Group criteria and assessed at 1, 3, 6, and 12 months. Univariable logistic regression was used to identify baseline predictors of achieving a platelet response.<\/jats:p>\n                  <jats:p>A total of 142 patients (median age, 54 years) were included. The cohort was heavily pretreated (median of three prior lines of therapy), with 36.6% previously exposed to another TPO-RA. The median baseline platelet count was 21.5 \u00d7109\/L. By month 1, 78.3% of patients achieved a platelet response, including 47.8% who achieved a complete response (CR), with a median time to first documented response of 30 days (corresponding to the first scheduled assessment). Among evaluable patients, response rates (CR\u2009+\u2009R) remained high at 3, 6, and 12 months (84.1, 90.8, and 96.8%, respectively). Four patients (2.8%) achieved TFR with stable platelet counts after discontinuation. Higher baseline hemoglobin levels and lower bleeding scores were associated with achieving a response. Avatrombopag was well tolerated, with infrequent adverse events, one event of hepatotoxicity, and rare thrombotic events.<\/jats:p>\n                  <jats:p>Avatrombopag induced rapid, sustained, and well-tolerated platelet responses in a heavily pretreated real-world Polish cohort. These findings support its use as an effective second-line therapy and highlight the potential for treatment de-escalation, including TFR, in selected patients.<\/jats:p>","DOI":"10.1055\/a-2868-3290","type":"journal-article","created":{"date-parts":[[2026,5,4]],"date-time":"2026-05-04T23:14:38Z","timestamp":1777936478000},"source":"Crossref","is-referenced-by-count":0,"special_numbering":"S","title":["Efficacy and Safety of Avatrombopag Treatment in Immune Thrombocytopenia Patients in Poland: A Multicenter Study"],"prefix":"10.1055","volume":"10","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-9845-6214","authenticated-orcid":false,"given":"Michal","family":"Witkowski","sequence":"additional","affiliation":[{"name":"Hematology Outpatient Clinic, Copernicus Memorial Hospital, Lodz, lodzkie, Poland"},{"name":"Department of Science and Technology, Foundation for Leukemia Patients, Lodz, Poland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jagoda","family":"Rytel","sequence":"additional","affiliation":[{"name":"Department of Hematology, Transplantation and Internal Medicine, Medical University of Warsaw, Warsaw, Masovian Voivodeship, Poland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Justyna","family":"Kozinska","sequence":"additional","affiliation":[{"name":"Department of Hematooncology and Bone Marrow Transplantation, Medical University of Lublin, Lublin, Lublin Voivodeship, Poland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jolanta","family":"Oleksiuk","sequence":"additional","affiliation":[{"name":"Hematology Clinic, University Clinical Hospital in Bialystok, Bialystok, Podlaskie Voivodeship, Poland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Weronika","family":"Piszczek","sequence":"additional","affiliation":[{"name":"Department of Hematology, Rydygier Provincial Polyclinical Hospital, Torun, Poland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Elzbieta","family":"Wiater","sequence":"additional","affiliation":[{"name":"Myeloma and Amyloidosis Treatment Center, Department of Hematology and Bone Marrow Transplantation, Nicolaus Copernicus Hospital, Torun, Poland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jacek","family":"Kwiatkowski","sequence":"additional","affiliation":[{"name":"Hematology and Transplantology Center, Lower Silesian Oncology, Pulmonology and Hematology Center, Wroclaw, Poland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Janusz","family":"Halka","sequence":"additional","affiliation":[{"name":"Department of Hematology and BMT, Clinical Hospital of the Ministry of Internal Affairs and Administration with the Warmia-Masuria Oncology Centre, Olsztyn, Poland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Radoslaw","family":"Bogucki","sequence":"additional","affiliation":[{"name":"Department of Hematology and BMT, Clinical Hospital of the Ministry of Internal Affairs and Administration with the Warmia-Masuria Oncology Centre, Olsztyn, Poland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Wiktoria","family":"Ryzewska","sequence":"additional","affiliation":[{"name":"Department of General Hematology and Internal Medicine, Copernicus Memorial Hospital, Lodz, Poland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Tadeusz","family":"Robak","sequence":"additional","affiliation":[{"name":"Department of General Hematology and Internal Medicine, Copernicus Memorial Hospital, Lodz, Poland"},{"name":"Haematology Clinic, Medical University of Lodz, Lodz, Lodz Voivodeship, Poland"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"194","published-online":{"date-parts":[[2026,5,13]]},"reference":[{"issue":"03","key":"ref1","doi-asserted-by":"crossref","first-page":"495","DOI":"10.1016\/j.hoc.2013.03.001","article-title":"Immune thrombocytopenia","volume":"27","author":"G Kistangari","year":"2013","journal-title":"Hematol Oncol Clin North Am"},{"issue":"03","key":"ref2","doi-asserted-by":"crossref","first-page":"238","DOI":"10.1055\/s-0039-1683416","article-title":"Epidemiology and clinical manifestations of immune thrombocytopenia","volume":"39","author":"R Kohli","year":"2019","journal-title":"Hamostaseologie"},{"key":"ref3","volume-title":"Immune Thrombocytopenia","author":"N M Pietras","year":"2025"},{"key":"ref4","doi-asserted-by":"crossref","first-page":"2445","DOI":"10.2147\/PPA.S505337","article-title":"Impact of primary chronic immune thrombocytopenia and thrombopoietin receptor agonists treatment instructions on daily living: results of a multinational cross-sectional survey","volume":"19","author":"J J Zwaginga","year":"2025","journal-title":"Patient Prefer Adherence"},{"issue":"02","key":"ref5","doi-asserted-by":"crossref","first-page":"51","DOI":"10.5603\/jtm.101708","article-title":"Polish recommendations for the management of ITP in adults developed by the Hemostasis Group of the Polish Society of Hematologists and Transfusiologists\u2014update 2024","volume":"17","author":"K Chojnowski","year":"2024","journal-title":"J Transfus Med Hemost"},{"issue":"23","key":"ref6","doi-asserted-by":"crossref","first-page":"3829","DOI":"10.1182\/bloodadvances.2019000966","article-title":"American Society of Hematology 2019 guidelines for immune thrombocytopenia","volume":"3","author":"C Neunert","year":"2019","journal-title":"Blood Adv"},{"issue":"01","key":"ref7","doi-asserted-by":"crossref","first-page":"86","DOI":"10.1111\/bjh.19077","article-title":"Is splenectomy a good strategy for refractory immune thrombocytopenia in adults?","volume":"203","author":"B Godeau","year":"2023","journal-title":"Br J Haematol"},{"issue":"05","key":"ref8","doi-asserted-by":"crossref","first-page":"963","DOI":"10.1007\/s00277-022-04786-y","article-title":"Refractory primary immune thrombocytopenia (ITP): current clinical challenges and therapeutic perspectives","volume":"101","author":"N Vianelli","year":"2022","journal-title":"Ann Hematol"},{"issue":"12","key":"ref9","doi-asserted-by":"crossref","first-page":"1547","DOI":"10.1182\/blood-2016-03-603365","article-title":"How I treat refractory immune thrombocytopenia","volume":"128","author":"A Cuker","year":"2016","journal-title":"Blood"},{"issue":"01","key":"ref10","doi-asserted-by":"crossref","first-page":"43","DOI":"10.5694\/mja2.51284","article-title":"Consensus guidelines for the management of adult immune thrombocytopenia in Australia and New Zealand","volume":"216","author":"P Y Choi","year":"2022","journal-title":"Med J Aust"},{"issue":"04","key":"ref11","doi-asserted-by":"crossref","first-page":"1267","DOI":"10.1111\/bjh.19777","article-title":"What is the best second-line treatment for immune thrombocytopenia?","volume":"205","author":"M Mah\u00e9vas","year":"2024","journal-title":"Br J Haematol"},{"issue":"02","key":"ref12","doi-asserted-by":"crossref","first-page":"239","DOI":"10.1007\/s00277-015-2556-z","article-title":"The use of thrombopoietin-receptor agonists (TPO-RAs) in immune thrombocytopenia (ITP): a \u201creal life\u201d retrospective multicenter experience of the Rete Ematologica Pugliese (REP)","volume":"95","author":"P Mazza","year":"2016","journal-title":"Ann Hematol"},{"issue":"11","key":"ref13","doi-asserted-by":"crossref","first-page":"1285","DOI":"10.1007\/s40265-021-01553-7","article-title":"Thrombopoietin receptor agonists (TPO-RAs): drug class considerations for pharmacists","volume":"81","author":"J Gilreath","year":"2021","journal-title":"Drugs"},{"issue":"11","key":"ref14","doi-asserted-by":"crossref","first-page":"4607","DOI":"10.1182\/blood-2006-10-019315","article-title":"New thrombopoietic growth factors","volume":"109","author":"D J Kuter","year":"2007","journal-title":"Blood"},{"issue":"10","key":"ref15","doi-asserted-by":"crossref","first-page":"3457","DOI":"10.1182\/blood.V100.10.3457","article-title":"Recombinant human thrombopoietin: basic biology and evaluation of clinical studies","volume":"100","author":"D J Kuter","year":"2002","journal-title":"Blood"},{"issue":"25","key":"ref16","doi-asserted-by":"crossref","first-page":"3887","DOI":"10.1182\/blood-2013-07-514398","article-title":"A randomized trial of avatrombopag, an investigational thrombopoietin-receptor agonist, in persistent and chronic immune thrombocytopenia","volume":"123","author":"J B Bussel","year":"2014","journal-title":"Blood"},{"issue":"03","key":"ref17","doi-asserted-by":"crossref","first-page":"255","DOI":"10.1007\/s12185-014-1731-7","article-title":"Treatment patterns and clinical outcomes in patients with chronic immune thrombocytopenia (ITP) switched to eltrombopag or romiplostim","volume":"101","author":"D J Kuter","year":"2015","journal-title":"Int J Hematol"},{"issue":"06","key":"ref18","doi-asserted-by":"crossref","first-page":"2414","DOI":"10.1111\/bjh.19800","article-title":"Real-world evidence of avatrombopag for the treatment of immune thrombocytopenia intolerant or ineffective to eltrombopag\/hetrombopag","volume":"205","author":"H Tian","year":"2024","journal-title":"Br J Haematol"},{"issue":"06","key":"ref19","doi-asserted-by":"crossref","first-page":"6419","DOI":"10.21037\/apm-21-1067","article-title":"Eltrombopag-induced liver dysfunction during the treatment of immune thrombocytopenia and its risk factors","volume":"10","author":"P Zhang","year":"2021","journal-title":"Ann Palliat Med"},{"issue":"18","key":"ref20","doi-asserted-by":"crossref","first-page":"3748","DOI":"10.1182\/blood-2009-05-224766","article-title":"Evaluation of bone marrow reticulin formation in chronic immune thrombocytopenia patients treated with romiplostim","volume":"114","author":"D J Kuter","year":"2009","journal-title":"Blood"},{"issue":"05","key":"ref21","doi-asserted-by":"crossref","first-page":"733","DOI":"10.1111\/ejh.14395","article-title":"Avatrombopag for the treatment of immune thrombocytopenia","volume":"114","author":"C Labanca","year":"2025","journal-title":"Eur J Haematol"},{"key":"ref22","first-page":"465","article-title":"Novel perspectives on thrombopoietin receptor agonists applications","volume":"42","author":"C Stafylidis","year":"2025","journal-title":"Hamostaseologie"},{"key":"ref23","doi-asserted-by":"crossref","first-page":"273","DOI":"10.2147\/TCRM.S251672","article-title":"Avatrombopag for the treatment of adult patients with chronic immune thrombocytopenia (cITP): focus on patient selection and perspectives","volume":"18","author":"G Tsykunova","year":"2022","journal-title":"Ther Clin Risk Manag"},{"key":"ref24","doi-asserted-by":"crossref","first-page":"313","DOI":"10.2147\/JBM.S191790","article-title":"Avatrombopag for the treatment of immune thrombocytopenia and thrombocytopenia of chronic liver disease","volume":"10","author":"A Z Cheloff","year":"2019","journal-title":"J Blood Med"},{"issue":"16","key":"ref25","doi-asserted-by":"crossref","first-page":"1905","DOI":"10.1007\/s40265-021-01613-y","article-title":"Avatrombopag: a review in thrombocytopenia","volume":"81","author":"A Markham","year":"2021","journal-title":"Drugs"},{"issue":"03","key":"ref26","doi-asserted-by":"crossref","first-page":"479","DOI":"10.1111\/bjh.15573","article-title":"Phase 3 randomised study of avatrombopag, a novel thrombopoietin receptor agonist for the treatment of chronic immune thrombocytopenia","volume":"183","author":"W Jurczak","year":"2018","journal-title":"Br J Haematol"},{"issue":"06","key":"ref27","doi-asserted-by":"crossref","first-page":"3113","DOI":"10.1007\/s12325-021-01752-4","article-title":"Efficacy and safety of avatrombopag in patients with chronic immune thrombocytopenia: a systematic literature review and network meta-analysis","volume":"38","author":"P Wojciechowski","year":"2021","journal-title":"Adv Ther"},{"issue":"03","key":"ref28","doi-asserted-by":"crossref","first-page":"44","DOI":"10.3892\/br.2024.1732","article-title":"Thrombopoietin receptor agonists use and risk of thrombotic events in patients with immune thrombocytopenic purpura: a systematic review and meta\u2013analysis of randomized controlled trials","volume":"20","author":"N Shen","year":"2024","journal-title":"Biomed Rep"},{"issue":"12","key":"ref29","doi-asserted-by":"crossref","first-page":"511","DOI":"10.1016\/j.medcli.2015.03.014","article-title":"Risk of thromboembolism with thrombopoietin receptor agonists in adult patients with thrombocytopenia: systematic review and meta-analysis of randomized controlled trials","volume":"145","author":"F Catal\u00e1-L\u00f3pez","year":"2015","journal-title":"Med Clin (Barc)"},{"key":"ref30","doi-asserted-by":"crossref","first-page":"103581","DOI":"10.1016\/j.critrevonc.2022.103581","article-title":"Risk of thrombosis with thrombopoietin receptor agonists for ITP patients: a systematic review and meta-analysis","volume":"171","author":"M Tjepkema","year":"2022","journal-title":"Crit Rev Oncol Hematol"},{"issue":"S 01","key":"ref34","doi-asserted-by":"crossref","first-page":"3952","DOI":"10.1182\/blood-2023-182848","article-title":"Real-world use, effectiveness and safety of romiplostim, eltrombopag and avatrombopag in immune thrombocytopenia: data from the Norwegian ITP Registry","volume":"142","author":"R Tomasello","year":"2023","journal-title":"Blood"},{"issue":"04","key":"ref35","doi-asserted-by":"crossref","first-page":"659","DOI":"10.3390\/medicina59040659","article-title":"Sustained remission off-treatment (SROT) of TPO-RAs: the Burgos Ten-Step Eltrombopag Tapering Scheme","volume":"59","author":"T J Gonz\u00e1lez-L\u00f3pez","year":"2023","journal-title":"Medicina (Kaunas)"},{"issue":"12","key":"ref36","doi-asserted-by":"crossref","first-page":"1501","DOI":"10.1002\/ajh.25275","article-title":"Thrombopoietin level predicts response to treatment with eltrombopag and romiplostim in immune thrombocytopenia","volume":"93","author":"H Al-Samkari","year":"2018","journal-title":"Am J Hematol"},{"issue":"12","key":"ref37","doi-asserted-by":"crossref","first-page":"2515","DOI":"10.1111\/jth.14295","article-title":"Avatrombopag increases platelet count but not platelet activation in patients with thrombocytopenia resulting from liver disease","volume":"16","author":"A D Michelson","year":"2018","journal-title":"J Thromb Haemost"}],"container-title":["TH Open"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/www.thieme-connect.de\/products\/ejournals\/pdf\/10.1055\/a-2868-3290.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,5,13]],"date-time":"2026-05-13T23:25:02Z","timestamp":1778714702000},"score":1,"resource":{"primary":{"URL":"http:\/\/www.thieme-connect.de\/DOI\/DOI?10.1055\/a-2868-3290"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,5,4]]},"references-count":34,"journal-issue":{"issue":"CP","published-online":{"date-parts":[[2026,1,9]]},"published-print":{"date-parts":[[2026]]}},"alternative-id":["a28683290"],"URL":"https:\/\/doi.org\/10.1055\/a-2868-3290","archive":["Portico","CLOCKSS"],"relation":{},"ISSN":["2512-9465"],"issn-type":[{"value":"2512-9465","type":"electronic"}],"subject":[],"published-other":{"date-parts":[[2026,5,4]]},"published":{"date-parts":[[2026,5,4]]}}}