{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,8]],"date-time":"2026-06-08T06:00:55Z","timestamp":1780898455431,"version":"3.54.1"},"reference-count":112,"publisher":"MDPI AG","issue":"12","license":[{"start":{"date-parts":[[2026,6,7]],"date-time":"2026-06-07T00:00:00Z","timestamp":1780790400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Saving Kids with Cancer Foundation","award":["KPOD.07.07-IW.07-0153\/24-00"],"award-info":[{"award-number":["KPOD.07.07-IW.07-0153\/24-00"]}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["IJMS"],"abstract":"<jats:p>Thrombopoietin receptor agonists (TPO-RAs) have become an important component of pediatric immune thrombocytopenia (ITP) management, with growing evidence supporting their use in other hematologic disorders. We conducted a retrospective single-center study evaluating TPO-RA use at a pediatric oncohematology center in Poland between 1 January 2016, and 31 March 2026. Clinical indications, treatment patterns, efficacy, and safety outcomes were analyzed. Treatment response was defined as initial response (IR, platelet count [PLT] &gt; 50 \u00d7 109\/L), complete response (CR, PLT &gt; 100 \u00d7 109\/L), and sustained response (SR, PLT &gt; 50 \u00d7 109\/L maintained in \u226575% of visits over 6 months). Thirty-five patients were included in the analysis. The most common indication was ITP (27\/35, 77%), followed by severe aplastic anemia (5\/35, 14%), poor graft function (2\/35, 6%), and inherited platelet disorders (1\/35, 3%). TPO-RA use increased over time, with more than half of patients (20\/35, 57%) initiating therapy between 2024 and 2026. Overall IR, CR, and SR rates were 77%, 63%, and 61%, respectively, with a median time to response of 7 days (interquartile range [IQR], 7\u201312.5). TPO-RAs were increasingly used earlier in the disease course, including in rescue and off-label settings. Treatment was well tolerated, with only grade 1\u20132 adverse events reported. Our real-world data confirm the high efficacy and favorable safety profile of TPO-RAs in pediatric patients and support their expanding role in pediatric hematology beyond disease-specific indications.<\/jats:p>","DOI":"10.3390\/ijms27125175","type":"journal-article","created":{"date-parts":[[2026,6,8]],"date-time":"2026-06-08T05:43:46Z","timestamp":1780897426000},"page":"5175","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Evolving Patterns of TPO-RA Use in Children: A Decade of Single-Centre Experience and Narrative Review"],"prefix":"10.3390","volume":"27","author":[{"ORCID":"https:\/\/orcid.org\/0009-0008-5399-4703","authenticated-orcid":false,"given":"Bartosz","family":"Urba\u0144ski","sequence":"first","affiliation":[{"name":"Department of Pediatrics, Oncology and Hematology, Medical University of Lodz, 251 Pomorska Street, 92-213 Lodz, Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ma\u0142gorzata","family":"Gaszy\u0144ska","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Oncology and Hematology, Medical University of Lodz, 251 Pomorska Street, 92-213 Lodz, Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Izabela","family":"Kasprzycka","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Oncology and Hematology, Medical University of Lodz, 251 Pomorska Street, 92-213 Lodz, Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Olga","family":"Kowalczyk","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Oncology and Hematology, Medical University of Lodz, 251 Pomorska Street, 92-213 Lodz, Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Olga","family":"Wegner","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Oncology and Hematology, Medical University of Lodz, 251 Pomorska Street, 92-213 Lodz, Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Magdalena","family":"Wojdalska","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Oncology and Hematology, Medical University of Lodz, 251 Pomorska Street, 92-213 Lodz, Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Monika","family":"Zjawiona","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Oncology and Hematology, Medical University of Lodz, 251 Pomorska Street, 92-213 Lodz, Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2714-5851","authenticated-orcid":false,"given":"Wojciech","family":"M\u0142ynarski","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Oncology and Hematology, Medical University of Lodz, 251 Pomorska Street, 92-213 Lodz, Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8007-6969","authenticated-orcid":false,"given":"Szymon","family":"Janczar","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Oncology and Hematology, Medical University of Lodz, 251 Pomorska Street, 92-213 Lodz, Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2026,6,7]]},"reference":[{"key":"ref_1","first-page":"350","article-title":"Demonstration and some properties of human thrombopoietin in thrombocythaemic sera","volume":"20","author":"Kelemen","year":"1958","journal-title":"Acta Haematol."},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"5640","DOI":"10.1073\/pnas.89.12.5640","article-title":"Molecular cloning and characterization of MPL, the human homolog of the v-mpl oncogene: Identification of a member of the hematopoietic growth factor receptor superfamily","volume":"89","author":"Vigon","year":"1992","journal-title":"Proc. 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