{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T14:02:00Z","timestamp":1790863320893,"version":"4.1.0"},"reference-count":0,"publisher":"American Society of Hematology","license":[{"start":{"date-parts":[[2023,3,9]],"date-time":"2023-03-09T00:00:00Z","timestamp":1678320000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"abstract":"<jats:p>Sustained response off-treatment (SROT) after thrombopoietin receptor agonist (TPO-RA) discontinuation has been reported in ITP. This prospective multicenter interventional study enrolled adults with persistent or chronic primary ITP and complete response on TPO-RAs. The primary endpoint was the proportion of patients achieving SROT (platelet count &amp;gt; 30x109\/L and no bleeding) at W24 with no other ITP-specific medications. Secondary endpoints included the proportion of sustained complete response off-treatment (SCROT, platelet count &amp;gt; 100x109\/L and no bleeding) and SROT at W52, bleeding events, and pattern of response to a new course of TPO-RAs. We included 48 patients with median (IQR) age 58.5 years (41-73.5); 30\/48 (63%) had chronic ITP at TPO-RA initiation. In the intention-to-treat analysis, 27\/48 (56.2%, 95% CI, 41.2-70.5) achieved SROT; 15\/48 (31.3%; 95% CI, 18.9-44.5) achieved SCROT at W24, and 25\/48 (52.1%; 95% CI, 37.2-66.7) achieved respectively SROT and 14\/48 (29.2%; 95% CI, 17.2-42.3) SCROT at W52. No severe bleeding episode occurred in patients who relapsed. Among patients re-challenged with TPO-RA, 11\/12 achieved CR. We found no significant clinical predictors of SROT at W24. Single-cell RNA-seq revealed enrichment of a \"TNF\u03b1 signaling via NF-\u03baB\" signature in CD8+ T cells of patients with no sustained response after TPO-RA discontinuation, which was further confirmed by a significant overexpression of CD69 on CD8+ T cells at baseline in these patients as compared with those achieving SCROT\/SROT. Our results strongly support a strategy based of progressive tapering and discontinuation of TPO-RAs for patients with chronic ITP who achieved a stable CR on treatment. Clinical trial number: NCT03119974<\/jats:p>","DOI":"10.1182\/blood.2022018665","type":"journal-article","created":{"date-parts":[[2023,3,9]],"date-time":"2023-03-09T22:14:31Z","timestamp":1678400071000},"update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":14,"title":["Prolonged response after TPO-RA discontinuation in primary ITP: results of a prospective multicenter study"],"prefix":"10.1182","author":[{"given":"Stephanie","family":"Guillet","sequence":"first","affiliation":[{"name":"Department of Internal Medecine, Henri Mondor University Hospital, Creteil, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3964-4968","authenticated-orcid":false,"given":"Etienne","family":"Crickx","sequence":"additional","affiliation":[{"name":"Institut Necker Enfants Malades (INEM), INSERM U1151\/CNRS UMS 8253, ATIP-Avenir TeamAI2B, 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