{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,1]],"date-time":"2026-06-01T18:08:16Z","timestamp":1780337296515,"version":"3.54.1"},"reference-count":28,"publisher":"MDPI AG","issue":"11","license":[{"start":{"date-parts":[[2026,5,28]],"date-time":"2026-05-28T00:00:00Z","timestamp":1779926400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JCM"],"abstract":"<jats:p>Background: Acute graft-versus-host disease (aGVHD) is a leading cause of morbidity and mortality following pediatric hematopoietic stem cell transplantation (HSCT). Approximately half of children achieve complete response (CR) to corticosteroids, whereas steroid-refractory (SR) disease carries a 1\u20132-year mortality of 41\u201344%. Mortality risk is 2.6-fold higher in children &gt; 13.9 years, and respiratory failure accounts for 26% of deaths. Existing second-line agents\u2014ruxolitinib, tocilizumab, or extracorporeal photopheresis\u2014have delayed onset or high toxicity. Begelomab (BEGESAND\u00ae), a monoclonal antibody targeting CD26\/dipeptidyl peptidase-4 (DPP4), inhibits CD26-mediated T-cell activation and has demonstrated 75% response in adults with minimal toxicity. However, pediatric data are lacking. Methods: We retrospectively reviewed five consecutive pediatric patients (ages 3\u201320 years) treated with Begelomab (BEGESAND\u00ae) for SR (n = 4) or steroid-dependent (SD; n = 1) aGVHD between 2017\u20132021 under emergency IND authorization. Begelomab (BEGESAND\u00ae) was administered intravenously at 2.7 mg\/m2\/day on days 1\u20135, 10, 14, 17, 21, 24, and 28. GVHD was graded by MAGIC criteria; flow cytometry and immunohistochemistry (IHC) assessed CD26 expression and immune effects. Results: All patients had grade IV disease after \u22652 prior agents. Two with pre-existing sepsis died early, before treatment response could be assessed. Of three evaluable patients, two (67%) achieved CR within 21 days and one achieved durable control by 6 months. All three remain alive; no Begelomab (BEGESAND\u00ae)-related toxicity, cytopenia, or new infections occurred. Flow cytometry showed preserved T-cell subsets, and IHC demonstrated CD26 localization at sites of epithelial injury. Conclusions: Begelomab (BEGESAND\u00ae) showed promising timely and durable responses with excellent safety in pediatric SR\/SD-aGVHD, supporting further evaluation in multicenter pediatric trials.<\/jats:p>","DOI":"10.3390\/jcm15114190","type":"journal-article","created":{"date-parts":[[2026,5,29]],"date-time":"2026-05-29T17:36:18Z","timestamp":1780076178000},"page":"4190","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Begelomab (BEGESAND\u00ae) Salvages Steroid-Resistant Acute GVHD in Pediatric Patients"],"prefix":"10.3390","volume":"15","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-1111-5335","authenticated-orcid":false,"given":"David","family":"Shyr","sequence":"first","affiliation":[{"name":"School of Medicine, Stanford University, Stanford, CA 94305, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3997-5652","authenticated-orcid":false,"given":"Steven M.","family":"Chirieleison","sequence":"additional","affiliation":[{"name":"School of Medicine, Stanford University, Stanford, CA 94305, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sebastian","family":"Fernandez-Pol","sequence":"additional","affiliation":[{"name":"School of Medicine, Stanford University, Stanford, CA 94305, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katja","family":"Weinacht","sequence":"additional","affiliation":[{"name":"School of Medicine, Stanford University, Stanford, CA 94305, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3865-8866","authenticated-orcid":false,"given":"Rajni","family":"Agarwal","sequence":"additional","affiliation":[{"name":"School of Medicine, Stanford University, Stanford, CA 94305, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5700-6188","authenticated-orcid":false,"given":"Ami J.","family":"Shah","sequence":"additional","affiliation":[{"name":"School of Medicine, Stanford University, Stanford, CA 94305, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0000-0122-8865","authenticated-orcid":false,"given":"Michela","family":"Spinelli","sequence":"additional","affiliation":[{"name":"Adienne Pharma & Biotech, 6900 Lugano, Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Renata","family":"Palmieri","sequence":"additional","affiliation":[{"name":"Adienne Pharma & Biotech, 6900 Lugano, Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Antonio Francesco","family":"Di Naro","sequence":"additional","affiliation":[{"name":"Adienne Pharma & Biotech, 6900 Lugano, Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alice","family":"Bertaina","sequence":"additional","affiliation":[{"name":"School of Medicine, Stanford University, Stanford, CA 94305, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2026,5,28]]},"reference":[{"key":"ref_1","first-page":"505","article-title":"Current Activity Trends and Outcomes in Hematopoietic Cell Transplantation and Cellular Therapy\u2014A Report from the CIBMTR","volume":"31","author":"Spellman","year":"2025","journal-title":"Transpl. 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