{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,4]],"date-time":"2026-08-04T04:11:28Z","timestamp":1785816688954,"version":"3.56.0"},"reference-count":20,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2022,1,19]],"date-time":"2022-01-19T00:00:00Z","timestamp":1642550400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pediatric Blood &amp; Cancer"],"published-print":{"date-parts":[[2022,4]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Prior to regulatory approval of blinatumomab in pediatric patients with relapsed\/refractory Philadelphia chromosome\u2010negative B\u2010cell precursor acute lymphoblastic leukemia (R\/R Ph\u2212 BCP\u2010ALL), blinatumomab was made available via an expanded access program (EAP).<\/jats:p><\/jats:sec><jats:sec><jats:title>Procedure<\/jats:title><jats:p>This retrospective observational study included patients receiving blinatumomab in the EAP between January 1, 2014 and June 30, 2017 who were followed until death, entry into a clinical trial, end of follow\u2010up, or end of the study period (December 31, 2017), whichever occurred first.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Among 113 children enrolled, 72 were diagnosed with R\/R Ph\u2212 BCP\u2010ALL and 41 were minimal residual disease positive (MRD+, either Ph\u2212 or Ph+). In the R\/R group, 38 (53%) patients achieved hematological response within two cycles. Of these, 19 (50%) proceeded to hematopoietic stem cell transplantation (HSCT) without bridging myelosuppressive therapy. Of 36 patients in the R\/R group evaluable for MRD, 30 (83%) had an MRD response. In the R\/R group, median relapse\u2010free survival was 5.4\u00a0months and median overall survival (OS) was 8.2\u00a0months. Of 36 patients in the MRD+ group who were evaluable for MRD after two cycles, 27 (75%) had an MRD response. Overall, 24 (59%) of the MRD+ patients proceeded to HSCT without other bridging therapy. Median disease\u2010free survival was 13.6\u00a0months; median OS was not reached.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>In this real\u2010world pediatric cohort, blinatumomab was effective within two cycles. Over half of patients with R\/R Ph\u2212 BCP\u2010ALL achieved hematological response and most achieved MRD response in the MRD+ group, confirming the efficacy of blinatumomab in pediatric trials.<\/jats:p><\/jats:sec>","DOI":"10.1002\/pbc.29562","type":"journal-article","created":{"date-parts":[[2022,1,19]],"date-time":"2022-01-19T10:15:03Z","timestamp":1642587303000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":23,"title":["Pediatric patients with acute lymphoblastic leukemia treated with blinatumomab in a real\u2010world setting: Results from the NEUF study"],"prefix":"10.1002","volume":"69","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-7976-3654","authenticated-orcid":false,"given":"Franco","family":"Locatelli","sequence":"first","affiliation":[{"name":"Department of Pediatric Hematology and Oncology IRCCS, Ospedale Pediatrico Bambino Ges\u00f9  Rome Italy"},{"name":"Sapienza, University of Rome  Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0016-6698","authenticated-orcid":false,"given":"Alexey","family":"Maschan","sequence":"additional","affiliation":[{"name":"Oncology and Immunology Dmitri Rogachev National Research Centre for Pediatric Hematology  Moscow Russian Federation"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicolas","family":"Boissel","sequence":"additional","affiliation":[{"name":"Division of Hematology EA3518 Saint\u2010Louis Institute for Research, Saint\u2010Louis Hospital  Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Luisa","family":"Strocchio","sequence":"additional","affiliation":[{"name":"Department of Pediatric Hematology and Oncology IRCCS, Ospedale Pediatrico Bambino Ges\u00f9  Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Naufil","family":"Alam","sequence":"additional","affiliation":[{"name":"Center for Observational Research, Amgen Ltd  Uxbridge UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Isabella","family":"Pezzani","sequence":"additional","affiliation":[{"name":"Amgen (Europe) GmbH  Rotkreuz Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alessandra","family":"Brescianini","sequence":"additional","affiliation":[{"name":"Amgen (Europe) GmbH  Rotkreuz Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Georg","family":"Kreuzbauer","sequence":"additional","affiliation":[{"name":"Amgen (Europe) GmbH  Rotkreuz Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andre","family":"Baruchel","sequence":"additional","affiliation":[{"name":"H\u00f4pital Universitaire Robert Debr\u00e9  Paris France"},{"name":"Universit\u00e9 de Paris  Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2022,1,19]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.pcl.2014.09.006"},{"key":"e_1_2_9_3_1","doi-asserted-by":"publisher","DOI":"10.1038\/srep04227"},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMra1400972"},{"key":"e_1_2_9_5_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41375\u2010020\u20100770\u20108"},{"key":"e_1_2_9_6_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2012-02-265884"},{"key":"e_1_2_9_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1470-2045(12)70580-6"},{"key":"e_1_2_9_8_1","doi-asserted-by":"publisher","DOI":"10.1080\/14737140.2017.1347507"},{"key":"e_1_2_9_9_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood\u20102017\u201008\u2010798322"},{"key":"e_1_2_9_10_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood\u20102015\u201003\u2010580027"},{"key":"e_1_2_9_11_1","doi-asserted-by":"publisher","DOI":"10.1080\/16078454.2019.1567654"},{"key":"e_1_2_9_12_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood\u20102015\u201003\u2010633685"},{"key":"e_1_2_9_13_1","doi-asserted-by":"publisher","DOI":"10.1200\/JCO.2016.67.3301"},{"key":"e_1_2_9_14_1","doi-asserted-by":"publisher","DOI":"10.1001\/jama.2021.0987"},{"key":"e_1_2_9_15_1","doi-asserted-by":"publisher","DOI":"10.1001\/jama.2021.0669"},{"key":"e_1_2_9_16_1","volume-title":"BLINCYTO summary of product characteristics","author":"European Medicines Agency"},{"key":"e_1_2_9_17_1","volume-title":"BLINCYTO: European public assessment report procedural steps","author":"European Medicines Agency","year":"2021"},{"key":"e_1_2_9_18_1","doi-asserted-by":"publisher","DOI":"10.1182\/bloodadvances.2021005579"},{"key":"e_1_2_9_19_1","volume-title":"Observational study of blinatumomab","author":"NIH"},{"issue":"1","key":"e_1_2_9_20_1","doi-asserted-by":"crossref","first-page":"24","DOI":"10.1182\/blood-2020-141336","article-title":"Blinatumomab in children with relapsed or refractory B\u2010precursor acute lymphoblastic leukemia (R\/R\u2010ALL): final results of 110 patients treated in an expanded access study (RIALTO)","volume":"136","author":"Locatelli F","year":"2020","journal-title":"Blood"},{"key":"e_1_2_9_21_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2019-121615"}],"updated-by":[{"DOI":"10.1002\/pbc.29870","type":"erratum","label":"Erratum","source":"publisher","updated":{"date-parts":[[2022,7,12]],"date-time":"2022-07-12T00:00:00Z","timestamp":1657584000000}}],"container-title":["Pediatric Blood &amp; Cancer"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/pbc.29562","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/pbc.29562","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/pbc.29562","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,8,27]],"date-time":"2023-08-27T16:54:13Z","timestamp":1693155253000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/pbc.29562"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,1,19]]},"references-count":20,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2022,4]]}},"alternative-id":["10.1002\/pbc.29562"],"URL":"https:\/\/doi.org\/10.1002\/pbc.29562","archive":["Portico"],"relation":{"erratum":[{"id-type":"doi","id":"10.1002\/pbc.29870","asserted-by":"object"}]},"ISSN":["1545-5009","1545-5017"],"issn-type":[{"value":"1545-5009","type":"print"},{"value":"1545-5017","type":"electronic"}],"subject":[],"published":{"date-parts":[[2022,1,19]]},"assertion":[{"value":"2021-08-11","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2021-12-20","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2022-01-19","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}],"article-number":"e29562"}}