{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,29]],"date-time":"2026-09-29T01:57:03Z","timestamp":1790647023639,"version":"4.1.0"},"reference-count":24,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2017,8,2]],"date-time":"2017-08-02T00:00:00Z","timestamp":1501632000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100004330","name":"GlaxoSmithKline","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004330","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100004336","name":"Novartis","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004336","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100008272","name":"Novartis Pharmaceuticals Corporation","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100008272","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Br J Haematol"],"published-print":{"date-parts":[[2017,10]]},"abstract":"<jats:title>Summary<\/jats:title><jats:p>Nelarabine is an antineoplastic agent approved for the treatment of relapsed\/refractory T\u2010lineage acute lymphoblastic leukaemia (T\u2010<jats:styled-content style=\"fixed-case\">ALL<\/jats:styled-content>) or T\u2010lineage acute lymphoblastic lymphoma (T\u2010<jats:styled-content style=\"fixed-case\">LBL<\/jats:styled-content>). The purpose of this phase 4, multicentre, single\u2010arm, observational, open\u2010label trial was to provide additional data on the safety and efficacy of nelarabine under licensed conditions of use in children and young adults \u226421\u00a0years of age. Patients (<jats:italic>N\u00a0<\/jats:italic>=<jats:italic>\u00a0<\/jats:italic>28) had a mean\u00a0\u00b1\u00a0standard deviation age of 11\u00b75\u00a0\u00b1\u00a04\u00b76\u00a0years; 71% were male and 61% had a diagnosis of T\u2010<jats:styled-content style=\"fixed-case\">ALL<\/jats:styled-content>. Adverse events (<jats:styled-content style=\"fixed-case\">AE<\/jats:styled-content>s) and treatment\u2010related <jats:styled-content style=\"fixed-case\">AE<\/jats:styled-content>s were experienced by 46% and 21%, respectively, and included few haematological <jats:styled-content style=\"fixed-case\">AE<\/jats:styled-content>s and no haematological serious <jats:styled-content style=\"fixed-case\">AE<\/jats:styled-content>s. Neurological <jats:styled-content style=\"fixed-case\">AE<\/jats:styled-content>s from one of four predefined categories (peripheral and central nervous systems, mental status change and uncategorized) were reported in four patients. There were no <jats:styled-content style=\"fixed-case\">AE<\/jats:styled-content>\u2010related treatment discontinuations\/withdrawals. The overall response rate was 39.3%: complete response (<jats:styled-content style=\"fixed-case\">CR<\/jats:styled-content>), 35.7%; <jats:styled-content style=\"fixed-case\">CR<\/jats:styled-content> without full haematological recovery (<jats:styled-content style=\"fixed-case\">CR<\/jats:styled-content>*), 3.6%. Post\u2010treatment stem cell transplantation was performed for 46% of the cohort. Median overall survival (<jats:styled-content style=\"fixed-case\">OS<\/jats:styled-content>) was 3\u00b735\u00a0months for non\u2010responders and not reached for responders (<jats:styled-content style=\"fixed-case\">CR<\/jats:styled-content>\u00a0+\u00a0<jats:styled-content style=\"fixed-case\">CR<\/jats:styled-content>*). The response rate, median <jats:styled-content style=\"fixed-case\">OS<\/jats:styled-content>, and safety profile of nelarabine in this disease setting and population were consistent with those reported previously.<\/jats:p>","DOI":"10.1111\/bjh.14874","type":"journal-article","created":{"date-parts":[[2017,8,3]],"date-time":"2017-08-03T19:30:52Z","timestamp":1501788652000},"page":"284-293","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":28,"title":["Safety and efficacy of nelarabine in children and young adults with relapsed or refractory T\u2010lineage acute lymphoblastic leukaemia or T\u2010lineage lymphoblastic lymphoma: results of a phase 4 study"],"prefix":"10.1111","volume":"179","author":[{"given":"Christian Michel","family":"Zwaan","sequence":"first","affiliation":[{"name":"Department of Paediatric Oncology Erasmus MC\u2010Sophia Children's Hospital Rotterdam The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jerzy","family":"Kowalczyk","sequence":"additional","affiliation":[{"name":"Department of Paediatric Haematology, Oncology and Transplantology Medical University Lublin Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Claudine","family":"Schmitt","sequence":"additional","affiliation":[{"name":"Department of Haematology\u2010Oncology\u2010Paediatrics Children's Hospital Nancy France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bella","family":"Bielorai","sequence":"additional","affiliation":[{"name":"Department of Paediatric Haematology\u2010Oncology &amp; BMT Sheba Medical Centre Ramat Gan Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mark W.","family":"Russo","sequence":"additional","affiliation":[{"name":"Novartis Pharmaceuticals Corporation East Hanover NJ USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mary","family":"Woessner","sequence":"additional","affiliation":[{"name":"Novartis Pharmaceuticals Corporation East Hanover NJ USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sulabha","family":"Ranganathan","sequence":"additional","affiliation":[{"name":"Adaptimmune LLC Philadelphia PA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Guy","family":"Leverger","sequence":"additional","affiliation":[{"name":"Department of Paediatric Onco\u2010Haematology APHP Armand Trousseau Hospital Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2017,8,2]]},"reference":[{"key":"e_1_2_10_2_1","doi-asserted-by":"publisher","DOI":"10.1111\/ejh.12722"},{"key":"e_1_2_10_3_1","first-page":"70","article-title":"Treatment of refractory t\u2010lymphoblastic leukaemia and lymphoma with nelarabine","volume":"2","author":"Bauters T.","year":"2011","journal-title":"Belgian Journal of Hematology"},{"key":"e_1_2_10_4_1","doi-asserted-by":"publisher","DOI":"10.1200\/JCO.2005.03.426"},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.1200\/JCO.2008.19.3367"},{"key":"e_1_2_10_6_1","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.14017"},{"key":"e_1_2_10_7_1","doi-asserted-by":"crossref","first-page":"660","DOI":"10.1182\/blood.V61.4.660.660","article-title":"Selective toxicity of deoxyguanosine and arabinosyl guanine for T\u2010leukemic cells","volume":"61","author":"Cohen A.","year":"1983","journal-title":"Blood"},{"key":"e_1_2_10_8_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2006-11-056754"},{"key":"e_1_2_10_9_1","doi-asserted-by":"publisher","DOI":"10.1200\/JCO.2011.40.8724"},{"key":"e_1_2_10_10_1","doi-asserted-by":"publisher","DOI":"10.1155\/2014\/359158"},{"key":"e_1_2_10_11_1","doi-asserted-by":"publisher","DOI":"10.1200\/JCO.2012.45.4728"},{"key":"e_1_2_10_12_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2011-02-337840"},{"key":"e_1_2_10_13_1","doi-asserted-by":"publisher","DOI":"10.1200\/JCO.2005.03.199"},{"key":"e_1_2_10_14_1","doi-asserted-by":"publisher","DOI":"10.1200\/JCO.2014.59.5827"},{"key":"e_1_2_10_15_1","unstructured":"National Cancer Institute(2013)FDA approval for nelarabine. 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