{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,10]],"date-time":"2026-09-10T00:37:12Z","timestamp":1789000632767,"version":"build-2803163510"},"reference-count":32,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2024,10,20]],"date-time":"2024-10-20T00:00:00Z","timestamp":1729382400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100000163","name":"Pediatric Oncology Group of Ontario","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100000163","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pediatric Blood &amp; Cancer"],"published-print":{"date-parts":[[2025,1]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>The Children's Oncology Group (COG)\u2010AALL0434 trial investigated the addition of nelarabine to the augmented Berlin\u2013Frankfurt\u2013M\u00fcnster (aBFM) protocol in patients (1.0\u201330.99\u00a0years) with newly diagnosed T\u2010cell acute lymphoblastic leukemia (T\u2010ALL). Despite demonstrating superior outcomes, nelarabine is not currently funded by many health systems, in part due to a lack of cost\u2010effectiveness data. We estimated the cost\u2013utility of nelarabine for this indication from a Canadian public healthcare payer perspective.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>We developed a microsimulation model that followed hypothetical patients with newly diagnosed T\u2010ALL from post\u2010induction therapy to death. Three health states were modeled: relapse\u2010free, post\u2010relapse, and death. Efficacy was estimated using AALL0434 and retrospective data from Ontario, Canada. Costs were obtained from Canadian sources. Utility estimates and long\u2010term mortality risks were sourced from literature. Total healthcare costs, quality\u2010adjusted life\u2010years (QALYs), and incremental cost\u2010effectiveness ratio (ICER) were reported. Probabilistic and scenario analyses were conducted.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Incorporating nelarabine in the aBFM protocol increased costs by $51,670 Canadian dollars per patient, but resulted in 1.97 more QALYs and an ICER of $26,184\/QALY. Most of the identified cost and benefit were accrued within the AALL0434 trial period (first 11\u00a0years post diagnosis) and while patients were in the relapse\u2010free health state. Across multiple scenarios, the ICER was stable under an assumed $50,000\/QALY threshold.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>Incorporating nelarabine into aBFM was cost\u2010effective across different scenarios and assumptions. These results support its funding by public and private payers.<\/jats:p><\/jats:sec>","DOI":"10.1002\/pbc.31393","type":"journal-article","created":{"date-parts":[[2024,10,21]],"date-time":"2024-10-21T04:29:18Z","timestamp":1729484958000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Cost\u2013utility of nelarabine for the first\u2010line treatment of newly diagnosed pediatric T\u2010cell acute lymphoblastic leukemia in Canada"],"prefix":"10.1002","volume":"72","author":[{"given":"Roaa","family":"Shoukry","sequence":"first","affiliation":[{"name":"The Hospital for Sick Children Research Institute Toronto Ontario Canada"},{"name":"Institute of Health Policy, Management and Evaluation, University of Toronto Toronto Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2715-1135","authenticated-orcid":false,"given":"Alexandra","family":"Moskalewicz","sequence":"additional","affiliation":[{"name":"The Hospital for Sick Children Research Institute Toronto Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicole","family":"Bradley","sequence":"additional","affiliation":[{"name":"The Pediatric Oncology Group of Ontario Toronto Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elizabeth","family":"Bond","sequence":"additional","affiliation":[{"name":"The Hospital for Sick Children Research Institute Toronto Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mandy","family":"Sala","sequence":"additional","affiliation":[{"name":"The Pediatric Oncology Group of Ontario Toronto Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1334-3670","authenticated-orcid":false,"given":"Sumit","family":"Gupta","sequence":"additional","affiliation":[{"name":"The Hospital for Sick Children Research Institute Toronto Ontario Canada"},{"name":"Institute of Health Policy, Management and Evaluation, University of Toronto Toronto Canada"},{"name":"Division of Haematology\/Oncology The Hospital for Sick Children Toronto Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paul","family":"Gibson","sequence":"additional","affiliation":[{"name":"The Pediatric Oncology Group of Ontario Toronto Ontario Canada"},{"name":"Division of Hematology and Oncology McMaster Children's Hospital Hamilton Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5090-7936","authenticated-orcid":false,"given":"Petros","family":"Pechlivanoglou","sequence":"additional","affiliation":[{"name":"The Hospital for Sick Children Research Institute Toronto Ontario Canada"},{"name":"Institute of Health Policy, Management and Evaluation, University of Toronto Toronto Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2024,10,20]]},"reference":[{"key":"e_1_2_10_2_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMra1400972"},{"key":"e_1_2_10_3_1","doi-asserted-by":"publisher","DOI":"10.1002\/mpo.2950090405"},{"key":"e_1_2_10_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1470\u20102045(19)30031\u20102"},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.1055\/s\u20100041\u20101730752"},{"key":"e_1_2_10_6_1","doi-asserted-by":"publisher","DOI":"10.1200\/JCO.2019.37.15_suppl.10008"},{"key":"e_1_2_10_7_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41375\u2010022\u201001714\u2010x"},{"key":"e_1_2_10_8_1","doi-asserted-by":"publisher","DOI":"10.1200\/JCO.2011.40.8724"},{"key":"e_1_2_10_9_1","doi-asserted-by":"publisher","DOI":"10.1200\/JCO.20.00256"},{"key":"e_1_2_10_10_1","doi-asserted-by":"publisher","DOI":"10.3390\/ijms22094502"},{"key":"e_1_2_10_11_1","doi-asserted-by":"publisher","DOI":"10.1002\/pbc.29238"},{"key":"e_1_2_10_12_1","doi-asserted-by":"publisher","DOI":"10.9778\/cmajo.20170144"},{"key":"e_1_2_10_13_1","doi-asserted-by":"publisher","DOI":"10.1093\/heapol\/czp002"},{"key":"e_1_2_10_14_1","unstructured":"Guidelines for the economic evaluation of health technologies: Canada.CADTH. 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