{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,1,24]],"date-time":"2026-01-24T06:43:05Z","timestamp":1769236985420,"version":"3.49.0"},"reference-count":31,"publisher":"American Society of Hematology","issue":"14","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2021,7,27]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:p>The phase 2, open-label study (DIALOG) of nilotinib in pediatric patients with Philadelphia chromosome-positive chronic myelogenous leukemia (CML) met its coprimary end points, showing sustained nilotinib efficacy in patients with newly diagnosed (ND) or imatinib\/dasatinib resistant\/intolerant (R\/I) CML. This update assessed growth and safety profiles in patients who had completed \u226548, 28-day treatment cycles of nilotinib 230 mg\/m2 twice daily, or previously discontinued the study. Height was assessed regularly and reported using standard deviation scores (SDSs) based on World Health Organization growth charts. All data were summarized descriptively (cutoff, 6 March 2019). Overall, 33 patients in the R\/I cohort and 25 patients in the ND cohort received nilotinib. Each cohort showed a negative slope in height SDS over the course of the study, indicating attenuated growth rates during nilotinib treatment: overall median change from baseline in height SDS after 48 cycles was \u22120.54 SDS (range, \u2212 1.6 to 0.4) and \u22120.91 SDS (\u22121.4 to \u22120.1) in R\/I and ND cohorts, respectively. Patients in the R\/I cohort were shorter at baseline than those in the ND cohort, and remained so throughout the study. The most common all-cause adverse events were increased blood bilirubin (53.4%), headache (46.6%), pyrexia (37.9%), and increased alanine transferase (36.2%). Apart from the impact on growth, the safety profile of nilotinib was generally consistent with previous reports. This study was registered on www.clinicaltrials.gov at #NCT01844765.<\/jats:p>","DOI":"10.1182\/bloodadvances.2020003759","type":"journal-article","created":{"date-parts":[[2021,7,26]],"date-time":"2021-07-26T15:37:27Z","timestamp":1627313847000},"page":"2925-2934","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":25,"title":["A phase 2 study of nilotinib in pediatric patients with CML: long-term update on growth retardation and safety"],"prefix":"10.1182","volume":"5","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-0199-054X","authenticated-orcid":false,"given":"Nobuko","family":"Hijiya","sequence":"first","affiliation":[{"name":"Division of Pediatric Hematology\/Oncology\/Stem Cell Transplantation, Columbia University Irving Medical Center, New York, NY;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0016-6698","authenticated-orcid":false,"given":"Alexey","family":"Maschan","sequence":"additional","affiliation":[{"name":"Dmitrii Rogachev Federal Research Center for Pediatric Hematology, Oncology and Immunology, Moscow, Russian Federation;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4828-3893","authenticated-orcid":false,"given":"Carmelo","family":"Rizzari","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Pediatric Hematology Oncology Unit, University of Milano Bicocca, Monza Brianza per il Bambino e la sua Mamma (MBBM) Foundation, Azienda Socio Sanitaria Territoriale (ASST), Monza, Italy;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5168-3222","authenticated-orcid":false,"given":"Hiroyuki","family":"Shimada","sequence":"additional","affiliation":[{"name":"Keio University School of Medicine, Shinjuku-ku, Japan;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Carlo","family":"Dufour","sequence":"additional","affiliation":[{"name":"Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS), Istituto Giannina Gaslini, Genoa, Italy;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hiroaki","family":"Goto","sequence":"additional","affiliation":[{"name":"Kanagawa Children\u2019s Medical Center, Yokohama, Japan;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hyoung Jin","family":"Kang","sequence":"additional","affiliation":[{"name":"Wide River Institute of Immunology, Hongcheon-gun, South Korea;"},{"name":"Department of Pediatrics, Seoul National University College of Medicine, Seoul, South Korea;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Terri","family":"Guinipero","sequence":"additional","affiliation":[{"name":"Nationwide Children's Hospital, The Ohio State University, Columbus, OH;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Zeynep","family":"Karakas","sequence":"additional","affiliation":[{"name":"Department of Pediatric Hematology\/Oncology, Istanbul Medical Faculty, Istanbul University, Istanbul, Turkey;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Francisco","family":"Bautista","sequence":"additional","affiliation":[{"name":"Hospital Ni\u00f1o Jesus, Madrid, Spain;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"St\u00e9phane","family":"Ducassou","sequence":"additional","affiliation":[{"name":"Groupe Hospitalier Pellegrin-H\u00f4pital des Enfants, Bordeaux, France;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Keon Hee","family":"Yoo","sequence":"additional","affiliation":[{"name":"Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, South Korea;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Christian Michel","family":"Zwaan","sequence":"additional","affiliation":[{"name":"Department of Pediatric Oncology\/Hematology, Erasmus Medical Center (MC)-Sophia Children\u2019s Hospital, Rotterdam, The Netherlands;"},{"name":"Innovative Therapies for Children with Cancer (ITCC) Consortium, The Netherlands;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Fr\u00e9d\u00e9ric","family":"Millot","sequence":"additional","affiliation":[{"name":"Unit\u00e9 d'Onco-H\u00e9matologie P\u00e9diatrique, Centre d'Investigation Clinique (CIC) 1402 INSERM, Centre Hospitalier Universitaire (CHU) de Poitiers, Poitiers, France;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Briana","family":"Patterson","sequence":"additional","affiliation":[{"name":"Aflac Cancer and Blood Disorders Center, Children\u2019s Healthcare of Atlanta and Emory University School of Medicine, Atlanta, GA;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9369-1661","authenticated-orcid":false,"given":"Jill","family":"Samis","sequence":"additional","affiliation":[{"name":"Ann and Robert H. 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