Chronic myeloid leukemia, pediatric

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This page contains studies that were specific to pediatric populations. For the more general CML page, follow this link.
Last updated on 2024-07-23:7 regimens on this page
8 variants on this page
=Chronic phase, first-line therapy=
==Bosutinib monotherapy [COG AAML1921]==
===Regimen===
Study 	Dates of enrollment	Evidence
Brivio et al. 2023 (BCHILD)	2016-11 to 2022-08 	Phase 1/2 (RT)
Note: this is the RP2D for newly diagnosed patients.
====Targeted therapy====
*Bosutinib (Bosulif) 300 mg/m2 (maximum dose of 500 mg) PO once per day
Continued indefinitely
===References===
#BCHILD: Brivio E, Pennesi E, Willemse ME, Huitema ADR, Jiang Y, van Tinteren HDR, van der Velden VHJ, Beverloo BH, den Boer ML, Rammeloo LAJ, Hudson C, Heerema N, Kowalski K, Zhao H, Kuttschreuter L, Bautista Sirvent FJ, Bukowinski A, Rizzari C, Pollard J, Murillo-Sanjuán L, Kutny M, Zarnegar-Lumley S, Redell M, Cooper S, Bertrand Y, Petit A, Krystal J, Metzler M, Lancaster D, Bourquin JP, Motwani J, van der Sluis IM, Locatelli F, Roth ME, Hijiya N, Zwaan CM. Bosutinib in Resistant and Intolerant Pediatric Patients With Chronic Phase Chronic Myeloid Leukemia: Results From the Phase I Part of Study ITCC054/COG AAML1921. J Clin Oncol. 2024 Mar 1;42(7):821-831. Epub 2023 Nov 30. link to original article link to PMC article dosing details in manuscript have been reviewed by our editors EuroPMC NCT04258943
==Imatinib monotherapy [COG AAML0123]==
===Regimen variant #1, 340 mg/m2 (stratum 1, 2, or 3)===
Study 	Dates of enrollment	Evidence
Champagne et al. 2011 (COG AAML0123)	2002-2004 	Phase 2 (RT)
====Targeted therapy====
*Imatinib (Gleevec) 340 mg/m2 PO once per day on days 1 to 28, rounded to the nearest 100 mg
28-day cycle for 13 cycles (1 year)
====Dose and schedule modifications====
*Patient evaluated per protocol after each 28-day cycle for response and toxicities
*Hold Imatinib (Gleevec) if a grade 2 nonhematologic adverse event fails to resolve or if a grade 3 or 4 nonhematologic adverse event occurs
*After resolution to < grade 1, Imatinib (Gleevec) dose may be reduced by 30%
===Regimen variant #2, 570 mg/m2 (stratum 4)===
Study 	Dates of enrollment	Evidence
Champagne et al. 2011 (COG AAML0123)	2002-2004 	Phase 2
====Targeted therapy====
*Imatinib (Gleevec) 570 mg/m2/day (maximum dose of 1000 mg/day) PO on days 1 to 28, divided twice per day and rounded to the nearest 100 mg
28-day cycle for 13 cycles (1 year)
====Dose and schedule modifications====
*Patient evaluated per protocol after each 28-day cycle for response and toxicities
*Hold Imatinib (Gleevec) if a grade 2 nonhematologic adverse event fails to resolve or if a grade 3 or 4 nonhematologic adverse event occurs
*After resolution to < grade 1, Imatinib (Gleevec) dose may be reduced by 30%
===References===
# COG AAML0123: Champagne MA, Fu CH, Chang M, Chen H, Gerbing RB, Alonzo TA, Cooley LD, Heerema NA, Oehler V, Wood C, French ME, Arceci RJ, Smith FO, Bernstein ML. Higher dose imatinib for children with de novo chronic phase chronic myelogenous leukemia: a report from the Children's Oncology Group. Pediatr Blood Cancer. 2011 Jul 15;57(1):56-62. Epub 2011 Apr 4. link to original article link to PMC article dosing details in abstract have been reviewed by our editors EuroPMC NCT00030394
==Nilotinib monotherapy [COG AAML1321]==
===Regimen===
Study 	Dates of enrollment	Evidence
Hijiya et al. 2019 (DIALOG)	2013-2015 	Phase 2 (RT)
====Targeted therapy====
*Nilotinib (Tasigna) 230 mg/m2 (maximum dose of 200 mg) PO twice per day on days 1 to 28, rounded to the nearest 50 mg dose
28-day cycle for up to 66 cycles
===References===
#DIALOG: Hijiya N, Maschan A, Rizzari C, Shimada H, Dufour C, Goto H, Kang HJ, Guinipero T, Karakas Z, Bautista F, Ducassou S, Yoo KH, Zwaan CM, Millot F, Aimone P, Allepuz A, Quenet S, Hourcade-Potelleret F, Hertle S, Sosothikul D. Phase 2 study of nilotinib in pediatric patients with Philadelphia chromosome-positive chronic myeloid leukemia. Blood. 2019 Dec 5;134(23):2036-2045. link to original article dosing details in manuscript have been reviewed by our editors link to PMC article EuroPMC NCT01844765
##Update: Hijiya N, Maschan A, Rizzari C, Shimada H, Dufour C, Goto H, Kang HJ, Guinipero T, Karakas Z, Bautista F, Ducassou S, Yoo KH, Zwaan CM, Millot F, Patterson B, Samis J, Aimone P, Allepuz A, Titorenko K, Sosothikul D. A phase 2 study of nilotinib in pediatric patients with CML: long-term update on growth retardation and safety. Blood Adv. 2021 Jul 27;5(14):2925-2934. link to original article dosing details in manuscript have been reviewed by our editors link to PMC article EuroPMC
=Chronic phase, relapsed or refractory=
==Bosutinib monotherapy [COG AAML1921]==
===Regimen===
Study 	Dates of enrollment	Evidence
Brivio et al. 2023 (BCHILD)	2016-11 to 2022-08 	Phase 1/2 (RT)
Note: this is the RP2D for refractory/intolerant patients.
====Targeted therapy====
*Bosutinib (Bosulif) 400 mg/m2 (maximum dose of 600 mg) PO once per day
Continued indefinitely
===References===
#BCHILD: Brivio E, Pennesi E, Willemse ME, Huitema ADR, Jiang Y, van Tinteren HDR, van der Velden VHJ, Beverloo BH, den Boer ML, Rammeloo LAJ, Hudson C, Heerema N, Kowalski K, Zhao H, Kuttschreuter L, Bautista Sirvent FJ, Bukowinski A, Rizzari C, Pollard J, Murillo-Sanjuán L, Kutny M, Zarnegar-Lumley S, Redell M, Cooper S, Bertrand Y, Petit A, Krystal J, Metzler M, Lancaster D, Bourquin JP, Motwani J, van der Sluis IM, Locatelli F, Roth ME, Hijiya N, Zwaan CM. Bosutinib in Resistant and Intolerant Pediatric Patients With Chronic Phase Chronic Myeloid Leukemia: Results From the Phase I Part of Study ITCC054/COG AAML1921. J Clin Oncol. 2024 Mar 1;42(7):821-831. Epub 2023 Nov 30. link to original article link to PMC article dosing details in manuscript have been reviewed by our editors EuroPMC NCT04258943
==Dasatinib monotherapy==
===Regimen===
Study 	Dates of enrollment	Evidence
Zwaan et al. 2013 (CA180-018)	2006-2009 	Phase 1, fewer than 20 pts in this cohort (RT)
Gore et al. 2018 (CA180-226) 	2009-2014 	Phase 2 (RT)
====Targeted therapy====
*Dasatinib (Sprycel) 60 mg/m2 PO once per day on days 1 to 28
28-day cycles
===References===
# CA180-018: Zwaan CM, Rizzari C, Mechinaud F, Lancaster DL, Lehrnbecher T, van der Velden VH, Beverloo BB, den Boer ML, Pieters R, Reinhardt D, Dworzak M, Rosenberg J, Manos G, Agrawal S, Strauss L, Baruchel A, Kearns PR. Dasatinib in children and adolescents with relapsed or refractory leukemia: results of the CA180-018 phase I dose-escalation study of the Innovative Therapies for Children with Cancer Consortium. J Clin Oncol. 2013 Jul 1;31(19):2460-8. Epub 2013 May 28. link to original article EuroPMC NCT00306202
# CA180-226: Gore L, Kearns PR, de Martino Lee ML, De Souza CA, Bertrand Y, Hijiya N, Stork LC, Chung NG, Cardos RC, Saikia T, Fagioli F, Seo JJ, Landman-Parker J, Lancaster D, Place AE, Rabin KR, Sacchi M, Swanink R, Zwaan CM. Dasatinib in Pediatric Patients With Chronic Myeloid Leukemia in Chronic Phase: Results From a Phase II Trial. J Clin Oncol. 2018 May 1;36(13):1330-1338. Epub 2018 Mar 2. link to original article link to PMC article dosing details in manuscript have been reviewed by our editors EuroPMC NCT00777036
==Imatinib monotherapy==
===Regimen===
FDA-recommended dose
Study 	Dates of enrollment	Evidence
Champagne et al. 2004 (COG P9973)	2000-2001 	Phase 1 (RT)
Note: this trial tested a variety of doses; this is the FDA-recommended dose.
====Targeted therapy====
*Imatinib (Gleevec) 340 mg/m2 PO once per day
Continued indefinitely
===References===
# COG P9973: Champagne MA, Capdeville R, Krailo M, Qu W, Peng B, Rosamilia M, Therrien M, Zoellner U, Blaney SM, Bernstein M; Children's Oncology Group phase 1 study. Imatinib mesylate (STI571) for treatment of children with Philadelphia chromosome-positive leukemia: results from a Children's Oncology Group phase 1 study. Blood. 2004 Nov 1;104(9):2655-60. Epub 2004 Jul 1. link to original article dosing details in manuscript have been reviewed by our editors EuroPMC NCT00004932
==Nilotinib monotherapy==
===Regimen===
Study 	Dates of enrollment	Evidence
Hijiya et al. 2019 (CAMN107A2120)	2011-2015 	Phase 1, fewer than 20 pts (RT)
Note: the study enrollment end date is from the sponsor website; it is not mentioned in the manuscript.
====Targeted therapy====
*Nilotinib (Tasigna) 230 mg/m2 PO twice per day on days 1 to 28
28-day cycles
===References===
#CAMN107A2120: Hijiya N, Zwaan CM, Rizzari C, Foà R, Abbink F, Lancaster D, Landman-Parker J, Millot F, Moppett J, Nelken B, Putti MC, Tian X, Sinclair K, Santanastasio H, Buchbinder A, Kearns P. Pharmacokinetics of Nilotinib in Pediatric Patients with Philadelphia Chromosome-Positive Chronic Myeloid Leukemia or Acute Lymphoblastic Leukemia. Clin Cancer Res. 2020 Feb 15;26(4):812-820. Epub 2019 Nov 1. link to original article dosing details in abstract have been reviewed by our editors EuroPMC NCT01077544
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