Chronic myeloid leukemia, pediatric

Section editor transclusions This page contains studies that were specific to pediatric populations. For the more general CML page, follow this link.

6 regimens on this page 7 variants on this page

Guidelines

"How I Treat"

• 2019: Hijiya & Suttorp How I treat chronic myeloid leukemia in children and adolescents

AAML0123

Stratum 1, 2, or 3

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Regimen

Study	Years of enrollment	Evidence
Champagne et al. 2011 (COG AAML0123)	2002-2004	Phase II (RT)

Chemotherapy

• Imatinib (Gleevec) 340 mg/m² PO rounded to the nearest 100 mg once daily.
• 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%.
• Imatinib (Gleevec) therapy continued until completion of 13 28-day courses (1 year).

28-Day Course

References

1. 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 contains protocol PubMed NCT00030394

Stratum 4

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Regimen

Study	Years of enrollment	Evidence
Champagne et al. 2011 (COG AAML0123)	2002-2004	Phase II (RT)

Chemotherapy

• Imatinib (Gleevec) 570 mg/m²/day PO divided BID rounded to the nearest 100 mg (maximum dose of 1000 mg/day).
• 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%.
• Imatinib (Gleevec) therapy continued until completion of 13 28-day courses (1 year).

28-Day Course

References

1. 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 contains protocol PubMed NCT00030394

AAML1321

Nilotinib monotherapy

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Regimen

Study	Years of enrollment	Evidence
Hijaya et al. 2019 (DIALOG)	2013-2015	Phase II (RT)

Targeted therapy

• Nilotinib (Tasigna) 230 mg/m² PO twice per day rounded to the nearest 50 mg dose (maximum single dose = 400 mg)

28-Day Course Up to 66 cycles

References

1. COG AAML1321: 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 contains verified protocol NCT01844765
2. COGAAML1321 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 articlecontains verified protocol NCT01844765

Chronic phase, first-line therapy

Imatinib monotherapy

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Regimen

Study	Years of enrollment	Evidence
Champagne et al. 2011 (COG AAML0123)	2002-2004	Phase II (RT)

Targeted therapy

• Imatinib (Gleevec) 340 mg/m² PO once per day

28-day cycles

References

1. 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 contains protocol PubMed NCT00030394

Nilotinib monotherapy

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Regimen

Study	Years of enrollment	Evidence
Hijaya et al. 2019 (DIALOG)	2013-2015	Phase II (RT)

Targeted therapy

• Nilotinib (Tasigna) 230 mg/m² PO twice per day

Continued indefinitely

References

1. 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 contains verified protocol link to PMC article PubMed NCT01844765

Chronic phase, relapsed or refractory

Dasatinib monotherapy

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Regimen

Study	Years of enrollment	Evidence
Zwaan et al. 2013 (CA180-018)	2006-2009	Phase I, <20 pts in this cohort (RT)
Gore et al. 2018 (CA180-226)	2009-2014	Phase II (RT)

Targeted therapy

• Dasatinib (Sprycel) 60 mg/m² PO once per day

Continued indefinitely

References

1. 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 PubMed NCT00306202
2. 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 contains verified protocol PubMed NCT00777036

Nilotinib monotherapy

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Regimen

Study	Years of enrollment	Evidence
(CAMN107A2120)	2011-NR	Phase 1, <20 pts (RT)

Note: there is no obvious publication of this trial.

Targeted therapy

• Nilotinib (Tasigna)

References

1. CAMN107A2120: NCT01077544