Abstract
The Mission of the Chronic Myeloid Leukemia group is to improve treatments for children and adolescents with chronic myeloid leukemia
Our objectives are:
To facilitate the development of interventional clinical studies and standards in children with CML including all relevant treatment modalities
To facilitate registries to promote continuity over successive trials
To facilitate the development of basic and translational research projects in children with hematological malignancies that help to define the therapeutic strategies in patients with CML
Identify and mentor next generation clinical scientists in the field of myeloproliferative diseases
Scientific aims
• Comparison of clinical and molecular signatures in Pediatric and Adult CML
• Differentiating CML-BP from CML-like ALL
• Identify predictive markers for successful TFR, dose reduction and stopping TKI
• Study the genetic predisposition in CML
• Understand the mechanism of the influence of TKI on bone growth
Open and completed clinical trials
International CML registry
Management of children and adolescents with chronic myeloid leukemia in blast phase: International pediatric CML expert panel recommendations.
Clinical trials
Bosutinib Study ITCC-054/AAML1921
Ponatinib Paediatric INCB84344-102 Study
Asciminib CABL001I12201 – ASC4KIDS
Selected publications (last 5 years)
Sembill S, Ampatzidou M, Chaudhury S, Dworzak M, Kalwak K, Karow A, Kiani A, Krumbholz M, Luesink M, Naumann-Bartsch N, De Moerloose B, Osborn M, Schultz KR, Sedlacek P, Giona F, Zwaan CM, Shimada H, Versluijs B, Millot F, Hijiya N, Suttorp M, Metzler M. Management of children and adolescents with chronic myeloid leukemia in blast phase: International pediatric CML expert panel recommendations. Leukemia. 2023 Mar;37(3):505-517.
Salmon M et al. Impact of BCR::ABL1 transcript type on RT-qPCR amplification performance and molecular response to therapy. Leukemia. 2022 Jul;36(7):1879-1886
White HE et al. Standardization of molecular monitoring of CML: results and recommendations from the European treatment and outcome study. Leukemia 2022, 36(7):1834-1842.
Shima H, Kada A, Tanizawa A, et al. Discontinuation of tyrosine kinase inhibitors in pediatric chronic myeloid leukemia. Pediatr Blood Cancer. 2022 Aug;69(8):e29699.
Shimada H, Tanizawa A, Kondo T, et al. Prognostic Factors for Outcomes of Allogeneic HSCT for Children and Adolescents/Young Adults With CML in the TKI Era. Transplant Cell Ther. 2022 Jul;28(7):376-389. doi: 10.1016/j.jtct.2022.04.011.
Shima H, Shimada H. Recent progress in the management of pediatric chronic myeloid leukemia. Int J Hematol. 2023 Feb;117(2):182-187. doi: 10.1007/s12185-022-03526-2. Epub 2022 Dec 27.PMID: 36574169
Leung WY et al. Outcome Prediction of Chronic Myeloid Leukemia in Children. Ann Haematol 2022 Aug
Frédéric Millot, Meinolf Suttorp, Stéphanie Ragot, Guy Leverger, Jean-Hugues Dalle, Caroline Thomas, Nathalie Cheikh, Brigitte Nelken, Marilyne Poirée, Geneviève Plat, Birgitta Versluys, Birgitte Lausen, Marina Borisevich. Discontinuation of Imatinib in Children with Chronic Myeloid Leukemia: A Study from the International Registry of Childhood CML Cancers 2021, 13, 4102
Gleixner K et al. Asciminib and ponatinib exert synergistic anti-neoplastic effects on CML cells expressing BCR-ABL1 T315I-compound mutations. Am J Cancer Res. 2021 Sep 15;11(9):4470-4484.
Heibl S et al. A phase 1 study to evaluate the feasibility and efficacy of the addition of ropeginterferon alpha-2b to imatinib treatment in patients with chronic phase chronic myeloid leukemia (CML) not achieving a deep molecular response (molecular remission 4.5)-AGMT_CML 1. Hematol Oncol. 2020.
Mughal TI et al. Illuminating novel biological aspects and potential new therapeutic approaches for chronic myeloproliferative malignancies. Hematol Oncol. 2020.
Comparison of the Transcriptomic Signatures in Pediatric and Adult CML. Youn M, Smith SM, Lee AG, Chae HD, Spiteri E, Erdmann J, Galperin I, Jones LM, Donato M, Abidi P, Bittencourt H, Lacayo N, Dahl G, Aftandilian C, Davis KL, Matthews JA, Kornblau SM, Huang M, Sumarsono N, Redell MS, Fu CH, Chen IM, Alonzo TA, Eklund E, Gotlib J, Khatri P, Sweet-Cordero EA, Hijiya N, Sakamoto KM. Cancers. 2021 Dec 14;13(24):6263.
Imatinib in the treatment of chronic myeloid leukemia in children and adolescents is effective and well tolerated: Report of the Polish Pediatric Study Group for the Treatment of Leukemias and Lymphomas. Janeczko-Czarnecka M, Krawczuk-Rybak M, Karpińska-Derda I, Niedźwiecki M, Musioł K, Ćwiklińska M, Drabko K, Mycko K, Ociepa T, Pawelec K, Januszkiewicz-Lewandowska D, Ussowicz M, Rybka B, Ryczan-Krawczyk R, Kołtan A, Karolczyk G, Zaucha-Prażmo A, Badowska W, Kałwak K. Adv Clin Exp Med. 2018 Jan;27(1):91-98.
