Management of children and adolescents with chronic myeloid leukemia in blast phase: International pediatric CML expert panel recommendations

Stephanie Sembill, Maria Ampatzidou, Sonali Chaudhury, Michael Dworzak, Krzysztof Kalwak, Axel Karow, Alexander Kiani, Manuela Krumbholz, Maaike Luesink, Nora Naumann-Bartsch, Barbara De Moerloose, Michael Osborn, Kirk R. Schultz, Petr Sedlacek, Fiorina Giona, Christian Michel Zwaan, Hiroyuki Shimada, Birgitta Versluijs, Frederic Millot, Nobuko HijiyaMeinolf Suttorp, Markus Metzler*

*Corresponding author for this work

Research output: Contribution to journalReview articleAcademicpeer-review

8 Citations (Scopus)
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Treatment of chronic myeloid leukemia has improved significantly with the introduction of tyrosine kinase inhibitors (TKIs), and treatment guidelines based on numerous clinical trials are available for chronic phase disease. However for CML in the blast phase (CML-BP), prognosis remains poor and treatment options are much more limited. The spectrum of treatment strategies for children and adolescents with CML-BP has largely evolved empirically and includes treatment principles derived from adult CML-BP and pediatric acute leukemia. Given this heterogeneity of treatment approaches, we formed an international panel of pediatric CML experts to develop recommendations for consistent therapy in children and adolescents with this high-risk disease based on the current literature and national standards. Recommendations include detailed information on initial diagnosis and treatment monitoring, differentiation from Philadelphia-positive acute leukemia, subtype-specific selection of induction therapy, and combination with tyrosine kinase inhibitors. Given that allogeneic hematopoietic stem cell transplantation currently remains the primary curative intervention for CML-BP, we also provide recommendations for the timing of transplantation, donor and graft selection, selection of a conditioning regimen and prophylaxis for graft-versus-host disease, post-transplant TKI therapy, and management of molecular relapse. Management according to the treatment recommendations presented here is intended to provide the basis for the design of future prospective clinical trials to improve outcomes for this challenging disease.

Original languageEnglish
Pages (from-to)505-517
Number of pages13
Issue number3
Publication statusPublished - Mar 2023

Bibliographical note

Funding Information:
Supported by the Interdisciplinary Center for Clinical Research (IZKF) at the University Hospital of the University of Erlangen-Nuremberg (Laboratory Rotation, SS).

Open Access funding enabled and organized by Projekt DEAL

Publisher Copyright:
© 2023, The Author(s).


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