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Review
. 2023 Mar;37(3):505-517.
doi: 10.1038/s41375-023-01822-2. Epub 2023 Jan 27.

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

Stephanie Sembill  1   2 , Maria Ampatzidou  3 , Sonali Chaudhury  4 , Michael Dworzak  5 , Krzysztof Kalwak  6 , Axel Karow  1   2 , Alexander Kiani  7 , Manuela Krumbholz  1   2 , Maaike Luesink  8 , Nora Naumann-Bartsch  1   2 , Barbara De Moerloose  9 , Michael Osborn  10 , Kirk R Schultz  11 , Petr Sedlacek  12 , Fiorina Giona  13 , Christian Michel Zwaan  8   14   15 , Hiroyuki Shimada  16 , Birgitta Versluijs  8 , Frederic Millot  17 , Nobuko Hijiya  18 , Meinolf Suttorp  19 , Markus Metzler  20   21
Affiliations
Review

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

Stephanie Sembill et al. Leukemia. 2023 Mar.

Abstract

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.

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Conflict of interest statement

AK consulted and received honoraria from Novartis, Bristol Myers Squibb, Pfizer, and research funding from Novartis. BM received institutional funding from Novartis and Incyte. MM received research funding from Roche and consulted for BMS and Novartis. MZ received institutional funding from BMS, Pfizer, Takeda, Abbvie, Kura, Syndax, Jazz, and was a consultant to Novartis, Incyte, Takeda, Gilead, and Sanofi. NH has received research funding from Pfizer and Novartis, served on the data monitoring committee for Incyte and Novartis, and has been a consultant for Stemline Therapeutics. All other authors declared no conflict of interest.

Figures

Fig. 1
Fig. 1. Timing of allogeneic hematopoietic stem cell transplantation (HSCT).
acc. according, CT chemotherapy, dx diagnosis, gen. generation, m month.
Fig. 2
Fig. 2
Approach in case of detectable BCR::ABL1 transcripts after allogeneic hematopoietic stem cell transplantation (HSCT).
See this image and copyright information in PMC

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