Frédéric Millot1, Christelle Dupraz1, Joelle Guilhot1, Meinolf Suttorp2, Françoise Brizard1, Thierry Leblanc3, Adalet Meral Güneş4, Petr Sedlacek5, Evelyne De Bont6,7, Chi Kong Li8, Krzysztof Kalwak9, Birgitte Lausen10, Srdjana Culic11, Michael Dworzak12, Emilia Kaiserova13, Barbara De Moerloose14, Farah Roula15, Andrea Biondi16, André Baruchel3, François Guilhot1. 1. INSERM Clinical Investigation Center 1402, Poitiers University, Poitiers, France. 2. Department of Pediatrics, Carl Gustav Carus University Hospital, Dresden, Germany. 3. Department of Pediatric Hematology, Robert Debré Hospital, Paris, France. 4. Department of Pediatric Hematology, Uludağ University Hospital, Görükle Bursa, Turkey. 5. Department of Pediatric Hematology-Oncology, Motol University Hospital, Charles University, Prague, Czech Republic. 6. Department of Pediatric Oncology-Hematology, University Medical Center Groningen, University of Groningen, Groningen, the Netherlands. 7. Dutch Childhood Oncology Group, the Hague, the Netherlands. 8. Department of Pediatrics, Prince of Wales Hospital, Chinese University of Hong Kong, Hong Kong, China. 9. Department of Pediatric Hematology-Oncology and Transplantation, Wroclaw Medical University, Wroclaw, Poland. 10. Department of Pediatrics, Rigshospitalet, University Hospital, Copenhagen, Denmark. 11. Department of Pediatric Hematology, Oncology, Immunology, and Medical Genetics, Clinical Hospital, Split, Croatia. 12. Children's Cancer Research Institute and St. Anna Children's Hospital, Vienna, Austria. 13. Department of Pediatric Oncology, University Children's Hospital, Bratislava, Slovakia. 14. Department of Pediatrics, Ghent University Hospital, Ghent, Belgium. 15. Department of Pediatrics, Saint George Hospital University Medical Centre, Beirut, Lebanon. 16. Department of Pediatrics, San Gerardo Hospital, University of Milano-Bicocca, Fondazione Monza e Brianza per il Bambino e la sua Mamma, Monza, Italy.
Abstract
BACKGROUND: In the adult population with newly diagnosed chronic myeloid leukemia (CML), variant translocations are usually not considered to be impairing the prognosis, whereas some additional cytogenetic abnormalities (ACAs) are associated with a negative impact on survival. Because of the rarity of CML in the pediatric population, such abnormalities have not been investigated in a large group of children with CML. METHODS: The prognostic relevance of variant t(9;22) and ACAs at diagnosis was assessed in 301 children with CML in the chronic phase who were enrolled in the International Registry for Chronic Myeloid Leukemia in Children and Adolescents. RESULTS: Overall, 19 children (6.3%) presented with additional cytogenetic findings at diagnosis: 5 children (1.7%) had a variant t(9;22) translocation, 13 children (4.3%) had ACAs, and 1 had both. At 3 years, for children with a classic translocation, children with ACAs, and children with a variant t(9;22) translocation who were treated with imatinib as frontline therapy, the probability of progression-free survival (PFS) was 95% (95% confidence interval [CI], 91%-97%), 100%, and 75% (95% CI, 13%-96%), respectively, and the probability of overall survival (OS) was 98% (95% CI, 95%-100%), 100% (95% CI, 43%-98%), and 75% (95% CI, 13%-96%), respectively. No statistical difference was observed between the patients with classic cytogenetic findings and those with additional chromosomal abnormalities in terms of PFS and OS. CONCLUSIONS: In contrast to adults with CML, additional chromosomal abnormalities observed at diagnosis do not seem to have a significant prognostic impact. Cancer 2017;123:3609-16.
BACKGROUND: In the adult population with newly diagnosed chronic myeloid leukemia (CML), variant translocations are usually not considered to be impairing the prognosis, whereas some additional cytogenetic abnormalities (ACAs) are associated with a negative impact on survival. Because of the rarity of CML in the pediatric population, such abnormalities have not been investigated in a large group of children with CML. METHODS: The prognostic relevance of variant t(9;22) and ACAs at diagnosis was assessed in 301 children with CML in the chronic phase who were enrolled in the International Registry for Chronic Myeloid Leukemia in Children and Adolescents. RESULTS: Overall, 19 children (6.3%) presented with additional cytogenetic findings at diagnosis: 5 children (1.7%) had a variant t(9;22) translocation, 13 children (4.3%) had ACAs, and 1 had both. At 3 years, for children with a classic translocation, children with ACAs, and children with a variant t(9;22) translocation who were treated with imatinib as frontline therapy, the probability of progression-free survival (PFS) was 95% (95% confidence interval [CI], 91%-97%), 100%, and 75% (95% CI, 13%-96%), respectively, and the probability of overall survival (OS) was 98% (95% CI, 95%-100%), 100% (95% CI, 43%-98%), and 75% (95% CI, 13%-96%), respectively. No statistical difference was observed between the patients with classic cytogenetic findings and those with additional chromosomal abnormalities in terms of PFS and OS. CONCLUSIONS: In contrast to adults with CML, additional chromosomal abnormalities observed at diagnosis do not seem to have a significant prognostic impact. Cancer 2017;123:3609-16.
Authors: F Nguyen-Khac; A Bidet; A Daudignon; M Lafage-Pochitaloff; G Ameye; C Bilhou-Nabéra; E Chapiro; M A Collonge-Rame; W Cuccuini; N Douet-Guilbert; V Eclache; I Luquet; L Michaux; N Nadal; D Penther; B Quilichini; C Terre; C Lefebvre; M-B Troadec; L Véronèse Journal: Leukemia Date: 2022-04-16 Impact factor: 12.883