Features and outcome of chronic myeloid leukemia at very young age: Data from the International Pediatric Chronic Myeloid Leukemia Registry

• Published in: Pediatric Blood & Cancer Vol. 68; no. 1; pp. e28706 - n/a
• Main Authors: Meral Günes, Adalet, Millot, Frédéric, Kalwak, Krzysztof, Lausen, Birgitte, Sedlacek, Petr, Versluys, A. Birgitta, Dworzak, Michael, De Moerloose, Barbara, Suttorp, Meinolf
• Format: Journal Article
• Language: English
• Published: United States Wiley 01.01.2021
• Subjects: Antineoplastic Combined Chemotherapy Protocols; Antineoplastic Combined Chemotherapy Protocols - therapeutic use; Child, Preschool; children; Combined Modality Therapy; Female; Follow-Up Studies; Hematopoietic Stem Cell Transplantation; Hematopoietic Stem Cell Transplantation - mortality; Humans; imatinib; Infant; Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Leukemia, Myelogenous, Chronic, BCR-ABL Positive - pathology; Leukemia, Myelogenous, Chronic, BCR-ABL Positive - therapy; Male; pediatric CML; Prognosis; Registries; Registries - statistics & numerical data; side effects; Survival Rate; very young age; imatinib; side effects; pediatric CML; children; very young age
• ISSN: 1545-5009; 1545-5017; 1545-5017
• DOI: 10.1002/pbc.28706

Introduction Chronic myeloid leukemia (CML) is rare in the first two decades of life comprising only 3% of newly diagnosed pediatric and adolescent leukemias. We studied the epidemiologic and clinical features of patients with CML diagnosed at younger than 3 years of age and evaluated treatment and long‐term outcome. Method Data from the International Pediatric I‐BFM/CML Registry were retrospectively analyzed using the European LeukemiaNet criteria of the year 2006. Characteristics and treatment outcome of patients <3 years old at diagnosis were evaluated from standardized forms. Results Twenty‐two patients (n = 22/479; 4.6%, male/female:14/8) were enrolled with a median age of 22 months (range, 10–34 m). Major symptoms comprised asthenia (30%), fever (30%), abdominal pain (20%), extramedullary signs (14%), hemorrhage (5%), and weight loss (5%). The extramedullary signs were specified in eight children: blueberry muffin (n = 1), sudden swollen abdomen (n = 1), sustained vomiting (n = 1), and cervical and inguinal lymph nodes (n = 5). Two of five children with cervical and inguinal lymph nodes were categorized as accelerated phase. Overall, 19 of 22 (86%) children were diagnosed in chronic phase, while the remaining three patients were in advanced phase. Median follow‐up was 78 months (range, 7–196 m). Twenty‐one out of 22 patients initially received imatinib, while one child received IFN + ARA‐C. Imatinib was changed to second‐line tyrosine kinase inhibitors (TKIs) in 29% of cases. During follow‐up, 41% patients underwent stem cell transplantation (SCT). While on TKI, major molecular response (MMR) was achieved in 48% of children. Among the remaining patients, 21% are alive on TKI without MMR and 22% achieved complete molecular response following SCT. Twenty‐one of 22 (95%) children are alive, while one patient died of posttransplant complications. Conclusion This report demonstrates for the first time the efficacy and long‐term effects of upfront imatinib in the so far largest cohort of children with CML diagnosed at very young age.