Features and outcome of chronic myeloid leukemia at very young age: Data from the International Pediatric Chronic Myeloid Leukemia Registry.
Pediatr Blood Cancer
; 68(1): e28706, 2021 01.
Article
in En
| MEDLINE
| ID: mdl-33034135
ABSTRACT
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/female14/8) 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.Key words
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
/
Antineoplastic Combined Chemotherapy Protocols
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Registries
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Hematopoietic Stem Cell Transplantation
Type of study:
Observational_studies
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Prognostic_studies
/
Risk_factors_studies
Limits:
Child, preschool
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Female
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Humans
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Infant
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Male
Language:
En
Journal:
Pediatr Blood Cancer
Journal subject:
HEMATOLOGIA
/
NEOPLASIAS
/
PEDIATRIA
Year:
2021
Type:
Article
Affiliation country:
Turkey