Features and outcome of chronic myeloid leukemia at very young age: Data from the International Pediatric Chronic Myeloid Leukemia Registry
Jazyk angličtina Země Spojené státy americké Médium print-electronic
Typ dokumentu časopisecké články, práce podpořená grantem
PubMed
33034135
DOI
10.1002/pbc.28706
Knihovny.cz E-zdroje
- Klíčová slova
- children, imatinib, pediatric CML, side effects, very young age,
- MeSH
- chronická myeloidní leukemie patologie terapie MeSH
- kojenec MeSH
- kombinovaná terapie MeSH
- lidé MeSH
- míra přežití MeSH
- následné studie MeSH
- předškolní dítě MeSH
- prognóza MeSH
- protokoly protinádorové kombinované chemoterapie terapeutické užití MeSH
- registrace statistika a číselné údaje MeSH
- transplantace hematopoetických kmenových buněk mortalita MeSH
- Check Tag
- kojenec MeSH
- lidé MeSH
- mužské pohlaví MeSH
- předškolní dítě MeSH
- ženské pohlaví MeSH
- Publikační typ
- časopisecké články MeSH
- práce podpořená grantem MeSH
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.
Department of Pediatric and Adolescent Medicine University of Copenhagen Copenhagen Denmark
Department of Pediatric Hematology and Oncology University Hospital Motol Prague Czech Republic
Department of Pediatric Hematology Medical Faculty Uludag University Nilüfer Bursa Turkey
Department of Pediatric Hematology Oncology and BMT Wroclaw Medical University Wroclaw Poland
Department of Pediatric Hematology Oncology Ghent University Hospital Ghent Belgium
Medical Faculty Pediatric Hematology Oncology Technical University of Dresden Dresden Germany
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