[Improved survival in children with neuroblastoma]. / Forbedret overlevelse hos børn med neuroblastom.

INTRODUCTION: The aims of the present study were to analyze whether changes in incidence and mortality rates have taken place in Denmark during the period 1981-2005, and whether the distribution of known prognostic factors has changed during this period. MATERIAL AND METHODS: A total of 206 children below 15 years of age with neuroblastoma or ganglioneuroblastoma who were diagnosed in Denmark between 1981 and 2005. RESULTS: The incidence was 8.68 per million children below 15 years of age (world standard 9.6) and 43.5 per million children below 12 months of age and these incidences have remained unchanged since 1970. The mortality rate has decreased steadily during the study period. The prognostic factors age, stage and site of primary tumour did not change during the study period and were not different from those reported by others. 32% of the children were below 12 months of age at diagnosis. 53% of the children had metastatic disease. The overall 5-year survival increased over the study period from 38% in 1981-1985 to 69% in 2001-2005. A significant increase in the survival of children > 12 months of age with stage 4 disease was also observed. Relapse/disease progression more than three years from diagnosis occurred in only 2% of patients. The median time from relapse to death was three months. CONCLUSION: The survival of children with neuroblastoma in Denmark has increased significantly over the last 25 years.

Myeloid neoplasm with prominent eosinophilia and PDGFRA rearrangement treated with imatinib mesylate.

The FIP1L1-PDGFRA fusion gene is the most frequent genetic aberration in myeloid neoplasms associated with eosinophilia and abnormalities of PDGFRA, PDGFRB, or FGFR1. Affected patients in adult populations are very sensitive to imatinib therapy. Pediatric cases are rare and so far only one case of FIP1L1-PDGFRA positive disease has been reported. We report a 2-year-old female with a myeloid neoplasm associated with eosinophilia and rearrangement of PDGFRA. Treatment with imatinib resulted in complete and durable clinical, hematological, and molecular remission within 3 months after starting treatment.

Late cardiac effects of anthracycline containing therapy for childhood acute lymphoblastic leukemia.

BACKGROUND: At present about 80% of children with acute lymphoblastic leukemia (ALL) will be cured following treatment with multi-drug chemotherapy. A major concern for this growing number of survivors is the risk of late effects of treatment. The aim of this study was to determine whether signs of cardiomyopathy were present in patients treated in childhood with cumulative anthracycline doses of less than 300 mg/m(2). PROCEDURE: Evaluation of cardiac function in a cohort of 63 long-term survivors in first continuous remission following treatment of ALL with multi-drug chemotherapy including anthracyclines was performed using standard M-mode echocardiography and tissue doppler imaging (TDI). Associations between age at diagnosis, cumulative dose of anthracycline, sex, length of follow-up, and deviations from normal values in M-mode echocardiograms were evaluated using univariate and multivariate regression analysis. TDI data were compared to normal values using Wilcoxon matched-pairs signed-ranks test. RESULTS: By standard M-mode echocardiography the most significant findings were diastolic dilation of the left ventricle, thinner interventricular septum (IVS), decreased left ventricular mass (LVM) in females, follow-up dependent dilation of the left ventricle in systole and follow-up dependent decrease in ejection fraction (EF). TDI abnormalities included signs of early diastolic dysfunction and myocardial hypertrophy, and were also found in structures that appeared normal by M-mode echocardiography. CONCLUSIONS: This study adds to the growing evidence that even low to moderate doses of anthracyclines might lead to progressive cardiac dysfunction. It is important that children treated with anthracyclines receive life long follow-up for signs of cardiomyopathy.