BACKGROUND AND OBJECTIVES:There is no consensus on optimal treatment duration for propranolol in infantile hemangioma (IH). We evaluated the efficacy and safety of oral propranolol solution administered for a minimum of 6 months up to a maximum of 12 months of age in high-risk IH. METHODS:This single-arm, open-label, phase 3 study was conducted in patients aged 35 to 150 days with high-risk IH in 10 hospitals between 2015 and 2017. The study comprised a 6-month initial treatment period (ITP) plus continuation up to 12 months of age if complete success was not achieved, a follow-up, and a retreatment period. Patients received oral propranolol twice daily (3 mg/kg per day). The primary end point was the success rate at the end of the ITP. Furthermore, the persistence of IH response and efficacy of retreatment was evaluated. RESULTS:The success rate after 6 months of treatment was 47%, increasing to 76% at the end of the ITP. Of the patients who achieved success, 68% sustained success for 3 months without treatment, and 24% required retreatment. Of the 8 patients who were retreated, 7 achieved success. Adverse events, reported by 80% of patients, were mild, which were expected in this population or known propranolol side effects. CONCLUSIONS:Oral propranolol administered beyond 6 months and up to 12 months of age meaningfully increases the success rate in high-risk IH. Success was sustained in most patients up to 3 months after stopping treatment. Retreatment was efficacious, and the safety profile satisfactory.abstract

(18)F-FDG PET demonstrates high diagnostic accuracy for response to initial chemotherapy in patients with ES and it correlates with outcome. The role of FDG PET in predicting response and outcome should be further investigated.

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The aim of this study was to assess bone mineral density (BMD) and biochemical bone metabolism markers in patients with bone tumors after anti-cancer treatment.The study included 27 patients (median age 15 years) with malignant bone tumors and 27 healthy children. In all subjects, BMD and body composition were measured by dual-energy X-ray absorptiometry. Serum bone markers were determined by immunoenzymatic assays. After completion of treatment, patients with bone tumors had significantly decreased total and lumbar spine BMD. We observed lower calcium and vitamin D levels in patients and comparable values of bone turnover markers (carboxyterminal telopeptide of collagen type I -CTX, bone alkaline phosphatase -BALP and osteocalcin -OC) in both groups of children. However, the level of carboxylated osteocalcin (cOC) was significantly lower (p<0.01) and undercarboxylated OC (ucOC) was higher (p<0.05) in patients than in the controls. Additionally, we observed similar values of anthropometric parameters in the subgroups of patients treated with methotrexate (MTX) or without MTX. In patients treated without MTX we found lower (p<0.05) ratio of cOC/ucOC, lower vitamin D level and higher CTX concentration.Patients with bone tumors after anticancer treatment had decreased bone mineral density and alterations in bone metabolism markers with potential decrease in bone formation.

PurposeSelected cell-cycle regulators and extracellular matrix proteins were found to play roles in malignant peripheral nerve sheath tumor (MPNST) biology. We aimed to analyze whether initial tumor tissue expressions of survivin, p53, cyclin D1, osteopontin (OPN) and fibronectin (FN) correlate with the response to neo-adjuvant CHT (naCHT) in children with advanced inoperable MPNST.MethodsThe study included 26 children with MPNST (M/F 14/12, median age 130 months) treated in Polish centers of pediatric oncology between 1992 and 2013. Tissue expression of markers was studied immunohistochemically in the manually performed tissue microarrays and assessed semi-quantitatively as low and high, based on the rate of positive cells and staining intensity.ResultsGood response to naCHT was noted in 47.6%, while poor—in 52.4% of patients. The response to naCHT was influenced negatively by the presence of neurofibromatosis NF1 and high initial tumor tissue expression of OPN, survivin, p53 and cyclin D1. Patients with high tumor expression of either OPN, survivin or p53 and those with simultaneous high expression of ≥ 3 of the markers, responded significantly worse to naCHT, than patients, in whom expression of ≤ 2 markers were detected at diagnosis. Nearly, 85% of patients expressing ≥ 3 markers, responded poor to CHT; while 87.5% of children, expressing ≤ 2 markers, were good responders.ConclusionThe initial tumor tissue expression of OPN, survivin, p53 and cyclin D1 may serve as markers to predict response to naCHT in pediatric advanced MPNST. Future studies in more numerous group of patients are needed to confirm these preliminary results.