Are all cases of paediatric essential thrombocythaemia really myeloproliferative neoplasms? Analysis of a large cohort

Sporadic essential thrombocythaemia (ET) is rare in paediatrics, and the diagnostic and clinical approach to paediatric cases cannot be simply copied from experience with adults. Here, we assessed 89 children with a clinical diagnosis of ET and found that 23 patients (258%) had a clonal disease. The JAK2 V617F mutation was identified in 14 children, 1 child had the MPL W515L mutation, and 6 had CALR mutations. The monoclonal X-chromosome inactivation pattern was seen in six patients (two with JAK2 V617F and two with CALR mutations). The other 66 patients (742%) had persistent thrombocytosis with no clonality. There were no clinical or haematological differences between the clonal and non-clonal patients. The relative proportion of ET-specific mutations in the clonal children was much the same as in adults. The higher prevalence of nonclonal cases suggests that some patients may not have myeloproliferative neoplasms, with significant implications for their treatment

Multicenter randomized, double-blind controlled trial to evaluate the efficacy of laser therapy for the treatment of severe oral mucositis induced by chemotherapy in children: laMPO RCT

Objectives: To demonstrate the efficacy of laser photobiomodulation (PBM) compared to that of placebo on severe oral mucositis (OM) in pediatric oncology patients. The primary objective was the reduction of OM grade (World Health Organization [WHO] scale) 7 days after starting PBM. Secondary objectives were reduction of pain, analgesic consumption, and incidence of side effects. Methods: One hundred and one children with WHO grade\ua0>\ua02 chemotherapy-induced OM were enrolled in eight Italian hospitals. Patients were randomized to either PBM or sham treatment for four consecutive days (days +1 to +4). On days +4, +7, and +11, OM grade, pain (following a 0\u201310 numeric pain rating scale, NRS) and need for analgesics were evaluated by an operator blinded to treatment. Results: Fifty-one patients were allocated to the PBM group, and 50 were allocated to the sham group. In total, 93.7% of PBM patients and 72% of sham patients had OM grade\ua0<\ua03 WHO on day +7 (P\ua0=\ua00.01). A significant reduction of pain was registered on day +7 in the PBM versus sham group (NRS 1 [0\u20133] vs. 2.5 [1\u20135], P\ua0<\ua00.006). Reduced use of analgesics was reported in the PBM group, although it was not statistically significant. No significant adverse events attributable to treatment were recorded. Conclusions: PBM is a safe, feasible, and effective treatment for children affected by chemotherapy-induced OM, as it accelerates mucosal recovery and reduces pain

Malignant tumors in children living in the province of Trieste, 1972-1993: descriptive epidemiology and the quality of diagnostic-therapeutic services [I tumori maligni nei bambini residenti nella provincia di Trieste, 1972-1993: epidemiologia descrittiva e qualit\ue0 dei servizi diagnostico-terapeutici.]

The study objectives were: 1) to analyse the incidence and death rates from cancer among children aged 0-14 years resident in the north-eastern Italian province of Trieste between 1972-1993, using data from the population-based Trieste Cancer Registry; 2) to evaluate the local diagnostic facilities by analysing the accuracy of histological diagnoses, the causes of delay in the diagnosis, and the interval between onset of symptoms and diagnosis of cancer; 3) to calculate the proportion of patients treated following the most effective therapy protocols known at the time of the tumour detection, and to compute the actuarial five-year survival rates since diagnosis. We recorded 123 new cases of cancer (93% microscopically verified) corresponding to a rate, age-standardized to the world population, of 161.9 (standard error [SE] = 15.1) per million child-years. The most common diagnostic group was that of primary brain tumours: 40 cases, rate = 51.0 (SE = 8.4). In 102 cases the diagnosis was made at hospitals in the province of Trieste, with a median time of seven days (25th-75th percentile = 1-16) between admission and diagnosis. In 37 cases the length of the interval between the advancing of the diagnostic hypothesis of cancer and the microscopic diagnosis ranged from eight to 57 days: 20% of the interval was spent in the identification of the lesion, 50% elapsed between the identification and the biopsy, and 30% was spent in performing the microscopic diagnosis. Out of 123 cases, 30 were partly treated or completely treated at centres not located in our province, i.e., at seven different Italian hospitals (14 cases), nine European hospitals (15 cases), and at one North-American centre (one case). The 40 children with brain tumours were spread among 12 institutions. The five-year survival rate increased from 52.4% (SE = 6.3) for the 63 children with cancers diagnosed in 1972-1981 to 62.5% (SE = 7.0) for the 48 with malignancies detected in 1982-1990

Secondary leukemia in a child with brain stem tumor.

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Methotrexate therapy and liver fibrosis: are human prolyl hydroxylase and type IV collagen reliable and sensitive markers?

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