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While irinotecan has been studied in various pediatric solid tumors, its potential role in Wilms tumor (WT) is less clear. We evaluated response and outcome of irinotecan-containing regimens in relapsed WT and compared our results to the available literature. Among 14 evaluable patients, one complete response (CR) and two partial responses (PRs) were observed in patients with initial intermediate-risk (CR and PR) and blastemal-type histologies (PR). Two patients were alive at last follow-up showing no evidence of disease. Our results and the reviewed literature suggest some effectiveness of irinotecan in the setting of relapsed WT., (© 2017 Wiley Periodicals, Inc.)

Background: Children with Wilms' tumor (WT) aged under 24 months (infants) have a better prognosis than older patients. Our aim was to study the epidemiology of this age group, with focus on the modality of diagnosis, tumor size, and association with malformations/syndromes, seeking to understand if any of these factors might be related to prognosis., Patients and Methods: Infants diagnosed with WT between 2003 and February 2010 were evaluated. A query form was used to collect data on the modality of WT diagnosis (symptomatic or incidental), tumor volume, maximum diameter, site, and stage., Results: Data were collected for 117 of 124 WT infants registered. Twenty-four cases had an incidental diagnosis (ID) of renal mass, usually arising from an abdominal ultrasound performed for other reasons, and 93 had been diagnosed based on clinical signs/symptoms. The incidental cohort displayed unifocal disease, mean tumor diameter 5.52 cm, mean tumor volume 84.30 ml, and 14 patients showed associated malformations. Symptomatic patients had mean maximum tumor diameter of 10.18 cm, mean tumor volume of 451.18 ml, and six had associated malformations., Conclusions: Our study showed that 20% of the infants had an ID of WT; they had a relatively smaller nonmetastatic tumor and a higher rate of malformations than infants of the symptomatically diagnosed group, but we did not detect any difference in age at diagnosis between the two groups. Conversely, we found a significant difference in the 5-year event-free survival rate (P = 0.018) between infants under 1 year (96%), more frequently associated with congenital malformations, and infants 1-2 years (80%)., (© 2017 Wiley Periodicals, Inc.)

Background: Various projects dedicated specifically to adolescents and young adults (AYA) with cancer have been developed in recent years. A critical aspect of such programs is the ability to demonstrate its value, and therefore how to measure desired outcomes., Methods: A list of metrics to consider for demonstrating the advantages of an AYA program was identified and used to assess the activity of the Youth Project operating at the Pediatric Oncology Unit of the Istituto Nazionale Tumori in Milan., Results: The number of newly diagnosed AYA patients seen at the Unit has increased since the formal launch of the Youth Project, from 65 to 81.2 cases/year. Concerning the 78 AYA patients presenting with malignant neoplasms in 2015, 82% were included in clinical trials (the other 18% in prospective observational studies). Fertility preservation measures were implemented for 59% of AYA patients considered at risk, and specific psychological support was provided in 70.6% of cases; 72.5% of patients actively participated in support activities. Other parameters considered were a preliminary satisfaction questionnaire administered to patients and the program's scientific recognition and acknowledgment by the community., Conclusions: The study proposed a number of potentially reproducible, practical parameters to consider in assessing the value of a program dedicated to AYA. These metrics were examined in terms of the activities of our Youth Project, and confirmed its efficacy. To be sustainable over time, AYA projects have to be accepted as a standard of care at the community and government levels., (© 2016 Wiley Periodicals, Inc.)

Background: The potential impact of diagnostic delays on patients' outcomes is a debated issue in pediatric oncology and discordant results have been published so far. We attempted to tackle this issue by analyzing a prospective series of 351 consecutive children and adolescents with solid malignancies using innovative statistical tools., Methods: To address the nonlinear complexity of the association between symptom interval and overall survival (OS), a regression tree algorithm was constructed with sequential binary splitting rules and used to identify homogeneous patient groups vis-à-vis functional relationship between diagnostic delay and OS., Results: Three different groups were identified: group A, with localized disease and good prognosis (5-year OS 85.4%); group B, with locally or regionally advanced, or metastatic disease and intermediate prognosis (5-year OS 72.9%), including neuroblastoma, Wilms tumor, non-rhabdomyosarcoma soft tissue sarcoma, and germ cell tumor; and group C, with locally or regionally advanced, or metastatic disease and poor prognosis (5-year OS 45%), including brain tumors, rhabdomyosarcoma, and bone sarcoma. The functional relationship between symptom interval and mortality risk differed between the three subgroups, there being no association in group A (hazard ratio [HR]: 0.96), a positive linear association in group B (HR: 1.48), and a negative linear association in group C (HR: 0.61)., Conclusions: Our analysis suggests that at least a subset of patients can benefit from an earlier diagnosis in terms of survival. For others, intrinsic aggressiveness may mask the potential effect of diagnostic delays. Based on these findings, early diagnosis should remain a goal for pediatric cancer patients., (© 2015 Wiley Periodicals, Inc.)

Background: Topotecan has been variably incorporated in the treatment of patients with relapsed Wilms tumour (WT) who failed initial treatment with three or more effective drugs. Our objective was to describe outcome and to retrospectively investigate the potential role of topotecan in relapsed WT patients., Methods: Children who were treated with topotecan as part of their chemotherapeutic regimens for relapsed WT were identified and included in our retrospective study. Patient charts were reviewed for general patient characteristics, histology and stage at initial diagnosis, number and type of relapse, salvage treatment schedules, toxicity, response to treatment and outcome., Results: From 2000 to 2012, 30 children (median age at relapse 5.5 years, range 1.6-14.5 years) were identified to have received topotecan as part of their salvage regimens (primary progressive disease n = 3, first, second and third relapse n = 13, 9 and 2 respectively, partial response n = 3). Topotecan was administered as a single agent (12 patients) or in combination with other drugs (18 patients). Sixteen patients had high-risk histology according to the SIOP classification, 15 died within 12 months because of progressive disease. Fourteen patients had SIOP intermediate-risk histology of which four patients displayed objective responses to topotecan. Overall, 6 out of 14 intermediate-risk patients survived (median follow up of 6 years), however, three of whom (stage V) had bilateral nephrectomy after topotecan treatment., Conclusions: Topotecan does not seem to show effectiveness in the treatment of relapsed WT patients with initial high-risk histology. In patients with intermediate-risk histology, the role of topotecan might deserve further attention, to prove its efficacy., (© 2014 Wiley Periodicals, Inc.)

Background: The awareness that adolescents can have cancer is probably insufficient, not only among teenagers and their families, but also among physicians, and adolescent patients are reportedly often referred to qualified cancer institutes after a considerable delay., Procedure: A prospective series of 425 patients (28% of them adolescents) with solid tumors was analyzed to investigate the correlation between symptom interval and age, and the different contributions to symptom interval in terms of the time from symptom onset to the first contact with a doctor (patient delay), referral to the oncologist (referral delay), and final diagnosis (oncologist delay)., Results: The median symptom interval was 47 days for 0 to 14-year-old patients and 137 for those ≥15 years (P < 0.001). The greatest delay in the adolescent group related to the patient delay (63.3% of the total symptom interval)., Conclusion: Adolescents are often diagnosed with longer delay as compared to children. The main contribution to symptom interval in adolescents appears to be due to the time they first go to a doctor; however, also the time taken by the physician to the patient to a specialist (oncologist or surgeon) able to define the diagnosis of cancer was longer for adolescents than for younger patients., (Copyright © 2012 Wiley Periodicals, Inc.)

Hypothyroidism remains a common late effect after irradiation of the neck/mediastinum for Hodgkins lymphoma (HL). We evaluated the protective effect of TSH suppression during neck/mediastinum irradiation. From 1998 to 2001, 14 consecutive euthyroid children were given, before and until the end of their radiotherapy on neck/mediastinum, L-thyroxine at TSH-suppressive doses. The 14 patients had adequate TSH suppression during irradiation in 8, inadequate in 6. The 8-year hypothyroidism-free-survival after irradiation was 75 ± 15% for the former group, 0% for the latter (P = 0.009). TSH suppression could have a protective effect on thyroid function as shown in a small group of patients with HL., (Copyright © 2011 Wiley-Liss, Inc.)

The combination of hyponatremia and renovascular hypertension is known as hyponatremic-hypertensive syndrome (HHS) and so rarely described in children but associated with various kinds of occlusions of the renal artery. We describe two children who presented HHS with severe hypokalemia, polyuria, and polydipsia associated with Wilms tumor, which required treatment with an angiotensin-converting enzyme inhibitor before nephrectomy. All HHS signs and symptoms resolved only following surgical resection of the tumor, allowing chemotherapy to be given., (2010 Wiley-Liss, Inc.)

Purpose: This analysis evaluates whether adjuvant chemotherapy can be recommended for high-risk, surgically-resected, adult-type non-rhabdomyosarcoma soft tissue sarcomas (NRSTS) within the new European Pediatric Soft Tissue Sarcoma Study Group (EpSSG) protocol. The Italian and German Cooperative Groups reviewed their data-bases, analyzing patients classified as group I-II, with high-grade tumor (G3) larger than 5 cm in size., Methods: The analysis included 36 patients, and compared the clinical features and outcome of the group of 21 patients who received chemotherapy versus the group of 15 patients treated with local therapies only., Results: For the series as a whole, 5-year event-free survival (EFS), metastasis-free survival (MFS), and overall survival (OS) were 26.2%, 34.0%, and 37.5%, respectively. In patients treated with chemotherapy, MFS and OS were 49.5% and 41.5% (median time to relapse: 13 months). In patients who did not receive chemotherapy, MFS and OS were 0% and 23.8% (median time to relapse: 3 months)., Conclusion: The role of adjuvant chemotherapy in NRSTS is still uncertain, however, the current retrospective analysis showed that: (1) despite the globally good prognosis of grossly-resected cases, patients with G3 and large-size have a high-risk of metastatic spread, and (2) MFS appears to be better in patients who had chemotherapy. Based in part on these results, and in accordance with recent suggestions coming from the literature on adult sarcomas, the EpSSG NRSTS protocol will recommend adjuvant chemotherapy in high-risk surgically-resected patients.