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Desmoplastic small round cell tumor (DSCRT) is a rare malignancy most often seen in the abdomen or pelvis of young men. Unfortunately, this disease is usually metastatic at diagnosis and has dismal outcomes. We describe a case of isolated paratesticular DSCRT in a 14-year-old male successfully treated with surgical resection, chemotherapy, and adjuvant radiation, and we present a review of the relevant literature. It appears that isolated, paratesticular DSCRTs have a markedly better outcome than the classic abdominal or pelvic location. We hypothesize that this is due to earlier detection and the relative ease of surgical resection., (© 2017 Wiley Periodicals, Inc.)

Background: Prior studies suggest that neuroblastomas that do not accumulate metaiodobenzylguanidine (MIBG) on diagnostic imaging (MIBG non-avid) may have more favorable features compared with MIBG avid tumors. We compared clinical features, biologic features, and clinical outcomes between patients with MIBG nonavid and MIBG avid neuroblastoma., Procedure: Patients had metastatic high- or intermediate-risk neuroblastoma and were treated on Children's Oncology Group protocols A3973 or A3961. Comparisons of clinical and biologic features according to MIBG avidity were made with chi-squared or Fisher exact tests. Event-free (EFS) and overall (OS) survival compared using log-rank tests and modeled using Cox models., Results: Thirty of 343 patients (8.7%) had MIBG nonavid disease. Patients with nonavid tumors were less likely to have adrenal primary tumors (34.5 vs. 57.2%; P = 0.019), bone metastases (36.7 vs. 61.7%; P = 0.008), or positive urine catecholamines (66.7 vs. 91.0%; P < 0.001) compared with patients with MIBG avid tumors. Nonavid tumors were more likely to be MYCN amplified (53.8 vs. 32.6%; P = 0.030) and had lower norepinephrine transporter expression. Patients with MIBG nonavid disease had a 5-year EFS of 50.0% compared with 38.7% for patients with MIBG avid disease (P = 0.028). On multivariate testing in high-risk patients, MIBG avidity was the sole adverse prognostic factor for EFS identified (hazard ratio 1.77; 95% confidence interval 1.04-2.99; P = 0.034)., Conclusions: Patients with MIBG nonavid neuroblastoma have lower rates of adrenal primary tumors, bone metastasis, and catecholamine secretion. Despite being more likely to have MYCN-amplified tumors, these patients have superior outcomes compared with patients with MIBG avid disease., (© 2017 Wiley Periodicals, Inc.)

Purpose: Prognosis for relapsed/refractory high-risk neuroblastoma (HR-NBL) remains poor. Bortezomib, a proteasome inhibitor, has shown preclinical activity against NBL as a single agent and in combination with cytotoxic chemotherapy including irinotecan., Patients and Methods: Eighteen HR-NBL patients with primary refractory (n = 8) or relapsed (n = 10) disease were enrolled in a Phase I study using modified Time To Event Continual Reassessment Method. Bortezomib (1.2 mg/m 2 /day) was administered on days 1, 4, 8, and 11 intravenously (IV) and irinotecan was given IV on days 1-5 (35, 40, or 45 mg/m 2 /day, on dose levels [DL] 1-3, respectively). The maximum tolerated dose (MTD), dose-limiting toxicity (DLT), and response rate were examined., Results: Eighteen NBL patients were evaluable for toxicity; 17 were evaluable for response assessment. A total of 142 courses were delivered (mean 8.2, median 2, range 1-48), with two patients receiving more than 40 courses of therapy. Two DLTs were reported, including a grade 4 thrombocytopenia (DL2) and a grade 3 irritability (DL3). MTD was estimated as DL3. Two of 17 (12%) evaluable patients showed objective responses (ORs) lasting more than 40 courses, including 1 partial remission and 1 complete remission. Four patients (23%) had prolonged stable disease (SD) lasting six or more courses, with a total of 35% study patients demonstrating clinical benefit in the form of prolonged OR or SD., Conclusion: The combination of bortezomib and irinotecan was well tolerated by patients with relapsed/refractory NBL with favorable toxicity profile. It also showed modest but promising clinical activity and merits further testing in Phase II studies., (© 2017 Wiley Periodicals, Inc.)

Pediatric spinal oligodendrogliomas are rare and aggressive tumors. They do not share the same molecular features of adult oligodendroglioma, and no previous reports have examined the molecular features of pediatric spinal oligodendroglioma. We present the case of a child with a recurrent spinal anaplastic oligodendroglioma. We performed whole exome (paired tumor and germline DNA) and transcriptome (tumor RNA) sequencing, which revealed somatic mutations in NF1 and FGFR1. These data allowed us to explore potential personalized therapies for this patient and expose molecular drivers that may be involved in similar cases., (© 2016 Wiley Periodicals, Inc.)

Background: Little is known about emergency department (ED) use among pediatric patients with cancer. We explored reasons prompting ED visits and factors associated with hospital admission., Procedure: A retrospective cohort analysis of pediatric ED visits from 2006 to 2010 using the Nationwide Emergency Department Sample, the largest all-payer database of United States ED visits. Pediatric patients with cancer (ages ≤19 years) were identified using Clinical Classification Software. Proportion of visits and disposition for the top ten-ranking non-cancer diagnoses were determined. Weighted multivariate logistic regression was performed to analyze factors associated with admission versus discharge., Results: There were 294,289 ED visits by pediatric patients with cancer in the U.S. over the study period. Fever and fever with neutropenia (FN) were the two most common diagnoses, accounting for almost 20% of visits. Forty-four percent of pediatric patients with cancer were admitted to the same hospital, with admission rates up to 82% for FN. Risk factors for admission were: FN (odds ratio (OR) 8.58; 95% confidence interval (CI) 5.97-12.34); neutropenia alone (OR 7.28; 95% CI 5.08-10.43), ages 0-4 years compared with 15-19 years (OR 1.19; 95% CI 1.08-1.31) and highest median household income ZIP code (OR 1.27; 95% CI 1.08-1.49) compared with lowest. "Self-pay" visits had lower odds of admission (OR 0.42; 95% CI 0.35-0.51) compared with public payer., Conclusion: FN was the most common reason for ED visits among pediatric patients with cancer and is the condition most strongly associated with admission. Socioeconomic factors appear to influence ED disposition for this population., (© 2014 Wiley Periodicals, Inc.)