Correlation of Technical and Adjunctive Factors with Quantitative Tumor Reduction in Children Undergoing Selective Ophthalmic Artery Infusion Chemotherapy for Retinoblastoma.

BACKGROUND AND PURPOSE: Selective ophthalmic artery infusion chemotherapy has improved ocular outcomes in children with retinoblastoma. Our aim was to correlate quantitative tumor reduction and dichotomous therapeutic response with technical and adjunctive factors during selective ophthalmic artery infusion chemotherapy for retinoblastoma. An understanding of such factors may improve therapeutic efficacy. MATERIALS AND METHODS: All patients with retinoblastoma treated by selective ophthalmic artery infusion chemotherapy at a single center during a 9-year period were reviewed. Only first-cycle treatments for previously untreated eyes were studied. Adjunctive factors (intra-arterial verapamil, intranasal oxymetazoline external carotid balloon occlusion) and technical factors (chemotherapy infusion time, fluoroscopy time) were documented by medical record review. Quantitative tumor reduction was determined by blinded comparison of retinal imaging acquired during examination under anesthesia before and 3-4 weeks after treatment. The dichotomous therapeutic response was classified according to quantitative tumor reduction as satisfactory (≥ 50%) or poor (<50%). RESULTS: Twenty-one eyes met the inclusion criteria. Patients ranged from 2 to 59 months of age. Adjuncts included intra-arterial verapamil in 15, intranasal oxymetazoline in 14, and external carotid balloon occlusion in 14. Quantitative tumor reduction ranged from 15% to 95%. Six showed poor dichotomous therapeutic response. A satisfactory dichotomous therapeutic response was correlated with intra-arterial verapamil (P = .03) in the aggregate cohort and in a subgroup undergoing treatment with single-agent melphalan at a dose of <5 mg (P = .02). In the latter, higher average quantitative tumor reduction correlated with intra-arterial verapamil (P < .01). CONCLUSIONS: Intra-arterial verapamil during selective ophthalmic artery infusion chemotherapy is correlated with an improved therapeutic response, particularly when treating with lower doses of single-agent melphalan.

Incidental inflammatory adenoma with ß-catenin activation in the setting of paediatric NASH.

Hepatocellular adenomas (HCA) are benign tumours with potential for malignant transformation with no recommendations regarding management in the paediatric population. We report a case of an inflammatory adenoma with ß-catenin activated pathway in an obese, paediatric patient with nonalcoholic steatohepatitis (NASH). CASE REPORT: An 11-year-old female presented with a microlobulated liver lesion measuring >5 cm in magnetic resonance imaging (MRI) with inflammatory adenoma with ß-catenin activated pathology arising in a background of NASH, nonalcoholic fatty liver disease (NAFLD) activity score 5/8. Imaging follow-up demonstrated stable disease without progression for 3 years. DISCUSSION: Malignant transformation of Hepatocellular adenomas in a child is approximately 4.2%. It is unknown if hepatic steatosis increases this risk. Obese patients mainly develop inflammatory and ß-catenin activated (highest risk for malignant transformation) adenomas. Our patient had inflammatory and ß-catenin activation, which led to monitoring for malignant transformation. CONCLUSION: We report a ß-catenin activated inflammatory adenoma in a child with obesity and NASH with ongoing expectant management.

Pediatric laryngeal carcinoma in a heterozygous carrier of Fanconi anemia.

A case of invasive, keratinizing squamous cell carcinoma of the larynx in an 8-year-old female treated with laryngectomy is presented. Perinatal exposure to human papilloma virus and constitutional heterozygosity for a FANCC mutation were identified, though FANCC heterozygosity is not known to be cancer predisposing. An additional tumor-associated mutation in NOTCH1 was also identified potentially contributing to oncogenesis. This case illustrates an exceedingly rare type of cancer in the pediatric population and discusses diagnostic workup, evaluation of risk factors for head and neck cancer, and treatment options.

Enzymatic analysis of 2,5-anhydro-D-mannitol and related compounds.

Two enzymatic assay procedures for the measurement of 2,5-anhydrohexitol fructose analogs have been devised. Both procedures are based on the measurement of ADP formed during enzymatic phosphorylation of the analogs either by hexokinase or by fructokinase. The actual measurement makes use of the coupled assay system using pyruvate kinase, PEP, lactate dehydrogenase, and NADH. Both systems can be used to measure fructose and appropriate analogs at cuvette concentrations up to 0.10 mM. The hexokinase procedures allows the measurement of fructose, 2,5-anhydromannitol, and 2,5-anhydromannose. Glucose, which also reacts, can be removed by pretreatment of the samples with glucose oxidase. The fructokinase procedure allows the measurement of fructose, 2,5-anhydromannitol, 2,5-anhydroglucitol, and 2,5-anhydrotalitol.