A B S T R A C T PurposeSurgery is curative therapy for pediatric low-grade gliomas (LGGs) in areas of the brain amenable to complete resection. However, LGGs located in areas where complete resection is not possible can threaten both function and life. The purpose of this study was to compare two chemotherapy regimens for LGGs in children younger than age 10 years for whom radiotherapy was felt by the practitioner to pose a high risk of neurodevelopmental injury. Patients and MethodsPreviously untreated children younger than age 10 years with progressive or residual LGGs were eligible. Children were randomly assigned to receive carboplatin and vincristine (CV) or thioguanine, procarbazine, lomustine, and vincristine (TPCV). Children with neurofibromatosis are reported separately. ResultsOf 274 randomly assigned patients who met eligibility requirements, 137 received CV and 137 received TPCV. The 5-year event-free survival (EFS) and overall survival (OS) rates for all eligible patients were 45% Ϯ 3.2% and 86% Ϯ 2.2%, respectively. The 5-year EFS rates were 39% Ϯ 4% for CV and 52% Ϯ 5% for TPCV (stratified log-rank test P ϭ .10; cure model analysis P ϭ .007). On multivariate analysis, factors independently predictive of worse EFS and OS were younger age and tumor size greater than 3 cm 2 . Tumor location in the thalamus was also associated with poor OS. ConclusionThe difference in EFS between the regimens did not reach significance on the basis of the stratified log-rank test. The 5-year EFS was higher for TPCV on the basis of the cure model analysis. Differences in toxicity may influence physician choice of regimens.

PURPOSE Atypical teratoid/rhabdoid tumor (AT/RT) is an aggressive, early-childhood brain tumor without standard effective treatment. To our knowledge, we conducted the first AT/RT-specific cooperative group trial, ACNS0333, to examine the efficacy and safety of intensive postoperative chemotherapy and focal radiation to treat AT/RT. PATIENTS AND METHODS Patients from birth to 22 years of age with AT/RT were eligible. After surgery, they received 2 courses of multiagent chemotherapy, followed by 3 courses of high-dose chemotherapy with peripheral blood stem cell rescue and involved-field radiation therapy. Timing of radiation was based on patient age and disease location and extent. Central testing of tumor and blood for SMARCB1 status was mandated. Tumor molecular subclassification was performed retrospectively. The primary analysis was event-free survival (EFS) for patients < 36 months of age compared with a cooperative groups’ historical cohort. Although accrual was based on the therapeutic question, potential prognostic factors, including age, tumor location, M stage, surgical resection, order of therapy, germline status, and molecular subtype, were explored. RESULTS Of 65 evaluable patients, 54 were < 36 months of age. ACNS0333 therapy significantly reduced the risk of EFS events in patients < 36 months of age compared with the historical cohort ( P < .0005; hazard rate, 0.43; 95% CI, 0.28 to 0.66). Four-year EFS and overall survival for the entire cohort were 37% (95% CI, 25% to 49%) and 43% (95% CI, 31% to 55%), respectively. Timing of radiation did not affect survival, and 91% of relapses occurred by 2 years from enrollment. Treatment-related deaths occurred in 4 patients. CONCLUSION The ACNS0333 regimen dramatically improved survival compared with historical therapies for patients with AT/RT. Clinical characteristics and molecular subgrouping suggest prognostic differences. ACNS0333 results lay a foundation on which to build future studies and incorporate testing of new therapeutic agents.

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A variety of congenital midface anomalies occur in children. High-resolution computed tomography (CT) and magnetic resonance (MR) imaging have proved helpful in determining the nature and extent of dysplasia, thereby facilitating treatment planning. A classification system has been developed that groups these anomalies into four categories based on embryogenesis and anatomic location. These categories comprise anomalies that are related to the nasal cavity, nasofrontal region, nasolacrimal apparatus, and craniofacial syndromes. CT is the imaging modality of choice in children with possible choanal atresia, pyriform aperture stenosis, or anomalies of the nasolacrimal duct (eg, nasolacrimal duct stenosis, dacryocystoceles). MR imaging is the modality of choice in patients with congenital midface masses (eg, dermoid and epidermoid cysts, nasal gliomas, encephaloceles) and craniofacial syndromes (eg, Apert syndrome, Crouzon syndrome, Treacher Collins syndrome). In many cases, however, both CT and MR imaging are required to adequately evaluate midface anomalies. Familiarity with the characteristic imaging features of these anomalies along with knowledge of midface embryogenesis and normal developmental anatomy is essential to prevent misinterpretation of anatomic variations that may simulate disease.