Optic pathway gliomas (OPGs) occur in 15%-20% of children with neurofibromatosis type 1 (NF1); up to half become symptomatic. There is little information regarding ophthalmologic outcomes after ...chemotherapy. A retrospective multicenter study was undertaken to evaluate visual outcomes following chemotherapy for NF1-associated OPG, to identify risks for visual loss, and to ascertain indications for treatment. Subjects included children undergoing initial treatment for OPGs with chemotherapy between January 1997 and December 2007. Of 115 subjects, visual acuity (VA) decline and tumor progression were the primary reasons to initiate treatment, although there were significant differences in the pattern of indications cited among the institutions. Eighty-eight subjects and 168 eyes were evaluable for VA outcome. At completion of chemotherapy, VA improved (32% of subjects), remained stable (40%), or declined (28%). Tumor location was the most consistent prognostic factor for poor VA outcome. There was poor correlation between radiographic and VA outcomes. Although visual outcomes for NF1-associated OPG are not optimal, approximately one-third of children regain some vision with treatment. Since radiographic outcomes do not predict visual outcomes, their use as the primary measure of treatment success is in question. The lack of consensus regarding the indications for treatment underlines the need for better standardization of care. Future clinical trials for OPG require standardized visual assessment methods and clear definitions of visual outcomes.
Survival rates for children diagnosed with posterior fossa brain tumors (PFBTs) have improved significantly over the past several decades, and long-term functioning assessments have become ...priorities. These evaluations have occurred frequently in adults but only rarely in children. This study describes a cross-sectional assessment of physical functioning in pediatric survivors of PFBTs using the Bruininks-Osteretsky Test of Motor Performance, Second Edition (BOT-2).
Primary analyses compared BOT-2 scores to normative data using 1-sample t tests for each gross motor subscale (Bilateral Coordination, Balance, Running Speed/Agility, Strength) and motor-area composite (Body Coordination and Strength and Agility). Second, the cohort was stratified by diagnostic or treatment variables. Group differences and groups vs norms were evaluated using independent 2-sample and 1-sample t tests, respectively. Primary analyses compared BOT-2 scores with normative data using 1-sample t tests for each gross motor subscale (Bilateral Coordinationcoordination, Balance, Running Speed/Agility, Strength) and motor-area composite (Body Coordination and Strength and Agility). Second, the cohort was stratified by diagnostic or treatment variables. Group differences and groups vs norms were evaluated using independent 2-sample and 1-sample t tests, respectively.
Mean age of 30 participants was 11.4 years (range, 4.9y-18.2y), and mean time from diagnosis was 6.1 years (range, 1.1y-16.7y). Cerebellar astrocytoma (43.3%) and medulloblastoma (40%) were the most common diagnoses. As a group, significantly decreased functioning, compared with norms, was observed in Balance (P < .001) and Running Speed/Agility (P = .005). Specifically in Balance, 21 (70%) participants performed below or well-below average. Participants with a non-astrocytoma performed significantly lower than norms in all areas, independent of age at diagnosis. Survivors with tumors infiltrating the vermis demonstrated significantly lower Body Coordination than norms (P < .001).
Pediatric survivors of PFBTs demonstrated decreased physical functioning, most notably in Balance. These data underscore the need for further research and implementation of physical activity programs aimed specifically at approaches to minimize physical limitations.
To evaluate the pattern of stability and change over time across multiple domains of neurocognitive function in radiated survivors of posterior fossa (PF) tumors.
Thirty-four children (25 males) ...treated for malignant PF tumors were observed with serial clinical neuropsychologic assessments. Thirty patients were treated for medulloblastoma and four patients were treated for ependymoma. Twelve patients were treated with reduced-dose and 21 patients were treated with standard-dose cranial radiation. All patients received an additional boost to the PF. One patient was treated with PF radiation only. Standardized neuropsychologic tests were administered at different times after diagnosis for each child. The rate of change in scores was determined using a mixed model regression.
Results showed a 2- to 4-point decline per year in intelligence scores. For our relatively young sample, intellectual function declined quickly in the first few years after treatment, and then more gradually. Significant declines in visual-motor integration, visual memory, verbal fluency, and executive functioning were also documented. No decline was evident for verbal memory and receptive vocabulary.
Cranial radiation is associated with a decline in multiple neurocognitive domains, with a few notable exceptions. Our results must be interpreted in the context of common limitations of clinical research, including patient variability, changes in test versions, small sample size, and clinical referral bias.
To determine the risk of ventriculoperitoneal (VP) shunt infections after percutaneous retrograde gastrostomy feeding tube (GT) placement in children with brain tumors.
All children (age 0-18 years) ...with primary brain tumors diagnosed and treated at the Hospital for Sick Children, Toronto, Canada, were subjected to a retrospective analysis. Two groups were identified: the study group included children with a VP shunt and a GT; the control group included children with VP shunts only. Each study patient was matched with 2 controls to compare the rate of infections (cohort comparative study).
There were 1,167 children diagnosed and treated with primary brain tumors during the study period (1988-2003); 174 (15%) had a VP shunt and 23 (2%) children had both, a VP shunt and a GT. In the study group (n=17), GTs were inserted at a median time of 80 days (range 6-204 days) after VP shunts. VP shunt infection rate was 23.5% (4/17) compared to 8.8% (3/34) in the control group (OR 3.18; 95% CI 0.622-16.54, p=0.16). Three (75%) of the infection episodes in the study group presented with an ascending VP shunt infection directly related to the GT insertion or manipulation in the first 6 weeks. These GTs were inserted at 13, 47 and 49 days after VP shunt insertion.
Placement of percutaneous retrograde GTs, in the acute phase, in children with brain tumors and VP shunts may increase the risk of ascending meningitis especially if there are early GT-related complications.
To tailor postinduction therapy for stage 4 neuroblastoma in children who are older than 1 year at diagnosis according to status after induction.
From March 1987 to December 1992, 99 patients who ...were consecutively admitted were included in the Lyon-Marseille-Curie East of France (LMCE)3 strategy. After induction with the French Society of Pediatric Oncology NB87 regimen and surgery, patients who were in complete remission immediately proceeded to consolidation therapy with vincristine, melphalan, and fractionated total-body irradiation (VMT). All other patients underwent a postinduction strategy before VMT, either an additional megatherapy regimen or further chemotherapy with etoposide/carboplatin.
The progression-free survival (PFS) is 29% at 7 years from diagnosis, which compares favorably with that of a similar cohort of 72 patients previously reported by our group (LMCE1; PFS of 20% at 5 years and 8% at 14 years, P =.004). In the multivariate analysis, only age younger than 3 years at diagnosis (P =.0085) and achievement of complete or very good partial remission after NB87 and surgery (P =.00024) remained significant. The PFS of the 87 patients who were included in the postinduction strategy was significantly better than that of the comparable 62 patients on the LMCE1 study (32% v 11% at 7 years; P =.005).
The progressive improvements in the LMCE results over the last 10 years suggest that improvements in supportive care measures and increases in each component of this strategy (induction, postinduction, consolidation) may all contribute to increased survival rates.
Purpose
The overall survival and prognostic factors for children with multiply recurrent posterior fossa ependymoma are not well understood. We aimed to assess prognostic factors associated with ...survival for relapsed pediatric posterior fossa ependymoma.
Methods
An institutional database was queried for children with a primary diagnosis of posterior fossa ependymoma from 2000 to 2019. Kaplan–Meier survival analysis and Cox-proportional hazard regression were used to assess the relationship between treatment factors and overall survival.
Results
There were 60 patients identified; molecular subtype was available for 56, of which 49 (87.5%) were PF-A and 7 (12.5%) were PF-B. Relapse occurred in 29 patients (48%) at a mean time of 24 months following primary resection. Median 50% survival was 12.3 years for all patients and 3.3 years following diagnosis of first relapsed disease. GTR was associated with significantly improved survival following primary resection (HR 0.373, 95% CI 0.14–0.96). Presence of recurrent disease was significantly associated with worse survival (p < 0.0001). At recurrent disease diagnosis, disseminated disease was a negative prognostic factor (HR 11.0 95% CI 2.7–44) while GTR at first relapse was associated with improved survival HR 0.215 (95% CI: 0.048–0.96, p = 0.044). Beyond first relapse, the impact of GTR was not significant on survival, though surgery compared to no surgery was favorable with HR 0.155 (95% CI: 0.04–0.59).
Conclusions
Disseminated disease at recurrence and extent of resection for first relapsed disease were important prognostic factors. Surgery compared to no surgery was associated with improved survival for the multiply recurrent ependymoma cohort.
PDF
