Faculty Bibliography

OBJECT: The authors set out to evaluate the quality of life (QOL), social-emotional functioning, and behavioral functioning of children treated surgically for craniopharyngiomas. METHODS: Twelve girls and 17 boys with a mean age at diagnosis of 8 +/- 3.8 years were surgically treated between 1985 and 1998 at the New York University Medical Center. After a mean follow-up period of 6.8 +/- 3.5 years, these 29 patients were administered either the 36-item Short Form Health Survey version 2 or the Child Health Questionnaire-Parent Form to assess QOL, as well as the Achenbach Child Behavior Checklist or Young Adult Checklist to measure social-emotional and behavioral functioning. Patients older than 19 years of age and parents of patients younger than 19 years of age reported low average overall physical QOL, with overall psychosocial QOL in the average range. Behavioral difficulties were noted, including internalizing, attention, somatic, and social difficulties. Further analyses indicated that retrochiasmatic tumor location, recurrence, and additional surgery were associated with poorer outcomes. In contrast, hydrocephalus, tumor size, and sex were not prognostic variables, and patients significantly improved as post-operative time increased. CONCLUSIONS: Attention toward late effects arising after the treatment of pediatric craniopharyngioma, including decreased postoperative physical health and behavioral functioning, is warranted. Future approaches to treatment should consider the documented effects of either gross-total resection or limited surgery followed by cranial irradiation on QOL, with specific evaluation for those with retrochiasmatic tumors, a recurrent tumor, or the need for additional surgery. Psychosocial QOL and social-emotional functioning should be maintained through ongoing counseling and education

PURPOSE: Clinicians often assume that children with posterior fossa tumors are at minimal risk for cognitive or adaptive deficits if they do not undergo cranial irradiation. However, small case series have called that assumption into question, and have also suggested that nonirradiated cerebellar tumors can cause location-specific cognitive and adaptive impairment. This study (1) assessed whether resected but not irradiated pediatric cerebellar tumors are associated with cognitive and adaptive functioning deficits, and (2) examined the effect of tumor location and medical complications on cognitive and adaptive functioning. PATIENTS AND METHODS: The sample was composed of 103 children aged 3 to 18 years with low-grade cerebellar astrocytomas, who underwent only surgical treatment as part of Children's Cancer Group protocol 9891 or Pediatric Oncology Group protocol 9130. The sample was divided into three groups based on primary tumor location: vermis, left hemisphere, or right hemisphere. Data were collected prospectively on intelligence, academic achievement, adaptive skills, behavioral functioning, and pre-, peri-, and postsurgical medical complications. RESULTS: The sample as a whole displayed an elevated risk for cognitive and adaptive impairment that was not associated consistently with medical complications. Within this group of children with cerebellar tumors, tumor location had little effect on cognitive, adaptive, or medical outcome. CONCLUSION: We did not replicate previous findings of location-specific effects on cognitive or adaptive outcome. However, the elevated risk of deficits in this population runs contrary to clinical lore, and suggests that clinicians should attend to the functional outcomes of children who undergo only surgical treatment for cerebellar tumors

OBJECT: Tectal gliomas are a distinct form of pediatric brainstem tumor that present in patients with symptoms related to increased intracranial pressure due to obstructive hydrocephalus. The natural history of these lesions is often uniquely indolent. Thus, initial surgical therapies are directed at treatment of hydrocephalus, usually with ventricular shunt placement. Recently, third ventriculostomy has been used in patients with tectal gliomas, both as an initial procedure and after shunt failures. In this report the authors review their experience with the treatment of hydrocephalus in patients with tectal gliomas. METHODS: The authors reviewed 31 consecutive cases of tectal gliomas and compared the success rates of ventricular shunt placement with the success rates of endoscopic third ventriculostomy (ETV). Shunt placement procedures were associated with a significant number of malfunctions, and most patients required shunt revisions. The ETV procedure was attempted both as an initial treatment and after shunt malfunction. Overall, ETV was attempted in 18 patients and was performed successfully in all cases. At the time of follow-up evaluation, 16 patients (89%) were shunt free. CONCLUSIONS: The authors found that ETV could be performed with good long-term success both as an initial treatment and after shunt failure. Overall, ETV was found to be superior to ventricular shunt placement in the management of hydrocephalus associated with tectal gliomas.

Medulloepithelioma is a rare primitive neuroectodermal tumor of the central nervous system usually developing in childhood, displaying highly malignant behavior, with early progression or recurrence. Once a recurrence develops, death reportedly follows with invariable rapidity. The purpose of this study was to evaluate the efficacy of high-dose, marrow-ablative chemotherapy with autologous hemopoietic stem cell rescue in the treatment of recurrent central nervous system medulloepithelioma. Three young children with recurrent central nervous system medulloepithelioma received high-dose marrow-ablative chemotherapy with thiotepa and etoposide either alone (one patient) or with the addition of carboplatin (two patients). This was followed by irradiation only in one patient. One child with residual radiographic tumor at the time of treatment could be evaluated for response and showed complete resolution of leptomeningeal disease after receiving marrow-ablative chemotherapy. Two children developed tumor recurrence at 2.0 and 5.5 months after receiving marrow-ablative chemotherapy. The third child continues free of tumor beyond 12 years from treatment. The authors' experience with marrow-ablative chemotherapy and autologous hemopoietic stem cell rescue suggests that this treatment strategy might be beneficially incorporated into the initial treatment approach for young children with medulloepithelioma

OBJECTIVE: Although optimal treatment for intrinsic focal tumors of the medulla remains controversial, many surgeons advocate radical surgery for patients with these tumors. Postoperative surgical morbidity may include loss of lower cranial nerve function and significant motor deficits. Recovery of lower cranial nerve dysfunction after radical surgery has not been reported previously. METHODS: Forty-one children and adolescents with tumors involving the medulla underwent operations between 1986 and 1997. Nineteen (46%) of these patients experienced loss of lower cranial nerve function requiring tracheostomy, ventilator support, and feeding gastrostomy. A retrospective analysis of this patient population and the time to cranial nerve recovery was undertaken. RESULTS: Thirteen (68%) of the 19 patients with loss of lower cranial nerve function had full recovery of lower cranial nerve function. Two patients (11%) have had significant improvement in their lower cranial nerve function, and four patients (21%) have remained without lower cranial nerve function. CONCLUSION: Lower cranial dysfunction is common after surgery for intrinsic medullary tumors. However, the majority of patients who require tracheostomy or gastrostomy tubes will recover cranial nerve function

PURPOSE: To evaluate the feasibility of and response rate to an intensified induction chemotherapy regimen for young children with newly diagnosed high-risk or disseminated medulloblastomas. PATIENTS AND METHODS: From January 1997 to March 2003, 21 patients with high-risk or disseminated medulloblastoma were enrolled. After maximal surgical resection, patients were treated with five cycles of vincristine (0.05 mg/kg/wk x three doses per cycle for three cycles), cisplatin (3.5 mg/kg per cycle), etoposide (4 mg/kg/d x 2 days per cycle), cyclophosphamide (65 mg/kg/d x 2 days per cycle) with mesna, and methotrexate (400 mg/kg per cycle) with leucovorin rescue. Following induction chemotherapy, eligible patients underwent a single myeloablative chemotherapy cycle with autologous stem-cell rescue. RESULTS: Significant toxicities of this intensified regimen, including gastrointestinal and infectious toxicities, are described. Among the 21 patients enrolled, there were 17 complete responses (81%), two partial responses, one stable disease, and one progressive disease. The 3-year event-free survival and overall survival are 49% (95% CI, 27% to 72%) and 60% (95% CI, 36% to 84%), respectively. CONCLUSION: This intensified induction chemotherapy regimen is feasible and tolerable. With the majority of patients with disseminated medulloblastoma having M2 or M3 disease at diagnosis, the encouraging high response rate of this intensified induction regimen suggests that such an addition of methotrexate should be explored in future studies