Survival in pediatric medulloblastoma: a population-based observational study to improve prognostication

Abstract

Medulloblastoma is the most common form of brain malignancy of childhood. The mainstay of epidemiological data regarding childhood medulloblastoma is derived from case series, hence population-based studies are warranted to improve the accuracy of survival estimates. To utilize a big-data approach to update survival estimates in a contemporary cohort of children with medulloblastoma. We performed a population-based retrospective observational cohort study utilizing the Surveillance, Epidemiology, and End Results Program database that captures all children, less than 20 years of age, between 1973 and 2012 in 18 geographical regions representing 28% of the US population. We included all participants with a presumed or histologically diagnosis of medulloblastoma. The main outcome of interest is survivors at 1, 5 and 10 years following diagnosis. A cohort of 1735 children with a median (interquartile range) age at diagnosis of 7 (4-11) years, with a diagnosis of medulloblastoma were identified. The incidence and prevalence of pediatric medulloblastoma has remained stable over the past 4 decades. There is a critical time point at 1990 when the overall survival has drastically improved. In the contemporary cohort (1990 onwards), the percentage of participants alive was 86, 70 and 63% at 1, 5 and 10 years, respectively. Multivariate Cox-Regression model demonstrated Radiation (HR 0.37; 95% CI 0.30-0.46, p < 0.001) and Surgery (HR 0.42; 95% CI 0.30-0.58, p < 0.001) independently predict survival. The probability of mortality from a neurological cause is <5% in patients who are alive 8 years following diagnosis. The SEER cohort analysis demonstrates significant improvements in pediatric medulloblastoma survival. In contrast to previous reports, the majority of patients survive in the modern era, and those alive 8 years following initial diagnosis are likely a long-term survivor. The importance of minimizing treatment-related toxicity is increasingly apparent given the likelihood of long-term survival.

Survival difference between brainstem and cerebellum medulloblastoma: the surveillance, epidemiology, and end results-based study

“…Another SEER-based study for the pediatric MB cohort drew a similar conclusion regarding radiotherapy improving survival (HR = 0.37). [ 13 ] However, the benefit of chemotherapy remains unclear. [ 19 ] A cohort of 66 adults with MB failed to show a benefit from chemotherapy for the cohort as a whole, but there was a trend for improvement in 5-year PFS from 36% to 71% and in 5-year OS from 49% to 100%.…”

Tumor Location and Treatment Modality are Associated with Overall Survival in Adult Medulloblastoma

“…We found that when further stratifying MB by location, that lesions found in the cerebellum had better outcomes than rarer MB tumors found in other infratentorial locations such as the brain stem or ventricles. In terms of treatment modality, a recent study by Weil et al demonstrated that radiation and surgery independently predict better survival outcome for children [11]. The influences of these treatments were not extensively studied in adults.…”

Posttreatment Maturation of Medulloblastoma into Gangliocytoma: Report of 2 Cases

“…Medulloblastoma occurs in the posterior fossa and presents most commonly with headaches, nausea, emesis, ataxia, hydrocephalus, and/or cranial nerve deficits. The overall survival for children in the USA diagnosed with medulloblastoma at 1, 5, and 10 years is estimated at 86, 70, and 63%, respectively [10]. Standard treatment includes surgical resection, craniospinal irradiation (in children above the age of 3 years), and combination chemotherapy.…”