title: Long-term neuropsychological follow-up of young children with medulloblastoma treated with sequential high-dose chemotherapy and irradiation sparing approach.
abstract: High-dose chemotherapy (HDC) strategies were developed in brain tumor protocols for young children to prevent neuropsychological (NP) impairments associated with radiotherapy. However, comprehensive NP evaluations of these children treated with such strategies remain limited. We examined the long-term neurocognitive outcomes of young children (<6 years) with medulloblastoma, treated similarly, with a HDC strategy "according to" the chemotherapy regimen of the protocol CCG 99703. This retrospective study included young children less than 6 years of age at diagnosis of medulloblastoma treated from 1998 to 2011 at 7 North American institutions. Twenty-four patients who had at least one NP assessment post-treatment are the focus of the current study. Of 24 patients in this review, 15 (63%) were male and the mean age at diagnosis was 29.4 months (SD = 13.5). Posterior fossa syndrome (PFs) was reported in five patients (21%). Nine (37.5%) received radiotherapy (5 focal, 4 craniospinal). On average, children were assessed 3.5 years (SD = 1.8) post-diagnosis, and full-scale intellectual quotient (FSIQ) scores ranged from 56 to 119 ([Formula: see text]= 92; SD = 16.8). The majority of children (74%) had low-average to average NP functioning. Very young children treated with radiotherapy, who needed hearing support or with PFs had worse neurocognitive outcomes. Clinically significant deficits (<10th percentile) in at least one area of NP functioning were found in 25% of the children. NP data obtained from this sample of survivors of medulloblastoma in early childhood, all treated with sequential HDC and 1/3 with radiotherapy, describe NP functioning within average normal limits overall. However, almost 25% of children had significant deficits in specific domains.
authors.affiliation: Division of Pediatric Hematology Oncology and Bone Marrow Transplantation, Alberta Children's Hospital, 2888 Shaganappi trail NW, Calgary, AB, T3B 6A8, Canada.
authors.forename: Taryn B
authors.initials: TB
authors.lastname: Fay-McClymont
authors.affiliation: Department of Neuropsychology, Kennedy Krieger Institute, Baltimore, Maryland, USA.
authors.forename: Danielle M
authors.initials: DM
authors.lastname: Ploetz
authors.affiliation: Division of Pediatric Hematology Oncology, Hospital for Sick Children, 555 University avenue, Toronto, ON, M5G 1X8, Canada.
authors.forename: Don
authors.initials: D
authors.lastname: Mabbott
authors.affiliation: Department of Neuropsychology, Children's National Medical Center, 111 Michigan Avenue NW, Washington, DC, 20010-2970, USA.
authors.forename: Karin
authors.initials: K
authors.lastname: Walsh
authors.affiliation: Arnold Palmer Hospital, 92 West Miller Street, Orlando, FL, 32806, USA.
authors.forename: Amy
authors.initials: A
authors.lastname: Smith
authors.affiliation: Pediatric Neuro-Oncology, Dana-Farber Cancer Institute, 450 Brookline Ave, Boston, MA, 02215, USA.
authors.forename: Susan N
authors.initials: SN
authors.lastname: Chi
authors.affiliation: Pediatrics, Neurology and Integrative Systems Biology Brain Tumor Institute, Children's National Health System, Washington, DC, USA.
authors.forename: Elizabeth
authors.initials: E
authors.lastname: Wells
authors.affiliation: CPNP, Children's Hospital Colorado, University of Colorado, 13123 E. 16th Avenue, Aurora, CO, 80045, USA.
authors.forename: Jennifer
authors.initials: J
authors.lastname: Madden
authors.affiliation: Children's Hospital Los Angeles, Keck School of Medicine of University of Southern California, 4650 Sunset Boulevard, MS #54, Los Angeles, CA, 90027-6016, USA.
authors.forename: Ashley
authors.initials: A
authors.lastname: Margol
authors.affiliation: Nationwide Children's Hospital and The Ohio State University, 700 Children's Drive, Columbus, OH, 43205, USA.
authors.forename: Jonathan
authors.initials: J
authors.lastname: Finlay
authors.affiliation: Pediatric Neuro-Oncology, Dana-Farber Cancer Institute, 450 Brookline Ave, Boston, MA, 02215, USA.
authors.forename: Mark W
authors.initials: MW
authors.lastname: Kieran
authors.affiliation: Division of Pediatric Hematology Oncology and Bone Marrow Transplantation, Alberta Children's Hospital, 2888 Shaganappi trail NW, Calgary, AB, T3B 6A8, Canada.
authors.forename: Douglas
authors.initials: D
authors.lastname: Strother
authors.affiliation: Children's Hospital Los Angeles, Keck School of Medicine of University of Southern California, 4650 Sunset Boulevard, MS #54, Los Angeles, CA, 90027-6016, USA.
authors.forename: Girish
authors.initials: G
authors.lastname: Dhall
authors.affiliation: Center for Neuroscience and Behavioral Medicine, Brain Tumor Institute Children's National Health System, 111 Michigan Ave, NW, Washington, DC, 20010, USA.
authors.forename: Roger J
authors.initials: RJ
authors.lastname: Packer
authors.affiliation: University of Colorado, 13123 East 16th avenue B115, Aurora, CO, 80045, USA.
authors.forename: Nicholas K
authors.initials: NK
authors.lastname: Foreman
authors.affiliation: Division of Pediatric Hematology Oncology, Hospital for Sick Children, 555 University avenue, Toronto, ON, M5G 1X8, Canada.
authors.forename: E
authors.initials: E
authors.lastname: Bouffet
authors.affiliation: Division of Pediatric Hematology Oncology and Bone Marrow Transplantation, Alberta Children's Hospital, 2888 Shaganappi trail NW, Calgary, AB, T3B 6A8, Canada. lucie.lafay-cousin@ahs.ca.
authors.forename: Lucie
authors.initials: L
authors.lastname: Lafay-Cousin
mesh_terms: D000293:Adolescent; D001932:Brain Neoplasms / Q000523:psychology* / Q000628:therapy*; D059248:Chemoradiotherapy; D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007223:Infant; D008297:Male; D008527:Medulloblastoma / Q000523:psychology* / Q000628:therapy*; D009483:Neuropsychological Tests; D012189:Retrospective Studies
keywords: High-dose chemotherapy; Medulloblastoma; Neurocognitive outcome; Radiation avoidance; Young children