title: Survival after chemotherapy and stem cell transplant followed by delayed craniospinal irradiation is comparable to upfront craniospinal irradiation in pediatric embryonal brain tumor patients.
abstract: Pediatric embryonal brain tumor patients treated with craniospinal irradiation (CSI) are at risk for adverse effects, with greater severity in younger patients. Here we compare outcomes of CSI vs. high-dose chemotherapy (HD), stem cell transplant (SCT) and delayed CSI in newly diagnosed patients. Two hundred one consecutive patients treated for medulloblastoma (72 %), supratentorial primitive neuroectodermal tumor (sPNET; 18 %) or pineoblastoma (10 %) at two institutions between 1988 and 2014 were retrospectively identified. Progression free survival (PFS) and overall survival (OS) were estimated using the Kaplan-Meier method and compared by log-rank tests. Adjuvant CSI regimens were used for 56 % of patients (upfront-CSI), and HD/SCT regimens were used in 32 % of patients. HD/SCT patients were significantly younger than those receiving upfront-CSI (2.9 vs. 7.8 years; P < 0.0001). There were no differences in metastases, extent of resection, or CSI dose between upfront-CSI and HD/SCT patients, but median follow-up was shorter in the HD/SCT group (6.2 vs. 3.9 years; P = 0.007). There were no significant outcome differences between upfront-CSI and HD/SCT patients who received CSI as a prophylaxis or following relapse (OS 66 % vs. 61 %, P = 0.13; PFS 67 % vs. 62 %, P = 0.12). Outcomes were equivalent when restricting analyses to HD/SCT patients who received prophylactic CSI prior to relapse (OS 66 % vs. 65 %, P = 0.5; PFS 67 % vs. 74 %, P = 0.8). At last follow-up, 48 % of HD/SCT patients had received neither definitive nor salvage radiotherapy. In this retrospective cohort, outcomes with adjuvant HD/SCT followed by delayed CSI are comparable to upfront-CSI for carefully surveyed pediatric embryonal brain tumor patients. Future prospective studies are required to validate this finding, and also to assess the impact of delayed CSI on neurocognitive outcomes.
authors: Department of Radiation Oncology, University of California San Francisco, San Francisco, CA, USA.
authors: David R
authors: DR
authors: Raleigh
authors: Department of Economics, California State University Chanel Islands, Camarillo, CA, USA.
authors: Bryan
authors: B
authors: Tomlin
authors: Department of Pediatrics, University of California San Francisco, San Francisco, CA, USA.
authors: Benedict Del
authors: BD
authors: Buono
authors: Department of Pediatrics, University of California San Francisco, San Francisco, CA, USA.
authors: Erika
authors: E
authors: Roddy
authors: Department of Pediatrics, University of California San Francisco, San Francisco, CA, USA.
authors: Katherine
authors: K
authors: Sear
authors: Department of Pediatrics, University of California San Francisco, San Francisco, CA, USA.
authors: Lennox
authors: L
authors: Byer
authors: Department of Pediatrics, University of California San Francisco, San Francisco, CA, USA.
authors: Erin
authors: E
authors: Felton
authors: Department of Pediatrics, University of California San Francisco, San Francisco, CA, USA.
authors: Anu
authors: A
authors: Banerjee
authors: Pediatric Hematology-Oncology, UCSF Benioff Children's Hospital Oakland, Oakland, CA, USA.
authors: Joseph
authors: J
authors: Torkildson
authors: Pediatric Hematology-Oncology, Valley Children's Healthcare, Madera, CA, USA.
authors: David
authors: D
authors: Samuel
authors: Department of Pediatrics, University of California San Francisco, San Francisco, CA, USA.
authors: Biljana
authors: B
authors: Horn
authors: Department of Radiation Oncology, University of California San Francisco, San Francisco, CA, USA.
authors: Steve E
authors: SE
authors: Braunstein
authors: Department of Radiation Oncology, Dana-Farber Cancer Institute, Boston, MA, USA.
authors: Daphne A
authors: DA
authors: Haas-Kogan
authors: Department of Pediatrics, University of California San Francisco, San Francisco, CA, USA. Sabine.Mueller@ucsf.edu.|Department of Neurology, Neurosurgery and Pediatrics, University of California San Francisco, 550 Sandler Neuroscience, 625 Nelson Rising Lane, 402B, Box 0434, San Francisco, CA, 94143, USA. Sabine.Mueller@ucsf.edu.
authors: Sabine
authors: S
authors: Mueller
mesh_terms: D000293:Adolescent; D000328:Adult; D001932:Brain Neoplasms / Q000532:radiotherapy / Q000628:therapy*; D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D003131:Combined Modality Therapy; D061888:Craniospinal Irradiation; D005260:Female; D006801:Humans; D007223:Infant; D053208:Kaplan-Meier Estimate; D008297:Male; D018714:Radiotherapy, Adjuvant; D012189:Retrospective Studies; D033581:Stem Cell Transplantation; D016896:Treatment Outcome; D055815:Young Adult
keywords: Chemotherapy; Medulloblastoma; Pineoblastoma; Radiation; Stem cell transplant; Supratentorial primitive neuroectodermal tumor