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Journal of Neuro-Oncology

, Volume 145, Issue 2, pp 375–383 | Cite as

Prognostic significance of molecular subgroups of medulloblastoma in young children receiving irradiation-sparing regimens

  • Kee Kiat Yeo
  • Ashley S. MargolEmail author
  • Rebekah J. Kennedy
  • Long Hung
  • Nathan J. Robison
  • Girish Dhall
  • Shahab Asgharzadeh
Clinical Study
  • 125 Downloads

Abstract

Purpose

Irradiation-avoiding strategies have been used with relative success in the treatment of infants and young children with medulloblastoma. While advances in cancer genomics have significantly improved our understanding of the tumor biology of medulloblastoma allowing for improved prognostication and risk-stratification, the molecular subgroup-specific outcomes of infants and young children with medulloblastoma treated with irradiation-avoiding strategies remains unknown.

Methods

Molecular and clinical features of children with medulloblastoma treated with irradiation-avoiding strategies at Children’s Hospital Los Angeles were analyzed. Molecular subgrouping of these patients was determined using a 31-gene TaqMan Low Density Array signature. Survival analyses were conducted based on 3 molecular subgroups (SHH, Group 3, and Group 4).

Results

Twenty-eight patients with medulloblastoma received irradiation-sparing regimens and were included in this analysis. Patients were divided into SHH (n = 16), Group 3 (n = 3) and Group 4 subgroups (n = 9). Subgroup specific 5-year progression-free and overall survival was 81.2% (95% CI 52.5–93.5) and 93.7% (95% CI 63.2–99.1) for SHH, 0% and 0% for Group 3 and 0% and 44.4% (95% CI 13.6–71.9) for Group 4.

Conclusion

The majority of young children with SHH-subgroup medulloblastoma can be treated effectively with irradiation-sparing regimens. Our results support the use of chemotherapy-only strategies for upfront treatment of young children with SHH medulloblastoma, while demonstrating the urgent need for intensification/augmentation of treatment for patients with group 3/4 medulloblastoma.

Keywords

Medulloblastoma Radiation Drug therapy Pediatrics Genomics 

Notes

Funding

This work was supported by a Grant to SA from the American Cancer Society, Alex’s Lemonade Stand Foundation, and Soccer for Hope Foundation.

Compliance with ethical standards

Conflict of interest

The authors report no conflicts of interest.

Supplementary material

11060_2019_3307_MOESM1_ESM.tif (306 kb)
Figure 1 Supplemental(A) Progression-free and overall survival of entire cohort. Overall survival according to (B) disease stage and (C) histology subgroup. (TIF 306 kb)
11060_2019_3307_MOESM2_ESM.tif (10.5 mb)
Figure 2 Supplemental(A&C) Progression-free and (B&D) overall survival of patients with SHH medulloblastoma according to (A&B) extent of resection (GTR vs STR) and (C&D) amount of residual disease (< vs > 1.5 cm2). (TIF 10721 kb)
11060_2019_3307_MOESM3_ESM.tif (127 kb)
Figure 3 SupplementalOverall survival after salvage radiation therapy according to molecular subgroup. (TIF 127 kb)

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Authors and Affiliations

  1. 1.Children’s Center for Cancer and Blood DiseasesChildren’s Hospital Los AngelesLos AngelesUSA
  2. 2.Dana-Farber/Boston Children’s Cancer and Blood Disorders CenterBostonUSA
  3. 3.The Alabama Center for Childhood Cancer and Blood Disorders at Children’s of AlabamaUniversity of Alabama at Birmingham (UAB)BirminghamUSA

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