Clinical, Radiologic, Pathologic, and Molecular Characteristics of Long-Term Survivors of Diffuse Intrinsic Pontine Glioma (DIPG): A Collaborative Report From the International and European Society for Pediatric Oncology DIPG Registries.
Date
2018-05-10ICR Author
Author
Hoffman, LM
Veldhuijzen van Zanten, SEM
Colditz, N
Baugh, J
Chaney, B
Hoffmann, M
Lane, A
Fuller, C
Miles, L
Hawkins, C
Bartels, U
Bouffet, E
Goldman, S
Leary, S
Foreman, NK
Packer, R
Warren, KE
Broniscer, A
Kieran, MW
Minturn, J
Comito, M
Broxson, E
Shih, C-S
Khatua, S
Chintagumpala, M
Carret, AS
Escorza, NY
Hassall, T
Ziegler, DS
Gottardo, N
Dholaria, H
Doughman, R
Benesch, M
Drissi, R
Nazarian, J
Jabado, N
Boddaert, N
Varlet, P
Giraud, G
Castel, D
Puget, S
Jones, C
Hulleman, E
Modena, P
Giagnacovo, M
Antonelli, M
Pietsch, T
Gielen, GH
Jones, DTW
Sturm, D
Pfister, SM
Gerber, NU
Grotzer, MA
Pfaff, E
von Bueren, AO
Hargrave, D
Solanki, GA
Jadrijevic Cvrlje, F
Kaspers, GJL
Vandertop, WP
Grill, J
Bailey, S
Biassoni, V
Massimino, M
Calmon, R
Sanchez, E
Bison, B
Warmuth-Metz, M
Leach, J
Jones, B
van Vuurden, DG
Kramm, CM
Fouladi, M
Type
Journal Article
Metadata
Show full item recordAbstract
Purpose Diffuse intrinsic pontine glioma (DIPG) is a brainstem malignancy with a median survival of < 1 year. The International and European Society for Pediatric Oncology DIPG Registries collaborated to compare clinical, radiologic, and histomolecular characteristics between short-term survivors (STSs) and long-term survivors (LTSs). Materials and Methods Data abstracted from registry databases included patients from North America, Australia, Germany, Austria, Switzerland, the Netherlands, Italy, France, the United Kingdom, and Croatia. Results Among 1,130 pediatric and young adults with radiographically confirmed DIPG, 122 (11%) were excluded. Of the 1,008 remaining patients, 101 (10%) were LTSs (survival ≥ 2 years). Median survival time was 11 months (interquartile range, 7.5 to 16 months), and 1-, 2-, 3-, 4-, and 5-year survival rates were 42.3% (95% CI, 38.1% to 44.1%), 9.6% (95% CI, 7.8% to 11.3%), 4.3% (95% CI, 3.2% to 5.8%), 3.2% (95% CI, 2.4% to 4.6%), and 2.2% (95% CI, 1.4% to 3.4%), respectively. LTSs, compared with STSs, more commonly presented at age < 3 or > 10 years (11% v 3% and 33% v 23%, respectively; P < .001) and with longer symptom duration ( P < .001). STSs, compared with LTSs, more commonly presented with cranial nerve palsy (83% v 73%, respectively; P = .008), ring enhancement (38% v 23%, respectively; P = .007), necrosis (42% v 26%, respectively; P = .009), and extrapontine extension (92% v 86%, respectively; P = .04). LTSs more commonly received systemic therapy at diagnosis (88% v 75% for STSs; P = .005). Biopsies and autopsies were performed in 299 patients (30%) and 77 patients (10%), respectively; 181 tumors (48%) were molecularly characterized. LTSs were more likely to harbor a HIST1H3B mutation (odds ratio, 1.28; 95% CI, 1.1 to 1.5; P = .002). Conclusion We report clinical, radiologic, and molecular factors that correlate with survival in children and young adults with DIPG, which are important for risk stratification in future clinical trials.
Collections
Subject
Humans
Glioma
Brain Stem Neoplasms
Registries
Adolescent
Adult
Child
Child, Preschool
Infant
Infant, Newborn
Young Adult
Kaplan-Meier Estimate
Cancer Survivors
Research team
Glioma Team
Language
eng
Date accepted
2018-03-21
License start date
2018-07
Citation
Journal of clinical oncology : official journal of the American Society of Clinical Oncology, 2018, 36 (19), pp. 1963 - 1972
Publisher
LIPPINCOTT WILLIAMS & WILKINS