TY - JOUR
T1 - Characteristics of patients ≥10 years of age with diffuse intrinsic pontine glioma
T2 - a report from the International DIPG/DMG Registry
AU - Erker, Craig
AU - Lane, Adam
AU - Chaney, Brooklyn
AU - Leary, Sarah
AU - Minturn, Jane E.
AU - Bartels, Ute
AU - Packer, Roger J.
AU - Dorris, Kathleen
AU - Gottardo, Nicholas G.
AU - Warren, Katherine E.
AU - Broniscer, Alberto
AU - Kieran, Mark W.
AU - Zhu, Xiaoting
AU - White, Peter
AU - Dexheimer, Phillip J.
AU - Black, Katie
AU - Asher, Anthony
AU - DeWire, Mariko
AU - Hansford, Jordan R.
AU - Gururangan, Sridharan
AU - Nazarian, Javad
AU - Ziegler, David S.
AU - Sandler, Eric
AU - Bartlett, Allison
AU - Goldman, Stewart
AU - Shih, Chie Schin
AU - Hassall, Tim
AU - Dholaria, Hetal
AU - Bandopadhayay, Pratiti
AU - Samson, Yvan
AU - Monje, Michelle
AU - Fisher, Paul G.
AU - Dodgshun, Andrew
AU - Parkin, Sarah
AU - Chintagumpala, Murali
AU - Tsui, Karen
AU - Gass, David
AU - Larouche, Valerie
AU - Broxson, Emmett
AU - Garcia Lombardi, Mercedes
AU - Wang, Stacie Shiqi
AU - Ma, Jie
AU - Hawkins, Cynthia
AU - Hamideh, Dima
AU - Wagner, Lars
AU - Koschmann, Carl
AU - Fuller, Christine
AU - Drissi, Rachid
AU - Jones, Blaise V.
AU - Leach, James
AU - Fouladi, Maryam
N1 - Publisher Copyright:
© The Author(s) 2021. Published by Oxford University Press on behalf of the Society for Neuro-Oncology. All rights reserved. For permissions, please e-mail: [email protected].
PY - 2022/1/1
Y1 - 2022/1/1
N2 - BACKGROUND: Diffuse intrinsic pontine gliomas (DIPG) generally occur in young school-age children, although can occur in adolescents and young adults. The purpose of this study was to describe clinical, radiological, pathologic, and molecular characteristics in patients ≥10 years of age with DIPG enrolled in the International DIPG Registry (IDIPGR). METHODS: Patients ≥10 years of age at diagnosis enrolled in the IDIPGR with imaging confirmed DIPG diagnosis were included. The primary outcome was overall survival (OS) categorized as long-term survivors (LTS) (≥24 months) or short-term survivors (STS) (<24 months). RESULTS: Among 1010 patients, 208 (21%) were ≥10 years of age at diagnosis; 152 were eligible with a median age of 12 years (range 10-26.8). Median OS was 13 (2-82) months. The 1-, 3-, and 5-year OS was 59.2%, 5.3%, and 3.3%, respectively. The 18/152 (11.8%) LTS were more likely to be older (P < .01) and present with longer symptom duration (P < .01). Biopsy and/or autopsy were performed in 50 (33%) patients; 77%, 61%, 33%, and 6% of patients tested had H3K27M (H3F3A or HIST1H3B), TP53, ATRX, and ACVR1 mutations/genome alterations, respectively. Two of 18 patients with IDH1 testing were IDH1-mutant and 1 was a LTS. The presence or absence of H3 alterations did not affect survival. CONCLUSION: Patients ≥10 years old with DIPG have a median survival of 13 months. LTS present with longer symptom duration and are likely to be older at presentation compared to STS. ATRX mutation rates were higher in this population than the general DIPG population.
AB - BACKGROUND: Diffuse intrinsic pontine gliomas (DIPG) generally occur in young school-age children, although can occur in adolescents and young adults. The purpose of this study was to describe clinical, radiological, pathologic, and molecular characteristics in patients ≥10 years of age with DIPG enrolled in the International DIPG Registry (IDIPGR). METHODS: Patients ≥10 years of age at diagnosis enrolled in the IDIPGR with imaging confirmed DIPG diagnosis were included. The primary outcome was overall survival (OS) categorized as long-term survivors (LTS) (≥24 months) or short-term survivors (STS) (<24 months). RESULTS: Among 1010 patients, 208 (21%) were ≥10 years of age at diagnosis; 152 were eligible with a median age of 12 years (range 10-26.8). Median OS was 13 (2-82) months. The 1-, 3-, and 5-year OS was 59.2%, 5.3%, and 3.3%, respectively. The 18/152 (11.8%) LTS were more likely to be older (P < .01) and present with longer symptom duration (P < .01). Biopsy and/or autopsy were performed in 50 (33%) patients; 77%, 61%, 33%, and 6% of patients tested had H3K27M (H3F3A or HIST1H3B), TP53, ATRX, and ACVR1 mutations/genome alterations, respectively. Two of 18 patients with IDH1 testing were IDH1-mutant and 1 was a LTS. The presence or absence of H3 alterations did not affect survival. CONCLUSION: Patients ≥10 years old with DIPG have a median survival of 13 months. LTS present with longer symptom duration and are likely to be older at presentation compared to STS. ATRX mutation rates were higher in this population than the general DIPG population.
KW - DIPG
KW - International DIPG Registry
KW - outcomes
KW - ≥10 years old
UR - http://www.scopus.com/inward/record.url?scp=85123324330&partnerID=8YFLogxK
U2 - 10.1093/neuonc/noab140
DO - 10.1093/neuonc/noab140
M3 - Article
C2 - 34114629
AN - SCOPUS:85123324330
SN - 1522-8517
VL - 24
SP - 141
EP - 152
JO - Neuro-Oncology
JF - Neuro-Oncology
IS - 1
ER -
