Characteristics of children ≤36 months of age with DIPG: A report from the international DIPG registry

Allison L. Bartlett; Adam Lane; Brooklyn Chaney; Nancy Yanez Escorza; Katie Black; Anne Cochrane; Jane Minturn; Ute Bartels; Kathy Warren; Jordan Hansford; David Ziegler; Blanca Diez; Stewart Goldman; Roger Packer; Mark Kieran; Mariko DeWire-Schottmiller; Craig Erker; Michelle Monje-Deisseroth; Lars Wagner; Carl Koschmann; Kathleen Dorris; Chie Schin Shih; Tim Hassall; Yvan Samson; Paul Fisher; Stacie S. Wang; Karen Tsui; Gustavo Sevlever; Xiaoting Zhu; Phillip Dexheimer; Anthony Asher; Christine Fuller; Rachid Drissi; Blaise Jones; James Leach; Maryam Fouladi

doi:10.1093/neuonc/noac123

Characteristics of children ≤36 months of age with DIPG: A report from the international DIPG registry

Allison L. Bartlett, Adam Lane, Brooklyn Chaney, Nancy Yanez Escorza, Katie Black, Anne Cochrane, Jane Minturn, Ute Bartels, Kathy Warren, Jordan Hansford, David Ziegler, Blanca Diez, Stewart Goldman, Roger Packer, Mark Kieran, Mariko DeWire-Schottmiller, Craig Erker, Michelle Monje-Deisseroth, Lars Wagner, Carl KoschmannKathleen Dorris, Chie Schin Shih, Tim Hassall, Yvan Samson, Paul Fisher, Stacie S. Wang, Karen Tsui, Gustavo Sevlever, Xiaoting Zhu, Phillip Dexheimer, Anthony Asher, Christine Fuller, Rachid Drissi, Blaise Jones, James Leach, Maryam Fouladi

Research output: Contribution to journal › Article › peer-review

8 Citations (Scopus)

Abstract

Background. Children ≤36 months with diffuse intrinsic pontine glioma (DIPG) have increased long-term survival (LTS, overall survival (OS) ≥24 months). Understanding distinguishing characteristics in this population is critical to improving outcomes. Methods. Patients ≤36 months at diagnosis enrolled on the International DIPG Registry (IDIPGR) with central imaging confirmation were included. Presentation, clinical course, imaging, pathology and molecular findings were analyzed. Results. Among 1183 patients in IDIPGR, 40 were eligible (median age: 29 months). Median OS was 15 months.Twelve patients (30%) were LTS, 3 (7.5%) very long-term survivors ≥5 years. Among 8 untreated patients, median OS was 2 months. Patients enrolled in the registry but excluded from our study by central radiology review or tissue diagnosis had median OS of 7 months. All but 1 LTS received radiation. Among 32 treated patients, 1-, 2-, 3-, and 5-year OS rates were 68.8%, 31.2%, 15.6% and 12.5%, respectively. LTS had longer duration of presenting symptoms (P =.018). No imaging features were predictive of outcome.Tissue and genomic data were available in 18 (45%) and 10 patients, respectively. Among 9 with known H3K27M status, 6 had a mutation. Conclusions. Children ≤36 months demonstrated significantly more LTS, with an improved median OS of 15 months; 92% of LTS received radiation. Median OS in untreated children was 2 months, compared to 17 months for treated children. LTS had longer duration of symptoms. Excluded patients demonstrated a lower OS, contradicting the hypothesis that children ≤36 months with DIPG show improved outcomes due to misdiagnosis.

Original language	English
Pages (from-to)	2190-2199
Number of pages	10
Journal	Neuro-oncology
Volume	24
Issue number	12
DOIs	https://doi.org/10.1093/neuonc/noac123
Publication status	Published or Issued - 1 Dec 2022
Externally published	Yes

Keywords

DIPG
International DIPG Registry
outcomes
survival
young

ASJC Scopus subject areas

Oncology
Clinical Neurology
Cancer Research

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10.1093/neuonc/noac123

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Bartlett, A. L., Lane, A., Chaney, B., Escorza, N. Y., Black, K., Cochrane, A., Minturn, J., Bartels, U., Warren, K., Hansford, J., Ziegler, D., Diez, B., Goldman, S., Packer, R., Kieran, M., DeWire-Schottmiller, M., Erker, C., Monje-Deisseroth, M., Wagner, L., ... Fouladi, M. (2022). Characteristics of children ≤36 months of age with DIPG: A report from the international DIPG registry. Neuro-oncology, 24(12), 2190-2199. https://doi.org/10.1093/neuonc/noac123

@article{79657c6cbc194d75899b15546a8803b2,

title = "Characteristics of children ≤36 months of age with DIPG: A report from the international DIPG registry",

abstract = "Background. Children ≤36 months with diffuse intrinsic pontine glioma (DIPG) have increased long-term survival (LTS, overall survival (OS) ≥24 months). Understanding distinguishing characteristics in this population is critical to improving outcomes. Methods. Patients ≤36 months at diagnosis enrolled on the International DIPG Registry (IDIPGR) with central imaging confirmation were included. Presentation, clinical course, imaging, pathology and molecular findings were analyzed. Results. Among 1183 patients in IDIPGR, 40 were eligible (median age: 29 months). Median OS was 15 months.Twelve patients (30%) were LTS, 3 (7.5%) very long-term survivors ≥5 years. Among 8 untreated patients, median OS was 2 months. Patients enrolled in the registry but excluded from our study by central radiology review or tissue diagnosis had median OS of 7 months. All but 1 LTS received radiation. Among 32 treated patients, 1-, 2-, 3-, and 5-year OS rates were 68.8%, 31.2%, 15.6% and 12.5%, respectively. LTS had longer duration of presenting symptoms (P =.018). No imaging features were predictive of outcome.Tissue and genomic data were available in 18 (45%) and 10 patients, respectively. Among 9 with known H3K27M status, 6 had a mutation. Conclusions. Children ≤36 months demonstrated significantly more LTS, with an improved median OS of 15 months; 92% of LTS received radiation. Median OS in untreated children was 2 months, compared to 17 months for treated children. LTS had longer duration of symptoms. Excluded patients demonstrated a lower OS, contradicting the hypothesis that children ≤36 months with DIPG show improved outcomes due to misdiagnosis.",

keywords = "DIPG, International DIPG Registry, outcomes, survival, young",

author = "Bartlett, {Allison L.} and Adam Lane and Brooklyn Chaney and Escorza, {Nancy Yanez} and Katie Black and Anne Cochrane and Jane Minturn and Ute Bartels and Kathy Warren and Jordan Hansford and David Ziegler and Blanca Diez and Stewart Goldman and Roger Packer and Mark Kieran and Mariko DeWire-Schottmiller and Craig Erker and Michelle Monje-Deisseroth and Lars Wagner and Carl Koschmann and Kathleen Dorris and Shih, {Chie Schin} and Tim Hassall and Yvan Samson and Paul Fisher and Wang, {Stacie S.} and Karen Tsui and Gustavo Sevlever and Xiaoting Zhu and Phillip Dexheimer and Anthony Asher and Christine Fuller and Rachid Drissi and Blaise Jones and James Leach and Maryam Fouladi",

year = "2022",

month = dec,

day = "1",

doi = "10.1093/neuonc/noac123",

language = "English",

volume = "24",

pages = "2190--2199",

journal = "Neuro-oncology",

issn = "1522-8517",

number = "12",

}

Bartlett, AL, Lane, A, Chaney, B, Escorza, NY, Black, K, Cochrane, A, Minturn, J, Bartels, U, Warren, K, Hansford, J, Ziegler, D, Diez, B, Goldman, S, Packer, R, Kieran, M, DeWire-Schottmiller, M, Erker, C, Monje-Deisseroth, M, Wagner, L, Koschmann, C, Dorris, K, Shih, CS, Hassall, T, Samson, Y, Fisher, P, Wang, SS, Tsui, K, Sevlever, G, Zhu, X, Dexheimer, P, Asher, A, Fuller, C, Drissi, R, Jones, B, Leach, J & Fouladi, M 2022, 'Characteristics of children ≤36 months of age with DIPG: A report from the international DIPG registry', Neuro-oncology, vol. 24, no. 12, pp. 2190-2199. https://doi.org/10.1093/neuonc/noac123

TY - JOUR

T1 - Characteristics of children ≤36 months of age with DIPG

T2 - A report from the international DIPG registry

AU - Bartlett, Allison L.

AU - Lane, Adam

AU - Chaney, Brooklyn

AU - Escorza, Nancy Yanez

AU - Black, Katie

AU - Cochrane, Anne

AU - Minturn, Jane

AU - Bartels, Ute

AU - Warren, Kathy

AU - Hansford, Jordan

AU - Ziegler, David

AU - Diez, Blanca

AU - Goldman, Stewart

AU - Packer, Roger

AU - Kieran, Mark

AU - DeWire-Schottmiller, Mariko

AU - Erker, Craig

AU - Monje-Deisseroth, Michelle

AU - Wagner, Lars

AU - Koschmann, Carl

AU - Dorris, Kathleen

AU - Shih, Chie Schin

AU - Hassall, Tim

AU - Samson, Yvan

AU - Fisher, Paul

AU - Wang, Stacie S.

AU - Tsui, Karen

AU - Sevlever, Gustavo

AU - Zhu, Xiaoting

AU - Dexheimer, Phillip

AU - Asher, Anthony

AU - Fuller, Christine

AU - Drissi, Rachid

AU - Jones, Blaise

AU - Leach, James

AU - Fouladi, Maryam

PY - 2022/12/1

Y1 - 2022/12/1

N2 - Background. Children ≤36 months with diffuse intrinsic pontine glioma (DIPG) have increased long-term survival (LTS, overall survival (OS) ≥24 months). Understanding distinguishing characteristics in this population is critical to improving outcomes. Methods. Patients ≤36 months at diagnosis enrolled on the International DIPG Registry (IDIPGR) with central imaging confirmation were included. Presentation, clinical course, imaging, pathology and molecular findings were analyzed. Results. Among 1183 patients in IDIPGR, 40 were eligible (median age: 29 months). Median OS was 15 months.Twelve patients (30%) were LTS, 3 (7.5%) very long-term survivors ≥5 years. Among 8 untreated patients, median OS was 2 months. Patients enrolled in the registry but excluded from our study by central radiology review or tissue diagnosis had median OS of 7 months. All but 1 LTS received radiation. Among 32 treated patients, 1-, 2-, 3-, and 5-year OS rates were 68.8%, 31.2%, 15.6% and 12.5%, respectively. LTS had longer duration of presenting symptoms (P =.018). No imaging features were predictive of outcome.Tissue and genomic data were available in 18 (45%) and 10 patients, respectively. Among 9 with known H3K27M status, 6 had a mutation. Conclusions. Children ≤36 months demonstrated significantly more LTS, with an improved median OS of 15 months; 92% of LTS received radiation. Median OS in untreated children was 2 months, compared to 17 months for treated children. LTS had longer duration of symptoms. Excluded patients demonstrated a lower OS, contradicting the hypothesis that children ≤36 months with DIPG show improved outcomes due to misdiagnosis.

AB - Background. Children ≤36 months with diffuse intrinsic pontine glioma (DIPG) have increased long-term survival (LTS, overall survival (OS) ≥24 months). Understanding distinguishing characteristics in this population is critical to improving outcomes. Methods. Patients ≤36 months at diagnosis enrolled on the International DIPG Registry (IDIPGR) with central imaging confirmation were included. Presentation, clinical course, imaging, pathology and molecular findings were analyzed. Results. Among 1183 patients in IDIPGR, 40 were eligible (median age: 29 months). Median OS was 15 months.Twelve patients (30%) were LTS, 3 (7.5%) very long-term survivors ≥5 years. Among 8 untreated patients, median OS was 2 months. Patients enrolled in the registry but excluded from our study by central radiology review or tissue diagnosis had median OS of 7 months. All but 1 LTS received radiation. Among 32 treated patients, 1-, 2-, 3-, and 5-year OS rates were 68.8%, 31.2%, 15.6% and 12.5%, respectively. LTS had longer duration of presenting symptoms (P =.018). No imaging features were predictive of outcome.Tissue and genomic data were available in 18 (45%) and 10 patients, respectively. Among 9 with known H3K27M status, 6 had a mutation. Conclusions. Children ≤36 months demonstrated significantly more LTS, with an improved median OS of 15 months; 92% of LTS received radiation. Median OS in untreated children was 2 months, compared to 17 months for treated children. LTS had longer duration of symptoms. Excluded patients demonstrated a lower OS, contradicting the hypothesis that children ≤36 months with DIPG show improved outcomes due to misdiagnosis.

KW - DIPG

KW - International DIPG Registry

KW - outcomes

KW - survival

KW - young

UR - http://www.scopus.com/inward/record.url?scp=85146596022&partnerID=8YFLogxK

U2 - 10.1093/neuonc/noac123

DO - 10.1093/neuonc/noac123

M3 - Article

C2 - 35552452

AN - SCOPUS:85146596022

SN - 1522-8517

VL - 24

SP - 2190

EP - 2199

JO - Neuro-oncology

JF - Neuro-oncology

IS - 12

ER -

Characteristics of children ≤36 months of age with DIPG: A report from the international DIPG registry

Abstract

Keywords

ASJC Scopus subject areas

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