TY - JOUR
T1 - Clinical, radiologic, pathologic, and molecular characteristics of long-term survivors of Diffuse Intrinsic Pontine Glioma (DIPG)
T2 - A collaborative report from the International and European Society for Pediatric Oncology DIPG registries
AU - Hoffman, Lindsey M.
AU - Van Zanten, Sophie E.M.Veldhuijzen
AU - Colditz, Niclas
AU - Baugh, Joshua
AU - Chaney, Brooklyn
AU - Hoffmann, Marion
AU - Lane, Adam
AU - Fuller, Christine
AU - Miles, Lili
AU - Hawkins, Cynthia
AU - Bartels, Ute
AU - Bouffet, Eric
AU - Goldman, Stewart
AU - Leary, Sarah
AU - Foreman, Nicholas K.
AU - Packer, Roger
AU - Warren, Katherine E.
AU - Broniscer, Alberto
AU - Kieran, Mark W.
AU - Minturn, Jane
AU - Comito, Melanie
AU - Broxson, Emmett
AU - Shih, Chie Schin
AU - Khatua, Soumen
AU - Chintagumpala, Murali
AU - Carret, Anne Sophie
AU - Escorza, Nancy Yanez
AU - Hassall, Timothy
AU - Ziegler, David S.
AU - Gottardo, Nicholas
AU - Dholaria, Hetal
AU - Doughman, Renee
AU - Benesch, Martin
AU - Drissi, Rachid
AU - Nazarian, Javad
AU - Jabado, Nada
AU - Boddaert, Nathalie
AU - Varlet, Pascale
AU - Giraud, Géraldine
AU - Castel, David
AU - Puget, Stephanie
AU - Jones, Chris
AU - Hulleman, Esther
AU - Modena, Piergiorgio
AU - Giagnacovo, Marzia
AU - Antonelli, Manila
AU - Pietsch, Torsten
AU - Gielen, Gerrit H.
AU - Jones, David T.W.
AU - Sturm, Dominik
AU - Pfister, Stefan M.
AU - Gerber, Nicolas U.
AU - Grotzer, Michael A.
AU - Pfaff, Elke
AU - Von Bueren, André O.
AU - Hargrave, Darren
AU - Solanki, Guirish A.
AU - Cvrlje, Filip Jadrijevic
AU - Kaspers, Gertjan J.L.
AU - Vandertop, William P.
AU - Grill, Jacques
AU - Bailey, Simon
AU - Biassoni, Veronica
AU - Massimino, Maura
AU - Calmon, Raphaël
AU - Sanchez, Esther
AU - Bison, Brigitte
AU - Warmuth-Metz, Monika
AU - Leach, James
AU - Jones, Blaise
AU - Van Vuurden, Dannis G.
AU - Kramm, Christof M.
AU - Fouladi, Maryam
N1 - Publisher Copyright:
© 2018 by American Society of Clinical Oncology.
PY - 2018/7/1
Y1 - 2018/7/1
N2 - 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% v23%, 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.
AB - 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% v23%, 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.
UR - https://www.scopus.com/pages/publications/85049646163
U2 - 10.1200/JCO.2017.75.9308
DO - 10.1200/JCO.2017.75.9308
M3 - Article
C2 - 29746225
AN - SCOPUS:85049646163
SN - 0732-183X
VL - 36
SP - 1963
EP - 1972
JO - Journal of Clinical Oncology
JF - Journal of Clinical Oncology
IS - 19
ER -