Browsing by Subject "International DIPG Registry"

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    Characteristics of children ≤36 months of age with DIPG: A report from the international DIPG registry
    (Oxford University Press, 2022) Bartlett, Allison L.; Lane, Adam; Chaney, Brooklyn; Yanez Escorza, Nancy; Black, Katie; Cochrane, Anne; Minturn, Jane; Bartels, Ute; Warren, Kathy; Hansford, Jordan; Ziegler, David; Diez, Blanca; Goldman, Stewart; Packer, Roger; Kieran, Mark; DeWire-Schottmiller, Mariko; Erker, Craig; Monje-Deisseroth, Michelle; Wagner, Lars; Koschmann, Carl; Dorris, Kathleen; Shih, Chie-Schin; Hassall, Tim; Fisher, Paul; Wang, Stacie S.; Tsui, Karen; Sevlever, Gustavo; Zhu, Xiaoting; Dexheimer, Phillip; Asher, Anthony; Fuller, Christine; Drissi, Rachid; Jones, Blaise; Leach, James; Fouladi, Maryam; Pediatrics, School of Medicine
    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.
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    Characteristics of patients ≥10 years of age with diffuse intrinsic pontine glioma: a report from the International DIPG/DMG Registry
    (Oxford University Press, 2022) Erker, Craig; Lane, Adam; Chaney, Brooklyn; Leary, Sarah; Minturn, Jane E.; Bartels, Ute; Packer, Roger J.; Dorris, Kathleen; Gottardo, Nicholas G.; Warren, Katherine E.; Broniscer, Alberto; Kieran, Mark W.; Zhu, Xiaoting; White, Peter; Dexheimer, Phillip J.; Black, Katie; Asher, Anthony; DeWire, Mariko; Hansford, Jordan R.; Gururangan, Sridharan; Nazarian, Javad; Ziegler, David S.; Sandler, Eric; Bartlett, Allison; Goldman, Stewart; Shih, Chie-Schin; Hassall, Tim; Dholaria, Hetal; Bandopadhayay, Pratiti; Samson, Yvan; Monje, Michelle; Fisher, Paul G.; Dodgshun, Andrew; Parkin, Sarah; Chintagumpala, Murali; Tsui, Karen; Gass, David; Larouche, Valerie; Broxson, Emmett; Garcia Lombardi, Mercedes; Shiqi Wang, Stacie; Ma, Jie; Hawkins, Cynthia; Hamideh, Dima; Wagner, Lars; Koschmann, Carl; Fuller, Christine; Drissi, Rachid; Jones, Blaise V.; Leach, James; Fouladi, Maryam; Pediatrics, School of Medicine
    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.