Subgroup and subtype-specific outcomes in adult medulloblastoma


Por: Coltin H, Sundaresan L, Smith KS, Skowron P, Massimi L, Eberhart CG, Schreck KC, Gupta N, Weiss WA, Tirapelli D, Carlotti C, Li KKW, Ryzhova M, Golanov A, Zheludkova O, Absalyamova O, Okonechnikov K, Stichel D, von Deimling A, Giannini C, Raskin S, Van Meir EG, Chan JA, Fults D, Chambless LB, Kim SK, Vasiljevic A, Faure-Conter C, Vibhakar R, Jung S, Leary S, Mora J, McLendon RE, Pollack IF, Hauser P, Grajkowska WA, Rubin JB, van Veelen MC, French PJ, Kros JM, Liau LM, Pfister SM, Kool M, Kijima N, Taylor MD, Packer RJ, Northcott PA, Korshunov A and Ramaswamy V

Publicada: 1 nov 2021 Ahead of Print: 1 ago 2021
Resumen:
Medulloblastoma, a common pediatric malignant central nervous system tumour, represent a small proportion of brain tumours in adults. Previously it has been shown that in adults, Sonic Hedgehog (SHH)-activated tumours predominate, with Wingless-type (WNT) and Group 4 being less common, but molecular risk stratification remains a challenge. We performed an integrated analysis consisting of genome-wide methylation profiling, copy number profiling, somatic nucleotide variants and correlation of clinical variables across a cohort of 191 adult medulloblastoma cases identified through the Medulloblastoma Advanced Genomics International Consortium. We identified 30 WNT, 112 SHH, 6 Group 3, and 41 Group 4 tumours. Patients with SHH tumours were significantly older at diagnosis compared to other subgroups (p < 0.0001). Five-year progression-free survival (PFS) for WNT, SHH, Group 3, and Group 4 tumours was 64.4 (48.0-86.5), 61.9% (51.6-74.2), 80.0% (95% CI 51.6-100.0), and 44.9% (95% CI 28.6-70.7), respectively (p = 0.06). None of the clinical variables (age, sex, metastatic status, extent of resection, chemotherapy, radiotherapy) were associated with subgroup-specific PFS. Survival among patients with SHH tumours was significantly worse for cases with chromosome 3p loss (HR 2.9, 95% CI 1.1-7.6; p = 0.02), chromosome 10q loss (HR 4.6, 95% CI 2.3-9.4; p < 0.0001), chromosome 17p loss (HR 2.3, 95% CI 1.1-4.8; p = 0.02), and PTCH1 mutations (HR 2.6, 95% CI 1.1-6.2; p = 0.04). The prognostic significance of 3p loss and 10q loss persisted in multivariable regression models. For Group 4 tumours, chromosome 8 loss was strongly associated with improved survival, which was validated in a non-overlapping cohort (combined cohort HR 0.2, 95% CI 0.1-0.7; p = 0.007). Unlike in pediatric medulloblastoma, whole chromosome 11 loss in Group 4 and chromosome 14q loss in SHH was not associated with improved survival, where MYCN, GLI2 and MYC amplification were rare. In sum, we report unique subgroup-specific cytogenetic features of adult medulloblastoma, which are distinct from those in younger patients, and correlate with survival disparities. Our findings suggest that clinical trials that incorporate new strategies tailored to high-risk adult medulloblastoma patients are urgently needed.

Filiaciones:
Coltin H:
 Division of Haematology/Oncology, Hospital for Sick Children, 555 University Avenue, Toronto, ON, Canada

 Programme in Developmental and Stem Cell Biology, Arthur and Sonia Labatt Brain Tumour Research Centre, Hospital for Sick Children, Toronto, ON, Canada

Sundaresan L:
 Programme in Developmental and Stem Cell Biology, Arthur and Sonia Labatt Brain Tumour Research Centre, Hospital for Sick Children, Toronto, ON, Canada

Smith KS:
 Department of Developmental Neurobiology, St. Jude Children's Research Hospital, MS 325, Room D2058, 262 Danny Thomas Place, Memphis, TN, 38105-3678, USA

Skowron P:
 Programme in Developmental and Stem Cell Biology, Arthur and Sonia Labatt Brain Tumour Research Centre, Hospital for Sick Children, Toronto, ON, Canada

Massimi L:
 Department of Neurosurgery, Fondazione Policlinico A. Gemelli IRCCS, Catholic University Medical School, Rome, Italy

Eberhart CG:
 Department of Neuropathology and Ophthalmic Pathology, Johns Hopkins University, Baltimore, MD, USA

Schreck KC:
 Department of Neurology, Johns Hopkins University, Baltimore, MD, USA

Gupta N:
 Departments of Neurological Surgery and Pediatrics, University of California, San Francisco, CA, USA

Weiss WA:
 Departments of Neurology, Neurological Surgery, and Pediatrics, University of California, San Francisco, CA, USA

Tirapelli D:
 Department of Surgery and Anatomy, Faculty of Medicine of Ribeirão Preto, University of Sao Paulo, São Paulo, Brazil

Carlotti C:
 Department of Surgery and Anatomy, Faculty of Medicine of Ribeirão Preto, University of Sao Paulo, São Paulo, Brazil

Li KKW:
 Department of Anatomical and Cellular Pathology, The Chinese University of Hong Kong, Shatin, New Territories, Hong Kong

Ryzhova M:
 NN Burdenko Neurosurgical Research Centre, Moscow, Russia

Golanov A:
 NN Burdenko Neurosurgical Research Centre, Moscow, Russia

Zheludkova O:
 NN Burdenko Neurosurgical Research Centre, Moscow, Russia

Absalyamova O:
 NN Burdenko Neurosurgical Research Centre, Moscow, Russia

Okonechnikov K:
 Hopp Children's Cancer Center Heidelberg (KiTZ) and Division of Pediatric Neurooncology, German Cancer Research Center (DKFZ), Heidelberg, Germany

Stichel D:
 Clinical Cooperation Unit Neuropathology (B300), German Cancer Research Center (DKFZ) and Department of Neuropathology, University of Heidelberg, University Hospital Heidelberg, Im Neuenheimer Feld 224, 69120, Heidelberg, Germany

von Deimling A:
 Clinical Cooperation Unit Neuropathology (B300), German Cancer Research Center (DKFZ) and Department of Neuropathology, University of Heidelberg, University Hospital Heidelberg, Im Neuenheimer Feld 224, 69120, Heidelberg, Germany

Giannini C:
 Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester, MN, USA

Raskin S:
 Center for Cancer and Blood Disorders, Children's National Medical Center, Washington, DC, USA

Van Meir EG:
 Department of Neurosurgery, O'Neal Comprehensive Cancer Center, University of Alabama at Birmingham (UAB), Birmingham, AL, USA

Chan JA:
 Department of Pathology and Laboratory Medicine, University of Calgary, Calgary, AB, Canada

Fults D:
 Department of Neurosurgery, University of Utah, Salt Lake City, UT, USA

Chambless LB:
 Department of Neurological Surgery, Vanderbilt Medical Center, Nashville, TN, USA

Kim SK:
 Department of Neurosurgery, Division of Pediatric Neurosurgery, Seoul National University Children's Hospital, Seoul, South Korea

Vasiljevic A:
 Centre de Pathologie et Neuropathologie Est, Centre de Biologie et Pathologie Est, Groupement Hospitalier Est, Hospices Civils de Lyon, Bron, France

 ONCOFLAM, Neuro-Oncologie Et Neuro-Inflammation Centre de Recherche en Neurosciences de Lyon, Lyon, France

Faure-Conter C:
 Department of Pediatrics, Institut d'Hemato-Oncologie Pediatrique, Lyon, France

Vibhakar R:
 Department of Pediatrics, University of Colorado Denver, Aurora, CO, USA

Jung S:
 Department of Neurosurgery, Chonnam National University Research Institute of Medical Sciences, Chonnam National University Hwasun Hospital and Medical School, Hwasun-gun, Chonnam, South Korea

Leary S:
 Cancer and Blood Disorders Center, Seattle Children's Hospital, Seattle, WA, USA

Mora J:
 Developmental Tumor Biology Laboratory, Hospital Sant Joan de Déu, Esplugues de Llobregat, Barcelona, Spain

McLendon RE:
 Department of Pathology, Duke University, Durham, NC, USA

Pollack IF:
 Department of Neurological Surgery, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA

Hauser P:
 2nd Department of Pediatrics, Semmelweis University, Budapest, Hungary

Grajkowska WA:
 Department of Pathology, The Children's Memorial Health Institute, Warsaw, Poland

Rubin JB:
 Departments of Pediatrics, Anatomy and Neurobiology, Washington University School of Medicine and St Louis Children's Hospital, St Louis, MO, USA

van Veelen MC:
 Department of Neurosurgery, Brain Tumour Center, Erasmus MC Cancer Institute, Rotterdam, The Netherlands

French PJ:
 Department of Neurology, Brain Tumour Center, Erasmus MC Cancer Institute, Rotterdam, The Netherlands

Kros JM:
 Department of Pathology, Erasmus University Medical Center, Rotterdam, Netherlands

Liau LM:
 Department of Neurosurgery, David Geffen School of Medicine at University of California at Los Angeles, University of California Los Angeles, Los Angeles, CA, 90095, USA

Pfister SM:
 Hopp Children's Cancer Center Heidelberg (KiTZ) and Division of Pediatric Neurooncology, German Cancer Research Center (DKFZ), Heidelberg, Germany

 Department of Pediatric Oncology, Hematology and Immunology, University of Heidelberg, Heidelberg, Germany

Kool M:
 Hopp Children's Cancer Center Heidelberg (KiTZ) and Division of Pediatric Neurooncology, German Cancer Research Center (DKFZ), Heidelberg, Germany

 Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands

Kijima N:
 Department of Neurosurgery, Osaka University Graduate School of Medicine, Suita, Japan

Taylor MD:
 Programme in Developmental and Stem Cell Biology, Arthur and Sonia Labatt Brain Tumour Research Centre, Hospital for Sick Children, Toronto, ON, Canada

Packer RJ:
 Center for Cancer and Blood Disorders, Children's National Medical Center, Washington, DC, USA

Northcott PA:
 Department of Developmental Neurobiology, St. Jude Children's Research Hospital, MS 325, Room D2058, 262 Danny Thomas Place, Memphis, TN, 38105-3678, USA.

Korshunov A:
 Clinical Cooperation Unit Neuropathology (B300), German Cancer Research Center (DKFZ) and Department of Neuropathology, University of Heidelberg, University Hospital Heidelberg, Im Neuenheimer Feld 224, 69120, Heidelberg, Germany.

Ramaswamy V:
 Division of Haematology/Oncology, Hospital for Sick Children, 555 University Avenue, Toronto, ON, Canada.

 Programme in Developmental and Stem Cell Biology, Arthur and Sonia Labatt Brain Tumour Research Centre, Hospital for Sick Children, Toronto, ON, Canada.

 Department of Medical Biophysics, University of Toronto, Toronto, ON, Canada.
ISSN: 00016322





ACTA NEUROPATHOLOGICA
Editorial
SPRINGER, ONE NEW YORK PLAZA, SUITE 4600 , NEW YORK, NY 10004, UNITED STATES, Alemania
Tipo de documento: Article
Volumen: 142 Número: 5
Páginas: 859-871
WOS Id: 000686088700002
ID de PubMed: 34409497
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