Outcomes of Infants and Young Children With Relapsed Medulloblastoma After Initial Craniospinal Irradiation-Sparing Approaches: An International Cohort Study.

Autor:	Erker C; Division of Hematology/Oncology, Department of Paediatrics, IWK Health Centre and Dalhousie University, Halifax, NS, Canada., Mynarek M; Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.; Mildred Scheel Cancer Career Center HaTriCS4, University Medical Center Hamburg-Eppendorf, Hamburg, Germany., Bailey S; Wolfson Childhood Cancer Research Centre, Newcastle University Centre for Cancer, Newcastle-upon-Tyne, United Kingdom., Mazewski CM; Children's Healthcare of Atlanta & Emory University, Atlanta, GA., Baroni L; Hospital of Pediatrics SAMIC Prof. Dr Juan P. Garrahan, Buenos Aires, Argentina., Massimino M; Fondazione Istituto Di Ricovero e Cura a Carattere Scientifico, Istituto Nazionale dei Tumori, Milan, Italy., Hukin J; Divisions of Neurology and Hematology, Oncology/ Bone Marrow Transplant, Department of Pediatrics, British Columbia Children's Hospital and University of British Columbia, Vancouver, BC, Canada., Aguilera D; Children's Healthcare of Atlanta & Emory University, Atlanta, GA., Cappellano AM; Division of Pediatric Oncology/BMT, Instituto de Oncologia Pediátrica-GRAACC-UNIFESP, São Paulo, Brazil., Ramaswamy V; Division of Haematology/Oncology, Department of Paediatrics, The Hospital for Sick Children and University of Toronto, Toronto, ON, Canada., Lassaletta A; Department of Pediatric Hematology and Oncology, Hospital Infantil Universitario Niño Jesús, Madrid, Spain., Perreault S; Centre Hospitalier Universitaire Sainte, Justine, Université de Montreal, Montreal, QC, Canada., Kline CN; Division of Oncology, The Children's Hospital of Philadelphia, Philadelphia, PA., Rajagopal R; Division of Hematology-Oncology, Department of Pediatrics, University Malaya Medical Center, Kuala Lumpur, Malaysia., Michaiel G; Cancer and Blood Disease Institute, Children's Hospital Los Angeles, Keck School of Medicine of University of Southern California, Los Angeles, CA., Zapotocky M; Department of Paediatric Haematology and Oncology, Second Faculty of Medicine, Charles University and University Hospital Motol, Prague, Czech Republic., Santa-Maria Lopez V; Department of Oncology, Hospital Sant Joan de Déu, Barcelona, Spain., La Madrid AM; Department of Oncology, Hospital Sant Joan de Déu, Barcelona, Spain., Cacciotti C; Division of Pediatric Hematology/Oncology, Western University, London, ON, Canada.; Dana Farber/Boston Children's Cancer and Blood Disorder Center, Boston, MA., Sandler ES; Nemours Children's Health, Wolfson's Children's Hospital & University of Florida, Jacksonville, FL., Hoffman LM; Center for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, AZ., Klawinski D; Nemours Children's Health, Wolfson's Children's Hospital & University of Florida, Jacksonville, FL., Khan S; Monash Children's Cancer Centre, Monash Children's Hospital. Monash Health. Center for Cancer Research, Hudson Institute of Medical Research, and Department of Molecular and Translational Science, School of Medicine, Nursing and Health Science, Monash University, Melbourne, Australia.; Division of Hematology, Oncology & Bone Marrow Transplant, Nationwide Children's Hospital, The Ohio State University, Columbus, OH., Salloum R; Division of Haematology/Oncology, Department of Paediatrics, The Hospital for Sick Children and University of Toronto, Toronto, ON, Canada., Hoppmann AL; Department of Pediatrics, Division of Hematology-Oncology, University of Alabama at Birmingham, Birmingham, AL., Larouche V; Department of Pediatrics, Centre Mère-enfant Soleil du CHU de Québec, CRCHU de Québec, Université Laval, Quebec City, QC, Canada., Dorris K; Children's Hospital of Colorado & University of Colorado School of Medicine, Denver, CO., Toledano H; Schneider Children's Medical Center of Israel, Petah Tikva, and Sackler faculty of Medicine, Tel Aviv University, Israel., Gilheeney SW; Department of Pediatrics, Memorial Sloan-Kettering Cancer Center, New York, NY., Abdelbaki MS; Division of Hematology, Oncology and Blood and Marrow Transplant, Nationwide Children's Hospital and The Ohio State University, Columbus, OH.; Division of Pediatric Hematology, Oncology, and Bone Marrow Transplant, Washington University School of Medicine in St Louis, St Louis, MO., Wilson B; Department of Pediatrics, Stollery Children's Hospital, University of Alberta, Edmonton, AB, Canada., Tsang DS; Radiation Medicine Program, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada., Knipstein J; Division of Pediatric Hematology/Oncology/BMT, Medical College of Wisconsin, Milwaukee, WI., Oren MY; Pediatric Hemato-Oncology Department, Sheba Medical Center at Tel HaShomer, Ramat Gan, Israel., Shah S; The University of Texas Health Science Center, Department of Pediatric Hematology-Oncology, San Antonio, TX., Murray JC; Division of Pediatric Hematology/Oncology, Cook Children's Medical Center, Fort Worth, TX., Ginn KF; Division of Pediatric Hematology and Oncology, Children's Mercy Hospital, Kansas City, MO., Wang ZJ; Division of Hematology and Oncology, Children's Hospital of Richmond and Virginia Commonwealth University, Richmond, VA., Fleischhack G; Pediatric Hematology and Oncology, Pediatrics III, University Hospital of Essen, Essen, Germany., Obrecht D; Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany., Tonn S; Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany., Harrod VL; Departments of Pediatric Hematology and Oncology, Dell Children's Medical Center of Central Texas and University of Texas, Austin, TX., Matheson K; Research Methods Unit, Nova Scotia Health Authority, Halifax, NS, Canada., Crooks B; Division of Haematology/Oncology, Department of Paediatrics, IWK Health Centre and Dalhousie University, Halifax, NS, Canada., Strother DR; Section of Pediatric Hematology and Bone Marrow Transplantation, Alberta Children's Hospital, Calgary, AB, Canada., Cohen KJ; Pediatric Oncology, The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, MD., Hansford JR; Children's Cancer Centre, Royal Children's Hospital; Murdoch Children's Research Institute; University of Melbourne, Melbourne, Australia., Mueller S; Department of Pediatrics, University of California San Francisco, San Francisco, CA., Margol A; Cancer and Blood Disease Institute, Children's Hospital Los Angeles, Keck School of Medicine of University of Southern California, Los Angeles, CA., Gajjar A; Department of Oncology, St Jude Children's Research Hospital, Memphis, TN., Dhall G; Division of Hematology, Oncology & Bone Marrow Transplant, Nationwide Children's Hospital, The Ohio State University, Columbus, OH., Finlay JL; Department of Pediatrics, Memorial Sloan-Kettering Cancer Center, New York, NY., Northcott PA; Department of Developmental Neurobiology, St Jude Children's Research Hospital, Memphis, TN., Rutkowski S; Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany., Clifford SC; Wolfson Childhood Cancer Research Centre, Newcastle University Centre for Cancer, Newcastle-upon-Tyne, United Kingdom., Robinson G; Department of Oncology, St Jude Children's Research Hospital, Memphis, TN., Bouffet E; Division of Haematology/Oncology, Department of Paediatrics, The Hospital for Sick Children and University of Toronto, Toronto, ON, Canada., Lafay-Cousin L; Section of Pediatric Hematology and Bone Marrow Transplantation, Alberta Children's Hospital, Calgary, AB, Canada.
Jazyk:	angličtina
Zdroj:	Journal of clinical oncology : official journal of the American Society of Clinical Oncology [J Clin Oncol] 2023 Apr 01; Vol. 41 (10), pp. 1921-1932. Date of Electronic Publication: 2022 Dec 22.
DOI:	10.1200/JCO.21.02968
Abstrakt:	Purpose: Infant and young childhood medulloblastoma (iMB) is usually treated without craniospinal irradiation (CSI) to avoid neurocognitive late effects. Unfortunately, many children relapse. The purpose of this study was to assess salvage strategies and prognostic features of patients with iMB who relapse after CSI-sparing therapy. Methods: We assembled a large international cohort of 380 patients with relapsed iMB, age younger than 6 years, and initially treated without CSI. Univariable and multivariable Cox models of postrelapse survival (PRS) were conducted for those treated with curative intent using propensity score analyses to account for confounding factors. Results: The 3-year PRS, for 294 patients treated with curative intent, was 52.4% (95% CI, 46.4 to 58.3) with a median time to relapse from diagnosis of 11 months. Molecular subgrouping was available for 150 patients treated with curative intent, and 3-year PRS for sonic hedgehog (SHH), group 4, and group 3 were 60%, 84%, and 18% ( P = .0187), respectively. In multivariable analysis, localized relapse ( P = .0073), SHH molecular subgroup ( P = .0103), CSI use after relapse ( P = .0161), and age ≥ 36 months at initial diagnosis ( P = .0494) were associated with improved survival. Most patients (73%) received salvage CSI, and although salvage chemotherapy was not significant in multivariable analysis, its use might be beneficial for a subset of children receiving salvage CSI < 35 Gy ( P = .007). Conclusion: A substantial proportion of patients with relapsed iMB are salvaged after initial CSI-sparing approaches. Patients with SHH subgroup, localized relapse, older age at initial diagnosis, and those receiving salvage CSI show improved PRS. Future prospective studies should investigate optimal CSI doses and the role of salvage chemotherapy in this population. Competing Interests: Craig ErkerConsulting or Advisory Role: Novartis Canada Pharmaceuticals Inc Martin MynarekEmployment: Novartis, BioNTech SE Maura MassiminoConsulting or Advisory Role: Oncoscience, Novartis Juliette HukinStock and Other Ownership Interests: AbbVieConsulting or Advisory Role: AstraZeneca, Novartis Vijay RamaswamyHonoraria: AstraZenecaConsulting or Advisory Role: AstraZeneca Canada Alvaro LassalettaStock and Other Ownership Interests: Gilead SciencesConsulting or Advisory Role: Jazz Pharmaceuticals, Servier, Alexion Pharmaceuticals Sébastien PerreaultLeadership: BayerStock and Other Ownership Interests: NovocureHonoraria: BayerConsulting or Advisory Role: BayerSpeakers' Bureau: BayerExpert Testimony: Bayer Cassie N. KlineResearch Funding: Regeneron (Inst), Curis (Inst), Midatech Pharma (Inst), Ipsen (Inst), Day One Therapeutics (Inst), Bristol Myers Squibb (Inst), Kazia Therapeutics (Inst), Chimerix (Inst) Eric S. SandlerConsulting or Advisory Role: Protara Therapeutics Lindsey M. HoffmanHonoraria: AstraZeneca Kathleen DorrisStock and Other Ownership Interests: Amgen, Gilead SciencesConsulting or Advisory Role: Day One Biopharmaceuticals Helen ToledanoConsulting or Advisory Role: AstraZeneca, Novartis Derek S. TsangTravel, Accommodations, Expenses: Mevion Medical Systems Jeffrey KnipsteinEmployment: PRA Health Sciences, ServierConsulting or Advisory Role: Atheneum Zhihong J. WangHonoraria: AstraZenecaConsulting or Advisory Role: AstraZenecaSpeakers' Bureau: AstraZeneca Kenneth J. CohenConsulting or Advisory Role: Novartis, Bristol Myers Squibb, DNAtrixResearch Funding: Novartis, Bristol Myers Squibb Jordan R. HansfordStock and Other Ownership Interests: AnteotechConsulting or Advisory Role: Bayer Sabine MuellerResearch Funding: Regeneron (Inst), DayOne Pharmaceuticals (Inst), Curis Pharamceuticals (Inst), Curis Pharamceuticals (Inst), Bristol Myers Squibb (Inst) Amar GajjarConsulting or Advisory Role: Roche/Genentech, QED Therapeutics, Day One Therapeutics, Geanno BioResearch Funding: Genentech (Inst), Kazia Therapeutics (Inst) Stefan RutkowskiConsulting or Advisory Role: Bristol Myers Squibb GmbH & Co. KGaA, Germany, Celgene, Roche Pharma AG, Grenzach-Wyhlen, Bayer Germany Giles RobinsonResearch Funding: Novartis (Inst), Genentech/Roche (Inst), Novartis (Inst), SpringWorks Therapeutics (Inst) Eric BouffetConsulting or Advisory Role: NovartisResearch Funding: Roche (Inst) Lucie Lafay-CousinHonoraria: Servier, Innomar StrategiesNo other potential conflicts of interest were reported.
Databáze:	MEDLINE
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