Retrospective observational studies in ultra-rare sarcomas: A consensus paper from the Connective Tissue Oncology Society (CTOS) community of experts on the minimum requirements for the evaluation of activity of systemic treatments.

Autor: Stacchiotti S; Department of Medical Oncology, IRCCS Fondazione Istituto Nazionale Tumori (INT), 20133 Milan, Italy. Electronic address: silvia.stacchiotti@istitutotumori.mi.it., Maria Frezza A; Department of Medical Oncology, IRCCS Fondazione Istituto Nazionale Tumori (INT), 20133 Milan, Italy., Demetri GD; Department of Medical Oncology, Sarcoma Center, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA 02215, USA., Blay JY; Department of Medicine, Centre Léon Bérard, Université Claude Bernard Lyon I, Unicancer, 69008 Lyon, France., Bajpai J; Medical Oncology Department, Tata Memorial Centre, Homi Bhabha National Institute, 400012 Mumbai, India., Baldi GG; Department of Medical Oncology, Ospedale Santo Stefano, 59100, Prato, Italy., Baldini EH; Department of Radiation Oncology, Dana-Farber Cancer Institute/ Brigham and Women's Hospital, Boston 02215, MA, USA., Benjamin RS; Department of Sarcoma Medical Oncology, The University of Texas M.D. Anderson Cancer Center, Houston 77030, TX, USA., Bonvalot S; Department of Surgical Oncology, Institut Curie, Université Paris Sciences et Lettres, 75005, France., Bovée JVMG; Departmen of Pathology, Leiden University Medical Center, 2333 ZA, Leiden, The Netherlands., Callegaro D; Department of Surgery, INT, 20133 Milan, Italy., Casali PG; Department of Medical Oncology, IRCCS Fondazione Istituto Nazionale Tumori (INT), 20133 Milan, Italy., D'Angelo SP; Department of Medicine, Memorial Sloan Kettering Cancer Center and Weill Cornell Medical College, 10065, New York, NY, USA., Davis EJ; Division of Hematology-Oncology, Vanderbilt University Medical Center, Nashville, TN 37232, USA., Dei Tos AP; Department of Pathology, Azienda Ospedaliera Università Padova, 35129, Padova, Italy., Demicco EG; Department of Laboratory Medicine and Pathobiology, University of Toronto & Pathology and Laboratory Medicine Mount Sinai Hospital, ON M5G 1X5, Toronto, Canada., Desai J; Department of Medical Oncology, Peter MacCallum Cancer Centre, Melbourne VIC 3000, Australia., Dileo P; Soft tissue and bone sarcoma service, University College Hospital, UCLH NHS Trust, NW1 2BU, London, United Kingdom., Eriksson M; Department of Oncology, Skåne University Hospital, and Lund University, 222 42, Lund, Sweden., Gelderblom H; Department of Medical Oncology, Leiden University Medical Center, 2333 ZA, Leiden, The Netherlands., George S; Department of Medical Oncology, Sarcoma Center, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA 02215, USA., Gladdy RA; Mount Sinai Hospital, Princess Margaret Hospital, University of Toronto, ON M5G 1X5, Toronto, ON, Canada., Gounder MM; Department of Medicine, Memorial Sloan Kettering Cancer Center and Weill Cornell Medical College, 10065, New York, NY, USA., Gupta AA; The Hospital for Sick Children and Princess Margaret Cancer Center, University of Toronto, ON M5G 2C1, Toronto, Canada., Haas R; Department of Radiotherapy, the Netherlands Cancer Institute, 1066 CX, Amsterdam and the Leiden University Medical Center, 2333 ZA, Leiden, The Netherlands., Hayes A; Department of Surgery, the Royal Marsden NHS Foundation Trust, SW3 6JJ, London, United Kingdom., Hohenberger P; Division of Surgical Oncology and Thoracic Surgery, Mannheim University Medical Center, Medical Faculty Mannheim, University of Heidelberg, 69117 Heidelberg, Germany., Jones KB; Departments of Orthopaedics and Oncological Sciences, Huntsman Cancer Institute, University of Utah, UT 84112, Salt Lake City, USA., Jones RL; Sarcoma Unit, the Royal Marsden NHS Foundation Trust and Institute of Cancer Research, SW3 6JJ, London, United Kingdom., Kasper B; Sarcoma Unit, Mannheim Cancer Center (MCC), Mannheim University Medical Center, University of Heidelberg, 68167 Mannheim, Germany., Kawai A; Department of Musculoskeletal Oncology, National Cancer Center Hospital, Tokyo, Japan., Kirsch DG; Department of Radiation Oncology, Duke University Medical Center, NC 27710 Durham, USA., Kleinerman ES; Division of Pediatrics, University of Texas M.D. Anderson Cancer Center, 77030 Huston, TX, USA., Le Cesne A; Medical Oncology, Insitut Gustave Roussy, 94805 Villejuif, Ile-de-France, France., Maestro R; Unit of Oncogenetics and Functional Oncogenomics, 33081 Aviano, Italy., Martin Broto J; Medical Oncology Department, University Hospital Fundacion Jimenez Diaz, University Hospital General de Villalba and Instituto de Investigacion Sanitaria FJD, 28040 Madrid, Spain., Maki RG; Abramson Cancer Center, University of Pennsylvania, 19104 Philadelphia, PA, USA., Miah AB; Department of Radiation Therapy, the Royal Marsden NHS Foundation Trust, SW3 6JJ, London, United Kingdom., Palmerini E; Osteoncology, Soft Tissue and Bone Sarcoma and Innovative Therapy Unit, IRCCS Istituto Ortopedico Rizzoli, 40136 Bologna, Italy., Patel SR; Department of Sarcoma Medical Oncology, The University of Texas M.D. Anderson Cancer Center, Houston 77030, TX, USA., Raut CP; Department of Surgery, Brigham and Women's Hospital, Center for Sarcoma and Bone Oncology, DFCC, Harvard Medical School, Boston 02215, MA, USA., Razak ARA; Princess Margaret Cancer Center, ON M5G 2C1, Toronto, Canada., Reed DR; Department of Individualized Cancer Management, Moffitt Cancer Center, FL 33612, Tampa, FL, USA., Rutkowski P; Department of Soft Tissue/Bone Sarcoma and Melanoma, Maria Sklodowska-Curie National Research Institute of Oncology, 00-001, Warsaw, Poland., Sanfilippo RG; Department of Medical Oncology, IRCCS Fondazione Istituto Nazionale Tumori (INT), 20133 Milan, Italy., Sbaraglia M; Department of Pathology, Azienda Ospedaliera Università Padova, 35129, Padova, Italy., Schaefer IM; Department of Pathology, Brigham and Women's Hospital, Harvard Medical School, MA 02215, Boston, MA, USA., Strauss DC; Department of Surgery, The Royal Marsden Hospital and The Institute of Cancer Research, SW3 6JJ, London, the United Kingdom of Great Britain and Northern Ireland., Strauss SJ; Soft tissue and bone sarcoma service, University College Hospital, UCLH NHS Trust, NW1 2BU, London, United Kingdom., Tap WD; Department of Medicine, Memorial Sloan Kettering Cancer Center and Weill Cornell Medical College, 10065, New York, NY, USA., Thomas DM; Garvan Institute of Medical Research, NSW 2010, Sydney, Australia., Trama A; Department of Research, Evaluative Epidemiology Unit, INT, 20133 Milan, Italy., Trent JC; Sylvester Comprehensive Cancer Center, University of Miami, 33136 Miami, FL, USA., van der Graaf WTA; Department of Medical Oncology, Netherlands Cancer Institute, 1066 CX, Amsterdam, The Netherlands., van Houdt WJ; Department of Surgical Oncology, the Netherlands Cancer Institute, 1066 CX, Amsterdam, The Netherlands., von Mehren M; Department of Hematology and Oncology, Fox Chase Cancer Center, 19111 Philadelphia, PA, USA., Wilky BA; Department of Medical Oncology, University of Colorado Cancer Center, 80045 Aurora, CO, USA., Fletcher CDM; Department of Pathology, Brigham and Women's Hospital, Harvard Medical School, MA 02215, Boston, MA, USA., Gronchi A; Department of Surgery, INT, 20133 Milan, Italy., Miceli R; Unit of Clinical Epidemiology and Trial Organization, Department of Applied Research and Technological Development, Fondazione IRCCS Istituto Nazionale Tumori, 20133 Milan, Italy., Wagner AJ; Department of Medical Oncology, Sarcoma Center, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA 02215, USA.
Jazyk: angličtina
Zdroj: Cancer treatment reviews [Cancer Treat Rev] 2022 Nov; Vol. 110, pp. 102455. Date of Electronic Publication: 2022 Aug 18.
DOI: 10.1016/j.ctrv.2022.102455
Abstrakt: Background: In ultra-rare sarcomas (URS) the conduction of prospective, randomized trials is challenging. Data from retrospective observational studies (ROS) may represent the best evidence available. ROS implicit limitations led to poor acceptance by the scientific community and regulatory authorities. In this context, an expert panel from the Connective Tissue Oncology Society (CTOS), agreed on the need to establish a set of minimum requirements for conducting high-quality ROS on the activity of systemic therapies in URS.
Methods: Representatives from > 25 worldwide sarcoma reference centres met in November 2020 and identified a list of topics summarizing the main issues encountered in ROS on URS. An online survey on these topics was distributed to the panel; results were summarized by descriptive statistics and discussed during a second meeting (November 2021).
Results: Topics identified by the panel included the use of ROS results as external control data, the criteria for contributing centers selection, modalities for ensuring a correct pathological diagnosis and radiologic assessment, consistency of surveillance policies across centers, study end-points, risk of data duplication, results publication. Based on the answers to the survey (55 of 62 invited experts) and discussion the panel agreed on 18 statements summarizing principles of recommended practice.
Conclusions: These recommendations will be disseminated by CTOS across the sarcoma community and incorporated in future ROS on URS, to maximize their quality and favor their use as control data when results from prospective studies are unavailable. These recommendations could help the optimal conduction of ROS also in other rare tumors.
Competing Interests: Declaration of Competing Interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.
(Copyright © 2022 Elsevier Ltd. All rights reserved.)
Databáze: MEDLINE
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