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Stacchiotti S , Frezza AM , Blay JY , Baldini EH , Bonvalot S , Bovée Jvmg , Callegaro D , Casali PG , Chiang RC , Demetri GD , Demicco EG , Desai J , Eriksson M , Gelderblom H , George S , Gounder MM , Gronchi A , Gupta A , Haas RL , Hayes-Jardon A , Hohenberger P , Jones KB , Jones RL , Kasper B , Kawai A , Kirsch DG , Kleinerman ES , Le Cesne A , Lim J , Chirlaque López MD , Maestro R , Marcos-Gragera R , Martin Broto J , Matsuda T , Mir O , Patel SR , Raut CP , Razak ARA , Reed DR , Rutkowski P , Sanfilippo RG , Sbaraglia M , Schaefer IM , Strauss DC , Sundby Hall K , Tap WD , Thomas DM , van der Graaf WTA , van Houdt WJ , Visser O , von Mehren M , Wagner AJ , Wilky BA , Won YJ , Fletcher CDM , Dei Tos AP , Trama A
Ultra-rare sarcomas: A consensus paper from the Connective Tissue Oncology Society community of experts on the incidence threshold and the list of entities
Cancer. 2021 Aug 15;127(16) :2934-2942
PMID: 33910263    PMCID: PMC8319065    URL: https://www.ncbi.nlm.nih.gov/pubmed/33910263
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BACKGROUND: Among sarcomas, which are rare cancers, many types are exceedingly rare; however, a definition of ultra-rare cancers has not been established. The problem of ultra-rare sarcomas is particularly relevant because they represent unique diseases, and their rarity poses major challenges for diagnosis, understanding disease biology, generating clinical evidence to support new drug development, and achieving formal authorization for novel therapies. METHODS: The Connective Tissue Oncology Society promoted a consensus effort in November 2019 to establish how to define ultra-rare sarcomas through expert consensus and epidemiologic data and to work out a comprehensive list of these diseases. The list of ultra-rare sarcomas was based on the 2020 World Health Organization classification, The incidence rates were estimated using the Information Network on Rare Cancers (RARECARENet) database and NETSARC (the French Sarcoma Network's clinical-pathologic registry). Incidence rates were further validated in collaboration with the Asian cancer registries of Japan, Korea, and Taiwan. RESULTS: It was agreed that the best criterion for a definition of ultra-rare sarcomas would be incidence. Ultra-rare sarcomas were defined as those with an incidence of approximately ≤1 per 1,000,000, to include those entities whose rarity renders them extremely difficult to conduct well powered, prospective clinical studies. On the basis of this threshold, a list of ultra-rare sarcomas was defined, which comprised 56 soft tissue sarcoma types and 21 bone sarcoma types. CONCLUSIONS: Altogether, the incidence of ultra-rare sarcomas accounts for roughly 20% of all soft tissue and bone sarcomas. This confirms that the challenges inherent in ultra-rare sarcomas affect large numbers of patients.
1097-0142 Stacchiotti, Silvia Orcid: 0000-0002-1742-8666 Frezza, Anna Maria Orcid: 0000-0003-2335-7224 Blay, Jean-Yves Orcid: 0000-0001-7190-120x Baldini, Elizabeth H Bonvalot, Sylvie Orcid: 0000-0001-9726-9732 Bovée, Judith V M G Callegaro, Dario Orcid: 0000-0002-3392-4002 Casali, Paolo G Chiang, RuRu Chun-Ju Demetri, George D Demicco, Elisabeth G Desai, Jayesh Eriksson, Mikael Gelderblom, Hans George, Suzanne Gounder, Mrinal M Gronchi, Alessandro Gupta, Abha Orcid: 0000-0002-0825-5070 Haas, Rick L Orcid: 0000-0003-4834-4331 Hayes-Jardon, Andrea Hohenberger, Peter Jones, Kevin B Jones, Robin L Kasper, Bernd Orcid: 0000-0001-8425-8356 Kawai, Akira Orcid: 0000-0003-2116-586x Kirsch, David G Orcid: 0000-0002-2086-205x Kleinerman, Eugene S Le Cesne, Axel Lim, Jiwon Chirlaque López, María Dolores Maestro, Roberta Marcos-Gragera, Rafael Martin Broto, Javier Matsuda, Tomohiro Mir, Olivier Patel, Shreyaskumar R Orcid: 0000-0002-0026-2348 Raut, Chandrajit P Razak, Albiruni R A Reed, Damon R Orcid: 0000-0002-8238-2465 Rutkowski, Piotr Sanfilippo, Roberta G Sbaraglia, Marta Schaefer, Inga-Marie Orcid: 0000-0001-9710-5500 Strauss, Dirk C Sundby Hall, Kirsten Tap, William D Thomas, David M van der Graaf, Winette T A van Houdt, Winan J Visser, Otto von Mehren, Margaret Wagner, Andrew J Orcid: 0000-0002-4384-9448 Wilky, Breelyn A Won, Young-Joo Orcid: 0000-0001-9861-6740 Fletcher, Christopher D M Dei Tos, Angelo P Trama, Annalisa K08 CA241085/CA/NCI NIH HHS/United States Journal Article United States Cancer. 2021 Apr 28. doi: 10.1002/cncr.33618.