RT Journal Article
T1 Ultra-rare sarcomas: A consensus paper from the Connective Tissue Oncology Society community of experts on the incidence threshold and the list of entities.
A1 Stacchiotti, Silvia
A1 Frezza, Anna Maria
A1 Blay, Jean-Yves
A1 Baldini, Elizabeth H
A1 Bonvalot, Sylvie
A1 Bovée, Judith V M G
A1 Callegaro, Dario
A1 Casali, Paolo G
A1 Chiang, RuRu Chun-Ju
A1 Demetri, George D
A1 Demicco, Elisabeth G
A1 Desai, Jayesh
A1 Eriksson, Mikael
A1 Gelderblom, Hans
A1 George, Suzanne
A1 Gounder, Mrinal M
A1 Gronchi, Alessandro
A1 Gupta, Abha
A1 Haas, Rick L
A1 Hayes-Jardon, Andrea
A1 Hohenberger, Peter
A1 Jones, Kevin B
A1 Jones, Robin L
A1 Kasper, Bernd
A1 Kawai, Akira
A1 Kirsch, David G
A1 Kleinerman, Eugene S
A1 Le Cesne, Axel
A1 Lim, Jiwon
A1 Chirlaque López, María Dolores
A1 Maestro, Roberta
A1 Marcos-Gragera, Rafael
A1 Martin Broto, Javier
A1 Matsuda, Tomohiro
A1 Mir, Olivier
A1 Patel, Shreyaskumar R
A1 Raut, Chandrajit P
A1 Razak, Albiruni R A
A1 Reed, Damon R
A1 Rutkowski, Piotr
A1 Sanfilippo, Roberta G
A1 Sbaraglia, Marta
A1 Schaefer, Inga-Marie
A1 Strauss, Dirk C
A1 Sundby Hall, Kirsten
A1 Tap, William D
A1 Thomas, David M
A1 van der Graaf, Winette T A
A1 van Houdt, Winan J
A1 Visser, Otto
A1 von Mehren, Margaret
A1 Wagner, Andrew J
A1 Wilky, Breelyn A
A1 Won, Young-Joo
A1 Fletcher, Christopher D M
A1 Dei Tos, Angelo P
A1 Trama, Annalisa
K1 drug development
K1 incidence
K1 rarity
K1 registry
K1 sarcoma
K1 ultra-rare
AB 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. 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. 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. 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.
YR 2021
FD 2021-04-28
LK http://hdl.handle.net/10668/17647
UL http://hdl.handle.net/10668/17647
LA en
DS RISalud
RD Apr 5, 2025