Ultra-rare sarcomas: A consensus paper from the Connective Tissue Oncology Society community of experts on the incidence threshold and the list of entities.
Ultra-rare sarcomas: A consensus paper from the Connective Tissue Oncology Society community of experts on the incidence threshold and the list of entities.
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作者：
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
在肉瘤中，这是一种罕见的癌症，许多类型是非常罕见的;然而，超罕见癌症的定义尚未建立。超罕见肉瘤的问题特别相关，因为它们代表独特的疾病，并且它们的罕见性对诊断，理解疾病生物学，产生支持新药开发的临床证据以及获得新疗法的正式授权构成了重大挑战。结缔组织肿瘤学会于2019年11月推动了一项共识努力，以确定如何通过专家共识和流行病学数据定义超罕见肉瘤，并制定出这些疾病的综合清单。超罕见肉瘤列表基于2020年世界卫生组织分类，使用罕见癌症信息网络（RARECARENet）数据库和NETSARC（法国肉瘤网络的临床病理登记处）估计发病率。与日本、韩国和台湾的亚洲癌症登记处合作，进一步验证了发病率。人们一致认为，定义超罕见肉瘤的最佳标准是发病率。超罕见肉瘤定义为发生率约≤1/1，000，000的肉瘤，包括罕见性极难进行有效性良好的前瞻性临床研究的实体。在此基础上，定义了一系列超罕见肉瘤，其中包括56种软组织肉瘤和21种骨肉瘤。总之，超罕见肉瘤的发病率约占所有软组织和骨肉瘤的20%。这证实了超罕见肉瘤固有的挑战影响了大量患者。
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.
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影响因子：
51.1
作者：
Gounder, Mrinal;Schoffski, Patrick;Stacchiotti, Silvia
通讯作者：
Stacchiotti, Silvia
影响因子：
3.7
作者：
Perrier L;Rascle P;Morelle M;Toulmonde M;Ranchere Vince D;Le Cesne A;Terrier P;Neuville A;Meeus P;Farsi F;Ducimetière F;Blay JY;Ray Coquard I;Coindre JM
通讯作者：
Coindre JM
影响因子：
254.7
作者：
DeSantis, Carol E.;Kramer, Joan L.;Jemal, Ahmedin
通讯作者：
Jemal, Ahmedin
影响因子：
1.9
作者：
Bustamante-Teixeira, Maria Teresa;Latorre, Maria do Rosario D. O.;Gatta, Gemma
通讯作者：
Gatta, Gemma
影响因子：
4
作者：
Botta, Laura;Gatta, Gemma;Mariotto, Angela B.
通讯作者：
Mariotto, Angela B.