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

Stacchiotti, Silvia; Frezza, Anna Maria; Demetri, George D.; Blay, Jean‐Yves; Bajpai, Jyoti; Baldi, Giacomo Giulio; Baldini, Elizabeth H.; Benjamin, Robert S.; Bonvalot, Sylvie; Bovée, Judith V.M.G.; Callegaro, Dario; Casali, Paolo; D’Angelo, Sandra P.; Davis, Elizabeth J.; Tos, Angelo Paolo Dei; Demicco, Elizabeth G.; Desai, Jayesh; Dileo, Palma; Eriksson, Mikael; Gelderblom, Hans; George, Suzanne; Gladdy, Rebecca A.; Gounder, Mrinal M.; Gupta, Abha; Haas, Rick L. M.; Hayes, Andrea Giráldez; Hohenberger, Peter; Jones, Kevin B.; Jones, Robin L.; Kasper, Bernd; Kawai, Akira; Kirsch, David G.; Kleinerman, Eugenie S.; Cesne, Axel Le; Maestro, Roberta; Broto, Javier Martín; Maki, Robert G.; Miah, Aisha; Palmerini, Emanuela; Shreyaskumar, R. Patal; Raut, Chandrajit P.; Razak, Albiruni R.A.; Reed, Damon R.; Rutkowski, Piotr; Sanfilippo, Roberta; Sbaraglia, Marta; Schaefer, Inga-Marie; Strauß, D.; Strauss, Sandra J.; Tap, William D.; Thomas, David M.; Trama, Annalisa; Trent, Jonathan C.; Graaf, Winette van der; Houdt, Winan J. van; vonMehren, Margaret; Wilky, Breelyn A.; Fletcher, Christopher D.�M.; Gronchi, Alessandro; Miceli, Rosalba; Wagner, Andrew J.

doi:10.1016/j.ctrv.2022.102455

Cited by 15 publications

(9 citation statements)

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“…1,14,15,26,27 In an attempt to resolve this ambiguity, we studied the clinical and biological characteristics of 42 patients with EH treated at a single institution. Although the retrospective design could be considered a limitation, the rarity of EH mandates that such a study be retrospective to have sufficient patients for analysis 28 and the relatively large case series is an advantage of the present study.…”

Section: Discussionmentioning

confidence: 99%

Fusion transcriptome profiling defines the monoclonal origin of multifocal epithelioid haemangioma of bone

et al. 2023

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AimsEpithelioid haemangioma (EH) of bone remains a highly controversial entity. Indeed, the WHO classifies EHs of soft tissues as benign tumours, whereas bone EHs are considered intermediate–locally aggressive tumours due to common multifocal presentation and local destructive growth. To gain insights into the clinical behaviour and biology of EH of bone we retrospectively analysed 42 patients treated in a single institution from 1978 to 2021.Methods and resultsMultifocal presentation was detected in 17 of 42 patients (40%) primarily as synchronous lesions. Patients were treated with curettage (57%), resection (29%) or biopsy, followed by radiotherapy or embolisation (14%). Follow‐up (minimum 24 months) was available for 38 patients, with only five local recurrences (13%) and no death of disease. To clarify whether the synchronous bone lesions in multifocal EH represent multicentric disease or clonal dissemination, four cases were profiled by RNA‐sequencing. Separate lesions from the same patient, which showed a similar transcriptional profile, expressed the same fusion transcript (involving FOS or FOSB) with identical gene breakpoints.ConclusionsThese results indicate that, in EH of bone, multifocal lesions are clonally related and therefore represent the spread of a same neoplastic clone rather than simultaneous independent tumours. This finding is in apparent contradiction with the benign clinical course of the disease, and suggests that tumour dissemination in bone EH probably reflects a phenomenon of passive spreading, with tumour cells colonising distal sites while maintaining their benign biological nature.

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Section: Discussionmentioning

confidence: 99%

Fusion transcriptome profiling defines the monoclonal origin of multifocal epithelioid haemangioma of bone

et al. 2023

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“…Consequently, a degree of uncertainty needs to be accepted in clinical management and regulatory matters. [9,10] We graded levels of evidence from I to V and used recommendation grades from A-D adapted from the Infectious Diseases Society of America-US Public Health Service Grading System 2 (Table 1).…”

Section: Methods Level Of Evidence and Grade Of Recommendationmentioning

confidence: 99%

Best clinical management of tenosynovial giant cell tumour (TGCT): A consensus paper from the community of experts

Stacchiotti

Roland

Schaefer

et al. 2023

Cancer Treatment Reviews

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“…Recently, after achievements in ALK‐ and ROS1‐positive non–small cell lung cancer, crizotinib was approved in 2022 for ALK‐translocated inflammatory myofibroblastic tumors 14,15 . The CTOS ultrarare sarcoma consensus group also made recommendations for evaluating treatments for sarcomas of ultrarare histologies through observational studies in tumor types 16 . The process of conducting sequential tumor‐by‐tumor clinical trials for the same drug to obtain approval for rare cancers can be expensive, time‐consuming, and challenging to execute.…”

Section: Actionable Target Drug Mechanism Fda‐approved Tumor Type(s)mentioning

confidence: 99%

“…14,15 The CTOS ultrarare sarcoma consensus group also made recommendations for evaluating treatments for sarcomas of ultrarare histologies through observational studies in tumor types. 16 The process of conducting sequential tumor-by-tumor clinical trials for the same drug to obtain approval for rare cancers can be expensive, time-consuming, and challenging to execute.…”

Section: Actionable Targetmentioning

confidence: 99%

TRKing sarcomas for precision medicine: Seek, and ye shall find!

Moyers,

Subbiah

2023

Cancer

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Plain language summary The way in which we treat different types of cancer has changed a lot because of genome‐driven precision oncology. This approach uses information from a person’s genes to personalize their treatment. A recent study conducted by Kummar and colleagues confirms that neurotrophic receptor tyrosine kinase (NTRK) inhibitors are beneficial in treating sarcomas with NTRK fusions. This study adds to the growing evidence that genomics, which is the study of genes and their functions, can help us to identify effective treatments even for rare targets such as NTRK fusions. This emphasizes the importance of using advanced DNA sequencing techniques in analyzing the genetic makeup of all sarcomas.

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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

Cited by 15 publications

References 15 publications

Fusion transcriptome profiling defines the monoclonal origin of multifocal epithelioid haemangioma of bone

Fusion transcriptome profiling defines the monoclonal origin of multifocal epithelioid haemangioma of bone

Best clinical management of tenosynovial giant cell tumour (TGCT): A consensus paper from the community of experts

TRKing sarcomas for precision medicine: Seek, and ye shall find!

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