Current status of biomarker testing in historically rare, high-unmet-need tumors: soft tissue sarcomas and thyroid cancers

Schroader, Bridgette Kanz; Kong, Sheldon X.; Андерсон, С.; Williamson, Todd; Sireci, Anthony; Shields, Kasia

doi:10.1080/14737140.2019.1682554

Cited by 3 publications

(4 citation statements)

References 79 publications

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“…As demonstrated in this case, NTRK ‐rearranged thyroid cancer is clinically aggressive with a high rate of metastatic spread, but with low mortality when treated with TRK inhibitor therapy 34 . Due to the prevalence and actionability of NTRK gene fusions, upfront molecular testing should be recommended for patients with advanced or metastatic RAI‐R DTC, if feasible 41‐43 …”

Section: Discussionmentioning

confidence: 84%

“…34 Due to the prevalence and actionability of NTRK gene fusions, upfront molecular testing should be recommended for patients with advanced or metastatic RAI-R DTC, if feasible. [41][42][43] Entrectinib, another recently developed TRK inhibitor, also targets NTRK1-3, but also has activity against protooncogene tyrosine-protein kinase ROS1 (ROS1), anaplastic lymphoma kinase (ALK), Janus kinase 2 (JAK2), and activated CDC42 kinase 1 (TNK2). 44,45 At the time of publication, entrectinib had been evaluated in five patients with TRK fusion thyroid cancer, with a recorded response in one patient (ORR 20%).…”

Section: F-fdg 2-deoxy-2-[fluorine-18] Fluoro-d-glucose; Pet/ct Positron Emission Tomography/computed Tomographymentioning

confidence: 99%

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Complete response to larotrectinib treatment in a patient with papillary thyroid cancer harboring an ETV6‐NTRK3 gene fusion

Pitoia

2021

Clinical Case Reports

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Section: Discussionmentioning

confidence: 84%

Section: F-fdg 2-deoxy-2-[fluorine-18] Fluoro-d-glucose; Pet/ct Positron Emission Tomography/computed Tomographymentioning

confidence: 99%

Complete response to larotrectinib treatment in a patient with papillary thyroid cancer harboring an ETV6‐NTRK3 gene fusion

Pitoia

2021

Clinical Case Reports

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“…2 Historically, sarcoma treatment has consisted primarily of surgery, chemotherapy, and radiation therapy with use of targeted therapy in selected subgroups. 3,4,58 Despite this, outcomes in the relapsed/ refractory setting remain poor 17,59 and chemotherapy-based regimens are associated with substantial toxicity and can be challenging to administer in elderly or frail patients. Even when loco-regional treatment with curative intent is possible, treatment such as radical resection can result in substantial morbidity (eg, amputations, especially in young patients), highlighting the need for safe and effective therapies.…”

Section: Discussionmentioning

confidence: 99%

“…The incidence of sarcoma is increasing relative to other cancers 2 . Historically, sarcoma treatment has consisted primarily of surgery, chemotherapy, and radiation therapy with use of targeted therapy in selected subgroups 3,4,58 . Despite this, outcomes in the relapsed/refractory setting remain poor 17,59 and chemotherapy‐based regimens are associated with substantial toxicity and can be challenging to administer in elderly or frail patients.…”

Section: Discussionmentioning

confidence: 99%

Canadian consensus on TRK‐inhibitor therapy for NTRK fusion‐positive sarcoma

Simmons

Deyell

MacNeill

et al. 2021

Intl Journal of Cancer

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Malignant sarcomas are rare accounting for <1% of all adult solid malignancies and approximately 11% to 13% of all pediatric malignancies. TRK‐inhibitors have demonstrated robust and long‐lasting responses in patients with NTRK fusion‐positive solid tumors, including sarcoma. Access to these agents in many jurisdictions such as Canada remains limited. We undertook a modified Delphi consensus to articulate and convey the clinical importance of these agents for the Canadian sarcoma community. A systematic search of published and presented literature was conducted to identify clinical trials reporting outcomes on the use of TRK‐inhibitors in relapsed/refractory NTRK fusion‐positive sarcoma. Three main consensus questions were identified: (a) is there currently an unmet clinical need for systemic therapy options in relapsed/refractory sarcoma? (b) do TRK‐inhibitors confer a clinical benefit to patients with NTRK fusion‐positive sarcoma? (c) do phase I/II basket trials provide sufficient evidence to justify funding of TRK‐inhibitors in NTRK fusion‐positive sarcoma? Response rates to the first and second surveys were 57% (n = 30) and 42% (n = 22), respectively. There was strong agreement among the Canadian sarcoma community that there was unmet clinical need for effective systemic therapy options in relapsed/refractory sarcoma, that TRK‐inhibitors are a safe and effective treatment option for patients with NTRK fusion‐positive sarcoma, and that available phase I/II basket trials provide sufficient evidence to support funding of these agents in relapsed/refractory NTRK fusion‐positive sarcoma. TRK‐inhibitors are a safe and effective systemic therapy option for patients with relapsed/refractory NTRK fusion‐positive sarcoma.

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Literature Review on Health Emigration in Rare Diseases—A Machine Learning Perspective

Skweres-Kuchta

Czerska

Szaruga

2023

IJERPH

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The article deals with one of the effects of health inequalities and gaps in access to treatments for rare diseases, namely health-driven emigration. The purpose of the paper is to systematize knowledge about the phenomenon of health emigration observed among families affected by rare diseases, for which reimbursed treatment is available, but only in selected countries. The topic proved to be niche; the issue of “health emigration in rare diseases” is an area for exploration. Therefore, the further analysis used text mining and machine learning methods based on a database selected based on keywords related to this issue. The results made it possible to systematize the guesses made by researchers in management and economic fields, to identify the most common keywords and thematic clusters around the perspective of the patient, drug manufacturer and treatment reimbursement decision-maker, and the perspective integrating all the others. Since the topic of health emigration was not directly addressed in the selected sources, the authors attempted to define the related concepts and discussed the importance of this phenomenon in managing the support system in rare diseases. Thus, they indicated directions for further research in this area.

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Current status of biomarker testing in historically rare, high-unmet-need tumors: soft tissue sarcomas and thyroid cancers

Cited by 3 publications

References 79 publications

Complete response to larotrectinib treatment in a patient with papillary thyroid cancer harboring an ETV6‐NTRK3 gene fusion

Complete response to larotrectinib treatment in a patient with papillary thyroid cancer harboring an ETV6‐NTRK3 gene fusion

Canadian consensus on TRK‐inhibitor therapy for NTRK fusion‐positive sarcoma

Literature Review on Health Emigration in Rare Diseases—A Machine Learning Perspective

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