Long-term follow-up of nilotinib in patients with advanced tenosynovial giant cell tumours

Spierenburg, Geert; Grimison, Peter; Chevreau, Christine; Stacchiotti, Silvia; Piperno‐Neumann, Sophie; Cesne, Axel Le; Ferraresi, Virginia; Italiano, Antoîne; Duffaud, Florence; Penel, Nicolas; Metzger, Séverine; Chabaud, Sylvie; Heijden, L. van der; Pérol, David; Sande, Michiel A. J. van de; Blay, Jean‐Yves; Gelderblom, Hans

doi:10.1016/j.ejca.2022.06.028

Cited by 13 publications

(8 citation statements)

References 33 publications

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“…ORR after one year of treatment was 6% [28]. Long-term analysis of the study showed that, after 8.5 years, half of the patients had experienced progression with a need for additional treatment, while disease control persisted in several patients after nilotinib cessation [29]. Stable disease after nilotinib cessation was also reported in case reports [30].…”

Section: Nilotinib (Table 2)mentioning

confidence: 66%

Overview of Pharmacological Therapies for Diffuse Tenosynovial Giant Cell Tumor

Stamatiou,

Nguyen-Ngoc,

Wetterwald

et al. 2023

Future Pharmacology

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Tenosynovial giant cell tumor (TGCT) is a rare and locally aggressive benign tumor arising from the synovium of joints, bursae, and tendon sheaths. It is classified into localized (L-TGCT) and diffuse (D-TGCT) forms based on the extent of involvement. Surgical resection is the primary treatment, though achieving a definitive cure remains challenging due to the high recurrence rates, especially in D-TGCT. Systemic therapies targeting the CSF1-CSF1R axis have emerged as promising treatment options. CSF1R tyrosine kinase inhibitors (TKIs) such as imatinib, nilotinib, pexidartinib, and vimseltinib, alongside anti-CSF1R antibodies like emactuzumab, cabiralizumab, and lacnotuzumab, have shown encouraging results in managing TGCT, particularly when surgery is not feasible or poses significant morbidity. Other potential therapies, including local treatments and anti-inflammatory drugs, are being explored for TGCT management. This review provides an overview of systemic treatment options for D-TGCT, highlighting emerging therapeutic modalities and their potential implications. Effective management is crucial due to TGCT’s significant morbidity despite its non-life-threatening nature, necessitating novel approaches to improve patient prognosis and quality of life.

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Section: Nilotinib (Table 2)mentioning

confidence: 66%

Overview of Pharmacological Therapies for Diffuse Tenosynovial Giant Cell Tumor

Stamatiou,

Nguyen-Ngoc,

Wetterwald

et al. 2023

Future Pharmacology

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“…CSF1R pathway inhibitors have shown activity in TGCT with substantial tumour shrinkage, symptomatic and functional improvement, and longterm disease control. [56][57][58][59][60].…”

Section: Tgct Resectable With Unacceptable Morbiditymentioning

confidence: 99%

“…In a phase II trial of inoperable TGCT, nilotinib achieved a RECIST ORR of 6 %, with a progression-free rate of 96 % and 53 % at 12 weeks and 5 years, respectively. [59,60] Nilotinib is currently under investigation in a randomized clinical trial (NCT02029001).…”

Section: Tgct Resectable With Unacceptable Morbiditymentioning

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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“…Pexidartinib was subsequently approved by the FDA for adult patients with symptomatic TGCT associated with severe morbidity or functional limitations who are not amenable to improvement with surgery [ 67 ]. Other notable therapeutic agents that also target the CSF-1 pathway in TGCT include imatinib, nilotinib, and vimseltinib, although pexidartinib remains the only treatment for TGCT currently approved by the FDA [ 68 ].…”

Section: Molecular Mechanisms and Targets For Stsmentioning

confidence: 99%

The Next Frontier in Sarcoma: Molecular Pathways and Associated Targeted Therapies

Kim

Bui

2023

Cancers

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Soft tissue sarcomas (STS) are a rare, complex, heterogeneous group of mesenchymal neoplasms with over 150 different histological subtypes. Treatments for this malignancy have been especially challenging due to the heterogeneity of the disease and the modest efficacy of conventional chemotherapy. The next frontier lies in discerning the molecular pathways in which these mesenchymal neoplasms arise, metastasize, and develop drug-resistance, thereby helping guide new therapeutic targets for the treatment of STS. This comprehensive review will discuss the current understanding of tumorigenesis of specific STS subtypes, including oncogenic pathway alterations involved in cell cycle regulation, angiogenesis, NOTCH signaling, and aberrant genetic rearrangements. It will then review current therapies that have been recently developed to target these pathways, including a review of ongoing clinical studies for targeted sarcoma treatment, as well as discuss new potential avenues for therapies against known molecular pathways of sarcomagenesis.

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Long-term follow-up of nilotinib in patients with advanced tenosynovial giant cell tumours

Cited by 13 publications

References 33 publications

Overview of Pharmacological Therapies for Diffuse Tenosynovial Giant Cell Tumor

Overview of Pharmacological Therapies for Diffuse Tenosynovial Giant Cell Tumor

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

The Next Frontier in Sarcoma: Molecular Pathways and Associated Targeted Therapies

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