A Timely Oral Option: Single-Agent Vinorelbine in Desmoid Tumors

Gennatas, Spyridon; Chamberlain, Florence; Smrke, Alannah; Stewart, James; Hayes, Andrew; Roden, Lorna; Messiou, Christina; Kowa, Jie-Ying; Estival, Anna; Chauhan, D; Thway, Khin; Fisher, Cyril; Graaf, Winette T.A. van der; Jones, Robin L.; Benson, Charlotte

doi:10.1002/onco.13516

Cited by 14 publications

(6 citation statements)

References 10 publications

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“…Low-dose MTX/VNL or VBL chemotherapy is effective and minimally toxic regimen but has significant impact on quality of life (QoL) for the lengthy duration of treatment. Single agent oral vinorelbine has a disease control rate of 86% with an excellent toxicity profile (46,64). Low dose chemotherapy regimens are an effective, safe, and affordable choice that can offer long term symptoms and disease control, however responses are delayed compared to other agents; their use is especially common in the pediatric and young adults' population for the well understood toxicity profile.…”

Section: Chemotherapymentioning

confidence: 99%

Current therapies and future prospective for locally aggressive mesenchymal tumors

et al. 2023

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Locally aggressive mesenchymal tumors comprise a heterogeneous group of soft tissue and bone tumors with intermediate histology, incompletely understood biology, and highly variable natural history. Despite having a limited to absent ability to metastasize and excellent survival prognosis, locally aggressive mesenchymal tumors can be symptomatic, require prolonged and repeat treatments including surgery and chemotherapy, and can severely impact patients’ quality of life. The management of locally aggressive tumors has evolved over the years with a focus on minimizing morbid treatments. Extensive oncologic surgeries and radiation are pillars of care for high grade sarcomas, however, play a more limited role in management of locally aggressive mesenchymal tumors, due to propensity for local recurrence despite resection, and the risk of transformation to a higher-grade entity following radiation. Patients should ideally be evaluated in specialized sarcoma centers that can coordinate complex multimodal decision-making, taking into consideration the individual patient’s clinical presentation and history, as well as any available prognostic factors into customizing therapy. In this review, we aim to discuss the biology, clinical management, and future treatment frontiers for three representative locally aggressive mesenchymal tumors: desmoid-type fibromatosis (DF), tenosynovial giant cell tumor (TSGCT) and giant cell tumor of bone (GCTB). These entities challenge clinicians with their unpredictable behavior and responses to treatment, and still lack a well-defined standard of care despite recent progress with newly approved or promising experimental drugs.

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Section: Chemotherapymentioning

confidence: 99%

Current therapies and future prospective for locally aggressive mesenchymal tumors

et al. 2023

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“…Several recent retrospective studies of oral singleagent vinorelbine have reported moderate response and clinical benefit rates in patients with DT. 68,69 Among 90 adults who had DT treated with oral vinorelbine with or without antiestrogen therapy, the best responses were 29% PR, 57% SD, and 14% progressive disease. 69 Concomitant antiestrogen therapy was associated with a significantly longer time to treatment failure in women (p = .03).…”

Section: Cytotoxic Chemotherapymentioning

confidence: 99%

Evolving strategies for management of desmoid tumor

Riedel

Agulnik

2022

Cancer

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Desmoid tumors (DTs) are rare soft tissue mesenchymal neoplasms that may be associated with impairments, disfigurement, morbidity, and (rarely) mortality. DT disease course can be unpredictable. Most DTs are sporadic, harboring somatic mutations in the gene that encodes for βcatenin, whereas DTs occurring in patients with familial adenomatous polyposis have germline mutations in the APC gene, which encodes for a protein regulator of βcatenin. Pathology review by an expert soft tissue pathologist is critical in making a diagnosis. Magnetic resonance imaging is preferred for most anatomic locations. Surgery, once the standard of care for initial treatment of DT, is associated with a significant risk of recurrence as well as avoidable morbidity because spontaneous regressions are known to occur without treatment. Consequently, active surveillance in conjunction with pain management is now recommended for most patients. Systemic medical treatment of DT has evolved beyond the use of hormone therapy, which is no longer routinely recommended. Current options for medical management include tyrosine kinase inhibitors as well as more conventional cytotoxic chemotherapy (e.g., anthracyclinebased or methotrexate-based regimens). A newer class of agents, γsecretase inhibitors, appears promising, including in patients who fail other therapies, but confirmation in Phase 3 trials is needed. In summary, DTs present challenges to physicians in diagnosis and prognosis, as well as in determining treatment initiation, type, duration, and sequence. Accordingly, evaluation by a multidisciplinary team with expertise in DT and patient-tailored management are essential. As management strategies continue to evolve, further studies will help clarify these issues and optimize outcomes for patients.

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“…No patient experienced at least grade 3 toxicity. Common toxicities were grades 1–2 nausea (48%), fatigue (31%), and diarrhea (14%) [19].…”

Section: Systemic Treatment Of Desmoid-type Fibromatosismentioning

confidence: 99%

Desmoid-type fibromatosis: toward a holistic management

Penel

Kasper

Graaf

2021

Current Opinion in Oncology

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Purpose of reviewDesmoid-type fibromatosis, a rare locally aggressive fibroblastic proliferation, is a treatment challenge. This review aimed to explore recent data about the management of desmoid-type fibromatosis. Recent findingsNew data underline the role of kinases and ]-secretase in stimulating cell proliferation and invasiveness in desmoid-type fibromatosis. This explains the proven activity of multikinase inhibitors (sorafenib or pazopanib) in the management of desmoid-type fibromatosis or the emerging role of a ]-secretase inhibitor. An international guideline for management was recently published, and this guideline take into account patient point of view. Lastly, recent studies highlight the multidimensional burden of desmoid-type fibromatosis, particularly health-related quality of life (HRQoL).

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A Timely Oral Option: Single-Agent Vinorelbine in Desmoid Tumors

Cited by 14 publications

References 10 publications

Current therapies and future prospective for locally aggressive mesenchymal tumors

Current therapies and future prospective for locally aggressive mesenchymal tumors

Evolving strategies for management of desmoid tumor

Desmoid-type fibromatosis: toward a holistic management

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