The Desmoid Tumor Research Foundation

May 2024

Real-word experience of pazopanib and sorafenib in patients with desmoid tumors: A CanSaRCC multi-center study

Author Information

Jonathan Noujaim 1, Abha A Gupta 2, Caroline L Holloway 3, Ramy Saleh 4, Amirrtha Srikanthan 5, Christopher Lemieux 6, Hagit Peretz Soroka 2, Pauline Tibout 7, Robert Turcotte 4, Xiaolan Feng 8, Albiruni R Abdul Razak 9, Philippos Costa 10

1Maisonneuve-Rosemont Hospital, Montreal, Qc, Canada. Electronic address: jonathan.noujaim.med@ssss.gouv.qc.ca. 2Department of Medical Oncology and Hematology, Princess Margaret Cancer Centre, Toronto, ON, Canada; Division of Hematology/Oncology, Hospital for Sick Children, Toronto, Ontario, Canada. 3BC Cancer, Victoria, Victoria, BC, Canada. 4McGill University Health Centre, Cedars Cancer Centre, Montreal, Qc, Canada. 5Ottawa Hospital, ON, Canada. 6Centre Hospitalier Universitaire de Québec, Quebec, Qc, Canada. 7Division of Hematology/Oncology, Hospital for Sick Children, Toronto, Ontario, Canada. 8Tom Baker Cancer Center, Calgary, AB, Canada. 9Department of Medical Oncology and Hematology, Princess Margaret Cancer Centre, Toronto, ON, Canada; Department of Medical Oncology, Mount Sinai Hospital, Toronto, ON, Canada. 10Yale University, New Haven, Connecticut, Canada.

Publication

Eur J Cancer

Summary

Background: Sorafenib and pazopanib, two tyrosine kinase inhibitors (TKI), are widely used in patients with progressive symptomatic desmoid tumors (DT). Limited real-word data is available on long-term outcomes of patients who progressed on, stopped, or continued TKIs. Methods: Patients diagnosed with DTs and treated with sorafenib or pazopanib between 2011 and 2022 at 11 institutions were reviewed. Patient history, response to therapy and toxicity were recorded. Statistical analyses utilized Kaplan-Meier and log-rank tests. Results: 142 patients with DT treated with sorafenib (n = 126, 88.7 %) or pazopanib (n = 16, 11.3 %) were analyzed. The median treatment duration was 10.8 months (range: 0.07- 73.9). The overall response rate and the disease control rate were 26.0 % and 95.1 %, respectively. The median tumor shrinkage was - 8.5 % (range -100.0 %- +72.5 %). Among responders, the median time to an objective response was 15.2 months (range: 1.1 to 33.1). The 1-year and 2-year progression-free survival rates were 82 % and 80 %. Dose reductions were necessary in 34 (23.9 %) patients. Grade 3 or higher adverse events were reported in 36 (25.4 %) patients. On the last follow-up, 55 (38.7 %) patients continued treatment. Treatment discontinuation (n = 85, 59.9 %) was mainly for toxicity (n = 35, 45.9 %) or radiological or clinical progression (n = 30, 35.3 %). For the entire cohort, 36 (25.4 %) patients required subsequent treatment. In the 32 responders, only 1 (3.1 %) patient required a subsequent treatment. In patients who discontinued TKI, 25 (44.6 %) with stable disease received subsequent treatment compared to 0 (0.0 %) of responders. Conclusion: This retrospective study represents the largest cohort of DT patients treated with sorafenib or pazopanib to date. Discontinuation of treatment in responders is safe. The optimal treatment duration in patients with stable disease remains to be defined.

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