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Pazopanib, a promising option for the treatment of aggressive fibromatosis.

ABSTRACT: Desmoid tumour/aggressive fibromatosis (DT/AF) is a rare soft-tissue neoplasm that is locally aggressive but does not metastasize. There is no standard systemic treatment for symptomatic patients, although a number of agents are used. Tyrosine kinase inhibitors have recently been reported to show useful activity. We reviewed our bi-institutional (Royal Marsden Hospital, Cambridge University Hospitals) experience with the tyrosine kinase inhibitor pazopanib in the treatment of progressing DT/AF. Eight patients with DT/AF were treated with pazopanib at Royal Marsden Hospital and Cambridge University Hospitals between June 2012 and June 2016. The median age of the patients was 37.5 (range: 27-60) years. The median duration of pazopanib treatment was 12 (range: 5-22) months and for three patients the treatment is ongoing. Three patients discontinued treatment early (patient preference, intolerable toxicity and logistical reasons, respectively). None of the patients showed radiological progression while on treatment, best responses according to Response Evaluation Criteria In Solid Tumors 1.1 were partial response in 3/8 and stable disease in 5/8 cases. Six patients derived clinical benefit from treatment in terms of improved function and/or pain reduction. Median progression-free survival was 13.5 (5-36) months. Only one patient experienced intolerable toxicity (grade 3 hypertension) leading to early treatment discontinuation. In our series of patients with DT/AF, pazopanib demonstrated important activity both in terms of symptom control (75%) and absence of radiological progression (100%). Results of ongoing confirmatory trials are eagerly awaited.

SUBMITTER: Szucs Z

PROVIDER: S-EPMC5469564 | biostudies-other | 2017 Apr

REPOSITORIES: biostudies-other

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Pazopanib, a promising option for the treatment of aggressive fibromatosis.

Szucs Zoltan Z Messiou Christina C Wong Han Hsi HH Hatcher Helen H Miah Aisha A Zaidi Shane S van der Graaf Winette T A WT Judson Ian I Jones Robin L RL Benson Charlotte C

Anti-cancer drugs 20170401 4

Desmoid tumour/aggressive fibromatosis (DT/AF) is a rare soft-tissue neoplasm that is locally aggressive but does not metastasize. There is no standard systemic treatment for symptomatic patients, although a number of agents are used. Tyrosine kinase inhibitors have recently been reported to show useful activity. We reviewed our bi-institutional (Royal Marsden Hospital, Cambridge University Hospitals) experience with the tyrosine kinase inhibitor pazopanib in the treatment of progressing DT/AF. ...[more]

PMID: 28099210

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Project description:BACKGROUND:Desmoid-type fibromatosis is a rare, potentially locally aggressive disease. Herein we present our experience in the treatment with radiotherapy. METHODS AND MATERIALS:In total 40 patients who received 44 treatments from 2009 to 2018 at the Heidelberg University Hospital with photons (N =?28) as well as protons (N =?15) and carbon ions (N =?1) were investigated. The median age at radiotherapy was 41?years [range 8-78]. Familial adenomatous polyposis (FAP) was confirmed for nine patients and 30 had a unifocal desmoid tumor. The localizations were abdominal wall, abdominopelvic cavity, thoracic wall, extremity, head and neck and trunk. The median prescribed dose was 54?Gy/ Gy (RBE) [range 39.6-66, IQR 50-60]. Eleven treatments were performed at the time of first diagnosis; 33 at the time of progression or recurrence. Post-operative radiotherapy was performed in 17 cases. The median planning target volume was 967?ml [84-4364?ml, IQR 447-1988]. Survival analysis was performed by the Kaplan-Meier Method. RESULTS:The median follow-up time was 32?months [1-153]. At the end of the follow-up interval all patients but one were alive. The estimated local progression free survival of the treated lesion in 3 and 5?years was 76.4% and 63,8%, respectively. The progression-free survival in 3 and 5?years was 72.3 and 58.4% and the overall survival was 97.4 and 97.4%, respectively. In case of macroscopic tumor (N =?31) before radiotherapy a partial remission was observed in 12 cases (38.7%) and a complete remission in 4 cases (12.9%). Progression was observed in 13 (29.5%) cases, predominantly at the margin of the planning target volume (PTV, N =?5, 38,4%) followed by progression within the PTV (N =?4, 30.8%). In univariate analysis multifocal localization was associated with impaired progression-free survival (p =?0.013). One patient developed a grade V gastrointestinal bleeding, otherwise no acute toxicity >°III was observed. Late toxicity was depending on the localization of the desmoid tumor and was especially severe in patients with FAP and abdominopelvine desmoids including gastrointesinal fistula, perforation and abscess. CONCLUSION:Radiotherapy in the treatment of desmoids can lead to long term control. Treatment of patients with abdominopelvine desmoids should be avoided, as the risk of higher-grade complications is substantial.

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Pazopanib, a promising option for the treatment of aggressive fibromatosis.

Publications

Pazopanib, a promising option for the treatment of aggressive fibromatosis.

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