Safety and efficacy of high-dose tamoxifen and sulindac for desmoid tumor in children: results of a Children's Oncology Group (COG) phase II study
Authors
Skapek, Stephen X.Anderson, James R.
Hill, D. Ashley
Henry, David
Spunt, Sheri L.
Meyer, William
Kao, Simon
Hoffer, Fredric A.
Grier, Holcombe E.
Hawkins, Douglas S.
Raney, R. Beverly
UMass Chan Affiliations
Quality Assurance Review CenterDocument Type
Journal ArticlePublication Date
2013-07-01
Metadata
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BACKGROUND: Desmoid fibromatosis (desmoid tumor, DT) is a soft tissue neoplasm prone to recurrence despite complete surgical resection. Numerous small retrospective reports suggest that non-cytotoxic chemotherapy using tamoxifen and sulindac may be effective for DT. We evaluated the safety and efficacy of tamoxifen and sulindac in a prospective phase II study within the Children's Oncology Group. PROCEDURES: Eligible patients were (PFS). Patients received tamoxifen and sulindac daily for 12 months or until disease progression or intolerable toxicity occurred. Response was assessed by magnetic resonance imaging. RESULTS: Fifty-nine eligible patients were enrolled from 2004 to 2009; 78% were 10-18 years old. Twenty-two (38%) were previously untreated; 15 (41%) of the remaining 37 enrolling with recurrent DT had prior systemic chemotherapy and six (16%) had prior radiation. No life-threatening toxicity was reported. Twelve (40%) of 30 females developed ovarian cysts, which were asymptomatic in 11 cases. Ten patients completed therapy without disease progression or discontinuing treatment. Responses included four partial and one complete (5/59, 8%). The estimated 2-year PFS and survival rates were 36% (95% confidence interval: 0.23-0.48) and 96%, respectively. All three deaths were due to progressive DT. CONCLUSIONS: Tamoxifen and sulindac caused few serious side effects in children with DT, although ovarian cysts were common. However, the combination showed relatively little activity as measured by response and PFS rates.Source
Pediatr Blood Cancer. 2013 Jul;60(7):1108-12. doi: 10.1002/pbc.24457. Epub 2012 Dec 31. Link to article on publisher's siteDOI
10.1002/pbc.24457Permanent Link to this Item
http://hdl.handle.net/20.500.14038/46474PubMed ID
23281268Related Resources
Link to Article in PubMedae974a485f413a2113503eed53cd6c53
10.1002/pbc.24457