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
Quality Assurance Review Center
Health Services Administration | Neoplasms | Oncology | Pediatrics | Radiology
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.
DOI of Published Version
Pediatr Blood Cancer. 2013 Jul;60(7):1108-12. doi: 10.1002/pbc.24457. Epub 2012 Dec 31. Link to article on publisher's site
Pediatric blood and cancer
Skapek SX, Anderson JR, Hill DA, Henry D, Spunt SL, Meyer W, Kao S, Hoffer FA, Grier HE, Hawkins DS, Raney RB. (2013). 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. IROC Rhode Island (QARC) Publications. https://doi.org/10.1002/pbc.24457. Retrieved from https://escholarship.umassmed.edu/qarc/27