Title

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

UMMS Affiliation

Department of Radiation Oncology

Date

7-2013

Document Type

Article

Medical Subject Headings

Adolescent; Antineoplastic Combined Chemotherapy Protocols; effects; Child; Disease-Free Survival; Female; Fibromatosis, Aggressive; Humans; Male; Sulindac; Tamoxifen

Disciplines

Neoplasms | Oncology

Abstract

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.

Rights and Permissions

Citation: Pediatr Blood Cancer. 2013 Jul;60(7):1108-12. doi: 10.1002/pbc.24457. Link to article on publisher's site

Comments

This study was supported in part by Grant CA-29511 from the National Cancer Institute for the IROC Rhode Island (QARC), a quality assurance vehicle and data management service for diagnostic imaging and radiation oncology for the National Cancer Institute Clinical Trials Program. QARC is a research program within the University of Massachusetts Medical School led by Dr. Thomas (TJ) FitzGerald.

Related Resources

Link to Article in PubMed