Journal article
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
Pediatric blood & cancer, Vol.60(7), pp.1108-1112
07/2013
DOI: 10.1002/pbc.24457
PMCID: PMC4646066
PMID: 23281268
Abstract
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.
Eligible patients were <19 years of age who had measurable DT that was recurrent or not amenable to surgery or radiation. The primary objective was to estimate progression-free survival (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.
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.
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.
Details
- Title: Subtitle
- 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
- Creators
- Stephen X Skapek - Division of Pediatric Hematology/Oncology, Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, Texas 75390-9063, USA. stephen.skapek@UTSouthwestern.eduJames R AndersonD Ashley HillDavid HenrySheri L SpuntWilliam MeyerSimon KaoFredric A HofferHolcombe E GrierDouglas S HawkinsR Beverly Raney
- Resource Type
- Journal article
- Publication Details
- Pediatric blood & cancer, Vol.60(7), pp.1108-1112
- DOI
- 10.1002/pbc.24457
- PMID
- 23281268
- PMCID
- PMC4646066
- NLM abbreviation
- Pediatr Blood Cancer
- ISSN
- 1545-5009
- eISSN
- 1545-5017
- Publisher
- United States
- Grant note
- U10 CA098413 / NCI NIH HHS U10 CA024507 / NCI NIH HHS CA-24507 / NCI NIH HHS CA-98543 / NCI NIH HHS U10 CA029511 / NCI NIH HHS U10 CA098543 / NCI NIH HHS CA-29511 / NCI NIH HHS U10 CA072989 / NCI NIH HHS
- Language
- English
- Date published
- 07/2013
- Academic Unit
- Radiology; Stead Family Department of Pediatrics
- Record Identifier
- 9984051737802771
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