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

Stephen X. Skapek, James R. Anderson, D. Ashley Hill, David Henry, Sheri L. Spunt, William Meyer, Simon Kao, Fredric A. Hoffer, Holcombe E. Grier, Douglas S. Hawkins, R. Beverly Raney

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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 <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. 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.

Original languageEnglish (US)
Pages (from-to)1108-1112
Number of pages5
JournalPediatric Blood and Cancer
Volume60
Issue number7
DOIs
StatePublished - Jul 2013

Fingerprint

Sulindac
Aggressive Fibromatosis
Tamoxifen
Safety
Neoplasms
Disease-Free Survival
Ovarian Cysts
Disease Progression
Survival Rate
Soft Tissue Neoplasms
Radiation
Drug Therapy
Fibroma
Magnetic Resonance Imaging
Confidence Intervals
Recurrence
Therapeutics

Keywords

  • Chemotherapy
  • Desmoid-type fibromatosis
  • Sulindac
  • Tamoxifen

ASJC Scopus subject areas

  • Oncology
  • Pediatrics, Perinatology, and Child Health
  • Hematology

Cite this

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. / 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.

In: Pediatric Blood and Cancer, Vol. 60, No. 7, 07.2013, p. 1108-1112.

Research output: Contribution to journalArticle

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', Pediatric Blood and Cancer, vol. 60, no. 7, pp. 1108-1112. https://doi.org/10.1002/pbc.24457
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. / 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. In: Pediatric Blood and Cancer. 2013 ; Vol. 60, No. 7. pp. 1108-1112.
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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 <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. 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.",
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T1 - Safety and efficacy of high-dose tamoxifen and sulindac for desmoid tumor in children

T2 - Results of a Children's Oncology Group (COG) Phase II Study

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AU - Anderson, James R.

AU - Hill, D. Ashley

AU - Henry, David

AU - Spunt, Sheri L.

AU - Meyer, William

AU - Kao, Simon

AU - Hoffer, Fredric A.

AU - Grier, Holcombe E.

AU - Hawkins, Douglas S.

AU - Raney, R. Beverly

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N2 - 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 <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. 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.

AB - 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 <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. 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.

KW - Chemotherapy

KW - Desmoid-type fibromatosis

KW - Sulindac

KW - Tamoxifen

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