Prognostic factors and clinical outcomes in children and adolescents with metastatic rhabdomyosarcoma-a report from the intergroup rhabdomyosarcoma study IV

John C. Breneman, Elizabeth Lyden, Alberto S. Pappo, Michael P. Link, James R. Anderson, David M. Parham, Stephen J. Qualman, Moody D. Wharam, Sarah S. Donaldson, Harold M. Maurer, William H. Meyer, K. Scott Baker, Charles N. Paidas, William M. Crist

Research output: Contribution to journalArticle

Abstract

Purpose: To identify risk factors associated with outcomes in children with metastatic rhabdomyosarcoma (RMS) treated on the fourth Intergroup Rhabdomyosarcoma Study (IRS-IV). Patients and Methods: Patients with metastatic RMS were treated with one of two regimens that incorporated a window of either ifosfamide and etoposide (IE) with vincristine, dactinomycin, and cyclophosphamide (VAC) or vincristine, melphalan (VM) and VAC. Study end points were failure-free survival (FFS) and overall survival (OS). Clinical factors including age, histology, sites of primary and metastatic disease, and number of sites of metastatic disease were correlated with those end points. Results: One hundred twenty-seven patients were eligible for analysis. The estimated 3-year OS and FFS for all patients were 39% and 25%, respectively. By univariate analysis, 3-year OS was significantly influenced by histology (47% for embryonal v 34% for all others, P = .026) and increasing number of metastatic sites (P =.028). By multivariate analysis, the presence of two or fewer metastatic sites was the only significant predictor (P = .007 and .006, respectively). The combination of embryonal histology with two or fewer metastatic sites identified a subgroup with 3-year FFS of 40% and OS of 47%. Conclusion: Children with group IV RMS treated on the IRS-IV study had improved OS and FFS if they had two or fewer metastatic sites and embryonal histology. This favorable subset of patients has outcomes approaching those observed in selected patients with localized, nonmetastatic disease. Thus, these patients might not be appropriate candidates for regimens that include experimental agents with substantial toxicities or unproven antitumor activity.

Original languageEnglish (US)
Pages (from-to)78-84
Number of pages7
JournalJournal of Clinical Oncology
Volume21
Issue number1
DOIs
StatePublished - Jan 1 2003

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Rhabdomyosarcoma
Survival
Histology
Vincristine
Dactinomycin
Cyclophosphamide
Ifosfamide
Melphalan
Age Factors
Etoposide
Multivariate Analysis

ASJC Scopus subject areas

  • Cancer Research
  • Oncology

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Prognostic factors and clinical outcomes in children and adolescents with metastatic rhabdomyosarcoma-a report from the intergroup rhabdomyosarcoma study IV. / Breneman, John C.; Lyden, Elizabeth; Pappo, Alberto S.; Link, Michael P.; Anderson, James R.; Parham, David M.; Qualman, Stephen J.; Wharam, Moody D.; Donaldson, Sarah S.; Maurer, Harold M.; Meyer, William H.; Baker, K. Scott; Paidas, Charles N.; Crist, William M.

In: Journal of Clinical Oncology, Vol. 21, No. 1, 01.01.2003, p. 78-84.

Research output: Contribution to journalArticle

Breneman, JC, Lyden, E, Pappo, AS, Link, MP, Anderson, JR, Parham, DM, Qualman, SJ, Wharam, MD, Donaldson, SS, Maurer, HM, Meyer, WH, Baker, KS, Paidas, CN & Crist, WM 2003, 'Prognostic factors and clinical outcomes in children and adolescents with metastatic rhabdomyosarcoma-a report from the intergroup rhabdomyosarcoma study IV', Journal of Clinical Oncology, vol. 21, no. 1, pp. 78-84. https://doi.org/10.1200/JCO.2003.06.129
Breneman, John C. ; Lyden, Elizabeth ; Pappo, Alberto S. ; Link, Michael P. ; Anderson, James R. ; Parham, David M. ; Qualman, Stephen J. ; Wharam, Moody D. ; Donaldson, Sarah S. ; Maurer, Harold M. ; Meyer, William H. ; Baker, K. Scott ; Paidas, Charles N. ; Crist, William M. / Prognostic factors and clinical outcomes in children and adolescents with metastatic rhabdomyosarcoma-a report from the intergroup rhabdomyosarcoma study IV. In: Journal of Clinical Oncology. 2003 ; Vol. 21, No. 1. pp. 78-84.
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abstract = "Purpose: To identify risk factors associated with outcomes in children with metastatic rhabdomyosarcoma (RMS) treated on the fourth Intergroup Rhabdomyosarcoma Study (IRS-IV). Patients and Methods: Patients with metastatic RMS were treated with one of two regimens that incorporated a window of either ifosfamide and etoposide (IE) with vincristine, dactinomycin, and cyclophosphamide (VAC) or vincristine, melphalan (VM) and VAC. Study end points were failure-free survival (FFS) and overall survival (OS). Clinical factors including age, histology, sites of primary and metastatic disease, and number of sites of metastatic disease were correlated with those end points. Results: One hundred twenty-seven patients were eligible for analysis. The estimated 3-year OS and FFS for all patients were 39{\%} and 25{\%}, respectively. By univariate analysis, 3-year OS was significantly influenced by histology (47{\%} for embryonal v 34{\%} for all others, P = .026) and increasing number of metastatic sites (P =.028). By multivariate analysis, the presence of two or fewer metastatic sites was the only significant predictor (P = .007 and .006, respectively). The combination of embryonal histology with two or fewer metastatic sites identified a subgroup with 3-year FFS of 40{\%} and OS of 47{\%}. Conclusion: Children with group IV RMS treated on the IRS-IV study had improved OS and FFS if they had two or fewer metastatic sites and embryonal histology. This favorable subset of patients has outcomes approaching those observed in selected patients with localized, nonmetastatic disease. Thus, these patients might not be appropriate candidates for regimens that include experimental agents with substantial toxicities or unproven antitumor activity.",
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T1 - Prognostic factors and clinical outcomes in children and adolescents with metastatic rhabdomyosarcoma-a report from the intergroup rhabdomyosarcoma study IV

AU - Breneman, John C.

AU - Lyden, Elizabeth

AU - Pappo, Alberto S.

AU - Link, Michael P.

AU - Anderson, James R.

AU - Parham, David M.

AU - Qualman, Stephen J.

AU - Wharam, Moody D.

AU - Donaldson, Sarah S.

AU - Maurer, Harold M.

AU - Meyer, William H.

AU - Baker, K. Scott

AU - Paidas, Charles N.

AU - Crist, William M.

PY - 2003/1/1

Y1 - 2003/1/1

N2 - Purpose: To identify risk factors associated with outcomes in children with metastatic rhabdomyosarcoma (RMS) treated on the fourth Intergroup Rhabdomyosarcoma Study (IRS-IV). Patients and Methods: Patients with metastatic RMS were treated with one of two regimens that incorporated a window of either ifosfamide and etoposide (IE) with vincristine, dactinomycin, and cyclophosphamide (VAC) or vincristine, melphalan (VM) and VAC. Study end points were failure-free survival (FFS) and overall survival (OS). Clinical factors including age, histology, sites of primary and metastatic disease, and number of sites of metastatic disease were correlated with those end points. Results: One hundred twenty-seven patients were eligible for analysis. The estimated 3-year OS and FFS for all patients were 39% and 25%, respectively. By univariate analysis, 3-year OS was significantly influenced by histology (47% for embryonal v 34% for all others, P = .026) and increasing number of metastatic sites (P =.028). By multivariate analysis, the presence of two or fewer metastatic sites was the only significant predictor (P = .007 and .006, respectively). The combination of embryonal histology with two or fewer metastatic sites identified a subgroup with 3-year FFS of 40% and OS of 47%. Conclusion: Children with group IV RMS treated on the IRS-IV study had improved OS and FFS if they had two or fewer metastatic sites and embryonal histology. This favorable subset of patients has outcomes approaching those observed in selected patients with localized, nonmetastatic disease. Thus, these patients might not be appropriate candidates for regimens that include experimental agents with substantial toxicities or unproven antitumor activity.

AB - Purpose: To identify risk factors associated with outcomes in children with metastatic rhabdomyosarcoma (RMS) treated on the fourth Intergroup Rhabdomyosarcoma Study (IRS-IV). Patients and Methods: Patients with metastatic RMS were treated with one of two regimens that incorporated a window of either ifosfamide and etoposide (IE) with vincristine, dactinomycin, and cyclophosphamide (VAC) or vincristine, melphalan (VM) and VAC. Study end points were failure-free survival (FFS) and overall survival (OS). Clinical factors including age, histology, sites of primary and metastatic disease, and number of sites of metastatic disease were correlated with those end points. Results: One hundred twenty-seven patients were eligible for analysis. The estimated 3-year OS and FFS for all patients were 39% and 25%, respectively. By univariate analysis, 3-year OS was significantly influenced by histology (47% for embryonal v 34% for all others, P = .026) and increasing number of metastatic sites (P =.028). By multivariate analysis, the presence of two or fewer metastatic sites was the only significant predictor (P = .007 and .006, respectively). The combination of embryonal histology with two or fewer metastatic sites identified a subgroup with 3-year FFS of 40% and OS of 47%. Conclusion: Children with group IV RMS treated on the IRS-IV study had improved OS and FFS if they had two or fewer metastatic sites and embryonal histology. This favorable subset of patients has outcomes approaching those observed in selected patients with localized, nonmetastatic disease. Thus, these patients might not be appropriate candidates for regimens that include experimental agents with substantial toxicities or unproven antitumor activity.

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