Abstract | PURPOSE: Patients with metastatic rhabdomyosarcoma (RMS), except those younger than 10 years with embryonal RMS, have an estimated long-term event-free survival (EFS) of less than 20%. The main goal of this study was to improve outcome of patients with metastatic RMS by dose intensification with interval compression, use of the most active agents determined in phase II window studies, and use of irinotecan as a radiation sensitizer. PATIENTS AND METHODS: RESULTS: One hundred nine eligible patients were enrolled, with a median follow-up of surviving patients of 3.8 years (3-year EFS for all patients, 38% [95% CI, 29% to 48%]; survival, 56% [95% CI, 46% to 66%]). Patients with one or no Oberlin risk factor (age > 10 years or < 1 year, unfavorable primary site of disease, ≥ three metastatic sites, and bone or bone marrow involvement) had a 3-year EFS of 69% (95% CI, 52% to 82%); high-risk patients with two or more risk factors had a 3-year EFS of 20% (95% CI, 11% to 30%). Toxicity was similar to that on prior RMS studies. CONCLUSION: Patients with metastatic RMS with one or no Oberlin risk factor had an improved 3-year EFS of 69% on ARST0431 compared with an historical cohort from pooled European and US studies; those with two or more risk factors have a dismal prognosis, and new approaches are needed for this very-high-risk group.
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Authors | Brenda J Weigel, Elizabeth Lyden, James R Anderson, William H Meyer, David M Parham, David A Rodeberg, Jeff M Michalski, Douglas S Hawkins, Carola A S Arndt |
Journal | Journal of clinical oncology : official journal of the American Society of Clinical Oncology
(J Clin Oncol)
Vol. 34
Issue 2
Pg. 117-22
(Jan 10 2016)
ISSN: 1527-7755 [Electronic] United States |
PMID | 26503200
(Publication Type: Clinical Trial, Journal Article, Research Support, N.I.H., Extramural)
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Copyright | © 2015 by American Society of Clinical Oncology. |
Chemical References |
- Vincristine
- Etoposide
- Irinotecan
- Doxorubicin
- Cyclophosphamide
- Ifosfamide
- Camptothecin
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Topics |
- Adolescent
- Adult
- Antineoplastic Combined Chemotherapy Protocols
(administration & dosage)
- Camptothecin
(administration & dosage, analogs & derivatives)
- Chemoradiotherapy
(adverse effects)
- Child
- Child, Preschool
- Cyclophosphamide
(administration & dosage)
- Disease Progression
- Doxorubicin
(administration & dosage)
- Drug Administration Schedule
- Etoposide
(administration & dosage)
- Feasibility Studies
- Female
- Follow-Up Studies
- Humans
- Ifosfamide
(administration & dosage)
- Infant
- Irinotecan
- Lymphatic Metastasis
- Male
- Middle Aged
- Patient Selection
- Radiotherapy, Adjuvant
- Rhabdomyosarcoma
(drug therapy, pathology, radiotherapy)
- Risk Assessment
- Risk Factors
- Severity of Illness Index
- Treatment Outcome
- Vincristine
(administration & dosage)
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