Rhabdomyosarcoma (RMS) is rare in adults and it is generally characterized by poor outcome. In a previous retrospective study, we demonstrated a better prognosis in adults treated with multimodality approach resembling pediatric protocols. Thereafter, we developed specific recommendations based on the principles adopted in pediatric oncology. The present analysis reports the results in a subsequent prospective series. The study included 95 consecutive patients (age 18–77 years) treated from 2002 to 2015 for embryonal and alveolar RMS. As in the previous series, patients were stratified by the appropriateness of their treatment according to therapeutic guidelines for childhood RMS. The 5-year event-free survival (EFS) and overall survival (OS) rates were 33.6% and 40.3%, respectively. The 5-year EFS was 40.8% for patients with the highest treatment score, and 15% for those with lower score, while OS was 44.4% and 24.5%, respectively. The developing of specific recommendations enabled an increase in the number of patients treated with intensive multimodal treatment resembling pediatric strategy (69.7% vs. 39.1% in the retrospective series). This study reinforced the idea that adherence to the principles of pediatric protocols, improves adult RMS outcomes. However, treating adults with pediatric-type strategy is not enough to achieve the results obtained in children. Issues in compliance and a more aggressive biology of adult RMS might have a role in the different outcome according to age. Improving the collaboration between pediatric and adult oncologists in promoting specific clinical and biological research is crucial to improve the outcome for this patient population.
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This research did not receive any specific grant from funding agencies in the public, commercial or not-for-profit sectors.
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Conflict of interest
The authors declare that they have no conflict of interest to disclose.
This study was performed in accordance with the Declaration of Helsinki. The project was approved by the Research Ethical Committee of the Fondazione IRCCS Istituto Nazionale Tumori, Milan, Italy (code 150053 of the Italian National Observatory on Clinical Trials). All the patients signed a written informed consent for their involvement in the project and for the use of their personal data.
Weiss SW, Goldblum J. Rhabdomyosarcoma. In: Weiss SW, Goldblum J, editors. Enzinger and Weiss’s soft tissue tumors. St. Louis: CV Mosby; 2001. p. 785–835.Google Scholar
Fletcher CDM, Bridge JA, Hogendoorn PCW, Mertens F. WHO classification of tumours of soft tissue and bone. 4th ed. Lyon: IARC Press; 2013.Google Scholar
Sultan I, Qaddoumi I, Yaser S, Rodriguez-Galindo C, Ferrari A. Comparing adult and pediatric rhabdomyosarcoma in the surveillance, epidemiology and end results program, 1973 to 2005: an analysis of 2600 patients. J Clin Oncol. 2009;27(20):3391–7.CrossRefGoogle Scholar
Ferrari A, Casanova M. Current chemotherapeutic strategies for rhabdomyosarcoma. Expert Rev Anticancer Ther. 2005;5(2):283–94.CrossRefGoogle Scholar
Esnaola NF, Rubin BP, Baldini EH, et al. Response to chemotherapy and predictors of survival in adult rhabdomyosarcoma. Cancer. 2001;91:794–803.CrossRefGoogle Scholar
Hawkins WG, Hoos A, Antonescu CR, et al. Clinicopathologic analysis of patients with adult rhabdomyosarcoma. Cancer. 2001;91:794–803.CrossRefGoogle Scholar
Little DJ, Ballo MT, Zagars GK, et al. Adult rhabdomyosarcoma: outcome following multimodality treatment. Cancer. 2002;95:377–88.CrossRefGoogle Scholar
Van Gaal JC, Van der Graaf WT, et al. The impact of age on outcome of embryonal and alveolar rhabdomyosarcoma patients. A multicenter study. Anticancer Res. 2012;32(10):4485–97.PubMedGoogle Scholar
Dumont SN, Araujo DM, Munsell MF, et al. Management and outcome of 239 adolescent and adult rhabdomyosarcoma patients. Cancer Med. 2013;2(4):553–63.CrossRefGoogle Scholar
Gerber NK, Wexler LH, Singer S, et al. Adult rhabdomyosarcoma survival improved with treatment on multimodality protocols. Int J Radiat Oncol Biol Phys. 2013;86(1):58–63.CrossRefGoogle Scholar
Fischer TD, Gaitonde SG, Bandera BC, et al. Pediatric-protocol of multimodal therapy is associated with improved survival in AYAs and adults with rhabdomyosarcoma. Surgery. 2018;163(2):324–9.CrossRefGoogle Scholar
Ferrari A, Dileo P, Casanova M, et al. Rhabdomyosarcoma in adults: a retrospective analysis of 171 patients treated at a single institution. Cancer. 2003;98:571–80.CrossRefGoogle Scholar
Harmer MH. TNM Classification of pediatric tumors. Geneva: International Union Against Cancer; 1982. p. 23–8.Google Scholar
Maurer HM, Beltangady M, Gehan EA, et al. The intergroup rhabdomyosarcoma study-I. A final report. Cancer. 1988;61(2):209–20.CrossRefGoogle Scholar
Therasse P, Arbuck SG, Eisenhauer EA, et al. New guidelines to evaluate the response to treatment in solid tumors (RECIST Guidelines). J Natl Cancer Inst. 2000;92:205–16.CrossRefGoogle Scholar
Eisenhauer EA, Therasse P, Bogaerts J, et al. New response evaluation criteria in solid tumours: revised RECIST guideline (version 1·1). Eur J Cancer. 2009;45:228–47.CrossRefGoogle Scholar
Kaplan E, Meier P. Non-parametric estimation from incomplete observation. J Am Stat Assoc. 1958;53:457–81.CrossRefGoogle Scholar
Conover WJ. Practical nonparametric statistics. New York: John Wiley & Sons; 1980. p. 153–69.Google Scholar
Cox DR. Regression models and life tables. J R Stat SocB. 1972;34:187–220.Google Scholar
Ferrari A, Miceli R, Meazza C, et al. Comparison of the prognostic value of assessing tumor diameter versus tumor volume at diagnosis or in response to initial chemotherapy in rhabdomyosarcoma. Clin Oncol. 2010;28:1322–8.CrossRefGoogle Scholar
Oberlin O, Rey A, Lyden E, et al. Prognostic factors in metastatic rhabdomyosarcomas: results of a pooled analysis from United States and European Cooperative Groups. J Clin Oncol. 2008;26:2384–9.CrossRefGoogle Scholar
Gupta AA, Anderson JR, Pappo AS, et al. Patterns of chemotherapy-induced toxicities in younger children and adolescents with rhabdomyosarcoma: a report from the Children’s Oncology Group Soft Tissue Sarcoma Committee. Cancer. 2012;118(4):1130–7.CrossRefGoogle Scholar
Kojima Y, Hashimoto K, Ando M, et al. Comparison of dose intensity of vincristine, d-actinomycin, and cyclophosphamide chemotherapy for child and adult rhabdomyosarcoma: a retrospective analysis. Cancer Chemother Pharmacol. 2012;70(3):391–7.CrossRefGoogle Scholar
Davicioni E, Anderson R, Buckley JD, et al. Gene expression profiling for survival prediction in pediatric rhabdomyosarcomas: a report from the Children’s Oncology Group. J Clin Oncol. 2010;28(7):1240–6.CrossRefGoogle Scholar
Missiaglia E, Williamson D, Chisholm J, et al. PAX3/FOXO1 fusion gene status is the key prognostic molecular marker in rhabdomyosarcoma and significantly improves current risk stratification. J Clin Oncol. 2012;30(14):1670–7.CrossRefGoogle Scholar
Parham DM, Barr FG. Classification of rhabdomyosarcoma and its molecular basis. Adv Anat Pathol. 2013;20:387–97.CrossRefGoogle Scholar
Shern JF, Chen L, et al. Comprehensive genomic analysis of rhabdomyosarcoma reveals a landscape of alterations affecting a common genetic axis in fusion-positive and fusion-negative tumors. Cancer Discov. 2014;4(2):216–31.CrossRefGoogle Scholar