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TABLE XXIII.A.3 (Continued)

Clinical endpoints

Study

Year LOE Study Design Study groups

Conclusions

DFS

1. 5-year DFS 47% 2. 94% of PM-RMSs stage 3

Koscielniak et al. 1248

1992 3

Prospective cohort

VACA, adjuvant

treatment based on response VACA or VAIA( n = 34) in PM-RMS IRSG 2 and3

Abbreviations: CEV, carboplatin + epirubicin + vincristine; CEVAIE, carboplatin + epirubicin + vincristine + actinomycin-D + ifosfamide + etoposide; CR, complete response rate; DFS, disease-free survival; DP, doxorubicin + cisplatin; EFS, event-free survival; FFS, failure- free survival; IVA/VAI, ifosfamide + vin cristine + actinomycin D; IVAd, ifosfamide + vincristine + doxorubicin; IVE, ifosfamide + vincristine + etoposide; PFS, progression-free survival; VACA, vincristine + dactinomycin + cyclophosphamide + doxorubicin; VAIA, vincristine + dactinomycin + ifosfamide + doxorubicin. a Five-year OS values measured from figure survival curves. b High-risk PM-RMS defined as skull base erosion, cranial nerve palsy, or intracranial extension.

OS of 73%). 1224 However, more recent data suggest a pos sible benefit in DMFS. 1238 The survival difference may be attributable to a higher ratio of adult patients with more aggressive histology, though this was not analyzed in the study. One additional study showed complete response in a limited number of patients who had an R0 surgical resection (Table XXIII.A.4). Role of surgery in adult rhabdomyosarcoma

Role of chemotherapy in pediatric rhabdomyosarcoma

Aggregate grade of evidence

B (Level 2: six studies; Level 3: nine studies)

Benefit

Gradual improvement in survival in more recent studies with VAC or VAI protocol. Chemotherapy side effects including pancytopenia and stomatitis. Some studies show higher rates of grade 3 and 4 toxicities with more aggressive chemotherapy regiments. Cost of chemotherapy administration. Preponderance of benefits over harms. There are no direct comparisons between chemotherapy and nonchemotherapy treatments. Failure to show survival benefit with addition of intrathecal chemotherapy.

Harm

Aggregate grade of evidence

C (Level 4: six studies)

Cost

Benefit

Comparable survival between surgical and nonsurgical approaches. Risk of surgical complications including anesthetic risks, blood loss, infection, CSF leak, and orbital injury. Potential disfiguring surgery for locally advanced cases. Additional costs of surgery, perioperative care, and long-term postoperative care. Patients treated with upfront surgery or surgery alone are likely to be highly selected for less aggressive, resectable tumors. Most studies do not differentiate between upfront and salvage surgery. Balance of benefits and harms.

Benefits–harm assessment

Harm

Value

judgments

Cost

Policy level Recommendation Intervention Administer VAC- or VAI-based chemotherapy

Benefits–harm assessment

protocols in treatment of sinonasal RMS. Intrathecal chemotherapy for sinonasal RMS is not recommended.

Value

judgments

5 Role of surgery in adult rhabdomyosarcoma

Policy level Option. Intervention May consider surgery in highly selected patients with resectable tumors and in salvage setting.

Six studies were identified that included surgery treat ment of adult RMS patients. All were retrospective and included both pediatric and adult patients. In three stud ies, there was no difference in outcomes between surgical and nonsurgical management options. 119,1235,1236 The tim ing of surgery was not clear, and surgical selection criteria were not available. Li et al. described their experience in a primarily adult population. 1237 The 5-year OS for this cohort was 46.5%. Notably, the survival for these patients is worse than that reported in IRS clinical trials (5-year

6 Role of chemoradiation therapy in adult rhabdomyosarcoma Only one study was identified that examined RT con trol rates prior to 2005 (pre-IMRT) and after 2005 (IMRT era) for nonmetastatic head and neck RMS. No patients