A B S T R A C T PurposeSome patients with rhabdomyosarcoma (RMS) achieve less than a complete response (CR) despite receiving all planned therapy. We assessed the impact of best response at the completion of all therapy on patient outcome. Patients and MethodsWe studied 419 clinical group III participants who completed all protocol therapy without developing progressive disease for Intergroup Rhabdomyosarcoma Study (IRS) IV. Response (complete resolution [CR], partial response [PR; Ն 50% decrease], or no response [NR; Ͻ 50% decrease and Ͻ 25% increase]) was determined by radiographic measurement and categorized by the best response. ResultsAt the end of therapy, 341 participants (81%) achieved a best response of CR and 78 (19%) had a best response of PR/NR. Five-year failure-free survival was similar for participants achieving CR (80%) and PR/NR (78%). After adjustment for age, nodal status, primary site, and histology, there was no significant indication of lower risk of failure (hazard ratio [HR], 0.77; 95% CI, 0.46 to 1.27; P ϭ .3) nor death (HR, 0.63; 95% CI, 0.36 to 1.09; P ϭ .1) for CR versus PR/NR participants. Seventeen participants with a best response of PR/NR had surgical procedures; eight (50%) of 16 with available pathology reports had residual viable tumor and only three achieved a complete resection. Resection of residual masses was not associated with improved outcome. ConclusionCR status at the end of protocol therapy in clinical group III participants was not associated with a reduction of disease recurrence and death. Aggressive alternative therapy may not be warranted for RMS patients with a residual mass at the end of planned therapy.

Significant histologic liver disease is common in cystic fibrosis, although the exact nature of the lesion cannot be predicted without liver biopsy. A clinical liver score that was developed for this may be useful in determining which patients require more definitive evaluation.