No abstract

Background Given the severe symptom burden and chronic nature of mycosis fungoides (MF) and S ezary syndrome (SS), effective assessment of quality of life (QoL) is essential to guiding patient-centred care in this population. In this study, we aim to provide a comprehensive assessment of QoL in early-and advanced-stage MF/SS and to assess the correlation of traditional measures of clinical severity with QoL measures. Methods Between July 2017 and April 2019, outpatients at an academic medical centre with either MF/SS (n = 115) or general dermatology concerns (n = 115) completed generic and dermatology-specific QoL instruments [Health Utilities Index Mark 3 (HUI3), RAND 36-Item Short-Form Health Survey (SF-36), Skindex-29, visual analogue scale for itch (VAS itch) and 5-D pruritus scale]. The mean scores of MF/SS patients were compared to that of controls using multivariable regression models adjusted for demographics and medical comorbidities. Cluster analysis of the QoL instruments and clinical severity measures (e.g. stage and body-surface-area involvement) was performed.Results Patients with MF/SS scored significantly worse than controls on all QoL instruments used, with advancedstage (IIB-IVB) disease having the worst QoL impairment. Early-stage (IA-IIA) and advanced-stage MF/SS patients had significantly reduced overall health status (HUI3; P < 0.05), with largest decrements in social functioning and usual role functioning due to physical and emotional health (SF-36; all P < 0.05). MF/SS had significantly worse skin-specific impairment than controls, with advanced-stage disease reporting the most severe skin-specific burden (Skindex-29, P < 0.05). Clinical severity measures had a weak correlation with generic (|r s | = 0.02-0.27) and moderate correlation with dermatology-specific instruments (|r s | = 0.41-0.53).Conclusions MF/SS have a significant impact on multiple domains of patients' QoL, including social, emotional and physical functioning. Current clinical measures do not adequately address QoL outcomes, underscoring the need for integrating formal disease-specific QoL assessment into the routine evaluation of MF/SS patients.

Conflict of interest: LAC and SD are co-inventors on a filed patent for the use of calcipotriol plus 5-fluorouracil for the treatment of precancerous skin lesions (PCT/US2015/049434).

Background The prognostic significance of response to induction therapy for rhabdomyosarcoma (RMS) by anatomic imaging [computerized tomographic (CT) or magnetic resonance imaging (MRI) scan] is controversial. We previously reported no relationship between early response and failure-free survival (FFS) on Intergroup Rhabdomyosarcoma Study IRS-IV. We repeated the same analysis using a more recent clinical trial as an independent cohort of patients with non-metastatic, initially unresected RMS. Methods A total of 338 patients enrolled in Children’s Oncology Group (COG) study D9803 met the inclusion criteria for this analysis: (1) non-metastatic, initially unresected (Group III); (2) embryonal (ERMS) or alveolar (ARMS) histology; (3) documented protocol week 12 response to induction chemotherapy (excluding progressive disease) based on anatomic imaging (CT/MRI); and (4) documented protocol therapy beyond week 12. Response at week 12 was determined by the treating institution as complete response (CR), partial response (PR), or no response (NR). FFS was estimated using the Kaplan-Meier method and comparisons between patient subsets were made using the log-rank test. Results Overall objective response rate (CR+PR) at week 12 of therapy was 85% and was similar between ERMS and ARMS. FFS was similar among all patients with CR, PR, or NR (p=0.49). Restricting the analysis to either ERMS or ARMS, there was no difference in FFS by response within either histology subset (p=0.89 and p=0.08, respectively). Conclusions These findings provide additional evidence that anatomic imaging to assess early response to therapy among patients with RMS does not predict outcome and has questionable use in tailoring subsequent therapy.