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Tuyển tập báo cáo các nghiên cứu khoa học quốc tế ngành y học dành cho các bạn tham khảo đề tài: Patient-recorded outcome to assess therapeutic efficacy in protoporphyria-induced dermal phototoxicity: a proposal | Minder et al. Health and Quality of Life Outcomes 2010 8 60 http www.hqlo.eom content 8 1 60 I HEALTH AND QUALITY OF LIFE OUTCOMES RESEARCH Open Access Patient-recorded outcome to assess therapeutic efficacy in protoporphyria-induced dermal phototoxicity a proposal Elisabeth I Minder 1 Xiaoye Schneider-Yin1 and Christoph E Minder2 Abstract Background Protoporphyria PP resulting from two rare inherited diseases of heme biosynthesis leads to dermal phototoxicity by accumulation of the heme precursor protoporphyrin IX. No standardized tools to quantify the degree of PP-related phototoxicity and its change by medical intervention have been published. Methods Results from a questionnaire completed by 17 affected individuals were used to determine the relative importance of two main components of PP-related phototoxicity skin pain and sunlight exposure time with respect to the effectiveness of any particular medical treatment. Results Inter-rater reliability was 0.71 n 490 repeated estimates by four identical individuals showed high reproducibility Slope 1 intercept 0 n 136 Passing-Bablock . Six different models were developed three of them showed good correlation with effectiveness estimates. Data from an unpublished trial indicated that the model with highest potential of responsiveness was the so called Exposure times multiplied by Freedom from Pain ETFP . The minimal clinically important difference MID was 15 10.2-20.4 ETFP scores representing 28 of the standard deviation of the clinical trial data and 2.9 of its total range. Conclusions Among the six models proposed to assess the effectiveness of therapeutic interventions in PP the ETFP model demonstrates the highest sensitivity using the existing data from a clinical trial of afamelanotide in PP The results of this study have provided sufficient validation of the ETFP model that is likely to prove useful in future clinical trials. Background Erythropoietic protoporphyria EPP OMIM 177000 a rare inherited disease of
