Validation of a novel patient reported tool to assess the impact of treatment in erythropoietic protoporphyria: the EPP-QoL

[1] Benjamin K(2017)Patient-reported outcome and observer-reported outcome assessment in rare disease clinical trials: An ISPOR COA emerging good practices task force report Value Health 20 838-855

[2] Vernon M(1994)Dermatology life quality index (DLQI)-a simple practical measure for routine clinical use Clin Exp Dermatol 19 210-216

[3] Patrick D(2006)Erythropoietic protoporphyria in the U.K.: Clinical features and effect on quality of life Br J Dermatol 155 574-581

[4] Finlay A(2005)Translating the science of quality of life into practice: What do dermatology life quality index scores mean? J Investig Dermatol 125 659-664

[5] Khan G(2008)The quality of life of 790 patients with photodermatoses Br J Dermatol 159 192-197

[6] Holme S(2009)Erythropoietic protoporphyria Orphanet J Rare Dis 4 19-900

[7] Anstey A(2000)Recommendations on health-related quality of life research to support labeling and promotional claims in the United States Qual Life Res 9 887-310

[8] Finlay A(2004)The World Health Organization's WHOQOL-BREF quality of life assessment: Psychometric properties and results of the international field trial. A Report from the WHOQOL Group Qual Life Res 13 299-766

[9] Hongbo Y(1994)Erythropoietic protoporphyria Br J Dermatol 131 751-483

[10] Thomas C(1992)The MOS 36-ltem short-form health survey (SF-36) Med Care 30 473-undefined

[1] Benjamin K(2017)Patient-reported outcome and observer-reported outcome assessment in rare disease clinical trials: An ISPOR COA emerging good practices task force report Value Health 20 838-855

[2] Vernon M(1994)Dermatology life quality index (DLQI)-a simple practical measure for routine clinical use Clin Exp Dermatol 19 210-216

[3] Patrick D(2006)Erythropoietic protoporphyria in the U.K.: Clinical features and effect on quality of life Br J Dermatol 155 574-581

[4] Finlay A(2005)Translating the science of quality of life into practice: What do dermatology life quality index scores mean? J Investig Dermatol 125 659-664

[5] Khan G(2008)The quality of life of 790 patients with photodermatoses Br J Dermatol 159 192-197

[6] Holme S(2009)Erythropoietic protoporphyria Orphanet J Rare Dis 4 19-900

[7] Anstey A(2000)Recommendations on health-related quality of life research to support labeling and promotional claims in the United States Qual Life Res 9 887-310

[8] Finlay A(2004)The World Health Organization's WHOQOL-BREF quality of life assessment: Psychometric properties and results of the international field trial. A Report from the WHOQOL Group Qual Life Res 13 299-766

[9] Hongbo Y(1994)Erythropoietic protoporphyria Br J Dermatol 131 751-483

[10] Thomas C(1992)The MOS 36-ltem short-form health survey (SF-36) Med Care 30 473-undefined