Linda X Yin1, Douglas D Reh2, Jeffrey B Hoag3, Sally E Mitchell4, Stephen C Mathai5, Gina M Robinson4, Christian A Merlo5. 1. Johns Hopkins School of Medicine, Johns Hopkins Medicine, Baltimore, Maryland. 2. Department of Otolaryngology/Head and Neck Surgery, Johns Hopkins Medicine, Baltimore, Maryland. 3. Division of Pulmonary, Critical Care, and Sleep Medicine, Drexel Medicine, Philadelphia, Pennsylvania, U.S.A. 4. Department of Radiology and Radiological Science, Johns Hopkins Medicine, Baltimore, Maryland. 5. Department of Medicine-Division of Pulmonary and Critical Care Medicine, Johns Hopkins Medicine, Baltimore, Maryland.
Abstract
OBJECTIVES/HYPOTHESIS: Hereditary hemorrhagic telangiectasia (HHT) is a disease of abnormal angiogenesis, causing epistaxis in over 96% of patients. The Epistaxis Severity Score (ESS) was developed as a standardized measurement of nasal symptoms among HHT patients. The minimal important difference (MID) of a disease index estimates the smallest change that a patient and clinician would identify as important. This study aims to establish the MID of the ESS in a diverse population of HHT patients. STUDY DESIGN: Retrospective cross-sectional study in patients with a diagnosis of HHT using Curacao criteria or genetic testing. METHODS: The ESS questionnaire and Medical Outcomes Study 36-Item Short Form (SF-36) were administered to participants recruited through the HHT Foundation Web site. Demographics and relevant medical histories were collected from all participants. An anchor-based method using a change of 5 in the Physical Component Summary (PCS) of the SF-36 and a distributional method were used to estimate the MID. RESULTS: A total of 604 subjects were recruited between April and August 2008. All participants reported epistaxis. An increasing ESS in the study cohort showed a significant negative correlation to the PCS (r = -0.43, P < 0.001). The MID was determined to be 0.41 via the anchor-based approach and 1.01 via the distribution-based approach, giving a mean MID of 0.71. CONCLUSION: Using both the anchor-based and distribution-based approaches, the estimated MID for the ESS in HHT is 0.71. Further implications include key metrics to help guide treatment responses in clinical care and essential information to calculate power and sample size for future clinical trials. LEVEL OF EVIDENCE: 4. Laryngoscope, 126:1029-1032, 2016.
OBJECTIVES/HYPOTHESIS: Hereditary hemorrhagic telangiectasia (HHT) is a disease of abnormal angiogenesis, causing epistaxis in over 96% of patients. The Epistaxis Severity Score (ESS) was developed as a standardized measurement of nasal symptoms among HHTpatients. The minimal important difference (MID) of a disease index estimates the smallest change that a patient and clinician would identify as important. This study aims to establish the MID of the ESS in a diverse population of HHTpatients. STUDY DESIGN: Retrospective cross-sectional study in patients with a diagnosis of HHT using Curacao criteria or genetic testing. METHODS: The ESS questionnaire and Medical Outcomes Study 36-Item Short Form (SF-36) were administered to participants recruited through the HHT Foundation Web site. Demographics and relevant medical histories were collected from all participants. An anchor-based method using a change of 5 in the Physical Component Summary (PCS) of the SF-36 and a distributional method were used to estimate the MID. RESULTS: A total of 604 subjects were recruited between April and August 2008. All participants reported epistaxis. An increasing ESS in the study cohort showed a significant negative correlation to the PCS (r = -0.43, P < 0.001). The MID was determined to be 0.41 via the anchor-based approach and 1.01 via the distribution-based approach, giving a mean MID of 0.71. CONCLUSION: Using both the anchor-based and distribution-based approaches, the estimated MID for the ESS in HHT is 0.71. Further implications include key metrics to help guide treatment responses in clinical care and essential information to calculate power and sample size for future clinical trials. LEVEL OF EVIDENCE: 4. Laryngoscope, 126:1029-1032, 2016.
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