Safety and impact of eculizumab withdrawal in patients with atypical haemolytic uraemic syndrome: protocol for a multicentre, open-label, prospective, single-arm study.

INTRODUCTION: Atypical haemolytic uraemic syndrome (aHUS) is a rare, life-threatening disease caused by excessive activation of part of the immune system called complement. Eculizumab is an effective treatment, controlling aHUS in 90% of patients. Due to the risk of relapse, lifelong treatment is currently recommended. Eculizumab treatment is not without problems, foremost being the risk of severe meningococcal infection, the burden of biweekly intravenous injections and the high cost.This paper describes the design of the Stopping Eculizumab Treatment Safely in aHUS trial that aims to establish whether a safety monitoring protocol, including the reintroduction of eculizumab for those who relapse, could be a safe, alternative treatment strategy for patients with aHUS. METHODS AND ANALYSIS: This is a multicentre, non-randomised, open-label study of eculizumab withdrawal with continuous monitoring of thrombotic microangiopathy-related serious adverse events using the Bayes factor single-arm design. 30 patients will be recruited to withdraw from eculizumab and have regular blood and urine tests for 24 months, to monitor for disease activity. If relapse occurs, treatment will be restarted within 24 hours of presentation. 20 patients will remain on treatment and complete health economic questionnaires only. An embedded qualitative study will explore the views of participants. ETHICS AND DISSEMINATION: A favourable ethical opinion and approval was obtained from the North East-Tyne & Wear South Research Ethics Committee. Outcomes will be disseminated via peer-reviewed articles and conference presentations. TRIAL REGISTRATION NUMBER: EudraCT number: 2017-003916-37 and ISRCTN number: ISRCTN17503205.

A randomized questionnaire-based study on the impact of providing numerical information on colorectal cancer screening.

OBJECTIVES: To establish whether the provision of numerical data using pictograms and framed as event rates affects subjects' attitudes to colorectal cancer (CRC) screening. DESIGN: Randomized questionnaire and telephone study comparing a control group given standard NHS CRC information leaflets with an intervention group given the same leaflet but enhanced with additional numerical and pictorial information. SETTING: District General hospital and two general practices in North East England. Study carried out immediately prior to the introduction of CRC screening. PARTICIPANTS: A total of 478 non-gastroenterological subjects (age range 60-70 years). MAIN OUTCOME MEASURES: The difference in the two groups' overall wish to be screened; comparison of the impact of enhanced vs. unenhanced summary points in the NHS information leaflet; the summary point that most influenced their decision on screening; the views of the intervention group on the additional numerical and pictorial information provided. RESULTS: A total of 256 (54%) responded (124 from the control group and 117 from the intervention group); 22% were interviewed by telephone; 90% of the control group and 85% of the intervention group wished to be screened (P = 0.34). Provision of numerical and pictorial information significantly changed the impact of five of the six summary points on the decision to be screened. Sixty-two percent of the intervention group found the pictograms helpful while 83% of those interviewed by telephone found the numerical data helpful; 73% of the control group when given by telephone the additional numerical information given to the intervention group said this would have been useful in aiding their decision-making. CONCLUSION: Providing additional numerical information would enhance the credibility of the screening programme without necessarily reducing the numbers screened.