The alliance relationship with the atypical Hemolytic-Uremic Syndrome( aHUS) Registry (NCT01522183) is now well into its fifth year. This observational non interventional long term study has a “patient representative” on its Scientific Advisory Board. The Registry has featured in alliance news items on many occasions and a blog about it appeared in the first week of the launch of this website see here.
The Registry has reached the half way stage in its planned existence to study the long term impact of aHUS on patients including those who have never received eculizumab.
Although it was expected to recruit 2000 patients to the study, following a review of enrolled patients’ status in 2018, when patient numbers were approaching 1800, it was decided to rebase the Registry to focus on 1000 or so active patient participants and thereby improve the quality of collected data for analysis and research.
The review illustrated the problems faced by all patient Registries as an “attrition” process takes effect.
Although in the early stages recruitment is positive and growing patient numbers, in time it becomes clear that not all enrolled patients continue to be followed. There may be several reasons for this. Sometimes the original diagnosis of aHUS is found to be incorrect. The enrolling site may decide that continuing to provide data is not worth its while or may persuade the patient it may not be in their interest to continue and so become lost to follow up. Some patients may just ask to be removed having agreed to participate. Or pediatric patients may move on to adult care in non Registry approved sites. Sadly some patients may die. In some cases so few are enrolled at a sites that it becomes uneconomic to maintain the site.
Although Alexion are compelled to maintain a post marketing Registry as part of its license approval it does not necessarily mean that each country compels Alexion to include all patients, even those on eculizumab. But some do, notably France and Belgium. Recruitment in those countries must continue. Recruitment is also taking place in The Republic of Korea , Turkey and Polandcountries in which aHUS patients have more recently accessed eculizumab.
So in some countries recruitment can be stopped and only those already recruited will be actively followed up and data collection continued. The status of each of the 128 hospitals where patients have been enrolled can be seen by following this link here. If the viewer is a patient treated at any of these hospitals it is likely that they are enrolled in this Registry.
Although its primary purpose is to collect data about the safety of eculizumab and clinical manifestations of aHUS following treatment, patients are asked for information about how they are feeling and how the illness is impacting on their lives in questionnaires which are completed at six monthly intervals. This feedback will provide valuable insight about patients throughout, and at the end of, up to 10 years following enrollment. It is important for aHUS patients everywhere that those enrolled say what it is like being treated for, and living with, aHUS.
Having made a big decision to rebase the patient participants, the Registry also faces the impact of the transition of patients from eculizumab to ravulizumab and to prepare itself for perhaps a longer haul to completion to maximise the value of the output of this crucial study of aHUS patients.
