Acquired immunodeficiency in ANCA associated vasculitis

  • Research type

    Research Study

  • Full title

    ACQUIVAS - Acquired immunodeficiency in ANCA associated vasculitis (AAV)

  • IRAS ID

    234724

  • Contact name

    Rona Smith

  • Contact email

    rms@medschl.cam.ac.uk

  • Sponsor organisation

    Cambridge University Hospital NHS Foundation Trust and University of Cambridge

  • Eudract number

    2017-003563-36

  • Duration of Study in the UK

    2 years, 7 months, 1 days

  • Research summary

    Research Summary

    Vaccines are recommended for patients with ANCA associated vasculitis (AAV) to reduce the risk of common infections such as flu or pneumonia. However, the medications used to treat vasculitis, particularly rituximab, may reduce the effectiveness of these vaccines. This study will measure responses to vaccines after rituximab, and look at possible ways to improve responses, by using different forms of the vaccine and boosting strategies. Improving vaccine responses may have a direct impact on patient outcomes, as poor vaccine responses have been shown to increase the risk of death in this patient group.

    Summary of Results

    Acquired immunodeficiency in ANCA Associated vasculitis (ACQUIVAS)

    Who carried out the research?
    The trial was sponsored by Cambridge University Hospitals NHS Foundation Trust (CUH) and The University of Cambridge.

    What public involvement there was in the study?
    Recruited patients all had a diagnosis of ANCA associated vasculitis and were receiving immunosuppression, either rituximab or other agents, such as azathioprine or mycophenolate mofetil after initial cyclophosphamide treatment (control group). Data from 52 patients in the rituximab treated group and 46 patients in the control group are included in the primary endpoint analysis.

    Was there any patient peer review?
    The study was funded by Versus Arthritis (formerly Arthritis Research UK) which includes patient members on their grant review panels.
    Where and when the study took place. Who participated in the study?
    Two sites took place in the study. Cambridge University Hospitals NHS Foundation Trust (Sponsor site) and University Hospitals Birmingham NHS Foundation Trust.

    Why was the research needed?
    Vasculitis can be very serious if left untreated. Usual treatment involves suppressing the immune system which can put patients at increased risk of infections. Consequently, it is recommended that patients with ANCA associated vasculitis (AAV) receive vaccinations against influenza (flu) and pneumococcus (a bacterium which causes pneumonia and meningitis).

    However, medications used to treat vasculitis, particularly rituximab, may reduce the effectiveness of these vaccines, meaning that patients are not protected against these infections. This trial measures responses to vaccines after patients with AAV have received rituximab therapy, and looks at possible ways to improve these, by using different forms of the vaccine and boosting strategies and comparing data with AAV patients who have never received rituximab and healthy controls.

    What were the main questions studied? What treatments or interventions did the participants take/receive?
    Hypothesis: Rituximab therapy leads to an acquired immune deficiency, as demonstrated by impaired vaccine responses, in AAV patients.

    Aims:
    1. To investigate whether rituximab leads to immune deficiency, and poorer vaccine responses, in patients with AAV when compared to both disease and healthy controls
    2. To investigate whether the degree of immune deficiency is associated with the degree of B cell depletion
    3. To investigate whether T-independent vaccine responses are more severely affected than T-dependent vaccine responses after rituximab and whether a conjugated vaccine will overcome this postulated deficit in T independent vaccine responses ie. Can the boost strategy employed in this study overcome the predicted sub-optimal vaccine responses?

    Participants received one dose of Pneumococcal Polysaccharide Conjugate Vaccine (Prevnar 13) at Month 0 and the one dose of a different Pneumococcal Polysaccharide vaccine (Pneumovax) at Month 6 unless participants were vaccinated within 5 years of screening in which case they only received the Month 0 dose.
    Blood samples were taken at baseline and day 28 to measure antibodies to 12 of the serotypes in the pneumococcal vaccine, with response defined as a 2 fold increase in antibody levels in at least 50% of serotypes tested. Samples were also taken before the boost vaccine at month 6 and again 28 days later to see if this approach improved response.

    What medical problems (adverse reactions) did the participants have?
    Only eight serious adverse events were recorded in the trial. All were deemed unlikely related or not related to the vaccine. Events included infections (lower respiratory, pneumonia and gall bladder), pulmonary fibrosis, raised inflammatory markers, vasculitis flare and osteoarthritis. All these events are to be expected in a group of patients with ANCA associated vasculitis who have received immunosuppression.

    What were the results of the study?
    Results of the study are expected later in 2024. Results will be presented at conferences, published in scientific journals and an article will be written for patient newsletters (Vasculitis UK) to disseminate the results to patients and an update will be made to clinicaltrials.gov when data is available.

    How has this study helped patients and researchers?
    Although results are still pending, this study remains highly relevant to immunocompromised patients and researchers. The COVID-19 pandemic has focused attention on the major unmet need to develop strategies to protect immunocompromised individuals because of their high risk of morbidity and mortality from infection, and by virtue of their immunocompromised state and sub-optimal responses to vaccination. Overall, despite vaccination, sub-optimal vaccine responses and subsequent vulnerability to infection remains a major issue for immunocompromised patients. As such, it is vital to understand vaccine responses not only in the general population, but also among immunocompromised populations. Detailed investigation into the immunologic mechanisms underlying responses to various immune challenges, particularly vaccination, longitudinally over time in this patient group may subsequently inform clinical approaches to protecting these vulnerable individuals. Understanding how different immunosuppressive therapies, immune-mediated diseases and vaccines interact and impact trajectories of immunity and responses to pathogens over time, may consequently inform the optimal type and timing of vaccine administration, vaccine-boosting strategies, and in which patients to prioritise other pre-exposure prophylactic approaches. Additionally, such an approach may also provide valuable mechanistic insights into underlying disease pathogenesis, and potentially identify novel therapeutic targets.

    Details of any further research planned
    Approval has been granted to the VARIED study (Chief investigator Dr Rona Smith; IRAS ID 328759), which brings together samples and clinical data from up to 5000 immunocompromised individuals, including patients recruited to the ACQUIVAS trial to facilitate ongoing research into understanding the immune response to vaccination in immunocompromised individuals. The overarching aim is to more accurately define the immune status of an individual in the context of their underlying health condition and immunosuppressive medication and to be able to risk stratify individuals in a more refined way and tailor approaches to infection prophylaxis.

  • REC name

    East of England - Cambridge East Research Ethics Committee

  • REC reference

    18/EE/0181

  • Date of REC Opinion

    30 Jul 2018

  • REC opinion

    Further Information Favourable Opinion