7 Recommendations for further research

7.1 The Committee recommended the use of eculizumab for atypical haemolytic uraemic syndrome (aHUS) only if all the following arrangements are in place:

  • coordination of eculizumab use through an expert centre

  • monitoring systems to record the number of people with a diagnosis of atypical haemolytic uraemic syndrome and the number who have eculizumab, and the dose and duration of treatment

  • a national protocol for starting and stopping eculizumab for clinical reasons

  • a research programme with robust methods to evaluate when stopping treatment or dose adjustment might occur.

    These arrangements include, but are not exclusive to, determining: which patient characteristics allow safe treatment withdrawal after an initial response; whether the patient's disease responds to retreatment with eculizumab if it has relapsed after stopping treatment; and whether the drug dose can be titrated to a marker of response, for example, platelet count, as well as data relating to patient experience of the value offered by eculizumab.

7.2 There is a need for further evidence on the long‑term outcomes of treatment with eculizumab, and on the effect of treatment in children, adolescents and during pregnancy. The Committee supports enrolment of patients into the aHUS registry and the collection of high‑quality information from these registries periodically to inform treatment decisions.