National observational study to monitor the new guideline concerning treatment of atypical hemolytic uremic syndrome

Atypical hemolytic uremic syndrome (aHUS) is a rare, but severe form of thrombotic

microangiopathy, and is considered the consequence of complement dysregulation. Atypical HUS has

a poor outcome with mortality up to 10% and over 50% of patients developing end stage renal

disease. Since the end of 2012, these outcomes have greatly improved with the introduction of

eculizumab.

The European Medicines Agency has approved eculizumab for the treatment of aHUS

patients. The guideline advocates lifelong treatment. However, there is no hard evidence to support

this advice. Historically, a substantial number of aHUS patients were weaned of plasma therapy,

often without disease recurrence. Moreover, the long-term consequences of eculizumab treatment

are unknown.

Recently, a new guideline concerning therapy in aHUS patients and hereby addressing

therapy adjustment and/or discontinuation in aHUS patients is implemented in the Netherlands. In

this national, multicenter, observational study we will include all aHUS, both pediatric and adults,

patients who are treated conform this new guideline. During four years this guideline will be

monitored and evaluated.

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Observational study for two years

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