NORD Physician Guide to Atypical Hemolytic Uremic Syndrome (aHUS). The NORD Online Physician Guides are written for physicians by physicians with expertise on specific rare disorders. This guide was reviewed by Larry Greenbaum, MD, PhD, Division Director of Pediatric Nephrology, Marcus Professor of Pediatrics, Emory University School of Medicine and Children’s Healthcare of Atlanta (see acknowledgements for additional information).
NORD is a nonprofit organization representing all patients and families affected by rare diseases. The information NORD provides to medical professionals is intended to facilitate timely diagnosis and treatment for patients.
aHUS, a form of thrombotic microangiopathy associated with excess activation or dysregulation of the alternate pathway of complement, is a life-threatening disorder that can lead to ischemic injury and damage to any organ.
A diagnosis of aHUS is suspected based on the classic triad of hemolytic anemia, thrombocytopenia, and acute kidney injury.
aHUS can be genetic or acquired and onset is often in childhood.
Eculizumab, an antibody to the fifth component of complement, is widely considered the first-line therapy for most patients with a confirmed diagnosis of aHUS.
There is a report on the following related topic in NORD’s online Rare Disease Database for patients, families and the public at: