A time for reappraisal of "atypical" hemolytic uremic syndrome: should all patients be treated the same?

Abstract

Atypical hemolytic uremic syndrome (HUS) refers to the triad of microangiopathic hemolytic anemia, thrombocytopenia, and acute kidney injury in the absence of Shiga toxin-producing Escherichia coli exposure or Streptococcus pneumoniae infection. Currently, approximately 50 % of the atypical cases have demonstrable mutations in complement regulatory proteins. Historically, the diagnosis of atypical HUS portends a poor prognosis with a high rate of disease recurrence, progression to end-stage renal disease, and death. However, it is now evident that atypical HUS actually encompasses a heterogeneous group of disorders, and there are reports suggesting that some cases of atypical HUS have a favorable prognosis, similar to that of diarrhea-associated disease. We present three patients with the atypical HUS phenotype who had complete renal recovery and no disease recurrence. We believe it is important to distinguish those cases of atypical HUS associated with disorders of complement regulatory proteins from other idiopathic causes of nondiarrheal HUS given the implications for prognosis and treatment.

Conclusion: Given the heterogeneous nature and variable prognosis of atypical HUS, treatment should be carefully considered prior to the use of long-term plasma therapy and/or eculizumab.