aHUS registry

Disease Definition



  • aHUS is a chronic and progressive disease. It is characterized by the development of TMA, which leads to systemic organ failure1
  • TMA comprises thrombosis, inflammation, and occlusion of microvasculature throughout the body, leading to ischemia and organ failure1
  • TMA is defined by its clinical characteristics1:
  • Decreased platelet count
  • Evidence of organ impairment/damage (e.g. serum creatinine > upper limit of normal (ULN))
  • Evidence off microangiopathic hemolysis (e.g. raised lactate dehydrogenase (LDH), decreased haptoglobin, schistocytes)
  • 30-40% of patients progress to end-stage renal disease (ESRD) or die with the first clinical manifestation despite plasma exchange or plasma infusion (PE/PI)2,3
  • 65% of all patients die, require dialysis, or have permanent renal damage within the first year after diagnosis despite plasma exchange/plasma infusion (PE/PI)2

1. Schmidtko J et al. Am J Kidney Dis 2013; 61: 289-299.
2. Caprioli J et al. Blood 2006; 108: 1267-1279.
3. Noris M et al. Clin J Am Soc Nephrol 2010;5:1844-1859

Incidence and prevalence of the disease



  • Incidence and prevalence1,2:
  • Very few sources of data are available regarding the incidence and prevalence of aHUS1
  • Incidence: 0.11-2 cases/million (as estimated in Europe and the United States)1
  • Prevalence: up to 3.3 cases/million/year (as estimated in Europe)1
  • At least 50% of patients have underlying inherited and/or acquired complement abnormality2
  • Gender distribution is similar (slight predominance in females when the disease appears later in life)1

1. Campistol JM, et al. Nefrologia. 2015;35(2):421-447
2. Goodship THJ, et al. Kidney International. 2017;91: 539-551



Diagnosis


Differential Diagnosis for TMA: aHUS, TTP, and STEC-HUS Laurence J et al. Clinical Advances in Hematology & Oncology. 2016; 14(11) Sup11. 1-15

Acronyms
TMA – Thrombotic microangiopathy
aHUS – atypical Hemolytic Uremic Syndrome
TTP – Thrombotic thrombocytopenic purpura
STEC-HUS - Shiga toxin-producing Escherichia coli – Hemolytic Uremic Syndrome