Soliris in atypical HUS

atypical HUS is a genetic, chronic, systemic, and life-threatening disease that affects both adults and children.1 It is caused by a genetic deficiency of natural complement inhibitors and is defined by the clinical characteristics of thrombotic microangiopathy (TMA), including thrombocytopenia, hemolysis, and organ dysfunction.1-5

atypical HUS has systemic impact: TMA occurs throughout the body and affects multiple vital organs, including the renal, nervous and cardiovascular systems.1,4,5,7,8 More than 60% of atypical HUS patients have at least one complication outside of the kidney, including neurological, cardiovascular, and gastrointestinal systems.8

The catastrophic consequences of atypical HUS make rapid diagnosis and clinical intervention critical.6,10 Due to the chronic nature of atypical HUS, all patients require continuous care. Patients surviving initial clinical manifestations remain at risk of progressive complications or sudden death.6 Supportive care approaches such as plasma exchange or plasma infusion (PE/PI) have not been shown to be either safe or effective in well-controlled clinical trials in patients with atypical HUS.5 Complement-mediated TMA persists despite PE/PI,9 dialysis,5,7,13-16 or kidney transplantation,17 resulting in extra-renal morbidities.8

Treating Patients Who Have atypical HUS with Soliris

Soliris is the only therapy approved for the treatment of atypical haemolytic uremic syndrome (atypical HUS), a life-threatening, ultra-rare disease in which a genetic deficiency in one or more complement regulatory genes causes chronic, uncontrolled complement activation that can result in sudden and progressive damage to vital organs, leading to stroke, heart attack, kidney failure and premature death. As a first-in-class treatment that specifically targets uncontrolled complement activation, Soliris represents a major step forward in the care of adults and children with aHUS. In clinical studies, 100 percent of patients had a reduction in terminal complement activity with sustained treatment with Soliris.

Soliris is approved in nearly 40 countries for the treatment of patients with atypical HUS. Soliris is indicated for the treatment of patients with atypical HUS to inhibit complement-mediated thrombotic microangiopathy (TMA), the formation of blood clots in small blood vessels throughout the body.

More Information

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atypical HUS Registry

Enroll your patients in the atypical HUS Registry today.

An observational, noninterventional, multicenter, multinational study of patients with atypical HUS

  • Collects and evaluates safety and effectiveness data for treatment options
  • Assesses long-term manifestations of TMA complications as well as other clinical outcomes, including morbidity and mortality, regardless of treatment approach

For more information, e-mail atypical HUSRegistry@Alxn.com, or contact your Alexion Representative

References:
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3. Fang CJ,Richards A, Liszewski MK, Kavanagh D, Atkinson JP. Br J Haematol. 2008;143:336-348.
4. Sallée M, Daniel L, Piercecchi MD, et al. Nephrol Dial Transplant. 2010;25:2028-2032.
5. Loirat C, Garnier A, Sellier-Leclerc AL, Kwon T. Semin Thromb Hemost. 2010;36:673-681.
6. Ståhl AL, Vaziri-Sani F, Heinen S, et al. Blood. 2008;111:5307-5315.
7. Neuhaus TJ, Calonder S, Leumann EP. Arch Dis Child. 1997;76:518-521.
8. Langman C. Presented at the 17th Congress of the European Hematology Association; June 14-17, 2012; Amsterdam, The Netherlands. Abstract 0490.
9. Caprioli J, Noris M, Brioschi S, et al; International Registry of Recurrent and Familial HUS/TTP. Blood. 2006;108:1267-1279.
10. Ariceta G, Besbas N, Johnson S, et al; European Paediatric Study Group for HUS. Pediatr Nephrol. 2009;24:687-696.
11. Data on File. Alexion Pharmaceuticals, Inc.
12. Mache CJ, Acham-Roschitz B, Frémeaux-Bacchi V, et al. Clin J Am Soc Nephrol. 2009;4:1312-1316.
13. Malina M, Gulati A, Majid MA, Bagga A, Schaefer F. Pediatr Nephrol. 2011;26:1678.
14. Larekeb A, Leroy S, Frémeaux-Bacchi V, et al. Pediatr Nephrol. 2007;22:1967-1970.
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16. Vergouwen MD, Adriani KS, Roos YB, Groothoff JW, Majoie CB. AJNR Am J Neuroradiol. 2008;29:e34.
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