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January 23, 2016

"Treatment of a Patient with Dense Deposit Disease with Eculizumab (Soliris)," M. Vivarelli, F. Emma.Results were reported on a 17-year-old patient who was diagnosed with DDD with normal renal function but significant proteinuria (3-5 grams/24 hrs). To date, the patient has been treated with Soliris for 16 months. The reported results indicate that Soliris treatment was associated with a significant increase in serum protein and albumin, as well as a decrease in both the urine protein/creatinine ratio and 24 hour protein. His creatinine and blood pressure remain normal. The investigators reported no drug-related adverse effects while on Soliris over the 16-month treatment period and concluded that Soliris may be an important therapeutic option for patients with DDD. "Membrano Proliferative Glomerulonephritis: 3 Case Reports," S. Hartl.Researchers report on three patients with MPGN, two with MPGN type I and one with MPGN type II or DDD, with different clinical courses and biopsy results. The activation of the complement system and antibodies against the C3 convertase play an important role in all three patients. The researchers suggest that the C5 convertase might be a sophisticated target in MPGN and a clinical trial with the anti-C5 antibody Soliris might be an important step in advancing the treatment in this severe disease. An international registry has been set-up to be the basis for further clinical trials using Soliris in patients with MPGN. "A Novel Disease Mechanism for MPGN II/DDD: Increased CFHR1 Expression Results in Competitive Loss of CFH Cofactor Activity," C. Licht.Researchers present a case of an 11 year-old boy with DDD who, despite treatment, progressed to end stage kidney disease within months and was started on peritoneal dialysis. After four years, he received a transplant but developed disease recurrence within one week. Plasma therapy was transiently successful, but after nine months the treatment effect was lost and renal function again severely deteriorated resulting in severe and uncontrollable hypertension requiring kidney removal. A genetic analysis identified 3 copies of CFHR1. The researchers identified that a surplus of CFHR1 could play a role in disease recurrence early post treatment, and that more efficient treatment strategies like targeted complement blockade are required for patients with DDD.

Other Clinical Experience with Soliris in TMA Diseases: PNH, CAPS and D+HUSSeveral encouraging case studies were reported and provide further insight into the potential benefits of Soliris in treating patients with other TMA-related diseases, including PNH, CAPS, and D+HUS.

"Current Experience with Eculizumab and Future Aspects," M. Riedl on behalf of L.B. Zimmerhackl.The report from Dr. Zimmerhackl's group highlighted a recent publication in the New England Journal of Medicine that described the investigational use of Soliris to achieve the first reported successful kidney transplant for a patient suffering from CAPS, another ultra rare life-threatening disease characterized by multi-organ failure as a consequence of TMA. "Eculizumab in Diarrhea-Associated Hemolytic Uremic Syndrome," C. Mache.A 28 year-old patient with positive serology for E. coli 0157, acute renal failure with hemolysis and platelet consumption, decreased early complement protein levels, and no identifiable complement mutation was treated with hemodialysis, steroids and frequent plasma exchange. With no improvement and continued dialysis requirement during 30 days of this treatment, Soliris treatment was commenced. The patient's platelet count was normalized after 29 days, serum haptoglobin levels after 85 days, and serum LDH after 99 days. In addition, renal function had improved permitting termination of hemodialysis after 22 days. At the time of this conference (five months post initiation of therapy), Soliris is still sustaining suppression of TMA, and maintaining renal function with a serum creatinine of 2.3 mg/dL. "Rescue Therapy with Eculizumab Fails to Prevent Graft Loss in a Renal Transplant Patient with Factor I Mutation: Chronic Rejection or Recurrence?" S. Loos.A patient with D-HUS (presumably aHUS), who had already had 2 failed kidney transplants, each with evidence of aHUS-associated TMA on kidney biopsy, was transplanted a third time with chronic plasma therapy in 2008. In 2009, the patient progressed to kidney failure despite intravenous steroids and addition of further immunosuppressive medications. At that time, the kidney biopsy showed interstitial fibrosis without biopsy evidence of TMA or humoral rejection in the kidney. The kidney allograft function had already declined significantly, and treatment with Soliris was commenced. This case highlights the problem of defining HUS recurrence in failing renal transplant.

SOURCE Alexion Pharmaceuticals, Inc.