News

The U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug discovery in rare diseases, with new guidance on natural history— how disorders such as spinal muscle atrophy(SMA) run their course if untreated — the choice of “efficacy endpoints” in clinical trials, and how…

A case of simultaneous cryoglobulinemic vasculitis and cold agglutinin disease (CAD) was reported for the first time in a patient, according to Chinese researchers. The rare occurrence was described in the study, “Concomitant cryoglobulinemic vasculitis and cold agglutinin disease successfully treated with bortezomib: A case report,”…

Treatment with low-dose Rituxan can effectively treat patients with warm autoimmune hemolytic anemia (wAIHA) but higher doses may be more beneficial for patients with cold agglutinin disease (CAD), results from a 10-year retrospective study suggest. The study, “Low dose rituximab in autoimmune hemolytic anemia: ten years…

Despite being extremely rare, cold type autoimmune hemolytic anemia, or cold agglutinin disease (CAD), should be considered in patients with infectious mononucleosis, especially those who also have anemia, a case study reports. The case report, “Cold type autoimmune hemolytic anemia- a rare manifestation of infectious mononucleosis; serum ferritin as…

New therapies that target the complement pathway — a type of immune response that involves the production of antibodies — could help improve the treatment of cold agglutinin disease and other hemolytic anemias, a review study says. Clinical trials testing such therapies are recruiting patients. The article, “…

Acute kidney injury triggered by excessive alcohol intake was described for the first time by Japanese researchers in a patient with cold agglutinin disease (CAD). The case was reported in the study “Acute Kidney Injury by Renal Hemosiderosis Secondary to Primary…

Off-label treatment with Soliris (eculizumab) was well-tolerated and lessened hemolysis — red blood cell destruction — and transfusion requirements in patients with cold agglutinin disease (CAD), a Phase 2 trial showed. The study, “Eculizumab in cold agglutinin disease (DECADE): an open-label, prospective, bicentric, nonrandomized phase 2…

Treatment with Soliris (eculizumab, marketed by Alexion) can effectively reverse cold agglutinin disease (CAD) attacks and induce rapid clinical remission, a case report shows. The study, “High efficacy of eculizumab treatment for fulminant hemolytic anemia in primary cold…

Sutimlimab, an antibody against complement C1, increases hemoglobin levels in patients with cold agglutinin disease and prevents blood transfusions, a first-in-human trial shows. The study, “Inhibition of Complement C1s improves severe hemolytic anemia in cold agglutinin disease: a first-in-human trial” was published in the journal Blood. Autoimmune…

With the U.S. midterm elections now less than two weeks away, patient advocacy groups are solidly focused on a range of hot-button issues, from the Orphan Drug Tax Creditand affordable health insurance to future funding for rare disease research. Yet “whether Democrats take over the House or Senate, or…