Apellis is currently evaluating APL-2 in clinical studies in patients with geographic atrophy (GA), in patients with paroxysmal nocturnal hemoglobinuria (PNH) who are being treated with eculizumab or who are naive to complement inhibitor treatment, in patients with warm autoimmune hemolytic anemia (wAIHA) or
cold agglutinin disease (CAD) and in patients with complement-dependent nephropathies.
Apellis Pharmaceuticals announced updated data from its Phase 2 PLAUDIT study of APL-2 in patients with autoimmune hemolytic anemia, including
cold agglutinin disease and warm antibody autoimmune hemolytic anemia.
In the case presented by the authors, IgM type autoantibodies lead to agglutination and hemolysis of erythrocytes by adhering to erythrocyte surface with complement (C3b) opsonization at low temperature (3-4 [degrees]C) in cold antibody AIHA (
cold agglutinin disease) which the authors were focused on and which frequently occurs secondary to infections (1, 2, 4, 5).
However, it goes without saying that certain nonmalignant diseases may also be associated with monoclonal gammopathy of undetermined significance, for instance
Cold agglutinin disease, Acquired von Willebrand syndrome and Membranous nephropathy, and they need to be ruled out.16 In addition, due consideration must be given to the prospect of overtreatment as well as to stress and anxiety of the patient and economic cost of MGUS follow-up.17
Cold agglutinin test was positive with a titer of 1:1024.
Beyond its two marketed products, Bioverativ's pipeline includes a program in Phase 3 testing for
cold agglutinin disease, and early stage research programs and collaborations in hemophilia, and other rare blood disorders, including sickle cell disease and beta thalassemia.
The first case report of autoimmune hemolytic anemia in brucellosis was described due to
cold agglutinin antibodies [7], but hemolysis was mild in their case which was resolved with antibiotic treatment for brucellosis.
Some differential diagnoses of this condition include
cold agglutinin disease, thromboangiitis obliterans, diabetic gangrene, and thromboembolic gangrene [7, 10, 14].
As part of the acquisition, Bioverativ will obtain worldwide rights to True North's lead candidate, TNT009, a monoclonal antibody in development to treat
cold agglutinin disease, a hemolytic condition.
The category of cold hemolysins includes 2 distinct disease entities:
cold agglutinin syndrome and paroxysmal cold hemoglobinuria, either of which should demonstrate a DAT positive only with C3 antisera.
Cultures were negative for typical and atypical mycobacteria, but the patient had elevated
cold agglutinin and M.
The child was leukopenic and was found to have
cold agglutinin associated autoimmune hemolytic anemia.