Autoimmune hemolytic anemia: evaluation of diagnostic features in correlation with the clinical course

Abstract

Autoimmune hemolytic anemia (AIHA) is caused by autoantibodies directed against a patient’s own red blood cells, leading to their destruction, a process known as hemolysis. This can result in life-threatening anemia. AIHA is diagnosed based on clinical signs of hemolysis and laboratory findings, particularly the detection of autoantibodies and complement components on the patient’s red blood cells. AIHA can be classified into warm AIHA (wAIHA), cold AIHA (cAIHA), mixed forms, and the rare paroxysmal cold AIHA. wAIHA is typically mediated by IgG autoantibodies, whereas cAIHA is usually driven by IgM autoantibodies that activate the classical complement pathway.

In this thesis, we focus on the complex pathophysiology underlying AIHA, together with the optimization and standardization of laboratory testing for autoantibodies. We further systematically evaluate the efficacy and safety of novel treatment options for patients with refractory AIHA, including complement inhibitors, B-cell–targeting Bruton’s tyrosine kinase inhibitors, and the plasma cell–targeting drug daratumumab.

We initiated a national prospective study, the Data Registry of AutoImmune Hemolytic Anemia (DRAIHA study). This study systematically collects serological and clinical data, along with patient outcomes, to improve understanding of AIHA pathophysiology and to contribute to the development of personalized, evidence-based diagnostic and treatment strategies.