Eculizumab is an effective treatment for
paroxysmal nocturnal hemoglobinuria (PNH). However, considering the risk of life-threatening
meningococcal disease, life-long duration and costs, there are strict criteria for initiation of
therapy. To evaluate the application and real-world effectiveness of
eculizumab in the Netherlands, a multicenter retrospective cohort study was conducted: indications and treatment outcomes were collected for 105 Dutch PNH patients. In all patients,
eculizumab was initiated conforming to indications as formulated in the Dutch PNH guideline. According to recently published response criteria, 23.4% of the patients had reached a complete hematological response, 53.2% a good or partial response, and 23.4% a minor response after 12 months of
therapy. In the majority of patients the response remained stable during long-term follow-up. The degree and relevance of
extravascular hemolysis significantly differed between response groups (p = 0.002). Improvements of EORTC-QLQc30 and FACIT-
fatigue scores were observed, however patients reported lower scores than the general population. A detailed evaluation of 18 pregnancies during
eculizumab showed no maternal or
fetal deaths, and no thromboembolic events during pregnancy. This study demonstrates that the majority of patients benefit from
eculizumab when adhering to the indications as formulated in the Dutch PNH guideline. However, novel
therapies are needed to further improve real-world outcomes, such as hematological responses and quality of life.