For a study, the researchers sought to determine how eculizumab affected the outcome of vascular liver disease (VLD) patients. The research considered a retrospective cohort of Paroxysmal nocturnal hemoglobinuria (PNH) patients in the Valdig registry who were diagnosed with VLD between 1997 and 2019. Eculizumab was the focus of attention. Death, venous thrombosis, haemorrhage, arterial ischemia event, infection, and liver-related problems were all studied outcomes. The PNH/VLD group was compared with the Envie2 non-PNH cohort in terms of survival and new thrombotic events. About 62  patients (33 women) were included, with a median age of 35 years (28–48) and a median follow-up VLD diagnosis of 4.7 years (1.2–9.5). The median haemoglobin content was 10.0 g/dl (8–11), while lactate dehydrogenase (LDH) was 736 IU (482–1744). Eculizumab was given to 42 patients (68%) with a median exposure time of 40.1 [9.3–72.6] months. The incidence rate ratio (IRR) was 0.29, 95% CI (0.1–0.9), P=.035. Mortality was considerably lower in the exposed versus nonexposed period: 2.6 versus 8.7 per 100 (PY). Thrombosis recurrence was less common when eculizumab was used: 0.5 versus 2.8 per 100 PY, IRR 0.22 (0.07–0.64). Other secondary endpoints (such as bleeding, arterial ischemia lesions, infection, and liver problems) were less common over the eculizumab treatment period, though not statistically significant. The PNH group had a 6-year thrombosis-free survival rate of 70%, with a 95% CI of [0.60–0.83], compared with 83%, with a 95% CI of [0.70–1.00] for non-PNH Envie 2 patients (P<.001). Finally, PNH and VLD patients have a higher risk of recurrent thrombosis than non-PNH patients. In individuals with PNH and VLD, eculizumab was linked to a decreased mortality rate and a lower risk of thrombotic recurrence.

 

Source:onlinelibrary.wiley.com/doi/10.1002/ajh.26474