Von Willebrand factor and factor VIII as potential biomarkers for diagnosis and disease monitoring in chronic graft-versus-host disease.

The identification of biomarkers of chronic Graft-versus-Host Disease (cGvHD) remains an unmet clinical need. Elevated von Willebrand factor (VWF) and factor VIII (FVIII) reflect inflammation and endothelial activation and might be interesting candidates for biomarkers of cGvHD. This prospective study evaluated 83 cGvHD patients and 39 allogeneic hematopoietic stem cell transplants recipients without cGvHD. VWF antigen (VWF:Ag), VWF activity (VWF:Ac), and FVIII activity were significantly elevated in cGvHD patients (p  <  0.001), with the highest levels observed in active disease. Higher VWF:Ag and VWF:Ac were associated with liver and oral cGvHD. In multivariate analysis, lower albumin was the strongest predictor of both higher VWF:Ag (R² = 0.576) and higher VWF:Ac (R² = 0.540), followed by older age, higher LDH, and number of affected organs. For higher FVIII, systemic immunosuppressive therapy emerged as the main predictor (R² = 0.247). Longitudinal analysis showed declining levels of these factors with cGvHD remission and persistent elevation in active disease. ROC analysis demonstrated diagnostic potential for early cGvHD of VWF:Ag >246.3% (AUC = 0.733) and VWF:Ac >271.4% (AUC = 0.728). These results show the potential of VWF and FVIII as novel biomarkers for diagnosis and disease activity in cGvHD. Additional validation in independent cohorts is warranted.