No abstract

One patient with an acquired factor VIII inhibitor is reported in which an acute lower intestinal hemorrhage was successfully managed using Desmopressin (DDAVP). The patient initially had a factor VIII level of 10% with a inhibitor titer of 1.9 Bethesda units. Following administration of DDAVP the factor VIII level rose to 86% and there was a decrease in the number and volume of bloody stools. The inhibitor disappeared following treatment with corticosteroids, however the patient ultimately expired due to complications of ischemic colitis. This case and 21 previously reported cases of acquired hemophilia treated with DDAVP are reviewed. The data support a role for DDAVP in the treatment of non life threatening hemorrhage in patients with acquired hemophilia and low titer factor VIII inhibitors (< 5 Bethesda units or factor VIII > or = 5%).

Background Accessible biomarkers are needed for immunotherapy in advanced non-small-cell lung cancer (NSCLC). We previously described a multivariate risk prediction model, the iSEND, which categorises advanced NSCLC patients treated with nivolumab into Good, Intermediate or Poor groups. This model was developed by using only clinical and analytical variables (sex, ECOG-performance status, neutrophil-to-lymphocyte ratio [NLR] and post-treatment delta NLR). Methods An international database of 439 patients who received post-platinum PD-1/L1 monotherapies was collected for validation. Performance of the iSEND to different PD-L1 groups was compared by using time-dependent positive predictive value (PPV) for their mortality events. Results Median follow-up was 18.2 months (95% CI: 15.9–19.6). The overall survival of the iSEND Good (HR = 0.31, 95% CI: 0.22–0.43, p < 0.0001) was superior to the iSEND Poor. Time-dependent PPV for mortality of iSEND Poor was superior to PD-L1 = 0% group at 12 (75 vs. 53%, p = 0.01) and 18 months (85 vs. 46%, p = 0.03). However, female gender did not independently associate with better outcome in the validation cohort. Conclusion The iSEND model is associated with the outcome of post-platinum PD-1/L1 monotherapy in advanced NSCLC patients. The iSEND Poor demonstrated a superior performance to PD-L1 = 0% in negative prognostication. Prospective investigation and modelling with other significant parameters in a larger cohort are warranted.