Ekaterina Ponomareva

Emicizumab has changed prophylactic treatment for HA, but the need for FVIII replacement still exists for the treatment of breakthrough bleeds, perioperative management, and participation in high-risk activities/sports. The primary objectives of this study were to describe the characteristics of individuals with congenital hemophilia A (HA) without inhibitors receiving emicizumab prophylaxis and to compare clinical outcomes, healthcare resource utilization (HCRU), and costs among those prescribed octocog alfa [Advate, standard half-life factor VIII (FVIII)] versus extended half-life (EHL) FVIII agents. Patients with congenital HA without inhibitors and emicizumab use were identified retrospectively from Inovalon claims data and stratified by concomitant octocog alfa vs. EHL FVIII use. Inverse probability treatment weighting (IPTW) and multivariable regression were used to balance baseline covariates and compare billable annualized rate of bleeding (ABR_b), healthcare utilization, and costs between subgroups. Among the 1,282 patients receiving emicizumab prophylaxis, 501 received concomitant FVIII prescription for octocog alfa (n = 274) or EHL (n = 227) during follow-up. Following IPTW adjustment, total and medical costs were comparable between groups; however, pharmacy costs associated with concomitant octocog alfa therapy were significantly lower compared with EHL products ($35,381 vs. $61,739; p < 0.001). Importantly, ABR_b (0.35 vs. 0.28; p = 0.34) and bleed-related healthcare utilization remained similar across cohorts. Findings are limited by the retrospective design and reliance on claims data, which may omit home-treated bleeds, and misrepresent FVIII utilization due to billing complexities and assumptions about dispensed product use. While the choice of concomitant FVIII does not substantially influence ABR_b or bleed-related HCRU, significantly lower pharmacy costs with octocog alfa compared with EHL agents highlight its potential cost saving and support its consideration as a preferred on-demand treatment option in patients with HA without inhibitors on emicizumab prophylaxis.

Belumosudil was approved by the Food and Drug Administration in the United States for the treatment of relapsed/refractory chronic graft-versus-host disease (cGVHD) based on a randomized phase 2 trial comparing 2 belumosudil doses. The efficacy and safety of belumosudil vs the best available therapy (BAT) has not been studied. Applying rigorous statistical methodology to real-world data, this study estimated the efficacy of belumosudil vs BAT in cGVHD patients whose disease failed to respond to 2 to 5 prior lines of therapy (LOTs). Retrospective data between March 2015 and 2024 were collected across 8 US sites for 196 patients, contributing 113 belumosudil and 245 BAT LOTs. The primary outcome was 6-month overall response rate (ORR), defined as the proportion of complete or partial responses based on 2014 National Institutes of Health consensus criteria, physician assessment, or corticosteroid dose taper of ≥50% without cGVHD progression. Death, relapse, and beginning a new LOT were considered a lack of response. Targeted maximum likelihood estimation (TMLE) was used to estimate the 6-month ORR following belumosudil vs BAT (38.7% vs 26.8%, respectively) or 44.2% improvement with belumosudil (1-sided 95% confidence interval [CI], [4.4 to ∞]; P = .031). TMLE was also used to estimate 1-year failure-free survival when treated with belumosudil (61.2%) or BAT (47.8%), a 13.5% difference (95% CI, 1.5-100; P = .032). Descriptive assessment of safety showed adverse events recorded in 27% of belumosudil and 36% of BAT LOTs. Findings demonstrated that belumosudil improved clinical outcomes compared to BAT in cGVHD patients with 2 to 5 prior LOTs, and safety was consistent with belumosudil’s established profile.