Belumosudil for chronic graft-versus-host disease after 2 or more prior lines of therapy: the ROCKstar Study.

Belumosudil, an investigational oral selective inhibitor of Rho-associated coiled-coil-containing protein kinase 2 (ROCK2), reduces type 17 and follicular T helper cells via downregulation of STAT3 and enhances regulatory T cells via upregulation of STAT5. Belumosudil may effectively treat patients with chronic graft-versus-host disease (cGVHD), a major cause of morbidity and late nonrelapse mortality after an allogeneic hematopoietic cell transplant. This phase 2 randomized multicenter registration study evaluated belumosudil 200 mg daily (n = 66) and 200 mg twice daily (n = 66) in subjects with cGVHD who had received 2 to 5 prior lines of therapy. The primary end point was best overall response rate (ORR). Duration of response (DOR), changes in Lee Symptom Scale score, failure-free survival, corticosteroid dose reductions, and overall survival were also evaluated. Overall median follow-up was 14 months. The best ORR for belumosudil 200 mg daily and 200 mg twice daily was 74% (95% confidence interval [CI], 62-84) and 77% (95% CI, 65-87), respectively, with high response rates observed in all subgroups. All affected organs demonstrated complete responses. The median DOR was 54 weeks; 44% of subjects have remained on therapy for ≥1 year. Symptom reduction with belumosudil 200 mg daily and 200 mg twice daily was reported in 59% and 62% of subjects, respectively. Adverse events (AEs) were consistent with those expected in patients with cGVHD receiving corticosteroids and other immunosuppressants. Sixteen subjects (12%) discontinued belumosudil because of possible drug-related AEs. Belumosudil, a promising therapy for cGVHD, was well tolerated with clinically meaningful responses. This trial was registered at www.clinicaltrials.gov as #NCT03640481.

Budget impact analysis of belumosudil for chronic graft-versus-host disease treatment in the United States.

AIMS: To assess the impact of belumosudil on the cost of care in chronic graft-versus-host disease (cGVHD) patients who have failed at least two prior lines of systemic therapy using a budget impact model. METHODS: A budget impact model with a 5-year time horizon was constructed in Microsoft Excel. The base case model uses the US prevalence rate of 3 L/4L + cGVHD patients from literature and secondary sources, with the potential for user-defined inputs, including model perspectives. The model includes data for two perspectives: the national US population and a hypothetical US private payer health insurance plan with 10 million (Mil) members. Additional model inputs include market share of cGVHD treatments, their associated adverse event rates, and healthcare resource utilization. RESULTS: The potential annual budget impact for the US national and payer plans was evaluated for cGVHD patients. Based on belumosudil utilization increasing to 55% in 3 L and 4 L + by 2026, cost savings of ∼5.5% and 6.7% ($128.8 and $4.9 Mil USD) were observed from national and payer perspectives, respectively. Cost savings in 2026 were derived from fewer AEs ($108.4 and $3.9 Mil USD, for national and payer perspectives; e.g. neutropenia, and thrombocytopenia) and reduced HCRU ($65.1 and $2.3 Mil USD, for national and payer perspectives; e.g. emergency room visits, ICU stays, etc.). LIMITATIONS: Results from the model were dependent on the available data inputs and assumptions. Real-world values may differ from the assumed performance of treatments, market growth, and treatment dosing and duration. CONCLUSION: The model results suggest that the introduction of belumosudil to treat cGVHD would be associated with substantial cost savings when evaluating a scenario with versus without belumosudil from a US payer perspective.

A retrospective analysis comparing small bowel follow-through with wireless capsule endoscopy in the evaluation of obscure gastrointestinal bleeding.

A comparative retrospective study was conducted to evaluate diagnostic findings between small bowel follow-through (SBFT) and wireless capsule endoscopy in the presence of obscure gastrointestinal bleeding. A convenience sample of 31 patients with previous negative upper and lower endoscopy was included in the study. Wireless capsule endoscopy established a significant source of obscure gastrointestinal bleeding 53% of the time. The diagnostic capacity of radiographic SBFT was measured at 17% as compared with that of the wireless capsule endoscopy. The clinical findings along with the digital images obtained from the wireless capsule endoscopy was found to be the optimum diagnostic tool in the evaluation of obscure gastrointestinal bleeding in the small bowel.