Real-world use of belumosudil: Key insights and future perspectives

Belumosudil, an oral selective Rho-associated coiled coil-containing protein kinase 2 (ROCK2) inhibitor, dually inhibits the inflammation and fibrosis that occurs in cGvHD.¹ Belumosudil has shown a response rate of 75% in steroid-refractory chronic graft-versus-host disease (SR-cGvHD) in the ROCKstar study (NCT03640481), with a low rate of adverse events beyond the first year of therapy. Further to these data collected in a clinical trial setting, promising data are also emerging in real-world use.

The GvHD Hub spoke to Corey Cutler, Dana-Farber Cancer Institute, Boston, US, about his key insights into real-world use of belumosudil.

Real-world use of belumosudil

• Several case series have demonstrated similar overall response rates as observed in the ROCKstar trial, suggesting the results from the trial can apply to patients in real-world settings.
• With long-term use, belumosudil treatment appears to prevent worsening of bronchiolitis obliterans syndrome and, in some cases, leads to reversal of airflow obstruction.
• A retrospective study of 66 patients who underwent allogeneic hematopoietic cell transplant demonstrated an overall response rate of 64% at 12 months.² The 12-month overall survival was 92%, with no Grade ≥3 toxicities identified.²

Future perspectives

• In the phase I dose-escalation study of belumosudil, patients with early increases in regulatory T cells were most likely to benefit from treatment, suggesting potential as a biomarker for patient selection in the steroid-refractory setting. Use of biomarkers will become increasingly important for treatment optimization.
• Belumosudil is approved in the US for third-line use and beyond; however, it may be beneficial for some patients to receive it earlier in their treatment sequence. The phase III ROCKnrol-1 study (NCT06143891) is currently recruiting patients to assess belumosudil in combination with steroids, compared with steroids alone, in newly diagnosed cGVHD.

This educational resource is independently supported by Sanofi. All content was developed by SES in collaboration with an expert steering committee; funders were allowed no influence on the content of this resource.