Real-World Characteristics and Ruxolitinib Treatment Patterns in 471 Patients with Chronic Graft-Versus-Host Disease in the United States

Background Chronic graft-versus-host disease (cGVHD) is a common and potentially life-threatening complication of allogeneic hematopoietic stem cell transplantation (HSCT). Ruxolitinib (RUX) is approved for cGVHD after failure of 1 or 2 lines of systemic therapy in adult and pediatric (ped) patients (pts) ≥12 y. Objectives To describe real-world characteristics and treatment patterns of RUX in pts with cGVHD post HSCT in the United States. Methods This retrospective study included commercial, Medicare and Medicare Advantage, and Medicaid health plan members with evidence of an allogeneic HSCT, cGVHD diagnosis, evidence of RUX use after cGVHD diagnosis (earliest claim was index date), and ≥6 mo of health insurance coverage before and after the index date (unless less due to death). Pts were followed until the earliest of death, end of continuous enrollment, or end of study period. Dosages and RUX treatment length were based on prescription refills in pharmacy claim records. Results A total of 471 pts who had cGVHD and started RUX treatment between Jul 2019 and Aug 2022 were included in the analysis. Mean age was 42.6 y, with 15.3% <18 y and 10.6% >65 y. Nearly 40% were female, 59.2% had commercial insurance, 28.0% had Medicaid, and 4.5% had Medicare. Most ped pts (66.7%) had Medicaid. Median baseline Charlson Comorbidity Index score was 2.0.Among all pts, 74.1% had ≥1 claim for acute GVHD any time during the study period. Over 62% of patients initiated RUX between 2021–2022. Patients were followed for a median of 465 d from RUX initiation.Pts started RUX after a median of 116 d (83.5 d in ped pts, 119 d in adults) from cGVHD diagnosis. Most pts started RUX as second-line treatment. In the 6 mo before RUX initiation, 88.3% of pts had ≥1 fill for corticosteroids (CS), and 76.0% were on calcineurin inhibitors. The median RUX starting dosages were 10 mg/d for both ped and adult pts (ped IQR: 5, 10; adult IQR: 10, 20). Among pts who had a RUX refill, 58.4% had a dose change; of those, 55.9% had dose increases, and 44.1% had dose decreases in their first dose change; median time from RUX initiation to first dose change was 82 d (50 d in ped pts, 89 d in adult pts). Within 6 mo after RUX initiation, 80.5% of pts had evidence of ≥1 fill for CS. A Kaplan-Meier analysis showed that the median length of RUX treatment was 245 d (95% CI: 210, 300). At the end of follow-up, 33.1% of pts had remained on RUX treatment for a median of 389 d (IQR: 260, 591). Conclusions RUX has been used to treat cGVHD in both ped and adult pts primarily as second-line treatment after CS in real-world clinical practice. Most pts, including ped pts, started RUX on a recommended dose and had dose adjustments. Pts received RUX for a median of ∼8 mo. One third of pts continued on RUX at the end of study period for a median duration of over 1 year, deriving ongoing clinical benefit from RUX. Dynamics of CS use are being evaluated to assess impact of RUX treatment on CS burden.