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

Introduction: Ruxolitinib was approved in September 2021 for chronic graft-versus-host disease (cGVHD) after failure of 1–2 lines of systemic therapy in adult and pediatric patients ≥12 years of age. Real-world data describing the use of ruxolitinib and how it impacts corticosteroid use in patients with cGVHD following allogeneic hematopoietic stem cell transplantation (alloHSCT) across various practices require further investigation. This retrospective study describes real-world characteristics and treatment patterns of ruxolitinib and corticosteroids in patients with cGVHD after alloHSCT in the United States.

Methods: This was a retrospective claims data analysis that included commercial, Medicare/Medicare Advantage, and Medicaid health plan members who received an alloHSCT and had a cGVHD diagnosis, ruxolitinib use for cGVHD treatment (earliest claim was the index date), and health insurance coverage ≥6 months before and ≥6 months after the index date (or less due to death). Patients were followed until the earliest of death, end of continuous plan enrollment, or end of study period. Ruxolitinib and corticosteroid dosages and treatment length were determined using prescription refills in pharmacy claim records.

Results: A total of 471 patients who had cGVHD and started ruxolitinib treatment between July 2019–August 2022 were included in this analysis. Median (IQR) age was 46 (26–60) years, with 15.3% <18 years. Approximately 60% were male, 59.2% had commercial insurance, 28.0% had Medicaid, and 4.5% had Medicare. Patients were followed for a median (IQR) of 465 (316–694) days from ruxolitinib initiation.

Patients initiated ruxolitinib after a median (IQR) of 116 (26–255) days (83.5 days in pediatric patients, 119 days in adults) from cGVHD diagnosis. A total of 213 (45%) patients started ruxolitinib as second-line treatment following treatment with corticosteroids. Median ruxolitinib starting dosage was 10 mg/day for pediatric and adult patients (pediatric IQR: 5–10; adult IQR: 10–20). Among patients who had a ruxolitinib refill, 58.4% had a dose change; of those, 55.9% had a dose increase and 44.1% had a dose decrease in their first dose change. Kaplan-Meier analysis showed median length of ruxolitinib treatment across all lines of therapy was 245 days (95% CI: 210–300). At the end of follow-up, 33.1% of patients remained on ruxolitinib treatment for a median of 389 days (IQR: 260–591).

Median corticosteroid daily dosing decreased 69% over time from median (IQR) 40.0 (18.4–61.0) mg pre-index to 12.5 (5.0–40.0) mg at 180 days post-index across all patients. Patients who initiated ruxolitinib early (1–29 days post-cGVHD) were more likely to taper steroids (72.3%) than those who initiated ruxolitinib later (30–100 or 101+ days; 59.3% and 58.5%, respectively; P=0.030). Median corticosteroid daily dose pre-index was 40 mg for early ruxolitinib initiators and day 30–100 ruxolitinib initiators, and 25 mg for day 101+ ruxolitinib initiators. Median corticosteroid daily dose by 180 days post-index was lowest for ruxolitinib early initiators (10 mg) versus 20 mg for day 30–100 ruxolitinib initiators and 15 mg for day 101+ ruxolitinib initiators. Furthermore, the number of patients with steroid complication-related healthcare resource utilization (HCRU) was numerically less after initiation of ruxolitinib compared with before ruxolitinib treatment for ambulatory visits (68.4% vs 78.8%), emergency room visits (12.5% vs 15.3%), and inpatient stays (37.2% vs 56.1%), respectively.

Conclusions: In real-world practice, patients treated with ruxolitinib for cGVHD start at a median dose of 10 mg daily, with most patients receiving dose adjustments as necessary. Overall, patients received ruxolitinib for a median of 8 months, with 33.1% of patients continuing ruxolitinib for a median duration of over 1 year, suggesting long-term safety and ongoing clinical benefit of treatment. Patients had a 69% reduction in corticosteroids over the 6 months after initiating ruxolitinib and a decrease in steroid complication-related HCRU, suggesting ongoing clinical benefit of ruxolitinib in reducing steroid dose and related complications.