Background: Ruxolitinib (RUX) is an oral, selective Janus kinase (JAK)1/JAK2 inhibitor approved for chronic graft-versus-host disease (cGVHD) after failure of 1–2 lines of systemic therapy in patients ≥12 years of age. Treatment patterns of RUX for cGVHD have evolved since approval, and real-world treatment characteristics and impact on corticosteroid (CS) management need further description. We report long-term data describing real-world characteristics in a large cohort treated with RUX to understand treatment patterns and impact on CS dosing in patients with cGVHD after allogeneic hematopoietic stem cell transplantation (allo-HSCT) in the United States.

Methods:This retrospective analysis of administrative claims data included commercial, Medicare/Medicare Advantage, and Medicaid health plan members of any age who received allo-HSCT, had cGVHD diagnosis, received RUX as cGVHD therapy (≥1 claim for RUX between July 1, 2021, and September 30, 2023; earliest claim was the index date), and had health insurance coverage ≥6 months before and ≥6 months after index (or less due to death). Follow-up was until earliest of death, end of continuous plan enrollment, or end of study period. Prescription refills in pharmacy claim records were used to determine RUX and prednisone-equivalent CS dosing and treatment length. Line of therapy for cGVHD medications was based on claims for CS, belumosudil, abatacept, alemtuzumab, RUX, etanercept, hydroxychloroquine, ibrutinib, imatinib, interleukin-2, methotrexate, mycophenolate mofetil, pentostatin, and rituximab after cGVHD diagnosis. Calcineurin inhibitors (CNIs), mTOR inhibitors, and extracorporeal photopheresis (ECP) were measured separately from the line of therapy.

Results:This analysis included 1147 patients who received RUX for cGVHD. Median (IQR) age was 52 (31–62) years (<18 years, 13.0%). Overall, 56.9% were male, 51.5% had commercial insurance, 31.0% had Medicaid, and 17.4% had Medicare. Patients were followed for a median (IQR) of 458 (254–740) days from initiating RUX. Patients initiated RUX a median (IQR) of 90 (20–256) days from cGVHD diagnosis (70 days in patients aged <18 years; 96 days in adults). Overall, RUX was administered as first-line therapy with/without CNIs/mTOR inhibitors in 29.0% (n=333) of patients, and second-line or later therapy in 71.0% (n=814). RUX monotherapy with/without CS was the most common regimen in patients receiving RUX as first- (84.7%; n=282), second- (93.7%; n=680), or third-line (77.3%; n=68) therapy. Other common therapies during RUX treatment were CNIs (52.2% of patients), mTOR inhibitors (14.5%), and ECP (12.6%). Median (IQR) RUX starting dosage was 10 mg/day (10–20) overall, in patients aged <18 years (5–10), and in adult patients (10–20). Among patients who had a RUX refill, 59.5% (n=615/1034) had a dose change; of those, 51.5% (n=317) had an increase and 48.5% (n=298) had a decrease in their first dose change. Median (IQR) time to first dose change was 71 (29–188) days. Kaplan-Meier analysis showed median (95% CI) length of RUX treatment was 242 days (218–279); 40.9% of patients remained on RUX at 1 year and 21.3% at 2 years.

Median (IQR) CS dosing was 40.0 (20.0–70.0) mg/day pre-index. By day 90, 71% of patients remained on CS; among these, median (IQR) CS dose decreased by 62.5% (15.0 [5.0–30.0] mg/day). By day 180, 55% had CS refill, with a median (IQR) CS dose of 15.0 (5.6–37.5) mg/day. By 360 days post-index, 35% had CS refill, with a median (IQR) dose of 17.5 (7.5–40.0) mg/day. Median (95% CI) time from earliest CS fill for cGVHD to discontinuation of CS (presence of a ≥90-day gap in supply) or reduction to low-dose (10 mg) CS was 77 (68–86) days overall, 84 (65–107) days in pediatric patients, and 74 (65–86) days in adult patients. By the end of 1-year follow-up, discontinuation or reduction to low-dose CS was achieved in 90% of patients (pediatrics, 87%; adults, 91%).

Conclusions:Patients treated with RUX for cGVHD in real-world clinical practice remained on treatment for a median of 8 months; 40.9% remained on RUX for 1 year and 21.3% for 2 years, suggesting long-term persistence of safety and ongoing clinical benefit. Median time from earliest CS fill for cGVHD to discontinuation or reduction to low-dose CS was 77 days. By the end of 1-year follow-up, 90% of patients achieved discontinuation or reduction to low-dose CS. These outcomes support RUX as a long-term and CS-sparing treatment for cGVHD.

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2025
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