Real-World Data Show Ruxolitinib Plus Extracorporeal Photopheresis Drives Responses in Steroid-Refractory cGVHD

Data from a single-center retrospective study demonstrated that the combination of ruxolitinib (Jakafi) and extracorporeal photopheresis (ECP) yielded responses in patients with steroid-refractory or -dependent chronic graft-vs-host disease (cGVHD).¹

Findings presented at the 2026 EHA Congress showed that evaluable patients treated between 2017 and 2026 (n = 80) experienced an overall response rate (ORR) of 85%, including a complete response (CR) rate of 40% and a partial response (PR) rate of 45%. The median time to first response was 30 days (interquartile range [IQR], 21-60).

Notably, severity of baseline cGVHD and donor type did not influence response outcomes with the combination. However, patients who received a haploidentical graft experienced a higher CR rate compared with those who underwent transplant via a matched unrelated donor or an HLA-identical related donor (x², 5.9; P = .0177).

Median overall survival (OS) was not reached in the overall population. Kaplan-Meier estimates produced OS rates of 79.35% at 1 year, 73.43% at 2 years, and 71.45% at 5 years.

“These findings support ruxolitinib plus ECP as a highly active and broadly applicable second-line strategy in cGVHD and warrant confirmation in prospective, controlled studies,” lead study author Andrea Varkonyi, MD, of the Central Hospital of Southern-Pest, National Institute of Hematology and Infectious Diseases, Department of Hematology and Stem Cell Transplantation, Innovative Cell Therapy Center in Budapest, Hungary, and colleagues wrote in a poster presentation of the data.

How was the study designed, and which patients were included?

In the United States, ECP is utilized for the treatment of skin symptoms in patients with cutaneous T-cell lymphoma who are unresponsive to other forms of treatment.² Additionally, the National Comprehensive Cancer Network Guidelines include ECP as an option for steroid-refractory acute and cGHVD.³

Ruxolitinib, a JAK1/2 inhibitor, is an established option for steroid-refractory cGVHD following 1 to 2 lines of systemic therapy.¹ Because the ruxolitinib and ECP target inflammatory signaling and promote immune tolerance through complementary mechanisms, investigators of the real-world study sought to evaluate their use in combination, noting that evidence on the combined approach in larger, well-characterized cohorts has been limited.

In the retrospective analysis, steroid-refractory cGVHD was defined as progression despite at least 1 mg/kg/day of prednisone for 1 to 2 weeks, or a lack of improvement on at least 0.5 mg/kg/day for 1 to 2 months. Steroid-dependent disease was defined as a flare when tapering prednisone below 0.25 mg/kg/day.

All patients received ruxolitinib at 5 mg to 10 mg twice daily combined with ECP, which was initiated twice weekly and then tapered individually. Responses were assessed using NIH consensus criteria.

Among the 80 patients treated at South-Pest Central Hospital between 2017 and 2025, the median age at the start of treatment was 45.5 years (IQR, 31.25-53). Underlying diseases included acute myeloid leukemia (47.5%) and B- or T-cell acute lymphoblastic leukemia (23.8%). Myeloablative conditioning was given to 73.6% of patients, and 26.2% received reduced-intensity conditioning. Donor types included matched unrelated donors (40.0%), HLA-identical related donors (33.8%), and haploidentical related donors (26.2%).

Furthermore, cGVHD most frequently involved the skin (95.0%), liver (37.5%), and gastrointestinal tract (31.3%), with other sites including mucosal, oral, or ocular (11.3%), lung (3.8%), and joint or fascia (3.8%) involvement. Multiorgan involvement was observed in most patients.

Investigators reported that the 85% ORR compared favorably with historical cohorts of ruxolitinib monotherapy, in which response rates have ranged from approximately 60% to 75%. Responses were consistent across the study population, independent of donor type, baseline cGVHD severity, and a history of acute GVHD, which the authors suggested indicates that the combination extends across clinically diverse risk groups.

References

1. Várkonyi A, Réti M, Király A, et al. The combination of ruxolitinib and extracorporeal photopheresis is a highly effective second line treatment for chronic graft-versus-host disease. Presented at: 2026 EHA Congress; June 11-14, 2026; Stockholm, Sweden. Abstract PF1137.
2. Prescribing ECP. Therakos. 2025. Accessed July 13, 2026. https://therakos.com/therakos-photopheresis/prescribing-ecp/
3. NCCN guidelines for patients: graft-versus-host disease. 2026. Accessed July 13, 2026. https://www.nccn.org/patients/guidelines/content/PDF/GVDH-patient-guideline.pdf