IC-02. The use of ruxolitinib in combination with extracorporeal photopheresis in patients with steroid-refractory acute and chronic graft-versus-host disease
Nune A. Kambaryan, Anna A. Dotsenko, Tatiana A. Bykova, Yulia Yu. Vlasova, Anna A. Osipova, Olesya V. Paina, Anna G. Smirnova, Elena V. Morozova, Ludmila S. Zubarovskaya, Ivan S. Moiseev
RM Gorbacheva Research Institute, Pavlov University, St. Petersburg, Russia
Contact: Dr. Nune A. Kambaryan, e-mail: firstname.lastname@example.org
The REACH III trial completed in May 2020 has shown ruxolitinib to be the best graft-versus-host disease (GVHD) therapy option currently available. Still, up to 30% of patients with GVHD are refractory to this therapy and are left without standard-of-care options.
Materials and methods
We conducted a retrospective, single-centered study in a cohort of patients refractory to ruxolitinib as monotherapy. A total of 26 patients were then treated by ruxolitinib in combination with extracorporeal photopheresis. The median age was 20 (3-46) years with 57.7% (n=15) of patients being adults and 42.3% (n=11) being children. M/F ratio was 13/13. Most patients with acute GVHD had Gr 3 (76.9%, n=20) or Gr 4 (7.7%, n=2) disease at therapy initiation. Also, 61.5% (n=16) of patients had moderate to severe chronic GVHD. In 92.3% (n=24) there was skin, in 73.1% mucosa, in 61.5% eyes, in 53.8% liver, and in 23% joints involvement.
During the treatment, 61.5% (n=16) patients achieved a partial response. The overall survival (OS) rate was 92.3% (n=24), progression-free survival was 88.5% (n=23). In 19.2% of patients (n=5) immunosuppressive therapy (IST) was successfully withdrawn. The median time to partial response (PR) was 42 (4-730) days and median therapy duration was 61 (7-1095) days. The best response rate was seen in patients with GVHD of the mucosa (30.8%, n=8). Skin and liver GVHD had the same response rate of 23.1% (n=6 for each). Patients with gastrointestinal tract (GIT) GVHD had a response rate of 26.9% (n=7). Patients with joint and genital GVHD had the same response rate of 11.5% (n=3). Only 15.4% (n=4) responses were seen in lung GVHD cases. Patients with ocular GVHD had shown disease progression in 19.2% (n=5). The patients with skin, joint, mucous membranes, and gastrointestinal tract GVHD, exhibited the disease progression in 7.7% of cases (n=2), accordingly. In patients with genital and pulmonary GVHD, disease progression was observed in only 3.8% cases (1 person). The vast majority of patients had no complications. In 11.5% (n=3) patients there was CMV reactivation, 7.7% of patients (n=2) developed hemorrhagic cystitis.
In total, 61.5% of patients respond to combined therapy with ruxolitinib and extracorporeal photopheresis. The vast majority of cases the symptoms show no signs of worsening. Thus, ruxolitinib combined with extracorporeal photopheresis seems to be a feasible therapy approach in patients with resistant GVHD. Due to the small sample size, validation on larger patient groups is necessary.
Graft-versus-host disease, ruxolitinb, extracorporeal photopheresis, steroid resistance.