scholarly journals Efficacy and Safety of Ruxolitinib in Treatment of Steroid Refractory/Refractory Acute Graft-Versus-Host Disease: A Prospective Multicenter Research

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 5724-5724
Author(s):  
Zhiping Fan ◽  
Lan Deng ◽  
Xudong Li ◽  
Qing Zhang ◽  
Li Xuan ◽  
...  

Abstract Objectives To explore the efficacy and safety of ruxolitinib in the treatment of steroid refractory/refractory acute graft versus host disease (aGVHD). Methods A prospective analysis was conducted in the 31 steroid refractory/refractory aGVHD cases who treated with ruxolitinib from October 2017 to June 2018. The first dose of ruxolitinib was 5-10mg bid, and the dose was adjusted to 5mg qd for maintenance treatment when complete remission (CR) status lasted for 2 weeks after steroid refractory/refractory aGVHD turned into CR. Results Of all the 29 steroid refractory/refractory aGVHD patients, 15 patients were treated with ruxolitinib in case of disease progression after receiving at least one second-line treatment, and 14 cases directly with ruxolitinib after the occurrence of corticosteroid refractoriness. The time of starting ruxolitinib treatment after the occurrence of aGVHD was 10 (4-81) d, the median time of onset was 8 (3-18) d. The overall response rate was 89.6% (26/29) including 22 complete responses (75.9%), and 4 partial responses (13.8%). 12 patients with thrombocytopenia need to reduce the dose of ruxolitinib, and 1 patient withdrew treatment for obvious bleeding tendency. With the median follow-up 3 (2-8) months after treatment with ruxolitinib, 28 patients survived and 1 died of severe pneumonia related to aGVHD, and the overall survival was 96.6% (28/29). Conclusion Ruxolitinib is safe and effective in the treatment of steroid refractory/refractory aGVHD, and can be used as a second-line treatment for refractory aGVHD. Disclosures No relevant conflicts of interest to declare.

2018 ◽  
Vol 101 (5) ◽  
pp. 676-683
Author(s):  
Frederick W. Thielen ◽  
Hedwig M. Blommestein ◽  
Liesbeth E.M. Oosten ◽  
Friso G. Calkoen ◽  
Arjan C. Lankester ◽  
...  

2018 ◽  
Vol 53 (7) ◽  
pp. 844-851 ◽  
Author(s):  
Marietta Nygaard ◽  
Niels Smedegaard Andersen ◽  
Claus Ernst Moser ◽  
Gitte Olesen ◽  
Ida Marie Schjødt ◽  
...  

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 4878-4878
Author(s):  
Yuan Suo ◽  
Jiapei Liu ◽  
Yiming Sun ◽  
Qiaoyuan Wu ◽  
Hua Jin ◽  
...  

Abstract Background Acute graft-versus-host disease (aGVHD) is the major cause of non-relapse morbidity and mortality after allogeneic hematopoietic stem cell transplantation(allo-HSCT). But only 40% of the patients will respond to first-line therapies Corticosteroids. Ruxolitinib, a selective Janus kinase (JAK) 1/2 inhibitor, reduces the incidence and severity of GVHD while preserving graft-versus-leukemia effects in preclinical models. The retrospective study evaluated the efficacy of ruxolitinib compared with other second-line therapies for steroid-refractory aGVHD (SR-aGVHD) in a single-center. Methods A total of 90 patients who developed SR-aGVHD after HSCT were evaluated in this retrospective study.They were treated with ruxolitinib (n=45) or other salvage-therapies (n=45) including MTX, basiliximab, etanercept and MSC at our institution. The primary endpoint was the overall response rate (ORR) at Day 28. Additional endpoints included overall survival (OS), cumulative incidence rates of failure-free survival (FFS), relapse, non-relapse mortality (NRM) and cGVHD. Treatment failure was defined as 1) addition of new systemic therapy for aGVHD, 2) NRM, 3) relapse, 4) progression of hematologic disease. FFS and OS were calculated from the day of starting the use of second-line treatments for aGVHD. Results The median age was 34 years (range 14-69). At the time of enrollment 25 patients had grade Ⅱ disease, 39 with grade Ⅲ disease, 26 with grade IV disease. The skin was involved in 54.4%, lower gastrointestinal (GI) tract in 78.9% and liver in 20.2%. With a median follow-up of 1.33 years, the ORR at day 28 was higher in ruxolitinib group than non-ruxolitinib group (62.2% [95% CI, 47.5%-77.0%] vs. 26.7% [95% CI, 13.2%-40.1%], P=0.001) (Table 1). The 1-year OS was 64.4 % and 45.5% in the two groups, respectively (P=0.0382). Ruxolitinib treatment also improved the 1-year cumulative incidence of FFS (57.8% vs. 26.6%, P=0.002), while the 1-year cumulative incidence of relapse did not differ significantly (9.6% vs. 20.0%, P=0.195). The 1-year cumulative incidence of NRM was lower in the ruxolitinib group than the non-ruxolitinib group (24.4% vs. 45.3%, P=0.023). The 1-year and 3-year cumulative incidence of cGVHD were 17.8% vs. 33.3% and 26.8% vs. 44.4% between the ruxolitinib group and non-ruxolitinib group (P=0.10 and P=0.04). Conclusions Our study demonstrated that ruxolitinib is effective than other second-line treatments in patients with SR-aGVHD due to the higher response rates and the improvement of prognosis. Furthermore, ruxolitinib could reduce the incidence and severity of chronic GVHD in aGVHD patients. Keywords: Ruxolitinib; Steroid-refractory; Acute graft-versus-host disease; Haploidentical hematopoietic stem cell transplantation Figure 1 Figure 1. Disclosures No relevant conflicts of interest to declare.


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