Since then, the GvHD Hub has continued to focus on novel treatment options for SR-aGvHD such as ruxolitinib, basiliximab, and MSC infusions, as well as other approaches.
Regarding ruxolitinib, an oral inhibitor of the Janus kinases (JAK) 1 and 2, we covered an article on the
phase II results
of the REACH1 study (
), which evaluated the use of ruxolitinib in combination with corticosteroids for patients with SR-aGvHD. Ruxolitinib responses were rapid and durable, with an overall response rate of 54.9% by Day 28 and a complete response rate of 56.3%.
Following on from this, the
study provided a comparison of ruxolitinib with nine other therapies for Grades 2–4 SR-aGvHD. These results demonstrated a significant improvement in response rates with ruxolitinib over control therapies, including a higher durable overall response, a longer duration of response, and a longer failure-free survival. Also, the safety profile of ruxolitinib was consistent with previously reported results; however, the data were not mature enough to evaluate the impact on patient survival as the data cutoff was Day 56.
We also reported on
the long-term clinical outcomes of patients with SR-aGvHD treated with basiliximab
,a chimeric monoclonal antibody that blocks the alpha-chain of the interleukin-2 receptor (IL-2Rα) complex. In this retrospective study, basiliximab proved to be an effective treatment, with high overall response rates after Days 28 and 56, and the infection rates were similar to those obtained with other second-line treatments for SR-GvHD. Furthermore, this study identified that severe aGvHD (Grades 3–4) before basiliximab treatment was an independent prognostic marker of poorer response to basiliximab.
For the treatment of pediatric patients with SR-aGvHD, we reported on a
phase III trial of remestemcel-L
ex-vivocultured adult human MSC infusion. The results demonstrated that remestemcel-L was a relatively safe and effective treatment option for this patient group, with a durable overall response rate of 70.4% and improved complete response rates from Day 28 up to Day 100.
covered a recent review
and colleagues, who evaluated both current and emerging treatment and management options for patients with SR-aGvHD. Second-line treatments were discussed, including extracorporeal photopheresis, anti-thymocyte globulin, anti-IL-2Rα monoclonal antibodies, tumor necrosis factor α inhibitors and monoclonal antibody combinations, as well as third-line treatments including MSCs and methotrexate.
Although significant progress has been made towards establishing effective treatment options for patients with SR-aGvHD, there is still a lack of routine guidelines for the treatment of patients with steroid refractory disease and a need for less toxic options to treat this condition.
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