Ruxolitinib Likely Safe, Effective in Chronic GVHD

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The study of 36 patients showed a response rate of 59% at 3 months, which increased to 62% at 6 months.

New study findings suggest that ruxolitinib is safe and effective for use against chronic forms of graft-vs-host-disease (GVHD), even in patients who have failed on multiple lines of treatment and have traditionally poor prognostic factors.

The single-center study of 36 patients showed a response rate of 59% at 3 months, which increased to 62% at 6 months. On average, patients received 3 previous treatments for a median of 8.6 months. Despite this response to treatment, however, the researchers did not observe a survival advantage associated with ruxolitinib.

Their findings were published recently in Leukemia Research.


According to the researchers, their findings are in line with previous retrospective findings, although large, prospective trials are needed to confirm what these groups have found and longer follow-up is needed to determine the durability of these responses, they said.

“Chronic graft-vs-host disease is diagnosed in about 50 % of allografted patients at a median of 6 months from transplant, representing the main cause of mortality in 2-year post–hematopoietic stem cell transplantationsurvivors,” explained the group. “For patients who are alive and struggling with chronic GVHD, quality of life is deeply compromised, impacting emotional health, restricting ability to work, and resulting in more frequent hospitalizations. Available salvage therapies are heterogeneous, but efficacy is often suboptimal, and none has demonstrated a strong superiority.”

The data also showed improved single-organ responses, with significant improvements seen in cutaneous, genital, and oral GVHD following 3 months of treatment. After longer follow-up, improvements were also seen in ocular GVHD.

The researchers noted that genital GVHD is often underrecognized and underdiagnosed, with research showing incidence rates of 24.9% to 45%. Genital GVHD has also been associated with genital neoplasia, which the researchers say highlights the importance of early and targeted intervention.

“We did not see a major improvement in fasciitis features, a not surprising data given the often-irreversible nature of this complication, and in gastrointestinal GVHD,” wrote the researchers. “We also did not see a significant improvement in pulmonary GVHD, which by now remains the most challenging GVHD presentation; however, among 11 patients with pulmonary GvHD, in 2 cases the forced expiratory volume in the first second values improved with a concomitant reduction of dyspnea, and subjective benefit was reported by other 6 patients, with 2 cases of decreased oxygen requirement.”

Across the patients, ruxolitinib was well tolerated. Although the treatment was not associated with a significant rate of malignancy relapse or infectious complications, 2 patients experienced fatal infections.


Xue E, Lorentino F, Pavesi F, et al. Ruxolitinib for chronic steroid-refractory graft versus host disease: a single center experience. Leuk Res. Published online June 11, 2021. doi:10.1016/j.leukres.2021.106642