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Report Describes Use of Ruxolitinib Over the Long-Term for Young Patient


Researchers discuss the case of a 32-year-old patient who is being treated with ruxolitinib over a period of years to treat steroid refractory graft-versus-host disease.

Long-term ruxolitinib use was well-tolerated in a 32-year-old male patient with steroid refractory graft-versus-host disease (srGVHD), according to a recent recently published case report.

Investigators described the treatment of the patient, who began with acute myeloid leukemia (AML) and developed srGVHD, in order to provide more context on the subject, which they describe as limited. There are clinical trials underway for ruxolitinib use in treating srGVHD, but there are no existing guidelines or protocols for long-term use.

Most srGVHD treatments with immunomodulatory drugs have side effects including lymphoproliferative disorders, relapse of disease, and opportunistic infections, the authors explained. But ruxolitinib can reduce side effects from srGVHD in acute settings up to 46% and in chronic settings up to 81%, according to some of the existing literature.

The patient had been on ruxolitinib for 5 years but could not tolerate a reduction in dosage due to flare-ups. At 26, the patient was diagnosed with AML and treated with 2 rounds of induction therapy with a 7 + 3 regimen. However, the authors said, the disease continued. He had a history of mucositis, pneumonia with sepsis, and decreased ejection fraction due to previous therapy. Because of these factors, he was given a high-dose cytarabine 1500 mg/m2 with sorafenib.

He received status postmatched unrelated donor stem cell transplant (MUD SCT) in November 2013 and began displaying acute GVHD after the transplant, for which he received topical steroids. He was also given sorafenib 200 mg and azacytidine to help with continued GVHD.

In May 2014, the patient presented with severe chronic GVHD involving the skin, eyes, and lungs; more than 50% of his skin was impacted. He was treated with prednisone 2 mg/kg given his severe GVHD and his skin symptoms improved.

When the authors attempted to taper below 20 mg, his skin flared up and he experienced worsening lung function. Steroid therapy was ineffective in 2014, the study authors said, so the patient began 5 mg of ruxolitinib twice a day and responded well.

Attempting to wean to once per day worsened his symptoms, the authors said, and a total discontinuation was not possible.

There are no reported identifiable issues or side effects from the long-term ruxolitinib use, they explained. They did note that there was one hospital admission for community-acquired pneumonia in 2018. That experience resolved with standard antibiotic therapy, they added.

The investigators still have a plan in place to wean the patient off ruxolitinib. They will attempt a taper every 3-4 months with 2.5 mg every 2 months to wean off therapy as the patient tolerates it. They will then weigh if the patient is gaining a benefit from ruxolitinib therapy, and if the risk for unknown long-term use outweighs the benefit of his current stability in suppressing his GVHD.

“With no seemingly therapeutic consequences of long-term use, we argue that there may be no reason not to continue our patient on therapy and monitor closely as with other immunosuppressive therapies,” the study authors concluded. “Our experience thus far has not shown any increased overall morbidity or mortality due to long-term ruxolitinib use.”

His flare ups were controlled when the therapy was restarted at 5 mg twice per day. The study authors said that the patient “seemingly tolerates the treatment with ruxolitinib.”


Sobash PT, Guddati AK, Kota V. Long-term use of ruxolitinib in an AML patient with posttransplant steroid refractory GVHD. Case Rep Oncol Med. Published online April 6, 2020. doi: 10.1155/2020/4936846

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