Ruxolitinib may be a promising second-line treatment option for pediatric patients with steroid refractory advanced graft-versus-host disease (aGVHD), according to a new study. Based on the findings, researchers suggest their results be validated in a prospective large-scale pharmacokinetic and efficacy trial.<\/p>\n
The small retrospective study of 29 patients recruited from 15 pediatric transplantation centers demonstrated that after 28 days, 6 patients achieved a complete response (CR). By 41 days, 19 (65.5%) patients achieved a CR. Another 2 patients had a partial response (PR). Among the 16 patients who were under the age of 6 years, there was a 75% CR and 6.3% PR. All patients who achieved a response discontinued corticosteroid treatment.<\/p>\n
Prior to ruxolitinib, patients received an average of 2 immunosuppressive agents, with a range of 1-6.<\/p>\n
The prognosis of patients with aGVHD depends mainly on the response of steroid treatment, which is known to be effective in approximately 50% of cases. Indeed, the mortality rate of steroid-refractory or recurrent aGVHD remains as high as 50% to 70%, wrote the researchers. There is currently no standardized second-line strategy for steroid resistant aGVHD.<\/p>\n
While several immunosuppressive therapies are used in the second line of treatment, they carry variable response rates. For example, Monoclonal anti-TNF have demonstrated response rates between 30% and 60%. But, relapse at discontinuation was high at approximately 80%.<\/p>\n
Results of the current study led the researchers to argue that ruxolitinib may be a possible treatment for these patients, including in cases of gastrointestinal involvement or severe GVHD.<\/p>\n
A total of 8 patients experienced treatment failure, and 23 patients were alive after a median follow-up of 685 days after their hematopoietic stem cell transplantation and a median 480 days after initiating ruxolitinib treatment.<\/p>\n
CR\/PR was a significant factor of survival with a rate of 90% versus 50% in case of treatment failure (P = 0.02), wrote the researchers. Nevertheless, we could not find any association of baseline characteristics and CR\/PR to ruxolitinib. Neither the number of immunosuppressive agents before using ruxolitinib nor involved organs was significant.<\/p>\n
The researchers were also unable to demonstrate a relationship between ruxolitinib dose and rate of efficacy nor the speed of efficacy. Throughout the study, the median dose of ruxolitinib was 12.6 mg\/m2\/day.<\/p>\n
The study also showed a favorable safety profile of ruxolitinib, with no observed severe hematologic adverse events and with all cytopenias requiring a dose modification resolving.<\/p>\n
Viral replication (including cytomegalovirus, Epstein-Barr virus, and adenovirus) was observed in 41.4% of cases indicating that viral replications need to be closely monitored, wrote the researchers. This rate should be interpreted with caution because a comparable frequency of viral replications was reported in children treated with other immunosuppressive drugs, including infliximab, alemtuzumab, and daclizumab.<\/p>\n
Reference<\/p>\n
Laisne L, Neven B, Dalle J, et al. Ruxolitinib in children with steroid-refractory acute graft-versus-host disease: a retrospective multicenter study of the pediatric group of SFGM-TC. Pediatr Blood Cancer. Published online July 2, 2020. doi: 10.1002\/pbc.28233.<\/p>\n
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