Results of a clinical trial recently published in the Journal of Allergy and Clinical Immunology: Global found that sarilumab does not significantly improve quality of life in patients with indolent systemic mastocytosis (SM).
Sarilumab is a drug used to treat patients with rheumatoid arthritis by blocking interleukin-6 (IL-6), a protein involved in inflammation. Because IL-6 levels are often elevated in patients with SM, it has been hypothesized that targeting IL-6 in patients with SM may improve symptoms and quality of life.
In their study, the authors randomized 16 patients with indolent SM to receive sarilumab or a placebo. The trial was double-blinded, meaning neither the patients nor the researchers knew which treatment each patient received until after the trial was over.
Quality of life was measured using the Mastocytosis Quality of Life Questionnaire (MC-QoL), where higher scores correspond to poorer quality of life.
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The average quality of life score at the start of the trial was 47.8 out of 100. The authors evaluated quality of life again after 16 weeks of treatment. The average MC-QoL was 38.98 among patients receiving sarilumab and 33.02 among patients receiving a placebo. Although these results appear to indicate that the placebo improved quality of life more than sarilumab, this difference was not statistically significant.
Further statistical analysis revealed that sarilumab could result in anywhere from a 9-unit benefit to a 21-unit deficit in average quality of life scores compared to the placebo. This range is so wide, the authors explained, due to the small sample size of the trial.
In both the placebo and treatment groups, seven of the eight participants experienced adverse events, including vomiting, dizziness and yeast infections. Two patients receiving sarilumab had to withdraw from the study due to significant adverse events that were possibly related to treatment.
“Results in this small trial did not support the use of sarilumab in this population and highlights the importance of double-blind randomized studies,” the authors concluded.
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