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Inhaled corticosteroids may benefit patients with sickle cell disease
Patients with sickle cell disease who did not have asthma experienced pain reduction after inhaling corticosteroids, according to results of a placebo-controlled trial.
“This study is an important step in identifying a new treatment that may benefit millions living with sickle cell disease,” Jeffrey Glassberg, MD, MA, assistant professor of emergency medicine at Icahn School of Medicine at Mount Sinai, said in a press release.
Because sickle cell disease alters pulmonary physiology — including increased inflammation, increased blood flow, and airway resistance and hyperactivity — comorbid asthma is a risk factor for increased mortality and morbidity.
However, individuals with sickle cell disease but without asthma have high rates of hyperreactivity and wheezing, suggesting there may be a link between respiratory symptoms and pain related to sickle cell disease.
Glassberg and colleagues studied the efficacy and feasibility of inhaled corticosteroids in 54 patients with sickle cell disease without asthma. Each patient received 220 mcg mometasone furoate dry powder inhalation once daily or placebo for 16 weeks.
The primary outcome was feasibility, defined as the number of patients who completed treatment divided by the total number enrolled. Prespecified efficacy outcomes included daily patient-reported pain score over time and change in soluble vascular cell adhesion molecule levels — a surrogate for vascular injury — between baseline and 8 weeks.
Patients were required to maintain at least 70% adherence to medication. They also completed an 8-week assessment of biological outcomes and at least 30 pain diaries for the per-protocol analysis.
Feasibility was 96% (95% CI, 87-99) on the intent-to-treat analysis and 83% (95% CI, 71-91) on the per-protocol analysis.
Over time, the adjusted treatment effect of mometasone led to a reduction in daily pain score of 1.42 points (95% CI, 0.61-2.21; P = .001).
Changes in ASCQ-Me pain impact were 5.2 points (95% CI, –18.9 to 8.49) better with mometasone than placebo; however, this difference did not reach statistical significance.
For patients who had at least one ED visit, the absolute difference in rate of admission to hospital was 12% in patients who received mometasone (RR = 0.79; 95% CI, 0.45-1.38). Patients treated with mometasone also spent an average of 1.88 fewer days (95% CI, –5.95 to 2.19) in the hospital if admitted than patients given placebo.
Patients who received placebo experienced a mean increase of 170.25 ng/mL in soluble vascular cell adhesion molecule compared with a decrease of 182.47 ng/mL among patients treated with mometasone, for an adjusted treatment effect of 526.94 ng/mL (95% CI, 1,003.23-50.66). The percent change soluble vascular cell adhesion molecule was 8.82% for placebo vs. –4.86% for mometasone.
No deaths or cases of pneumonia or acute chest syndrome were reported during the study period. More patients treated with mometasone experienced hoarseness of voice, thrush and sore throat than patients in the placebo group, but none were statistically significant.
“To our knowledge, there are no other published clinical trials of asthma controller medications for treatment of [sickle cell disease]. With respect to protocol fidelity, we demonstrated high rates of adherence to study medication with low rates of loss to follow-up,” the researchers wrote. “Future studies to improve generalizability of these findings should be conducted at multiple centers and should evaluate longer durations of treatment.” – by Melinda Stevens
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Disclosure: The researchers report no relevant financial disclosures.