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  • Myo-Inositol Lacked Efficacy and Safety in a Multicenter Trial

    By Lynda Seminara
    Selected By: Deepak P. Edward, MD
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    Journal Highlights

    JAMA
    2018;320(16):1649-1658

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    In studies of preterm infants with respi­ratory distress, myo-inositol appeared to reduce the severity of retinopathy of prematurity (ROP) and the frequency of ROP, death, and intraventricular hemorrhage. However, its efficacy and safety had not been tested in large trials until a recent multicenter study by Phelps et al. In their large population of infants, myo-inositol did not reduce the risk of death or type 1 ROP relative to placebo, suggesting that it is not a viable treatment for this age group. The study was terminated early because the mortality rate was significantly higher in the myo-inositol arm.

    This randomized trial included 638 infants (gestational age [GA] <28 weeks) who were enrolled from 18 U.S. neo­natal intensive care centers in 2014 and 2015. (The planned enrollment was 1,760 participants, which would have been sufficient to detect an absolute reduction in death or type 1 ROP of 7% with 90% power.)

    Participants received either myo-inositol 40 mg/kg (n = 321) or placebo (n = 321) for up to 10 weeks. Adminis­tration was every 12 hours, intravenous­ly and then enterally (when feeding). The main outcomes were type 1 ROP or death before the determination of an unfavorable ROP status. The designated favorable outcome was survival without type 1 ROP. The final month of follow-up was February 2016.

    In the study population (mean GA, 26 weeks; 50% male), 92% had a documented outcome. Death or type 1 ROP occurred more frequently in the myo-inositol group (29% vs. 21%; adjusted relative risk, 1.41; p = .01). Before 55 weeks’ postmenstrual age, death (any cause) had occurred in 18% of the myo-inositol group and 11% of the placebo group (adjusted relative risk, 1.66; p = .007). The most common serious adverse events with active treatment versus placebo, respectively, were systemic infection (16% vs. 11%), respiratory distress (15% vs. 13%), intraventricular hemorrhage (10% vs. 9%), poor perfusion or hypotension (7% vs. 4%), and necrotizing enteroco­litis (6% vs. 4%).

    Although these findings do not sup­port the efficacy or safety of myo-inosi­tol in premature infants, the trial’s early termination does not allow for defini­tive conclusions.

    The original article can be found here.