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  1. Lai NM, Ong JM, Chen KH, Chaiyakunapruk N, Ovelman C, Soll R
    Neonatology, 2020;117(1):125-126.
    PMID: 31487740 DOI: 10.1159/000502492
  2. Lai NM, Ong JMJ, Chen KH, Chaiyakunapruk N, Ovelman C, Soll R
    Neonatology, 2019;116(2):123-131.
    PMID: 31108494 DOI: 10.1159/000497423
    BACKGROUND: The introduction of Neonatology as a subspecialty in 1960 has stimulated an enormous amount of neonatal research. A large proportion of neonatal randomized-controlled trials (RCTs) have been included in the Cochrane reviews, within which methodological quality or risk-of-bias (ROB) assessment is an integral feature.

    OBJECTIVES: We described the ROB profile of neonatal RCTs published since the 1950s.

    METHODS: We analyzed individual studies within the Cochrane Neonatal reviews published up to December 2016. We extracted the reviewers' judgments on the ROB domains including random sequence generation, allocation concealment, blinding, incomplete outcome data, and selective reporting. We evaluated blinding of personnel in trials in which blinding was considered feasible.

    RESULTS: We assessed 1980 RCTs published between 1952 and 2016 from 294 Cochrane Neonatal systematic reviews, with full ROB assessments performed in 848 trials (42.8%). Among the ROB domains, the highest proportion of trials (73%) were judged as satisfactory ("low risk") in handling incomplete outcome data, while fewest trials achieved blinding of outcome assessor (38.4%). In the last 6 decades, a progressive increase has been observed in the proportion of trials that were rated as low risk in random sequence generation, allocation concealment, and selective reporting. However, blinding was achieved in less than half of the trials with no clear improvement across decades (23-44% since the 1980s).

    CONCLUSIONS: Despite steady improvement in the overall quality of neonatal RCTs over the last 6 decades, blinding remained unsatisfactory in the majority of the trials.

  3. Lai NM, Lee SWH, Wai SX, Teh ZW, Chan MY, Lim YS, et al.
    Neonatology, 2020;117(6):687-693.
    PMID: 33264799 DOI: 10.1159/000511656
    BACKGROUND: Neonates with jaundice are usually managed according to their serum bilirubin despite an unclear overall correlation between bilirubin levels and patient-important outcomes (PIOs) such as kernicterus spectrum disorder (KSD).

    OBJECTIVES: We examined data from Cochrane Neonatal reviews to assess whether conditions that constituted KSD were included as key outcomes and how commonly they occurred in the population studied.

    METHODS: We identified Cochrane reviews, published till November 2017 that evaluated interventions for neonatal jaundice (NNJ). We extracted the following information at the review and study levels: included population, outcomes assessed (in particular, whether PIOs such as KSD were listed as the primary outcomes), as well as their cumulative incidence in the reviews.

    RESULTS: Out of 311 reviews, 11 evaluated interventions for NNJ with 78 randomized controlled trials (RCTs) included. Among the reviews, a total number of 148 outcomes were predefined and 30 (20.3%) were PIOs related to KSD, with 11 (36.7%) listed as primary outcomes. Among the 78 included RCTs (total participants = 8,232), 38 (48.7%) enrolled predominantly high-risk and 40 (51.3%) enrolled predominantly low-risk population. A total number of 431 outcomes were reported, and 40 (9.2%) were PIOs related to KSD (of which 37 were from studies with high-risk infants), with 13 (32.5%) listed as primary outcome. Cumulatively, no infant developed KSD across all studies.

    CONCLUSIONS: There is suboptimal representation of PIOs such as KSD in neonatal trials and Cochrane reviews on NNJ. Over half of the trials included populations with very low risk of KSD, which does not represent judicious use of resources. Amidst our continued search for a more reliable surrogate marker for NNJ, studies should evaluate the whole spectrum KSD alongside serum bilirubin in high-risk populations with sufficiently significant event rates, as this will make the trial methodologically feasible, with findings that will impact the population concerned.

  4. Lai NM, Leom DYX, Chow WL, Chen KH, Lin PH, Chaiyakunapruk N, et al.
    Neonatology, 2020;117(4):428-435.
    PMID: 32209794 DOI: 10.1159/000506703
    BACKGROUND: Research findings based on patient-important outcomes (PIOs) provide more useful conclusions than those that are based on surrogate outcomes. It is unclear to what extent PIOs are represented in neonatal randomized controlled trials (RCTs).

    OBJECTIVES: We determined the proportion of PIOs in neonatal RCTs included in Cochrane Neonatal reviews.

    METHODS: We extracted up to 5 outcomes from each RCT included in Cochrane Neonatal reviews published until January 2018, with independent determination of PIOs among authors followed by a discussion leading to a consensus. We defined PIOs as outcomes that matter to patient care, such as clinical events or physiological or laboratory parameters that are widely used to guide management.

    RESULTS: Among 6,832 outcomes extracted from 1,874 RCTs included in 276 reviews, 5,349 (78.3%) were considered PIOs; 461 studies (24.5%) included 5 or more PIOs, 1,278 (68.2%) included 1-4 PIOs, while 135 (7.2%) had no PIO included. PIOs were observed more often among dichotomous than among continuous outcomes (94.9 vs. 61.5%; RR: 1.54; 95% CI: 1.50-1.58), and more among subjective than among objective outcomes (95.9 vs. 76.8%; RR: 1.25; 95% CI: 1.22-1.28). Newer studies were more likely to have a greater number of PIOs (adjusted OR: 1.033 [95% CI: 1.025-1.041] with each publication year).

    CONCLUSIONS: The large and increasing representation of PIOs over the years suggests an improving awareness by neonatal trialists of the need to incorporate important outcomes in order to justify the utilization of resources. Further research should explore the reasons for non-inclusion or non-reporting of PIOs in a small proportion of RCTs.

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