Improved residual fat malabsorption and growth in children with cystic fibrosis treated with a novel oral structured lipid supplement: A randomized controlled trial.

新型口服结构脂质补充剂治疗囊性纤维化儿童改善残余脂肪吸收不良和生长: 一项随机对照试验。

  • 影响因子:3.02
  • DOI:10.1371/journal.pone.0232685
  • 作者列表:"Stallings VA","Tindall AM","Mascarenhas MR","Maqbool A","Schall JI
  • 发表时间:2020-05-08

BACKGROUND:In the primary analysis of a 12-month double-blind randomized active placebo-controlled trial, treatment of children with cystic fibrosis (CF) and pancreatic insufficiency (PI) with a readily absorbable structured lipid (Encala™, Envara Health, Wayne, PA) was safe, well-tolerated and improved dietary fat absorption (stool coefficient of fat absorption [CFA]), growth, and plasma fatty acids (FA). OBJECTIVE:To determine if the Encala™ treatment effect varied by severity of baseline fat malabsorption. METHODS:Subjects (n = 66, 10.5±3.0 yrs, 39% female) with baseline CFA who completed a three-month treatment with Encala™ or a calorie and macronutrient-matched placebo were included in this subgroup analysis. Subjects were categorized by median baseline CFA: low CFA (<88%) and high CFA (≥88%). At baseline and 3-month evaluations, CFA (72-hour stool, weighed food record) and height (HAZ), weight (WAZ) and BMI (BMIZ) Z-scores were calculated. Fasting plasma fatty acid (FA) concentrations were also measured. RESULTS:Subjects in the low CFA subgroup had significantly improved CFA (+7.5±7.2%, mean 86.3±6.7, p = 0.002), and reduced stool fat loss (-5.7±7.2 g/24 hours) following three months of EncalaTM treatment. These subjects also had increased plasma linoleic acid (+20%), α-linolenic acid (+56%), and total FA (+20%) (p≤0.005 for all) concentrations and improvements in HAZ (0.06±0.08), WAZ (0.17±0.16), and BMIZ (0.20±0.25) (p≤0.002 for all). CFA and FA were unchanged with placebo in the low CFA group, with some WAZ increases (0.14±0.24, p = 0.02). High CFA subjects (both placebo and Encala™ groups) had improvements in WAZ and some FA. CONCLUSIONS:Subjects with CF, PI and more severe fat malabsorption experienced greater improvements in CFA, FA and growth after three months of Encala™ treatment. Encala™ was safe, well-tolerated and efficacious in patients with CF and PI with residual fat malabsorption and improved dietary energy absorption, weight gain and FA status in this at-risk group.


背景: 在一项为期12个月的双盲随机活性安慰剂对照试验的主要分析中,囊性纤维化 (CF) 和胰腺功能不全 (PI) 儿童用易吸收结构脂质 (encla) 治疗™,Envara Health,Wayne,PA) 是安全的,耐受性良好,改善了膳食脂肪吸收 (粪便脂肪吸收系数 [CFA]),生长和血浆脂肪酸 (FA)。 目的: 确定Encala™治疗效果因基线脂肪吸收不良的严重程度而异。 方法: 基线CFA的受试者 (n = 66,10.5 ± 3.0岁,39% 为女性) 完成了encla三个月治疗™或热量和常量营养素匹配的安慰剂被包括在这个亚组分析中。根据中位基线CFA对受试者进行分类: 低CFA (<88%) 和高CFA (≥ 88%)。在基线和3个月评估时,计算CFA (72小时粪便,称重食物记录) 和身高 (HAZ) 、体重 (WAZ) 和BMI (BMIZ) Z-评分。还测量了空腹血浆脂肪酸 (FA) 浓度。 结果: 低CFA亚组的受试者在EncalaTM治疗三个月后,CFA显著改善 (+ 7.5 ± 7.2%,平均值86.3 ± 6.7,p = 0.002),粪便脂肪减少 (-5.7 ± 7.2g/24小时)。这些受试者的血浆亚油酸 (+ 20%),α-亚麻酸 (+ 56%) 和总FA (+ 20%) (p ≤ 0.005) 浓度也有所增加,HAZ (0.06 ± 0.08),WAZ (0.17 ± 0.16) 和BMIZ (0.20 ± 0.25) 也有所改善 (全部p ≤ 0.002)。在低CFA组中,安慰剂组的CFA和FA无变化,一些WAZ增加 (0.14 ± 0.24,p = 0.02)。高CFA受试者 (安慰剂和恩卡拉™组) 在WAZ和一些FA方面有改善。 结论: 患有CF,PI和更严重的脂肪吸收不良的受试者在encla三个月后,CFA,FA和生长有更大的改善™治疗。恩卡拉™在该风险组中,CF和PI患者的残余脂肪吸收不良和改善的饮食能量吸收,体重增加和FA状态是安全的,良好的耐受性和有效的。




METHODS::Background: Early onset sepsis (EOS) is potentially life-threatening problem especially in preterm. EOS diagnosis is challenging due to its non-specific signs and laboratory tests. Mean platelet volume (MPV) has been used as predictor of many inflammatory diseases.Objectives: To assess the correlation between serial MPV measurement and EOS occurrence in preterm infants and to determine MPV effectiveness in combination with C reactive protein (CRP) to diagnose EOS and mortality prediction.Methods: The study was carried out on 95 preterm infants with antenatal risk factor for EOS. Blood samples were taken for complete blood count (CBC) including MPV evaluated at birth (cord blood) and at 72 h of life. CRP analyzed on days 1 and 3, subsequently patients were identified in two groups: sepsis (n = 28) and no-sepsis (n = 67).Results: MPV was significantly higher on both day 1 (10.23 ± 0.92) fl and day 3 (10.77 ± 1.16) fL in the sepsis group compared with no-sepsis (8.11 ± 0.29) fl and (8.53 ± 0.42) fl, respectively. MPV of 8.6 fL was identified as cut off value in patients probably resulting in sepsis with a sensitivity of 97.14% and a specificity of 100%. MPV of 10.4 fl was determined as cut off value in patients possibly resulting in death with a sensitivity of 70% and a specificity of 82.5%. The combination of both MPV and CRP on day 1 resulted in improving performance of MPV with higher negative predictive value (93.1%) and higher sensitivity (80%).Conclusion: High cord blood and day 3 MPV can be used as surrogate marker for prediction of EOS and associated mortality in preterm neonates.

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METHODS::Objective: This study used near-infrared spectroscopy (NIRS) to detect the pulmonary regional oxygen saturation (rSO2) of premature infants. The oxygenation state of the lung tissue was also evaluated, which provided preliminary evidence regarding the application of NIRS in oxygen therapy for premature infants.Methods: NIRS was used to measure the pulmonary rSO2 of 26 premature infants (gestational age <32 weeks). The correlations between pulmonary rSO2 and the arterial partial pressure of oxygen (PaO2), arterial oxygen saturation (SaO2), and pulse oxygen saturation (SpO2) were analyzed. The diagnostic value of NIRS was evaluated via both Pearson's correlation and receiver operating characteristic (ROC) curve analyses.Results: Pulmonary rSO2 was positively correlated with both PO2 and SaO2; the linear correlation coefficients (r) were 0.544 (p = .004) and 0.515 (p = .007), respectively. No significant correlation was found between rSO2 and SpO2 (p = .098). SpO2 was positively correlated with PO2 (r = 0.402, p = .042) and SaO2 (r = 0.625, p = .001). NIRS could be used to predict hypoxemia (area under the curve [AUC] = 0.843; Youden's index =0.654) when the pulmonary rSO2 was 62.39%, the sensitivity was 88.9%, and the specificity was 23.5% (p = .005) as well as predict hyperoxemia (AUC = 0.775; Youden's index = 0.65) when the pulmonary rSO2 was 61.99%, the sensitivity was 100%, and the specificity was 35% (p = .045). SpO2 predicted hypoxemia (AUC = 0.784, p = .019) but not hyperoxemia (AUC = 0.7, p = .144).Conclusion: NIRS objectively reflects the changes in oxygenation in the lung tissue. This study provides evidence for the clinical application of NIRS.

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METHODS::The global incidence of diabetes mellitus, including diabetes in pregnant women, is on the rise. Diabetes mellitus in a pregnant woman jeopardizes not only maternal health but can also have significant implications on the child to be born. Therefore, timely diagnosis and strict glycemic control are of utmost importance in achieving a safe outcome for both the mother and fetus. The treating physician should be aware of the complications that can arise due to poor glycemic control during pregnancy. The objective of this article is to discuss the key concerns in a neonate born to diabetic mother, the underlying pathogenesis, and the screening schedule during pregnancy.

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