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The Cochrane Database of Systematic... Mar 2023Phototherapy is a widely accepted, effective first-line therapy for neonatal jaundice. It is traditionally used continuously but intermittent phototherapy has been... (Review)
Review
BACKGROUND
Phototherapy is a widely accepted, effective first-line therapy for neonatal jaundice. It is traditionally used continuously but intermittent phototherapy has been proposed as an equally effective alternative with practical advantages of improved maternal feeding and bonding. The effectiveness of intermittent phototherapy compared with continuous phototherapy is unknown.
OBJECTIVES
To assess the safety and effectiveness of intermittent phototherapy compared with continuous phototherapy.
SEARCH METHODS
Searches were conducted on 31 January 2022 in the following databases: CENTRAL via CRS Web, MEDLINE and Embase via Ovid. We also searched clinical trials databases and the reference lists of retrieved articles for randomised controlled trials (RCTs) and quasi-randomised trials.
SELECTION CRITERIA
We included RCTs, cluster-RCTs and quasi-RCTs comparing intermittent phototherapy with continuous phototherapy in jaundiced infants (both term and preterm) up to the age of 30 days. We compared intermittent phototherapy with continuous phototherapy by any method and at any dose and duration as defined by the authors.
DATA COLLECTION AND ANALYSIS
Three review authors independently selected trials, assessed trial quality and extracted data from included studies. We performed fixed-effect analyses and expressed treatment effects as mean difference (MD), risk ratio (RR) and risk difference (RD) with 95% confidence intervals (CIs). Our primary outcomes of interest were rate of decline of serum bilirubin, and kernicterus. We used the GRADE approach to assess the certainty of evidence.
MAIN RESULTS
We included 12 RCTs (1600 infants) in the review. There is one ongoing study and four awaiting classification. There was little or no difference between intermittent phototherapy and continuous phototherapy with respect to rate of decline of bilirubin in jaundiced newborn infants (MD -0.09 micromol/L/hr, 95% CI -0.21 to 0.03; I² = 61%; 10 studies; 1225 infants; low-certainty evidence). One study involving 60 infants reported no incidence of bilirubin induced brain dysfunction (BIND). It is uncertain whether either intermittent or continuous phototherapy reduces BIND because the certainty of this evidence is very low. There was little or no difference in treatment failure (RD 0.03, 95% CI 0.08 to 0.15; RR 1.63, 95% CI 0.29 to 9.17; 1 study; 75 infants; very low-certainty evidence) or infant mortality (RD -0.01, 95% CI -0.03 to 0.01; RR 0.69, 95% CI 0.37 to 1.31 I² = 0%; 10 studies, 1470 infants; low-certainty evidence). AUTHORS' CONCLUSIONS: The available evidence detected little or no difference between intermittent and continuous phototherapy with respect to rate of decline of bilirubin. Continuous phototherapy appears to be more effective in preterm infants, however, the risks of continuous phototherapy and the potential benefits of a slightly lower bilirubin level are unknown. Intermittent phototherapy is associated with a decrease in the total number of hours of phototherapy exposure. There are theoretical benefits to intermittent regimens but there are important safety outcomes that were inadequately addressed. Large, well designed, prospective trials are needed in both preterm and term infants before it can be concluded that intermittent and continuous phototherapy regimens are equally effective.
Topics: Infant; Infant, Newborn; Humans; Jaundice, Neonatal; Phototherapy; Bilirubin; Family
PubMed: 36867730
DOI: 10.1002/14651858.CD008168.pub2 -
Mayo Clinic Proceedings Nov 2008To determine whether an elevated homocysteine level is an independent risk factor for the development of coronary heart disease (CHD) to aid the US Preventive Services... (Meta-Analysis)
Meta-Analysis Review
OBJECTIVE
To determine whether an elevated homocysteine level is an independent risk factor for the development of coronary heart disease (CHD) to aid the US Preventive Services Task Force in its evaluation of novel risk factors for incident CHD.
METHODS
Studies of homocysteine and CHD were identified by searching MEDLINE (1966 through March 2006). We obtained additional articles by reviewing reference lists from prior reviews, original studies, editorials, and Web sites and by consulting experts. We included prospective cohort studies that measured homocysteine and Framingham risk factors and the incidence of CHD in the general adult population without known CHD. Each study was quality rated using criteria developed by the US Preventive Services Task Force. We conducted a meta-analysis using a random-effects model to determine summary estimates of the risk of major CHD associated with each 5-micromol/L increase in homocysteine level. The systematic review and meta-analysis were conducted between January 25, 2005, and September 17, 2007.
RESULTS
We identified 26 articles of good or fair quality. Most studies found elevations of 20% to 50% in CHD risk for each increase of 5 micromol/L in homocysteine level. Meta-analysis yielded a combined risk ratio for coronary events of 1.18 (95% confidence interval, 1.10-1.26) for each increase of 5 micromol/L in homocysteine level. The association between homocysteine and CHD was similar when analyzed by sex, length of follow-up, outcome, study quality, and study design.
CONCLUSION
Each increase of 5 micromol/L in homocysteine level increases the risk of CHD events by approximately 20%, independently of traditional CHD risk factors.
Topics: Adult; Case-Control Studies; Cohort Studies; Coronary Disease; Female; Homocysteine; Humans; Male; Odds Ratio; Prospective Studies; Risk Factors; Sex Factors
PubMed: 18990318
DOI: 10.4065/83.11.1203 -
Effects of betaine supplementation on cardiovascular markers: A systematic review and Meta-analysis.Critical Reviews in Food Science and... 2022Controversy regarding the effects of betaine supplementation on cardiovascular markers has persisted for decades. This systematic review and meta-analysis compared the... (Meta-Analysis)
Meta-Analysis
Controversy regarding the effects of betaine supplementation on cardiovascular markers has persisted for decades. This systematic review and meta-analysis compared the effects of betaine supplementation on cardiovascular disease (CVD) markers. Studies examining betaine supplementation on CVD markers published up to February 2021 were identified through PubMed, the Cochrane Library, Web of Science, Embase, and SCOPUS. Betaine supplementation had a significant effect on concentrations of betaine (MD: 82.14 μmol/L, 95% CI: 67.09 to 97.20), total cholesterol (TC) (MD: 14.12 mg/dl, 95% CI%: 9.23 to 19.02), low-density lipoprotein (LDL) (MD: 10.26 mg/dl, 95% CI: 6.14 to 14.38)], homocysteine (WMD: -1.30 micromol/L, 95% CI: -1.61 to -0.98), dimethylglycine (DMG) (MD: 21.33 micromol/L, 95% CI: 13.87 to 28.80), and methionine (MD: 2.06 micromol/L, 95% CI: 0.23 to 3.88). Moreover, our analysis indicated that betaine supplementation did not affect serum concentrations of triglyceride (TG), high-density lipoprotein (HDL), fasting blood glucose (FBG), C-reactive protein (CRP), liver enzymes [alanine aminotransferase (ALT), aspartate aminotransferase (AST), gamma-glutamyl transferase (GGT)], and blood pressure. Our subgroup analysis suggested that a maximum dose of 4 g/d might have homocysteine-lowering effects without any adverse effect on lipid profiles reported with doses of ≥4 g/d. In conclusion, the present systematic review and meta-analysis supports the advantage of a lower dose of betaine supplementation (<4 g/d) on homocysteine concentrations without the lipid-augmenting effect observed with a higher dosage.
Topics: Betaine; Biomarkers; Cardiovascular Diseases; Dietary Supplements; Homocysteine; Humans; Triglycerides
PubMed: 33764214
DOI: 10.1080/10408398.2021.1902938 -
Transplantation May 2006Target of rapamycin inhibitors (TOR-I) have a novel mode of action but uncertain clinical role. We performed a systematic review of randomized trials where... (Meta-Analysis)
Meta-Analysis Review
Target of rapamycin inhibitors (sirolimus and everolimus) for primary immunosuppression of kidney transplant recipients: a systematic review and meta-analysis of randomized trials.
BACKGROUND
Target of rapamycin inhibitors (TOR-I) have a novel mode of action but uncertain clinical role. We performed a systematic review of randomized trials where immunosuppressive regimens containing TOR-I were compared with other regimens as initial therapy for kidney transplant recipients.
METHODS
Databases (inception, June 2005) and conference proceedings (1996-2005) were searched. Two independent reviewers assessed trials for eligibility and quality. Results at 1 year, are expressed as relative risk (RR), where values<1 favor TOR-I, or lower dose of TOR-I, and for continuous outcomes are expressed as weighted mean difference (WMD), both expressed with 95% confidence intervals (CI).
RESULTS
Thirty-three trials (142 reports) were included (27 trials of sirolimus, 5 of everolimus, and 1 of head-to-head comparison). When TOR-I replaced calcineurin inhibitors (CNI) (8 trials with 750 participants), there was no difference in acute rejection (RR, 1.03; 95% CI, 0.74-1.44), but serum creatinine was lower (WMD, -18.31 micromol/L; 95% CI, -30.96 to -5.67) and bone marrow more suppressed (leukopenia: RR 2.02; 95% CI, 1.12-3.66; thrombocytopenia: RR, 6.97; 95% CI, 2.97-16.36; and anaemia: RR, 1.67; 95% CI, 1.27-2.20). When TOR-I replaced antimetabolites (11 trials with 3966 participants), acute rejection and cytomegalovirus infection (CMV) were reduced (RR, 0.84; 95% CI, 0.71-0.99; RR, 0.49; 95% CI, 0.37-0.65, respectively), but hypercholesterolemia was increased (RR, 1.65; 95% CI, 1.32-2.06). When low- was compared with high-dose TOR-I, with equal CNI dose (10 trials with 3,175 participants), rejection was increased (RR, 1.23; 95% CI, 1.06-1.43) but calculated glomerular filtration rate (GFR) higher (WMD, 4.27 mL/min; 95% CI, 1.12-7.41), and when lower-dose TOR-I and standard-dose CNI were compared with higher-dose TOR-I and reduced CNI, acute rejection was reduced (RR, 0.67; 95% CI, 0.52-0.88), but calculated GFR was also reduced (WMD, -9.46 mL/min; 95% CI, -12.16 to -6.76). There was no significant difference in mortality, graft loss, or malignancy risk for TOR-I in any comparison.
CONCLUSIONS
TOR-I have been evaluated in four different primary immunosuppressive algorithms: as replacement for CNI and antimetabolites, in combination with CNI at low and high doses, and with a variable dose of CNI. Generally, surrogate endpoints for graft survival favor TOR-I (lower risk of acute rejection and higher GFR), and surrogate endpoints for patient outcomes are worsened by TOR-I (bone marrow suppression and lipid disturbance). Long-term hard-endpoint data from methodologically robust randomized trials are still required.
Topics: Creatinine; Humans; Immunosuppressive Agents; Kidney Transplantation; Randomized Controlled Trials as Topic; Sirolimus; Survival Analysis; Time Factors; Treatment Outcome
PubMed: 16699448
DOI: 10.1097/01.tp.0000219703.39149.85 -
Clinical Endocrinology May 2009Elevated circulating androgens are risk factors for several chronic, metabolic and reproductive disorders. Metformin is an insulin-sensitizing agent that may lower... (Meta-Analysis)
Meta-Analysis
OBJECTIVES
Elevated circulating androgens are risk factors for several chronic, metabolic and reproductive disorders. Metformin is an insulin-sensitizing agent that may lower androgen levels. To evaluate the effects of metformin on endogenous androgens and SHBG levels in women, we conducted a systematic review and meta-analysis of randomized controlled trials (RCTs) comparing metformin with placebo or no treatment.
DATA SOURCE
We used OVID to search MEDLINE, EMBASE and CENTRAL until March 2007.
REVIEW METHODS
Two reviewers independently extracted data on methodological quality, participants, interventions and outcomes of interest. Our a priori primary outcome was post-treatment measurements. In a secondary analysis, we evaluated the difference between the pre- and post-treatment levels. We computed the weighted mean difference (WMD) as a measure of effect for each outcome using the DerSimonian-Laird random effects method. We used the I2 statistic to assess heterogeneity and explored its causes in subgroup analyses of features related to participants' characteristics and study design. Based on a regression model, we conducted sensitivity analyses by investigating the use of placebo as a predictor of effect size.
RESULTS
Twenty RCTs fulfilled the inclusion criteria. Pooled WMDs in post-treatment levels between the metformin and control group were -0.31 nmol/l (95% CI -0.65 to 0.03) for total testosterone (TT), 0.10 pmol/l (95% CI -0.89 to 1.10) for free testosterone (FT), 0.14 micromol/l (95% CI -0.34 to 0.62) for dehydroepiandrosteronesulfate (DHEAS), -0.60 nmol/l (95% CI -1.67 to 0.46) for androstenedione (AND) and 5.88 nmol/l (95% CI 2.01-9.75) for SHBG. Pooled WMDs of the pre- to post-treatment differences (i.e. with adjustment for baseline hormone levels) were -0.38 (95% CI -0.51 to -0.25) for TT, -2.71 (95% CI -10.35 to 4.93) for FT, -0.50 (95% CI -0.83 to -0.16) for DHEAS, -1.39 (95% CI -2.30 to -0.49) for AND and 6.63 (95% CI 2.32-10.94) for SHBG. In subgroup analyses, features related to the administered treatment (i.e. metformin as a single agent or as part of combined regimens) partly explained the heterogeneity. Sensitivity analyses of studies using placebo showed similar results to those not using placebo.
CONCLUSIONS
Our systematic review and meta-analysis provides evidence of metformin-induced changes in circulating androgens and SHBG levels in women but the quality of evidence is not high. However, there are no data from RCTs regarding these effects in postmenopausal women or healthy premenopausal women. High-quality RCTs are required to evaluate whether metformin has effects on surrogate markers and patient-important outcomes in these patient groups.
Topics: Androgens; Cardiovascular Diseases; Diabetes Mellitus, Type 2; Female; Humans; Hypoglycemic Agents; Insulin Resistance; Metformin; Obesity; Polycystic Ovary Syndrome; Randomized Controlled Trials as Topic; Risk Factors; Sex Hormone-Binding Globulin
PubMed: 19178532
DOI: 10.1111/j.1365-2265.2008.03459.x -
Archives of General Psychiatry Nov 2008The prevalence of depression in later life increases with plasma total homocysteine concentration (tHcy). High tHcy accounts for about 15% of prevalent cases, but... (Meta-Analysis)
Meta-Analysis Review
CONTEXT
The prevalence of depression in later life increases with plasma total homocysteine concentration (tHcy). High tHcy accounts for about 15% of prevalent cases, but observational studies are prone to confounding and bias. Genetic association studies are not prone to the same sources of error and offer an opportunity to explore the consistency and external validity of this association.
OBJECTIVE
To determine if tHcy is causally related to depression in later life.
DESIGN
Cross-sectional study (Health in Men Study), systematic review, and meta-analysis. Patients Community sample of 3752 men aged 70 years or older (Health in Men Study).
MAIN OUTCOME MEASURES
Fifteen-Item Geriatric Depression Scale and self-reported past or current treatment for depression (Health in Men Study).
RESULTS
In the Health in Men Study, the odds ratio (OR) of prevalent depression increased 4% (OR, 1.04; 95% confidence interval [CI], 1.02-1.05) with every unit increase of tHcy (micromoles per liter). The tHcy was 0.19 mg/L higher among participants with the MTHFR C677T TT genotype compared with the CC genotype. The meta-analysis showed that older adults with high tHcy had increased risk of depression (OR, 1.70; 95% CI, 1.38-2.08) and TT carriers were 22% more likely than CC carriers to have current depression or a history of depression (OR, 1.22; 95% CI, 1.01-1.47).
CONCLUSIONS
The triangular association between the MTHFR genotype, tHcy, and depression implies that higher concentrations of tHcy increase the risk of depression and that lowering tHcy by 0.19 mg/L could reduce the odds of depression by about 20%. Confirmatory data from sufficiently powered randomized trials of homocysteine-lowering therapy are now required to test if the relationship between tHcy and depression is truly causal.
Topics: Age Factors; Aged; Cohort Studies; Depressive Disorder; Folic Acid; Genetic Predisposition to Disease; Genotype; Homocysteine; Humans; Male; Methylenetetrahydrofolate Reductase (NADPH2); Odds Ratio; Personality Inventory; Polymorphism, Genetic; Pyridoxine; Randomized Controlled Trials as Topic; Risk Factors; Vitamin B 12
PubMed: 18981340
DOI: 10.1001/archpsyc.65.11.1286 -
The Cochrane Database of Systematic... Jan 2010Primary sclerosing cholangitis is a chronic cholestatic disease of intrahepatic and extrahepatic biliary ducts, characterised by chronic periductal inflammation and... (Meta-Analysis)
Meta-Analysis Review
BACKGROUND
Primary sclerosing cholangitis is a chronic cholestatic disease of intrahepatic and extrahepatic biliary ducts, characterised by chronic periductal inflammation and sclerosis of the ducts, which results in segmental stenoses of bile ducts, cholestasis, fibrosis, and ultimately, liver cirrhosis. Patients with primary sclerosing cholangitis are at higher risk of cholangiocarcinoma as well as of colonic neoplasia, since primary sclerosing cholangitis is associated with inflammatory bowel disease in more than 80% of the patients. Several therapeutic modalities have been proposed for primary sclerosing cholangitis, like ursodeoxycholic acid, glucocorticosteroids, and immunomodulatory agents, but none has been successful in reversing the process of the disease. To date, liver transplantation is the only definite therapeutic solution for patients with advanced primary sclerosing cholangitis with liver cirrhosis.
OBJECTIVES
To assess the beneficial and harmful effects of glucocorticosteroids for patients with primary sclerosing cholangitis.
SEARCH STRATEGY
We searched The Cochrane Hepato-Biliary Group Controlled Trials Register, The Cochrane Central Register of Controlled Trials in The Cochrane Library, MEDLINE, EMBASE, and LILACS from their inception until September 2009, as well as reference lists.
SELECTION CRITERIA
Randomised clinical trials comparing any dose or duration of glucocorticosteroids versus placebo, no intervention, or other immunosuppressive agents. We included trials irrespective of language, blinding, or publication status.
DATA COLLECTION AND ANALYSIS
Authors extracted data independently and assessed the methodological quality by the generation of the allocation sequence, allocation concealment, double blinding, follow-up, incomplete outcome data reporting, selective reporting, baseline imbalance, and early stopping. The results of the meta-analyses were presented as relative risks (RR) or mean difference (MD), both with 95% confidence intervals (CI). The primary outcome measures were mortality and liver-related morbidity.
MAIN RESULTS
Two randomised clinical trials were eligible for inclusion. One trial compared biliary lavage with hydrocortisone versus saline in 17 patients. Hydrocortisone tended to increase adverse events (pancreatitis, cholangitis with septicaemia, paranoid ideas, fluid retention) (RR 3.43, 95% CI 0.51 to 22.9) and had no cholangiographic improvement, which led to termination of the trial. The other trial compared budesonide versus prednisone in 18 patients. Patients had statistically significant higher serum bilirubin concentration after treatment with prednisone compared with budesonide (MD 10.4 micromol/litre, 95% CI 1.16 to 19.64 micromol/litre). No other statistically significant effects on clinical or biochemical outcomes were reported on any of the evaluated interventions.
AUTHORS' CONCLUSIONS
There is no evidence to support or refute peroral glucocorticosteroids for patients with primary sclerosing cholangitis. The intrabiliary application of corticosteroids via nasobiliary tube seems to induce severe adverse effects.
Topics: Anti-Inflammatory Agents; Budesonide; Cholangitis, Sclerosing; Humans; Hydrocortisone; Prednisone; Randomized Controlled Trials as Topic; Therapeutic Irrigation
PubMed: 20091555
DOI: 10.1002/14651858.CD004036.pub3 -
Intensive Care Medicine Jul 2002To determine the magnitude of the treatment effect of low-dose dopamine on renal function in patients at risk of and in patients with early renal injury. (Meta-Analysis)
Meta-Analysis Review
OBJECTIVE
To determine the magnitude of the treatment effect of low-dose dopamine on renal function in patients at risk of and in patients with early renal injury.
DATA SOURCES
MEDLINE, citation review of relevant primary and review articles, personal files, and contact with expert informants.
STUDY SELECTION
Randomized controlled studies that compared low-dose dopamine with placebo for the prevention or treatment of acute renal dysfunction. From 122 articles screened, 21 met the inclusion criteria for this meta-analysis. Of these six, were excluded.
DATA EXTRACTION
Fifteen studies containing 970 subjects were analyzed. Descriptive and outcome data were extracted. The main outcome measure was the absolute change in serum creatinine. In addition the number of patients who developed an acute decline in renal function was recorded. The meta-analysis was performed using the random effects model.
DATA SYNTHESIS
The meta-analysis demonstrated no significant difference between the absolute change in serum creatinine (5.1 micromol/l, 95% CI of -6.5 to +16.7) and the incidence of acute renal dysfunction (31% vs 33%, relative risk 1.01, 95% CI of 0.79-1.28) between those patients receiving low-dose dopamine and the control group. In addition, no sub-group of patients showed improved renal function with low-dose dopamine.
CONCLUSIONS
The results of this meta-analysis confirms that low-dose dopamine has no reno-protective effect. Considering the potential side-effects of dopamine this agent should not be used for this indication.
Topics: Creatine; Dopamine; Dose-Response Relationship, Drug; Female; Humans; Kidney; Kidney Function Tests; Male; Placebos; Randomized Controlled Trials as Topic; United States
PubMed: 12122525
DOI: 10.1007/s00134-002-1346-y -
Critical Care Medicine Jan 2003To summarize and compare different prognostic criteria used to determine need for liver transplantation in patients with fulminant hepatic failure secondary to... (Meta-Analysis)
Meta-Analysis Review
Fulminant hepatic failure secondary to acetaminophen poisoning: a systematic review and meta-analysis of prognostic criteria determining the need for liver transplantation.
OBJECTIVES
To summarize and compare different prognostic criteria used to determine need for liver transplantation in patients with fulminant hepatic failure secondary to acetaminophen poisoning.
DATA SOURCES
Studies published in the literature that investigated criteria for hepatic transplantation secondary to acetaminophen-induced liver failure as identified by a preestablished MEDLINE strategy (1966 through October 2001).
STUDY SELECTION
Studies were included if 2 x 2 tables could be reconstructed and if they did not assume that patients undergoing transplantation would have eventually died had they not received the transplant.
DATA EXTRACTION
Relevant articles were reviewed by two authors independently. Discrepancies or disagreements, if any, on the inclusion or exclusion of studies were resolved by consulting the third author.
DATA SYNTHESIS
King's criteria (pH < 7.30 or prothrombin time of >100 secs plus creatinine of >300 micromol/L plus encephalopathy grade of > or =3) were evaluated in nine studies, pH < 7.30 in four, prothrombin time of >100 secs in three, prothrombin time of >100 secs plus creatinine of >300 micromol/L plus encephalopathy grade of > or =3 in three, creatinine of >300 micromol/L in two, and one each for increase in prothrombin time day 4, factor V of <10%, Acute Physiology and Chronic Health Evaluation (APACHE) II score of >15, and Gc-globulin of <100 mg/L. King's criteria were more sensitive than pH: 69% (95% confidence interval, 63-75) vs. 57% (95% confidence interval, 44-68). Their specificities were, however, comparable: 92% (95% confidence interval, 81-97) vs. 89% (95% confidence interval, 62-97). APACHE II score of >15 had the highest positive likelihood ratio (16.4) and the lowest negative likelihood ratio (0.19) but was evaluated in only one study. The accuracy measures of all other criteria were lower than that of King's criteria or pH < 7.30.
CONCLUSIONS
Presently, available criteria are not very sensitive and may miss patients requiring transplantation. Future studies should further evaluate the efficacy of the APACHE II criteria.
Topics: Acetaminophen; Health Status Indicators; Humans; Liver Failure; Liver Transplantation; Patient Selection; Prognosis; Sensitivity and Specificity
PubMed: 12545033
DOI: 10.1097/00003246-200301000-00048 -
Molecular Genetics and Metabolism 2007Blood phenylalanine (Phe) levels provide a practical and reliable method for the diagnosis and monitoring of metabolic status in patients with phenylketonuria (PKU). To... (Meta-Analysis)
Meta-Analysis
Blood phenylalanine (Phe) levels provide a practical and reliable method for the diagnosis and monitoring of metabolic status in patients with phenylketonuria (PKU). To assess the reliability of blood Phe levels as a predictive biomarker of clinical outcomes in the development of treatments for PKU, a systematic literature review and meta-analysis of published trials of PKU, which included Phe level and neurological and dietary compliance outcome measures, was conducted. Within-study correlations between Phe level and intelligence quotient (IQ) were extracted from 40 studies. Significant, proportional correlations were found during critical periods (from 0 to 12 years of age) for early-treated patients with PKU (r=-0.35; 95% confidence interval [CI]: -0.44 to -0.27), where each 100 micromol/l increase in Phe predicted a 1.3- to 3.1-point reduction in IQ. Similar significant correlations were observed between IQ and mean lifetime Phe level for early-treated patients (r=0.34; 95% CI: -0.42 to -0.25), where each 100 micromol/l increase in Phe predicted a 1.9- to 4.1-point reduction in IQ. Moderate correlations were found between concurrent Phe level and IQ for early-treated patients. In conclusion, these results confirm a significant correlation between blood Phe level and IQ in patients with PKU, and support the use of Phe as a predictive biomarker for IQ in clinical trials.
Topics: Humans; Phenylalanine; Phenylketonurias
PubMed: 17591452
DOI: 10.1016/j.ymgme.2007.05.006