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In phenylketonuria (PKU), variable dietary advice provided by health professionals and social media leads to uncertainty for patients/caregivers reliant on accurate, evidence based dietary information. Over four years, 112 consensus statements concerning the allocation of foods in a low phenylalanine diet for PKU were developed by the British Inherited Metabolic Disease Dietitians Group (BIMDG-DG) from 34 PKU treatment centres, utilising 10 rounds of Delphi consultation to gain a majority (≥75%) decision. A mean of 29 UK dietitians (range: 18–40) and 18 treatment centres (range: 13–23) contributed in each round. Statements encompassed all foods/food groups divided into four categories based on defined protein/phenylalanine content: (1) foods high in protein/phenylalanine (best avoided); (2) foods allowed without restriction including fruit/vegetables containing phenylalanine ≤75 mg/100 g and most foods containing protein ≤0.5 g/100 g; (3) foods that should be calculated/weighed as an exchange food if they contain protein exchange ingredients (categorized into foods with a protein content of: >0.1 g/100 g (milk/plant milks only), >0.5 g/100 g (bread/pasta/cereal/flours), >1 g/100 g (cook-in/table-top sauces/dressings), >1.5 g/100 g (soya sauces)); and (4) fruit/vegetables containing phenylalanine >75 mg/100 g allocated as part of the protein/phenylalanine exchange system. These statements have been endorsed and translated into practical dietary management advice by the medical advisory dietitians for the National Society for PKU (NSPKU).

Background: Metabolic control in phenylketonuria (PKU) is known to deteriorate with age, but national-level data describing blood phenylalanine (Phe) control across the United Kingdom (UK) are limited. Objective: To characterise blood Phe control in individuals with PKU attending UK metabolic centres. Methods: Sixteen UK centres (nine paediatric, six adult, one mixed) retrospectively extracted blood Phe results collected between 2012 and 2018. Demographic, phenotypic and monitoring-related variables were analysed. Written consent for data collection was obtained from all patients or their caregivers. Results: Data were available for 871 individuals (55% female), of whom 744 (85%) were classified as follows: classical PKU, 75%, mild PKU, 22% and hyperphenylalaninaemia, 3%. Mean blood Phe concentrations were significantly higher in adults than children (491 ± 308 vs. 303 ± 199 µmol/L; p < 0.001), and the proportion of samples within target range declined steadily with age, from 78% in children < 2 years to 36% in adults ≥ 41 years. Individuals with classical PKU had higher mean Phe concentrations and lower target attainment than those with HPA (386 vs. 300 µmol/L; 61% vs. 78%; p < 0.001), while mild PKU and HPA showed comparable control. Females generally demonstrated better metabolic control than males. More frequent dried blood spot sampling for blood Phe was strongly associated with improved metabolic control: weekly (254 ± 175 µmol/L; 82% within target), fortnightly (319 ± 207 µmol/L; 70%), monthly (397 ± 231 µmol/L; 61%), and less than monthly (624 ± 349 µmol/L; 44%). Nearly half of the blood Phe samples (47%) with recorded timing were taken in a non-fasting state. Conclusions: Achieving lifelong metabolic stability on a Phe-restricted diet alone remains challenging. These national data highlight the need for broader therapeutic options to support individuals with PKU across the lifespan.

Patients with phenylketonuria (PKU) are reliant on special low protein foods (SLPFs) as part of their dietary treatment. In England, several issues regarding the accessibility of SLPFs through the national prescribing system have been highlighted. Therefore, prescribing patterns and expenditure on all SLPFs available on prescription in England (n = 142) were examined. Their costs in comparison to regular protein-containing (n = 182) and ‘free-from’ products (n = 135) were also analysed. Similar foods were grouped into subgroups (n = 40). The number of units and costs of SLPFs prescribed in total and per subgroup from January to December 2020 were calculated using National Health Service (NHS) Business Service Authority (NHSBSA) ePACT2 (electronic Prescribing Analysis and Cost Tool) for England. Monthly patient SLPF units prescribed were calculated using patient numbers with PKU and non-PKU inherited metabolic disorders (IMD) consuming SLPFs. This was compared to the National Society for PKU (NSPKU) prescribing guidance. Ninety-eight percent of SLPF subgroups (n = 39/40) were more expensive than regular and ‘free-from’ food subgroups. However, costs to prescribe SLPFs are significantly less than theoretical calculations. From January to December 2020, 208,932 units of SLPFs were prescribed (excluding milk replacers), costing the NHS £2,151,973 (including milk replacers). This equates to £962 per patient annually, and prescribed amounts are well below the upper limits suggested by the NSPKU, indicating under prescribing of SLPFs. It is recommended that a simpler and improved system should be implemented. Ideally, specialist metabolic dietitians should have responsibility for prescribing SLPFs. This would ensure that patients with PKU have the necessary access to their essential dietary treatment, which, in turn, should help promote dietary adherence and improve metabolic control.

Funding Information: Conflicts of Interest: A.M. (Anita MacDonald) is a member of, the advisory board ELEMENT Danone-Nutricia, the advisory board for Arla and Applied Pharma Research, and received research funding and honoraria from Nutricia, Vitaflo International, Biomarin, MetaHealth, Metax and Merck Serono. S.E. receives research funding from Nutricia, and has received financial support and honoraria from Nutricia and Vitaflo to attend/speak at study days and conferences. A.P. received an educational grant from Cambrooke Therapeutics and grants from Vitaflo International, Nutricia, Merck Serono, Biomarin and Mevalia to attend scientific meetings. A.D. received research funding from Vitaflo International, financial support from Nutricia, Mevalia and Vitaflo International to attend study days and conferences. J.C.R. is a member of the European Nutritionist Expert Panel (Biomarin), the Advisory Board for Applied Pharma Research and Nutricia, and has received honoraria as a speaker from APR, Merck Serono, Biomarin, Nutricia, Vitaflo, Cambrooke, PIAM, and Lifediet. S.F. (Suzanne Ford) is a member of the advisory board for Nutricia, and MetaHealth and has received financial support and honoraria from Cambrooke and Vitaflo. R.S. has received sponsorship to attend conferences and study days, payment to present at conferences from Nutricia Metabolics, Vitaflo Internation and Mevalia. C.E. has received honoraria and educational grants to attend events from Vitaflo, Nutricia, Meta Healthcare and SOBI and is a member of the Advisory Commitee on Borderline Substances. S.F. (Sarah Firman) has received funding to attend conferences and study days from Nutricia, Vitaflo International and Dr. Schär UK Ltd., and consulting fees from Vitaflo International and Meta Healthcare Ltd. C.N. has received financial support and honoraria from Nutricia and Vitaflo to attend/speak at study days and conferences. L.R. is a member of the Nutricia adult advisory board and received honorarium from Nutricia and Vitaflo in the past. F.J.W. receives honoraria, educational and travel grants from Nutricia and Vitaflo. M.H. is a member of advisory board Nutricia Danone and Applied Pharma and has received financial support and honoraria from Nutricia, Vitaflo, Cambrooke, Mevalia, Promin for attendance at conferences/meetings and speakers fee. A.T. (Allyson Terry) has received payment from Vitaflo International for speaking at a patient event. K.vW. receives honoraria and educational grants from Nutricia, Vitaflo and Meta Healthcare. S.T. is expected to receive honoraria from Vitaflo International for providing feedback and reviewing guidelines for PKU Sphere. G.W., M.F., L.G., C.P., S.A., A.M. (Anita MacDonald), A.T. (Alison Tooke), A.W., E.G., L.W., I.H., H.S., J.G., A.E., S.C., R.P. have no conflicts of interest to declare. Publisher Copyright: © 2021 by the authors. Licensee MDPI, Basel, Switzerland. Patients with phenylketonuria (PKU) are reliant on special low protein foods (SLPFs) as part of their dietary treatment. In England, several issues regarding the accessibility of SLPFs through the national prescribing system have been highlighted. Therefore, prescribing patterns and expenditure on all SLPFs available on prescription in England (n = 142) were examined. Their costs in comparison to regular protein-containing (n = 182) and ‘free-from’ products (n = 135) were also analysed. Similar foods were grouped into subgroups (n = 40). The number of units and costs of SLPFs prescribed in total and per subgroup from January to December 2020 were calculated using National Health Service (NHS) Business Service Authority (NHSBSA) ePACT2 (electronic Prescribing Analysis and Cost Tool) for England. Monthly patient SLPF units prescribed were calculated using patient numbers with PKU and non-PKU inherited metabolic disorders (IMD) consuming SLPFs. This was compared to the National Society for PKU (NSPKU) prescribing guidance. Ninety-eight percent of SLPF subgroups (n = 39/40) were more expensive than regular and ‘free-from’ food subgroups. However, costs to prescribe SLPFs are significantly less than theoretical calculations. From January to December 2020, 208,932 units of SLPFs were prescribed (excluding milk replacers), costing the NHS £2,151,973 (including milk replacers). This equates to £962 per patient annually, and prescribed amounts are well below the upper limits suggested by the NSPKU, indicating under prescribing of SLPFs. It is recommended that a simpler and improved system should be implemented. Ideally, specialist metabolic dietitians should have responsibility for prescribing SLPFs. This would ensure that patients with PKU have the necessary access to their essential dietary treatment, which, in turn, should help promote dietary adherence and improve metabolic control.

To determine if text message–based behavioral interventions could increase response rates to a patient-reported outcomes survey in the emergency department (ED). We conducted a study within a trial (SWAT), within the NUDG-ED trial. The NUDG-ED trial aimed to reduce low-value care for patients with back pain presenting to eight EDs in Sydney, Australia. This SWAT was a 3-arm randomized controlled trial (RCT) nested within the NUDG-ED trial. After discharge from the ED, patients were randomized to receive one of the three text message invitations to complete a follow-up patient-reported outcome survey: a standard control message, or one of two behaviorally informed messages including either a prize draw incentive or prosocial framing. Our primary outcome measure was the response rate in each study group. We performed a linear mixed-effects model controlling for hospital heterogeneity and patient characteristics to estimate the mean difference (MD) in proportions with 95% CI, to determine the effectiveness of the behavioral interventions. A total of 1494 patients were randomized between May 15, 2024 and January 29, 2025. Of these, 52% were women, the median age was 46 years (IQR 35, 62), 43% were from disadvantaged areas and 51% were triaged with a clinically urgent condition. Baseline characteristics were balanced across all groups. Our primary analysis found that compared to the control, the prize draw incentive increased response rates (n = 997 patients, MD = 6.9%, 95% CI: 1.8% to 11.9%, P = .007). Our adjusted mixed-effects model also found a significant increase in response rates (n = 979 patients, MD = 6.4%, 95% CI 1.3% to 11.4%, P = .013). Compared to the control, the prosocial framing message may have slightly increased response rates, but the results were not statistically significant (n = 996 patients, 17.2% vs 21.1%, MD = 3.9%, 95% CI: −1.1% to 8.9%). In this randomized trial, a prize draw incentive modestly improved response rates to a patient-reported outcomes survey in routine emergency care settings. Prosocial framing may have slightly increased response rates, but the effect was uncertain. Both behavioral approaches warrant further testing in routine care settings. Patient-reported outcomes, such as surveys about how people feel and recover after care, are important for understanding what matters most to patients. However, response rates to these surveys are often very low, especially in real clinical settings. This makes it difficult to draw strong conclusions about whether treatments are helping patients. So, researchers and health services need to find ways to improve response rates. This study looked at whether simple text message strategies could encourage more patients to complete follow-up surveys after visiting the ED for low back pain. We conducted a RCT involving 1494 patients in Sydney. After being discharged from the ED, patients were randomly assigned to receive one of the three text message invitations to complete a survey: a standard message, a message offering entry into a prize draw, or a message using prosocial wording (emphasizing how their response could help others). We found that patients who received the prize draw message were more likely to complete the survey. About 24% responded to this message, compared to 17% who received the standard message, an increase of around 7%. We are uncertain whether the prosocial message was effective. These findings suggest that adding a prize draw incentive to a text message-based study invitation can modestly increase survey participation in routine health-care settings. Improving response rates helps ensure that patient perspectives are more accurately represented in research and health-care improvement efforts. Further studies are needed to confirm these results and explore other effective ways to increase participation. [Display omitted] Key findings•Response rates to patient-reported outcome surveys are typically low.•Text message based behavioral strategies increased response rates in emergency care patients. What this adds to what is known?•This study shows that behavioral strategies are feasible and effective in clinical settings. What is the implication and what should change now?•Such strategies could enhance data quality and facilitate patient-centred evaluations of care.

IntroductionArtemisinin-based combination therapies (ACTs) remain the WHO-recommended treatment for uncomplicated Plasmodium falciparum malaria. However, the emergence and spread of artemisinin resistance (ART-R) threatens ACT efficacy. ART-R is phenotypically expressed as delayed parasite clearance, which can facilitate ACT partner drug resistance. ART-R has been causally linked to specific mutations in the Pfkelch13 gene.Methods and analysisThe systematic review and associated meta-analysis aim to determine the correlation between Pfkelch13 (alleles present in the Kelch13 gene region of the P. falciparum parasite) genotypes and clinical and parasitological response to ACTs from a globally representative data set pooling individual patient data (IPD) from eligible published and unpublished studies. The eligibility criteria include Pfkelch13 genotyping results at baseline complemented by individually linked parasitological and clinical assessments following artemisinin-based treatment. The data will be curated, standardised and analysed using this proposed statistical analysis plan (SAP), adhering to PRISMA-IPD (PRISMA, Preferred Reporting Items for Systematic reviews and Meta-Analyses) guidelines. Our SAP will apply hierarchical modelling to assess the effect of the P. falciparum parasite Pfkelch13 mutations on parasite clearance half-life and therapeutic efficacy across different regions. This will include study sites as random effects in the model and potential predictors such as age, sex, baseline parasite load and other potential effect modifiers as fixed effects. This analysis will enhance the understanding of the influence of Pfkelch13 mutations on malaria treatment outcomes.Ethics and disseminationData were obtained with informed consent and ethical approvals from the relevant countries and were pseudonymised before curation in the Infectious Diseases Data Observatory (IDDO)/WorldWide Antimalarial Resistance Network (WWARN) repository. Data ownership remains with contributors. This IPD meta-analysis met the Oxford Tropical Research Ethics Committee criteria for waiving ethical review, as it is a secondary analysis of existing pseudonymised data. The resulting peer-reviewed publication and conference proceedings will help strengthen and enhance the efficiency of ART-R surveillance and response and support policy decisions.PROSPERO registration numberCRD42019133366.

We compared the change in child household contact management of pulmonary tuberculosis (TB) cases before and after the implementation of an isoniazid preventive therapy (IPT) register in an urban clinic setting in Cape Town, South Africa. We determined if the presence of an IPT register was associated with an increase in the number of child contacts identified per infectious case and the proportion of identified children who were started on IPT. We reviewed routine programme data on IPT delivery to children during two time periods (May 2008-October 2008 and May 2011-October 2011), before and after the implementation of an IPT register used by routine clinic personnel. Adult TB case demographic and clinical characteristics from the two observation periods were similar. During the post-register period, more child contacts per adult case were identified (0.7 (54 children) vs. 0.3 (24 children)), more of the identified children were started on IPT (54 vs. 4) and 37% of those who started, completed six months of treatment compared to the pre-register period where no adherence information was recorded. After pilot implementation of an IPT register, documented identification of child contacts, IPT initiation and IPT adherence documentation in TB exposed children was improved. Our findings support further exploration of the potential impact of using standardised IPT recording and reporting in routine clinics in high-burden TB settings to improve TB prevention efforts targeted at young children. Future efforts to improve IPT delivery should be systematic and comprehensive in order to support a change in current operational IPT delivery practices in TB programs.

Background : Increasing urbanisation and rising unhealthy lifestyle risk factors are contributing to a growing diabetes epidemic in South Africa. In 2000, a study estimated diabetes prevalence to be 5.5% in those aged over 30. Accurate, up-to-date information on the epidemiology and burden of disease due to diabetes and its sequelae is essential in the planning of health services for diabetes management. Objective : To calculate the non-fatal burden of disease in Years Lost due to Disability (YLD) due to diabetes and selected sequelae in South Africa in 2009. YLD measures the equivalent loss of life due to ill-health. Methods : A series of systematic literature reviews identified data on the epidemiology of diabetes and its sequelae in South Africa. The data identified were then applied to Global Burden of Disease (GBD) methodology to calculate the burden attributable to diabetes. Results : Prevalence of type 2 diabetes in South Africa in 2009 is estimated at 9.0% in people aged 30 and older, representing approximately 2 million cases of diabetes. We modelled 8,000 new cases of blindness and 2,000 new amputations annually caused by diabetes. There are 78,900 YLD attributed to diabetes, with 64% coming from diabetes alone, 24% from retinopathy, 6% from amputations, 9% from attributable stroke disability, and 7% from attributable ischemic heart disease disability. Conclusions : We estimate that the prevalence of diabetes is increasing in South Africa. Significant disability associated with diabetes is demonstrated. Some of the attributed burden can be prevented through early detection and treatment. Appendices available online under Reading Tools.

Background: Low-value care exposes patients to unnecessary risk and wastes scarce health resources. We aimed to determine if patient- or clinician-directed nudges could reduce low-value care for low back pain in the emergency department. Methods: We conducted a 2 * 2 factorial, cluster randomized controlled trial involving patients with low back pain presenting to emergency departments. Eight emergency departments were randomized to receive patient nudges (6 electronic information posters discouraging unnecessary imaging and opioids, displayed on 55-inch screens in waiting rooms), clinician nudges (3 electronic health record alerts that provided indications for lumbar spine imaging and suggested alternatives to opioids), both patient and clinician nudges, or no nudges. The primary outcome was the proportion of encounters for low back pain with low-value care, defined as non-indicated lumbar spine imaging test, opioid prescription at discharge, or both. We calculated odds ratios (ORs), adjusted for baseline and clustering. Results: There were 3770 encounters for low back pain during the study period. The overall baseline prevalence of low-value care was 41.6%. During the intervention period, the proportion of encounters with low-value care reduced to 36.4% with patient nudges versus 38.1% without, but the difference was not significant (adjusted OR 0.80, 95% confidence interval [CI] 0.51 to 1.27). The proportion of encounters with low-value care was 39.4% with clinician nudges versus 35.0% without (adjusted OR 1.31, 95% CI 0.84 to 2.05). We did not observe an interaction effect between the interventions (p = 0.4). The patient nudge may have reduced strong beliefs among patients in the value of imaging for low back pain. We found no important differences in secondary outcomes. Interpretation: Nudges--including waiting room information posters targeting patients and electronic health record alerts targeting clinicians--did not reduce low-value care in emergency departments. Trial registration: www.anzctr.org.au, ACTRN12623001000695