Unleashing patient voices: empowering adverse event assessment with complete patient-reported outcomes.

The 124-item patient reported-outcome common terminology criteria for adverse events (PRO-CTCAE) questionnaire, assessing 78 symptoms, is widely used in cancer clinical trials to identify side effects. However, its regular use in routine cancer care is rarely reported. We aimed to investigate the feasibility of weekly PRO-CTCAE completion over 9 weeks in a prospective study with 30 patients with cancer undergoing chemotherapy. Participants were asked to complete electronic surveys with reminders, but no feedback or incentives. Only 136 (50%) of the planned 270 time points at which a PRO-CTCAE self-report was expected were completed, with an additional 21 (8%) partially completed, and represents a failure to achieve the expected level of completion. Patients reported experiencing up to 51 and a median of 30 symptoms across all time points, highlighting the complexity of symptom assessment in acute cancer care. While weekly implementation of the PRO-CTCAE may not be feasible outside of clinical trial settings, this study highlights the breadth of symptoms experienced.

Examining ACCURE's Nurse Navigation Through an Antiracist Lens: Transparency and Accountability in Cancer Care.

There are persistent disparities in the delivery of cancer treatment, with Black patients receiving fewer of the recommended cancer treatment cycles than their White counterparts on average. To enhance racial equity in cancer care, innovative methods that apply antiracist principles to health promotion interventions are needed. The parent study for the current analysis, the Accountability for Cancer Care through Undoing Racism and Equity (ACCURE) intervention, was a system-change intervention that successfully eliminated the Black-White disparity in cancer treatment completion among patients with early-stage breast and lung cancer. The intervention included specially trained nurse navigators who leveraged real-time data to follow-up with patients during their treatment journeys. Community and academic research partners conducted thematic analysis on all clinical notes (n = 3,251) written by ACCURE navigators after each contact with patients in the specialized navigation arm (n = 162). Analysis was informed by transparency and accountability, principles adapted from the antiracist resource Undoing Racism and determined as barriers to treatment completion through prior research that informed ACCURE. We identified six themes in the navigator notes that demonstrated enhanced accountability of the care system to patient needs. Underlying these themes was a process of enhanced data transparency that allowed navigators to provide tailored patient support. Themes include (1) patient-centered advocacy, (2) addressing system barriers to care, (3) connection to resources, (4) re-engaging patients after lapsed treatment, (5) addressing symptoms and side effects, and (6) emotional support. Future interventions should incorporate transparency and accountability mechanisms and examine the impact on racial equity in cancer care.

The cancer nursing workforce in Australia: a national survey exploring determinants of job satisfaction.

BACKGROUND: To maintain and improve the quality of the cancer nursing workforce, it is crucial to understand the factors that influence retention and job satisfaction. We aimed to investigate the characteristics of cancer nurses in Australia and identify predictors of job satisfaction. METHODS: We analysed data from an anonymous cross-sectional survey distributed through the Cancer Nurses Society Australia membership and social media platforms from October 2021 to February 2022. The survey was compared to national nursing registration data. Data were analysed with non-parametric tests, and a stepwise, linear regression model was developed to best predict job satisfaction. RESULTS: Responses were received from 930 cancer nurses. Most respondents (85%) described themselves as experienced nurses, and more than half had post-graduate qualifications. We identified individual, organizational, and systemic factors that contribute to job satisfaction and can impact in workforce shortages. The findings include strategies to address and prioritize workforce challenges. There were 89 different titles for advanced practice nursing roles. Managing high workload was a reported challenge by 88%. Intention to stay less than 10 years was reported by nearly 60%; this was significantly correlated with job satisfaction and age. Significantly higher scores for job satisfaction were associated with those who had career progression opportunities, career development opportunities, adequate peer support and a clearly defined scope of role. Conversely, job satisfaction scores decreased the more people agreed there was a lack of leadership and they had insufficient resources to provide quality care. CONCLUSION: Cancer nurses are critical to the delivery of cancer care however, the workforce faces multiple challenges. This study provides an understanding of the Australian cancer nursing workforce characteristics, their roles and activities, and highlights important considerations for retaining nurses in the profession.

Development and validation of a novel model for characterizing migraine outcomes within real-world data.

BACKGROUND: In disease areas with 'soft' outcomes (i.e., the subjective aspects of a medical condition or its management) such as migraine or depression, extraction and validation of real-world evidence (RWE) from electronic health records (EHRs) and other routinely collected data can be challenging due to how the data are collected and recorded. In this study, we aimed to define and validate a scalable framework model to measure outcomes of migraine treatment and prevention by use of artificial intelligence (AI) algorithms within EHR data. METHODS: Headache specialists defined descriptive features based on routinely collected clinical data. Data elements were weighted to define a 10-point scale encompassing headache severity (1-7 points) and associated features (0-3 points). A test data set was identified, and a reference standard was manually produced by trained annotators. Automation (i.e., AI) was used to extract features from the unstructured data of patient encounters and compared to the reference standard. A threshold of 70% close agreement (within 1 point) between the automated score and the human annotator was considered to be a sufficient extraction accuracy. The accuracy of AI in identifying features used to construct the outcome model was also evaluated and success was defined as achieving an F1 score (i.e., the weighted harmonic mean of the precision and recall) of 80% in identifying encounters. RESULTS: Using data from 2,006 encounters, 11 features were identified and included in the model; the average F1 scores for automated extraction were 92.0% for AI applied to unstructured data. The outcome model had excellent accuracy in characterizing migraine status with an exact match for 77.2% of encounters and a close match (within 1 point) for 82.2%, compared with manual extraction scores-well above the 70% match threshold set prior to the study. CONCLUSION: Our findings indicate the feasibility of technology-enabled models for validated determination of soft outcomes such as migraine progression using the data elements typically captured in the real-world clinical setting, providing a scalable approach to credible EHR-based clinical studies.

The utility of the implementation science framework "Integrated Promoting Action on Research Implementation in Health Services" (i-PARIHS) and the facilitator role for introducing patient-reported outcome measures (PROMs) in a medical oncology outpatient department.

PURPOSE: We evaluated the utility of the implementation science framework "Integrated Promoting Action on Research Implementation in Health Services" (i-PARIHS) for introducing patient-reported outcome measures (PROMs) into a medical oncology outpatient department. The i-PARIHS framework identifies four core constructs for implementation, including Facilitation, Innovation, Context and Recipients. METHODS: A pilot study used the i-PARIHS framework to identify PROM implementation barriers and enablers to inform facilitation support strategies, such as training clinicians and staff, workflow support, technical support and audit and feedback. Pre- and post-implementation surveys were completed by 83 and 72 staff, respectively, (nurses, doctors and allied health), to assess perceived knowledge, enablers, barriers and utility of PROMs; and acceptability of the PROM intervention was also assessed post-implementation. RESULTS: Important barriers included time constraints and previous experiences with technology. Enablers included good leadership support and a culture of learning. Facilitation strategies were used to overcome barriers identified in the i-PARIHS core domains. Compared to before the intervention, staff surveys showed improvement in perceived usefulness, perceived understanding and interpretation skills for PROMs. Staff perceptions about lack of time to use PROMs during visits remained a major perceived barrier post-implementation. CONCLUSION: The i-PARIHS framework was useful for guiding the implementation of PROMs in routine oncology care. The four core i-PARIHS constructs (Facilitation, Innovation, Context and Recipients) identified factors that directly impacted implementation, with Facilitation having a particularly important role to overcome these barriers. Oncology clinics and health systems considering implementing PROMs should consider having a dedicated Facilitator available during PROM implementation.

Optimising symptom management in children with cancer using a novel mobile phone application: protocol for a controlled hybrid effectiveness implementation trial (RESPONSE).

BACKGROUND: Intense and aggressive treatment regimens for most children's cancer have achieved vast improvements in survival but are also responsible for both a high number and burden of symptoms. The use of Patient Reported Outcome Measures (PROMs) demonstrates a range of benefits for improved symptom management in adults with cancer. There are, however, multiple barriers to integrating PROMs into routine care in children and adolescents with cancer. This study aims to evaluate: (1) the effectiveness of electronic PROMs to generate stratified alerts, symptom management recommendations and graphical summaries (the RESPONSE system) to improve health outcomes and (2) the implementation of the RESPONSE system by assessing feasibility, acceptability, satisfaction, and sustainability. METHODS: A pragmatic hybrid II effectiveness-implementation controlled trial, using mixed methods, will be undertaken, advancing both knowledge of the effectiveness of the intervention and implementation factors. One-hundred and sixty children with cancer receiving active treatment will be recruited 1:1 to a non-randomised study involving two groups with an equal number of participants in each group. The intervention group (n = 80) will be prospectively recruited to receive the RESPONSE system intervention over eight weeks, versus the historical matched control group (n = 80) who will complete the ePROMs without access to the RESPONSE system. The primary outcome of the effectiveness trial is change between groups in total symptom burden. Secondary outcomes include child health-related quality-of-life and implementation outcomes. Trial data will be analysed using linear mixed-effects models. Formative implementation evaluation is informed by CFIR and ERIC frameworks and implementation outcomes will be mapped to the RE-AIM framework and include interviews, field notes, as well as administrative data to evaluate feasibility, acceptability, satisfaction and sustainability. TRIAL REGISTRATION NUMBER: ACTRN12621001084875 . Retrospectively Registered 16 August 2021.

Factors associated with time to first analgesic medication in the emergency department.

AIM AND OBJECTIVE: To examine the factors associated with time to first analgesic medication in the emergency department. BACKGROUND: Pain is the most common symptom presenting to the emergency department, and the time taken to deliver analgesic medication is a common outcome measure. Factors associated with time to first analgesic medication are likely to be multifaceted, but currently poorly described. DESIGN: Retrospective cohort study. METHODS: Cox proportional hazards regression modelling was undertaken to evaluate the associations between person, environment, health and illness variables within Symptom Management Theory and time to first analgesic medication in a sample of adult patients presenting with moderate-to-severe pain to an emergency department over twelve months. This study was completed in line with the STROBE statement. RESULTS: 383 patients were included in the study, 290 (75.92%) of these patients received an analgesic medication in a median time of 45 minutes (interquartile range, 70 minutes). A model containing nine explanatory variables associated with time to first analgesic medication was identified. These nine variables (employment status, discharge location, triage score, Charlson score, arrival pain score, socio-economic status, first location, daily total treatment time and patient time to be seen) represent all of the domains of the Symptom Management Theory. CONCLUSIONS: Person, environment, health and illness factors are associated with the time taken to deliver analgesic medication to those in pain in the emergency department. This study demonstrates the complexity of factors associated with pain care and the applicability of Symptom Management Theory to pain care in the emergency department. RELEVANCE TO CLINICAL PRACTICE: Identifying a model of factors that are associated with the time in which the most common symptom presenting to the emergency department is treated allows for targeted interventions to groups likely to receive poor care and a framework for its evaluation.

Phenotypic and molecular characteristics associated with various domains of quality of life in oncology patients and their family caregivers.

PURPOSE: Not all oncology patients and their family caregivers (FCs) experience the same quality of life (QOL). The purposes of this study were to identify latent classes of oncology patients (n = 168) and their FCs (n = 85) with distinct physical, psychological, social, and spiritual well-being trajectories from prior to through 4 months after the completion of radiation therapy and to evaluate for demographic, clinical, and genetic characteristics that distinguished between these latent classes. METHODS: Using growth mixture modeling, two latent classes were found for three (i.e., physical, psychological, and social well-being) of the four QOL domains evaluated. RESULTS: Across these three domains, the largest percentage of participants reported relatively high well-being scores across the 6 months of the study. Across these three QOL domains, patients and FCs who were younger, female, belonged to an ethnic minority group, had children at home, had multiple comorbid conditions, or had a lower functional status, were more likely to be classified in the lower QOL class. The social well-being domain was the only domain that had a polymorphism in nuclear factor kappa beta 2 (NFKB2) associated with latent class membership. Carrying one or two doses of the rare allele for rs7897947 was associated with a 54 % decrease in the odds of belonging to the lower social well-being class [OR (95 % CI) = .46 (.21, .99), p = .049]. CONCLUSIONS: These findings suggest that a number of phenotypic and molecular characteristics contribute to differences in QOL in oncology patients and their FCs.

Clinical and economic burden of idiopathic pulmonary fibrosis: a retrospective cohort study.

BACKGROUND: Idiopathic pulmonary fibrosis (IPF) is a devastating condition with a variable course. Not uncommonly, IPF patients are hospitalized for respiratory-related causes, including disease worsening. This study aimed to characterize the prevalence, and economic and health care burden of IPF. METHODS: Retrospective insurance claims data collected yearly between January 1, 2009 and December 31, 2011, were used to determine prevalence and calculate all-cause and respiratory-related resource utilization and costs. Patients had at least one inpatient claim or two outpatient claims for IPF (ICD-9-CM code 516.3). Results for health care burden are reported for the 2011 cohort (similar findings in 2009-2010). Costs are reported in 2011 US dollars ($). RESULTS: Patients with IPF had a mean age of 69.8-71.3 years. Overall prevalence for IPF was 28.8, 28.1 and 19.8 per 100,000 insured persons in 2009, 2010 and 2011. In each year, prevalence increased with age. In 2011, 37.7% of patients were hospitalized at least once for any cause; 19.5% for respiratory-related reasons. Also in 2011, the mean number of all-cause outpatient visits and respiratory-related office visits was 18.5 and 5.7 per patient, respectively. All-cause health care costs in 2011 were $59,379 per patient; 36.6% of costs ($21,732) were respiratory related. CONCLUSIONS: The prevalence of IPF in this claims database increased with age, with a notable increase in those over 65 years. IPF is associated with a large economic and health care burden. Additional research is needed to determine how such burden might be reduced.

Association between single-nucleotide polymorphisms in growth factor genes and quality of life in men with prostate cancer and the general population.

PURPOSE: Improved survival for men with prostate cancer has led to increased attention to factors influencing quality of life (QOL). As protein levels of vascular endothelial growth factor (VEGF) and insulin-like growth factor 1 (IGF-1) have been reported to be associated with QOL in people with cancer, we sought to identify whether single-nucleotide polymorphisms (SNPs) of these genes were associated with QOL in men with prostate cancer. METHODS: Multiple linear regression of two data sets (including approximately 750 men newly diagnosed with prostate cancer and 550 men from the general population) was used to investigate SNPs of VEGF and IGF-1 (10 SNPs in total) for associations with QOL (measured by the SF-36v2 health survey). RESULTS: Men with prostate cancer who carried the minor 'T' allele for IGF-1 SNP rs35767 had higher mean Role-Physical scale scores (≥0.3 SD) compared to non-carriers (p < 0.05). While this association was not identified in men from the general population, one IGF-1 SNP rs7965399 was associated with higher mean Bodily Pain scale scores in men from the general population that was not found in men with prostate cancer. Men from the general population who carried the rare 'C' allele had higher mean Bodily Pain scale scores (≥0.3 SD) than non-carriers (p < 0.05). CONCLUSIONS: Through identifying SNPs that are associated with QOL in men with prostate cancer and men from the general population, this study adds to the mapping of complex interrelationships that influence QOL and suggests a role for IGF-I in physical QOL outcomes. Future research may identify biomarkers associated with increased risk of poor QOL that could assist in the provision of pre-emptive support for those identified at risk.

Non-pharmacological interventions for chemotherapy-induced diarrhoea and constipation management: A scoping review.

PURPOSE: Chemotherapy-induced diarrhoea (CID) and constipation (CIC) are among the most common and severe gastrointestinal symptoms related to chemotherapy. This review aimed to identify and describe the evidence for non-pharmacological interventions for the management of CID and CIC. METHODS: The scoping review was based on the Joanna Briggs Institute methodology and reported in line with the Preferred Reporting Items for Systematic Reviews and Meta-Analyses extension for Scoping Reviews checklist. Evidence from five databases were included: CINAHL, MEDLINE, Embase, PubMed, and APA PsycInfo. Data were systematically identified, screened, extracted and synthesised narratively to describe the evidence for non-pharmacological interventions and their effects on CID and CIC. RESULTS: We included 33 studies, of which 18 investigated non-pharmacological interventions for CID management, six for CIC management, and nine for both CID and CIC management. Interventions were categorized into five groups, including (1) digital health interventions, (2) physical therapies, (3) diet and nutrition therapies, (4) education, and (5) multimodal. Diet and nutrition therapies were the most common to report potential effectiveness for CID and CIC outcomes. Most of the interventions were implemented in hospitals under the supervision of healthcare professionals and were investigated in randomised control trials. CONCLUSIONS: The characteristics of non-pharmacological interventions were diverse, and the outcomes were inconsistent among the same type of interventions. Diet and nutritional interventions show promise but further research is needed to better understand their role and to contribute to the evidence base. Nurses are well placed to assess and monitor for CIC and CID, and also deliver effective non-pharmacological interventions.

A Comparison of the Application of Two Different Scoring Systems in a Patient With Upper Limb Necrotizing Fasciitis.

Necrotizing fasciitis (NF) is a life-threatening soft-tissue infection that requires early recognition and surgical debridement to ensure the best outcome for patients. The Laboratory Risk Indicator for Necrotizing Fasciitis (LRINEC) score and the SIARI (Site other than lower limb, Immunosuppression, Age <60 years, Renal Impairment and Inflammatory markers) score are clinical predictor tools that can aid in the timely diagnosis of NF. This case report discusses a male patient who presented with a rash on his arm that was initially thought to be cellulitis. It examines how the application of scoring systems can be beneficial for earlier identification or when the diagnosis is uncertain.

Differences in the symptom experience of older versus younger oncology outpatients: a cross-sectional study.

BACKGROUND: Mortality rates for cancer are decreasing in patients under 60 and increasing in those over 60 years of age. The reasons for these differences in mortality rates remain poorly understood. One explanation may be that older patients received substandard treatment because of concerns about adverse effects. Given the paucity of research on the multiple dimensions of the symptom experience in older oncology patients, the purpose of this study was to evaluate for differences in ratings of symptom occurrence, severity, frequency, and distress between younger (< 60 years) and older ( ≥ 60 years) adults undergoing cancer treatment. We hypothesized that older patients would have significantly lower ratings on four symptom dimensions. METHODS: Data from two studies in the United States and one study in Australia were combined to conduct this analysis. All three studies used the MSAS to evaluate the occurrence, severity, frequency, and distress of 32 symptoms. RESULTS: Data from 593 oncology outpatients receiving active treatment for their cancer (i.e., 44.4% were < 60 years and 55.6% were ≥ 60 years of age) were evaluated. Of the 32 MSAS symptoms, after controlling for significant covariates, older patients reported significantly lower occurrence rates for 15 (46.9%) symptoms, lower severity ratings for 6 (18.9%) symptoms, lower frequency ratings for 4 (12.5%) symptoms, and lower distress ratings for 14 (43.8%) symptoms. CONCLUSIONS: This study is the first to evaluate for differences in multiple dimensions of symptom experience in older oncology patients. For almost 50% of the MSAS symptoms, older patients reported significantly lower occurrence rates. While fewer age-related differences were found in ratings of symptom severity, frequency, and distress, a similar pattern was found across all three dimensions. Future research needs to focus on a detailed evaluation of patient and clinical characteristics (i.e., type and dose of treatment) that explain the differences in symptom experience identified in this study.

The effects of cancer clinical decision support systems on patient-reported outcomes: A systematic review.

PURPOSE: The implementation of high-quality decision-making support are integral to ensuring the delivery of quality cancer care and subsequently achieving positive patient outcomes. Decision Support Systems (DSS) are increasingly used, however it is not known what the effects are beyond supporting the decision-making process. We aimed to identify and synthesize the available literature regarding the effects of DSS on patient-reported outcomes both during and after cancer treatment. METHODS: A systematic review was conducted using dual processes to identify empirical literature that reported an evaluation of DSS interventions and patient-reported outcomes. We appraised study quality using the Mixed Methods Appraisal Tool (MMAT). Data were narratively synthesized. RESULTS: We included 15 studies, categorized as symptom assessment interventions or interactive educational interventions. Findings were mixed regarding the effectiveness of DSS interventions in improving total symptom distress and severity, whereas the majority were effective in reducing mean scores for worst and usual pain. Interventions were not effective in improving other health-related patient-reported outcomes including quality of life, global distress, depression, or self-efficacy and there were mixed effects for reducing decisional conflict. There was moderate to high patient adherence to the interventions and generally high satisfaction and acceptability, yet minimal evidence for the effect of DSS interventions in clinician adherence to intervention recommendations. CONCLUSIONS: Including patient-reported outcomes in the evaluation of DSS is critical to understand their impact. Inconsistencies in reporting of interventions may, however, be a contributing factor to heterogeneous effects of clinical DSS regarding a broad range of patient-reported outcomes.

Energy conservation education intervention for people with end-stage kidney disease receiving haemodialysis (EVEREST): protocol for a cluster randomised control trial.

INTRODUCTION: Multiple symptoms occur in people with kidney failure receiving haemodialysis (HD) and these symptoms have a negative impact on health-related quality of life (HRQoL). Fatigue, the most common symptom, is debilitating and difficult to manage. Educational interventions involving energy conservation strategies are helpful in reducing fatigue, however the effectiveness of energy conservation has not been previously studied in those receiving HD. The aim of this study is to evaluate the effectiveness of an energy conservation education intervention for people with end-stage kidney disease receiving HD (EVEREST trial). METHODS AND ANALYSIS: A pragmatic cluster randomised control trial with repeated measure will be used. One hundred and twenty-six participants from tertiary level dialysis centre will be cluster randomised to the intervention and control group according to HD treatment day. The intervention group will receive usual care along with a structured energy conservation education programme over 12 weeks comprising three individual face-to-face educational intervention sessions, one booster session and a booklet. The control group will receive usual care from their healthcare providers and a booklet at the end of the study. The primary outcome is fatigue, and the secondary outcomes are other Chronic Kidney Disease (CKD) symptoms, occupational performance and HRQoL. Intention-to-treat analysis will occur and will include a change in primary and secondary outcomes. ETHICS AND DISSEMINATION: Ethical approval has been obtained from the Human Research Committee of the Griffith University and Nepal Health Research Council. The results of this research will be published and presented in a variety of forums. TRIAL REGISTRATION NUMBER: NCT04360408.

Persistent Symptoms, Quality of Life, and Correlates with Health Self-Efficacy in Adolescent and Young Adult Survivors of Childhood Cancer.

Purpose: The numbers of adolescent and young adult (AYA) survivors of childhood cancer are exponentially growing. To ensure suitable services are available to meet the needs of this growing population, understanding the experience of late effects, quality of life, and potentially modifiable factors, such as self-efficacy, is required. Methods: AYA survivors of childhood cancer recruited through an After Cancer Therapy Service at a Children's Hospital rated their symptoms experience, quality of life, and self-efficacy using the Patient Reported Outcome Common Terminology Criteria for Adverse Events, Functional Assessment of Cancer Therapy-General (FACT-G), and Patient-Reported Outcomes Measurement Information System (PROMIS®), respectively. Descriptive statistics were used to characterize the sample. Quality-of-life scores were compared with population norms. Regression analyses were used to explore the relationships between symptom experience, quality of life, and self-efficacy. Results: Thirty participants (mean age 22 ± 4.4 years) reported an average of nine symptoms as persistently experienced at moderate or higher rated intensity among participants (standard deviation ±8.7; range: 0-32; interquartile range: 2-16); over half (n = 17, 56.7%) had finished treatment 10 or more years ago. Participants scored lower on the FACT-G Physical Well-being and Emotional Well-being, and higher on Social Well-being subscales than the general population. Around two-thirds of participants were confident in their ability to self-manage their health based on their health self-efficacy score. Bivariate linear regression identified a statistically significant increase in the overall quality of life with increased self-efficacy, adjusted for age and sex (0.60, 95% confidence interval [CI] 0.30-0.90, p < 0.01). Higher symptom burden was associated with a lower overall quality of life after adjusting for age and sex (-0.95, 95% CI: -1.35 to -0.54, p < 0.001). Conclusion: Young cancer survivors experience a substantial number of persistent symptoms related to their cancer treatment that may negatively impact aspects of their quality of life. Health self-efficacy is a potential target for future interventions.

Intracranial hypertension in pediatric patients treated with recombinant human growth hormone: data from 25 years of the Genentech National Cooperative Growth Study.

Intracranial hypertension (IH) is a rare condition in children. However, a relationship between recombinant human growth hormone (rhGH) therapy and IH has been well documented. Risk factors were assessed for 70 rhGH-naive patients enrolled in the National Cooperative Growth Study with reports of IH after treatment initiation. Patients with severe growth hormone deficiency, Turner syndrome, chronic renal insufficiency (CRI), and obesity (particularly in the CRI group) were at highest risk of developing IH during the first year of therapy, suggesting initiation of careful early monitoring. In some patients, factors such as corticosteroid use or other chromosomal abnormalities appear to confer a delayed risk of IH, and these patients should be monitored long-term for signs and symptoms of IH.

Birth prevalence of phenylalanine hydroxylase deficiency: a systematic literature review and meta-analysis.

BACKGROUND: Phenylalanine hydroxylase (PAH) deficiency is an autosomal recessive disorder that results in elevated concentrations of phenylalanine (Phe) in the blood. If left untreated, the accumulation of Phe can result in profound neurocognitive disability. The objective of this systematic literature review and meta-analysis was to estimate the global birth prevalence of PAH deficiency from newborn screening studies and to estimate regional differences, overall and for various clinically relevant Phe cutoff values used in confirmatory testing. METHODS: The protocol for this literature review was registered with PROSPERO (International prospective register of systematic reviews). Pubmed and Embase database searches were used to identify studies that reported the birth prevalence of PAH deficiency. Only studies including numeric birth prevalence reports of confirmed PAH deficiency were included. RESULTS: From the 85 publications included in the review, 238 birth prevalence estimates were extracted. After excluding prevalence estimates that did not meet quality assessment criteria or because of temporal and regional overlap, estimates from 45 publications were included in the meta-analysis. The global birth prevalence of PAH deficiency, estimated by weighting regional birth prevalences relative to their share of the population of all regions included in the study, was 0.64 (95% confidence interval [CI] 0.53-0.75) per 10,000 births and ranged from 0.03 (95% CI 0.02-0.05) per 10,000 births in Southeast Asia to 1.18 (95% CI 0.64-1.87) per 10,000 births in the Middle East/North Africa. Regionally weighted global birth prevalences per 10,000 births by confirmatory test Phe cutoff values were 0.96 (95% CI 0.50-1.42) for the Phe cutoff value of 360 ± 100 µmol/L; 0.50 (95% CI 0.37-0.64) for the Phe cutoff value of 600 ± 100 µmol/L; and 0.30 (95% CI 0.20-0.40) for the Phe cutoff value of 1200 ± 200 µmol/L. CONCLUSIONS: Substantial regional variation in the birth prevalence of PAH deficiency was observed in this systematic literature review and meta-analysis of published evidence from newborn screening. The precision of the prevalence estimates is limited by relatively small sample sizes, despite widespread and longstanding newborn screening in much of the world.

Occurrence and perceived effectiveness of activities used to decrease chemotherapy-induced peripheral neuropathy symptoms in the feet.

PURPOSE: Investigate the reported use and perceived effectiveness of self-care activities for chemotherapy-induced peripheral neuropathy (CIPN) symptoms in the feet. METHODS: Cancer survivors with CIPN (n = 405) completed a questionnaire that assessed the use and perceived effectiveness of 25 self-care activities. Effectiveness was rated on a 0 (not at all) to 10 (completely effective) numeric rating scale. Descriptive statistics and regression analyses were conducted to identify demographic, clinical, and pain characteristics associated with the use and effectiveness of selected self-care activities. RESULTS: The five most commonly used activities were: went for a walk (73.8%); watched television (67.8%); read a book, newspaper or magazine (64.4%); listened to radio, music (60.0%); and did exercises (jogging, swimming) (58.6%). The five most effective self-care activities were: had a trigger point injection (8.3 ( ± 1.3)); took tranquilizers (4.8 ( ± 2.6)); went for ultrasonic stimulation treatment (4.3 ( ± 3.1)); used a heating pad or hot water bottle (4.3 ( ± 2.5)); and used a transcutaneous electric nerve stimulator (4.2 ( ± 2.6)). Demographic, clinical, and pain characteristics influenced use and perceived effectiveness of selected self-care activities to varying degrees. CONCLUSIONS: Two-thirds of the survivors used at least seven self-care activities to manage CIPN symptoms. The most commonly used activities did not receive the highest effectiveness ratings. Some activities that were rated as highly effective warrant more rigorous evaluation. Survivors can try a range of activities to decrease CIPN symptoms in the feet following discussion of their potential risks and benefits with their clinicians.

Evaluation of a new culture medium for isolation of nontuberculous mycobacteria from environmental water samples.

Nontuberculous mycobacteria (NTM) are waterborne pathogens commonly found in building water systems where they are a primary concern to vulnerable patient populations and can cause severe disease. The recovery of NTM from environmental samples can be a laborious undertaking and current pre-treatment methods and selective media lack sensitivity. We explored the use of the highly selective Rapidly Growing Mycobacteria (RGM) medium for culturing NTM from environmental water samples compared to existing methods. In total, 223 environmental water samples, including potable and non-potable water, were cultured for NTM using three culture media. In addition to direct culture on RGM medium, each sample was cultured on Middlebrook 7H10 medium and Mitchison 7H11 medium after pre-treatment with 0.2M KCl-HCl. Additionally, 33 distinct species of NTM were inoculated onto RGM medium and 7H10 medium in parallel to directly compare their growth. The use of RGM medium alone without pre-treatment provided a sensitivity (91%) comparable to that offered by culture on both 7H10 and 7H11 with acid pretreatment (combined sensitivity; 86%) with significantly less overgrowth and interference from other organisms on RGM medium. The average concentration of NTM observed on RGM medium alone was comparable to or greater than the NTM concentration on either medium alone or combined. Thirty-three species were examined in parallel and all tested strains of 27 of these species successfully grew on RGM medium, including 19 of 21 from the CDC's healthcare-associated infections species list. RGM medium was successful at recovering environmental NTM without a pre-treatment, greatly reducing labor and materials required to process samples. Simplification of culture processing for environmental NTM will allow for a better assessment of their presence in building water systems and the potential for reduced exposure of susceptible populations.