Nevin Manimala

J Pediatr Gastroenterol Nutr. 2022 Apr 20. doi: 10.1097/MPG.0000000000003456. Online ahead of print.

ABSTRACT

BACKGROUND: Ileocecectomy related to stricturing, fistula formation, or medically refractory disease is commonly required in patients with Crohn’s disease (CD). Limited research exists in endoscopic recurrence (ER) in pediatric inflammatory bowel disease (IBD). In this study, we sought to determine ER rates and the impact of therapy duration prior to surgery in pediatric patients with CD.

METHODS: This was a single-center retrospective review of patients with CD between the ages of 2-20 years who required ileocecectomy between January 2015 and December 2019 at Nationwide Children’s Hospital. Follow-up endoscopies, laboratory values, medications, and sPCDAI scores were recorded at 6, 12, 24, and 36 months post-resection where available. Modified Rutgeert scores (mRS) were independently assigned to post-resection colonoscopy images by 3 trained investigators. Post-resection outcomes were compared between patients on CD therapy >30 days prior to resection (late surgery) to those started on CD therapy <30 days prior to resection (early surgery).

RESULTS: A total of 48 patients underwent ileocecectomy, with a mean age at time of resection of 17 years (+/- 2.3). In total, 88% of patients had a post-resection endoscopy and 57% had an endoscopy within 12 months of resection. Twenty-nine percent had ER with a mRS ≥ i2. There was no statistical difference in endoscopic and clinical outcomes after resection between the early and late surgery groups.

CONCLUSIONS: Post-resection endoscopic recurrence after ileocecectomy was found in 29% of our center’s pediatric CD population based on mRS. Post-resection outcomes were not affected by therapy duration prior to resection.

PMID:35442239 | DOI:10.1097/MPG.0000000000003456

J Pediatr Gastroenterol Nutr. 2022 Apr 20. doi: 10.1097/MPG.0000000000003452. Online ahead of print.

ABSTRACT

OBJECTIVES: Sebelipase alfa is approved for treatment of lysosomal acid lipase deficiency (LAL-D). This single-arm, open-label study (NCT02112994) evaluated sebelipase alfa efficacy and safety in patients with LAL-D.

METHODS: Patients >8 months of age diagnosed with LAL-D received sebelipase alfa 1.0 mg/kg by intravenous infusion every other week (qow) for up to 144 weeks. Dose escalation to 3.0 mg/kg qow and subsequently to 3.0 mg/kg weekly was permitted, per protocol; dose reductions for tolerability were permitted to 0.35 mg/kg qow. Descriptive statistical analyses were conducted.

RESULTS: Thirty-one patients were enrolled and treated. Baseline median alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels were 63.5 and 65.5 U/L, respectively. Twenty-eight patients completed 96 weeks of treatment, and 25 continued into the extended treatment period; 19 completed 144 weeks. From baseline to week 144, median ALT and AST levels changed by -42.0 and -22.0 U/L, respectively, median liver and spleen volumes changed from 1.4 to 1.3 and from 2.6 to 2.3 multiples of normal, respectively, median low-density lipoprotein cholesterol levels decreased by 52.6 mg/dL, and median high-density lipoprotein cholesterol increased by 9.8 mg/dL. Liver biopsies showed mostly improved or stable histopathology at 48 and 96 weeks versus baseline. Infusion-associated reactions were mild (n = 1) or moderate (n = 2). One patient (a candidate for liver transplant at baseline) discontinued treatment due to liver transplant (unrelated to treatment). Two patients tested positive for non-neutralizing, anti-drug antibodies on 1 occasion each.

CONCLUSION: Sebelipase alfa was well tolerated and resulted in sustained improvements in liver and lipid parameters.

PMID:35442238 | DOI:10.1097/MPG.0000000000003452

J Pediatr Gastroenterol Nutr. 2022 Apr 20. doi: 10.1097/MPG.0000000000003462. Online ahead of print.

ABSTRACT

BACKGROUND: Studies assessing adult inflammatory bowel disease (IBD) patient perspectives on biosimilar use revealed that most were unfamiliar with biosimilars and had a negative perception. The objective of this study was to evaluate the perspectives of pediatric patients with IBD and their caregivers regarding biosimilar use and non-medical switches.

METHODS: A survey was given to a cross section of patients with IBD ages 11-21 years receiving the intravenous anti-TNF originator and caregivers of patients with IBD ages 3-21 years receiving the originator. Recruitment occurred via mail, during clinic visits, and infusions. Fisher’s exact tests were used to test for statistically significant differences.

RESULTS: Response rate amongst caregivers was 49% (n = 98) and amongst patients was 35% (n = 67). Sixty-four percent of caregivers and 79% of patients had never heard of biosimilars. There was increased discomfort surrounding the use of biosimilars and switching to a biosimilar amongst caregivers who had previously heard of biosimilars compared to caregivers who had not previously heard of biosimilars (p < 0.05). Similar concerns were not seen in patient respondents. The length of time on the originator had no effect on patient or caregiver concerns related to biosimilar efficacy, side effects, or switches.

CONCLUSION: The majority of pediatric patients and caregivers had never heard of biosimilars. Caregivers that had heard of biosimilars prior to the study were more likely to have a negative perception of them. This study highlights the importance of providing thorough and accurate education to pediatric patients and families regarding the safety and efficacy of biosimilars.

PMID:35442227 | DOI:10.1097/MPG.0000000000003462

JMIR Mhealth Uhealth. 2022 Apr 20;10(4):e29985. doi: 10.2196/29985.

ABSTRACT

BACKGROUND: Drug-referencing apps are among the most frequently used by emergency health professionals. To date, no study has analyzed the quantity and quality of apps that provide information on emergency drugs.

OBJECTIVE: This study aimed to identify apps designed to assist emergency professionals in managing drugs and to describe and analyze their characteristics.

METHODS: We performed an observational, cross-sectional, descriptive study of apps that provide information on drugs for adult emergency care. The iOS and Android platforms were searched in February 2021. The apps were independently evaluated by 2 hospital clinical pharmacists. We analyzed developer affiliation, cost, updates, user ratings, and number of downloads. We also evaluated the main topic (emergency drugs or emergency medicine), the number of drugs described, the inclusion of bibliographic references, and the presence of the following drug information: commercial presentations, usual dosage, dose adjustment for renal failure, mechanism of action, therapeutic indications, contraindications, interactions with other medicinal products, use in pregnancy and breastfeeding, adverse reactions, method of preparation and administration, stability data, incompatibilities, identification of high-alert medications, positioning in treatment algorithms, information about medication reconciliation, and cost.

RESULTS: Overall, 49 apps were identified. Of these 49 apps, 32 (65%) were found on both digital platforms; 11 (22%) were available only for Android, and 6 (12%) were available only for iOS. In total, 41% (20/49) of the apps required payment (ranging from €0.59 [US $0.64] to €179.99 [US $196.10]) and 22% (11/49) of the apps were developed by non-health care professionals. The mean weighted user rating was 4.023 of 5 (SD 0.71). Overall, 45% (22/49) of the apps focused on emergency drugs, and 55% (27/49) focused on emergency medicine. More than half (29/47, 62%) did not include bibliographic references or had not been updated for more than a year (29/49, 59%). The median number of drugs was 66 (range 4 to >5000). Contraindications (26/47, 55%) and adverse reactions (24/47, 51%) were found in only half of the apps. Less than half of the apps addressed dose adjustment for renal failure (15/47, 32%), interactions (10/47, 21%), and use during pregnancy and breastfeeding (15/47, 32%). Only 6% (3/47) identified high-alert medications, and 2% (1/47) included information about medication reconciliation. Health-related developer, main topic, and greater amount of drug information were not statistically associated with higher user ratings (P=.99, P=.09, and P=.31, respectively).

CONCLUSIONS: We provide a comprehensive review of apps with information on emergency drugs for adults. Information on authorship, drug characteristics, and bibliographic references is frequently scarce; therefore, we propose recommendations to consider when developing an app of these characteristics. Future efforts should be made to increase the regulation of drug-referencing apps and to conduct a more frequent and documented review of their clinical content.

PMID:35442212 | DOI:10.2196/29985

JMIR Med Inform. 2022 Apr 20;10(4):e28696. doi: 10.2196/28696.

ABSTRACT

BACKGROUND: Clinical trials are the gold standard for advancing medical knowledge and improving patient outcomes. For their success, an appropriately sized cohort is required. However, patient recruitment remains one of the most challenging aspects of clinical trials. Information technology (IT) support systems-for instance, patient recruitment systems-may help overcome existing challenges and improve recruitment rates, when customized to the user needs and environment.

OBJECTIVE: The goal of our study is to describe the status quo of patient recruitment processes and to identify user requirements for the development of a patient recruitment system.

METHODS: We conducted a web-based survey with 56 participants as well as semistructured interviews with 33 participants from 10 German university hospitals.

RESULTS: We here report the recruitment procedures and challenges of 10 university hospitals. The recruitment process was influenced by diverse factors such as the ward, use of software, and the study inclusion criteria. Overall, clinical staff seemed more involved in patient identification, while the research staff focused on screening tasks. Ad hoc and planned screenings were common. Identifying eligible patients was still associated with significant manual efforts. The recruitment staff used Microsoft Office suite because tailored software were not available. To implement such software, data from disparate sources will need to be made available. We discussed concrete technical challenges concerning patient recruitment systems, including requirements for features, data, infrastructure, and workflow integration, and we contributed to the support of developing a successful system.

CONCLUSIONS: Identifying eligible patients is still associated with significant manual efforts. To fully make use of the high potential of IT in patient recruitment, many technical and process challenges have to be solved first. We contribute and discuss concrete technical challenges for patient recruitment systems, including requirements for features, data, infrastructure, and workflow integration.

PMID:35442203 | DOI:10.2196/28696

JMIR Med Inform. 2022 Apr 20;10(4):e33395. doi: 10.2196/33395.

ABSTRACT

BACKGROUND: As a major health hazard, the incidence of coronary heart disease has been increasing year by year. Although coronary revascularization, mainly percutaneous coronary intervention, has played an important role in the treatment of coronary heart disease, major adverse cardiovascular events (MACE) such as recurrent or persistent angina pectoris after coronary revascularization remain a very difficult problem in clinical practice.

OBJECTIVE: Given the high probability of MACE after coronary revascularization, the aim of this study was to develop and validate a predictive model for MACE occurrence within 6 months based on machine learning algorithms.

METHODS: A retrospective study was performed including 1004 patients who had undergone coronary revascularization at The People’s Hospital of Liaoning Province and Affiliated Hospital of Liaoning University of Traditional Chinese Medicine from June 2019 to December 2020. According to the characteristics of available data, an oversampling strategy was adopted for initial preprocessing. We then employed six machine learning algorithms, including decision tree, random forest, logistic regression, naïve Bayes, support vector machine, and extreme gradient boosting (XGBoost), to develop prediction models for MACE depending on clinical information and 6-month follow-up information. Among all samples, 70% were randomly selected for training and the remaining 30% were used for model validation. Model performance was assessed based on accuracy, precision, recall, F1-score, confusion matrix, area under the receiver operating characteristic (ROC) curve (AUC), and visualization of the ROC curve.

RESULTS: Univariate analysis showed that 21 patient characteristic variables were statistically significant (P<.05) between the groups without and with MACE. Coupled with these significant factors, among the six machine learning algorithms, XGBoost stood out with an accuracy of 0.7788, precision of 0.8058, recall of 0.7345, F1-score of 0.7685, and AUC of 0.8599. Further exploration of the models to identify factors affecting the occurrence of MACE revealed that use of anticoagulant drugs and course of the disease consistently ranked in the top two predictive factors in three developed models.

CONCLUSIONS: The machine learning risk models constructed in this study can achieve acceptable performance of MACE prediction, with XGBoost performing the best, providing a valuable reference for pointed intervention and clinical decision-making in MACE prevention.

PMID:35442202 | DOI:10.2196/33395

JMIR Ment Health. 2022 Apr 20;9(4):e21208. doi: 10.2196/21208.

ABSTRACT

BACKGROUND: Patient-controlled granular information sharing (PC-GIS) allows a patient to select specific health information “granules,” such as diagnoses and medications; choose with whom the information is shared; and decide how the information can be used. Previous studies suggest that health professionals have mixed or concerned opinions about the process and impact of PC-GIS for care and research. Further understanding of behavioral health professionals’ views on PC-GIS are needed for successful implementation and use of this technology.

OBJECTIVE: The aim of this study was to evaluate changes in health professionals’ opinions on PC-GIS before and after a demonstrative case study.

METHODS: Four focus groups were conducted at two integrated health care facilities: one serious mental illness facility and one general behavioral health facility. A total of 28 participants were given access to outcomes of a previous study where patients had control over medical record sharing. Participants were surveyed before and after focus groups on their views about PC-GIS. Thematic analysis of focus group output was paired with descriptive statistics and exploratory factor analysis of surveys.

RESULTS: Behavioral health professionals showed a significant opinion shift toward concern after the focus group intervention, specifically on the topics of patient understanding (P=.001), authorized electronic health record access (P=.03), patient-professional relationship (P=.006), patient control acceptance (P<.001), and patient rights (P=.02). Qualitative methodology supported these results. The themes of professional considerations (2234/4025, 55.5% of codes) and necessity of health information (260/766, 33.9%) identified key aspects of PC-GIS concerns.

CONCLUSIONS: Behavioral health professionals agreed that a trusting patient-professional relationship is integral to the optimal implementation of PC-GIS, but were concerned about the potential negative impacts of PC-GIS on patient safety and quality of care.

PMID:35442199 | DOI:10.2196/21208

JMIR Aging. 2022 Apr 20;5(2):e35075. doi: 10.2196/35075.

ABSTRACT

BACKGROUND: eHealth technologies for self-management can improve quality of life, but little is known about whether the benefits gained outweigh their costs. The electronic patient-reported outcome (ePRO) mobile app and portal system supports patients with multiple chronic conditions to collaborate with primary health care providers to set and monitor health-related goals.

OBJECTIVE: This study aims to estimate the cost of ePRO and the cost utility of the ePRO intervention compared with usual care provided to patients with multiple chronic conditions and complex needs living in the community, from the perspective of the publicly funded health care payer in Ontario, Canada.

METHODS: We developed a decision tree model to estimate the incremental cost per quality-adjusted life year (QALY) gained for the ePRO tool versus usual care over a time horizon of 15 months. Resource utilization and effectiveness of the ePRO tool were drawn from a randomized clinical trial with 6 family health teams involving 45 participants. Unit costs associated with health care utilization (adjusted to 2020 Canadian dollars) were drawn from literature and publicly available sources. A series of sensitivity analyses were conducted to assess the robustness of the findings.

RESULTS: The total cost of the ePRO tool was CAD $79,467 (~US $ 63,581; CAD $1733 [~US $1386] per person). Compared with standard care, the ePRO intervention was associated with higher costs (CAD $1710 [~US $1368]) and fewer QALYs (-0.03). The findings were consistent with the clinical evidence, suggesting no statistical difference in health-related quality of life between ePRO and usual care groups. However, the tool would be considered a cost-effective option if it could improve by at least 0.03 QALYs. The probability that the ePRO is cost-effective was 17.3% at a willingness-to-pay (WTP) threshold of CAD $50,000 (~US $40,000)/QALY.

CONCLUSIONS: The ePRO tool is not a cost-effective technology at the commonly used WTP value of CAD $50,000 (~US $40,000)/QALY, but long-term and the societal impacts of ePRO were not included in this analysis. Further research is needed to better understand its impact on long-term outcomes and in real-world settings. The present findings add to the growing evidence about eHealth interventions’ capacity to respond to complex aging populations within finite-resourced health systems.

TRIAL REGISTRATION: ClinicalTrials.gov NCT02917954; https://clinicaltrials.gov/ct2/show/NCT02917954.

PMID:35442194 | DOI:10.2196/35075

Microb Genom. 2022 Apr;8(4). doi: 10.1099/mgen.0.000675.

ABSTRACT

A hospital outbreak of carbapenem-resistant Enterobacterales was detected by routine surveillance. Whole genome sequencing and subsequent analysis revealed a conserved promiscuous bla _OXA-48 carrying plasmid as the defining factor within this outbreak. Four different species of Enterobacterales were involved in the outbreak. Escherichia coli ST399 accounted for 35 of all the 55 isolates. Comparative genomics analysis using publicly available E. coli ST399 genomes showed that the outbreak E. coli ST399 isolates formed a unique clade. We developed a mathematical model of pOXA-48-like plasmid transmission between host lineages and used it to estimate its conjugation rate, giving a lower bound of 0.23 conjugation events per lineage per year. Our analysis suggests that co-evolution between the pOXA-48-like plasmid and E. coli ST399 could have played a role in the outbreak. This is the first study to report carbapenem-resistant E. coli ST399 carrying blaOXA-48 as the main cause of a plasmid-borne outbreak within a hospital setting. Our findings suggest complementary roles for both plasmid conjugation and clonal expansion in the emergence of this outbreak.

PMID:35442183 | DOI:10.1099/mgen.0.000675

Hemodial Int. 2022 Apr 19. doi: 10.1111/hdi.13014. Online ahead of print.

ABSTRACT

BACKGROUND: Home hemodialysis (HHD) remains underused in patients with kidney failure. Current literature on HHD mostly originates from non-European countries, making generalization difficult. The present study describes patients’ profile and practice patterns from a Belgian HHD center, and assesses patient and technique survival and complications associated with HHD.

METHODS: We analyzed data from all our incident patients during a 6-year period. The patient’s characteristics were summarized using descriptive statistics. Transition to another therapeutic modality, estimated using a risk model with death and transplantation as competing events, episodes of respite cares and hospitalizations, and access complications were analyzed.

RESULTS: Eighty patients (mean age: 47 years; male: 64%) met the inclusion criteria. Fifty-one percent of patients initiated dialysis with a central venous catheter (CVC) and 96% were not assisted. Arterio-venous fistula (AVF) cannulation was performed using buttonhole technique. Standard-frequent HD (47%) and short-frequent low-flow dialysate HD (34%) were mostly used at HHD initiation. Cumulative incidences of technique failure and death were 15%, 24%, and 32% at 1, 2, and 5 years. Incidence rates for respite dialysis and hospitalizations were 2.39 and 0.54 per patient-year of HHD. In comparison with AVF, incidence rate ratios of overall access complications and access-related infections for CVC were 4.3 (95% CI: 3.1-6, p < 0.01) and 4.4 (95% CI: 2.1-10, p < 0.01), respectively. Buttonhole cannulation was complicated by 0.26 (95% CI: 0.15-0.46) infections per 1000 AVF-days.

CONCLUSIONS: This present study provides important information about patient’s profile and practice patterns and safety in a cohort of 80 incident Belgian HHD patients, with encouraging techniques and patient survival.

PMID:35441473 | DOI:10.1111/hdi.13014