Search

BACKGROUND/OBJECTIVES: The phase 3 REGAIN study and its open-label extension demonstrated the efficacy of the complement C5 inhibitor eculizumab in patients with treatment-refractory, acetylcholine receptor antibody-positive generalized myasthenia gravis (gMG). The aim of the ELEVATE study was to assess the effectiveness of eculizumab in clinical practice in adults with MG in the United States. METHODS: A retrospective chart review was conducted in adults with MG who initiated eculizumab treatment between October 23, 2017 and December 31, 2019. Outcomes assessed before and during eculizumab treatment using a pre- versus post-treatment study design included Myasthenia Gravis-Activities of Daily Living (MG-ADL) total scores; minimal symptom expression (MSE); physician impression of clinical change; minimal manifestation status (MMS); and concomitant medication use. RESULTS: In total, 119 patients were included in the study. A significant reduction was observed in mean MG-ADL total score, from 8.0 before eculizumab initiation to 5.4 at 3 months and to 4.7 at 24 months after eculizumab initiation (both p

Older adults often present with multiple comorbidities and face significant postoperative complications. This study aimed to describe the role of Nurse Practitioner (NP)-led orthogeriatric services in managing hip fracture patients. We conducted a review of health records of older adults with hip and proximal femoral fractures between July 2017 and June 2018, presenting descriptive statistics on patient characteristics, surgical outcomes, and the involvement of orthogeriatric services. A total of 197 participants were included, with a majority being female (n = 132; 67.0%). Most patients (53.8%; n = 106) had between five and nine pre-existing conditions. Among the 192 patients who underwent surgery, 69.8% (n = 134) experienced up to four surgical complications. The Nurse Practitioner provided care to 89.1% (n = 163) of the patients within the orthogeriatric service, with half of the patients (n = 82) requiring at least five NP interventions to manage complex pre- and postoperative needs. Refining the NP-led model could potentially help reduce the burden on physicians and surgeons in treating complex medical conditions, especially in settings where geriatricians may not be readily available. [ABSTRACT FROM AUTHOR]

Background: Effective management of hearing loss in adults is fundamental for communication, relationships, employment, and learning. This study examined the rates and management of self-reported hearing loss in urban Aboriginal and Torres Strait Islander adults. Methods: A retrospective, observational study of Aboriginal and Torres Strait Islander people aged ≥15 years who had annual health checks at an urban Aboriginal and Torres Strait Islander primary healthcare clinic in Inala, Queensland, was conducted to determine self-reported hearing loss rates by age and ethnic groups stratified by sex. A medical record audit of patients who self-reported hearing loss from January to June 2021 was performed to identify current management approaches, and the proportion of patients that were appropriately managed. Results: Of the 1735 patients (average age 40.7 years, range 15.0–88.5 years, 900 [52.0%] women) who completed 3090 health checks between July 2018 and September 2021, 18.8% self-reported hearing loss. Rates did not differ between men and women. However, significant effects were noted for age, with rates increasing from 10.7% for patients aged 15–24 years to 38.7% for those aged ≥65 years. An audit of 73 patient medical records revealed that 39.7% of patients with self-reported hearing loss were referred to Ear, Nose and Throat/audiology or received other management. A total of 17.8% of patients owned hearing aids. Conclusions: Only 40% of Aboriginal and Torres Strait Islander adults who self-reported hearing loss were referred for management. Significant changes to clinical management and government-funded referral options for hearing services are required to improve the management of self-reported hearing loss in this population. Limited evidence on hearing loss in Aboriginal and Torres Strait Islander adults exists. In this primary care adult population, the rate of self-reported hearing loss was 18.8%. Only two in five adults received further action to manage their hearing loss with successful hearing outcomes evident. Hearing loss in Aboriginal and Torres Strait Islander adults is underreported, undermanaged, and requires urgent attention from health professionals and government policymakers to enhance its clinical management leading to improved quality of life. [ABSTRACT FROM AUTHOR]

Introduction: Gastrointestinal (GI) bleeding events (BEs) in von Willebrand disease (VWD) are difficult to diagnose and often recurrent. Limited data from clinical trials has led to lack of consensus on treatment options. Aim: Describe current treatments and outcomes for GI BEs in people with VWD. Methods: This retrospective, observational, multicentre chart review study was conducted from January 2018 through December 2019 and included patients with inherited VWD with ≥1 GI BE in the preceding 5 years. Baseline characteristics, number and aetiology of BEs, associated GI‐specific morbidities/lesions, treatment and outcomes were analysed descriptively. Results: Sixty bleeds were reported in 20 patients with type 1 (20%), type 2 (50%) and type 3 (30%) VWD. During the 5‐year study period, 31 (52%) BEs had one identified or suspected cause; multiple causes were reported in 11 (18%). Most GI BEs (72%) were treated with a combination of von Willebrand factor (VWF), antifibrinolytics and/or other haemostatic or non‐haemostatic treatments. Time to resolution did not differ by VWF treatment use; however, BEs treated with non‐VWF treatments tended to resolve later. In patients with GI‐specific morbidities/lesions, 84% resolved with first‐line treatment; time to resolution tended to be longer than in patients without such morbidities/lesions. Thirteen BEs occurred in patients receiving prophylaxis and 47 in patients receiving on‐demand treatment; 18 BEs resulted in a switch to prophylaxis after bleed resolution. Conclusions: This study confirms the unmet need for the management of recurrent GI BEs in people with VWD and the need for prospective data, especially on prophylaxis. [ABSTRACT FROM AUTHOR]

School absenteeism among children and adolescents has been associated with a myriad of adverse outcomes. Despite a substantial amount of research on school attendance, our understanding of absenteeism in elementary-aged children with mental health difficulties is limited. The current study used a mixed-method sequential explanatory design to gain a better understanding of the links between children's mental health symptoms, age, gender and school absenteeism. The study included two phases: Phase I presented a quantitative investigation of the links between absenteeism and mental health symptoms in a large sample (N = 750) of primarily White (74%) clinic-referred children aged 5–12 years. Phase II used a participant selection model and chart review design to garner a deeper understanding of how school absenteeism presents in children with mental health problems in the elementary years. Results suggested that internalizing symptoms were significantly related to school attendance problems, and this association worsens as children age and as internalizing symptoms increase in severity. Externalizing symptoms were associated with absenteeism as well, but these findings showed that school attendance problems were worse for younger children with low to moderate levels of externalizing symptoms. The qualitative chart review illustrates the complex interplay of school absenteeism and child mental health, particularly for children experiencing co-occurring internalizing and externalizing problems. Children often struggled academically and socially, with some exhibiting increasing levels of school refusal and truancy and others displaying aggressive behaviors resulting in suspensions. Future studies that examine additional contextual factors, such as school and family variables, with longitudinal cross-lagged models and diverse families are needed to develop a comprehensive understanding of causal associations and their impact on children's school attendance across children's development. [ABSTRACT FROM AUTHOR]

Purpose: Lenvatinib was approved for the treatment of patients with radioiodine-refractory differentiated thyroid cancer (RAI-R DTC) in the United States (US) in 2015. The main objective of the current study was to assess real-world clinical effectiveness in RAI-R DTC patients treated with first line lenvatinib monotherapy in the US. Methods: A retrospective chart review was conducted in RAI-R DTC patients who initiated lenvatinib monotherapy as first line treatment between February 2015 and September 2020. Anonymized data were abstracted by prescribing physicians from individual patient's electronic health records. Clinical outcomes included provider-reported real-world best overall response (rwBOR), real-world progression-free survival (rwPFS), and overall survival (OS). Time-to-event endpoints were assessed using Kaplan–Meier methods. Results: Our study included 308 RAI-R DTC patients treated with first line lenvatinib. At lenvatinib initiation, patients' median age was 60 years, 51.6% were female, and 26.0% of patients had an ECOG performance score of ≥2. Over the follow-up period, 32.5% of patients discontinued first line lenvatinib permanently, with others remaining on treatment. The median duration of lenvatinib therapy was 17.5 months overall. Provider-reported rwBOR (complete or partial response) to lenvatinib was 72.4%. Median rwPFS was 49.0 months. Estimated rwPFS rates at 24 and 48 months were 68.5% and 55.0%, respectively. Estimated OS rates at 24 and 72 months were 78.4% and 57.0%, respectively; median OS was not reached. Conclusion: The current study reinforces the clinical effectiveness of first line lenvatinib as standard of care in patients with RAI-R DTC in real-world clinical practice in the US. [ABSTRACT FROM AUTHOR]

AbstractPurposeMethodsResultsConclusion\nIMPLICATIONS FOR REHABILITATIONEvidence on co-occurring mental health problems in youth with physical disabilities is growing, however how services are provided remains unclear. This study examined current interprofessional rehabilitation practices for physical and mental health services.Youth (aged 15–24) followed for a physical disability that had mental health problems were identified. Chart reviews were used to identify practices. Mental health-related diagnoses/symptoms, assessments, goals, interventions, and referrals were extracted for inductive content analysis.Sixty charts were reviewed. Mental health problems included anxiety (n = 53), depression (n = 25), neurodevelopmental (n = 19) and personality disorders (n = 8), often (n = 36) citing more than one. No mental health assessments were found, and in 43%, no goals or interventions were evident. Relevant goals (n = 98) targeted emotional management, autonomy/communication of needs, acceptance of physical condition, socialization, routines/energy levels, school/work supports, and leisure/calming environments. Interventions (n = 104) included emotional management, formal individual/group therapy, links with external supports, routines/activities, reflection/acceptance, and school/work support. Mental health services were received in-house (n = 24) and/or externally (n = 30), plus 18 referrals pending and 14 not referred.Many had more than one mental health problem, suggesting the complexity of their condition. While some mental health goals/interventions are documented, problems may often not be reported or addressed in this context.Further attention can be directed to the needs of youth with physical disabilities and co-occurring mental health problems as they are not fully addressed by current interdisciplinary rehabilitation practices.Follow-up (services and referrals) should be adapted to the holistic needs of youth and their goals within the rehabilitation context.Rehabilitation professionals can be provided with training to build workforce capacity in mental health screening and have access to guidance when addressing situations related to mental health or referring to external services.Further attention can be directed to the needs of youth with physical disabilities and co-occurring mental health problems as they are not fully addressed by current interdisciplinary rehabilitation practices.Follow-up (services and referrals) should be adapted to the holistic needs of youth and their goals within the rehabilitation context.Rehabilitation professionals can be provided with training to build workforce capacity in mental health screening and have access to guidance when addressing situations related to mental health or referring to external services. [ABSTRACT FROM AUTHOR]

Epilepsy is a common, and often genetic, neurological disorder. Few guidelines exist to help medical providers or insurance companies decide when to order or cover epilepsy panels for patients with epilepsy. The most recent guidelines were published by NSGC after this study's data collection. Since 2017, the Genetic Testing Stewardship Program (GTSP) at UPMC Children's Hospital of Pittsburgh (CHP) has been utilizing a set of internally developed epilepsy panel (EP) testing criteria to facilitate appropriate EP ordering practices. The purpose of this study was to assess these testing criteria by determining their sensitivities and positive predictive values (PPV). Retrospective chart review of the electronic medical record (EMR) was performed for 1242 CHP Neurology patients that were evaluated for a primary diagnosis of epilepsy between 2016 and 2018. One hundred and nine patients had EPs at various testing laboratories. Of the patients that met criteria, 17 had diagnostic EPs and 54 had negative EPs. Criteria were organized into category groupings (C1‐C4), and analyzed alone for C1, in pairs for C2, etc. The highest sensitivity and PPV results in each category grouping were: C1 (64.7%, 60%); C2, (88%, 30.3%); C3, (94.1%, 27.1%); C4, (94.1%, 25.4%). Family history was crucial to increasing sensitivity. Confidence intervals (CIs) narrowed as category grouping level increased, though this was not statistically significant due to the considerable CI overlap across category groupings. The PPV from C4 was applied to the untested population cohort and predicted 121 patients with unidentified positive EPs. This study presents data supporting the predictive capabilities of EP testing criteria and suggests the addition of a family history criterion. This study impacts public health by encouraging the adoption of evidence‐driven insurance policies and by suggesting guidelines to ease EP ordering and coverage decisions, which could potentially improve patient access to EP testing. [ABSTRACT FROM AUTHOR]

Background: Acupuncture is a potentially effective non-pharmacological treatment for insomnia. Objective: We observed the responses of patients with insomnia to acupuncture in routine clinical practice. In addition, we explored patient characteristics that might affect the treatment response to acupuncture for insomnia. Methods: Medical records of patients with insomnia in a Korean medicine clinic with baseline Insomnia Severity Index (ISI) scores ⩾8 and Pittsburgh Sleep Quality Index (PSQI) scores ⩾5 were reviewed. Acupuncture was applied at ST43, GB41, ST41, SI5, HT3, KI10, HT7 and ST3, for 1–2 months. The ISI and PSQI were measured monthly to assess insomnia severity. The effect of acupuncture over time was analyzed using a multilevel linear model for repeated measures. In addition, logistic regression was used to explore predictors of treatment response. Results: A total of 91 patients with insomnia aged 59.2 ± 12.5 years (mean ± standard deviation (SD)) (90.1% female) were included in the analysis. After the acupuncture treatment, ISI scores were significantly reduced by −3.75 (95% confidence interval (CI) = −4.99, −2.50) and −4.69 (95% CI = −6.22, −3.16) after the first and second month, respectively. The PSQI global scores also improved, and sleep duration showed a tendency to increase by 0.35 h (95% CI = −0.17, 0.86) after acupuncture treatment. Three cases of mild fatigue were reported. In addition, higher baseline pain/discomfort predicted a greater likelihood of response after acupuncture treatment (odds ratio (OR) = 1.66, 95% CI = 1.10, 2.60). Conclusion: In a real-world setting, the insomnia of outpatients in a clinic was slightly alleviated after acupuncture treatment. These findings require validation by randomized controlled trials. [ABSTRACT FROM AUTHOR]

Objective: Claims data can be leveraged to study rare diseases such as Guillain-Barré Syndrome (GBS), a neurological autoimmune condition. It is difficult to accurately measure and distinguish true cases of disease with claims without a validated algorithm. Our objective was to identify the best-performing algorithm for identifying incident GBS cases in Medicare fee-for-service claims data using chart reviews as the gold standard. Study design and setting: This was a multi-center, single institution cohort study from 2015 to 2019 that used Medicare-linked electronic health record (EHR) data. We identified 211 patients with a GBS diagnosis code in any position of an inpatient or outpatient claim in Medicare that also had a record of GBS in their electronic medical record. We reported the positive predictive value (PPV = number of true GBS cases/total number of GBS cases identified by the algorithm) for each algorithm tested. We also tested algorithms using several prevalence assumptions for false negative GBS cases and calculated a ranked sum for each algorithm's performance. Results: We found that 40 patients out of 211 had a true case of GBS. Algorithm 17, a GBS diagnosis in the primary position of an inpatient claim and a diagnostic procedure within 45 days of the inpatient admission date, had the highest PPV (PPV = 81.6%, 95% CI (69.3, 93.9). Across three prevalence assumptions, Algorithm 15, a GBS diagnosis in the primary position of an inpatient claim, was favored (PPV = 79.5%, 95% CI (67.6, 91.5). Conclusions: Our findings demonstrate that patients with incident GBS can be accurately identified in Medicare claims with a chart-validated algorithm. Using large-scale administrative data to study GBS offers significant advantages over case reports and patient repositories with self-reported data, and may be a potential strategy for the study of other rare diseases.

Abstract Background Clinical practice guidelines emphasize shared decision-making for kidney replacement treatment, yet little is known about the influence of cultural differences on that process. We undertook a retrospective chart review to explore the process and timing of dialysis decision making and initiation in Chinese American patients to provide quality kidney care for this population. Design Participants received outpatient care at Tufts Medical Center and dialysis at Dialysis Clinic, Inc. Boston or Somerville, MA from 2001–2021. Clinic chart review sourced demographic, clinical, and end-of-life care information from 180 participants (82 Chinese American, 98 other) from stage 4 chronic kidney disease (CKD) and dialysis initiation. Results Chinese American participants were older (mean 70 vs. 59, p

Locally advanced or metastatic urothelial carcinoma (aUC) presents a significant challenge with high mortality rates. Platinum-based chemotherapy remains the established frontline standard of care, and a switch-maintenance strategy with immunotherapy has now emerged as a new standard for aUC patients without disease progression, following initial platinum therapy. Examining the treatment patterns is imperative, given the evolving therapeutic landscape. In this study, we conducted a retrospective medical chart review of 17 Canadian oncologists treating patients with aUC to assess unmet needs in Canadian aUC patient care. Data from 146 patient charts were analyzed, revealing important clinical insights about the management of aUC. A substantial proportion of patients (53%) presented with de novo metastatic disease, which was possibly influenced by pandemic-related care disruptions. Variability was evident in the cisplatin eligibility criteria, with a majority (70%) of oncologists utilizing a 50 mL/min threshold. Most favored four cycles of platinum-based chemotherapy to spare the bone marrow for future therapies and prevent patient fatigue. Notably, some eligible patients were kept under surveillance rather than receiving maintenance therapy, suggesting a potential gap in awareness regarding evidence-based recommendations. Furthermore, managing treatment-related adverse events was found to be one of the biggest challenges in relation to maintenance immunotherapy. In conclusion, our findings provide the first comprehensive overview of aUC treatment patterns in Canada following the approval of maintenance immunotherapy, offering insights into the decision-making process and underscoring the importance of evidence-based guidelines in aUC patient management. [ABSTRACT FROM AUTHOR]

Background: Clinical practice guidelines emphasize shared decision-making for kidney replacement treatment, yet little is known about the influence of cultural differences on that process. We undertook a retrospective chart review to explore the process and timing of dialysis decision making and initiation in Chinese American patients to provide quality kidney care for this population. Design: Participants received outpatient care at Tufts Medical Center and dialysis at Dialysis Clinic, Inc. Boston or Somerville, MA from 2001–2021. Clinic chart review sourced demographic, clinical, and end-of-life care information from 180 participants (82 Chinese American, 98 other) from stage 4 chronic kidney disease (CKD) and dialysis initiation. Results: Chinese American participants were older (mean 70 vs. 59, p < 0.0001), less likely to speak English (12% vs. 87%, p < 0.0001), and used interpreter services more (80% vs. 11%, p < 0.0001). Chinese American participants had more visits (median 14 vs. 10, p = 0.005); were more often accompanied by family members (75% vs. 40%, p < 0.001); and had significantly lower rates of healthcare proxy documentation (35% vs. 55%, p = 0.006). There was no statistical difference in months between first CKD 4 visit and first dialysis. Both groups started dialysis at the same average eGFR and with similar rates of permanent dialysis access. Chinese American participants had significantly lower serum albumin at dialysis initiation (mean 3.3 g/dL vs 3.7 g/dL, p = 0.0003). Documentation reflected a low number of conversations about non-dialytic care, end-of-life planning, or palliative care in both groups across all visits. Conclusion: The time between CKD 4 and dialysis initiation was the same in both groups, suggesting a similar overall outcome of care. Chart documentation suggests that Chinese American participants had a significantly higher number of visits with nephrologists where discussion about dialysis was noted and were more likely to have a family member present at the visit. Fewer Chinese American participants completed healthcare proxies. Among all study participants, healthcare proxy, code status, and palliative care discussions were reported less frequently than expected. These findings highlight opportunities for collaboration between palliative care clinicians and nephrologists. [ABSTRACT FROM AUTHOR]

Introduction: There are limited data from the United States regarding the real-world signs and symptoms of Wilson disease (WD). This retrospective, observational medical chart review was conducted to identify real-world characteristics of patients with WD in the United States, as well as WD signs and symptoms at diagnosis and over time. Methods: De-identified clinical data were abstracted from medical charts of US patients diagnosed with WD between January 1, 2012, and June 30, 2017. Hepatic, neurologic, and psychiatric biochemical findings, signs, and symptoms were characterized at diagnosis and follow-up/during treatment. Results: In total, 225 WD patients were included in the study. The mean (SD) age at diagnosis was 24.7 (9.8) years, and 65.3% were male. Median (Q1-Q3) follow-up after diagnosis was 39.5 (33.8-60.4) months. The most common disease presentation at WD diagnosis was combined neurologic/psychiatric and hepatic (52.9%), followed by neurologic/psychiatric (20.0%), hepatic (16.9%), and asymptomatic (10.2%). Common clinical characteristics at diagnosis were Kayser-Fleischer rings (77.2%), low ceruloplasmin levels (95.2%), high hepatic copper (97.8%), elevated 24-hour urinary copper excretion (90.2%), and abnormal liver function tests (38.7%-85.1%). At diagnosis, the most common biochemical findings or hepatic sign/symptoms were abnormal liver enzymes (50.7%), abdominal pain (16.6%), and fatigue (15.7%). The most common neurologic signs/symptoms were headache (18.3%), dysarthria (17.4%), and ataxia (17.0%). Common psychiatric signs/symptoms included anxiety/depression/other mood changes (36.2%), emotional lability (12.8%), and increased irritability/anger outbursts (9.2%). Prevalence of biochemical abnormalities or signs/symptoms among patients at diagnosis and after ~1-year follow-up were neurologic (60.1% and 44.0%), hepatic (69.6% and 37.8%), and psychiatric (53.7% and 37.6%), respectively. Common new onset symptoms at ~1-year post-WD diagnosis were abnormal liver enzymes (5.6%), headache (6.2%), and anxiety/depression/other mood changes (7.2%). Conclusions: These real-world, descriptive data highlight the clinical complexity and heterogeneity of WD and the need for better education about diagnostic testing and multidisciplinary support. Although rare, the neurologic, psychiatric, and hepatic signs/symptoms of WD have a substantial clinical impact. [ABSTRACT FROM AUTHOR]

Objectives Chart review as the current gold standard for phenotype evaluation cannot support observational research on electronic health records and claims data sources at scale. We aimed to evaluate the ability of structured data to support efficient and interpretable phenotype evaluation as an alternative to chart review. Materials and Methods We developed Knowledge-Enhanced Electronic Profile Review (KEEPER) as a phenotype evaluation tool that extracts patient's structured data elements relevant to a phenotype and presents them in a standardized fashion following clinical reasoning principles. We evaluated its performance (interrater agreement, intermethod agreement, accuracy, and review time) compared to manual chart review for 4 conditions using randomized 2-period, 2-sequence crossover design. Results Case ascertainment with KEEPER was twice as fast compared to manual chart review. 88.1% of the patients were classified concordantly using charts and KEEPER, but agreement varied depending on the condition. Missing data and differences in interpretation accounted for most of the discrepancies. Pairs of clinicians agreed in case ascertainment in 91.2% of the cases when using KEEPER compared to 76.3% when using charts. Patient classification aligned with the gold standard in 88.1% and 86.9% of the cases respectively. Conclusion Structured data can be used for efficient and interpretable phenotype evaluation if they are limited to relevant subset and organized according to the clinical reasoning principles. A system that implements these principles can achieve noninferior performance compared to chart review at a fraction of time. [ABSTRACT FROM AUTHOR]

Background: Most prefer to die at home, but the Medicare Hospice Benefit does not cover custodial care, making it difficult for terminally ill patients with housing insecurity and/or caregiver instability to access hospice care at home. Objectives: To examine the characteristics of patients who received end-of-life care in community-run, residential care homes (RCHs) operating under the social model hospice. Methods: A retrospective chart review of 500 residents who were admitted to one of three RCHs in Upstate New York over a 15-year period (2004-2019). Results: Patients served by the RCHs included 318 (63.6%) women and 182 (36.4%) men aged 34-101 (M = 77.8). The majority (94.9%) were Caucasian and most had cancer diagnoses (71.6%). Prior to admission, most (93%) patients resided in a private residence, and nearly half (47%) lived alone, but most (81.7%) had full- or part-time caregivers. Nearly all patients were admitted either directly from a hospital (47.5%) or private home (47.2%). Over half (52%) were admitted to RCHs within a month of hospice enrollment, and 20.1% enrolled concurrent with admission. While the average length of stay was 21 days, 50% died within 10 days of admission. Conclusions: Community-run RCHs represent a unique approach for improving access to hospice home care for patients with home insecurity and/or caregiver instability, yet most patients had prior caregiver coverage and were admitted from a hospital or home setting, suggesting there is a need for community care settings for patients unable to remain at home in the final weeks or days prior to death. [ABSTRACT FROM AUTHOR]

Background: Acid sphingomyelinase deficiency (ASMD), historically known as Niemann–Pick disease type A, A/B, and B, is a rare lysosomal storage pathology with multisystemic clinical manifestations. The aims of this study were to estimate the survival probability in patients in the United States with chronic ASMD (ASMD types B and A/B), and to describe the disease characteristics of these patients. Methods: This observational retrospective study included medical chart records of patients with chronic ASMD with retrievable data abstracted by 69 participating physicians from 25 medical centers in the United States. Included patients had a date of ASMD diagnosis or first presentation to a physician for ASMD symptoms (whichever occurred first) between January 01, 1990, and February 28, 2021. Medical chart records were excluded if patients were diagnosed with ASMD type A. Eligible medical chart records were abstracted to collect demographic, medical and developmental history, and mortality data. Survival outcomes were analyzed using Kaplan–Meier survival analyses from birth until death. Results: The overall study population (N = 110) included 69 patients with ASMD type B, nine with type A/B, and 32 with ASMD “non-type A'' (ASMD subtype was unknown, but patients were confirmed as not having ASMD type A). The majority of patients were male with a median age at diagnosis of 3.8 years. Thirty-eight patients died during the study observation period, at a median age of 6.8 years. The median (95% confidence interval) survival age from birth was 21.3 (10.2; 60.4) years. At diagnosis or first presentation, 42.7% patients had ≥1 ASMD-related complication; splenic (30.0%) and hepatobiliary (20.9%) being the most common, and 40.9% required ≥1 medical visit due to complications. Conclusion: Patients with chronic ASMD in the United States have poor survival and significant burden of illness.

BackgroundPreoperative medical evaluation serves to identify risk factors and optimize patients before surgery. Providing a telehealth option in the perioperative setting has played a significant role in reducing barriers to quality perioperative health care.ObjectiveWe aimed to evaluate how telemedicine preoperative evaluations using Clinical Video Telehealth (CVT) impact hospital length of stay.MethodsWe performed a retrospective chart review between 2016 and 2017 of adult patients who underwent evaluations in our hospitalist-run preoperative medicine clinic. Patients seen in our preoperative CVT program were compared to patients seen in person to evaluate the association of visit type (preoperative CVT versus in-person evaluation) with hospital length of stay, defined as hospital stay from postoperative day 0 to discharge. There were 62 patients included in this retrospective study.ResultsThe adjusted incidence rate ratio (IRR) for hospital length of stay was significantly shorter in patients who underwent preoperative CVT compared to an in-person visit (IRR 0.52, 95% CI 0.29-0.92, P=.02).ConclusionsAfter adjusting for age and comorbidities, we show that preoperative telemedicine in the perioperative setting is associated with a shorter hospital length of stay compared to in-person visits. This suggests that telemedicine can play a viable role in this clinical setting.

Aim: Traumatic brain injury (TBI) is a common neurological condition, which can present with a wide range of neuropsychological symptoms. Treating this broad spectrum of symptoms represents a significant medical challenge. In part because of this, there is growing interest in the use of medical cannabis to treat the sequelae of TBI, as medical cannabis has been used to treat multiple associated conditions, such as pain. However, medical cannabis represents a heterogeneous collection of therapies, and relatively little is known about their effectiveness in treating TBI symptoms. The aim of the present study was therefore to assess medical cannabis use in patients with TBI. Methods: In the present study, a retrospective chart review was conducted of patterns of cannabis use and TBI symptoms in individuals who used medical cannabis to treat TBI-related symptoms. All subjects were recruited from a medical cannabis clinic, where cannabis was authorized by physicians, using licensed cannabis products. A total of 53 subjects provided written consent to have their charts reviewed. Results: Neuropsychiatric conditions, including depression, pain, and anxiety were frequent in this group. The most common forms of medical cannabis consumption at intake included smoking, vaping, and oral ingestion. Patients used a combination of high tetrahydrocannabinol (THC)/low cannabidiol (CBD) and low THC/high CBD products, typically 1–3 times per day. Medical cannabis appeared to be relatively well-tolerated in subjects, with few serious side effects. At follow-up, subjects self-reported improvements in TBI symptoms, although these were not statistically significant when assessed using validated questionnaires. Conclusions: Overall findings indicate modest potential benefits of medical cannabis for TBI, but further research will be required to validate these results.

Introduction In recent years, there has been a national push to incorporate high-fidelity quality improvement and patient safety (QIPS) education into physician training programs. In fact, integration of robust patient safety education became an Accreditation Council for Graduate Medical Education (ACGME) Common Program Requirement for residency programs in 2017. We developed a curriculum to not only fulfill the ACGME's requirement but also provide PGY 1 internal medicine residents with the skills needed to become active participants in ongoing patient safety work throughout their training and careers. Methods Our patient safety curriculum was woven into residents’ existing protected educational time and supported by a standardized facilitator guide and participant workbook. It combined didactic prework with the review of recent near-miss or low-harm patient safety events, empowering residents to identify root causes and propose interventions. Results We successfully delivered our patient safety curriculum to 80 PGY 1 residents over the course of 2 academic years. Residents rated the curriculum as a valuable educational experience, and the event reviews they completed met most of the criteria for high-quality patient safety reviews according to the Strong String Assessment. Discussion Implementation of this standardized curriculum has allowed us to reliably and consistently incorporate experiential patient safety education into the first year of training for internal medicine residents. Unlike purely didactic sessions, our curriculum encourages active learning, building muscle memory for event reviews that enables future engagement in patient safety activities.

IntroductionThere are limited data from the United States regarding the real-world signs and symptoms of Wilson disease (WD). This retrospective, observational medical chart review was conducted to identify real-world characteristics of patients with WD in the United States, as well as WD signs and symptoms at diagnosis and over time.MethodsDe-identified clinical data were abstracted from medical charts of US patients diagnosed with WD between January 1, 2012, and June 30, 2017. Hepatic, neurologic, and psychiatric biochemical findings, signs, and symptoms were characterized at diagnosis and follow-up/during treatment.ResultsIn total, 225 WD patients were included in the study. The mean (SD) age at diagnosis was 24.7 (9.8) years, and 65.3% were male. Median (Q1–Q3) follow-up after diagnosis was 39.5 (33.8–60.4) months. The most common disease presentation at WD diagnosis was combined neurologic/psychiatric and hepatic (52.9%), followed by neurologic/psychiatric (20.0%), hepatic (16.9%), and asymptomatic (10.2%). Common clinical characteristics at diagnosis were Kayser-Fleischer rings (77.2%), low ceruloplasmin levels (95.2%), high hepatic copper (97.8%), elevated 24-hour urinary copper excretion (90.2%), and abnormal liver function tests (38.7%–85.1%). At diagnosis, the most common biochemical findings or hepatic sign/symptoms were abnormal liver enzymes (50.7%), abdominal pain (16.6%), and fatigue (15.7%). The most common neurologic signs/symptoms were headache (18.3%), dysarthria (17.4%), and ataxia (17.0%). Common psychiatric signs/symptoms included anxiety/depression/other mood changes (36.2%), emotional lability (12.8%), and increased irritability/anger outbursts (9.2%). Prevalence of biochemical abnormalities or signs/symptoms among patients at diagnosis and after ~1-year follow-up were neurologic (60.1% and 44.0%), hepatic (69.6% and 37.8%), and psychiatric (53.7% and 37.6%), respectively. Common new onset symptoms at ~1-year post-WD diagnosis were abnormal liver enzymes (5.6%), headache (6.2%), and anxiety/depression/other mood changes (7.2%).ConclusionsThese real-world, descriptive data highlight the clinical complexity and heterogeneity of WD and the need for better education about diagnostic testing and multidisciplinary support. Although rare, the neurologic, psychiatric, and hepatic signs/symptoms of WD have a substantial clinical impact.

Purpose: This study aimed to describe the clinical characteristics and wellness programming preferences of cancer survivors from Acres Homes, a historically Black neighborhood in Houston, Texas, with areas of persistent poverty. The goal of this study was to identify opportunities to increase cancer survivor utilization of healthy eating and active living interventions aligned to cancer center community outreach and engagement efforts. Methods: This multiple methods study included a retrospective review of electronic health record data (n = 413) and qualitative interviews with cancer survivors (n = 31) immediately preceding initiation of healthy eating, active living programming in Acres Homes. Results: This study found Acres Homes survivors have high rates of co-occurrent cardiometabolic disease including obesity (45.0%), diabetes (30.8%), and other related risk factors as well as treatment-related symptoms. Four major concepts emerged from interviews: (1) Factors that influence survivors' ability to eat well and exercise, (2) Current usage of community resources, (3) Interest in relevant programming, and (4) Specific programming preferences. Opportunities for current and future health promotion programming for cancer survivors were explored. Conclusion: Strategically tailoring community resources for cancer survivors can provide a more robust network of support to promote healthy eating and active living in this population. This work informed community implementation of evidence-based health interventions in Acres Homes and may support future projects aiming to enhance community-led cancer prevention efforts in historically underserved communities. [ABSTRACT FROM AUTHOR]

Aims and Objectives: To enhance the practice of a person‐centred palliative approach in long‐term care. Background: Implementing a person‐centred palliative approach in long‐term care entails placing residents at the centre of care planning that attends to the 'whole' person, rather than prioritising biomedical needs. Design: We conducted a four‐stage directed content analysis of long‐term care progress notes to meet our study aims and applied the EQUATOR guidelines for qualitative research publication (COREQ). Methods: We qualitatively analysed 78 resident charts across three long‐term care homes in southern Ontario to capture the extent to which person‐centred care was absent, initiated or implemented in different types of documented care interactions. Results: Most residents had interactions related to daily care activities (65/78, 83%), social concerns (65/78, 83%) and treatment decisions (53/78, 68%). By contrast, interactions around pain and discomfort (34/78, 44%) and spirituality (27/78, 35%) were documented for less than half of the residents. Almost all (92%) residents had at least one progress note where staff initiated person‐centred care by documenting their preference for a certain type of care, but only a third had at least one progress note that suggested their preference was implemented (35%). Conclusions: While person‐centred care is often initiated by nurses and other allied health professionals, changes to care plans to address resident preferences are implemented less often. Nurses and other allied health professionals should be encouraged to elicit care preferences crucial for holistic care planning and equipped with the skills and support to enact collaborative care planning. Relevance to Clinical Practice: Collaborative care planning appears relatively absent in charted progress notes, constraining the full implementation of a person‐centred palliative approach to care. Patient or Public Contribution: An advisory group consisting of long‐term care resident and staff representatives informed the overall study design and dissemination of the results. [ABSTRACT FROM AUTHOR]

Purpose: The safety and efficacy of subcutaneous immune globulin 20% (human) solution (Ig20Gly) were demonstrated in clinical trials. However, real-world evidence of the tolerability of self-administered Ig20Gly in elderly patients is lacking. We describe real-world patterns of Ig20Gly usage for 12 months in patients with primary immunodeficiency diseases (PIDD) in the USA. Methods: This retrospective chart review of longitudinal data from 2 centers included patients aged ≥ 2 years with PIDD. Ig20Gly administration parameters, tolerability, and usage patterns were assessed at initial and subsequent 6- and 12-month infusions. Results: Of 47 enrolled patients, 30 (63.8%) received immunoglobulin replacement therapy (IGRT) within 12 months before starting Ig20Gly, and 17 (36.2%) started IGRT de novo. Patients were predominantly White (89.1%), female (85.1%), and elderly (aged > 65 years, 68.1%; median age = 71.0 years). Most adults received at-home treatment during the study, and most self-administered at 6 months (90.0%) and 12 months (88.2%). Across all time points, infusions were administered at a mean rate of 60–90 mL/h/infusion, using a mean of 2 sites per infusion, on a weekly or biweekly frequency. No emergency department visits occurred, and hospital visits were rare (n = 1). Forty-six adverse drug reactions occurred in 36.4% of adults, mostly localized site reactions; none of these or any adverse events led to treatment discontinuation. Conclusion: These findings demonstrate tolerability and successful self-administration of Ig20Gly in PIDD, including elderly patients and patients starting IGRT de novo. [ABSTRACT FROM AUTHOR]

This study aimed to understand current treatment patterns and healthcare resource utilization (HRU) of women with locally advanced or metastatic breast cancer (advanced breast cancer [ABC]) in Taiwan overall and within the subgroup of patients who were postmenopausal women with no previous systemic therapy in the ABC setting. A chart review of anonymized data on patient characteristics, treatment patterns, and HRU was conducted via an online physician survey including 118 patient charts from women ≥ 18 years old with hormone receptor positive/human epidermal growth receptor negative ABC, diagnosed between 2015 and 2017. The mean age of all patients was 56.6 years (range 29-83). Among the 118 patients, the most common first-line systemic therapy group after diagnosis of ABC was endocrine-based therapy (39.0%) or endocrine therapy (ET) plus chemotherapy (ChT) combinations (38.1%). In the postmenopausal subgroup (n = 56), ET-based therapy was the most common (44.6%). Oncologist visits, at annual rate of 9.20 (95% confidence interval 8.81-9.60), and hospitalizations, at annual rate of 1.08 (95% confidence interval 0.96-1.22), were key drivers of HRU. Of the 118 patients, the 72 with at least one ChT agent in their first-line regimen had an annual hospitalization rate of 1.4 versus 0.45 admissions compared with the 46 patients on first-line ET-based therapy. Current treatment patterns suggest an unmet need for new medications that lead to reduction in high rate of ChT use. Results can inform future evaluations of new ABC treatments that estimate the health economic impact of their adoption in Taiwan. • This study presents an up-to-date overview of the current treatment patterns and healthcare resource utilization in patients with advanced breast cancer (ABC) in the real-world setting in Taiwan. Anticancer treatments since diagnosis of ABC included a variety of therapies, including hormonal therapy, chemotherapy (ChT), other targeted and biologic therapies, ovarian suppression, ovarian ablation, and other radiation and surgery. • Healthcare resource utilization data indicated that oncologist visits and hospitalizations were the key drivers of health resource utilization. These results can inform future evaluations of new ABC treatments that estimate the health economic impact of their adoption in Taiwan. • The study also highlighted that current treatment patterns suggest an unmet need for new medications, including for the overall patient population and the subgroups of patients based on postmenopausal status, presence of visceral disease, and first-line ChT use, that can lead to reduction in high rate of ChT use and frequent severe adverse events associated with them. [ABSTRACT FROM AUTHOR]

Abstract Objective A beta version (2018) of International Classification of Diseases, 11th Revision for MMS (ICD-11), needed testing. Field-testing involves real-world application of the new codes to examine usability. We describe creating a dataset and characterizing the usability of ICD-11 code set by coders. We compare ICD-11 against ICD-10-CA (Canadian modification) and a reference standard dataset of diagnoses. Real-world usability encompasses code selection and time to code a complete inpatient chart using ICD-11 compared with ICD-10-CA. Methods and results A random sample of inpatient records previously coded using ICD-10-CA was selected from hospitals in Calgary, Alberta (N = 2896). Nurses examined these charts for conditions and healthcare-related harms. Clinical coders re-coded the same charts using ICD-11 codes. Inter-rater reliability (IRR) and coding time improved with ICD-11 coding experience (23.6 to 9.9 min average per chart). Code structure comparisons and challenges encountered are described. Overall, 86.3% of main condition codes matched. Coder comments regarding duplicate codes, missing codes, code finding issues enabled improvements to the ICD-11 Browser, Coding Tool, and Reference Guide. Training is essential for solid IRR with 17,000 diagnostic categories in the new ICD-11. As countries transition to ICD-11, our coding experiences and methods can inform users for implementation or field testing.

Abstract Background Impaired self-awareness (i.e., a lack of insight) is experienced by most individuals who have sustained a moderate to severe traumatic brain injury (TBI). During the early recovery period post-injury, these individuals may not be able to recognize their abilities and limitations, hence, negatively impacting their daily life and function. Although there are assessments and interventions to improve self-awareness after TBI, little is known about how clinicians assess and address this impairment in an inpatient rehabilitation setting. Objective To examine how clinicians assess, report, and provide interventions for impaired self-awareness after TBI. Methods A retrospective chart review was conducted on interdisciplinary rehabilitation clinician entries for individuals with TBI (n = 67) who received inpatient rehabilitation within a five-year period (2014–2019). A reflexive thematic analysis was used to identify themes pertaining to self-awareness. Results Three themes were generated to explore clinician responses to their clients’ impaired self-awareness: 1) ‘recalling and understanding’ described clinician observations of client behaviors and expressions of self-awareness, 2) ‘applying and analyzing’ identified clinicians providing relevant tasks and advice to clients, and 3) ‘evaluating and creating’ described clinicians actively interacting with clients by providing feedback, guided prompts, and a follow-up plan. Conclusion Clinicians produced varied responses to clients’ impaired self-awareness after TBI. Findings may help to develop research priorities and integrated knowledge translation initiatives to increase evidence-based practice for impaired self-awareness after TBI.

This study aimed to evaluate the risk factors and occurrence of pulpal disease in patients who received either full-coverage (crowns) or large noncrown restorations (fillings, inlays, or onlays involving ≥3 surfaces). A retrospective chart review identified 2177 cases of large restorations placed on vital teeth. Based on the restoration type, patients were stratified into various groups for statistical analysis. After restoration placement, those who required endodontic intervention or extraction were classified as having pulpal disease. Over the course of the study, 8.77% (n = 191) of patients developed pulpal disease. Pulpal disease was slightly more common in the large noncrown group than the full-coverage group (9.05% vs 7.54%, respectively). For patients who received large fillings, there was not a statistically significant difference based on operative material (amalgam vs composite: odds ratio = 1.32 [95% confidence interval, 0.94–1.85], P >.05) or the number of surfaces involved (3 vs 4: odds ratio = 0.78 [95% confidence interval, 0.54–1.12], P >.05). The association between the restoration type and the pulpal disease treatment performed was statistically significant (P <.001). The full-coverage group more frequently underwent endodontic treatment than extraction (5.78% vs 3.37%, respectively). Only 1.76% (n = 7) of teeth in the full-coverage group were extracted compared with 5.68% (n = 101) in the large noncrown group. It appears that ∼9% of patients who receive large restorations will go on to develop pulpal disease. The risk of pulpal disease tended to be highest in older patients who receive large (4 surface) amalgam restorations. However, teeth with full-coverage restorations were less likely to be extracted. [ABSTRACT FROM AUTHOR]

Abstract Background Adults with attention-deficit hyperactivity disorder (ADHD) often cycle through multiple treatments for reasons that are not well documented. This study analyzed the reasons underlying treatment changes among adults treated for ADHD in a real-world setting. Methods Data were collected via an online reporting form completed by eligible physicians between October and November 2020. Data for adult patients in the United States who were diagnosed with ADHD and initiated a treatment regimen within 1 to 5 years of chart abstraction were obtained. Reason for a treatment change was described for a randomly selected regimen episode, which spanned from treatment initiation until the earliest among treatment add-on/switch or discontinuation, death, or date of chart abstraction. The overall rate of ADHD/treatment-related complications were also described. Physician satisfaction with current treatment options for adult ADHD and opinions on areas for improvement were assessed. Results Data on 320 patients were reported by 152 physicians specializing in psychiatry (40.1%), pediatrics (25.0%), family medicine (21.7%), and internal medicine (13.2%). Patients had a mean age of 29.3 years; most were diagnosed with ADHD as adults (57.5%) and within the previous 5 years (56.5%). Selected treatment regimens included stimulants (79.1%), nonstimulants (14.7%), and combination therapy (5.6%) for an average duration of 1.9 years. Among patients with treatment discontinuation (N = 59), the most common reasons for discontinuation were suboptimal symptom management (55.9%), occurrence of ADHD/treatment-related complications (25.4%), and patient attitude/dislike of medication (25.4%). The main reasons for other key treatment changes were inadequate/suboptimal management of symptoms and cost considerations. Over 40% of patients had ≥ 1 documented ADHD/treatment-related complication, irrespective of whether they led to a treatment change. One in 5 physicians (19.8%) were very dissatisfied, moderately dissatisfied, or neither satisfied nor dissatisfied with current treatment options for ADHD in adults; the top 3 suggested improvements were lower risk of abuse (71.7%), longer effect duration (65.1%), and fewer ADHD/treatment-related complications (61.2%). Conclusions The top reasons for treatment changes among adults with ADHD are lack of efficacy and ADHD/treatment-related complications, highlighting the importance of developing more effective and safer treatments to alleviate the burden of ADHD.

A retrospective study published in the International Journal of Dermatology examined the prevalence of malignancy and associated risk factors in patients with prurigo nodularis (PN), a debilitating skin disease characterized by itchy nodules. The study found that 27% of PN patients were diagnosed with concomitant malignancy, including non-melanoma skin cancer (NMSC) and other types of cancer such as melanoma, ovarian, breast, lung, uterine, cervical, and colon cancer, as well as cutaneous T-cell lymphoma and non-Hodgkin's lymphoma. The study also identified increased age at PN diagnosis and the presence of pain as significant risk factors for malignancy development. However, the study had limitations due to its retrospective nature and sample size. [Extracted from the article]

Knowledge about work-related occupational therapy in psychiatric inpatient and day hospital clients is limited. The aim of the study was to explore the desired and achieved productivity status in mental health service users participating in a pre-vocational therapy (PVT) intervention. Outcome variables were productivity status and achievement of desired productivity status at discharge, and change or persistence of productivity status between admission and discharge. This is a short-term prospective study based on observational data from health records (N = 98). At admission, 53.2% of participants named a clear desired productivity status; 46.8% did not. Of the former, 76.9% expressed a desire for employment or education on the regular job market; 28.0% achieved their desired outcome at discharge, whereas 72.0% did not. Overall, 58.5% retained, established or increased productive activities, while 41.5% were not engaged in or had reduced productive activities at discharge. These two groups differed in socio-economic and social-security-related characteristics, treatment-related characteristics and psychiatric diagnosis. Some, but not all kinds of clients in acute psychiatric inpatient and day hospital settings manage to take a first step towards productive activities during the first phase of treatment. [ABSTRACT FROM AUTHOR]

Importance: Vaginoplasty is a relatively common gender‐affirming surgery with approximately 200 Ontarians seeking this surgery annually. Although Ontario now offers vaginoplasty in province, the capacity is not meeting demand; the majority of trans and gender‐diverse patients continue to seek vaginoplasty out of province. Out‐of‐province surgery presents a barrier to accessing postsurgical follow‐up care leaving most patients to seek support from their primary care providers or providers with little experience in gender‐affirming surgery. Objective: To provide an account of the common postoperative care needs and neovaginal concerns of Ontarians who underwent penile inversion vaginoplasty out of province and presented for care at a gender‐affirming surgery postoperative care clinic. Design, Settings, and Participants: A retrospective chart review of the first 80 patients presenting to a gender‐affirming surgery postoperative care clinic who had undergone vaginoplasty at an outside surgical center was performed. Descriptive analyses were performed for all variables. Results: The sample consisted of 80 individuals with the mean age of 39 years (19–73). Most patients had surgery at another surgical center in Canada (76.3%). Many patients (22.5%) accessed care in the first 3 months after surgery, with the majority (55%) seeking care within the first perioperative year. Most patients (61.3%) were seen for more than one visit and presented with more than two symptoms or concerns. Common patient‐reported symptoms during clinical visit included pain (53.8%), dilation concerns (46.3%), and surgical site/vaginal bleeding (42.5%). Sexual function concerns were also common (33.8%) with anorgasmia (11.3%) and dyspareunia (11.3%) being the most frequent complications. The most common adverse outcomes identified by health care providers included hypergranulation (38.8%), urinary dysfunction (18.8%), and wound healing issues (12.5%). Conclusions and Relevance: Findings from chart review offer valuable insights into the postoperative needs and neovaginal concerns of Ontarians who have had vaginoplasty out of province. This study demonstrates the need for routine postoperative care in patients undergoing vaginoplasty. Patients experience numerous symptoms and concerns that often correlate with clinical findings and require multiple follow‐up appointments. Health care providers may benefit from further education on the more common nonsurgical issues identified in this study. [ABSTRACT FROM AUTHOR]

Mixed-methods were used to investigate treatment outcomes for youth enrolled in an innovative sports-based group therapy intervention. Chart review was conducted for 65 youth, aged 5 to 13 years over one academic year at an elementary school serving high-needs youth in an urban district. Analysis revealed symptom reduction for 72% of participants. For a subset of students (n = 34) with available Child and Adolescent Needs and Strengths (CANS) Assessments, the average number of adverse experiences was 2.74 (SD : 2.11). Qualitative coding of progress notes and discharge summaries was used to assess symptom presentation and treatment outcomes. This sports-based group therapy intervention promotes access to services while minimizing stigma. [ABSTRACT FROM AUTHOR]

This European observational chart review assessed the efficacy/safety of recombinant von Willebrand factor (rVWF) for on-demand treatment of spontaneous/traumatic bleeds and prevention and/or treatment of surgery-related bleeding in adults with von Willebrand disease (VWD). Patients (n = 91) were enrolled at first rVWF administration (index). Data were collected for the 12 months before index and until death, loss to follow-up, or end of study (3-12 months after index). Fifteen patients reported an rVWF-treated spontaneous/traumatic bleed at index. Bleed resolution was obtained for 14 patients (unknown status, n = 1), and investigators assessed treatment satisfaction for 13 rVWF prescriptions (2 moderate, 5 good, and 6 excellent). rVWF was used to prevent/treat surgery-related bleeds at index in 76 patients. Bleed resolution was achieved in 25/58 rVWF-treated surgeries; bleed resolution was not applicable for 33 surgeries. In both groups, there were no reports of treatment-emergent adverse events after initiating rVWF, including hypersensitivity reactions, thrombotic events, and VWF inhibitor development. rVWF was shown to be effective for the on-demand treatment of spontaneous/traumatic bleeds, and for the prevention and treatment of surgical bleeds in this real-world VWD population. [ABSTRACT FROM AUTHOR]

Objective To provide high-quality data for coronavirus disease 2019 (COVID-19) research, we validated derived COVID-19 clinical indicators and 22 associated machine learning phenotypes, in the Mass General Brigham (MGB) COVID-19 Data Mart. Methods Fifteen reviewers performed a retrospective manual chart review for 150 COVID-19-positive patients in the data mart. To support rapid chart review for a wide range of target data, we offered a natural language processing (NLP)-based chart review tool, the Digital Analytic Patient Reviewer (DAPR). For this work, we designed a dedicated patient summary view and developed new 127 NLP logics to extract COVID-19 relevant medical concepts and target phenotypes. Moreover, we transformed DAPR for research purposes so that patient information is used for an approved research purpose only and enabled fast access to the integrated patient information. Lastly, we performed a survey to evaluate the validation difficulty and usefulness of the DAPR. Results The concepts for COVID-19-positive cohort, COVID-19 index date, COVID-19-related admission, and the admission date were shown to have high values in all evaluation metrics. However, three phenotypes showed notable performance degradation than the positive predictive value in the prepandemic population. Based on these results, we removed the three phenotypes from our data mart. In the survey about using the tool, participants expressed positive attitudes toward using DAPR for chart review. They assessed that the validation was easy and DAPR helped find relevant information. Some validation difficulties were also discussed. Conclusion Use of NLP technology in the chart review helped to cope with the challenges of the COVID-19 data validation task and accelerated the process. As a result, we could provide more reliable research data promptly and respond to the COVID-19 crisis. DAPR's benefit can be expanded to other domains. We plan to operationalize it for wider research groups. [ABSTRACT FROM AUTHOR]

To understand current treatment patterns and health care resource utilization (HRU) of women with locally advanced or metastatic breast cancer (advanced breast cancer; ABC) in Korea overall and within patients who had progressed with prior endocrine therapy (as first-line treatment for metastatic disease) and patients with no prior systemic treatment (for advanced disease). A chart review was conducted in 109 patients (women ≥ 18 years old with HR+/HER2- ABC diagnosed between 2015 and 2017) from 11 hospitals. Anonymized data on patient characteristics, treatment patterns and HRU was abstracted. Mean (range) age of all patients was 57.5 (40–81) years. Overall, the most common first-, second- and third-line systemic therapy after diagnosis of ABC were letrozole ± palbociclib (51%), endocrine therapy (ET)±everolimus (42%) or chemotherapy (ChT) (39%), and ChT (68%), respectively. In patients progressed with ET (n = 33) and those with no prior systemic treatment (n = 52), the most common first-line treatments were letrozole (82%) and letrozole + palbociclib (42%), respectively. The percentage of patients with at least one grade 3 or higher adverse event during first-line therapy was 93.1% vs 39.2% in patients on a ChT based regimen (N = 29) vs. ET (N = 74). Overall, oncologist visits, at an annual rate of 9.27 (95% CI: 8.87, 9.69) visits per month, and hospitalizations, with an annual rate of 0.44 (95% CI: 0.36, 0.54), and mean (SD) length of stay of 14.3 (10.32) days, were the key drivers of HRU. These findings on real world HRU reflected clinical guidelines and severity of ABC. Results can inform future evaluations of new ABC treatments that estimate the health economic impact of their adoption in Korea. [ABSTRACT FROM AUTHOR]

Introduction: This study aimed to examine the reasons underlying treatment changes among pediatric patients with attention-deficit/hyperactivity disorder (ADHD). Methods: Data were obtained through online medical chart abstraction (08/2021–09/2021). Eligible patients with ADHD had initiated a treatment regimen at ages 6–17 and within 1–5 years of chart abstraction. Reasons contributing to treatment changes were analyzed for a randomly selected treatment episode. ADHD/treatment-related complication rate was also described. Results were reported overall and among children (ages 6–12) and adolescents (ages 13–17), separately. Physicians' perspective on adherence among their child, adolescent, and adult patients was assessed through an online survey. Results: A total of 156 physicians abstracted 434 patient charts (235 children + 199 adolescents). Mean patient age was 11.3 years, and 68.7% were male. Inadequate/suboptimal symptom management was the most common reason for treatment discontinuation (50/83 [60.2%]), add-on (17/21 [81.0%]), and dose increase (189/237 [79.7%]). Patient/parent/family attitude/dislike of medication and ADHD/treatment-related complications were common reasons for treatment discontinuation, add-on, switch, and dose decrease. Overall, 42.4% of patients had ≥ 1 documented ADHD/treatment-related complication, insomnia/sleep disturbances being the most common (9.7%). Among patients with ≥ 1 complication, 75.5% reported the experience/fear of complications had a negative impact on their treatment adherence. Results were similar among children and adolescents. Physicians reported taking actions toward patients' non-adherence by further educating patients, closer monitoring, and changing the prescribed ADHD medication. Conclusion: Lack of effectiveness and ADHD/treatment-related complications are important reasons for treatment changes among children and adolescents with ADHD, highlighting the need for more effective and tolerable treatments to mitigate the burden of ADHD. [ABSTRACT FROM AUTHOR]

Natalie Woods,1,2 Kristy Wittmeier,1– 3 Kathy Mulder,1 Brenden Dufault,4 Brian Black5,6 1Physiotherapy Department, Health Sciences Centre Winnipeg – Shared Health, Winnipeg, MB, Canada; 2Children’s Hospital Research Institute of Manitoba, Winnipeg, MB, Canada; 3Department of Pediatrics and Child Health, Rady Faculty of Health Sciences, University of Manitoba, Winnipeg, MB, Canada; 4Data Sciences Platform, George & Fay Yee Centre for Healthcare Innovation, Winnipeg, MB, Canada; 5Department of Pediatric Orthopedic Surgery, University of Manitoba, Winnipeg, MB, Canada; 6Child Health Program, Health Sciences Centre Winnipeg – Shared Health, Winnipeg, MB, CanadaCorrespondence: Brian Black, Child Health Program, Health Sciences Centre Winnipeg – Shared Health, 840 Sherbrook Street, Winnipeg, MB, R3A 1S1, Canada, Tel +1-204-787-4204, Fax +1-204-787-2460, Email BBlack@hsc.mb.caPurpose: To evaluate the relationship between body mass index (BMI) and spinal curvature in patients presenting with idiopathic scoliosis at a major pediatric tertiary care centre.Patients and Methods: Retrospective chart review (2015– 2019). Data extracted from patient’s first visit included age, sex, height, weight, spinal curvature (magnitude, location), referral source, physical activity participation (yes/no), pain (yes/no). Demographics were analyzed descriptively. The relationship between BMI and spinal curve magnitude was analyzed using Spearman correlation coefficient. Linear regression was applied to determine the relationship between BMI, curve magnitude, and curve location. Exploratory univariate analyses were conducted for BMI and referral source, pain, and skeletal maturity, and physical activity and pain.Results: A total of 206 patient charts were included (177 females, 29 males). Patients presented with double major (41.3%), thoracic (26.7%), thoracolumbar (22.8%), and lumbar (9.2%) curves. Mean (SD) BMI percentile was 48.3 (30.5). No relationship existed between BMI percentile and curve magnitude with curve locations combined. However, a test for interaction revealed a positive relationship between BMI percentile and curve magnitude for adolescents with double major curves, and a negative relationship for adolescents with thoracic curves. Exploratory analyses suggested a relationship between BMI percentile and presence of pain, and between referral source and curve magnitude. No relationship was observed between BMI and skeletal maturity, or physical activity and pain.Conclusion: The relationship between BMI varied by curve location within this cohort, in which most patients presented with a BMI < 85th percentile. Findings highlight the importance of sensitive history taking and careful physical examination for early detection of scoliosis.Keywords: scoliosis, body mass index, curve magnitude, screening, chart review

Abstract Background Fremanezumab, a fully humanized monoclonal antibody (mAb; IgG2Δa) that selectively targets calcitonin gene-related peptide (CGRP), is approved for the preventive treatment of migraine in adults. The efficacy and safety of fremanezumab for migraine prevention have been demonstrated in randomized, double-blind, placebo-controlled trials. Real-world effectiveness data are needed to complement clinical trial data. This study assessed the effectiveness of fremanezumab across different subgroups of adult patients with episodic migraine (EM), chronic migraine (CM), or difficult-to-treat (DTT) migraine in real-world clinical settings. Methods This retrospective, panel-based online chart review used electronic case report forms. Patient inclusion criteria were a physician diagnosis of EM or CM; age ≥ 18 years at the time of first fremanezumab initiation; ≥ 1 dose of fremanezumab treatment; ≥ 1 follow-up visit since first initiation; and ≥ 2 measurements of monthly migraine days (MMD; with 1 within a month before or at first initiation and ≥ 1 after first initiation). Changes in MMD and monthly headache days were assessed during the follow-up period. These endpoints were evaluated in subgroups of patients by migraine type (EM/CM) and in subgroups with DTT migraine (diagnosis of medication overuse [MO], major depressive disorder [MDD], generalized anxiety disorder [GAD], or prior exposure to a different CGRP pathway–targeted mAb [CGRP mAb]). Results Data were collected from 421 clinicians and 1003 patients. Mean (percent) reductions from baseline in MMD at Month 6 were − 7.7 (77.0%) in EM patients, − 10.1 (68.7%) in CM patients, − 10.8 (80.6%) in the MO subgroup, − 9.9 (68.3%) in the MDD subgroup, − 9.5 (66.4%) in the GAD subgroup, and − 9.0 (68.7%) in the prior CGRP mAb exposure subgroup. Improvements in MDD or GAD severity were reported by 45.5% and 45.8% of patients with comorbid MDD or GAD, respectively. Conclusions In this real-world study, fremanezumab demonstrated effectiveness for migraine regardless of migraine type or the presence of factors contributing to DTT migraine (MO, GAD, MDD, or prior exposure to a different CGRP mAb).

Abstract Background The efficacy and tolerability of fremanezumab, a fully humanized monoclonal antibody (IgG2Δa) that selectively targets calcitonin gene-related peptide (CGRP) and is approved for the preventive treatment of migraine in adults, have been demonstrated in randomized, double-blind, placebo-controlled trials. Real-world data can further support those clinical trial data and demonstrate the full clinical benefits of fremanezumab. This chart review assessed the effectiveness of fremanezumab for improving clinical outcomes in adult patients with migraine treated according to real-world clinical practice. Methods This retrospective, panel-based, online physician chart review study used electronic case report forms with US physicians. Patient inclusion criteria were a physician diagnosis of migraine, fremanezumab treatment initiation at ≥ 18 years of age after US Food and Drug Administration approval, ≥ 1 dose of fremanezumab treatment, and ≥ 2 assessments of monthly migraine days (MMD; 1 within 30 days before treatment initiation and ≥ 1 after initiation). Changes from baseline in MMD, monthly headache days (MHD), and Migraine Disability Assessment (MIDAS) and 6-item Headache Impact Test (HIT-6) scores were assessed over 6 months. These endpoints were evaluated in the overall population and subgroups divided by dosing schedule and number of prior migraine preventive treatment failures. Results This study included data from 421 clinicians and 1003 patients. Mean age at fremanezumab initiation was 39.7 years, and most patients were female (75.8%). In the overall population, mean baseline MMD and MHD were 12.7 and 14.0, respectively. Mean (percent) reductions from baseline in MMD and MHD, respectively, were − 4.6 (36.2%) and − 4.7 (33.6%) at Month 1, − 6.7 (52.8%) and − 6.8 (48.6%) at Month 3, and − 9.2 (72.4%) and − 9.8 (70.0%) at Month 6. Mean (percent) reductions from baseline in MIDAS and HIT-6 scores also increased over the 6-month study period, from − 6.2 (21.6%) and − 8.4 (14.0%) at Month 1 to − 18.1 (63.1%) and − 16.2 (27.0%) at Month 6, respectively. Improvements in these outcomes over 6 months were observed across all evaluated subgroups. Conclusions This real-world study demonstrated effectiveness of fremanezumab treatment for up to 6 months, irrespective of dosing regimen or number of prior migraine preventive treatment failures, reflecting ongoing, clinically meaningful improvements in patient outcomes.

Kelly K Nichols,1 Eric D Donnenfeld,2 Charis Lau,3 Annie Syntosi,4 Paul Karpecki,5 John A Hovanesian6,7 1School of Optometry, University of Alabama at Birmingham, Birmingham, AL, USA; 2New York University Medical Center, New York, NY, USA; 3Retina Global Medical Affairs, Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA; 4Retina Global Patient Access, Novartis Pharma AG, Basel, Switzerland; 5Kentucky Eye Institute, Lexington, KY, USA; 6Harvard Eye Associates, Laguna Hills, CA, USA; 7UCLA Jules Stein Eye Institute, Los Angeles, CA, USACorrespondence: John A Hovanesian, Harvard Eye Associates, 24401 Calle de la Louisa, Suites 300-312, Laguna Hills, CA, 92653, USA, Tel +1 949-742-3937, Fax +1 844-479-0584, Email johnhova@gmail.com; jhovanesian@HarvardEye.comPurpose: To assess the frequency of patients reducing the use of artificial tears (ATs) among patients with dry eye disease (DED) following lifitegrast treatment.Patients and Methods: Two independent analyses were performed using the data from the 1-year, randomized, multicenter, Phase 3 SONATA trial and a noninterventional, real-world evidence (RWE) study conducted in patients with DED who were treated with lifitegrast in the United States and Canada. In SONATA, patients who had used ATs in the lifitegrast and placebo groups were included. The RWE study reviewed patients’ electronic medical records, prescribing patterns, and practices of physicians throughout the survey. These data were then used to compare the proportion of patients using ATs in the 6-month pre-index period versus the 12-month post-index period.Results: Of 293 patients (lifitegrast, n=195; placebo, n=98) from SONATA, 107 (lifitegrast, n=64; placebo, n=43) used ATs during the on-therapy period while 186 (lifitegrast, n=131; placebo, n=55) did not. Of those not using ATs, the proportion of patients in the lifitegrast group at any time was higher (∼ 67% [n=131]) versus placebo (∼ 56% [n=55]); this was the case at all study time-points (Days 90, 180, 270, and 360). The RWE study included 600 patient charts (US, n=550; Canada, n=50); 75.5% (n=453) reported AT use. There was ∼ 40% decrease in the proportion of patients using ATs as adjunct DED therapy to lifitegrast in the post-index period (n=273) versus those in the pre-index period (n=453).Conclusion: The findings show that the reliance on AT use can be gradually reduced with lifitegrast treatment, eventually leading to a reduction in disease burden.Keywords: dry eye disease, lifitegrast, artificial tears, SONATA, real-world evidence, chart review

[Display omitted] Phenotypic misclassification in genetic association analyses can impact the accuracy of PRS-based prediction models. The bias reduction method proposed by Tong et al. (2019) has demonstrated its efficacy in reducing the effects of bias on the estimation of association parameters between genotype and phenotype while minimizing variance by employing chart reviews on a subset of the data for validating phenotypes, however its improvement of subsequent PRS prediction accuracy remains unclear. Our study aims to fill this gap by assessing the performance of simulated PRS models and estimating the optimal number of chart reviews needed for validation. To comprehensively assess the efficacy of the bias reduction method proposed by Tong et al. in enhancing the accuracy of PRS-based prediction models, we simulated each phenotype under different correlation structures (an independent model, a weakly correlated model, a strongly correlated model) and introduced error-prone phenotypes using two distinct error mechanisms (differential and non-differential phenotyping errors). To facilitate this, we used genotype and phenotype data from 12 case-control datasets in the Alzheimer's Disease Genetics Consortium (ADGC) to produce simulated phenotypes. The evaluation included analyses across various misclassification rates of original phenotypes as well as quantities of validation set. Additionally, we determined the median threshold, identifying the minimal validation size required for a meaningful improvement in the accuracy of PRS-based predictions across a broad spectrum. This simulation study demonstrated that incorporating chart review does not universally guarantee enhanced performance of PRS-based prediction models. Specifically, in scenarios with minimal misclassification rates and limited validation sizes, PRS models utilizing debiased regression coefficients demonstrated inferior predictive capabilities compared to models using error-prone phenotypes. Put differently, the effectiveness of the bias reduction method is contingent upon the misclassification rates of phenotypes and the size of the validation set employed during chart reviews. Notably, when dealing with datasets featuring higher misclassification rates, the advantages of utilizing this bias reduction method become more evident, requiring a smaller validation set to achieve better performance. This study highlights the importance of choosing an appropriate validation set size to balance between the efforts of chart review and the gain in PRS prediction accuracy. Consequently, our study establishes a valuable guidance for validation planning, across a diverse array of sensitivity and specificity combinations. [ABSTRACT FROM AUTHOR]

Abstract Background Medullary thyroid cancer (MTC) accounts for approximately 1.6% of new cases of thyroid cancer. The objective of this study was to describe patient characteristics, biomarker testing, treatment patterns, and clinical outcomes among patients with advanced/metastatic MTC in a real-world setting in the United States and to identify potential gaps in the care of these patients. Methods Selected oncologists retrospectively reviewed medical records of patients aged ≥ 12 years diagnosed with advanced MTC. Patients must have initiated ≥ 1 line of systemic treatment for advanced/metastatic MTC between January 2013–December 2018 to be eligible. Patient characteristics, biomarker testing, and treatment patterns were summarized descriptively; progression-free survival (PFS) and overall survival (OS) were estimated using the Kaplan–Meier method. Results The 203 patients included in this study had a mean (SD) age of 52.2 (10.4) years; mean (SD) duration of follow-up from start of first-line treatment was 24.5 (16.0) months. Most patients (82.8%) were initially diagnosed with stage IVA, IVB, or IVC disease. Among all patients, 121 (59.6%) had testing for RET mutations, of whom 37.2% had RET-mutant MTC. The RET-mutation type was reported for 28 patients; the most common mutations reported were M918T (64.3%) and C634R (32.1%). Of the 203 patients, 75.9% received only one line of systemic treatment for advanced disease, and 36% were still undergoing first-line therapy at the time of data extraction. Cabozantinib (30.0%), vandetanib (30.0%), sorafenib (17.2%), and lenvatinib (4.9%) were the most common first-line treatments. Among 49 patients who received second-line treatment, most received cabozantinib (22.4%), vandetanib (20.4%), lenvatinib (12.2%), or sunitinib (12.2%). Median PFS (95% confidence interval [CI]) from start of first- and second-line treatments was 26.6 months (20.8–60.8) and 15.3 months (6.6-not estimable [NE]), respectively. Median OS from initiation of first- and second-line treatment was 63.8 months (46.3-NE) and 22.4 months (12.4-NE), respectively. Conclusions For the treatment of advanced/metastatic MTC, no specific preference of sequencing systemic agents was observed in the first- and second-line settings. Considering the recent approval of selective RET inhibitors for patients with RET-mutant MTC, future research should investigate how treatment patterns evolve for these patients.

Abstract Background Lenvatinib monotherapy was approved in the United States for first‐line treatment of patients with unresectable hepatocellular carcinoma (uHCC) in 2018. This study assessed real‐world treatment patterns and outcomes of lenvatinib beyond first‐line systemic treatment in the United States. Methods A retrospective study was conducted among US adults (≥18 years) with uHCC. Eligible patients initiated lenvatinib monotherapy as second‐ or later‐line systemic therapy (2L‐plus) from August 2018 to September 2019. Clinical outcomes included physician‐reported best response, progression‐free survival (PFS), and overall survival (OS). Results Of 164 patients who received lenvatinib in 2L‐plus, most (n = 133; 81.1%) received lenvatinib in 2 L. There were 109 patients (66.4%) who initiated lenvatinib after immunotherapy. At lenvatinib initiation, only 31.1% of patients had Child‐Pugh class A, while half (49.4%) had Child‐Pugh class B. Most patients had Barcelona Clinic Liver Cancer stage B (23.8%) or C (38.4%) uHCC. Median duration of lenvatinib treatment was 6.9 months, with 42.7% of patients still on treatment at the end of follow‐up. Physician‐reported best response was complete and partial response for 8.5% and 44.5% of patients, respectively. PFS and OS rate estimates from lenvatinib initiation at 12 months were 51.7% and 57.8%, respectively. Among patients treated after immunotherapy, complete and partial responses were 10.1% and 43.1%, respectively, and PFS and OS estimates from lenvatinib initiation at 12 months were 52.8% and 60.0%, respectively. Conclusion This retrospective study suggests clinical effectiveness of lenvatinib monotherapy in a real‐world setting among previously treated patients with uHCC, including among those previously treated with immunotherapy.

Introduction: Left ventricular assist device (LVAD) insertion is an increasingly common intervention for patients with advanced heart failure; however, published literature on the emergency department (ED) presentation of this population is limited. The objective of this study was to characterize ED presentations of patients with LVADs with a focus on device-specific complications to inform provider education and preparation initiatives. Methods: This was a retrospective chart review of all patients with LVADs followed at an urban academic medical center presenting to the ED over a five-year period (July 1, 2009, to June 30, 2014). Two abstractors reviewed 45 randomly selected charts to standardize the abstraction process and establish a priori categories for reason for presentation to the ED. Remaining charts were then divided evenly for review by one of the two abstractors. Primary outcomes for this study were (1) frequency of and (2) reason for presentation to the ED by patients with LVADs. Results: Of 349 patients with LVADs identified, 143 (41.0%) had ED encounters during the study period. There were 620 total ED encounters, (range 1 to 32 encounters per patient, median=3, standard deviation=5.3). Among the encounters, 431 (69.5%) resulted in admission. The most common reasons for presentation were bleeding (e.g., gastrointestinal, epistaxis) (182, 29.4%); infection (127, 20.5%); heart failure exacerbation (68, 11.0%); pain (56, 9.0%); other (45, 7.3%); and arrhythmias (40, 6.5%). Fifty-two encounters (8.4%) were device-specific; these patients frequently presented with abnormal device readings (37, 6.0%). Interventions for device-specific presentations included anticoagulation regimen adjustment (16/52, 30.8%), pump exchange (9, 17.3%), and hardware repair (6, 11.5%). Pump thrombosis occurred in 23 cases (3.7% of all encounters). No patients required cardiopulmonary resuscitation or died in the ED. Conclusion: This is the largest study known to the investigators to report the rate of ED presentations of patients with LVADs and provide analysis of device-specific presentations. In patients who do have device-specific ED presentations, pump thrombosis is a common diagnosis and can present without device alarms. Specialized LVAD education and preparation initiatives for ED providers should emphasize the recognition and management of the most common and critical conditions for this patient population, which have been identified in this study as bleeding, infection, heart failure, and pump thrombosis.