Lay healthcare worker financial toxicity intervention: a pilot financial toxicity screening and referral program.

PURPOSE: Financial toxicity is a source of significant distress for patients with urologic cancers, yet few studies have addressed financial burden in this patient population. METHODS: We developed a financial toxicity screening program using a lay health worker (LHW) and social worker (SW) to assess and mitigate financial toxicity in a single academic medical clinic. As part of a quality improvement project, the LHW screened all newly diagnosed patients with advanced stages of prostate, kidney, or urothelial cancer for financial burden using three COST tool questions and referred patients who had significant financial burden to an SW who provided personalized recommendations. The primary outcome was feasibility defined as 80% of patients with financial burden completing the SW consult. Secondary outcomes were patient satisfaction, change in COST Tool responses, and qualitative assessment of financial resources utilized. RESULTS: The LHW screened a total of 185 patients for financial toxicity; 82% (n = 152) were male, 65% (n = 120) White, and 75% (n = 139) reported annual household income >$100,000 US Dollars; 60% (n = 114) had prostate cancer. A total of 18 (9.7%) participants screened positive for significant financial burden and were referred to the SW for consultation. All participants (100%) completed and reported satisfaction with the SW consultation and had 0.83 mean lower scores on the COST Tool post-intervention assessment compared to pre-intervention (95% confidence interval [0.26, 1.41]). CONCLUSION: This multidisciplinary financial toxicity intervention using an LHW and SW was feasible, acceptable, and associated with reduced financial burden among patients with advanced stages of urologic cancers. Future work should evaluate the effect of this intervention among cancer patients in diverse settings.

The Pediatric Craniofacial Collaborative Group (PCCG) Consensus Conference Methodology.

OBJECTIVE: This article describes the methodology used for the Pediatric Craniofacial Collaborative Group (PCCG) Consensus Conference. DESIGN: This is a novel Consensus Conference of national experts in Pediatric Craniofacial Surgery and Anesthesia, who will follow standards set by the Institute of Medicine and using the Research and Development/University of California, Los Angeles appropriateness method, modeled after the Pediatric Critical Care Transfusion and Anemia Expertise Initiative. Topics related to pediatric craniofacial anesthesia for open cranial vault surgery were divided into twelve subgroups with a systematic review of the literature. SETTING: A group of 20 content experts met virtually between 2019 and 2020 and will collaborate in their selected topics related to perioperative management for pediatric open cranial vault surgery for craniosynostosis. These groups will also identify where future research is needed. CONCLUSIONS: Experts in pediatric craniofacial surgery and anesthesiology are developing recommendations on behalf of the Pediatric Craniofacial Collaborative Group for perioperative management of patients undergoing open cranial vault surgery for craniosynostosis and identifying future research priorities.

Heart rate changes following the administration of sugammadex in children: a prospective, observational study.

INTRODUCTION: Sugammadex is a novel agent to reverse steroidal neuromuscular blocking agents (NMBA) with potential clinical advantages over acetylcholinesterase inhibitors such as neostigmine. However, rare instances of bradycardia were reported during its initial clinical trials. To better define this issue, its incidence and mitigating factors, we prospectively evaluated heart rate changes after sugammadex administration in pediatric-aged patients. METHODS: Patients less than 18 years of age who were to receive sugammadex were included. After sugammadex administration, heart rate (HR) was recorded every minute for 15 min and then every 5 min for the next 15 min or until the patient was transferred from the operating room. Bradycardia was defined as HR below the 5th percentile for age. RESULTS: The study cohort included 221 children. Bradycardia was noted in 18 cases (8%; 95% confidence interval 5%, 13%), occurring at a median of 2 min (IQR: 1, 6) after sugammadex administration. Among patients developing bradycardia, 7 of 18 (38%) had comorbid cardiac conditions (congenital heart disease). No patient required treatment for bradycardia and no clinically significant blood pressure (BP) changes were noted. On bivariate analysis, initial sugammadex dose was not associated with bradycardia onset. In multivariable analysis, cardiac comorbid conditions and male gender were associated with an increased incidence of bradycardia. CONCLUSIONS: The incidence of bradycardia following the administration of sugammadex is low, is not associated with BP changes or other clinically significant effects, and did not require treatment. A higher incidence of bradycardia was noted in patients with cardiac comorbid conditions.

Decrease in heart rate following the administration of sugammadex in adults.

BACKGROUND AND AIMS: Sugammadex is a novel agent for reversal of steroidal neuromuscular blocking agents (NMBAs) with potential advantages over acetylcholinesterase inhibitors. In preclinical trials, there have been rare instances of bradycardia with progression to cardiac arrest. To better define this issue, its incidence and mitigating factors, we prospectively evaluated the incidence of bradycardia after sugammadex administration in adults. MATERIAL AND METHODS: Patients ≥ 18 years of age who received sugammadex were included in this prospective, open label trial. After administration, heart rate (HR) was continuously monitored. HR was recorded every minute for 15 minutes and then every five minutes for the next 15 minutes or until patient was transferred out of the operating room. Bradycardia was defined as HR less than 60 beats/minute (bpm) or decrease in HR by ≥ 10 beats per minute (bpm) if the baseline HR was <70 bpm. RESULTS: The study cohort included 200 patients. Bradycardia was observed in 13 cases (7%; 95% confidence interval: 4, 11), occurring a median of 4 minutes after sugammadex administration (IQR: 4, 9, range: 2-25). Among patients developing bradycardia, two (15%) had cardiac comorbid conditions. One patient received treatment for bradycardia with ephedrine. No clinically significant blood pressure changes were noted. On bivariate analysis, patients receiving a higher initial sugammadex dose were more likely to develop bradycardia. On multivariable logistic regression, initial sugammadex dose was not associated with the risk of bradycardia. CONCLUSION: The incidence of bradycardia after administration of sugammadex in our study was low and not associated with significant hemodynamic changes.

Hospital course of underweight youth with ARFID treated with a meal-based behavioral protocol in an inpatient-partial hospitalization program for eating disorders.

OBJECTIVE: Information on nutritional rehabilitation for underweight patients with avoidant/restrictive food intake disorder (ARFID) is scarce. This study characterized hospitalized youth with ARFID treated in an inpatient (IP)-partial hospitalization behavioral eating disorders (EDs) program employing an exclusively meal-based rapid refeeding protocol and compared weight restoration outcomes to those of patients with anorexia nervosa (AN). METHOD: Data from retrospective chart review of consecutive underweight admissions (N = 275; age 11-26 years) with ARFID (n = 27) were compared to those with AN (n = 248) on clinical features, reason for discharge, and weight restoration variables. For patients with ARFID, presenting phenomenology was further characterized by detailed chart review. RESULTS: At admission, 53% of patients with ARFID were vomiting regularly. The predominant ARFID subtype was ARFID-aversive, with close to a third being mixed subtype. Gastrointestinal (GI) symptomatology (81.5%) was the most commonly endorsed reason for restriction. A third had undergone unsuccessful parenteral or enteral tube feeding. Patients with ARFID were more likely male, had higher admission BMI, and slower IP weight gain (1.36 kg /week vs 1.92) compared to patients with AN. Fewer patients with ARFID transitioned to the partial hospitalization program, although the proportion discharged for clinical improvement did not differ and both groups had a mean program discharge BMI >18.5. DISCUSSION: GI symptoms appear a common contributor to restrictive eating amongst hospitalized youth with ARFID. Despite a slightly lower rate of IP weight gain, clinical improvement and weight restoration at discharge were similar for patients with ARFID compared to AN.

Predictive factors for adverse outcomes in pediatric patients undergoing low-risk skin and soft tissue surgery: A database analysis of 6730 patients.

BACKGROUND: There is a paucity of data regarding risk stratification of pediatric patients presenting for low-risk skin and soft tissue surgery. AIMS: We sought to determine the incidence and independent predictors of postoperative complications and unplanned 30-day readmission in a cohort of children undergoing low-risk skin and soft tissue surgery. METHODS: The study included pediatric patients who underwent minor procedures of the skin and soft tissue at continuously enrolled American College of Surgeons National Surgical Quality Improvement Program Pediatric hospitals over a two-year period. The primary outcome was a 30-day postoperative complication composite. The secondary outcome was unplanned 30-day readmission. RESULTS: The final analysis included 6,730 patients. There were a total of 170 postoperative complications among 152 patients (2.23%) with the majority of complications being either wound-related or postoperative mechanical ventilation. The independent predictors for an increased risk of postoperative complication were American Society of Anesthesiologists classification ≥3 and nutritional deficiency. There were 41 unplanned readmissions (0.61%). The presence of a postoperative wound complication or a postoperative pulmonary complication during the index hospital stay was an independent risk factor for unplanned 30-day readmission. CONCLUSION: Pediatric patients with American Society of Anesthesiologists classification ≥3 and nutritional deficiency undergoing low-risk surgery are at risk for the development of postoperative complications. Patients who develop wound and postoperative pulmonary complications are at higher risk for unplanned 30-day readmission. Identification of these higher risk patients may allow the anesthesiologist to implement targeted therapies to minimize the likelihood of occurrence of these complications.

Endoscopic Versus Open Repair for Craniosynostosis in Infants Using Propensity Score Matching to Compare Outcomes: A Multicenter Study from the Pediatric Craniofacial Collaborative Group.

BACKGROUND: The North American Pediatric Craniofacial Collaborative Group (PCCG) established the Pediatric Craniofacial Surgery Perioperative Registry to evaluate outcomes in infants and children undergoing craniosynostosis repair. The goal of this multicenter study was to utilize this registry to assess differences in blood utilization, intensive care unit (ICU) utilization, duration of hospitalization, and perioperative complications between endoscopic-assisted (ESC) and open repair in infants with craniosynostosis. We hypothesized that advantages of ESC from single-center studies would be validated based on combined data from a large multicenter registry. METHODS: Thirty-one institutions contributed data from June 2012 to September 2015. We analyzed 1382 infants younger than 12 months undergoing open (anterior and/or posterior cranial vault reconstruction, modified-Pi procedure, or strip craniectomy) or endoscopic craniectomy. The primary outcomes included transfusion data, ICU utilization, hospital length of stay, and perioperative complications; secondary outcomes included anesthesia and surgical duration. Comparison of unmatched groups (ESC: N = 311, open repair: N = 1071) and propensity score 2:1 matched groups (ESC: N = 311, open repair: N = 622) were performed by conditional logistic regression analysis. RESULTS: Imbalances in baseline age and weight are inherent due to surgical selection criteria for ESC. Quality of propensity score matching in balancing age and weight between ESC and open groups was assessed by quintiles of the propensity scores. Analysis of matched groups confirmed significantly reduced utilization of blood (26% vs 81%, P < .001) and coagulation (3% vs 16%, P < .001) products in the ESC group compared to the open group. Median blood donor exposure (0 vs 1), anesthesia (168 vs 248 minutes) and surgical duration (70 vs 130 minutes), days in ICU (0 vs 2), and hospital length of stay (2 vs 4) were all significantly lower in the ESC group (all P < .001). Median volume of red blood cell administered was significantly lower in ESC (19.6 vs 26.9 mL/kg, P = .035), with a difference of approximately 7 mL/kg less for the ESC (95% confidence interval for the difference, 3-12 mL/kg), whereas the median volume of coagulation products was not significantly different between the 2 groups (21.2 vs 24.6 mL/kg, P = .73). Incidence of complications including hypotension requiring treatment with vasoactive agents (3% vs 4%), venous air embolism (1%), and hypothermia, defined as <35°C (22% vs 26%), was similar between the 2 groups, whereas postoperative intubation was significantly higher in the open group (2% vs 10%, P < .001). CONCLUSIONS: This multicenter study of ESC versus open craniosynostosis repair represents the largest comparison to date. It demonstrates striking advantages of ESC for young infants that may result in improved clinical outcomes, as well as increased safety.

Retinal Hemorrhages: Abusive Head Trauma or Not?

Abusive head trauma is an important cause of morbidity and mortality in infants and young children. Retinal hemorrhages (RHs) are frequently seen, particularly during dilated eye examination of these children. This review focuses on the evaluation of children with RH, with emphasis on the differential diagnosis, pathophysiology, and distinguishing features of RHs due to abusive head trauma. Many causes exist for RHs in infants and children. Most medical and accidental traumatic causes result in a pattern of RH that is nonspecific and not typical of the pattern and distribution of RHs seen in children with abusive head trauma. In children with intracranial hemorrhage and concerns for abuse, the finding of severe, multilayered RHs extending to the periphery of the retina is very specific for abuse as the cause of the findings, especially if retinoschisis is present. There are few other accidental traumatic mechanisms associated with retinoschisis, and the history of such a traumatic event is readily apparent. The indications for ophthalmologic consult, optimal timing of the eye examination, and significance of the findings are specifically discussed.

Self-selection bias in eating disorders outcomes research: Does treatment response of underweight research participants and non-participants differ?

Observational treatment studies provide a valuable alternative to RCTs but are often criticized due to potential self-selection biases. Studies comparing those who do and do not participate in research on eating disorder treatment are scarce, but necessary to evaluate the impact of self-selection bias on outcomes. All consecutive underweight adult first admissions (N = 392) to an integrated inpatient (IP)-partial hospital (PH) behavioral specialty program were invited to participate in a longitudinal study of eating disorder treatment. Demographic and hospital course data were collected on participants (n = 234) and non-participants (n = 158). Participants and non-participants had similar BMI at admission, lengths of stay, and weight gain rates. Participants were less likely than non-participants to end treatment prematurely from IP and were discharged at a higher BMI; the effect size was small. Few differences in hospital course were observed between participants and non-participants. Although participants were more likely to transition to PH and were discharged at a higher BMI, completion of step-down to PH within this integrated IP-PH program rather than research participation status at admission was a better indicator of discharge BMI, which remains the strongest predictor of long-term weight-maintenance in eating disorders.

Intensive treatment for adults with anorexia nervosa: The cost of weight restoration.

OBJECTIVE: Weight restoration in anorexia nervosa (AN) is associated with lower relapse risk; however rate of weight gain and percent of patients achieving weight restoration (BMI ≥ 19 at discharge) vary among treatment programs. We compared both cost/pound of weight gained and cost of weight restoration in a hospital-based inpatient (IP)-partial hospitalization (PH) eating disorders program to estimates of these costs for residential treatment. METHOD: All adult first admissions to the IP-PH program with AN (N = 314) from 2003 to 2015 were included. Cost of care was based on hospital charges, rates of weight gain, and weight restoration data. Results were compared with residential treatment costs extracted from a national insurance claims database and published weight gain data. RESULTS: Average charge/day in the IP-PH program was $2295 for IP and $1567 for PH, yielding an average cost/pound gained of $4089 and $7050, respectively, with 70% of patients achieving weight restoration. Based on published mean weight gain data and conservative cost/day estimates, residential treatment is associated with higher cost/pound, and both higher cost and lower likelihood of weight restoration for most patients. DISCUSSION: The key metrics used in this study are recommended for comparing the cost-effectiveness of intensive treatment programs for patients with AN.

Reducing Postoperative Bleeding After Craniosynostosis Repair Utilizing a Low-Dose Transexamic Acid Infusion Protocol.

BACKGROUND: Craniosynostosis surgery is associated with large volume blood loss and the need for blood transfusion. Recently, the use of tranexamic acid (TXA) has been demonstrated to be helpful in reducing perioperative blood loss in many pediatric procedures. This study used a low-dose pharmacokinetic TXA dosing protocol and assessed its ability to limit perioperative blood loss for craniosynostosis repairs. METHODS: A retrospective chart review was conducted of pediatric craniosynostosis surgeries performed at our institution between September 2011 and December 2014. Outcome measures included comparisons of perioperative blood loss and transfusion rates in patients who had surgery with or without TXA. RESULTS: Twenty-five patients met inclusion criteria. Nine patients had craniosynostosis surgery without TXA (no-TXA group) and 16 patients received TXA (TXA group). The TXA group had significantly higher postoperative hemoglobin levels than the no-TXA group (P = 0.009). This finding was supported by significantly higher postoperative estimated red cell volume in the TXA group (P = 0.017). Postoperative 24-hour drain output was significantly lower in the TXA group (P = 0.042). The volume of packed red blood cells transfused perioperatively was not significantly different between groups. CONCLUSIONS: Patients who received TXA during craniosynostosis surgery at our institution had higher postoperative hemoglobin levels and lower 24-hour drain output. However, TXA was not associated with lower perioperative blood transfusion rates.

Perceived coercion in inpatients with Anorexia nervosa: Associations with illness severity and hospital course.

OBJECTIVE: The use of coercion in the treatment for anorexia nervosa (AN) is controversial and the limited studies to date have focused on involuntary treatment. However, coercive pressure for treatment that does not include legal measures is common in voluntarily admitted patients with AN. Empirical data examining the effect of non-legal forms of coerced care on hospital outcomes are needed. METHOD: Participants (N = 202) with AN, Avoidant/Restrictive Food Intake Disorder (ARFID), or subthreshold AN admitted to a hospital-based behavioral specialty program completed questionnaires assessing illness severity and perceived coercion around the admissions process. Hospital course variables included inpatient length of stay, successful transition to a step-down partial hospitalization program, and achievement of target weight prior to program discharge. RESULTS: Higher perceived coercion at admission was associated with increased drive for thinness and body dissatisfaction, but not with admission BMI. Perceived coercion was not related to inpatient length of stay, rate of weight gain, or achievement of target weight although it was predictive of premature drop-out prior to transition to an integrated partial hospitalization program. DISCUSSION: These results, from an adequately powered sample, demonstrate that perceived coercion at admission to a hospital-based behavioral treatment program was not associated with rate of inpatient weight gain or achieving weight restoration, suggesting that coercive pressure to enter treatment does not necessarily undermine formation of a therapeutic alliance or clinical progress. Future studies should examine perceived coercion and long-term outcomes, patient views on coercive pressures, and the effect of different forms of leveraged treatment.

Comparison of Positive End-Expiratory Pressure of 8 versus 5 cm H2O on Outcome After Cardiac Operations.

PURPOSE: Postoperative positive end-expiratory pressure (PEEP) selection in patients who are mechanically ventilated after cardiac operations often seems random. The aim of this investigation was to compare the 2 most common postoperative initial PEEP settings at our institution, 8 and 5 cm H2O, on postoperative initial tracheal intubation time (primary outcome); cardiovascular intensive care unit (CVICU); hospital length of stay (LOS); occurrence of pneumonia; and hospital mortality (secondary outcomes). MATERIALS AND METHODS: The electronic medical records of patients who were mechanically ventilated after isolated coronary artery bypass grafting (CABG) or combined CABG and valve operations were reviewed. Propensity score matching was used to compare patients with an initial postoperative PEEP setting of 8 cm H2O (n = 4722 [25.9%]) with those who had PEEP of 5 cm H2O (n = 13 535 [74.1%]) on the primary and secondary outcomes listed earlier. RESULTS: There was no difference in initial postoperative intubation time between the PEEP of 8 cm H2O and the PEEP of 5 cm H2O patient groups (mean 11.9 vs 12.0 hours [median 8.2 vs 8.8 hours], P = .89). The groups did not differ on the occurrence of pneumonia (0.43% vs 0.60%, P = .25) nor on hospital mortality (0.47% vs 0.43%, P = .76). Aspiration pneumonia occurrence approached a significant difference (0.06% vs 0.21%, P value = .052), as did CVICU LOS (mean: 47.9 vs 49.8 hours [median: 28.5 vs 28.4 hours], P = .057), but were not statistically different. There was a slight but likely clinically unimportant difference in hospital LOS (7.7 vs 7.4 days, PEEP = 8 vs 5, P < .001). CONCLUSION: Patients being mechanically ventilated after cardiac operations with an initial postoperative PEEP setting of 8 versus 5 cm H2O differed significantly only on hospital LOS but the difference was likely clinically unimportant. Thus, use of 8 cm H2O PEEP in these patients without a clinical indication, although likely not harmful, does not seem beneficial.

The Effect of Systemic Magnesium on Postsurgical Pain in Children Undergoing Tonsillectomies: A Double-Blinded, Randomized, Placebo-Controlled Trial.

BACKGROUND: Tonsillectomy is a frequently performed surgical procedure in children; however, few multimodal analgesic strategies have been shown to improve postsurgical pain in this patient population. Systemic magnesium infusions have been shown to reliably improve postoperative pain in adults, but their effects in pediatric surgical patients remain to be determined. In the current investigation, our main objective was to evaluate the use of systemic magnesium to improve postoperative pain in pediatric patients undergoing tonsillectomy. We hypothesized that children who received systemic magnesium infusions would have less post-tonsillectomy pain than the children who received saline infusions. METHODS: The study was a prospective, randomized, double-blinded, clinical trial. Subjects were randomly assigned using a computer-generated table of random numbers to 1 of the 2 intervention groups: systemic magnesium infusion (initial loading dose 30 mg/kg given over 15 minutes followed by a continuous magnesium infusion 10 mg/kg/h) and the same volume of saline. The primary outcome was pain scores in the postanesthesia care unit (PACU) measured by FLACC (Face, Legs, Activity, Cry, Consolability) pain scores. Pain reduction was measured by the decrement in the area under the pain scale versus 90-minute postoperative time curve using the trapezoidal method. Secondary outcomes included opioid consumption in the PACU, emergence delirium scores (measured by the pediatric anesthesia emergence delirium scale), and parent satisfaction. RESULTS: Sixty subjects were randomly assigned and 60 completed the study. The area under pain scores (up to 90 minutes) was not different between the study groups, median (interquartile range [IQR]) of 30 (0-120) score × min and 45 (0-135) score × min for the magnesium and control groups, respectively (P = 0.74). Similarly, there was no clinically significant difference in the morphine consumption in the PACU between the magnesium group, median (IQR) of 2.0 (0-4.44) mg IV morphine, compared with the control, median (IQR) of 2.5 (0-4.99) mg IV morphine (P = 0.25). The serum level of magnesium was significantly lower in the control group than in the treatment group at the end of the surgery (P < 0.001). CONCLUSIONS: Despite a large number of studies demonstrating the efficacy of systemic magnesium for preventing postsurgical pain in adults, we could not find evidence for a significant clinical benefit of systemic magnesium infusion in children undergoing tonsillectomies. Our findings reiterate the importance of validating multimodal analgesic strategies in children that have been demonstrated to be effective in the adult population.

Child maltreatment in children with medical complexity and disability.

Child maltreatment is common and pediatric healthcare providers are becoming increasingly aware of risk factors and signs of abuse.1-4 Children with disabilities and those with special medical needs are recognized as a population at increased risk of child maltreatment. Understanding this risk and recognizing that not all disabilities confer the same risks can provide deeper insight for pediatric providers regarding the supports these children and their families need to prevent maltreatment.

Comparative healthcare use by adolescents screening positive for sexual exploitation.

BACKGROUND: Although screens for commercial sexual exploitation of children (CSEC) have been developed, little is known about how adolescents at high risk for, or victims of, CSEC compared to non-CSEC adolescents in healthcare utilization as previous studies have not included a control group. OBJECTIVE: Identify where and how often CSEC adolescents presented to medical care in 12 months prior to being identified as compared to non-CSEC adolescents. PARTICIPANTS AND SETTING: Adolescents between 12 and 18 years seen in a tertiary pediatric health care system in a Midwestern city with a metropolitan population of >2 million. METHODS: This was a 46-month retrospective case-control study. Cases included adolescents who screened high risk or positive for CSEC. Control group 1 included adolescents who screened negative for CSEC. Control group 2 were adolescents who were not screened for CSEC, matched to cases and to control group 1. The three study groups were compared for frequency of, location of, and diagnosis given for medical visits. RESULTS: There were 119 CSEC adolescents, 310 CSEC negative, and 429 unscreened adolescents. Compared to the controls, CSEC positive adolescents sought care less frequently (p < 0.001) and were more likely to present to an acute care setting (p < 0.0001). CSEC cases sought medical care in the acute setting more commonly for inflicted injuries (p < 0.001), mental health (p < 0.001), and reproductive health (p = 0.003). In primary care, CSEC adolescents were more commonly seen for reproductive health (p = 0.002) and mental health (p = 0.006). CONCLUSIONS: CSEC adolescents differ from non-CSEC adolescents in frequency, location, and reasons for seeking healthcare.

Equitable and accessible informed healthcare consent process for people with intellectual disability: a systematic literature review.

OBJECTIVE: To identify factors acting as barriers or enablers to the process of healthcare consent for people with intellectual disability and to understand how to make this process equitable and accessible. DATA SOURCES: Databases: Embase, MEDLINE, PsychINFO, PubMed, SCOPUS, Web of Science and CINAHL. Additional articles were obtained from an ancestral search and hand-searching three journals. ELIGIBILITY CRITERIA: Peer-reviewed original research about the consent process for healthcare interventions, published after 1990, involving adult participants with intellectual disability. SYNTHESIS OF RESULTS: Inductive thematic analysis was used to identify factors affecting informed consent. The findings were reviewed by co-researchers with intellectual disability to ensure they reflected lived experiences, and an easy read summary was created. RESULTS: Twenty-three studies were included (1999 to 2020), with a mix of qualitative (n=14), quantitative (n=6) and mixed-methods (n=3) studies. Participant numbers ranged from 9 to 604 people (median 21) and included people with intellectual disability, health professionals, carers and support people, and others working with people with intellectual disability. Six themes were identified: (1) health professionals' attitudes and lack of education, (2) inadequate accessible health information, (3) involvement of support people, (4) systemic constraints, (5) person-centred informed consent and (6) effective communication between health professionals and patients. Themes were barriers (themes 1, 2 and 4), enablers (themes 5 and 6) or both (theme 3). CONCLUSIONS: Multiple reasons contribute to poor consent practices for people with intellectual disability in current health systems. Recommendations include addressing health professionals' attitudes and lack of education in informed consent with clinician training, the co-production of accessible information resources and further inclusive research into informed consent for people with intellectual disability. PROSPERO REGISTRATION: CRD42021290548.

Use of Machine Learning and Lay Care Coaches to Increase Advance Care Planning Conversations for Patients With Metastatic Cancer.

PURPOSE: Patients with metastatic cancer benefit from advance care planning (ACP) conversations. We aimed to improve ACP using a computer model to select high-risk patients, with shorter predicted survival, for conversations with providers and lay care coaches. Outcomes included ACP documentation frequency and end-of-life quality measures. METHODS: In this study of a quality improvement initiative, providers in four medical oncology clinics received Serious Illness Care Program training. Two clinics (thoracic/genitourinary) participated in an intervention, and two (cutaneous/sarcoma) served as controls. ACP conversations were documented in a centralized form in the electronic medical record. In the intervention, providers and care coaches received weekly e-mails highlighting upcoming clinic patients with < 2 year computer-predicted survival and no prior prognosis documentation. Care coaches contacted these patients for an ACP conversation (excluding prognosis). Providers were asked to discuss and document prognosis. RESULTS: In the four clinics, 4,968 clinic visits by 1,251 patients met inclusion criteria (metastatic cancer with no prognosis previously documented). In their first visit, 28% of patients were high-risk (< 2 year predicted survival). Preintervention, 3% of both intervention and control clinic patients had ACP documentation during a visit. By intervention end (February 2021), 35% of intervention clinic patients had ACP documentation compared with 3% of control clinic patients. Providers' prognosis documentation rate also increased in intervention clinics after the intervention (2%-27% in intervention clinics, P < .0001; 0%-1% in control clinics). End-of-life care intensity was similar in intervention versus control clinics, but patients with ≥ 1 provider ACP edit met fewer high-intensity care measures (P = .04). CONCLUSION: Combining a computer prognosis model with care coaches increased ACP documentation.

An expanded access program of risdiplam for patients with Type 1 or 2 spinal muscular atrophy.

OBJECTIVE: The US risdiplam expanded access program (EAP; NCT04256265) was opened to provide individuals with Type 1 or 2 spinal muscular atrophy (SMA) who had no satisfactory treatment options access to risdiplam prior to commercial availability. The program was designed to collect safety data during risdiplam treatment. METHODS: Patients were enrolled from 23 non-preselected sites across 17 states and treated with risdiplam orally once daily. Eligible patients had a 5q autosomal recessive Type 1 or 2 SMA diagnosis, were aged ≥2 months at enrollment, and were ineligible for available and approved SMA treatments or could not continue treatment due to a medical condition, lack/loss of efficacy, or the COVID-19 pandemic. RESULTS: Overall, 155 patients with Type 1 (n = 73; 47.1%) or 2 SMA (n = 82; 52.9%) were enrolled and 149 patients (96.1%) completed the EAP (defined as obtaining access to commercial risdiplam, if desired). The median treatment duration was 4.8 months (range, 0.3-9.2 months). The median patient age was 11 years (range, 0-50 years), and most patients (n = 121; 78%) were previously treated with a disease-modifying therapy. The most frequently reported adverse events were diarrhea (n = 10; 6.5%), pyrexia (n = 7; 4.5%), and upper respiratory tract infection (n = 5; 3.2%). The most frequently reported serious adverse event was pneumonia (n = 3; 1.9%). No deaths were reported. INTERPRETATION: In the EAP, the safety profile of risdiplam was similar to what was reported in pivotal risdiplam clinical trials. These safety data provide further support for the use of risdiplam in the treatment of adult and pediatric patients with SMA.