Prevalence and Risk Factors for Cerebral Palsy in Children With Congenital Heart Disease Based on Risk of Surgical Mortality.

BACKGROUND: Children with congenital heart disease (CHD) have a higher prevalence of motor impairment secondary to brain injury, resulting in cerebral palsy (CP). The purpose of this study is to determine the prevalence of CP in CHD in a single-center cohort, stratify risk based on surgical mortality using Society of Thoracic Surgeons-European Association for Cardio-Thoracic Surgery (STAT) categories and identify risk factors. METHODS: Retrospective cohort study of pediatric patients registered in the University of Florida (UF) Society of Thoracic Surgeons Congenital Heart Surgery database from 2006 to 2017 with a diagnosis of CHD who continued follow-up for more than two years at UF. RESULTS: A total of 701 children with CHD met inclusion criteria. Children identified to have CP were 54 (7.7%). Most common presentation was spastic hemiplegic CP with a Gross Motor Function Classification System of level 2. Analysis of surgical and intensive care factors between the two groups showed that children with CHD and CP had longer time from admission to surgery (P = 0.003), higher STAT categories 4 and 5 (P = 0.038), and higher frequency of brain injury and seizures (P < 0.001). Developmental disabilities and rehabilitation needs were significantly greater for children with CHD and CP when compared with those with CHD alone (P < 0.001). CONCLUSIONS: In our cohort, 7.7% children with CHD develop CP; this is significantly higher than the 2010 US population estimate of 0.3%. Our study suggests higher STAT categories, brain injury, and seizures are associated with developing CP in children with CHD.

Access to Financial Assistance Programs and Their Impact on Overall Spending on Oral Anticancer Medications at an Integrated Specialty Pharmacy.

PURPOSE: Financial assistance (FA) programs are increasingly used to help patients afford oral anticancer medications (OAMs), but access to such programs and their impact on out-of-pocket (OOP) spending has not been well explored. This study aimed to (1) characterize the impact of receipt of FA on both OOP spending and likelihood of catastrophic spending on OAMs and (2) evaluate racial/ethnic disparities in access to FA programs. METHODS: Patients with a cancer diagnosis prescribed an OAM anytime between January 1, 2021, and December 31, 2021 were included in this retrospective, single-center study at an integrated specialty pharmacy affiliated with a tertiary academic cancer center. Fixed-effect regression models were used to characterize the impact of receipt of FA on overall spending and likelihood of catastrophic spending on OAMs, as well as explore the association of race/ethnicity with receipt of FA. RESULTS: Across 1,186 patients prescribed an OAM, 37% received FA. Receipt of FA was associated with lower annual spending on OAMs (ß = -$1,236 US dollars [USD; 95% CI, -$1,841 to -$658], P < .001) but not reduced risk of catastrophic spending (odds ratio [OR], 0.442 [95% CI, 0.755 to 3.199], P = .23). Non-White patients (OR, 0.60 [95% CI, 0.43 to 0.85], P = .004) and patients who spoke English as a second language (OR, 0.46 [95% CI, 0.23 to 0.90], P = .02) were less likely to receive FA compared with White and English-speaking patients, respectively. CONCLUSION: FA programs can mitigate high OOP spending but not for patients who spend at catastrophic levels. There are racial/ethnic and language disparities in access to such programs. Future studies should evaluate access to FA programs across diverse delivery settings.

Projected impact of population aging on non-communicable disease burden and costs in the Kingdom of Saudi Arabia, 2020-2030.

BACKGROUND: The number of people aged greater than 65 years per 100 people aged 20-64 years is expected to almost double in The Kingdom of Saudi Arabia (KSA) between 2020 and 2030. We therefore aimed to quantify the growing non-communicable disease (NCD) burden in KSA between 2020 and 2030, and the impact this will have on the national health budget. METHODS: Ten priority NCDs were selected: ischemic heart disease, stroke, type 2 diabetes, chronic obstructive pulmonary disease, chronic kidney disease, dementia, depression, osteoarthritis, colorectal cancer, and breast cancer. Age- and sex-specific prevalence was projected for each priority NCD between 2020 and 2030. Treatment coverage rates were applied to the projected prevalence estimates to calculate the number of patients incurring treatment costs for each condition. For each priority NCD, the average cost-of-illness was estimated based on published literature. The impact of changes to our base-case model in terms of assumed disease prevalence, treatment coverage, and costs of care, coming into effect from 2023 onwards, were explored. RESULTS: The prevalence estimates for colorectal cancer and stroke were estimated to almost double between 2020 and 2030 (97% and 88% increase, respectively). The only priority NCD prevalence projected to increase by less than 60% between 2020 and 2030 was for depression (22% increase). It is estimated that the total cost of managing priority NCDs in KSA will increase from USD 19.8 billion in 2020 to USD 32.4 billion in 2030 (an increase of USD 12.6 billion or 63%). The largest USD value increases were projected for osteoarthritis (USD 4.3 billion), diabetes (USD 2.4 billion), and dementia (USD 1.9 billion). In scenario analyses, our 2030 projection for the total cost of managing priority NCDs varied between USD 29.2 billion - USD 35.7 billion. CONCLUSIONS: Managing the growing NCD burden in KSA's aging population will require substantial healthcare spending increases over the coming years.

Implementation of a Multisite Financial Reimbursement Program in Cancer Clinical Trials Integrated With Patient Navigation: A Pilot Randomized Clinical Trial.

PURPOSE: Cancer clinical trial participants face considerable indirect costs associated with participation, such as travel and lodging, which may contribute to poor enrollment. Here, we report the findings in IMproving Patient Access to Cancer clinical Trials, a pilot feasibility study investigating the efficacy of offering a financial reimbursement program (FRP) during a therapeutic clinical trial discussion with or without additional outreach in improving patient enrollment. METHODS: Study participants for this study were recruited at two National Cancer Institute-designated comprehensive cancer centers (CCCs) from April 8, 2019, to September 19, 2019. Eligible participants were adults with a cancer diagnosis being approached to consider enrollment in a clinical trial. Participants were randomly assigned 1:1 to receive no follow-up (usual care) or a follow-up telephone call to facilitate FRP utilization stratified by study site. The target enrollment was 132 patients, with 66 patients in each study arm. The primary outcome was the consent rate to the multisite interventional study on the FRP among participants enrolling in clinical trials. RESULTS: The study had a 78% consent rate and enrolled a total of 132 participants, of whom 51% were non-White compared with 28% of CCC treatment clinical trial participants in 2019. No difference in enrollment in clinical trials between the two study arms was observed as the proportion of enrollment was 70% for both study arms. The most common reason for not enrolling in a clinical trial was due to ineligibility determined through screening procedures (75%). CONCLUSION: The current study observed that implementation of FRP at CCCs is feasible and serves a diverse patient population. Future studies will measure the impact of programs on overall clinical trial accrual and among racial/ethnic minorities.

Accelerating cancer clinical trial recruitment through a financial reimbursement program integrated with patient navigation: an interrupted time series analysis.

BACKGROUND: Cancer treatment clinical trials face major challenges with patient recruitment. Strategies to address patient indirect costs associated with clinical trial participation may accelerate accrual overall. The current study examined the effect of the IMproving Patient Access to Clinical Trials (IMPACT) intervention on patient accrual to cancer treatment clinical trials at an academic medical center. The IMPACT intervention was an onsite patient navigator combined with a financial reimbursement program to address patient out of pocket costs and began on September 2018. METHODS: This analysis measured frequency of patient enrollment in cancer treatment clinical trials and available cancer treatment clinical trials per month between January 1, 2016 and March 31, 2020. An interrupted time-series analysis (ITSA) was conducted to estimate changes in patient enrollment attributable to the IMPACT intervention. RESULTS: During the study period, a mean of 69 patients enrolled in clinical trials per month (standard deviation (SD = 13), with 27 (SD = 7) in early phase vs 41 (SD = 12) in late phase clinical trials. The number of available clinical trials per month was 51 (SD = 2) overall, with 23 (SD = 1) in early phase vs 28 (SD = 1) in late phase context. A total of 3470 patients were enrolled in cancer treatment clinical trials during the evaluated time period, the majority of whom were men (1895, 55 %) and racially white (2267, 65 %). A statistically significant increase in the number of patients accrued as compared to the pre-intervention trend was observed; with approximately 1 additional patient accrued per month, with a larger effect on increase patient accrual for late phase clinical trials. DISCUSSION: This study observed that the IMPACT intervention accelerated clinical trial recruitment, especially among late phase clinical trials. Future research will examine strategies to leverage this infrastructure to optimize recruitment among underrepresented patients. POLICY SUMMARY: To improve clinical trial recruitment and ensure that trial results are representative of a diverse population it is critical for health policies consider patient out-of-pocket costs and potential reimbursement to alleviate financial burden associated with clinical trial participation. Furthermore, policies for facilitating clinical trial recruitment and participant retention should budget for and incorporate a navigation component to assist patients who may not be familiar with the healthcare system and available financial assistance.

Evaluating determinants of receipt of molecular imaging in biochemical recurrent prostate cancer.

BACKGROUND: Molecular imaging with novel radiotracers is changing the treatment landscape in prostate cancer (PCa). Currently, standard of care includes either conventional and molecular imaging at time of biochemical recurrence (BCR). This study evaluated the determinants of and cost associated with utilization of molecular imaging for BCR PCa. METHODS: This is a retrospective observational cohort study among men with BCR PCa from June 2018 to May 2019. Multivariate logistic regression models were employed to analyze the primary outcome: receipt of molecular imaging (e.g. Fluciclovine PET and Prostate Specific Membrane Antigen PET) as part of diagnostic work-up for BCR PCa. Multivariate linear regression models were used to analyze the secondary outcome: overall healthcare cost within a 1-year time frame. RESULTS: The study sample included 234 patients; 79.1% White, 2.1% Black, 8.5% Asian/Pacific Islander, and 10.3% Other. The majority were 55 years or older (97.9%) and publicly insured (74.8%). Analysis indicated a one-unit reduction in PSA is associated with 1.3 times higher likelihood of receiving molecular imaging (p < 0.01). Analysis found that privately insured patients were associated with approximately $500,000 more in hospital reimbursement (p < 0.01) as compared to the publicly insured. Additionally, a one-unit increase in PSA is associated with $6254 increase in hospital reimbursement or an increase in total payments by 2.1% (p < 0.05). CONCLUSIONS: Higher PSA was associated with lower likelihood for molecular imaging and higher cost in a one-year time frame. Higher cost was also associated with private insurance, but there was no clear relationship between insurance type and imaging type.

Liposomal Bupivacaine Decreases Postoperative Length of Stay and Opioid Use in Patients Undergoing Radical Cystectomy.

OBJECTIVE: To analyze differences in length of stay, opioid use, and other perioperative outcomes in patients undergoing radical cystectomy with urinary diversion who received either liposomal bupivacaine (LB) or epidural analgesia. METHODS: This was a single center, retrospective cohort study of patients undergoing open radical cystectomy with urinary diversion from 2015-2019 in the early recovery after surgery (ERAS) pathway. Patients received either LB or epidural catheter analgesia for post-operative pain control. LB was injected at the time of fascial closure to provide up to 72 hours of local analgesia. The primary outcome was post-operative length of stay. Secondary outcomes were post-operative opioid use, time to solid food, time to ambulation, and direct hospitalization costs. Multivariable Cox proportional hazards regression was used to determine associations between analgesia type and discharge. RESULTS: LB use was independently associated with shorter post-operative length of stay compared to epidural use (median (IQR) 4.9 days (3.9-5.8) vs 5.9 days (4.9-7.9), P<.001), less total opioid use (mean 188.3 vs 612.2 OME, P <.001), earlier diet advancement (mean 1.6 vs 2.4 days, P <.001), and decreased overall direct costs ($23,188 vs $29,628, P <.001). 45% of patients who received LB were opioid-free after surgery, none in the epidural group. On multivariable Cox proportional hazards regression modeling, LB use was independently associated with earlier discharge (HR 2.1, IQR 1.0-4.5). CONCLUSION: Use of LB in open radical cystectomy is associated with reduced LOS, less opioid exposure, and earlier diet advancement.

Disparities in PET Imaging for Prostate Cancer at a Tertiary Academic Medical Center.

The purpose of this study was to evaluate differences between patients receiving 18F-fluciclovine and 68Ga-prostate-specific membrane antigen (68Ga-PSMA-11) for biochemically recurrent prostate cancer at a tertiary medical center. Methods: All 18F-fluciclovine and 68Ga-PSMA-11 PET studies performed at the University of California San Francisco from October 2015 to January 2020 were reviewed. Age, race/ethnicity, primary language, body mass index, insurance type, and home address were obtained through the electronic medical record. A logistic regression model was used to evaluate the predictor variables. Results: In total, 1,502 patients received 68Ga-PSMA-11 and 254 patients received 18F-fluciclovine. Black patients had increased odds of receiving imaging with 18F-fluciclovine versus 68Ga-PSMA-11 compared with non-Hispanic White patients (odds ratio, 3.88; 95% CI, 1.90-7.91). There were no other statistically significant differences. Conclusion: In patients receiving molecular imaging for prostate cancer at a single U.S. tertiary medical center, access to 68Ga-PSMA-11 for Black patients was limited, compared with non-Hispanic White patients, by a factor of nearly 4.

Development of a theory-driven implementation strategy for cancer management guidelines in sub-Saharan Africa.

BACKGROUND: Despite recent international efforts to develop resource-stratified clinical practice guidelines for cancer, there has been little research to evaluate the best strategies for dissemination and implementation in low- and middle-income countries (LMICs). Guideline publication alone is insufficient. Extensive research has shown that structured, multifaceted implementation strategies that target barriers to guideline use are most likely to improve adherence; however, most of this research has been conducted in high-income countries. There is a pressing need to develop and evaluate guideline implementation strategies for cancer management in LMICs in order to address stark disparities in cancer outcomes. METHODS: In preparation for the launch of Tanzania's first National Cancer Treatment Guidelines, we developed a theory-driven implementation strategy for guideline-based practice at Ocean Road Cancer Institute (ORCI). Here, we use the Intervention Mapping framework to provide a detailed stepwise description of our process. First, we conducted a needs assessment to identify barriers and facilitators to guideline-based practice at ORCI. Second, we defined both proximal and performance objectives for our implementation strategy. Third, we used the Capability, Opportunity, Motivation and Behavior/Behavior Change Wheel (COM-B/BCW) framework to categorize the barriers and facilitators, choose behavior change techniques most likely to overcome targeted barriers and leverage facilitators, and select a feasible mode of delivery for each technique. Fourth, we organized these modes of delivery into a phased implementation strategy. Fifth, we operationalized each component of the strategy. Sixth, we identified the indicators of the process, outcome, and impact of our intervention and developed an evaluation plan to measure them using a mixed methods approach. DISCUSSION: We developed a robust, multifaceted guideline implementation strategy derived from a prominent behavior change theory for use in Tanzania. The barriers and strategies we generated are consistent with those well established in the literature, enhancing the validity and generalizability of our process and results. Through our rigorous evaluation plan and systematic account of modifications and adaptations, we will characterize the transferability of "proven" guideline implementation strategies to LMICs. We hope that by describing our process in detail, others may endeavor to replicate it, meeting a widespread need for dedicated efforts to implement cancer guidelines in LMICs.

Evaluation of the ASCO Value Framework for Anticancer Drugs at an Academic Medical Center.

BACKGROUND: Anticancer drug prices have increased by an average of 12% each year from 1996 to 2014. A major concern is that the increasing cost and responsibility of evaluating treatment options are being shifted to patients. This research compared 2 value-based pricing models that were being considered for use at the University of California, San Francisco (UCSF) Medical Center to address the growing burden of high-cost cancer drugs while improving patient-centered care. PROGRAM DESCRIPTION: The Medication Outcomes Center (MOC) in the Department of Clinical Pharmacy, University of California, San Francisco (UCSF), School of Pharmacy focuses on assessing the value of medication-related health care interventions and disseminating findings to the UCSF Medical Center. The High Cost Oncology Drug Initiative at the MOC aims to assess and adopt tools for the critical assessment and amelioration of high-cost cancer drugs. The American Society of Clinical Oncology (ASCO) Value Framework (2016 update) and a cost-effectiveness analysis (CEA) framework were identified as potential tools for adoption. To assess 1 prominent value framework, the study investigators (a) asked 8 clinicians to complete the ASCO Value Framework for 11 anticancer medications selected by the MOC; (b) reviewed CEAs assessing the drugs; (c) generated descriptive statistics; and (d) analyzed inter-rater reliability, convergence validity, and ranking consistency. OBSERVATIONS: On the scale of -20 to 180, the mean ASCO net health benefit (NHB) total score across 11 drugs ranged from 7.6 (SD = 7.8) to 53 (SD = 9.8). The Kappa coefficient (κ) for NHB scores across raters was 0.11, which is categorized as "slightly reliable." The combined κ score was 0.22, which is interpreted as low to fair inter-rater reliability. Convergent validity indicates that the correlation between NHB scores and CEA-based incremental cost-effectiveness ratios (ICERs) was low (-0.215). Ranking of ICERs, ASCO scores, and wholesale acquisition costs indicated different results between frameworks. IMPLICATIONS: The ASCO Value Framework requires further specificity before use in a clinical setting, since it currently results in low inter-rater reliability and validity. Furthermore, ASCO scores were unable to discriminate between drugs providing the most and least value. RECOMMENDATIONS: The evaluation provides specific areas of weakness that can be addressed in future updates of the ASCO framework to improve usability. Meanwhile, the UCSF Medical Center should rely on CEAs, which are highly accessible for the highlighted cancer drugs. The MOC role can include summarizing and disseminating available CEA studies for interpretation by clinicians and financial counselors around drug value. DISCLOSURES: Funding for this research was contributed by the University of California, San Francisco, Medical Center Campus Strategic Initiative Program. The authors have no conflicts of interest to disclose. Study concept and design were contributed primarily by Wilson, along with Wang and Patel. Kim, Dacey, and Yuen collected the data, and data interpretation was performed by Wilson and Lin. The manuscript was written by Wilson, Lin, Wang, and Tran and revised by Lin, Redondi, Brodowy, and Kroon.

Use of Conjoint Analysis to Determine Patient Preferences for Surgical Treatment of Urethral Stricture Disease.

INTRODUCTION: Understanding patient preferences for characteristics of treatments facilitates patient participation and doctor-patient communication and enhances patient-centered care. Patient participation is especially important for urethral stricture disease, which has no definitive treatment guidelines favoring either endoscopic incision or open reconstruction, making patient preference an important factor in treatment choice. However, to date, there have been no studies assessing factors that patients value when choosing a treatment option. METHODS: We employ choice-based conjoint analysis to assess patient preferences in the trade-offs of treatment attributes for urethral stricture disease. Male patients undergoing treatment or follow-up examination for urethral stricture disease were recruited through a University Medical Practice. We included 169 patients in the analysis. Six attributes of both risk and benefit were examined: treatment type, success rate, number of future procedures, post-treatment catheter duration, recovery time, and copayment amount. RESULTS: The treatment success rate was by far the most important attribute. Relative to a 25% success rate (OR = 1) an 85% success rate (OR = 26.72, p<.01) increased patient preference by approximately 27 times. Furthermore, patients are willing to pay a $10,000 copayment to double the success rate from 25% to 50%. Patients demonstrated a strong aversion to time with a urinary catheter. Catheter duration for 1 week or less (OR = .67, p<.01) reduced patient preference by about 1.5 times when compared to requiring no catheter. We also found that patients place low importance on both how invasive the treatment seems and low copayment amounts but are willing to pay $10,000 copayment for an open reconstruction surgery compared with an endoscopic incision procedure. CONCLUSION: The findings highlight the importance of shared and detailed physician/patient discussions of all the risk and benefits of each treatment choice and suggest that conjoint analysis may be helpful as a decision aid to guide discussions with individual patients deciding on a treatment.

Patient-centered Treatment Decisions for Urethral Stricture: Conjoint Analysis Improves Surgical Decision-making.

OBJECTIVE: To determine whether the use of a choice-based conjoint analysis (CA) exercise decreased patients' decisional conflict about treatment preferences for surgical management of urethral stricture disease. Understanding patient preferences for treatment decisions assists in shared decision-making and emphasizes patient-centered outcomes. CA offers a method to understand what risks patients are willing to take for what gains. METHODS: The CA methodology was used by providing participants with case-based choices to elucidate the relative importance that individuals place on various treatment aspects. Patients' decisional conflict regarding surgery for urethral stricture was assessed before and after the CA exercise to assess the impact the exercise had on their decisional conflict. RESULTS: Completion of the CA exercise resulted in a significant decrease in decisional conflict (P < .001). The majority (59.5%) of participants with decisional conflict before the CA exercise experienced a decrease in decisional conflict afterwards, with only a minority (16.5%) experiencing new decisional conflict after the exercise. Participants felt the choice-based CA exercise was helpful in deciding what was important in making treatment decisions (70%) and in expressing their priorities and treatment preferences (82%). The number needed to counsel to achieve a decrease in decisional conflict was 1.69 and to achieve no decisional conflict was 3.65. CONCLUSION: Choice-based CA improves patients' ability to express their treatment preferences and decreases decisional conflict. CA may be a new tool that physicians and patients can use to aid in shared decision-making with a focus on patient-centered outcomes.

Type of bone graft or substitute does not affect outcome of spine fusion with instrumentation for adolescent idiopathic scoliosis.

STUDY DESIGN: Retrospective cohort analysis. OBJECTIVE: To compare clinical outcomes after spine instrumentation and fusion using 3 different bone grafts in children with adolescent idiopathic scoliosis (AIS). SUMMARY OF BACKGROUND DATA: Autogenous iliac crest bone graft (AIC) is the "gold standard" to promote fusion in posterior AIS operations, although the morbidity of harvest is a concern. There is limited data comparing outcomes after AIS surgery based on types of bone grafts. METHODS: Children (10-18 yr) with AIS who underwent deformity correction via a posterior approach were identified in the Spinal Deformity Study Group database. All had a minimum of 2-year follow-up. Patients were subdivided into 3 groups based on bone graft used: AIC, allograft, and bone substitute (BS). Clinical data included patient demographics, operative details, postoperative analgesic use, and perioperative complications. Lenke curve type and curve magnitude changes were radiographically analyzed. The Scoliosis Research Society-30 questionnaire was used to assess clinical outcomes. RESULTS: 461 patients met inclusion criteria (girls: 381, boys: 80; average age 14.7 ± 1.7) and consisted of 152 AIC patients (124 girls, 28 boys), 199 allograft patients (167 girls, 32 boys), and 110 BS patients (90 girls, 20 boys). There was no difference in age (P = 0.41) or gender (P = 0.82). The BS group had significantly smaller preoperative curves and shorter operative times. Postoperatively, patients who received BS had significantly longer hospital stays, used higher quantities of patient-controlled intravenous analgesia and used epidurals longer. The AIC group used patient-controlled intravenous analgesia significantly longer. There were no differences between the groups in regards to curve type, number of levels fused, postoperative infections, pseudarthrosis, reoperations for any indication, and Scoliosis Research Society-30 scores at the latest follow-up. CONCLUSION: Outcomes after primary posterior spinal fusion with instrumentation are not influenced by type of bone graft or substitute. LEVEL OF EVIDENCE: 3.