Cost-Effectiveness of Lovotibeglogene Autotemcel (Lovo-Cel) Gene Therapy for Patients with Sickle Cell Disease and Recurrent Vaso-Occlusive Events in the United States.

BACKGROUND AND OBJECTIVE: Gene therapies for sickle cell disease (SCD) may offer meaningful benefits for patients and society. This study evaluated the cost-effectiveness of lovotibeglogene autotemcel (lovo-cel), a one-time gene therapy administered via autologous hematopoietic stem cell transplantation, compared with common care for patients in the United States (US) with SCD aged ≥ 12 years with ≥ 4 vaso-occlusive events (VOEs) in the past 24 months. METHODS: We developed a patient-level simulation model accounting for lovo-cel and SCD-related events, complications, and mortality over a lifetime time horizon. The pivotal phase 1/2 HGB-206 clinical trial (NCT02140554) served as the basis for lovo-cel efficacy and safety. Cost, quality-of-life, and other clinical data were sourced from HGB-206 data and the literature. Analyses were conducted from US societal and third-party payer perspectives. Uncertainty was assessed through probabilistic sensitivity analysis and extensive scenario analyses. RESULTS: Patients treated with lovo-cel were predicted to survive 23.84 years longer on average (standard deviation [SD], 12.80) versus common care (life expectancy, 62.24 versus 38.40 years), with associated discounted patient quality-adjusted life-year (QALY) gains of 10.20 (SD, 4.10) and direct costs avoided of $1,329,201 (SD, $1,346,446) per patient. Predicted societal benefits included discounted caregiver QALY losses avoided of 1.19 (SD, 1.38) and indirect costs avoided of $540,416 (SD, $262,353) per patient. Including lovo-cel costs ($3,282,009 [SD, $29,690] per patient) resulted in incremental cost-effectiveness ratios of $191,519 and $124,051 per QALY gained from third-party payer and societal perspectives, respectively. In scenario analyses, the predicted cost-effectiveness of lovo-cel also was sensitive to baseline age and VOE frequency and to the proportion of patients achieving and maintaining complete resolution of VOEs. CONCLUSIONS: Our analysis of lovo-cel gene therapy compared with common care for patients in the US with SCD with recurrent VOEs estimated meaningful improvements in survival, quality of life, and other clinical outcomes accompanied by increased overall costs for the health care system and for broader society. The predicted economic value of lovo-cel gene therapy was influenced by uncertainty in long-term clinical effects and by positive spillover effects on patient productivity and caregiver burden.

Opioid Use Among Children and Adults With Sickle Cell Disease in North Carolina Medicaid Enrollees in the Era of Opioid Harm Reduction.

Adults and children with sickle cell disease (SCD) are predominantly African American, with pain-related health disparities. We examined opioid prescription fill patterns in adults and children with SCD and compared factors associated with fills in North Carolina Medicaid enrollees. Our retrospective cohort study included 955 enrollees diagnosed with SCD having at least one opioid fill. Associations were measured between two cohorts (12 and 24 mo of continuous enrollment) for the following characteristics: sex, age, enrollee residence, hydroxyurea adherence, comanagement, enrollment in Community Care North Carolina, prescription for short versus short and long-acting opioids, and emergency department reliance. The majority of individuals did not have an opioid claim over a 12 or 24-month period. Claims increased at ages 10 to 17, peaking at ages 18 to 30. The increased number of claims was associated with the following factors: increasing age, male, short versus long-acting opioids, and Medicaid enrollment for 24 versus 12 months. Community Care North Carolina enrollees in the 12-month cohort had higher opioid days of supply per month; the inverse was true of the 24-month cohort.

Feasibility of Implementation of a Mobile Digital Personal Health Record to Coordinate Care for Children and Youth With Special Health Care Needs in Primary Care: Protocol for a Mixed Methods Study.

BACKGROUND: Electronic health record (EHR)-integrated digital personal health records (PHRs) via Fast Healthcare Interoperability Resources (FHIR) are promising digital health tools to support care coordination (CC) for children and youth with special health care needs but remain widely unadopted; as their adoption grows, mixed methods and implementation research could guide real-world implementation and evaluation. OBJECTIVE: This study (1) evaluates the feasibility of an FHIR-enabled digital PHR app for CC for children and youth with special health care needs, (2) characterizes determinants of implementation, and (3) explores associations between adoption and patient- or family-reported outcomes. METHODS: This nonrandomized, single-arm, prospective feasibility trial will test an FHIR-enabled digital PHR app's use among families of children and youth with special health care needs in primary care settings. Key app features are FHIR-enabled access to structured data from the child's medical record, families' abilities to longitudinally track patient- or family-centered care goals, and sharing progress toward care goals with the child's primary care provider via a clinician dashboard. We shall enroll 40 parents or caregivers of children and youth with special health care needs to use the app for 6 months. Inclusion criteria for children and youth with special health care needs are age 0-16 years; primary care at a participating site; complex needs benefiting from CC; high hospitalization risk in the next 6 months; English speaking; having requisite technology at home (internet access, Apple iOS mobile device); and an active web-based EHR patient portal account to which a parent or caregiver has full proxy access. Digital prescriptions will be used to disseminate study recruitment materials directly to eligible participants via their existing EHR patient portal accounts. We will apply an intervention mixed methods design to link quantitative and qualitative (semistructured interviews and family engagement panels with parents of children and youth with special health care needs) data and characterize implementation determinants. Two CC frameworks (Pediatric Care Coordination Framework; Patient-Centered Medical Home) and 2 evaluation frameworks (Consolidated Framework for Implementation Research; Technology Acceptance Model) provide theoretical foundations for this study. RESULTS: Participant recruitment began in fall 2022, before which we identified >300 potentially eligible patients in EHR data. A family engagement panel in fall 2021 generated formative feedback from family partners. Integrated analysis of pretrial quantitative and qualitative data informed family-centered enhancements to study procedures. CONCLUSIONS: Our findings will inform how to integrate an FHIR-enabled digital PHR app for children and youth with special health care needs into clinical care. Mixed methods and implementation research will help strengthen implementation in diverse clinical settings. The study is positioned to advance knowledge of how to use digital health innovations for improving care and outcomes for children and youth with special health care needs and their families. TRIAL REGISTRATION: ClinicalTrials.gov NCT05513235; https://clinicaltrials.gov/study/NCT05513235. INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID): DERR1-10.2196/46847.

Outcomes of End-User Testing of a Care Coordination Mobile App With Families of Children With Special Health Care Needs: Simulation Study.

BACKGROUND: Care for children with special health care needs relies on a network of providers who work to address the medical, behavioral, developmental, educational, social, and economic needs of the child and their family. Family-directed, manually created visual depictions of care team composition (ie, care mapping) and detailed note-taking curated by caregivers (eg, care binders) have been shown to enhance care coordination for families of these children, but they are difficult to implement in clinical settings owing to a lack of integration with electronic health records and limited visibility of family-generated insights for care providers. Caremap is an electronic health record-integrated digital personal health record mobile app designed to integrate the benefits of care mapping and care binders. Currently, there is sparse literature describing end-user participation in the co-design of digital health tools. In this paper, we describe a project that evaluated the usability and proof of concept of the Caremap app through end-user simulation. OBJECTIVE: This study aimed to conduct proof-of-concept testing of the Caremap app to coordinate care for children with special health care needs and explore early end-user engagement in simulation testing. The specific aims included engaging end users in app co-design via app simulation, evaluating the usability of the app using validated measures, and exploring user perspectives on how to make further improvements to the app. METHODS: Caregivers of children with special health care needs were recruited to participate in a simulation exercise using Caremap to coordinate care for a simulated case of a child with complex medical and behavioral needs. Participants completed a postsimulation questionnaire adapted from 2 validated surveys: the Pediatric Integrated Care Survey (PICS) and the user version of the Mobile Application Rating Scale (uMARS). A key informant interview was also conducted with a liaison to Spanish-speaking families regarding app accessibility for non-English-speaking users. RESULTS: A Caremap simulation was successfully developed in partnership with families of children with special health care needs. Overall, 38 families recruited from 19 different US states participated in the simulation exercise and completed the survey. The average rating for the survey adapted from the PICS was 4.1 (SD 0.82) out of 5, and the average rating for the adapted uMARS survey was 4 (SD 0.83) out of 5. The highest-rated app feature was the ability to track progress toward short-term, patient- and family-defined care goals. CONCLUSIONS: Internet-based simulation successfully facilitated end-user engagement and feedback for a digital health care coordination app for families of children with special health care needs. The families who completed simulation with Caremap rated it highly across several domains related to care coordination. The simulation study results elucidated key areas for improvement that translated into actionable next steps in app development.

Improving Pain Assessment Using Vital Signs and Pain Medication for Patients With Sickle Cell Disease: Retrospective Study.

BACKGROUND: Sickle cell disease (SCD) is the most common inherited blood disorder affecting millions of people worldwide. Most patients with SCD experience repeated, unpredictable episodes of severe pain. These pain episodes are the leading cause of emergency department visits among patients with SCD and may last for several weeks. Arguably, the most challenging aspect of treating pain episodes in SCD is assessing and interpreting a patient's pain intensity level. OBJECTIVE: This study aims to learn deep feature representations of subjective pain trajectories using objective physiological signals collected from electronic health records. METHODS: This study used electronic health record data collected from 496 Duke University Medical Center participants over 5 consecutive years. Each record contained measures for 6 vital signs and the patient's self-reported pain score, with an ordinal range from 0 (no pain) to 10 (severe and unbearable pain). We also extracted 3 features related to medication: medication type, medication status (given or applied, or missed or removed or due), and total medication dosage (mg/mL). We used variational autoencoders for representation learning and designed machine learning classification algorithms to build pain prediction models. We evaluated our results using an accuracy and confusion matrix and visualized the qualitative data representations. RESULTS: We designed a classification model using raw data and deep representational learning to predict subjective pain scores with average accuracies of 82.8%, 70.6%, 49.3%, and 47.4% for 2-point, 4-point, 6-point, and 11-point pain ratings, respectively. We observed that random forest classification models trained on deep represented features outperformed models trained on unrepresented data for all pain rating scales. We observed that at varying Likert scales, our models performed better when provided with medication data along with vital signs data. We visualized the data representations to understand the underlying latent representations, indicating neighboring representations for similar pain scores with a higher resolution of pain ratings. CONCLUSIONS: Our results demonstrate that medication information (the type of medication, total medication dosage, and whether the medication was given or missed) can significantly improve subjective pain prediction modeling compared with modeling with only vital signs. This study shows promise in data-driven estimated pain scores that will help clinicians with additional information about the patient's condition, in addition to the patient's self-reported pain scores.

Perspectives of individuals with sickle cell disease on barriers to care.

INTRODUCTION: Sickle cell disease (SCD) is an inherited hemoglobinopathy that predominantly affects African Americans in the United States. The disease is associated with complications leading to high healthcare utilization rates, including emergency department (ED) visits and hospitalizations. Optimal SCD care requires a multidisciplinary approach involving SCD specialists to ensure preventive care, minimize complications and prevent unnecessary ED visits and hospitalizations. However, most individuals with SCD receive sub-optimal care or are unaffiliated with care (have not seen an SCD specialist). We aimed to identify barriers to care from the perspective of individuals with SCD in a multi-state sample. METHODS: We performed a multiple methods study consisting of surveys and interviews in three comprehensive SCD centers from March to June 2018. Interviews were transcribed and coded, exploring themes around barriers to care. Survey questions on the specific themes identified in the interviews were analyzed using summary statistics. RESULTS: We administered surveys to 208 individuals and conducted 44 in-depth interviews. Barriers to care were identified and classified according to ecological level (i.e., individual, family/interpersonal, provider, and socio-environmental/organizational level). Individual-level barriers included lack of knowledge in self-management and disease severity. Family/interpersonal level barriers were inadequate caregiver support and competing life demands. Provider level barriers were limited provider knowledge, provider inexperience, poor provider-patient relationship, being treated differently, and the provider's lack of appreciation of the patient's SCD knowledge. Socio-environmental/organizational level barriers included limited transportation, lack of insurance, administrative barriers, poor care coordination, and reduced access to care due to limited clinic availability, services provided or clinic refusal to provide SCD care. CONCLUSION: Participants reported several multilevel barriers to SCD care. Strategies tailored towards reducing these barriers are warranted. Our findings may also inform interventions aiming to locate and link unaffiliated individuals to care.

A Needs Assessment of Persons With Sickle Cell Disease in a Major Medical Center in North Carolina.

BACKGROUND Sickle cell disease (SCD) is a complex disease associated with many complications and a shortened lifespan. In 2016, the National Heart, Lung and Blood Institute funded 8 centers in the United States to form the Sickle Cell Disease Implementation Consortium, with the goal of improving SCD care. The aim of our study was to describe SCD self-efficacy, pain interference, and barriers to care from the perspective of persons with SCD in the North Carolina center.METHODS Persons with SCD, aged 15 and older were recruited from a large SCD center in North Carolina. Surveys, focus groups, and interviews were completed.RESULTS Fifty-one people completed a survey, and 14 people completed an interview or focus group. Barriers identified in the survey included self-care barriers, misconceptions related to hydroxyurea (an oral medication that reduces rates of pain crisis), limited provider knowledge, and stigma. Concerning self-efficacy, participants reported that they were able to manage their pain symptoms most of the time. Pain interfered most with the ability to participate in social and day-to-day activities.Common themes from the focus groups and interviews included misconceptions about hydroxyurea, pain, provider knowledge, stigma, co-management, transportation, and insurance. Recommendations to improve care included the use of case managers, utilization of treatment guidelines, individualized pain protocols, and effective co-management by providers.LIMITATIONS Participants were recruited from 1 SCD center and may not be representative of the entire SCD population in North Carolina.CONCLUSIONS Participants described many perceived barriers to care, and their responses suggest a need for improvements in patient hydroxyurea education, provider knowledge, and care coordination.

Pain Intensity Assessment in Sickle Cell Disease Patients Using Vital Signs During Hospital Visits.

Pain in sickle cell disease (SCD) is often associated with increased morbidity, mortality, and high healthcare costs. The standard method for predicting the absence, presence, and intensity of pain has long been self-report. However, medical providers struggle to manage patients based on subjective pain reports correctly and pain medications often lead to further difficulties in patient communication as they may cause sedation and sleepiness. Recent studies have shown that objective physiological measures can predict subjective self-reported pain scores for inpatient visits using machine learning (ML) techniques. In this study, we evaluate the generalizability of ML techniques to data collected from 50 patients over an extended period across three types of hospital visits (i.e., inpatient, outpatient and outpatient evaluation). We compare five classification algorithms for various pain intensity levels at both intra-individual (within each patient) and inter-individual (between patients) level. While all the tested classifiers perform much better than chance, a Decision Tree (DT) model performs best at predicting pain on an 11-point severity scale (from 0-10) with an accuracy of 0.728 at an inter-individual level and 0.653 at an intra-individual level. The accuracy of DT significantly improves to 0.941 on a 2-point rating scale (i.e., no/mild pain: 0-5, severe pain: 6-10) at an inter-individual level. Our experimental results demonstrate that ML techniques can provide an objective and quantitative evaluation of pain intensity levels for all three types of hospital visits.

Dissemination of Evidence-Based Recommendations for Sickle Cell Disease to Primary Care and Emergency Department Providers in North Carolina: A Cost Benefit Analysis.

Background: Sickle cell disease (SCD) is a genetic condition affecting primarily individuals of African descent, who happen to be disproportionately impacted by poverty and who lack access to health care. Individuals with SCD are at high likelihood of high acute care utilization and chronic pain episodes. The multiple complications seen in SCD contribute to significant morbidity and premature mortality, as well as substantial costs to the healthcare system. Objectives: SCD is a complex chronic disease resulting in the need for primary, specialty and emergency care. Many providers do not feel prepared to care for individuals with SCD, despite the existence of evidence-based guidelines. We report the development of a SCD toolbox and the dissemination process to primary care and emergency department (ED) providers in North Carolina (NC). We report the effect of this dissemination on health-care utilization, cost of care, and overall cost-benefit. Methods: The SCD toolbox was adapted from the National Heart, Lung, and Blood Institute recommendations. Toolbox training was provided to quality improvement specialists who then disseminated the toolbox to primary care providers (PCPs) affiliated with the only NC managed care coordination system and ED providers. Tools were made available in paper, online, and in app formats to participating managed care network practices (n=1 800). Medicaid claims data were analyzed for total costs and benefits of the toolbox dissemination for a 24-month pre- and 18-month post-intervention period. Results: There was no statistically significant shift in the number of outpatient specialty visits, ED visits or hospitalizations. There was a small decrease in the number of PCP visits in the post-implementation period. The dissemination resulted in a net cost-savings of $361 414 ($14.03 per-enrollee per-month on average). However, the estimated financial benefit associated with the dissemination of the SCD toolbox was not statistically significant. Conclusions: Although we did not find the expected shift to increased PCP visits and decreased ED visits and hospitalizations, there were many lessons learned.

Vaso-occlusive crises and costs of sickle cell disease in patients with commercial, Medicaid, and Medicare insurance - the perspective of private and public payers.

AIM: To characterize vaso-occlusive crises (VOCs) and describe healthcare costs among commercially-insured, Medicaid-insured, and Medicare-insured patients with sickle cell disease (SCD). MATERIALS AND METHODS: The IBM Truven Health MarketScan Commercial (2000-2018), Medicaid Analytic eXtract (2008-2014), and Medicare Research Identifiable Files (2012-2016) databases were used to identify patients with ≥2 SCD diagnoses. Study measures were evaluated during a 12-month follow-up period, stratified by annual number of VOCs (i.e. 0, 1, and ≥2). RESULTS: Among 16,092 commercially-insured patients (mean age = 36.7 years), 35.3% had 1+ VOCs. Mean annual total all-cause healthcare costs were $15,747, $27,194, and $64,555 for patients with 0, 1, and 2+ VOCs, respectively. Total all-cause healthcare costs were mainly driven by inpatient (0 VOC = 31.0%, 1 VOC = 53.1%, 2+ VOCs = 65.4%) and SCD-related costs (0 VOC = 56.4%, 1 VOC = 78.4%, 2+ VOCs = 93.9%). Among 18,287 Medicaid-insured patients (mean age = 28.5 years, fee-for-service = 50.2%), 63.9% had 1+ VOCs. Mean annual total all-cause healthcare costs were $16,750, $29,880, and $64,566 for patients with 0, 1, and 2+ VOCs, respectively. Inpatient costs (0 VOC = 37.2%, 1 VOC = 64.3%, 2+ VOCs = 72.9%) and SCD-related costs (0 VOC = 60.9%, 1 VOC = 73.8%, 2+ VOCs = 92.2%) accounted for a significant proportion of total all-cause healthcare costs. Among 15,431 Medicare-insured patients (mean age = 48.2 years), 55.1% had 1+ VOCs. Mean annual total all-cause healthcare costs were $21,877, $29,250, and $58,308 for patients with 0, 1, and ≥2 VOCs, respectively. Total all-cause healthcare costs were mainly driven by inpatient (0 VOC = 47.9%, 1 VOC = 54.9%, 2+ VOCs = 67.5%) and SCD-related costs (0 VOC = 74.9%, 1 VOC = 84.4%, 2+ VOCs = 95.3%). LIMITATIONS: VOCs managed at home were not captured. Analyses were descriptive in an observational setting; thus, no causal relationships can be inferred. CONCLUSIONS: A high proportion of patients experienced VOCs across payers. Furthermore, inpatient and SCD-related costs accounted for a significant proportion of total all-cause healthcare costs, which increased with VOC frequency.

Medical Resource Use and Costs of Treating Sickle Cell-related Vaso-occlusive Crisis Episodes: A Retrospective Claims Study.

BACKGROUND: The study investigated the economic burden of vaso-occlusive crisis (VOC) among sickle cell disease (SCD) patients, through assessment of overall utilization and costs and costs per VOC episode (regarding the number of VOC episodes and health care setting, respectively). METHODS: Using the Medicaid Analytic Extracts database, the first SCD-related diagnosis claim (index claim) between June 1, 2009-December 31, 2012 was identified among eligible adults. Patients were required to have continuous medical and pharmacy benefits for 6 months pre- and 12 months post-index. Discrete VOC claims identified within a 3-day gap were combined as a single VOC episode. Annual all-cause and SCD-related medical resources and costs were identified and stratified by number of VOC episodes during the 1-year follow-up period. Health care costs per VOC episode were also examined, stratified by care setting. RESULTS: Enrollees included 8521 eligible patients with a mean age of 32.88 years (SD=12.21). Of these, 66.5% had a Charlson Comorbidity index (CCI) score of 0 (no comorbidities) and 67.3% were female. The average total medical costs were US$34 136 (median=US$12 691) annually, and SCD accounted for 60% of the total costs (mean=US$20 206, median=US$1204). Patients with >3 episodes had the highest annual SCD-related costs (mean=US$58 950) across all settings. Health care resource utilization (HCRU) and costs increased substantially as the number of VOC episodes increased. This study was limited to observation of associations rather than causal inference, and by possible coding and identification discrepancies and the restricted generalizability of the population. CONCLUSIONS: VOC has a severe impact on medical resource use and costs among the adult SCD population. Further research among broader study populations is needed to facilitate the reduction of VOC episodes and thereby improve clinical and economic outcomes for SCD patients.

Mobile Health Technology for Pediatric Symptom Monitoring: A Feasibility Study.

BACKGROUND: Pediatric blood and marrow transplant (PBMT) patients experience significant symptom distress. Mobile health (mHealth) technologies can be leveraged to improve understanding of the patient's symptom experience by providing continuous, real-time, in situ, patient-generated symptom data. This rich data stream can subsequently enhance symptom management strategies. However, limited research has been conducted in this area. OBJECTIVES: This pilot study seeks to (a) explore the feasibility of integrating mHealth technologies to monitor symptom data for PBMT patients and (b) evaluate the study design, measures, and procedures. METHODS: An exploratory longitudinal design was employed to assess the feasibility of monitoring 10 PBMT patients' symptoms using data from two mHealth technologies: (a) a smartphone mHealth application (app) to collect symptom data and (b) a wearable tracking device (Apple watch) to collect physiological data. Feasibility was measured as usability and acceptability. Monthly patient interviews and an end-of-study feasibility survey were employed and analyzed to further understand reasons for sustained interest in and attrition from the study. RESULTS: Overall usability of the wearable was 51%, and app was 56%. Children reported devices were easy to use and acceptable. The study demonstrated acceptability with an enrollment rate of 83% and an attrition rate of 30%, with 70% of the children remaining in the study for at least 40 days. DISCUSSION: This pilot study is among the first to explore the feasibility of using mobile technologies to longitudinally obtain patient-generated symptom data to enhance understanding of the PBMT symptom experience. In addition, it will improve our understanding of how these data present, interact, and cluster together throughout the posttransplant period.

Sickle-Cell Disease Co-Management, Health Care Utilization, and Hydroxyurea Use.

BACKGROUND: Sickle-cell disease (SCD) causes significant morbidity, premature mortality, and high disease burden, resulting in frequent health care use. Comanagement may improve utilization and patient adherence with treatments such as Hydroxyurea. The purpose of this study was to describe acute-care utilization in Medicaid-enrolled patients with SCD, patient factors associated with comanagement, and adherence to Hydroxyurea. METHODS: Data from 2790 patients diagnosed with SCD, age 1 to 65+ years, enrolled at least 1 month in North Carolina Medicaid between March 2016 and February 2017, were analyzed. Outpatient visits were categorized as primary care, hematologist, and nonhematologist specialist. Nurse practitioners or physician assistants with unidentified specialty type or family practice were categorized separately. Comanagement was defined as a minimum of 1 primary care and 1 hematologist visit/patient during the study period. RESULTS: There were notable age-related differences in utilization of health care services. Only 34.82% of the sample was comanaged. Comanagement was higher in the 1-to-9-year-old (44.88%) and 10-to-17-year-old groups (39.22%) versus the 31-to-45-year-old (30.26%) and 65+-year-old (18.75%) age groups. Age had the greatest influence (AUC = 0.599) on whether or not a patient was comanaged. Only a third of the sample (32.24%) had at least 1 Hydroxyurea (HU) prescription. Age was the most predictive factor of good HUadherence (AUC = 0.6503). Prediction by comanagement was minimal with an AUC = 0.5615. CONCLUSION: Comanagement was a factor in predicting HUadherence, but further studies are needed to identify the frequency and components of comanagement needed to increase adherence and reduce acute care utilization.

Treatment patterns and economic burden of sickle-cell disease patients prescribed hydroxyurea: a retrospective claims-based study.

BACKGROUND: This study aimed to evaluate sickle-cell disease (SCD) treatment patterns and economic burden among patients prescribed hydroxyurea (HU) in the US, through claims data. METHODS: SCD patients with pharmacy claims for HU were selected from the Medicaid Analytic Extracts (MAX) from January 1, 2009 - December 31, 2013. The first HU prescription during the identification period was defined as the index date and patients were required to have had continuous medical and pharmacy benefits for ≥6 months baseline and 12 months follow-up periods. Patient demographics, clinical characteristics, treatment patterns, health care utilization, and costs were examined, and variables were analyzed descriptively. RESULTS: A total of 3999 SCD patients prescribed HU were included; the mean age was 19.24 years, most patients were African American (73.3%), and the mean Charlson comorbidity index (CCI) score was 0.6. Asthma (20.3%), acute chest syndrome (15.6%), and infectious and parasitic diseases (20%) were the most prevalent comorbidities. During the 12-month follow-up period, 58.9% (N = 2357) of patients discontinued HU medication. The mean medication possession ratio (MPR) was 0.52, and 22.3% of patients had MPR ≥80%. The average length of stay (LOS) for SCD-related hospitalization was 13.35 days; 64% of patients had ≥1 SCD-related hospitalization. The mean annual total SCD-related costs per patient were $27,779, mostly inpatient costs ($20,128). CONCLUSIONS: Overall, the study showed the patients had significant unmet needs manifest as poor medication adherence, high treatment discontinuation rates, and high economic burden.

Continuous Pain Assessment Using Ensemble Feature Selection from Wearable Sensor Data.

Sickle cell disease (SCD) is a red blood cell disorder complicated by lifelong issues with pain. Management of SCD related pain is particularly challenging due to its subjective nature. Hence, the development of an objective automatic pain assessment method is critical to pain management in SCD. In this work, we developed a continuous pain assessment model using physiological and body movement sensor signals collected from a wearable wrist-worn device. Specifically, we implemented ensemble feature selection methods to select robust and stable features extracted from wearable data for better understanding of pain. Our experiments showed that the stability of feature selection methods could be substantially increased by using the ensemble approach. Since different ensemble feature selection methods prefer varying feature subsets for pain estimation, we further utilized stacked generalization to maximize the information usage contained in the selected features from different methods. Using this approach, our best performing model obtained the root-mean-square error of 1.526 and the Pearson correlation of 0.618 for continuous pain assessment. This indicates that subjective pain scores can be estimated using objective wearable sensor data with high precision.

Abstract Animations for the Communication and Assessment of Pain in Adults: Cross-Sectional Feasibility Study.

BACKGROUND: Pain is the most common physical symptom requiring medical care, yet the current methods for assessing pain are sorely inadequate. Pain assessment tools can be either too simplistic or take too long to complete to be useful for point-of-care diagnosis and treatment. OBJECTIVE: The aim was to develop and test Painimation, a novel tool that uses graphic visualizations and animations instead of words or numeric scales to assess pain quality, intensity, and course. This study examines the utility of abstract animations as a measure of pain. METHODS: Painimation was evaluated in a chronic pain medicine clinic. Eligible patients were receiving treatment for pain and reported pain more days than not for at least 3 months. Using a tablet computer, participating patients completed the Painimation instrument, the McGill Pain Questionnaire (MPQ), and the PainDETECT questionnaire for neuropathic symptoms. RESULTS: Participants (N=170) completed Painimation and indicated it was useful for describing their pain (mean 4.1, SE 0.1 out of 5 on a usefulness scale), and 130 of 162 participants (80.2%) agreed or strongly agreed that they would use Painimation to communicate with their providers. Animations selected corresponded with pain adjectives endorsed on the MPQ. Further, selection of the electrifying animation was associated with self-reported neuropathic pain (r=.16, P=.03), similar to the association between neuropathic pain and PainDETECT (r=.17, P=.03). Painimation was associated with PainDETECT (r=.35, P<.001). CONCLUSIONS: Using animations may be a faster and more patient-centered method for assessing pain and is not limited by age, literacy level, or language; however, more data are needed to assess the validity of this approach. To establish the validity of using abstract animations ("painimations") for communicating and assessing pain, apps and other digital tools using painimations will need to be tested longitudinally across a larger pain population and also within specific, more homogenous pain conditions.