Mental health, pain and likelihood of opioid misuse among adults with sickle cell disease.

Depressive symptoms are prevalent in individuals living with sickle cell disease (SCD) and may exacerbate pain. This study examines whether higher depressive symptoms are associated with pain outcomes, pain catastrophizing, interference and potential opioid misuse in a large cohort of adults with SCD. The study utilized baseline data from the 'CaRISMA' trial, which involved 357 SCD adults with chronic pain. Baseline assessments included pain intensity, daily mood, the Patient Health Questionnaire (PHQ), the Generalized Anxiety Disorders scale, PROMIS Pain Interference, Pain Catastrophizing Scale, the Adult Sickle Cell Quality of Life Measurement Information System and the Current Opioid Misuse Measure. Participants were categorized into 'high' or 'low' depression groups based on PHQ scores. Higher depressive symptoms were significantly associated with increased daily pain intensity, negative daily mood, higher pain interference and catastrophizing, poorer quality of life and a higher likelihood of opioid misuse (all p < 0.01). SCD patients with more severe depressive symptoms experienced poorer pain outcomes, lower quality of life and increased risk of opioid misuse. Longitudinal data from this trial will determine whether addressing depressive symptoms may potentially reduce pain frequency and severity in SCD.

Primary care use and depression screening among young adults with sickle cell disease during their final year of pediatric hematology care.

Depression may contribute to transition risk among young adults with sickle cell disease (SCD). It is unclear if they receive depression screening because primary care providers (PCPs) routinely perform this screening, but PCP use declines with age. This retrospective study of young adults with SCD during their final year of pediatric hematology care identified 51 (91%) had PCPs. Among those with hospital system PCPs, 20% saw their PCP and 50% of those were screened for depression by the PCP. This suggests young adults with SCD may not receive depression screening or see PCPs, leading to potential missed opportunities for intervention.

Identifying erroneous height and weight values from adult electronic health records in the All of Us research program.

INTRODUCTION: Electronic Health Records (EHR) are a useful data source for research, but their usability is hindered by measurement errors. This study investigated an automatic error detection algorithm for adult height and weight measurements in EHR for the All of Us Research Program (All of Us). METHODS: We developed reference charts for adult heights and weights that were stratified on participant sex. Our analysis included 4,076,534 height and 5,207,328 wt measurements from âˆ¼ 150,000 participants. Errors were identified using modified standard deviation scores, differences from their expected values, and significant changes between consecutive measurements. We evaluated our method with chart-reviewed heights (8,092) and weights (9,039) from 250 randomly selected participants and compared it with the current cleaning algorithm in All of Us. RESULTS: The proposed algorithm classified 1.4 % of height and 1.5 % of weight errors in the full cohort. Sensitivity was 90.4 % (95 % CI: 79.0-96.8 %) for heights and 65.9 % (95 % CI: 56.9-74.1 %) for weights. Precision was 73.4 % (95 % CI: 60.9-83.7 %) for heights and 62.9 (95 % CI: 54.0-71.1 %) for weights. In comparison, the current cleaning algorithm has inferior performance in sensitivity (55.8 %) and precision (16.5 %) for height errors while having higher precision (94.0 %) and lower sensitivity (61.9 %) for weight errors. DISCUSSION: Our proposed algorithm outperformed in detecting height errors compared to weights. It can serve as a valuable addition to the current All of Us cleaning algorithm for identifying erroneous height values.

Design and Implementation of the All of Us Research Program COVID-19 Participant Experience (COPE) Survey.

In response to the rapidly evolving coronavirus disease 2019 (COVID-19) pandemic, the All of Us Research Program longitudinal cohort study developed the COVID-19 Participant Experience (COPE) survey to better understand the pandemic experiences and health impacts of COVID-19 on diverse populations within the United States. Six survey versions were deployed between May 2020 and March 2021, covering mental health, loneliness, activity, substance use, and discrimination, as well as COVID-19 symptoms, testing, treatment, and vaccination. A total of 104,910 All of Us Research Program participants, of whom over 73% were from communities traditionally underrepresented in biomedical research, completed 275,201 surveys; 9,693 completed all 6 surveys. Response rates varied widely among demographic groups and were lower among participants from certain racial and ethnic minority populations, participants with low income or educational attainment, and participants with a Spanish language preference. Survey modifications improved participant response rates between the first and last surveys (13.9% to 16.1%, P < 0.001). This paper describes a data set with longitudinal COVID-19 survey data in a large, diverse population that will enable researchers to address important questions related to the pandemic, a data set that is of additional scientific value when combined with the program's other data sources.

Pregnancy-related thromboembolism in women with sickle cell disease: An analysis of National Medicaid Data.

Pregnancy and sickle cell disease (SCD) both individually carry a risk of thromboembolism (TE). Pregnancy in people with SCD may further enhance the prothrombotic effect of the underlying disease. The objectives of this study were to determine the rate and risk factors for arterial and venous thrombosis in pregnant people with SCD. Administrative claims data from the United States Centers for Medicare and Medicaid Service Analytic eXtract from 2006 to 2018 were used. The study population included people with SCD from the start of their first identified pregnancy until 1 year postpartum and a control cohort of pregnant people without SCD of similar age and race. Outcomes of interest were identified with ICD-9 or 10 codes. Logistic regression analyses were used to analyze risk factors. We identified infant deliveries in 6388 unique people with SCD and 17 110 controls. A total of 720 venous thromboembolism (11.3%) and 335 arterial TE (5.2%) were observed in people with SCD compared to 202 (1.2%) and 95 (0.6%) in controls. People with SCD had an 8-11 times higher odds of TE compared to controls (p < .001). Within the SCD cohort, age, hemoglobin SS (HbSS) genotype, hypertension, and history of thrombosis were identified as independent risk factors for pregnancy-related TE. Pregnancy-specific factors (pre-eclampsia, eclampsia, multigestational pregnancy) were not associated with TE. In conclusion, the risk of pregnancy-related TE is considerably higher in people with SCD compared with controls without SCD. Hence, people with SCD, particularly those with multiple risk factors may be candidates for thromboprophylaxis during pregnancy and the postpartum period.

Parent Activation in the Neonatal Intensive Care Unit.

OBJECTIVE: Patient activation is the knowledge, skills, and confidence to manage one's health; parent activation is a comparable concept related to a parent's ability to manage a child's health. Activation in adults is a modifiable risk factor and associated with clinical outcomes and health care utilization. We examined activation in parents of hospitalized newborns observing temporal trends and associations with sociodemographic characteristics, neonate characteristics, and outcomes. STUDY DESIGN: Participants included adult parents of neonates admitted to a level-IV neonatal intensive care unit in an academic medical center. Activation was measured with the 10-item Parent version of the Patient Activation Measure (P-PAM) at admission, discharge, and 30 days after discharge. Associations with sociodemographic variables, health literacy, clinical variables, and health care utilization were evaluated. RESULTS: A total of 96 adults of 64 neonates were enrolled. The overall mean P-PAM score on admission was 81.8 (standard deviation [SD] = 18), 88.8 (SD = 13) at discharge, and 86.8 (SD = 16) at 30-day follow-up. Using linear mixed regression model, P-PAM score was significantly associated with timing of measurement. Higher P-PAM scores were associated with higher health literacy (p = 0.002) and higher in mothers compared to fathers (p = 0.040). There were no significant associations of admission P-PAM scores with sociodemographic characteristics. Parents of neonates who had a surgical diagnosis had a statistically significant (p = 0.003) lower score than those who did not. There were no associations between discharge P-PAM scores and neonates' lengths of stay or other indicators of illness severity. CONCLUSION: Parental activation in the NICU setting was higher than reported in the adult and limited pediatric literature; scores increased from admission to discharge and 30-day postdischarge. Activation was higher in mothers and parents with higher health literacy. Additional larger scale studies are needed to determine whether parental activation is associated with long-term health care outcomes as seen in adults. KEY POINTS: · Little is known about activation in parents of neonates.. · Activation plays a role in health outcomes in adults.. · Larger studies are needed to explore parent activation..

Patient-Centered Digital Health Records and Their Effects on Health Outcomes: Systematic Review.

BACKGROUND: eHealth tools such as patient portals and personal health records, also known as patient-centered digital health records, can engage and empower individuals with chronic health conditions. Patients who are highly engaged in their care have improved disease knowledge, self-management skills, and clinical outcomes. OBJECTIVE: We aimed to systematically review the effects of patient-centered digital health records on clinical and patient-reported outcomes, health care utilization, and satisfaction among patients with chronic conditions and to assess the feasibility and acceptability of their use. METHODS: We searched MEDLINE, Cochrane, CINAHL, Embase, and PsycINFO databases between January 2000 and December 2021. PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analyses) guidelines were followed. Eligible studies were those evaluating digital health records intended for nonhospitalized adult or pediatric patients with a chronic condition. Patients with a high disease burden were a subgroup of interest. Primary outcomes included clinical and patient-reported health outcomes and health care utilization. Secondary outcomes included satisfaction, feasibility, and acceptability. Joanna Briggs Institute critical appraisal tools were used for quality assessment. Two reviewers screened titles, abstracts, and full texts. Associations between health record use and outcomes were categorized as beneficial, neutral or clinically nonrelevant, or undesired. RESULTS: Of the 7716 unique publications examined, 81 (1%) met the eligibility criteria, with a total of 1,639,556 participants across all studies. The most commonly studied diseases included diabetes mellitus (37/81, 46%), cardiopulmonary conditions (21/81, 26%), and hematology-oncology conditions (14/81, 17%). One-third (24/81, 30%) of the studies were randomized controlled trials. Of the 81 studies that met the eligibility criteria, 16 (20%) were of high methodological quality. Reported outcomes varied across studies. The benefits of patient-centered digital health records were most frequently reported in the category health care utilization on the "use of recommended care services" (10/13, 77%), on the patient-reported outcomes "disease knowledge" (7/10, 70%), "patient engagement" (13/28, 56%), "treatment adherence" (10/18, 56%), and "self-management and self-efficacy" (10/19, 53%), and on the clinical outcome "laboratory parameters," including HbA1c and low-density lipoprotein (LDL; 16/33, 48%). Beneficial effects on "health-related quality of life" were seen in only 27% (4/15) of studies. Patient satisfaction (28/30, 93%), feasibility (15/19, 97%), and acceptability (23/26, 88%) were positively evaluated. More beneficial effects were reported for digital health records that predominantly focus on active features. Beneficial effects were less frequently observed among patients with a high disease burden and among high-quality studies. No unfavorable effects were observed. CONCLUSIONS: The use of patient-centered digital health records in nonhospitalized individuals with chronic health conditions is potentially associated with considerable beneficial effects on health care utilization, treatment adherence, and self-management or self-efficacy. However, for firm conclusions, more studies of high methodological quality are required. TRIAL REGISTRATION: PROSPERO (International Prospective Register of Systematic Reviews) CRD42020213285; https://www.crd.york.ac.uk/prospero/display_record.php?RecordID=213285.

Digital behavioural interventions for people with sickle cell disease.

This is a protocol for a Cochrane Review (intervention). The objectives are as follows: To identify and assess the effects of digital behavioural interventions focused on behavioural change in people with SCD on: medication adherence or disease management (such as managing acute and chronic pain), or both, on health- and other-related outcomes;specific subgroups defined by age (i.e. children, adolescents and adults) and type of modality or delivery (e.g. cell phone, the Internet).

Development of the Initial Surveys for the All of Us Research Program.

BACKGROUND: The All of Us Research Program is building a national longitudinal cohort and collecting data from multiple information sources (e.g., biospecimens, electronic health records, and mobile/wearable technologies) to advance precision medicine. Participant-provided information, collected via surveys, will complement and augment these information sources. We report the process used to develop and refine the initial three surveys for this program. METHODS: The All of Us survey development process included: (1) prioritization of domains for scientific needs, (2) examination of existing validated instruments, (3) content creation, (4) evaluation and refinement via cognitive interviews and online testing, (5) content review by key stakeholders, and (6) launch in the All of Us electronic participant portal. All content was translated into Spanish. RESULTS: We conducted cognitive interviews in English and Spanish with 169 participants, and 573 individuals completed online testing. Feedback led to over 40 item content changes. Lessons learned included: (1) validated survey instruments performed well in diverse populations reflective of All of Us; (2) parallel evaluation of multiple languages can ensure optimal survey deployment; (3) recruitment challenges in diverse populations required multiple strategies; and (4) key stakeholders improved integration of surveys into larger Program context. CONCLUSIONS: This efficient, iterative process led to successful testing, refinement, and launch of three All of Us surveys. Reuse of All of Us surveys, available at http://researchallofus.org, may facilitate large consortia targeting diverse populations in English and Spanish to capture participant-provided information to supplement other data, such as genetic, physical measurements, or data from electronic health records.

Increased Patient Activation Is Associated with Fewer Emergency Room Visits and Hospitalizations for Pain in Adults with Sickle Cell Disease.

OBJECTIVE: Recurrent vaso-occlusive pain episodes, the most common complication of sickle cell disease (SCD), cause frequent health care utilization. Studies exploring associations between patient activation and acute health care utilization for pain are lacking. We tested the hypothesis that increased activation and self-efficacy are associated with decreased health care utilization for pain in SCD. METHODS: In this cross-sectional study of adults with SCD at a tertiary medical center, we collected demographics, SCD phenotype, Patient Activation Measure levels, and self-efficacy scores using structured questionnaires. We reviewed charts to obtain disease-modifying therapy and acute health care utilization, defined as emergency room visits and hospitalizations, for vaso-occlusive pain episodes. Negative binomial regression analyses were used to test the hypothesis. RESULTS: We surveyed 67 adults with SCD. The median age was 27.0 years, 53.7% were female, and 95.5% were African American. Median health care utilization for pain over one year (range) was 2.0 (0-24). Only one-third of participants (38.8%) were at the highest activation level (median [range] = 3 [1-4]). Two-thirds (65.7%) of participants had high self-efficacy (median [range] = 32.0 [13-45]). Regressions showed significant association between health care utilization and activation (incidence rate ratio [IRR] = 0.663, P = 0.045), self-efficacy (IRR = 0.947, P = 0.038), and male sex (IRR = 0.390, P = 0.003). Two outliers with high activation, self-efficacy, and health care utilization also had addictive behavior. CONCLUSIONS: Many individuals with SCD have suboptimal activation and reduced self-efficacy. Higher activation and self-efficacy were associated with lower health care utilization for pain. Additional studies are needed to evaluate interventions to improve activation and self-efficacy and reduce acute health care utilization for pain.

Patient-Centered eHealth Interventions for Children, Adolescents, and Adults With Sickle Cell Disease: Systematic Review.

BACKGROUND: Sickle cell disease is an inherited blood disorder that affects over 100,000 Americans. Sickle cell disease-related complications lead to significant morbidity and early death. Evidence supporting the feasibility, acceptability, and efficacy of self-management electronic health (eHealth) interventions in chronic diseases is growing; however, the evidence is unclear in sickle cell disease. OBJECTIVE: We systematically evaluated the most recent evidence in the literature to (1) review the different types of technological tools used for self-management of sickle cell disease, (2) discover and describe what self-management activities these tools were used for, and (3) assess the efficacy of these technologies in self-management. METHODS: We reviewed literature published between 1995 and 2016 with no language limits. We searched MEDLINE, EMBASE, CINAHL, PsycINFO, and other sources. We followed the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines. Two independent reviewers screened titles and abstracts, assessed full-text articles, and extracted data from articles that met inclusion criteria. Eligible studies were original research articles that included texting, mobile phone-based apps, or other eHealth interventions designed to improve self-management in pediatric and adult patients with sickle cell disease. RESULTS: Of 1680 citations, 16 articles met all predefined criteria with a total of 747 study participants. Interventions were text messaging (4/16, 25%), native mobile apps (3/16, 19%), Web-based apps (5/16, 31%), mobile directly observed therapy (2/16, 13%), internet-delivered cognitive behavioral therapy (2/16, 13%), electronic pill bottle (1/16, 6%), or interactive gamification (2/16, 13%). Interventions targeted monitoring or improvement of medication adherence (5/16, 31%); self-management, pain reporting, and symptom reporting (7/16, 44%); stress, coping, sleep, and daily activities reporting (4/16, 25%); cognitive training for memory (1/16, 6%); sickle cell disease and reproductive health knowledge (5/16, 31%); cognitive behavioral therapy (2/16, 13%); and guided relaxation interventions (1/16, 6%). Most studies (11/16, 69%) included older children or adolescents (mean or median age 10-17 years; 11/16, 69%) and 5 included young adults (≥18 years old) (5/16, 31%). Sample size ranged from 11 to 236, with a median of 21 per study: <20 in 6 (38%), ≥20 to <50 in 6 (38%), and >50 participants in 4 studies (25%). Most reported improvement in self-management-related outcomes (15/16, 94%), as well as high satisfaction and acceptability of different study interventions (10/16, 63%). CONCLUSIONS: Our systematic review identified eHealth interventions measuring a variety of outcomes, which showed improvement in multiple components of self-management of sickle cell disease. Despite the promising feasibility and acceptability of eHealth interventions in improving self-management of sickle cell disease, the evidence overall is modest. Future eHealth intervention studies are needed to evaluate their efficacy, effectiveness, and cost effectiveness in promoting self-management in patients with sickle cell disease using rigorous methods and theoretical frameworks with clearly defined clinical outcomes.

Classifying patient portal messages using Convolutional Neural Networks.

OBJECTIVE: Patients communicate with healthcare providers via secure messaging in patient portals. As patient portal adoption increases, growing messaging volumes may overwhelm providers. Prior research has demonstrated promise in automating classification of patient portal messages into communication types to support message triage or answering. This paper examines if using semantic features and word context improves portal message classification. MATERIALS AND METHODS: Portal messages were classified into the following categories: informational, medical, social, and logistical. We constructed features from portal messages including bag of words, bag of phrases, graph representations, and word embeddings. We trained one-versus-all random forest and logistic regression classifiers, and convolutional neural network (CNN) with a softmax output. We evaluated each classifier's performance using Area Under the Curve (AUC). RESULTS: Representing the messages using bag of words, the random forest detected informational, medical, social, and logistical communications in patient portal messages with AUCs: 0.803, 0.884, 0.828, and 0.928, respectively. Graph representations of messages outperformed simpler features with AUCs: 0.837, 0.914, 0.846, 0.884 for informational, medical, social, and logistical communication, respectively. Representing words with Word2Vec embeddings, and mapping features using a CNN had the best performance with AUCs: 0.908 for informational, 0.917 for medical, 0.935 for social, and 0.943 for logistical categories. DISCUSSION AND CONCLUSION: Word2Vec and graph representations improved the accuracy of classifying portal messages compared to features that lacked semantic information such as bag of words, and bag of phrases. Furthermore, using Word2Vec along with a CNN model, which provide a higher order representation, improved the classification of portal messages.

Rapid growth in surgeons' use of secure messaging in a patient portal.

BACKGROUND: Use of secure messaging through patient portals has risen substantially in recent years due to provider incentives and consumer demand. Secure messaging may increase patient satisfaction and improve outcomes, but also adds to physician workload. Most prior studies of secure messaging focused on primary care and medical specialties. We examined surgeons' use of secure messaging and the contribution of messaging to outpatient interactions in a broadly deployed patient portal. METHODS: We determined the number of clinic visits and secure messages for surgical providers in the first 3 years (2008-2010) after patient portal deployment at an academic medical center. We calculated the proportion of outpatient interaction conducted through messaging for each specialty. Logistic regression models compared the likelihood of message-based versus clinic outpatient interaction across surgical specialties. RESULTS: Over the study period, surgical providers delivered care in 648,200 clinic visits and received 83,912 messages, with more than 200% growth in monthly message volume. Surgical specialties receiving the most messages were orthopedics/podiatry (25.1%), otolaryngology (20.1%), urology (10.8%), and general surgery (9.6%); vascular surgery (0.8%) and pediatric general surgery (0.2%) received the fewest. The proportion of outpatient interactions conducted through secure messaging increased significantly from 5.4% in 2008 to 15.3% in 2010 (p < 0.001) with all specialties experiencing growth. Heart/lung transplantation (74.9%), liver/kidney/pancreas transplantation (69.5%), and general surgery (48.7%) had the highest proportion of message-based outpatient interaction by the end of the study. CONCLUSIONS: This study demonstrates rapid adoption of online secure messaging across surgical specialties with significant growth in its use for outpatient interaction. Some specialties, particularly those with long-term follow-up, interacted with patients more through secure messaging than in person. As surgeons devote more time to secure messaging, additional research will be needed to understand the care delivered through online interactions and to develop models for reimbursement.

Comparing super-utilizers and lower-utilizers among commercial- and Medicare-insured adults with sickle cell disease.

ABSTRACT: Sickle cell disease (SCD) is a rare but costly condition in the United States. Super-utilizers have been defined as a subset of the population with high health care encounters or expenditures. Although super-utilizers have been described in other disease states, little is known about super-utilizers among adults with SCD. This study aimed to characterize the differences in expenditures, overall health care encounters, and pain episode encounters between super-utilizers (top 10% expenditures) and lower-utilizers with SCD (high, top 10%-24.9%; moderate, 25%-49.9%; and low, bottom 50% expenditures). A retrospective longitudinal cohort of adults with SCD were identified using validated algorithms in MarketScan and Medicare claim databases from 2016 to 2020. Encounters and expenditures were analyzed from inpatient, outpatient, and emergency department settings. Differences in encounters and expenditures between lower-utilizers and super-utilizers were compared using logistic regression. Among super-utilizers, differences in encounters and expenditures were compared according to incidences of pain episode encounters. The study population included 5666 patients with commercial insurance and 8600 with Medicare. Adjusted total annual health care expenditure was 43.46 times higher for super-utilizers than for low-utilizers among commercial-insured and 13.37 times higher in Medicare-insured patients. Among super-utilizers, there were patients with few pain episode encounters who had higher outpatient expenditures than patients with a high number of pain episode encounters. Our findings demonstrate the contribution of expensive outpatient care among SCD super-utilizers, in which analyses of high expenditure have largely focused on short-term care. Future studies are needed to better understand super-utilizers in the SCD population to inform the effective use of preventive interventions and/or curative therapies.

Burden of employment loss and absenteeism in adults and caregivers of children with sickle cell disease.

ABSTRACT: Sickle cell disease (SCD) is a genetic disorder affecting 100 000 people with an estimated annual medical cost of $3 billion in the United States; however, the economic impact on patients is not well described. We aimed to examine the indirect economic burden and test the hypothesis that socioeconomic status and greater social vulnerability risks are associated with increased absenteeism and employment loss. We surveyed adults and caregivers of children with SCD at 5 US centers from 2014 to 2021. Logistic regression models were used to examine the associations of employment loss and missed days of work with demographics and social determinants. Indirect costs were estimated by multiplying the self-reported missed days of work and job loss by 2022 average wages by the state of the participating institution. Of the 244 participants, 10.3% reported employment loss in the last 5 years, and 17.5% reported missing 10 or more days of work. Adults had 3 times more employment loss compared with caregivers of children with SCD (OR, 3.18; 95% CI, 1.12-9.01) but fewer missed days of work (OR, 0.24; 95% CI, 0.11-0.0.51). Participants who did not live with a partner reported increased employment loss (OR, 4.70; 95% CI, 1.04-21.17) and more missed days of work (OR, 4.58; 95% CI, 1.04-20.15). The estimated annual indirect economic burden was $2 266 873 ($9290 per participant). Adults with SCD and caregivers of children with SCD commonly report employment loss and missed days of work as important risk factors. The high indirect economic burden suggests that future economic evaluations of SCD should include SCD-related indirect economic burden.

Measuring social determinants of health in the All of Us Research Program.

To accelerate medical breakthroughs, the All of Us Research Program aims to collect data from over one million participants. This report outlines processes used to construct the All of Us Social Determinants of Health (SDOH) survey and presents the psychometric characteristics of SDOH survey measures in All of Us. A consensus process was used to select SDOH measures, prioritizing concepts validated in diverse populations and other national cohort surveys. Survey item non-response was calculated, and Cronbach's alpha was used to analyze psychometric properties of scales. Multivariable logistic regression models were used to examine associations between demographic categories and item non-response. Twenty-nine percent (N = 117,783) of eligible All of Us participants submitted SDOH survey data for these analyses. Most scales had less than 5% incalculable scores due to item non-response. Patterns of item non-response were seen by racial identity, educational attainment, income level, survey language, and age. Internal consistency reliability was greater than 0.80 for almost all scales and most demographic groups. The SDOH survey demonstrated good to excellent reliability across several measures and within multiple populations underrepresented in biomedical research. Bias due to survey non-response and item non-response will be monitored and addressed as the survey is fielded more completely.

A feasibility randomized controlled trial of an mHealth app vs booklets for patient-facing guidelines in adults with SCD.

Despite the increased number of evidence-based guidelines for sickle cell disease (SCD), dissemination of evidence-based guidelines in lay language for individuals or families with SCD has not been evaluated. We conducted a feasibility randomized controlled trial to determine the acceptability of a mobile health (mHealth) app with patient-facing guidelines to improve the knowledge of individuals with SCD about SCD-specific knowledge and reduce hospitalizations. Primary outcome measures include recruitment, retention, and adherence rates. Adults with SCD were enrolled at 2 sickle cell centers between 2018 and 2022. Participants were randomized to receive either an mHealth app + booklet with patient-facing guidelines or a booklet with the guidelines alone. Participants completed surveys at baseline and a final 6-month visit. Approximately 67 of 74 (91%) agreed to participate and were randomized, with 50 of 67 (75%) completing all the study components. All participants who completed the study in the treatment arm used the app. Our results demonstrated high recruitment, retention, and adherence rate for the first randomized trial for an mHealth app with patient-facing guidelines in adults with SCD. This clinical trial was registered at https://www.clinicaltrials.gov/ as #NCT03629678.

Comparison of thromboembolism outcomes in patients with sickle cell disease prescribed hormonal contraception.

Patients with sickle cell disease (SCD) are at a risk of thromboembolism (TE), and use of hormonal contraception can further increase that risk. This study aims to assess patterns of hormonal contraceptive use and compare risk of contraception-related TE between combined hormonal contraceptives (CHCs) and progestin-only contraceptives (POCs). Patients with SCD aged between 12 and 44 years with a new prescription of a hormonal contraceptive in the Centers for Medicare and Medicaid Services Medicaid Analytic eXtract database (2006-2018) were followed up to 1 year. We identified 7173 new users: 44.6% initiated CHC and 55.4% initiated POC. Combined oral contraceptive pills (OCPs; 36.5%) and progestin-only depot medroxyprogesterone acetate (33.9%) were the most frequently prescribed agents. A total of 1.8% of contraception users had a new diagnosis of TE within 1 year of the first identified contraception prescription. There were no significant differences in TE event rates between CHC and POC users (17.2 and 24.7 events per 1000 person-years, respectively). In patients prescribed OCP, there were no differences in TE event rates based on estrogen dose or progestin generation. Transdermal patch had a 2.4-fold increased risk of TE as compared with that of OCP. Although limited by the retrospective study design and use of administrative claims data, this study found no significant differences in TE rates between new users of CHC and POC in patients with SCD. Careful evaluation of underlying TE risk factors should be considered for each patient with SCD before initiation of hormonal contraception.

Creating an automated contemporaneous cohort in sickle cell anemia to predict survival after disease-modifying therapy.

The Food and Drug Administration requires contemporaneous controls to compare clinical outcomes for participants receiving experimental gene therapy or gene editing clinical trials. However, developing a contemporaneous cohort of rare diseases requires multiple person-hours. In a single referral center for sickle cell disease, we tested the hypothesis that we could create an automated contemporaneous cohort of children and adults with sickle cell anemia (SCA) to predict mortality. Data were obtained between 1 January 2004 and 30 April 2021. We identified 419 individuals with SCA with consistent medical care defined as followed continuously for >0.5 years with no visit gaps >3.0 years. The median age was 10.2 years (IQR, 1-24 years), with a median follow-up of 7.4 years (IQR, 3.6-13.5 years) and 47 deaths. A total of 98% (274 of 277) of the children remained alive at 18 years of age, and 34.3% (94 of 274) of those children were followed into adulthood. For adults, the median age of survival was 49.3 years. Treatment groups were mutually exclusive and in a hierarchical order: hematopoietic stem cell transplant (n = 22)>regular blood transfusion for at least 2 years (n = 56)>hydroxyurea for at least 1 year (n = 243)>no disease-modifying therapy (n = 98). Compared to those receiving no disease-modifying treatment, those treated with hydroxyurea therapy had a significantly lower hazard of mortality (hazard ratio = 0.38; P = 0.016), but no statistical difference for those receiving regular blood transfusions compared to no disease-modifying therapy (hazard ratio = 0.71; P = 0.440). An automated contemporaneous SCA cohort can be generated to estimate mortality in children and adults with SCA.