The utility of a fidelity measure to monitor early psychosis services across Australia

Aims: This project aims to describe the development, implementation, and adaptation of a fidelity tool to measure service quality in a national network of early psychosis services across Australia-the headspace Early Psychosis program. Method(s): An 80-item Early Psychosis Prevention and Intervention Centre Model Integrity Tool (EMIT) was developed. The tool assesses adherence in six sites across Australia. Ratings were informed by interviews, routine data and site policies. The EMIT has been adapted for use virtually during Covid-19 restrictions on in-person site visits. A review is underway to enhance the tool to capture quality of services. The Revised EPPIC Model Integrity Tool (REMIT) will be utilized to assess fidelity and service quality in 2023. Result(s): All six sites participated in five fidelity assessments since 2017. In the initial visits, average scores were in the 'low' fidelity range. By the fifth fidelity visit, the network average improved to 'superior' fidelity. The EMIT was successfully adapted for use virtually during Covid-19 and sites were able to maintain scores of high to superior fidelity. The results from the use of the redeveloped tool, the REMIT will be presented. Conclusion(s): The Australian Early Psychosis model has been successfully implemented across the headspace Early Psychosis program. Ongoing fidelity assessments are an effective method to improve and maintain fidelity. The review and development of the REMIT reflects the sites state of implementation and ensures services are of high quality. The findings of the initial use of the REMIT tool in fidelity assessments in early 2023.

What impact did the COVID-19 pandemic have on psychosis and the delivery of early intervention psychosis (EIP) services in Australia?

Background: The COVID-19 pandemic had a catastrophic impact worldwide, the significance of which continues to be explored. For EIP services the implications were two fold. First, that the pandemic and associated psychosocial stressors would lead to an increased incidence of psychosis and secondly, social distancing measures would negatively impact the detection and treatment of people with psychosis. 18 months on and we now have the data to explore these questions. Method(s): Participants included in this study are young people who attended one of the six headspace Early Psychosis (hEP) services across Australia and met the criteria for being at Ultra High Risk (UHR) of psychosis or experiencing a First Episode of Psychosis (FEP), were aged between 12 and 25 years, and provided informed consent for their data to be used. We will analyse the following routinely collected data from young people accessing EIP services and compared outcomes collected the year before the COVID-19 pandemic occurred (March 2019-February 2020) with data collected during the pandemic (March 2020 onwards): a. Clinical and functional outcomes of young people at UHR or with FEP, b. Duration of untreated psychosis (DUP), c. patterns of substance use, and d. source and rates of referrals. Results and Conclusion(s): I will present the differences in the above outcomes for those young people accessing treatment pre and during COVID-19 pandemic. Confounding factors such as age, gender, sexuality, ATSI status, CALD background will be controlled for. Discussion of the results ad implication for clinical practice will follow.

Humoral immune responses to SARS-CoV-2 vaccines in an ozanimod open-label extension study

Background: Ozanimod, an oral sphingosine 1-phosphate receptor modulator, is approved in the European Union and United States for the treatment of moderately to severely active ulcerative colitis (UC) and relapsing multiple sclerosis (RMS). A previous analysis of data from UC and multiple sclerosis (MS) open-label extension (OLE) studies showed that most patients with confirmed coronavirus infection (COVID-19) had nonserious infections, recovered, and did not require ozanimod discontinuation. Some immunomodulators and biologics may attenuate severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) vaccine response;therefore, this analysis evaluated humoral immune responses and predictors of response to SARS-CoV-2 vaccination in patients with RMS treated with ozanimod. Method(s): RMS participants who completed a phase 1-3 ozanimod trial could enter an OLE trial (DAYBREAK;NCT02576717) of ozanimod 0.92 mg/d. This analysis (January 2020-October 2021) included DAYBREAK participants receiving mRNA or non-mRNA SARS-CoV-2 vaccines (1-2 doses, vaccine-dependent) with no evidence of recent infection (ie, nucleocapsid antibody negative). Receptor binding domain (RBD) antibody titers were analysed (Elecsys Anti-SARS-CoV-2 assay;Roche Diagnostics, Basel, Switzerland) prevaccination, after 1 dose, and <4, 4-8, 8-12, and >12 weeks after full vaccination. Fisher's exact tests and regression models determined association with seroconversion and log2 antibody levels. Result(s): Demographics were similar between the mRNA and nonmRNA vaccine recipients (Table). Seroconversion (>=0.8 U/mL spike RBD antibody) occurred in 100% (80/80) of fully vaccinated mRNA recipients and 62% (18/29) of fully vaccinated non-mRNA vaccine recipients. Higher spike RBD antibody levels were seen with mRNA (grand mean: 512.6 U/mL, range: 1.3-4572.0) vs non-mRNA (grand mean: 39.3 U/mL, range: 0.4-368.5) vaccines at all time points studied. Vaccination with a non-mRNA vaccine predicted lower antibody levels (beta: -5.90 [95% CI: -6.99 to -4.82];P<0.0001) and less seroconversion (Fisher's exact: P<0.0001), whereas age, sex, body mass index, and absolute lymphocyte count (ALC) did not. Conclusion(s): Participants receiving ozanimod developed humoral immune response to SARS-CoV-2 vaccines, with 100% seroconversion after mRNA vaccination;this was independent of demographic characteristics and ALC levels at time of vaccination. However, some participants developed lower antibody concentrations and may benefit from booster doses. These findings provide important information for physicians managing ozanimod-treated patients with UC or MS.

SUPPORTING DEMENTIA CAREGIVERS DURING COVID-19 WITH CAREHEROES IT: USAGE PATTERNS AND OUTCOMES

Caregivers of people with dementia often experience negative physical and mental health outcomes due to the complex challenges posed by dementia symptoms and navigating care systems. The COVID-19 pandemic posed even greater hurdles for caregivers as many health care and support services transferred to virtual formats and required increased use of information technologies (IT). This research examined usage data and psychosocial outcomes of dementia caregivers who used a new mobile-enabled web-based app that was offered as an adjunct to clinical care at two memory clinics (Birmingham, AL;Miami, FL) during the pandemic. This app, called CareHeroes: (1) allows caregivers to self-assess their emotional and mental health;(2) offers a secure platform for tracking and communicating patient-related information among caregivers and providers;and (3) offers caregiver education. CareHeroes is also available in Spanish. Effort was made to recruit digitally underserved populations of caregivers (e.g., rural dwelling, underserved racial/ethnic groups). This presentation will review caregiver outcomes (e.g., burden, depression, user data) at baseline and 3-month follow-up. Among the 22 caregivers who enrolled in the study, 17 completed baseline and 3-month follow-up interviews. Over a 14-month period, participants logged onto CareHeroes 131 times, which varied by month. Caregivers most often used CareHeroes to complete assessment tools. Findings indicate that depression and burden were lower at 3-month follow-up compared to baseline, though this reduction was not statistically significant. The presentation will also review challenges of integrating a new technology intervention designed to promote telehealth during the COVID-19 pandemic.

Serologic Response and Clinical Outcomes of SARSCoV-2 Infection and Vaccination in Ozanimod-Treated Participants With Relapsing Multiple Sclerosis

Introduction: Multiple sclerosis disease-modifying therapies, including sphingosine 1-phosphate receptor modulators, may attenuate the response to severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection or vaccination. Objective(s): To describe the serological response and clinical outcomes of SARS-CoV-2 infection and vaccination in ozanimodtreated participants with relapsing multiple sclerosis (RMS) in an open-label extension (OLE) trial. Method(s): Participants with RMS who completed a phase 1-3 ozanimod trial could enter an OLE trial (DAYBREAK-NCT02576717) of ozanimod 0.92 mg/d. This analysis (January 2020-October 2021 [serology] and January 2022 [clinical outcomes]) included DAYBREAK participants who received SARSCoV-2 vaccines (fully vaccinated) and/or had COVID-19 adverse events. Receptor binding domain (RBD) antibody levels and nucleocapsid antibody positivity were analysed using Roche Elecsys assays. Log2 RBD antibody levels were compared between groups using t-tests. Result(s): Among the 148 vaccinated participants with serological data, 39 participants had serologically confirmed SARS-CoV-2 exposure. After full vaccination, RBD seroconversion occurred in 100% (n=39/39) of nucleocapsid antibody positive and most (n=98/109) nucleocapsid antibody negative participants (with 100% seroconversion in nucleocapsid antibody negative participants receiving mRNA vaccines [n=80/80]). Significantly higher RBD antibody levels were observed in the vaccinated nucleocapsid antibody positive vs negative vaccinated participants (median [range], U/mL: 2259 [12.4-44260.0] vs 138 [0.4-4572.0], respectively, P<0.0001). COVID-19 adverse events were reported in 15/148 participants, all nonserious events (confirmed=12, suspected= 3). Ozanimod treatment was continued in 9 participants and interrupted in 5 (1 unknown). Eleven participants recovered by the time of data cut off, and one recovered with sequelae (cough and loss of sense of smell). Conclusion(s): Participants with RMS receiving ozanimod mount a serologic response to SARS-CoV-2 infection and vaccination. COVID-19 events in these fully vaccinated participants were nonserious. A limitation of this research is its retrospective nature and the potential for selection bias towards higher-risk individuals.

Online Programming for Dancers and Dance Educators: A Response to COVID-19 and the Racial Justice Movement

Upon the convergence of the twin epidemics of COVID-19 and racial injustice in 2020, the Delaware Dance Education Organization (DDEO) responded by creating and presenting interactive professional development webinars that ranged from sharing how dance history is a microcosm of the world to the impact culture has on personal behaviors. Through the lens of public scholarship, the virtual programming format allowed DDEO to reach dance populations in Delaware and beyond. Groundswell, a series of four webinars presented a consideration of the impact racism has in the dance world. Solos@Home invited participants to create, share, and virtually perform dance works based on material presented in Groundswell. Solos@Home II continued in this modality highlighting the theme of environmental justice. The value of activism was a key enduring understanding for these projects. DDEO continues to support and advance dance education while listening and responding to the immediate needs of the community. © 2022, Pennsylvania State University Libraries. All rights reserved.

INTERIM RESULTS OF A PHASE 3b, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED TRIAL EVALUATING 24-MONTH SAFETY OF ELAGOLIX WITH ADD-BACK FOR HEAVY MENSTRUAL BLEEDING ASSOCIATED WITH UTERINE FIBROIDS

Objective: To evaluate the long-term safety of oral gonadotropin-releasing hormone receptor elagolix (ELA) with hormonal add-back therapy (AB) in women with heavy menstrual bleeding (HMB) associated with uterine fibroids (UF). Material(s) and Method(s): This is a multicenter, phase 3b, sequential, randomized, 12-month (M) double-blind, placebo-controlled, 36M open-label study to evaluate the safety of ELA 300 mg twice a day (BID) with estradiol 1 mg/norethindrone acetate 0.5 mg (E2/NETA) one a day AB (NCT03271489) in women with HMB associated with UF. For the first 12M of the study, the study group received ELA+AB and the control group received placebo (PBO) in a 2:1 ratio. After 12M, patients in both groups entered the open-label treatment period, whereby all patients in the study received ELA+AB. This study is ongoing. Herein, we report safety data at 12M and 24M. Result(s): A total of 478 women were randomly assigned to receive ELA+AB (n = 319) or PBO (n = 159). Patient demographics were balanced between groups with a study mean (SD) age of 42.3 (5.3) years, mean (SD) BMI 32.8 (7.2) kg/m2, and the majority of participants (57%) were Black. At the end of 12M, overall adverse events (AEs) were slightly higher with ELA+AB compared with PBO (63.3% versus 52.2%, respectively). Serious AEs occurred in 3.1% of women taking ELA+AB compared with 1.3% taking PBO. The most common AEs (>=5%) were hot flush (ELA+AB, 9.4%;PBO, 3.8%), headache (ELA+AB, 5.6%;PBO, 3.8%), and back pain (ELA+AB, 3.8%;PBO, 5.0%);key AEs of special interest (AESI) included bone mineral density decrease (ELA+AB, 5.0%;PBO, 1.3%) and intermenstrual bleeding (ELA+AB, 3.8%;PBO, 0%). A total of 278 patients entered the open-label treatment period. AE frequency was similar to the placebo control period (ELA+AB/ELA+AB, 54.1%;PBO/ELA+AB, 66.7%). The serious AE rate was ELA+AB/ELA+AB, n=7 (3.6%);PBO/ELA+AB, n=4 (4.8%). The most common AEs during the open-label treatment period were COVID-19 (ELA+AB/ELA+AB, 3.1%;PBO/ELA+AB, 8.3%), decreased bone density (ELA+AB/ELA+AB, 7.7%;PBO/ELA+AB, 6.0%), heavy menstrual bleeding (ELA+AB/ELA+AB, 1.5%;PBO/ELA+AB, 7.1%), hot flush (ELA+AB/ELA+AB, 1.0%;PBO/ELA+AB, 6.0%) and hypertension (ELA+AB/ELA+AB, 3.6%;PBO/ELA+AB, 6.0%). In general, the AESI rates observed at 24M were consistent with those observed at 12M. Conclusion(s): The overall safety profile of ELA 300 mg BID with E2/NETA is consistent with its profile established from the previous Elagolix Uterine Fibroid Phase 3 studies in terms of overall AE rates and most frequent AEs. Safety findings at 24M were similar and consistent to those observed at the end of 12M with no new safety signals identified. Impact Statement: The safety results from this study represent the longest duration of exposure to a GnRH agonist with E2/NETA add-back. Results show that treatment up to 24M with ELA plus E2/NETA is well-tolerated in women with HMB associated with UF. Support: AbbVie Inc. participated in the study design;study research;collection, analysis, and interpretation of data;and writing, reviewing, and approving this for submission. All authors had access to the data;participated in the development, review, and approval of the ;and agreed in the decision to submit this to ASRM for consideration as a poster or oral presentation. AbbVie funded the research for this study and provided writing support for this . No honoraria or payments were made for authorship. Medical writing assistance, funded by AbbVie, was provided by Mayoni Ranasinghe, MBBS, MPH and Kersten Reich, MPH, CMPP, of JB Ashtin. Copyright © 2022

Enhancing Pandemic-Sensitive Recruitment Strategies for a Remote, Randomized Placebo Controlled Trial for Fracture Prevention in Patients with Neurodegenerative Parkinsonisms

Objective: To develop a diversified recruitment model for the ongoing Trial of Parkinson's and Zoledronic Acid (TOPAZ) during the COVID-19 pandemic. Background: TOPAZ is a home-based trial examining the efficacy of zoledronate in preventing fractures in people with neurodegenerative parkinsonisms, who have up to 4-fold increased fracture risk. Design/Methods: Consent is obtained online (https://www.topazstudy.org). After eligibility is determined by movement disorders specialists using medical records and/or telemedicine, study drug is infused by research nurses at home. Fractures are ascertained by email or telephone. The 2/2020 onset of recruitment coincided with COVID-19 restrictions, with a nearly 7 months pause. To randomize 3,500 participants by 12/2023, we developed multiple methods to recruit potential participants via: 1) 46 Parkinson Study Group (PSG) sites, 2) 11 health care systems with integrated research networks, 3) community outreach organizations (i.e. support groups, social media, newsletters, etc.), 4) outreach by the Parkinson's Foundation (PF), 5) Fox Trial Finder (FTF), and 6) the 23andMe Parkinson's disease research program. Results: By 10/1/2021, 2002 had registered on the website, 1333 consented, 992 were eligible per expert diagnostic confirmation, and 632 were randomized, exceeding our goal of 600 for 9/30/21. Registered participants came from the multiple sources: 1) 609 (27.7%) from PSG sites, 2) 529 (24%) from health care systems with integrated research networks, 3) 213 (9.7%) from community outreach, 4) 34 (1.5%) from PF, 5) 16 (0.7%) from FTF and 6) 601 (27.3%) from 23andMe. The largest source of recruitment was PSG. A single study invitation emailed from 23andMe to its 19,733 PD research participants led to nearly the same number of referrals as PSG but in only a few weeks'time. Conclusions: Using diverse referral sources to the TOPAZ study website, we are succeeding in achieving enrollment targets for a Parkinson's trial amidst the challenges of the COVID-19 pandemic.

Why We Need More Data before the Next Pandemic

Attempts to control the current pandemic through public health interventions have been driven by predictions based on modelling, thus bringing epidemiological models to the forefront of policy and public interest. It is almost inevitable that there will be further pandemics and controlling, suppressing and ameliorating their effects will undoubtedly involve the use of models. However, the accuracy and usefulness of models are highly dependent on the data that are used to calibrate and validate them. In this article, we consider the data needed by the two main types of epidemiological modelling (compartmental and agent-based) and the adequacy of the currently available data sources. We conclude that at present the data for epidemiological modelling of pandemics is seriously deficient and we make suggestions about how it would need to be improved. Finally, we argue that it is important to initiate efforts to collect appropriate data for modelling now, rather than waiting for the next pandemic. Copyright © 2021 Nigel Gilbert, Edmund Chattoe-Brown, Christopher Watts, Duncan Robertson

The Large-Scale, Systematic and Iterated Comparison of Agent-Based Policy Models

Vital to the increased rigour (and hence reliability) of Agent-based modelling are various kinds of model comparison. The reproduction of simulations is an essential check that models are as they are described. Here we argue that we need to go further and carry out large-scale, systematic and persistent model comparison—where different models of the same phenomena are compared against standardised data sets and each other. Lessons for this programme can be gained from the Model Intercomparison Projects (MIP) in the Climate Community and elsewhere. The benefits, lessons and particular difficulties of implementing a similar project in social simulation are discussed, before sketching what such a project might look like. It is time we got our act together! © 2022, The Author(s), under exclusive license to Springer Nature Switzerland AG.

Portable Ultraviolet-C Chambers for Inactivation of SARS-CoV-2

The goal of this project was to create and optimize the performance of portable chambers for reliable ultraviolet (UV) disinfection of personal protective equipment (PPE) and enable its safe reuse. During unforeseen times of high demand for PPE, such as during the coronavirus disease 2019 (COVID-19) pandemic, caused by the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), single-use PPE supply can be quickly depleted. UV radiation has been shown to disinfect materials with high efficacy. This paper reports the design and construction of two 280 nm ultraviolet-C (UV-C) disinfection chambers in the form of portable chambers with 46 cm x 46 cm x 46 cm interior dimensions, one using light-emitting diodes and the other using mercury vapor lamps. This paper summarizes and presents a review of SARS-CoV-2 UV deactivation research during 2020 to 2021. Additionally, this paper discusses efforts to increase the uniformity and overall intensity of the UV-C radiation within the chambers through the installation of a UV-reflective, porous polytetrafluoroethylene (PTFE) material. A calculator prototype was additionally designed to calculate the reduction of SARS-CoV-2 as a result of UV-C disinfection, and the prototype code is presented. The paper describes the selection of UV-C radiation sources for the chambers and the chambers' mechanical and electrical design, PTFE installation, testing, and safety considerations.

ARE STATE PUBLIC OPTION HEALTH PLANS WORTH IT?

The COVID-19 pandemic exposed the weaknesses of the U.S. health care system’s reliance on private, employer-based health insurance. The crisis in health care access and affordability has increased support for a public option— the choice to purchase a state-initiated health plan with publicly determined rates. Congressional gridlock, however, may dim the chances for a federal public option. States have stepped into the policy vacuum, proposing forty-nine bills to establish state public options since 2010, including three that became law. This article provides a comprehensive survey and taxonomy of state public option proposals from 2010–2021, identifying three main models: (1) Medicaid Buy-In Public Options;(2) Marketplace-Based Public Options;and (3) Comprehensive Public Options. Though each model serves different policy goals and varies in scope, the defining aim of all public option plans is to improve access to affordable health coverage by applying public payment rates to the private insurance market. We seek to answer whether state public option plans are le-gally viable and “worth it” for states to pursue. The answer is yes to both, but, surprisingly, the degree of legal difficulty is inversely related to the scope of the plan’s reach—the broadest plans have fewer legal hurdles than narrower plans. Moreover, the policy effects increase with the scope of the plan and the robust-ness of the controls on provider payment rates. Public options with modest provider rate controls may have too little impact on affordability and costs, falling short of their defining goal of improving affordability. As a result, the legal and political difficulty of enacting such plans may not be worth it. State public option plans may be most effective when they cover a broad swath of the population and pursue robust provider rate controls. In short, for state public option plans to be worth it, bigger is better. © 2022, Harvard University. All rights reserved.

Current issues in the design and analysis of COVID-19 vaccine efficacy trials

The global COVID-19 pandemic has posed unprecedented challenges to public health, not to mention the global economy and way of life. The pace of vaccine development has been unprecedented and major successes have been realized, with three vaccines having received authorization for use in several countries around the globe following efficacy trials demonstrating high efficacy at preventing COVID-19 disease. However, considerable challenges remain. The immunological predictors of and mechanisms for the efficacy have yet to be defined, and identifying such immune correlates will be critical for bridging the efficacy to populations not included in the original efficacy trials. Additional vaccines also require evaluation to meet global demand. And evaluating the effects of vaccines on onward transmission of infection is imperative to guide policy and optimize vaccine uptake. The talks in this session will pertain to these topics of discovering immunological correlates, designing non-inferiority studies of new candidate COVID-19 vaccines, bridging efficacy of efficacious vaccines to new populations, and designing studies of vaccine efficacy against transmission. This session will include presentations by both academic and industry partners in the field of COVID-19 vaccine development. As leaders in the field, the speakers will articulate the scientific and statistical challenges faced for a given problem, and articulate and illustrate the practical solutions based on ongoing and planned studies. The session chair will work with speakers to ensure that the talks are appropriately sequenced and complementary. Each talk will be 17 min long, followed by 3 min for question and answer, with 10 min discussion at the end of the session. Given the rapid pace with which the COVID-19 field is moving, the topics will be re-evaluated in coming months to ensure that they are relevant and timely. The development and deployment of effective COVID-19 vaccines is currently among the highest public health priorities. The general topics for the talks (immunological correlates of vaccine protection;noninferiority trial design;bridging studies;and transmission studies), are applicable to multiple pathogens, and the statistical problems encountered have features common to clinical trials in other fields.

First Year Comparison of Sickle Cell Pediatric Cohorts from Haiti and Miami (CSHSCD Multicenter Study): Baseline Data

Background: The NIH-sponsored observational study “Comparative Study of Haiti and Miami Cohorts of Sickle Cell Disease CSHSCD” (R01HL149121) coordinates the follow up of children with sickle cell disease (SCD) in Haiti and compares it to a Miami cohort of children of either Haitian or African American ethnicity for the purpose of assessing barriers through questionnaires and examining differences in the care received in their respective environments. Methods: Children less than 6 years of age with SCD are eligible for enrollment in five participating sites: University of Miami (UM, Miami, Florida), Hôpital Saint Damien (HSD, Tabarre, Haiti), Hôpital de l' Université d'Etat d'Haïti (HUEH, Port-au-Prince, Haiti), Hôpital Universitaire Justinien (HUJ, Cap Haitien, Haiti), and Hôpital Sacré Coeur (HSC, Milot, Haiti). Medians and interquartile ranges or percentages were compared at baseline regarding demographics, clinical and growth parameters, laboratory tests, and the children's hydroxyurea (HU) utilization during the first year of enrollment (May 25, 2020-May 24,2021). A Likert-scale barrier questionnaire was distributed at baseline to assess differences in healthcare access. A P value <0.05 was considered statistically significant to establish differences. Results: 130 children were enrolled during the reported period. Significant differences were observed in age, weight percentiles, hemoglobin levels, pain rates, HU treatment, and pneumococcal vaccination. Penicillin prophylaxis was always given by oral route in Miami, but only 39.8% times in Haiti, with 58% of children receiving prophylaxis by intramuscular injection every month and 2.2% (N=2 children) with either unknown or not receiving prophylaxis. Previous medication outsourcing accounted for the oral tablet form in Haiti. Parents in Haiti had more barriers regarding not able to afford treatment (21.5% compared to 8.1% in Miami) and had similar responses regarding not able to afford coming to clinic (21.5% vs. 18.9%). Parents in Miami expressed living far away from clinic (70.2% compared to 25.8% in Haiti), but had more help from other family members (78.4% vs. 33.3%). Interestingly, parents in Miami did not know sometimes what to do when the child was sick (40% respondents vs. 11% in Haiti). There were no major differences between the responses from the African Americans and Haitians living in Miami, except for not knowing sometimes what to do when child is sick (African-Americans having less doubts than Haitians;25% vs. 52.6%). In short-term follow up, no enrolled children died, although two eligible children in Haiti died before enrollment. One child developed COVID-19 in Miami with only mild symptoms, which resolved. Conclusion: At entry children in Haiti are older, weigh less, are more anemic, have more pain episodes, and fewer receive hydroxyurea treatment. Under-vaccination with pneumococcal 13-valent conjugate (Prevnar-13) is notable in Haiti. There were significant differences detected on the barrier questionnaire among respondents in both countries. Acknowledgment: We acknowledge NHLBI for supporting this work. [Formula presented] Disclosures: Alvarez: GBT: Membership on an entity's Board of Directors or advisory committees;Forma Therapeutics: Membership on an entity's Board of Directors or advisory committees.

Comparative Study of Haiti and Miami Cohorts of Sickle Cell Disease (CSHSCD): Methods, Accomplishments, and Implementation

[Formula presented] Background: There are significant limitations in Haiti for the diagnosis and management of sickle cell disease (SCD), including the non-availability of universal newborn screening (NBS) and transcranial Doppler (TCD) ultrasound screening, and the lack of diagnostic laboratory resources, oral penicillin and hydroxyurea (HU). Methods: Beginning in September 2019, CSHSCD (R01HL149121), a 5-year NIH-sponsored observational comparative study of children with SCD from Haitian ethnicity in Miami and in Haiti compared to children of African American ethnicity with SCD, was designed to increase access to care in Haiti. The study aims are 1) to compare the incidence of SCD among newborns from Haitian and African American ethnicity in Miami, 2) to establish NBS programs for hemoglobinopathies in Haiti, and 3) to compare cohorts of children in SCD at the study sites. The participating sites are the University of Miami (UM, Miami, Florida), Hôpital Saint Damien (HSD, Tabarre, Haiti), Hôpital de l'Université d'Etat d'Haïti (HUEH, Port-au-Prince, Haiti), Hôpital Universitaire Justinien (HUJ, Cap Haitien, Haiti), and Hôpital Sacré Coeur (HSC, Milot, Haiti). HUJ and HSC use two NBS screening methods (isoelectric focusing and Sickle SCAN rapid test) and HSD and HUEH use isoelectric focusing only. CSHSCD supplies penicillin and HU and trains TCD examiners to implement stroke risk screening. Data are collected in REDCap. Results: During the first 2 years and despite the COVID-19 pandemic, we established NBS sites with a cohesive network of physicians and nurses trained in the care of children with SCD in Haiti. This capacity building will support sustainability of the program. We successfully identified at least 15 new cases of SCD via newborn screening, trained six TCD examiners, and enrolled 130 children with SCD in follow up, providing them with penicillin prophylaxis and hydroxyurea for severe cases according to local protocols. Implementation activities which have helped are close communications between the investigators, monthly Zoom meetings to coordinate efforts with enrollment updates every month, the availability of rapid tests (Sickle SCAN and Gazelle miniature cellulose acetate electrophoresis) for the diagnosis of SCD, especially when there is no laboratory equipment on site. Implementation challenges we have faced are mostly two. The first is the timely completion of DUNS and SAM registration for the two public hospitals, with one site achieving this after 9 months and the other site taking 18 months to complete. The reasons for the delay are the inability for the UM site to direct these efforts, following strict rules, and the Haitian hospital officers' lack of familiarity with website requirements. We were able to achieve these registrations with the assistance of one Haitian study staff who is very acquainted with internet navigation and became familiarized with requirements. Outsourcing materials to Haiti is another major challenge, with either gaps in the delivery of supplies because of multiple steps involved in ordering and shipping or with delays in releasing equipment once it is at the Port-au-Prince customs, resulting in gaps in NBS in one of the sites for 8 weeks. We have minimized these issues by opening a one-year ticket to order materials from the different companies involved. Also, Haiti's lack of infrastructure, available materials and medications, and political instability limit health care delivery. Conclusion: Since its inception, we have achieved major milestones, including capacity building and implementation of NBS, TCD training, and enrollment of children with SCD into the prospective cohorts despite the current COVID-19 pandemic. Material outsourcing challenges have been the major implementation problem we have faced due to systemic factors. We anticipate that these factors will be corrected or minimized as we have learned how to handle them. These problems were expected as part of conducting an international study in a low-resource setting. Acknowledgment: We acknowledg NHLBI for supporting this work. Disclosures: Alvarez: Forma Therapeutics: Membership on an entity's Board of Directors or advisory committees;GBT: Membership on an entity's Board of Directors or advisory committees. Romano: Genentech: Research Funding;Vycor: Current holder of individual stocks in a privately-held company;NovaVision: Consultancy.