Background: Eliminating maternal and perinatal morbidity and mortality are global health priorities. Cameroon has some of the highest levels of maternal and neonatal mortality in the world. Evidence-based maternal healthcare is vital to ensuring women's and infants' health; however, offering such services in low-income countries is often challenging. Mobile health interventions have the potential to overcome longstanding public health barriers to fill gaps in maternal and infant care. This is among the first qualitative studies in Cameroon that examines individual and interpersonal barriers to maternity care to inform mHealth intervention adaptation. Methods: We conducted qualitative in-depth interviews (n=38) and three focus groups (n=18) with key stakeholders including previously pregnant women who experienced an adverse event, currently pregnant women, maternity care providers, healthcare administrators, and Ministry of Health representatives. Thematic analysis was performed using NVivo12 software. Themes were categorized on the individual and interpersonal levels. The current study is part of a larger, long-term implementation project to adapt MIST to the local contexts of Cameroon using mobile phones. Results: A total of 56 respondents, 14 men and 42 women, participated in this qualitative research. Participants reported individual-level barriers for women's timely and appropriate maternity care related to antenatal care initiation, antenatal care utilization, health literacy, and blind trust in providers. Participants suggested that women waited to initiate antenatal care, underutilized antenatal care, lacked perinatal health literacy, and trusted their providers, regardless of health outcomes and complications. At the interpersonal level, husbands, religious leaders, and family were identified by participants as influencing women's perinatal healthcare decisions-making, both positively and negatively. Conclusion: Our data reveal individual and interpersonal forces that affect maternal and perinatal care in Cameroon. Future public health efforts can focus on improving health literacy during pregnancy and reproductive years among women as well as in their close social networks.

Fabry disease (FD) is an X-linked lysosomal storage disease that results in the accumulation of glycosphingolipids, such as globotriaosylceramide (Gb3) in a variety of cells. FD most prominently involves cardiac, nervous, and renal tissue, with cardiac complications representing the most common cause of death. Fabry disease has a prevalence ranging between 1:8454 to 1:117,000 among men. The higher prevalence included patients with the A143T mutation, which was shown to be a non-disease causing variant. Due to its rarity, and wide array of phenotypic presentations, especially in women, FD is often misdiagnosed. Advances in echocardiographic techniques and magnetic resonance imaging can play a crucial role in raising suspicion for Fabry disease and identifying early Fabry cardiomyopathy. Identification of end-organ involvement can, in turn, permit treatment initiation in patients who did not previously qualify for advanced therapies and in screened family members who are still too early in the disease process to manifest specific symptoms.

In Fabry disease (FD, OMIM #301500), a rare lysosomal storage disorder, the glucosylceramide synthase inhibitor lucerastat acts as substrate reduction therapy. The Phase 3, prospective, double-blind, placebo-controlled, 6-month, randomized clinical trial, MODIFY (NCT03425539), aimed to evaluate the efficacy, safety, and tolerability of lucerastat in adults with FD with moderate-to-severe neuropathic pain. The single-arm, open-label extension (OLE) (NCT03737214) evaluated the longer-term safety and tolerability of lucerastat over 72 months. Lucerastat 1000 mg twice daily (n = 80), compared with placebo (n = 37), failed to affect neuropathic pain at Month-6, with no significant difference between treatment groups (LSM difference –0.42 [95

Enzyme replacement therapies (ERTs) approved for Fabry disease require infusions every 2 weeks (E2W). Pegunigalsidase alfa, a PEGylated ERT with a prolonged half-life vs. other ERTs, may allow extension of the dosing interval to every 4 weeks (E4W). BRIGHT F51 (NCT03614234) is an ongoing phase III, open-label extension study evaluating long-term efficacy and safety of pegunigalsidase alfa 2 mg/kg E4W in adults with Fabry disease previously treated with agalsidase alfa or beta E2W for ≥ 3 years who completed one year of pegunigalsidase alfa treatment in the BRIGHT study. This interim analysis reports results following 3–5 years of treatment (cutoff date December 31, 2022). Twenty-nine patients were enrolled. Median (interquartile range [IQR]) annualized eGFR slope during treatment was ‒2.2 (‒2.9; ‒1.1) mL/min/1.73 m2/year (males: ‒2.4 [‒2.9; ‒1.0, n = 23]; females: ‒1.8 [‒2.4; ‒1.3, n = 6]; anti-drug antibody [ADA]-positive: ‒2.6 [‒4.0; ‒1.7, n = 9 all male]; ADA-negative: ‒1.8 [‒2.7; ‒0.6, n = 20]). Median (IQR) change in plasma lyso-Gb3 from baseline to Week 208 was 3.2 (‒3.9; 8.5, n = 17) nM in males; concentrations remained low and stable in females. Overall, 51/477 treatment-emergent adverse events in 13 patients (45

BACKGROUND:Remote blood pressure (BP) monitoring has emerged as a promising solution to overcome barriers to hypertension care access, yet validation of devices is lacking. METHODS:We conducted the validation of the Ideal Life BP Manager BP device, a remote BP monitoring device, using the AAMI/ESH/ISO protocol. From June 2021 to May 2022, participants were screened and systolic BP (SBP) and diastolic BP (DBP) measurements were conducted by trained staff using the Ideal Life BP Manager and a mercury sphygmomanometer. Statistical analyses were performed to assess agreement of SBP and DBP. RESULTS:Of the 90 individuals screened, 79 were included, with a mean age of 50.9 years (standard deviation [SD] 15.6) and the majority being female (68.8%). The mean ± SD differences between the Ideal Life BP Manager and mercury sphygmomanometer measurements were 2.20 ± 7.37 mm Hg for SBP and 1.49 ± 7.31 mm Hg for DBP, satisfying AAMI/ESH/ISO protocol criterion 1. Criterion 2 was also met, with participant-level standard deviations of the mean paired differences (6.54 mm Hg for SBP, 6.30 mm Hg for DBP) falling below the protocol-specified thresholds. Bland-Altman plots demonstrated limits of agreement of -12.0 to 16.9 mm Hg for systolic blood pressure and -12.8 to 15.9 mm Hg for diastolic blood pressure. CONCLUSIONS:The Ideal Life BP Manager met the AAMI/ESH/ISO validation criteria.

Background: Fabry disease (FD) is a rare, progressive disorder caused by pathogenic variants of the GLA gene resulting in the accumulation of toxic metabolites. Pain is a hallmark of FD, and patients often present with heterogeneous pain profiles. This cross-sectional, web-based survey was conducted to characterize pain and pain crises in patients with FD in the United States and explore the effects of sex, disease phenotypes, and treatment on pain. Results: A total of 66 participants (mean [±SD] age: 44.0 [±12.7] years; females: 59.1%) completed the survey. Participants reported experiencing pain in upper (34.8%) and lower (43.9%) extremities several times a day and abdominal pain (31.8%) a few times a week. Overall, participants reported the nature of their pain as triggered (upper extremities: 47.0%; abdomen: 51.5%) or sudden (lower extremities: 57.6%). Female participants reported experiencing pain in upper (46.2%) and lower (48.7%) extremities several times a day and described it as sudden or triggered (48.7%) in upper extremities and sudden (61.5%) in lower extremities. Pain crises were reported in the lower extremities (80.0%), followed by the upper extremities (66.7%) and the abdomen (51.1%), and were often characterized as burning, tingling, or stabbing. A higher proportion of female participants (84.6%) than that of male participants (73.7%) reported pain crises in lower extremities. The duration of pain crises varied from 30 minutes to several days for different subgroups depending on sex and FD phenotypes. Most participants (81.0%) reported symptom improvement after 12 months of FD-specific treatment. With agalsidase beta as the most recent medication, participants reported improvement in neuropathic symptoms (burning in hands, 45.9%), with an overall mean (±SD) satisfaction score of 7.2 (±1.7). Conclusions: Pain was largely reported to be triggered across all subgroups. Consistent pain profiles were noted in participants across sex and FD phenotypes. Female participants reported pain burden similar to that of male participants, and pain crisis experience was heterogeneous across the subgroups. Most participants reported improvement in symptoms after FD-specific treatment and a high treatment satisfaction score with agalsidase beta.

BACKGROUND:In the U.S. Deep South, Black adults experience disproportionate rates of type 2 diabetes (T2D) and associated complications, driven in part by adverse social determinants of health (SDoH). Addressing these disparities requires multilevel interventions that can be optimized for both effectiveness and cost. The Food Delivery, Remote Monitoring, & Coaching-Enhanced Education for Optimized Diabetes Management (FREEDOM) trial aims to identify an optimized, scalable intervention package that improves glycemic control among Black adults with T2D. METHODS:FREEDOM is a multicenter, hybrid type 1 optimization-implementation trial uses a 2 × 2 × 2 factorial design to test three intervention components-digital health coaching, food box delivery, and remote patient monitoring (RPM)-among 304 adults recruited from three health systems in Alabama and Mississippi. Interventions are delivered over six months with follow-up assessments through 12 months. The primary clinical outcome is change in HbA1c at 12 months. Secondary outcomes include within-trial cost-utility using net monetary benefit, RE-AIM outcomes, and CFIR-guided qualitative assessment of contextual determinants. Mixed methods will evaluate fidelity and context. Optimization will be determined using the net monetary benefit framework based on quality-adjusted life years (QALYs). DISCUSSION:This protocol describes the design and methods of the FREEDOM trial, which seeks to address key gaps in optimizing multilevel interventions for adults with T2D in underserved regions of the Deep South. Findings will guide the selection of scalable, cost-effective intervention strategies to improve glycemic control among adults with T2D. REGISTRATION:ClinicalTrials.gov identifier: NCT05288452; first posted December 29, 2022.