Long-Term Efficacy and Safety of Acoramidis in ATTR-CM: Initial Report From the Open-Label Extension of the ATTRibute-CM Trial

Circulation, Ahead of Print. Background: In the phase 3 randomized controlled study, ATTRibute-CM, acoramidis, a transthyretin (TTR) stabilizer, demonstrated significant efficacy on the primary endpoint. Participants with transthyretin amyloid cardiomyopathy (ATTR-CM) who completed ATTRibute-CM were invited to enroll in an open-label extension study (OLE). We report efficacy and safety data of acoramidis in participants who completed ATTRibute-CM and enrolled in the ongoing OLE.Methods: Participants who previously received acoramidis through Month 30 (M30) in ATTRibute-CM continued to receive it (continuous acoramidis), and those who received placebo through M30 were switched to acoramidis (placebo to acoramidis). Participants who received concomitant tafamidis in ATTRibute-CM were required to discontinue it to be eligible to enroll in the OLE. Clinical efficacy outcomes analyzed through Month 42 (M42) included time to event for all-cause mortality (ACM) or first cardiovascular-related hospitalization (CVH), ACM alone, first CVH alone, ACM or recurrent CVH, change from baseline in N-terminal pro-B-type natriuretic peptide (NT-proBNP), 6-minute walk distance (6MWD), serum TTR, and the Kansas City Cardiomyopathy Questionnaire Overall Summary score (KCCQ-OS). Safety outcomes were analyzed through M42.Results: Overall, 438 of 632 participants in ATTRibute-CM completed treatment and 389 enrolled in the ongoing OLE (263 continuous acoramidis, 126 placebo to acoramidis). The hazard ratio (HR) (95% CI) for ACM or first CVH was 0.57 (0.46, 0.72) at M42 based on a stratified Cox proportional hazards model (P-value < 0.0001) favoring continuous acoramidis. Similar analyses were performed on ACM alone and first CVH alone, with HRs (95% CI) of 0.64 (0.47, 0.88) and 0.53 (0.41, 0.69), respectively, at M42. Treatment effects for NT-proBNP and 6MWD also favored continuous acoramidis. Upon initiation of open-label acoramidis in the placebo-to-acoramidis arm there was a prompt increase in serum TTR. Quality of life assessed by KCCQ-OS was well preserved in continuous acoramidis participants compared with the placebo to acoramidis participants. No new clinically important safety issues were identified in this long-term evaluation.Conclusions: Early initiation and continuous use of acoramidis in the ATTRibute-CM study through M42 of the ongoing OLE study was associated with sustained clinical benefits in a contemporary ATTR-CM cohort, with no clinically important safety issues newly identified.

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Novembre 2024

Determinants of underweight among returnee lactating mothers in Gedeb District, Southern Ethiopia: a case-control study

Objective
This study aimed to assess the determinants of underweight among returnees lactating mothers in governmental health facilities of Gedeo zone, Southern Ethiopia.

Design
Case–control study.

Setting
Gedeb District governmental health facilities.

Participants
A total of 264 returnees lactating mothers (88 cases and 176 controls) were randomly selected from four health institutions of Gedeb District.

Outcome measures
Underweight was measured by anthropometric measurement weight/height2. An institutional-based unmatched case–control study was conducted from 11 July 2022 to 20 September 2022. The study involved 264 returnee lactating mothers, with 88 classified as cases (body mass index, BMI

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Novembre 2024

Burden and determinants of scabies in a pastoralist community: a case-control study from Southwest Ethiopia

Background
Scabies is a skin infestation caused by the human itch mite, affecting people globally across all demographics. However, it is more prevalent among children, individuals with poor hygiene, those in overcrowded or slum areas and people affected by drought and war. There is limited research on scabies in Ethiopia and none specifically focused on marginalised communities. This study aimed to assess the burden of scabies and its contributing factors among pastoralist communities in Ethiopia.

Methods
A community-based, unmatched case–control study (1:2 ratio) was conducted in Meinit Goldiya District to assess risk factors for scabies. The researchers collected line-listed data on scabies and conducted face-to-face interviews from January to February 2023, with a sample of 156 cases and 312 controls. Data were gathered using a structured questionnaire and analysed with SPSS V.22. OR, p values and 95% CI were calculated to identify associated factors. Independent factors were determined using a p value of

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Novembre 2024

Pilot study protocol evaluating the impact of telerobotics interactions with autistic children during a Denver intervention on communication skills using single-case experimental design

Introduction
For several years, studies have been conducted on the contribution of social robots as an intervention tool for children with autism spectrum disorder (ASD). One of the early intervention models recommended by the French National Authority for Health is the Early Start Denver Model, an individualised, intensive programme based on play activities chosen by the child. While studies published in recent years suggest that robots provide benefits for autistic children in learning social interactions within a clinical setting, there is no scientific consensus on the widespread contribution and maintenance of their effects over time. On the other hand, a robotic solution controlled directly by a practitioner (ie, on-site telepresence system) enables greater adaptability to children’s responses and choices during interventions. We believe that such a solution would enable better assessment of progress in the fundamental skills of expressive communication and imitation as well as greater engagement during interventions.

Methods and analysis
This is a prospective, monocentric, descriptive and evaluative pilot study based on single-case experimental design (SCED) methodology. The study will recruit eight children diagnosed with ASD aged between 2 and 5 years. The intervention will take place 15 min after the usual weekly care. The SCED methodology is constructed in three stages: (A) 4 weekly sessions at baseline without the robot, (B) 9 weekly sessions with intervention modification using a social robot as cotherapist and (C) 4 weekly sessions without the robot for follow-up.

Ethics and dissemination
Ethical approval was obtained from the South East IV Ethics Committee (CPP Sud-Est IV) (number: 2023-A00895-40) in France. Explicit consent is required from all legal representatives (parents) of children participating in this study. We aim to disseminate the results of this study through national and international conferences, international peer-reviewed journals and social media.

Trial registration number
NCT05991791.

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Novembre 2024

Prevalence of comorbidities among patients with rheumatoid arthritis in the UAE: a case-control study

Objectives
Data on the rate of comorbidities in Arab patients with rheumatoid arthritis (RA) are limited, and extrapolating the prevalence of comorbidities from international studies is challenging. This study aimed to investigate the prevalence of comorbidities in patients with RA, compare it with that in non-RA controls and explore the association between the body mass index of patients with RA and comorbidities.

Design
This is a retrospective, case–control study.

Setting
This study included patients receiving secondary care at the Rheumatology Department of a public hospital in the Emirate of Dubai. The controls were recruited from patients receiving primary and secondary care at the Dubai Academic Health Corporation in the fourth quarter of 2022.

Participants
This study included all consecutive UAE national patients with RA who visited the rheumatology clinic. The study included 1756 participants in an age-matched and sex-matched control group and 439 patients with RA. Of these, 88.6% were female, and the median age was 55 years. Each RA case was randomly matched with four controls of the same age and sex. All relevant information, including case and control demographics and comorbidities, was retrieved from the electronic medical record.

Primary and secondary outcome measures
The relative risk of comorbidities was compared between patients with RA and age-matched controls. The relationship between obesity in RA and the frequency of comorbidities was determined.

Results
This study revealed that 188 (42.8%) patients with RA had at least one comorbidity, whereas only 636 (36.2%) individuals in the control group had at least one comorbidity (OR 1.3; 95% CI 1.1 to 1.6, p

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Novembre 2024