Safety and Efficacy of Metabolic Modulation With Ninerafaxstat in Patients With Nonobstructive Hypertrophic Cardiomyopathy.

BACKGROUND: In nonobstructive hypertrophic cardiomyopathy (nHCM), there are no approved medical therapies. Impaired myocardial energetics is a potential cause of symptoms and exercise limitation. Ninerafaxstat, a novel cardiac mitotrope, enhances cardiac energetics. OBJECTIVES: This study sought to evaluate the safety and efficacy of ninerafaxstat in nHCM. METHODS: Patients with hypertrophic cardiomyopathy and left ventricular outflow tract gradient <30 mm Hg, ejection fraction ≥50%, and peak oxygen consumption <80% predicted were randomized to ninerafaxstat 200 mg twice daily or placebo (1:1) for 12 weeks. The primary endpoint was safety and tolerability, with efficacy outcomes also assessed as secondary endpoints. RESULTS: A total of 67 patients with nHCM were enrolled at 12 centers (57 ± 11.8 years of age; 55% women). Serious adverse events occurred in 11.8% (n = 4 of 34) in the ninerafaxstat group and 6.1% (n = 2 of 33) of patients in the placebo group. From baseline to 12 weeks, ninerafaxstat was associated with significantly better VE/Vco2 (ventilatory efficiency) slope compared with placebo with a least-squares (LS) mean difference between the groups of -2.1 (95% CI: -3.6 to -0.6; P = 0.006), with no significant difference in peak VO2 (P = 0.90). The Kansas City Cardiomyopathy Questionnaire Clinical Summary Score was directionally, though not significantly, improved with ninerafaxstat vs placebo (LS mean 3.2; 95% CI: -2.9 to 9.2; P = 0.30); however, it was statistically significant when analyzed post hoc in the 35 patients with baseline Kansas City Cardiomyopathy Questionnaire Clinical Summary Score ≤80 (LS mean 9.4; 95% CI: 0.3-18.5; P = 0.04). CONCLUSIONS: In symptomatic nHCM, novel drug therapy targeting myocardial energetics was safe and well tolerated and associated with better exercise performance and health status among those most symptomatically limited. The findings support assessing ninerafaxstat in a phase 3 study.

Factors associated with prolonged cardiopulmonary resuscitation attempts in out-of-hospital cardiac arrest patients presenting to the emergency department.

BACKGROUND: There is high variability among clinicians' decision of appropriate cardiopulmonary resuscitation (CPR) duration before deciding for termination of resuscitation. This study attempted to investigate factors associated with the decision to prolong resuscitation attempts in cardiac arrest patients treated in an emergencydepartment (ED). METHODS: A retrospective study that evaluated two years of mortality registry starting in 2015 was conducted in the ED of University Kebangsaan Malaysia Medical Centre (UKMMC), Kuala Lumpur, Malaysia. Adult out-hospital cardiac arrest (OHCA) patients treated in the ED were included. Multivariate logistic regression analysis was utilized for the exploration of factors associated with prolonged CPR attempts (> 30min). RESULTS: The median CPR duration was 24min (range 2-68min). Four variables were independently associated with prolonged CPR attempts: younger age (OR, 0.97; 95% CI, 0.95-0.99; p<0.001), pre-existing heart disease (OR, 1.97; 95% CI, 1.07-3.65; p=0.031), occurrence of transient return of spontaneous circulation (ROSC) (OR, 2.38; 95% CI, 1.05-5.36; p=0.037), and access to the ED by nonemergency medical services (EMS) transport (OR, 1.92; 95% CI, 1.09-3.37; p=0.024). CONCLUSION: Patient-related and access-related factors were associated with prolonged CPR attempts among OHCA patients resuscitated in the ED.

A Malaysian Medical Non-Governmental Organization's (NGO) Experience in the Emergency Response for COVID-19, Using the Whole-of-Society Collaborative Concept.

Non-governmental organizations (NGOs) are one of the important players during a pandemic, including the Islamic Medical Association of Malaysia (IMAM) Response and Relief Team (IMARET). During the coronavirus disease (COVID-19) pandemic, IMARET played a key role in assisting health relief efforts in Malaysia. We are sharing this experience as a medical NGO's response to the pandemic. This report presents data from the March 18 to June 10, 2020, retrieved from IMARET's database with approval from the Executive Committee and the IMARET COVID-19 Task Force. We report that IMARET's task force consists of 30 people, mostly medical doctors. Supplies distributed included personal protective equipment with other medical equipment, such as portable ultrasounds and ventilators. IMARET engaged with 33 collaborators and 92 partners and funders. There were 135 volunteers with the majority being medical volunteers. IMARET raised more than RM $3 million (US $740 000) garnering support from over 40 000 donors in 85 days. In conclusion, NGOs play a significant role that effectively enhance and complement the consolidated works by the authorities and public in the effort to overcome COVID-19 challenges.

Pneumatosis intestinalis: An important radiological clue in a case of missed perforated appendicitis.

Pneumatosis intestinalis (PI) is a rare radiological finding, characterized by the presence of gas in the bowel wall. It has a wide spectrum of possible underlying diagnosis ranging from benign to life-threatening conditions. We present a case of a previously healthy male who was initially presented with missed diagnosis of acute appendicitis which had led to perforated appendix. Failure to recognize the significance of PI and its correlation with other clinical information had caused the delay in his surgical intervention. Fortunately, the patient made a steady recovery and was discharged well. It is important to have a high clinical suspicion of life-threatening conditions whenever patients presented with a radiological clue of PI. Failure to incorporate this finding with other clinical clues might lead to devastating consequences and delay the necessary treatment.

Knowledge and Confidence Level Among Emergency Healthcare Workers in Airway Management and Resuscitation of Suspected COVID-19 Patients: A Cross Sectional Study in Malaysia.

INTRODUCTION: This study aims to evaluate the knowledge and confidence of emergency healthcare workers (EHCW) in facing the COVID-19 pandemic. MATERIALS AND METHODS: A cross-sectional online study using a validated questionnaire was distributed to doctors (MD), assistant medical officers (AMO), and staff nurses (SN) at an urban tertiary Emergency Department. It comprised of 40 knowledge and 10 confidence-level questions related to resuscitation and airway management steps. RESULTS: A total of 135 from 167 eligible EHCW were enrolled. 68.9% (n = 93) had high knowledge while 53.3% (n = 72) possessed high confidence level. Overall knowledge mean score was 32.96/40 (SD = 3.63) between MD (33.88±3.09), AMO (32.28±4.03), and SN (32.00±3.60), P= 0.025. EHCWs with a length of service (LOS) between 4-10 years had the highest knowledge compared to those with LOS <4-year (33.71±3.39 versus 31.21±3.19 P = 0.002). Airway-related knowledge was significantly different between the designations and LOS (P = 0.002 and P = 0.003, respectively). Overall, EHCW confidence level against LOS showed significant difference [F (2, 132) = 5.46, P = 0.005] with longer LOS showing better confidence. MD showed the highest confidence compared to AMO and SN (3.67±0.69, 3.53±0.68, 3.26±0.64) P = 0.049. The majority EHCW were confident in performing high-quality chest-compression, and handling of Personal Protective Equipment but less than half were confident in resuscitating, leading the resuscitation, managing the airway or being successful in first intubation attempt. CONCLUSIONS: EHCW possessed good knowledge in airway and resuscitation of COVID-19 patients, but differed between designations and LOS. A longer LOS was associated with better confidence, but there were some aspects in airway management and resuscitation that needed improvement.