Real-world effectiveness of elexacaftor/tezacaftor/ivacaftor on the burden of illness in adolescents and adults with cystic fibrosis.

Background: Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) has been shown to be safe and efficacious in people with cystic fibrosis (CF) aged ≥2 years. Here, we describe results from an observational study assessing change in burden of illness following initiating ELX/TEZ/IVA in real-world settings. Methods: This US-based, multicenter, observational study used data from electronic medical records to evaluate real-world burden of illness before and after ELX/TEZ/IVA initiation in people with CF aged ≥12 years heterozygous for F508del and a minimal function mutation (F/MF) or an uncharacterized CFTR mutation. Endpoints included absolute change from baseline in percent predicted forced expiratory volume in 1 s (ppFEV1), body mass index (BMI) and BMI-for-age z-score, glycated hemoglobin (HbA1c), and numbers of pulmonary exacerbations (PEx). Results: Overall, 206 people with CF were enrolled (mean [SD] age 22.5 [11.1] years; 192 [93.2%] with F/MF genotype). Mean follow-up was 15.6 (SD, 1.6) months. Improvements in ppFEV1 (7.3 [95% CI: 5.7, 8.8] percentage points) were observed from baseline through follow-up. Increases in BMI (1.40 [95% CI: 1.07, 1.77] kg/m2) and BMI-for-age z-score (0.14 [95% CI: 0.00, 0.28]) were also observed from baseline at 12 months. The estimated annualized rate of any PEx was 1.31 at baseline and 0.61 over follow-up (rate ratio 0.47 [95% CI: 0.39, 0.55]), with annualized rates of PEx requiring antibiotics and hospitalizations of 0.55 and 0.88 in the baseline period and 0.12 and 0.36 over follow-up (rate ratios 0.22 [95% CI: 0.15, 0.31] and 0.41 [95% CI: 0.32, 0.51]), respectively. Absolute change in HbA1c was -0.22 (95% CI: -0.38, -0.06) from baseline through follow-up. Conclusions: ELX/TEZ/IVA treatment was associated with improved lung function, increased BMI, reduced frequency of PEx, and improved (i.e., reduced) HbA1c. These results confirm the broad clinical benefits of ELX/TEZ/IVA seen in clinical trials and show the potential for ELX/TEZ/IVA to improve markers of glucose metabolism.

Prevalence of Lipid Abnormalities and Cholesterol Target Value Attainment in Patients with Stable and Acute Coronary Heart Disease in the United Arab Emirates.

BACKGROUND: Careful management of lipid abnormalities in patients with coronary heart disease (CHD) or an acute coronary syndrome (ACS) can reduce the risk of recurrent cardiovascular events. The extent of hyperlipidemia in these very high-risk patients in the United Arab Emirates (UAE), along with the treatment strategies employed, is not clear. METHODS: The Dyslipidemia International Study II was a multinational observational analysis carried out from 2012 to 2014. Patients were enrolled if they had either stable CHD or an ACS. Patient characteristics, lipid levels, and use of lipid-lowering therapy (LLT) were recorded at enrollment. For the ACS patients, the LLT used during the 4 months' follow-up period was documented, as were any cardiovascular events. RESULTS: A total of 416 patients were recruited from two centers in the UAE, 216 with stable CHD and 200 hospitalized with an ACS. Comorbidities and cardiovascular risk factors were extremely common. A low-density lipoprotein cholesterol level of <70 mg/dl, recommended for patients at very high cardiovascular risk, was attained by 39.3% of the LLT-treated CHD patients and 33.3% of the LLT-treated ACS patients at enrollment. The mean atorvastatin-equivalent daily statin dose was 29 ± 15 mg for the CHD patients, with 13.7% additionally using ezetimibe. For the ACS patients, the daily dosage was 23 ± 13 mg at admission, rising to 39 ± 12 mg by the end of the 4-month follow-up. The use of nonstatin agents was extremely low in this group. CONCLUSIONS: Despite LLT being widely used, hyperlipidemia was found to be prevalent in ACS and CHD patients in the UAE. Treatment strategies need to be significantly improved to reduce the rate of cardiovascular events in these very high-risk patients.

Prevalence of lipid abnormalities and cholesterol target value attainment in patients with stable coronary heart disease or an acute coronary syndrome in Saudi Arabia.

OBJECTIVES: To provide an overview of the extent of hyperlipidemia in very high-risk patients, and how lipid-lowering therapy (LLT) is used in a real-world setting. Methods: In this multicenter observational study, data were collected from LLT-treated patients with stable CHD or an ACS in Saudi Arabia between 2013 and 2014. Individuals were included if they were greater than 18 years and had a full lipid profile available, recorded either prior to the baseline physician visit (CHD patients) or within 24-hours of admission to hospital (ACS patients).  Results: A total of 737 patients were included in the study, 597 with stable CHD and 140 with ACS. Few patients in either group had an LDL-C level of greater than 70 mg/dl, which is advocated for very high-risk patients (24.3% and 11.4%, respectively). The median distances to this value were 19.0 mg/dl (CHD) and 25.0 mg/dl (ACS). Low doses of statins were being utilized (31 and 24 mg/day for CHD and ACS, respectively), with only minimal intensification for the ACS patients after hospital admission (41 mg/day at follow-up). Conclusions: Achievement of recommended LDL-C levels was poor for patients with stable CHD or an ACS. Statin intensity was low, indicating huge scope for intensifying the treatment of these very high-risk patients.

Driving and diabetes mellitus in the Gulf Cooperation Council countries: Call for action.

The aim of the present article is to increase awareness concerning safe driving for patients with diabetes in the Gulf Cooperation Council (GCC) countries and to provide recommendations concerning the management of these patients. The cognitive, motor, and sensory skills required for driving can be adversely affected by diabetes as well as the side effects of anti-diabetic medications, particularly hypoglycemia. The prevalence of diabetes in the GCC countries is among the highest in the world. As the number of diabetic drivers in these countries continues to increase, the number at risk of having a motor vehicle accident is also expected to increase. We reviewed the available literature concerning driving and diabetes, particularly in relation to the current situation in the GGC countries. Unfortunately, very little published information is available addressing this issue in the GCC countries. Most of the GCC countries lack legislation on driving and diabetes. We have proposed recommendations to help diabetic drivers in the GCC countries as well as to provide guidance to health care professionals managing these patients.