A systematic review and meta-analysis of the efficacy and safety of Mavacamten therapy in international cohort of 524 patients with hypertrophic cardiomyopathy.

Hypertrophic cardiomyopathy (HCM) is the most common heritable myocardial disorder worldwide. Current pharmacological treatment options are limited. Mavacamten, a first-in-class cardiac myosin inhibitor, targets the main underlying pathology of HCM. We conducted a systematic review and meta-analysis to evaluate the efficacy and safety of Mavacamten in patients with HCM. PRISMA flow chart was utilized using PubMed, SCOPUS, and Cochrane databases for all up-to-date studies using pre-defined keywords. Pre-specified efficacy outcomes comprised several parameters, including an improvement in peak oxygen consumption (pVO2) and ≥ 1 NYHA class, the need for septal reduction therapy (SRT), change from baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ), changes in biochemical markers and LVEF, along with peak left ventricular outflow tract gradient at rest and after Valsalva maneuver. Safety outcomes included morbidity and serious adverse events. This systematic review included five studies, four RCTs and one non-randomized control trial comprised a total of 524 (Mavacamten [273, 54.3%] vs placebo [230, 45.7%] adult (≥ 18 years) patients with a mean age of 56 years. The study. comprised patients with Caucasian and Chinese ethnicity and patients with obstructive (oHCM) and non-obstructive (nHCM) HCM. Most baseline characteristics were similar between the treatment and placebo groups. Mavacamten showed a statistically significant increase in the frequency of the primary composite endpoint (RR = 1.92, 95% CI [1.28, 2.88]), ≥ 1 NYHA class improvement (RR = 2.10, 95% CI [1.66, 2.67]), a significant decrease in LVEF, peak left ventricular outflow tract gradient at rest and after Valsalva maneuver. Mavacamten also showed a significant reduction in SRT rates (RR = 0.29, 95% CI [0.21, 0.40], p < 0.00001), KCCQ clinical summary scores (MD = 8.08, 95% CI [4.80, 11.37], P < 0.00001) troponin levels and N-terminal pro-B-type natriuretic peptide levels. However, there was no statistically significant difference between Mavacamten and placebo regarding the change from baseline peak oxygen consumption. Mavacamten use resulted in a small increase in adverse events but no statistically significant increment in serious adverse events. Our study showed that Mavacamten is a safe and effective treatment option for Caucasian and Chinese patients with HCM on the short-term. Further research is needed to explore the long-term safety and efficacy of Mavacamten with HCM. In addition, adequately powered studies including patients with nHCM is needed to ascertain befits of Mavacamten in those patients.

Preoperative training education with incentive spirometry may reduce postoperative pulmonary complications.

OBJECTIVE: To assess whether preoperative incentive spirometer training would influence the development of postoperative pulmonary complications after lung resection. METHODS: Sixty-two lung resection patients were prospectively investigated; 17 were given an incentive spirometer preoperatively and 45 did not have an incentive spirometer preoperatively. Postoperatively, both arms exercised with an incentive spirometer. The number of repetitions per day, balls raised per repetition, correct technique of exercising, and postoperative pulmonary complications were compared between the 2 groups. Univariate binary logistic regression analysis of potential predictors of postoperative pulmonary complications led to multivariate analysis of independent predictors. Receiver operating characteristic analysis established the cutoff points of predictors. RESULTS: The group with no preoperative incentive spirometer developed more postoperative pulmonary complications than the preoperative incentive spirometer group (24.4% vs. 5.9%, respectively, p = 0.045). The preoperative incentive spirometer arm achieved more repetitions per day, balls per repetition, and correct incentive spirometer technique (p = 0.002, p < 0.001, p = 0.034, respectively). Balls raised per repetition and repetitions per day postoperatively were identified as independent predictors of postoperative pulmonary complications (p = 0.032 and p = 0.021, respectively). Less than 5 repetitions per day (sensitivity 93%, specificity 77%, p < 0.001) and less than 2 balls per repetition (sensitivity 93%, specificity 77%, p < 0.001) were predictive of postoperative pulmonary complications. CONCLUSION: Preoperative incentive spirometer exposure ensured better compliance with postoperative treatment and a more accurate technique (balls raised per repetition, repetitions per day). These variables correlated with a lower postoperative pulmonary complication rate.

Pathological fracture of non-ossifying fibroma associated with neurofibromatosis type 1.

We report the management of a pathological fracture through a proximal tibial non-ossifying fibroma (NOF) in a 13-year-old girl with neurofibromatosis type 1 (NF1). The fracture was minimally displaced, and the lesion had clinical features of a NOF, and therefore biopsy was not required. Operative fixation has been the preferred method of treatment for pathological fractures through NOF associated with NF1. Multiple NOFs associated with NF1 are rare but can coalesce resulting in large lesions with an increased risk of pathological fracture. In cases which permit, non-operative treatment with cast immobilisation can yield satisfactory results.

Association Between Abdominal Visceral Artery Calcification and All-Cause Mortality-A Computerized Tomography Imaging-Based Longitudinal Follow-Up Study.

Arterial calcification in different arterial beds has been observed to be an independent predictor of mortality. The association of abdominal visceral artery calcium with all-cause mortality remains unexplored. Patients who had undergone contrast-enhanced computerized tomography (CT) imaging for routine assessment of peripheral arterial disease (PAD) were considered for this study. A novel calcium score (abdominal visceral arteries calcium [AVAC]) for the abdominal visceral arteries (celiac axis, superior mesenteric, and renal arteries) was calculated using a modified Agatston score. Cumulative AVAC was defined as sum total of the calcium score of above individual arteries. The primary outcome was all-cause mortality. The association of AVAC with all-cause mortality was assessed. Of the 134 consecutive patients, 89 were included for analysis. Median follow-up duration was 72 (47-91) months since CT imaging; 35 (39%) patients died during this period. Hypertension and cumulative AVAC score had a significant association with all-cause mortality (P < .05). Cumulative visceral abdominal artery calcification is associated with all-cause mortality in patients with PAD. Future prospective studies are warranted to investigate this relationship in PAD and other patient cohorts.

Development of an Arabic version of the National Eye Institute Visual Function Questionnaire as a tool to study eye diseases patients in Egypt.

AIM: To develop and test an Arabic version of the National Eye Institute Visual Function Questionnaire-25 (NEI-VFQ-25). METHODS: NEI-VFQ-25 was translated into Arabic according to WHO translation guidelines. We enrolled adult consenting patients with bilateral chronic eye diseases who presented to 14 hospitals across Egypt from October to December 2012, and documented their clinical findings. Psychometric properties were then tested using STATA. RESULTS: We recruited 379 patients, whose mean age was (54.5±15)y. Of 46.2% were males, 227 had cataract, 31 had glaucoma, 23 had retinal detachment, 37 had diabetic retinopathy, and 61 had miscellaneous visual defects. Non-response rate and the floor and ceiling numbers of the Arabic version (ARB-VFQ-25) were calculated. Internal consistency was high in all subscales (except general health), with Cronbach-α ranging from 0.702-0.911. Test-retest reliability was high (intraclass correlation coefficient 0.79). CONCLUSION: ARB-VFQ-25 is a reliable and valid tool for assessing visual functions of Arabic speaking patients. However, some questions had high non-response rates and should be substituted by available alternatives. Our results support the importance of including self-reported visual functions as part of routine ophthalmologic examination.