Alpha-mannosidosis is an inherited rare disorder of mannose-containing oligosaccharides metabolism that is currently treated by enzyme replacement therapy (ERT), bone marrow transplantation (BMT), or ...supportive therapy (ST). However, the relative cost-effectiveness of these treatment options is yet unknown. Our study aimed to compare the cost-effectiveness of the treatment options for mild to moderate alpha-mannosidosis. The study is based on a modeling approach using a Discrete-Event Simulation model to generate and simulate the course of the disease under the influence of each of the treatment options: ERT, BMT, and ST. The model had a lifetime horizon and was made from the perspective of the Serbian Health Insurance Fund. Currently, available causal therapy of mild to moderate alpha-mannosidosis with velmanase alpha enzyme replacement is not cost-effective compared with supportive therapy (ICER = 941,587,152 RSD) or bone marrow transplantation (ICER = −398,412,755 RSD). Bone marrow transplantation can be cost-effective compared to supportive therapy (ICER = 6,032,689 RSD), but only if the willingness-to-pay threshold is increased to 9 gross domestic products (GDP) per capita per QALY gained. According to the current threshold, velmanase-alfa is not cost-effective compared to BMT or ST. To make alfa-mannosidosis therapy widely accessible to patients, criteria for assessing the cost-effectiveness of orphan drugs must include not only the absolute value of ICER but other aspects like equity weightings of QALYs, risk-sharing, reimbursement of severe forms of a disease only, or availability of dedicated funding.
Aim
To evaluate the efficacy (rate of recanalization) of therapy with novel oral anticoagulants (NOAC; rivaroxaban, apixaban)
compared to conventional treatment (low molecular weight heparin - LMWH ...and vitamin K antagonist) in the treatment of deep vein thrombosis (DVT) of the proximal segments of lower extremities.
Methods
The first group consisted of patients diagnosed with DVT and treated with NOAC (n = 100), while the second group consisted of patients diagnosed with DVT, who were treated by conventional treatment (low molecular weight heparin and vitamin K antagonists) (n = 100). In the first group, NOAC was included in the initial treatment. Patients in the second group were treated with LMWH for four days, and on the fifth day vitamin K antagonist was included in therapy, international ratio (INR) was titrated to therapeutic values (2.0-3.0), and then low molecular weight heparin was excluded from the therapy.
Results
There was a statistically significant difference in the estimated values of free lumen of the blood vessel between the examined groups after 30 days (p=0.0001), after 90 days (p=0.0001) and after 180 days (p=0.0001). After 180 days, the average free lumen values in the NOAC group were 85% (81-89%), which was significantly higher than the free lumen values in the second group, 73% (69-79%).
Conclusion
The use of NOAC represents more efficient treatment of DVT comparing to vitamin K antagonists.
HIV infection is characterized by progressive depletion of CD4
T cells due to their reduced synthesis and increased destruction followed by marked activation and expansion of CD8
T lymphocytes. ...CD4/CD8 ratio was traditionally described as a marker of immune system ageing in the general population, but it increasingly appears as a marker of different outcomes in the HIV-infected population. The main objective of this study is to examine the power of CD4/CD8 ratio in predicting the occurrence of metabolic syndrome (MetS) in HIV-positive patients receiving cART therapy.
80 HIV/AIDS subjects were included in a retrospective case-control study. Flow cytometry was used to determine the percentage of CD4
and CD8
cells in peripheral blood of these patients. The values of biochemical parameters (triglycerides, HDL, blood sugar, blood counts), immunological parameters (CD4/CD8, PCR), anthropometric measurements and type of cART therapy were evaluated in this study.
After six months of cART therapy 19 (23.8%) subjects had all the elements necessary for making the diagnosis of MetS. Using multivariate analysis CD4/CD8 ratio was statistically significant (p < 0.05) and had the largest effect on development of MetS (Wald = 9.01; OR = 0.45), followed by cART (Wald = 7.87; OR = 0.10) and triglycerides (Wald = 5.27; OR = 1.7). On the other hand, body weight and waist circumference showed no statistically significant effect on the development of MetS after six months of cART, p > 0.05.
CD4/CD8 ratio proved to be a significant marker for prediction of metabolic syndrome in HIV/AIDS patients.
IntroductionLevosimendan as a new generation vasoactive coronary and peripheral dilatation drug, has been recently developed for cardiac decompensation treatment.AimTo evaluate the effects on cardiac ...function during treatment with Levosimendan in cardiac decompensated severely ill infants and children.MethodsRetrospective-prospective study was performed in Paediatric Clinic CC University of Sarajevo, from 01.01.2013. till 30.12.2014. Study included 7 pts; with congenital/post surgery Tetralogia Fallot/ and acquired heart diseases/dilated cardiomyopathy-myocarditis/pts. Patients were divided into two groups: I Group pre treatment and II Group, post Levosimendan treatment. Parameters evaluated were: age, sex, diagnosis, left ventricle EF /LV EF/pre and post-treatment (EF1 and EF2/and use of inotropic drugs.ResultsThe evaluated variables were: age in I Group of pts /4/ with congenital heart disease: mean 7.5 months/ratio: 6.5–8.5 months of age/and in second with acquired heart diseases /3/, mean: 8.5 years of age/3–14 years/, 3 boys; inotropic drugs used in both Groups of pts: Noradrenalin, Dobutamin, Dopamine and Corotrop which were decreased and stopped in the First Group 2nd day post Levosimendan start and in second Group, 3rd day stopped post Levosimendan start. LV EF evaluated in both Groups; difference of mean EF1 in relation to mean EF2 values, showed statistically significant improvement of LV function /p=0,002/ and decrease in inotropic support.ConclusionLevosimendan has positive effects on myocardial function restoration regardless on congenital or acquired heart diseases in paediatric population as well as in decrease in inotropic support and rapid myocardial cell recovery.
Introduction: Adequate prenatal and postnatal care for preterm neonates not only affects the survival rate, but also the occurrence of chronic diseases, and in the future also affects the quality of ...life of that children.
Aim: To examine the influence of independent predictors (weeks of gestation, body weight, sex) on the outcome of the disease and to analyze the influence of the applied ventilatory mode on the final outcome of treatment.
Material and methods: The study included neonates (n = 248) born prematurely who were treated in the neonatal intensive care unit for a period of one year due to immaturity-related difficulties.
Results: The mean age of male neonates (n = 119) at birth was 31.13 ± 3.3 weeks of gestation (WG), and females (n = 129) 31.59 ± 3.2 WG. Weeks of gestation have a statistically significant effect on survival (p = 0.0001), for each more week of gestation, the chances of survival increase by 21%. There was no significant difference between birth weight and sex (p = 0.289), and the birth weight of the neonates had a statistically significant effect on survival (p = 0.0001). For every 10 grams of body weight, in our sample, the chance of survival increases by 2%. Ventilation mode showed a statistically significant effect on neonatal survival (p < 0.05), and intubation mode was used as an indicator. If neonates are switched from non-invasive to invasive ventilation mode, the chance of survival in our sample is reduced by 88%.
Conclusion: Weeks of gestation, birth weight, and the use of a noninvasive mode of ventilation are predictors of a positive outcome for preterm neonates.
Translations of instruments for measuring quality of life developed in certain, mostly more developed, parts of the world usually do not cover regionally specific aspects of health-related quality of ...life, even after transcultural validation. The aim of this study was to develop and validate a reliable questionnaire in Serbian, Croatian, Bosnian, and Montenegrin languages suitable for measuring health-related quality of life in adults.
The study was of a cross-sectional type, assessing the reliability and validity of a newly developed questionnaire for measuring health-related quality of life (HRQoL) in adults residing in western Balkan states (WB-HRQoL). It was conducted on a sample of 489 adults from Serbia, Croatia, Bosnia & Herzegovina, and Montenegro, with a mean age of 52.2±14.4 years and a male/female ratio of 195/294 (39.9%/60.1%).
The definitive version of the WB-HRQoL scale with 19 items showed very good reliability, with Cronbach's alpha 0.905. The scale was temporally stable, and satisfactory results were obtained for divergent and convergent validity tests. Exploratory factorial analysis brought to the surface four domains of health-related quality of life, namely the physical, psychical, social, and environmental.
The WB-HRQoL scale is a reliable and valid generic instrument for measuring HRQoL that takes into account the cultural specifics of the western Balkan region.
Introduction: A tricuspid annular plane systolic excursion (TAPSE) reflects longitudinal myocardial shortening and represents an echocardiographic parameter to assess right ventricular systolic ...function. Aim: To determine relationship between TAPSE and gestational age and body weight in neonates, establishing method for prediction the normal TAPSE values in neonates based on gestational age and body weight. Patients and methods: The prospective study group consisted of 97 neonates from 24 to 40 weeks of gestation, with a weight of 625-4,340 g and normal echocardiographic results with determination of TAPSE based on gestational age and body weight. Results: The TAPSE range was 6.45-9.80 mm (with average value 8.07 ± 1.89 mm) in male and 6.95-8.50 mm (with average value 7.9 ± 1.86 mm) in female babies, depending on gestational age. There was no statistically significant difference of normal TAPSE values between female and male patients (p = 0.586). TAPSE is affected by increasing birth weight and increasing gestational age. The TAPSE values have had strong and positive correlation with gestational age (p = 0.0001, rho = 0.692) as well with body weight (p = 0.0001, rho = 0.786). Regression equation relating body weight and TAPSE is: TAPSE predicted = 4.738 + (body weight * 0.002); equation relating gestational age and TAPSE is: TAPSE predicted = -4.163 + (gestational weeks * 0.385). Conclusions: It is possible to adequately predict TAPSE based on gestational age. As TAPSE is easy to measure and highly reproducible, we consider it a useful quantitative parameter to assess right ventricular longitudinal function in premature baby.
Juvenile scleroderma (JS) represents a rarely seen group of connective tissue diseases with multiple organ involvement. Cardiac involvement in JSS is well known and, although rare in children, it may ...be an important cause of mortality and morbidity. Therefore, an early determination of cardio-vascular and pulmonary involvement is of the most relevance to reduce the mortality in patients with juvenile scleroderma. The aim of the study was to explore the non-invasive methods (Doppler echocardiography, pulmonary function tests), Forced vital capacity (FVC) and Carbon monoxide diffusion capacity (DLCO) in the assessment of the cardiopulmonary involvement in patients with JS. The assessment of pulmonary arterial pressure (PAP) and risk factors for pulmonary arterial hypertension (PAH) were made by the measurement of maximum tricuspid insufficiency (TI), end-diastolic pulmonary insufficiency (PI), ratio of acceleration time (AT) to ejection time (ET) (AT/ET), right atrial pressure (RAP) and contraction of vena cava inferior during inspiration. Thirty-five patients with confirmed JS were included in the study. The mean age of onset of the disease was 9.57 years (median 10 years, range 2-18 years). The mean disease duration and follow-up time was 2 years (median 1 year, range 0.5-8 years) and 3.57 years (median 2 years, range 0.5-14.5 years), respectively.The values of all the analyzed parameters including TI, PI, AT/ET, PAP, FVC and DLCO were found to be within normal ranges in all the patients tested, confirming an uncommonness of cardiopulmonary involvement in patients with juvenile scleroderma.
Aim
To examine the effects of therapeutic hypothermia on the outcome of patients with the diagnosis of out-of-hospital cardiac arrest (OHCA).
Methods
The study included 76 patients who were ...hospitalised at the Medical Intensive Care Unit (MICU) of the Clinical Centre University of Sarajevo, with the diagnosis of out-of-hospital cardiac arrest, following the return of spontaneous circulation. Therapeutic hypothermia was performed with an average temperature of 33 o C (32.3-34.1 o C) on the patients who had coma, according to the Glasgow Coma Scale (GCS).
Results
Multiple organ dysfunction syndrome (MODS) significantly affected survival (p=0.0001), as its presence reduced patients' survival by 96%. In addition, ventricular fibrillation (VF) as the presenting rhythm, also significantly affected survival (p=0.019). A degree of patient's coma, as measured by the GCS, significantly affected survival (p=0.011). For each increasing point on the GCS, the chance for survival increased twice. Moreover, other physiological factors such as the pH and the lactate serum levels significantly affected patients' survival (p=0.012 and p=0.01, respectively).
Conclusion
In patients with the diagnosis of OHCA who underwent to the treatment with therapeutic hypothermia, verified VF as a presenting rhythm was a positive predictive factor for their outcome. Therefore, therapeutic hypothermia represents an option of therapeutic modality for this type of patients.
Although some myocardial bridges can be asymptomatic, their presence often causes coronary disease either through direct compression of the "tunnel" segment or through stimulation and accelerated ...development of atherosclerosis in the segment proximally to the myocardial bridge. The studied material contained 30 human hearts received from the Department of Anatomy. The hearts were preserved 3 to 5 days in 10% formalin solution. Thereafter, the fatty tissue was removed and arterial blood vessels prepared by careful dissection with special reference to the presence of the myocardial bridges. Length and thickness of the bridges were measured by the precise electronic caliper. The angle between the myocardial bridge fibre axis and other axis of the crossed blood vessel was measured by a goniometer. The presence of the bridges was confirmed in 53.33% of the researched material, most frequently (43.33%) above the anterior interventricular branch. The mean length of the bridges was 14.64 ± 9.03 mm and the mean thickness was 1.23 ± 1.32 mm. Myocardial bridge fibres pass over the descending blood vessel at the angle of 10-90 degrees. The results obtained on a limited sample suggest that the muscular index of myocardial bridge is the highest for bridges located on RIA, but that the difference is not significant in relation to bridges located on other branches. The results obtained suggest that bridges located on other branches, not only those on RIA, could have a great contractive power and, consequently, a great compressive force, which would be exerted on the wall of a crossed blood vessel.
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