scholarly journals Ipertensione arteriosa polmonare: stima della casistica italiana e considerazioni sull’impatto sul budget dell’introduzione di tadalafil

2010 ◽  
Vol 11 (2) ◽  
pp. 77-82
Author(s):  
Luca Guidi ◽  
Mariano Alejandro Mignini
Keyword(s):  
Pulmonary Arterial Hypertension ◽  
Budget Impact ◽  
Drug Market ◽  
Fixed Dose ◽  
International Literature ◽  
Daily Cost ◽  
Dose Administration ◽  
Number Of Patients ◽  
Wide Range ◽  
Pulmonary Arterial

This contribution is an attempt to estimate a range of Pulmonary Arterial Hypertension (PAH) prevalence in Italy using international literature and Italian drug market sales data and to evaluate the budget impact of the introduction of tadalafil among the actual drugs with specific indication for this pathology. The final epidemiological figure obtained shows a wide range of prevalence of PAH in Italy (900-3,000 cases). The introduction of tadalafil as PAH treatment should not cause a cost increase for the pharmaceutical budget considering that this new therapy will occupy essentially the patient segment actually treated with sildenafil and that it reduces, in a significant number of patients, the daily cost of PAH therapy through a stable, fixed dose, administration.

PP238 Budget Impact Of Methionine-Free Amino Acid Formula For Homocystinuria

2019 ◽  
Vol 35 (S1) ◽  
pp. 75-75
Author(s):  
Eduardo Mulinari ◽  
Nayara Castelano Brito ◽  
Lays Pires Marra
Keyword(s):  
Amino Acid ◽  
Free Amino Acid ◽  
Free Amino ◽  
Budget Impact ◽  
National Committee ◽  
Budgetary Impact ◽  
Health Technologies ◽  
Number Of Patients ◽  
Wide Range ◽  
Amino Acid Formula

IntroductionThe National Committee for Health Technology Incorporation (CONITEC) evaluates health technologies to recommend their inclusion or exclusion within the Brazilian Public Health System (SUS), and uses the budget impact assessment to estimate costs to the system. This study estimated the budget impact of the supply of methionine-free amino acid formula (MFAAf) for patients with classical homocystinuria (HCU) in the SUS.MethodsThe incidence of one case per 250,000 live births in Brazil and the registration of a Brazilian association of patients with HCU was assumed to calculate the population. Mortality and responsiveness to pyridoxine rates were applied. The costs of treatment were estimated according to the recommended dosage in literature and public purchasing prices. For calculating the dose of MFAAf patients, a median age of 19 years and weight of 60 kg were assumed, according to Brazilian study data.ResultsThe annual cost of treatment was estimated at BRL 77,000 (USD 21,084) per patient. The incorporation of MFAAf for HCU would generate a budget impact in SUS of around BRL 37 million (USD 10.1 million) in 2019 and BRL 188 million (USD 51.5 million) after five years which considers the epidemiological data, and a budget impact of around BRL 6.4 million (USD 1.75 million) in 2019 and BRL 33 million (USD 9 million) after five years which considers the information of a Brazilian association of patients with HCU. The wide range of values in the incremental budgetary impact is due to the lack of information on the epidemiology of the disease in Brazil.ConclusionsThe incorporation of the MFAAf in the SUS represents an important budgetary impact and covers a small number of patients. CONITEC recommended the incorporation of the MFAAf in the SUS, according to clinical protocol.

A Bayesian Network Meta-analysis of Add-on Drug Therapies Specific for Pulmonary Arterial Hypertension

2019 ◽  
Vol 54 (5) ◽  
pp. 423-433 ◽  
Author(s):  
Maja Petrovič ◽  
Igor Locatelli
Keyword(s):  
Pulmonary Arterial Hypertension ◽  
Combination Therapy ◽  
Arterial Hypertension ◽  
Meta Analysis ◽  
Phosphodiesterase Type 5 Inhibitors ◽  
Number Of Patients ◽  
All Cause Mortality ◽  
Pulmonary Arterial ◽  
Meta Analyses ◽  
Better Than

Background: Recently published meta-analyses did not discriminate between drug agents used for initial and sequential combination therapy. Objective: To assess the comparative efficacy of drugs specific for the treatment of pulmonary arterial hypertension (PAH) as add-on therapies based on 6-minute walk distance (6MWD), all-cause mortality, and discontinuation due to adverse events (AEs). Methods: EMBASE, PubMed, Cochrane Library, and ClinicalTrials.gov were searched until December 9, 2018, for the randomized, placebo-controlled clinical trials (RCTs) conducted on primarily adult patients diagnosed with PAH. Data extracted from applicable RCTs were as follows: for 6MWD mean change from baseline, the total number of patients, and the number of patients with events, per treatment. Network meta-analysis (NMA) was conducted in a Bayesian framework. Results: A total of 16 RCTs were eligible for analysis, with 4112 patients. Add-on therapy with tadalafil or inhaled treprostinil performed better than endothelin receptor antagonists alone [27 m; 95% credible interval (CrI): (11, 43); and 19 m; 95% CrI: (10, 27); respectively]. Add-on therapy with macitentan or bosentan performed better than phosphodiesterase type 5 inhibitors alone [26 m; 95% CrI: (6.4, 45); and 22 m; 95% CrI: (5.1, 38); respectively]. Differences in all-cause mortality and discontinuation due to AEs were nonsignificant. Conclusion and Relevance: Our NMA evaluated efficacy and safety of add-on therapies in patients with PAH. None of the previous meta-analyses evaluated RCTs focusing solely on patients pretreated with another PAH-specific drug therapy. Our results support guideline recommendations on combination therapy in PAH patients and add the quantitative perspective on which sequential therapy demonstrated the greatest effect size.

scholarly journals Echocardiography and Pulmonary Arterial Hypertension

2016 ◽  
Vol 68 (4) ◽  
Author(s):  
Eduardo Bossone ◽  
Rodolfo Citro ◽  
Alberto Ruggiero ◽  
Bettina Kuersten ◽  
Giovanni Gregorio ◽  
...  
Keyword(s):  
Pulmonary Arterial Hypertension ◽  
Arterial Hypertension ◽  
Structural Changes ◽  
Pulmonary Arteries ◽  
Progressive Increase ◽  
Accurate Estimate ◽  
Therapeutic Interventions ◽  
Wide Range ◽  
Advanced Stages ◽  
Pulmonary Arterial

Pulmonary Arterial Hypertension (PAH) is an heterogeneous condition brought on by a wide range of causes. It is characterized by structural changes in small pulmonary arteries, that produce a progressive increase in pulmonary artery pressure and pulmonary vascular resistance, ultimately leading to right ventricle failure and death. Given the non-specific nature of its early symptoms and signs, PAH is often diagnosed in its advanced stages. Along with a careful clinical assessment and an accurate electrocardiogram/Chest X-ray interpretation, echocardiography is an essential test in the evaluation of patient with PAH. In fact it not only provides an accurate estimate of pulmonary pressure at rest and during exercise, but may also help to exclude any secondary causes, predict the prognosis, monitor the efficacy of specific therapeutic interventions and detect the preclinical stage of the disease.

Levels of lipoprotein (a) in pulmonary arterial hypertension

2001 ◽  
Vol 11 (1) ◽  
pp. 25-29 ◽  
Author(s):  
Raul D. Santos ◽  
Antonio Foronda ◽  
José A. F. Ramires ◽  
Raul C. Maranhão
Keyword(s):  
Pulmonary Arterial Hypertension ◽  
Arterial Hypertension ◽  
Normal Population ◽  
Lipoprotein A ◽  
Eisenmenger’S Syndrome ◽  
Number Of Patients ◽  
Caucasian Patients ◽  
Pulmonary Arterial ◽  
Turbidimetric Assay ◽  
Genetically Determined

AbstractWe compared the levels of lipoprotein (a) in 48 Caucasian patients with pulmonary arterial hypertension, comprising 32 females and 16 males, aged 28.0 ± 12.0 years, with a range from 4 through 52 years, with 48 normal Caucasian subjects matched for age and sex. Pulmonary hypertension was secondary in 41 patients with Eisenmenger's syndrome, these comprising 27 females and 14 males aged 27.0 ± 12.0 years, with a range from 4 through 51 years, and primary in the other 7 patients, 5 females and 2 males, whose age was 30.0 ± 14.0 years, with a range from 9 through 52 years. Lipoprotein (a) was measured using an immunoprecipitation and turbidimetric assay after a 12 hour fast. Levels of the protein, expressed as the median (% 25; % 75), were higher in those with Eisenmenger's syndrome than in normal controls (p = 0.003). In addition, there was a greater prevalence of levels of lipoprotein greater than 30.0 mg/dl in those with secondary pulmonary arterial hypertension patients than in our normal population (p = 0.03). We have found no differences, however, in the levels of lipoprotein(a) in those who had primary pulmonary arterial hypertension when compared with their matched controls, albeit that the number of patients studied was small. We conclude that increased levels of lipoprotein (a) may be secondary to pulmonary arterial hypertension as a marker of tissue damage or may be genetically determined. In either way, the increase in lipoprotein (a) could be an additional factor predisposing to the vascular alterations known to occur in this disease.

scholarly journals Chemical and biological assessment of metal organic frameworks (MOFs) in pulmonary cells and in an acute in vivo model: relevance to pulmonary arterial hypertension therapy

2017 ◽  
Vol 7 (3) ◽  
pp. 643-653 ◽  
Author(s):  
Nura A. Mohamed ◽  
Robert P. Davies ◽  
Paul D. Lickiss ◽  
Blerina Ahmetaj-Shala ◽  
Daniel M. Reed ◽  
...  
Keyword(s):  
Endothelial Cells ◽  
Pulmonary Arterial Hypertension ◽  
Side Effects ◽  
Arterial Hypertension ◽  
Therapeutic Window ◽  
Endothelin 1 ◽  
Wide Range ◽  
Metal Organic ◽  
Pulmonary Arterial

Pulmonary arterial hypertension (PAH) is a progressive and debilitating condition. Despite promoting vasodilation, current drugs have a therapeutic window within which they are limited by systemic side effects. Nanomedicine uses nanoparticles to improve drug delivery and/or reduce side effects. We hypothesize that this approach could be used to deliver PAH drugs avoiding the systemic circulation. Here we report the use of iron metal organic framework (MOF) MIL-89 and PEGylated MIL-89 (MIL-89 PEG) as suitable carriers for PAH drugs. We assessed their effects on viability and inflammatory responses in a wide range of lung cells including endothelial cells grown from blood of donors with/without PAH. Both MOFs conformed to the predicted structures with MIL-89 PEG being more stable at room temperature. At concentrations up to 10 or 30 µg/mL, toxicity was only seen in pulmonary artery smooth muscle cells where both MOFs reduced cell viability and CXCL8 release. In endothelial cells from both control donors and PAH patients, both preparations inhibited the release of CXCL8 and endothelin-1 and in macrophages inhibited inducible nitric oxide synthase activity. Finally, MIL-89 was well-tolerated and accumulated in the rat lungs when given in vivo. Thus, the prototypes MIL-89 and MIL-89 PEG with core capacity suitable to accommodate PAH drugs are relatively non-toxic and may have the added advantage of being anti-inflammatory and reducing the release of endothelin-1. These data are consistent with the idea that these materials may not only be useful as drug carriers in PAH but also offer some therapeutic benefit in their own right.

The enzymatic degradation of hyaluronan is associated with disease progression in experimental pulmonary hypertension

2010 ◽  
Vol 298 (2) ◽  
pp. L148-L157 ◽  
Author(s):  
Mark L. Ormiston ◽  
Graham R. D. Slaughter ◽  
Yupu Deng ◽  
Duncan J. Stewart ◽  
David W. Courtman
Keyword(s):  
Pulmonary Hypertension ◽  
Pulmonary Arterial Hypertension ◽  
Arterial Hypertension ◽  
Molecular Mass ◽  
Disease Progression ◽  
Enzymatic Degradation ◽  
Early Stage ◽  
Wide Range ◽  
Pulmonary Arterial ◽  
Ha Synthase

Hyaluronan (HA) degradation fragments have been linked to inflammation in a wide range of lung diseases. In idiopathic pulmonary arterial hypertension, HA accumulation has been associated with advanced disease. In this study, we investigated the potential role of HA degradation in the early stages of disease by examining HA distribution, molecular mass, synthesis, and enzymatic degradation at different stages of disease progression in a rat model of monocrotaline (MCT)-induced pulmonary hypertension (PH). At 28 days post-MCT, severe PH was associated with increased total lung HA ( P = 0.04). In contrast, a significant decrease in total lung HA was observed on day 10, before the onset of PH ( P = 0.02). Molecular mass analysis revealed a loss of high molecular mass (HMM) HA at 10 and 24 days post-MCT, followed by an increase in HMM HA at 28 days. Expression of HA synthase 2 ( HAS2) was elevated in MCT-challenged animals at 24 and 28 days, consistent with increased synthesis of HMM HA. Analysis by Morgan Elson assay and zymography demonstrated increased hyaluronidase-1 activity in the lungs of MCT-challenged rats, indicating that the observed increases in HAS2 expression and HA synthesis were counterbalanced, in part, by enhanced degradation. The present data demonstrate that, in the MCT model, early-stage PH is associated with enhanced hyaluronidase-1 activity, while both degradation and synthesis are increased at later stages. Thus an early increase in the generation of proinflammatory HA fragments may play a role in the onset and progression of pulmonary arterial hypertension.

scholarly journals Outpatient specialist clinics for pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension in the Nordic countries

2020 ◽  
Vol 10 (4) ◽  
pp. 204589401989749 ◽  
Author(s):  
Barbro Kjellström ◽  
Henrik Ryftenius ◽  
Lise-Lotte Landenfelt-Gestre ◽  
Bodil Ivarsson
Keyword(s):  
Pulmonary Hypertension ◽  
Pulmonary Arterial Hypertension ◽  
Arterial Hypertension ◽  
Complex Interventions ◽  
Chronic Thromboembolic Pulmonary Hypertension ◽  
Nordic Countries ◽  
Multidisciplinary Teams ◽  
Number Of Patients ◽  
Thromboembolic Pulmonary Hypertension ◽  
Pulmonary Arterial

Pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension are rare conditions that require complex interventions by multidisciplinary teams. The European Society of Cardiology (ESC)/the European Respiratory Society (ERS) 2015 guidelines included recommendations for pulmonary hypertension (PH) referral centers including minimum number of patients, staff, facilities, and network. The aim of the present study was to investigate how the PH-specialist centers in the Nordic countries are presently organized. A descriptive, questionnaire was sent to all PH-specialist centers in the Nordic countries in 2018. Sixteen of 20 PH-specialist centers completed the questionnaire. Seven centers (43%) followed less than 50 patients and three centers (19%) followed 125 patients or more. All had a physician or nurse attending or available at the clinic and eight had support staff such as physiotherapists, counsellors, dieticians, or psychologists directly connected to the center. Twelve centers were available by telephone five days or more per week. Nine centers offered a nurse-led outpatient clinic and of those, six had nurses delegated to make protocol-led changes in pulmonary arterial hypertension-specific treatment. Half of the centers had cooperation with a patient organization. All centers except one used international guidelines to guide care and treatment. More than half of the Nordic PH-specialist centers adhered to the ESC/ERS 2015 guidelines recommendations for volumes and staff in 2018, but there is potential for improvement. However, when formulating recommendations of patient volumes in guidelines, the situation for the geographical large but sparsely populated Nordic countries needs to be considered.

scholarly journals Vascular Compromise and Hemodynamics in Pulmonary Arterial Hypertension: Model Predictions

2012 ◽  
Vol 19 (3) ◽  
pp. 209-215 ◽  
Author(s):  
Zoheir Bshouty
Keyword(s):  
Cardiac Output ◽  
Pulmonary Arterial Hypertension ◽  
Arterial Hypertension ◽  
Disease Process ◽  
Early Detection Of Disease ◽  
Alternative Approach ◽  
Wide Range ◽  
Model Predictions ◽  
Vascular Compromise ◽  
Pulmonary Arterial

A previously validated computer model of the normal pulmonary circulation is adapted to simulate pulmonary arterial hypertension (PAH) in humans. Model predictions are used to explore the suitability of currently accepted criteria for diagnosing PAH by correlating hemodynamic data with the degree of vascular compromise (disease severity).Model predictions demonstrate a hyperbolic relationship between vascular compromise, mean pulmonary artery pressure (PAPm) and pulmonary vascular resistance (PVR). PAPm and PVR change very little from disease initiation until a vascular compromise of 65% to 70% (surface area of 0.35 to 0.3 of baseline, respectively) is reached. Following that, further compromise is associated with a steep rise in PAPm and PVR.The relationship between vascular compromise and hemodynamics may explain the relative stability of cardiac output early in this disease process and, therefore, the lack of symptoms. It also explains the rapid deterioration following diagnosis if the disease remains untreated.Model predictions demonstrate the inadequacy of the current hemodynamic criteria for diagnosing PAH over a wide range of left atrial pressure and cardiac output combinations and for early detection of disease. The model provides an alternative approach to diagnosing PAH by translating hemodynamic data to degree of vascular compromise.

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