Cost-Effectiveness Analysis Of Ambrisentan Versus Bosentan In The Treatment Of Patients With Pulmonary Arterial Hypertension Functional Class III

The article presents a clinical case of successful triple combination therapy in a female patient with functional class III idiopathic pulmonary arterial hypertension. Supplementing the previous macitentan and riociguat treatment with selexipag reduced the severity of clinical manifestations of pulmonary hypertension. Also, the treatment efficacy was demonstrated by improvement of laboratory and instrumental indexes. Time-related changes were evaluated at 3 months after initiation of the selexipag treatment.

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Phenotype and outcome of pulmonary arterial hypertension patients carrying a TBX4 mutation

European Respiratory Journal ◽

10.1183/13993003.02340-2019 ◽

2020 ◽

Vol 55 (5) ◽

pp. 1902340 ◽

Cited By ~ 3

Author(s):

Pierre Thoré ◽

Barbara Girerd ◽

Xavier Jaïs ◽

Laurent Savale ◽

Maria-Rosa Ghigna ◽

...

Keyword(s):

New York ◽

Pulmonary Arterial Hypertension ◽

Arterial Hypertension ◽

Survival Rates ◽

Heart Association ◽

Lung Parenchyma ◽

Functional Class ◽

Class Iii ◽

High Resolution Computed Tomography ◽

Pulmonary Arterial

IntroductionTBX4 mutation causes small patella syndrome (SPS) and/or pulmonary arterial hypertension (PAH). The characteristics and outcomes of PAH associated with TBX4 mutations are largely unknown.MethodsWe report the clinical, functional, radiologic, histologic and haemodynamic characteristics and outcomes of heritable PAH patients carrying a TBX4 mutation from the French pulmonary hypertension (PH) network.Results20 patients were identified in 17 families. They were characterised by a median age at diagnosis of 29 years (0–76 years) and a female to male ratio of three. Most of the patients (70%) were in New York Heart Association (NYHA) functional class III or IV with a severe haemodynamic impairment (median pulmonary vascular resistance (PVR) of 13.6 (6.2–41.8) Wood units). Skeletal signs of SPS were present in 80% of cases. Half of the patients had mild restrictive or obstructive limitation and diffusing capacity of the lung for carbon monoxide (DLCO) was decreased in all patients. High-resolution computed tomography (HRCT) showed bronchial abnormalities, peri-bronchial cysts, mosaic distribution and mediastinal lymphadenopathies. PAH therapy was associated with significant clinical improvement. At follow-up (median 76 months), two patients had died and two had undergone lung transplantation. One-year, three-year and five-year event-free survival rates were 100%, 94% and 83%, respectively. Histologic examination of explanted lungs revealed alveolar growth abnormalities, major pulmonary vascular remodelling similar to that observed in idiopathic pulmonary arterial hypertension (IPAH) and accumulation of cholesterol crystals within the lung parenchyma.ConclusionPAH due to TBX4 mutations may occur with or without skeletal abnormalities across a broad age range from birth to late adulthood. PAH is usually severe and associated with bronchial and parenchymal abnormalities.

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The Modified Borg Dyspnea Scale does not predict hospitalization in pulmonary arterial hypertension

Pulmonary Circulation ◽

10.1177/2045893217695568 ◽

2017 ◽

Vol 7 (2) ◽

pp. 384-390 ◽

Cited By ~ 2

Author(s):

Debasree Banerjee ◽

Jane Kamuren ◽

Grayson L. Baird ◽

Amy Palmisciano ◽

Ipsita Krishnan ◽

...

Keyword(s):

Pulmonary Arterial Hypertension ◽

Arterial Hypertension ◽

Functional Class ◽

World Health ◽

Pulmonary Vascular Disease ◽

Common Symptom ◽

Class Iii ◽

Patient Reported ◽

Pulmonary Arterial ◽

Minute Walk

Background Breathlessness is the most common symptom reported by patients with pulmonary arterial hypertension (PAH). The Modified Borg Dyspnea Scale (MBS) is routinely obtained during the six-minute walk test in the assessment of PAH patients, but it is not known whether the MBS predicts clinical outcomes such as hospitalizations in PAH. Methods We performed a retrospective study of World Health Organization (WHO) Group 1 PAH patients followed at our center. The dates of the first three MBS and hospitalizations that occurred within three months of a documented MBS were collected. Marginal Cox hazard regression modeling was used to assess for a relationship between MBS and all-cause as well as PAH-related hospitalization. Results A total of 50 patients were included; most (92%) were functional class III/IV, 44% and 65% were treatment-naïve prior to their first MBS and hospitalization, respectively. The first recorded MBS was inversely correlated with the first recorded six-minute walk distance (6MWD) (r = –0.41, P < 0.01) but did not track with WHO functional class (r = 0.07, P = 0.63). MBS did not predict all-cause (hazard ratio [HR], 0.91; 95% confidence interval [CI], 0.76–1.08; P = 0.28) or PAH-related hospitalization (HR, 1.04; 95% CI, 0.89–1.23; P = 0.61), though there was a strong relationship between 6MWD and PAH-related hospitalization ( P = 0.01). These findings persisted after multivariable adjustment. Conclusions Breathlessness as assessed by MBS does not predict all-cause or PAH-related hospitalization. Robust and validated patient-reported outcomes are needed in pulmonary vascular disease.

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Bosentan-based, treat-to-target therapy in patients with pulmonary arterial hypertension: results from the COMPASS-3 study

Pulmonary Circulation ◽

10.1177/2045893217741480 ◽

2017 ◽

Vol 8 (1) ◽

pp. 204589321774148 ◽

Cited By ~ 4

Author(s):

Raymond L. Benza ◽

Amresh Raina ◽

Himanshu Gupta ◽

Srinivas Murali ◽

Annie Burden ◽

...

Keyword(s):

Pulmonary Arterial Hypertension ◽

Arterial Hypertension ◽

Target Therapy ◽

Left Ventricular ◽

Functional Class ◽

Treat To Target ◽

Heart Catheterization ◽

Class Iii ◽

Pulmonary Arterial ◽

Clinical Worsening

The phase 4 COMPASS-3 study evaluated whether a singular endpoint produces clinically meaningful outcomes in patients with pulmonary arterial hypertension (PAH). The relationship between cardiac magnetic resonance imaging (cMRI)-derived parameters and right heart catheterization (RHC) measurements was also examined. In COMPASS-3 (ClinicalTrials.gov NCT00433329), 100 patients with PAH received bosentan monotherapy for 16 weeks. Patients continued monotherapy if their 6-min walk distance (6MWD) was ≥380 m, or otherwise received add-on sildenafil for an additional 12 weeks. 6MWD, RHC, and cMRI were performed at baseline, week 16, and week 28 (6MWD and cMRI). Baseline median 6MWD was 274 m and 82% of patients had WHO Functional Class III/IV. At week 16, 17% (n = 16) of remaining patients achieved the 6MWD threshold and 78 (83%) did not. In the intention-to-treat population, median 6MWD increased significantly relative to baseline (week 16 = 308 m; week 28 = 327 m; P < 0.001). At week 28, 9/16 (monotherapy) and 15/76 (20%; add-on sildenafil) patients met the target threshold. Baseline cMRI-derived and RHC-derived parameters showed moderate-to-strong correlations (e.g. right to left ventricular end-diastolic ratio [RVEDV:LVEDV] correlated strongly with pulmonary vascular resistance [r = +0.729, P < 0.0001]). cMRI-derived parameters predicted clinical worsening/decline (e.g. week 16 RVEDV:LVDEV [ P = 0.0172]). Time to clinical worsening/decline did not differ between patients based on 6MWD threshold achievement. No unexpected safety events were reported. A substantial proportion of patients failed to achieve the goal of 380 m, regardless of treatment. Several cMRI parameters predicted clinical worsening/decline and its non-invasive nature further supports its use in future clinical trials.

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