AB0697 Systemic Sclerosis-Associated Pulmonary Arterial Hypertension: Is it Feasible to Diagnose and Maintain Patients in World Health Organization (WHO) Functional Class II?

Treprostinil, a prostacyclin analogue, is approved for the treatment of pulmonary arterial hypertension (PAH) in adults. Transition from parenteral to oral treprostinil has been successfully accomplished in adults with PAH but not in children. In this multicenter study, pediatric patients treated with parenteral (Cohort 1) or inhaled (Cohort 2) treprostinil were transitioned to oral treprostinil. Prostacyclin-naïve individuals on background oral PAH therapy received oral treprostinil as add-on therapy (Cohort 3). Successful transition was oral treprostinil dose maintenance through week 24. Patients were monitored for adverse events (AEs), 6-min walk distance (6MWD), PAH symptoms, World Health Organization (WHO) Functional Class (FC), cardiac magnetic resonance imaging (cMRI), cardiopulmonary exercise testing (CPET), and quality of life through 24 weeks. A total of 32 patients were enrolled in the study; 23 (72%) were girls (mean age = 12.2 years). All patients were on background oral PAH therapy. Overall, patients (96.9%) maintained transition to oral treprostinil; one patient (Cohort 1) transitioned to oral treprostinil, then back to parenteral after experiencing syncope and WHO FC change from II to III. Cohorts 1, 2, and 3 received a final mean oral treprostinil dose of 5.6, 3.3, and 4.5 mg t.i.d., respectively. All cohorts had variable changes in 6MWD, cMRI, and CPET. Overall, 12 serious AEs were reported. All patients had drug-related AEs including headache (81%), diarrhea (69%), nausea (66%), vomiting (66%), and flushing (56%). Pediatric patients maintained transition to oral treprostinil with preservation of exercise capacity and WHO FC. Prostanoid-related AEs were most common and similar to those reported in adults.

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Epidemiology and initial management of pulmonary arterial hypertension: real-world data from the Hellenic pulmOnary hyPertension rEgistry (HOPE)

Pulmonary Circulation ◽

10.1177/2045894019877157 ◽

2019 ◽

Vol 9 (3) ◽

pp. 204589401987715 ◽

Cited By ~ 8

Author(s):

Alexandra Arvanitaki ◽

Maria Boutsikou ◽

Anastasia Anthi ◽

Sotiria Apostolopoulou ◽

Aikaterini Avgeropoulou ◽

...

Keyword(s):

Pulmonary Hypertension ◽

Pulmonary Arterial Hypertension ◽

Arterial Hypertension ◽

Functional Class ◽

World Health ◽

Real World Data ◽

Treatment Data ◽

Risk Patients ◽

Pulmonary Arterial ◽

Health Organization

Pulmonary arterial hypertension (PAH) is a heterogenous clinical entity with poor prognosis, despite recent major pharmacological advances. To increase awareness about the pathophysiology, epidemiology, and management of the disease, large national registries are required. The Hellenic pulmOnary hyPertension rEgistry (HOPE) was launched in early 2015 and enrolls patients from all pulmonary hypertension subgroups in Greece. Baseline epidemiologic, diagnostic, and initial treatment data of consecutive patients with PAH are presented in this article. In total, 231 patients with PAH were enrolled from January 2015 until April 2018. At baseline, about half of patients with PAH were in World Health Organization functional class II. The majority of patients with PAH (56.7%) were at intermediate 1-year mortality risk, while more than one-third were low-risk patients, according to an abbreviated risk stratification score. Half of patients with PAH were on monotherapy, 38.9% received combination therapy, while prostanoids were used only in 12.1% of patients. In conclusion, baseline data of the Greek PAH population share common characteristics, but also have some differences with other registries, the most prominent being a better functional capacity. This may reflect earlier diagnosis of PAH that in conjunction with the increased proportion of patients with atypical PAH could partially explain the preference for monotherapy and the limited use of prostanoids in Greece. Nevertheless, early, advanced specific therapy is strongly recommended.

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Phase 2 Clinical Study to Evaluate the Efficacy and Safety of Inhaled GB002 for the Treatment of World Health Organization Group 1 Pulmonary Arterial Hypertension

The Journal of Heart and Lung Transplantation ◽

10.1016/j.healun.2021.01.346 ◽

2021 ◽

Vol 40 (4) ◽

pp. S107

Author(s):

R. Frantz ◽

L.S. Howard ◽

V.V. McLaughlin ◽

O. Sitbon ◽

R.T. Zamanian ◽

...

Keyword(s):

Clinical Study ◽

Pulmonary Arterial Hypertension ◽

Arterial Hypertension ◽

World Health Organization ◽

World Health ◽

Phase 2 ◽

Efficacy And Safety ◽

Pulmonary Arterial ◽

Health Organization ◽

Group 1

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Dasatinib-Induced Pulmonary Arterial Hypertension Treated with Upfront Combination Therapy

Case Reports in Cardiology ◽

10.1155/2018/3895197 ◽

2018 ◽

Vol 2018 ◽

pp. 1-5 ◽

Cited By ~ 1

Author(s):

Makoto Nishimori ◽

Tomoyuki Honjo ◽

Kenji Kaihotsu ◽

Naohiko Sone ◽

Sachiko Yoshikawa ◽

...

Keyword(s):

Pulmonary Arterial Hypertension ◽

Combination Therapy ◽

Arterial Hypertension ◽

Rare Complication ◽

Functional Class ◽

World Health ◽

First Line Therapy ◽

Mean Pulmonary Arterial Pressure ◽

Pulmonary Arterial ◽

Health Organization

Pulmonary arterial hypertension (PAH) is a rare complication of dasatinib that was approved as a first-line therapy for chronic myelocytic leukemia (CML). A 24-year-old man presenting dyspnea at rest and leg edema was admitted to our hospital. He had been diagnosed with CML and prescribed dasatinib for 4 years. Chest X-ray showed significant bilateral pleural effusion and heart enlargement. Echocardiography revealed interventricular septal compression and elevated peak tricuspid regurgitation pressure gradient of 66.7 mmHg indicating severe pulmonary hypertension. After the other specific diseases to provoke PAH were excluded, he was diagnosed with dasatinib-induced PAH. Despite discontinuation of dasatinib and intravenous administration of diuretic for two weeks, World Health Organization (WHO) functional class was still II and mean pulmonary arterial pressure (PAP) was high at 37 mmHg. Therefore, we administered sildenafil and bosentan together as an upfront combination therapy three weeks after dasatinib discontinuation. Six months later, his symptoms improved to WHO functional class I and mean PAP was decreased to 31 mmHg. Although PAH is a rare complication of dasatinib, symptomatic patients prescribed with dasatinib should have an echocardiogram for PAH screening. Moreover, the upfront combination therapy would be a useful option for symptomatic patients after discontinuation of dasatinib.

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Risk assessment and treatment goals in patients presenting with pulmonary arterial hypertension

ESC CardioMed ◽

10.1093/med/9780198784906.003.0603_update_001 ◽

2018 ◽

pp. 2560-2562

Author(s):

Marius M. Hoeper

Keyword(s):

Risk Assessment ◽

Pulmonary Arterial Hypertension ◽

Arterial Hypertension ◽

Right Ventricular ◽

Right Ventricular Function ◽

Functional Class ◽

Low Risk ◽

World Health ◽

Pulmonary Arterial ◽

Health Organization

Risk stratification in patients with pulmonary arterial hypertension is based on a multidimensional approach that utilizes clinical features, World Health Organization functional class, exercise capacity, biomarkers, and evaluation of right ventricular function by imaging and/or invasive measurements. Risk assessment at the time of diagnosis determines the initial treatment strategy, while risk assessment during follow-up is performed to identify disease progression and the need for treatment modifications. A low-risk category is characterized by non-progressive disease, absence of signs of right heart failure and syncope, World Health Organization functional class I or II with normal or near normal exercise capacity, normal or near normal plasma levels of B-type natriuretic peptide, and well-preserved right ventricular function as determined by echocardiography, cardiovascular magnetic resonance imaging, or right heart catheterization. Achieving and maintaining a low-risk status is a major objective in the treatment of patients with pulmonary arterial hypertension.

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