Long-term real world clinical outcomes of macitentan therapy in chronic thromboembolic pulmonary hypertension

Thor, M.C.J. van; Klooster, Liesbeth ten; Snijder, Repke J.; Mager, Johannes J.; Post, Martijn C.

doi:10.1016/j.rmed.2020.105966

Cited by 7 publications

(11 citation statements)

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“…This beneficial development was found in etiologies and clinical approaches, and regardless of previous therapies. [ 10 11 12 ] Progressively, clinical changes were also reported in the 5 patients who were previously treated with bosentan and ambrisentan and were associated with reasonable tolerability, consistent with recent publication reports, without elevation of liver enzymes. [ 10 ]…”

Section: Discussionsupporting

confidence: 85%

The clinical experience of macitentan in pulmonary hypertension in Indian cohort: 12-month follow-up

et al. 2021

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Background: The effectiveness and safety of macitentan, an endothelin-receptor antagonist (ERA) in the treatment of pulmonary arterial hypertension (PAH), has been demonstrated in numerous randomized clinical trials including SERAPHIN, focused on the reduction of morbidity and mortality. Objectives: Our aim was to demonstrate the clinical and echocardiographic progression using macitentan in Indian patients with PAH. Settings and Design: It was a retrospective study of 20 patients with multiple etiologies of PAH who had begun macitentan in routine clinical practice from a single center. There were 55% of patients with existing PAH therapies. Subjects and Methods: The World Health Organization functional class (WHO-FC), 6-min walking distance, N-terminal prohormone of brain natriuretic peptide level (NT-pro-BNP), and echocardiographic data such as tricuspid annular plane systolic excursion (TAPSE), systolic pulmonary artery pressure (sPAP), and the occurrence of pericardial effusion were collected at baseline and 12-month follow-up. The statistical analysis was performed using SPSS software. Results: Of the 20 PAH patients, 70% were women. The majority were in WHO FC II (50%), while 35% were in FC III and 15% were in FC IV. The mean age was 43.4 years at the start of the therapy with macitentan. After 6 months of macitentan therapy, 85% showed substantial improvement in their FC, each increased its 6-min walking distance test ( P < 0.0001), and there was a significant reduction in plasma levels of NT-pro BNP ( P < 0.0001). There has also been an improvement in echocardiographic parameters such as TAPSE and sPAP ( P < 0.0001). Conclusions: Our findings indicate that macitentan has been well tolerated and beneficial in Indian patients with PAH and further, future research is required to verify these results.

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Section: Discussionsupporting

confidence: 85%

The clinical experience of macitentan in pulmonary hypertension in Indian cohort: 12-month follow-up

et al. 2021

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“…14 The combination of macitentan and riociguat was also found to be safe in a real-world study with inoperable and residual CTEPH patients. 31 Although TEAEs of hemoptysis and pulmonary hemorrhage, including cases with fatal outcome, have been observed in patients with CTEPH or PAH treated with riociguat, 10,32,33 no such TEAEs were reported in the present study.…”

Section: Safety Findingscontrasting

confidence: 47%

Results of Macitentan in Japanese Patients With Chronic Thromboembolic Pulmonary Hypertension　― A Prospective, Multicenter, Open-Label, Single-Arm, Phase 3 Study ―

Sakurai

Ukyo²

2021

Circ Rep

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Background: Macitentan, an endothelin-receptor antagonist, is approved in Japan for the treatment of pulmonary arterial hypertension (PAH). This study evaluated the use of macitentan for chronic thromboembolic pulmonary hypertension (CTEPH) in Japanese patients. Methods and Results:This open-label single-arm Phase 3 study evaluated the efficacy and safety of oral macitentan 10 mg (once daily) in Japanese CTEPH patients. The study was prematurely discontinued due to the sponsor's decision to not develop macitentan 10 mg further for the indication of CTEPH (unrelated to safety concerns). Of the 9 patients enrolled in the study, 4 completed 24 weeks of treatment. The mean (± SD) ratio of pulmonary vascular resistance (PVR) at Week 16 to baseline was 71.9±34.3%. The mean (± SD) decreases in PVR and the PVR index (PVRI) from baseline to Week 16 were 181.4±243.9 dyn • s/cm 5 and 280.6±366.0 dyn • s • m 2 /cm 5 , respectively. The mean (± SD) increase in the 6-min walk distance from baseline to Week 24 was 44.3±46.8 m. All treatment-emergent adverse events (TEAEs) were mild or moderate in severity, except for 1 serious TEAE of angioplasty reported in 1/9 patients that was severe in intensity.Conclusions: Definite conclusions regarding the efficacy of macitentan 10 mg in Japanese patients with CTEPH cannot be drawn because of premature study discontinuation. No safety concerns were observed, and the safety profile was consistent with previously reported studies in CTEPH and PAH patients.

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“…The diagnosis and management of CTEPH have been assessed in the International CTEPH Registry in Canada and Western/Central European countries [ 7 , 19 , 38 ], while the worldwide prospective CTEPH Registry encompasses the USA, Japan, Taiwan, and many countries in Europe, including the Russian Federation [ 39 ]. The US CTEPH Registry has also reported results [ 40 ], and a retrospective observational single-center study in the Netherlands assessed clinical outcomes of macitentan therapy in patients with non-operated CTEPH or with residual PH after PEA [ 41 ]. The Russian National Registry reported demographic and clinical characteristics of 727 newly diagnosed patients with PH including 206 with CTEPH [ 42 ].…”

Section: Introductionmentioning

confidence: 99%

Diagnosis and Treatment Patterns of Chronic Thromboembolic Pulmonary Hypertension in Russia, Kazakhstan, Turkey, Lebanon, and Saudi Arabia: A Registry Study

Öngen,

Akdeniz,

Düzenli

et al. 2024

Drugs - Real World Outcomes

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Background Patients with chronic thromboembolic pulmonary hypertension (CTEPH) in countries with limited resources have, to date, been poorly represented in registries. Objective This work assesses the epidemiology, diagnosis, hemodynamic and functional parameters, and treatment of CTEPH in Russia, Kazakhstan, Turkey, Lebanon, and Saudi Arabia. Methods A prospective, cohort, phase IV, observational registry with 3-year follow-up ( n = 212) in patients aged ≥ 18 years diagnosed with CTEPH was created. Clinical, hemodynamic, and functional parameters were obtained at an initial visit, follow-up visits, and a final visit at the end of 3 years’ observation or end of follow-up. Data were recorded on electronic case report forms. Parameters evaluated included 6-minute walking distance (6MWD), use of pulmonary endarterectomy (PEA), balloon pulmonary angioplasty (BPA), pulmonary hypertension (PH)-targeted therapy, and survival. All statistical analyses were exploratory and descriptive, and were performed in the overall population. Results The most common symptoms were typical of those expected for CTEPH. Almost 90% of patients underwent right heart catheterization at diagnosis or initial study visit. In total, 66 patients (31%) underwent PEA before the initial visit; 95 patients (45%) were considered operable, 115 (54%) were inoperable, and two (1%) had no operability data. Only 26 patients (12%) had been assessed for BPA at their initial visit. PH-targeted therapy was documented at diagnosis for 77 patients (36%), most commonly a phosphodiesterase type 5 inhibitor (23%). Use of PH-targeted therapy increased to 142 patients (67%) at the initial visit, remaining similar after 3 years. Use of riociguat increased from 6% of patients at diagnosis to 38% at 3 years. Between baseline and end of observation, results for patients with paired data showed an increase in 6MWD. Survival at the end of observation was 88%. Conclusions These data highlight the current diagnosis and management of CTEPH in the participating countries. They show that early CTEPH diagnosis remains challenging, and use of off-label PH-targeted therapy is common. ClinicalTrials.gov: NCT02637050; registered December 2015. Graphical Abstract Supplementary Information The online version contains supplementary material available at 10.1007/s40801-023-00407-w.

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Long-term real world clinical outcomes of macitentan therapy in chronic thromboembolic pulmonary hypertension

Cited by 7 publications

References 20 publications

The clinical experience of macitentan in pulmonary hypertension in Indian cohort: 12-month follow-up

The clinical experience of macitentan in pulmonary hypertension in Indian cohort: 12-month follow-up

Results of Macitentan in Japanese Patients With Chronic Thromboembolic Pulmonary Hypertension　― A Prospective, Multicenter, Open-Label, Single-Arm, Phase 3 Study ―

Diagnosis and Treatment Patterns of Chronic Thromboembolic Pulmonary Hypertension in Russia, Kazakhstan, Turkey, Lebanon, and Saudi Arabia: A Registry Study

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Long-term real world clinical outcomes of macitentan therapy in chronic thromboembolic pulmonary hypertension

Cited by 7 publications

References 20 publications

The clinical experience of macitentan in pulmonary hypertension in Indian cohort: 12-month follow-up

The clinical experience of macitentan in pulmonary hypertension in Indian cohort: 12-month follow-up

Results of Macitentan in Japanese Patients With Chronic Thromboembolic Pulmonary Hypertension ― A Prospective, Multicenter, Open-Label, Single-Arm, Phase 3 Study ―

Diagnosis and Treatment Patterns of Chronic Thromboembolic Pulmonary Hypertension in Russia, Kazakhstan, Turkey, Lebanon, and Saudi Arabia: A Registry Study

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Results of Macitentan in Japanese Patients With Chronic Thromboembolic Pulmonary Hypertension　― A Prospective, Multicenter, Open-Label, Single-Arm, Phase 3 Study ―