Opsumit (macitentan) / Nippon Shinyaku, J&J - LARVOL DELTA

BUSULFAN ASSOCIATED PULMONARY TOXICITY AND INFECTIOUS COMPLICATIONS FOLLOWING AUTOLOGOUS SCT IN A 3-YEAR-OLD CHILD WITH NEUROBLASTOMA (EBMT 2026) - "Background: High-dose busulfan/melphalan (Bu/Mel) remains a standard conditioning regimen for autologous stem cell transplantation in high-risk neuroblastoma but is associated with significant organ toxicity, particularly hepatic and pulmonary injury...A treatment with Defibrotide, Ganciclovir and Azithromycin was initiated...We started a treatment with Sildenafil, Macitentan, Epoprostenol in addition to Methylprednisolone pulse therapy and antifibrotic treatment with Nintedanib, Hydroxychloroquin and Azithromycin...We started a treatment with Sildenafil, Macitentan, Epoprostenol in addition to Methylprednisolone pulse therapy and antifibrotic treatment with Nintedanib, Hydroxychloroquin and Azithromycin...We started a treatment with Sildenafil, Macitentan, Epoprostenol in addition to Methylprednisolone pulse therapy and antifibrotic treatment with Nintedanib, Hydroxychloroquin and Azithromycin... This case illustrates the complex interplay between infectious..."

Clinical • Acute Respiratory Distress Syndrome • Cardiovascular • Cystic Fibrosis • Cytomegalovirus Infection • Gastroenterology • Genetic Disorders • Hepatology • Immunology • Infectious Disease • Inflammation • Interstitial Lung Disease • Neuroblastoma • Pneumonia • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases • Septic Shock • Solid Tumor

MAC-NOCA: Efficacy and Mechanisms of Macitentan for Non-Coronary Obstructive Angina (clinicaltrials.gov) - P1 | N=40 | Recruiting | Sponsor: China-Japan Friendship Hospital

New P1 trial • Cardiovascular

Macitentan for Pulmonary Arterial Hypertension Associated with Systemic Sclerosis: results from the European Scleroderma Trials And Research Group (EUSTAR) database (SSWC 2026) - No abstract available

Cardiovascular • Immunology • Pulmonary Arterial Hypertension • Respiratory Diseases • Scleroderma • Systemic Sclerosis

Real-world observational study on pulmonary arterial hypertension: Italian cohort treated with macitentan and/or selexipag as a part of a combination treatment (INSPECTIO). (PubMed, Vascul Pharmacol) - "The INSPECTIO study supports the effectiveness of guideline-directed therapy and regular risk assessment to optimize treatment strategies in PAH. The increase in non-invasive low-risk criteria suggests a stabilization of disease over 12 months."

Journal • Observational data • Real-world evidence • Cardiovascular • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

Case Report: Immune checkpoint inhibitor-associated pulmonary hypertension. (PubMed, Front Cardiovasc Med) - "We report a case of a 65-year-old male who developed severe pulmonary arterial hypertension (PAH) following treatment with the programmed cell death protein 1 inhibitor tislelizumab. Following the initiation of a triple combination targeted therapy for PAH comprising macitentan, riociguat, and treprostinil, the patient's condition improved significantly...To date, no specific diagnostic or treatment guidelines exist for this condition due to its rarity. Therefore, there is an urgent need for more case reports, shared experiences, and clinical research to assist clinicians in identifying optimal strategies for the diagnosis and management of this complication."

Checkpoint inhibition • Journal • Cardiovascular • Gastrointestinal Disorder • Hypertension • Oncology • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

Overall Patient Characteristics from UNISUS, a Phase 3 Superiority Study Comparing Macitentan 75 mg vs Macitentan 10 mg in Patients with Pulmonary Arterial Hypertension (PAH) (ISHLT 2026) - "Abstract is embargoed at this time."

Clinical • Head-to-Head • P3 data • Cardiovascular • Pulmonary Arterial Hypertension • Respiratory Diseases

PHARMACOKINETICS (PK) AND SAFETY OF MACITENTAN 75 MG: PHASE I DATA FROM CHINESE AND WHITE HEALTHY ADULTS (ACC 2026) - "Abstract is embargoed at this time."

Clinical • P1 data • PK/PD data • Cardiovascular

Macitentan and phosphodiesterase-5 inhibitor alone or in combination in newly diagnosed pulmonary arterial hypertension: a pooled analysis. (PubMed, JHLT Open) - "This large, pooled analysis suggests an observed statistical association indicating a potential survival benefit for early macitentan+PDE5i versus either monotherapy in newly diagnosed PAH."

Journal • Retrospective data • Cardiovascular • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

Liver Transplantation for a Patient With Hemophilia and HIV/HCV Coinfection Complicated by Pulmonary Hypertension. (PubMed, Hepatol Res) - "Preoperative evaluation revealed pulmonary arterial hypertension, which improved with sildenafil citrate, macitentan, and home oxygen therapy. The patient was discharged on postoperative day 51 with normal liver function and good overall condition. This case underscores the importance of multidisciplinary collaboration and careful perioperative planning in achieving successful outcomes in liver transplantation for patients with complex comorbidities such as hemophilia, HIV/HCV coinfection, and pulmonary hypertension."

Journal • Cardiovascular • Hematological Disorders • Hemophilia • Hemophilia B • Hepatitis C • Human Immunodeficiency Virus • Hypertension • Infectious Disease • Pulmonary Arterial Hypertension • Pulmonary Disease • Rare Diseases • Respiratory Diseases • Transplantation

A Study to Assess Whether Macitentan Delays Disease Progression in Children With Pulmonary Arterial Hypertension (PAH) (clinicaltrials.gov) - P3 | N=165 | Completed | Sponsor: Actelion | Active, not recruiting ➔ Completed

Trial completion • Cardiovascular • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

A patient-derived CRISPR platform reveals selective dependencies in Acute Myeloid Leukemia (ASH 2025) - "Our results indicate that EDNRAknockout inhibits AML cell proliferation and growth, highlighting its potential as a therapeutic target.Furthermore, EDNRA is overexpressed in AML compared to normal cells, and since FDA approved EDNRAinhibitors such as Macitentan, Bosentan, Ambrisentan, and Clazosentan are already being used to treatpulmonary arterial hypertension, this presents a promising opportunity to evaluate these inhibitors aspotential chemotherapy-sensitizing agents for high-risk AML.SOD1 plays a critical role in eliminating toxicradicals generated within biological systems and has been associated with poor outcomes in AML. This study demonstrates the establishment of a scalable platform for CRISPR screening inprimary AML cells for identification of more conserved vulnerabilities that may be exploitedtherapeutically, with EDNRA as a potential target. Ongoing work includes validating key targets with thegoal of advancing therapeutic strategies. Future studies will..."

Clinical • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Pulmonary Arterial Hypertension • Respiratory Diseases • EDNRA • HOXA9 • MEIS1 • PPM1D • SUZ12

Persistent Pulmonary Hypertension in Corrected Congenital Left Circumflex Artery to Coronary Sinus Fistula: A Case Report and Literature Review. (PubMed, Case Rep Cardiol) - "Postoperatively, the patient remained symptomatic, with elevated pulmonary arterial pressure persisting, prompting the initiation of triple therapy comprising macitentan, selexipag, and sildenafil. The case illustrates a rare case of persistent PHTN in a corrected LCx-CS fistula. Thorough follow-up, early diagnosis, and timely interventions, complemented by pharmacotherapy when necessary, are essential in managing these complex clinical scenarios."

Journal • Cardiovascular • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

Selection of Endothelin Receptor Antagonists in the Treatment of Pulmonary Arterial Hypertension: A Comprehensive Narrative Review. (PubMed, Adv Ther) - "Endothelin receptor antagonists (ERAs) are established treatments for PAH, and three agents-bosentan, ambrisentan, and macitentan-are approved for use in adults in the USA. It also discusses the differing physicochemical and pharmacokinetic properties of each ERA and how these unique characteristics influence their receptor binding and kinetics, mechanisms of action, therapeutic effects, dosing frequency, and safety in PAH. In the absence of head-to-head clinical trials assessing their comparative efficacy and safety, it is important to understand both the similarities and the distinguishing characteristics of the three ERAs approved in PAH, to inform individualized treatment selection."

Journal • Review • Cardiovascular • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

Acute Decompensated Heart Failure in a Woman With Eisenmenger Syndrome: A Case Report. (PubMed, Cureus) - "It emphasizes the importance of maintaining a high index of suspicion in the presence of chronic hypoxia and abnormal hematologic parameters. We discuss the role of echocardiography and adjunctive imaging in diagnosis, review the pathophysiologic mechanisms underlying ES, and outline current therapeutic strategies, including the use of endothelin receptor antagonists in the management of pulmonary arterial hypertension (PAH)."

Journal • Cardiovascular • Congestive Heart Failure • Heart Failure • Hematological Disorders • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

The Involvement of the Peptidergic Systems in Breast Cancer Development. (PubMed, Cancers (Basel)) - "Breast cancer cells overexpress peptide receptors; at the same time they are known to interact with peptides that (a) exert an oncogenic action (adrenomedullin 2, endothelin, gastrin-releasing peptide, neurokinin A, neuromedin, neuropeptide Y, neurotensin, substance P, vasoactive intestinal peptide), (b) exert an anticancer action (angiotensin (1-7), ghrelin, peptide YY) or (c) exert dual oncogenic and anticancer effects (adrenomedullin, angiotensin II, bradykinin, corticotropin-releasing factor, β-endorphin, glucagon-like peptide 1, gonadotropin-releasing hormone, kisspeptin, methionine-enkephalin, oxytocin)...Future lines of research are suggested in breast cancer using promising anti-breast-cancer peptide receptor antagonists (HOE-140, exendin (9-39), bosentan, macitentan, PD168,368, CGP71,683A, SR48,692, aprepitant) or agonists (FR190,997, semaglutide, exendin 4, goserelin) mentioned in this review...Taken together, the available data highlight the enormous promise..."

Journal • Review • Breast Cancer • Oncology • Solid Tumor • GRP-10

Results of the MACiTEPH study of macitentan for the treatment of inoperable or persistent/recurrent chronic thromboembolic pulmonary hypertension. (PubMed, J Heart Lung Transplant) - P3 | "MACiTEPH was discontinued for futility, as no treatment effect on 6MWD (primary endpoint) was observed. Despite the higher discontinuation rate in the macitentan 75 mg group, no unexpected safety signals were observed in CTEPH patients."

Journal • Cardiovascular • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Pulmonary Embolism • Respiratory Diseases

Characteristics, Treatment Patterns and Outcomes of Patients with Pulmonary Arterial Hypertension by Race and Ethnicity Using Real-World Data from the Combined OPUS/OrPHeUS Studies. (PubMed, Adv Ther) - P | "OPUS/OrPHeUS provides real-world insights into racial/ethnic minority groups receiving macitentan and other PAH-specific treatments in the USA. These data on treatment patterns and outcomes could help inform treatment decisions in the reported minority groups."

Journal • Real-world evidence • Cardiovascular • Heart Failure • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases • Rheumatology

Macitentan Plus Tadalafil Single-Tablet Combination Therapy in Chinese Patients With Pulmonary Arterial Hypertension: A Subgroup Analysis of the A DUE Study. (PubMed, Pulm Circ) - P3 | "Our findings support the use of M/T STCT for PAH in China. Trial registration: ClinicalTrials.gov https://clinicaltrials.gov/ NCT03904693 (April 5, 2019)."

Journal • Cardiovascular • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

Sotatercept-Enhanced Quadruple Therapy Reverses Hemodynamic Decline in Severe Systemic Lupus Erythematosus-Associated Pulmonary Arterial Hypertension (AHA 2025) - "Treatment included high-dose corticosteroids, mycophenolate mofetil, rituximab, tadalafil, macitentan, and continuous epoprostenol infusion. This case demonstrates that a proactive, multidisciplinary approach incorporating novel therapies such as sotatercept can reverse the trajectory of life-threatening SLE-PAH. Timely diagnosis, aggressive immunosuppression, and layered vasodilator regimens can restore cardiopulmonary function and delay or avoid transplantation in patients with advanced disease."

Cardiovascular • Glomerulonephritis • Immunology • Inflammatory Arthritis • Lupus • Lupus Nephritis • Nephrology • Pulmonary Arterial Hypertension • Respiratory Diseases • Systemic Lupus Erythematosus

Case report: Successful use of sotatercept to treat pulmonary arterial hypertension in a patient with hereditary hemorrhagic telangiectasia. (PubMed, Respir Med Case Rep) - "Despite long-term treatment with epoprostenol, treprostinil, macitentan, and tadalafil, her condition progressed, leading to evaluation for lung transplantation. This is the first reported case of sotatercept use in HHT-associated PAH, highlighting its potential efficacy but also the need for careful monitoring due to increased bleeding risk. Further studies are required to establish the safety and efficacy of sotatercept in this population."

Journal • Cardiovascular • Heart Failure • Hematological Disorders • Hypertension • Otorhinolaryngology • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases • Transplantation • TGFB1

Interactions between Integrin α9β1 and VCAM-1 Promote Neutrophil Hyperactivation and Deep Vein Thrombosis in a Murine Model of Lung Cancer (ASH 2023) - "Virtual screening and cell-free assay identified macitentan as a potent inhibitor of integrin α9-VCAM-1 interactions... Using genetic and pharmacological approaches in combination with transcriptomic and pharmacological studies, we described a previously unknown pathway involving integrin α9 and VCAM-1 in promoting cancer-associated thrombosis. We found that integrin α9-VCAM1 axis is critical for neutrophil hyperactivation, and its pharmacological inhibition significantly reduces DVT severity. The results of this study suggest that neutrophil integrin α9 is a key factor regulating neutrophil hyperactivation and cancer-associated thrombosis."

Preclinical • Cardiovascular • Lung Cancer • Oncology • Solid Tumor • Thrombosis • Venous Thromboembolism • ELANE • MPO • VCAM1

Macitentan in the Treatment of Digital Ulcers in Patients with Systemic Rheumatic Autoimmune Diseases: A National Multicenter Study of 42 Patients. (PubMed, J Clin Med) - "GI and higher ulcer burden predicted diminished treatment response in SSc patients. These results support the use of MACI as a valuable therapeutic option for severe digital vasculopathy in SARDs, although further prospective studies are warranted to confirm these observations."

Clinical • Journal • Cardiovascular • Immunology • Rheumatology • Scleroderma • Systemic Sclerosis

Preferences for pulmonary arterial hypertension (PAH) medications: plain language summary on insights from interviews with patients and doctors in the A DUE phase 3 clinical study. (PubMed, Future Cardiol) - No abstract available

Interview • Journal • P3 data • Cardiovascular • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

Severe Pulmonary Hypertension in a Young Adult with Congenital Diaphragmatic Hernia: A Challenging Case of Multifactorial Developmental Lung Disease. (AHA 2025) - "He developed severe PH requiring extracorporeal membrane oxygenation (ECMO), prostaglandins, and prolonged dual therapy with endothelin receptor antagonists (bosentan, macitentan)...At age 20, clinical deterioration followed the introduction of selexipag, with worsening hypoxemia, increased oxygen requirements, and suspicion of pulmonary vascular disease progression...This case underscores the complex trajectory of PH in survivors of CDH and the risks associated with transitions in pulmonary vasodilator therapy. It highlights the need for long-term multidisciplinary follow-up and raises concerns about late-onset pulmonary vascular deterioration. Novel strategies and biomarkers are needed to better phenotype and risk-stratify this unique patient population.Use of AI tools:No automated editing assistance was used."

Clinical • Cardiovascular • Developmental Disorders • Gastroenterology • Psychiatry • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

DOUBLE TROUBLE: A CASE OF DASATINIB-INDUCED PULMONARY ARTERIAL HYPERTENSION AND RECURRENT CHYLOTHORAX (CHEST 2025) - "She was started on tadalafil, macitentan, and orenitram...Bosutinib was discontinued, and she was started on asciminib, with no further recurrence of effusions... While generally well tolerated, this case highlights rare manifestations of TKI side effects and underscores importance of evaluating pulmonary symptoms that coincide with the initiation of these therapies."

Clinical • Cardiovascular • Chronic Myeloid Leukemia • Congestive Heart Failure • Diabetes • Genetic Disorders • Heart Failure • Hematological Malignancies • Leukemia • Metabolic Disorders • Obesity • Obstructive Sleep Apnea • Pulmonary Arterial Hypertension • Respiratory Diseases • Sleep Disorder • Type 2 Diabetes Mellitus