Oral Selexipag Safe, Effective in Children With Pulmonary Arterial Hypertension - Pulmonology Advisor
Oral Selexipag Safe, Effective in Children With Pulmonary Arterial Hypertension - Pulmonology Advisor |
| Posted: 27 May 2020 08:30 AM PDT  With close monitoring, the use of oral add-on treatment with selexipag in children with pulmonary arterial hypertension (PAH) was safe and well tolerated, according to the results of a prospective, observational study published in The Journal of Heart and Lung Transplantation. Researchers from the European Pediatric Pulmonary Vascular Disease Network (EPPVDN) conducted in consecutive children with PAH who were treated with oral selexipag at 3 tertiary pulmonary hypertension (PH) centers. The researchers sought to evaluate the efficacy and safety of the oral prostacyclin receptor agonist selexipag, which has been approved for use in adult patients with PAH. The current analysis is the largest, exploratory pediatric cohort to date. A total of 15 children from the 3 PH centers were assessed: 7 from Hannover, Germany; 6 from Graz, Austria; and 2 from Gothenburg, Sweden. The age range of the participants was 7 months to 17 years. Patients' body weight ranged from 5 kg to 73 kg. All of the patients underwent cardiac catheterization at baseline and at a median of 8 months' follow-up. Moreover, the participants all received clinical, echocardiographic, and N-terminal pro B-type natriuretic peptide evaluations, including EPPVDN pediatric PH risk score. No deaths were reported with the use of selexipag. Of the 15 patients, 2 ultimately received a lung transplantation. There was 1 death reported in a patient with heritable PAH who had previously received selexipag therapy and was being treated with intravenous treprostinil. Of the 10 participants who received comparable sedation at both times of cardiac catheterization, the use of selexipag was associated with a significant improvement in mean right arterial pressure (-2 mm Hg), mean pulmonary arterial pressure to mean systemic arterial pressure ratio (-17%; P <.05), mean transpulmonary pressure gradient (-17%; P <.01), and diastolic TPG (-5.8 mm Hg; P <.05) at 5 to 18 months of follow-up vs baseline. Additionally, at follow-up, patients treated with selexipag tended to have a lower pulmonary vascular resistance (PVR) index (-13%; P =.131) and PVR to systemic vascular resistance ratio (-20%; P =.097), as well as a higher cardiac index (Qsi, +18%; P =.322). On the whole, the efficacy of selexipag on invasive hemodynamics was variable and was frequently better in those patients who were less ill. The investigators concluded that the results from this study need to be confirmed in larger prospective studies of PAH to clarify the broader applicability and utility of selexipag in clinical care. Disclosure: Several study authors declared affiliations with the pharmaceutical industry. Please see the original reference for a full list of authors' disclosures. Reference Hansmann G, Meinel K, Bukova M, Chouvarine P, Wåhlander H, Koestenberger M; on behalf of the European Pediatric Pulmonary Vascular Disease Network (EPPVDN). Selexipag for the treatment of children with pulmonary arterial hypertension: first multicenter experience in drug safety and efficacy [published online April 7, 2020]. J Heart Lung Transplant. doi:10.1016/j.healun.2020.03.029  |
| Kalytera to Acquire R-107, Potential Liquid Nitric Oxide Treatment... - Pulmonary Hypertension News Posted: 27 May 2020 07:00 AM PDT Kalytera Therapeutics intends to acquire Salzman Group, a company developing R-107 — a liquid form of nitric oxide — to possibly treat pulmonary arterial hypertension (PAH), lung disease associated with COVID-19, and other lung disorders, the company announced. Its acquisition of the Massachusetts-based company should be completed by June 15. R-107 is a liquid prodrug of nitric oxide (NO), a strong vasodilator that opens blood vessels and is used to treat multiple respiratory conditions. In the lung, NO reduces blood flow resistance to improve oxygenation. At high concentrations, NO is involved in the activity of the immune system in fighting infections. A prodrug is a medication or compound that, after administration, is metabolized (i.e., converted within the body) into a pharmacologically active drug. Nitric oxide gas is usually administered via high-pressure cylinders in a hospital, and require handling by trained respiratory therapists. Companies are working to design easier ways of allowing for its use by patients. R-107 is a liquid formulation of NO administered by a single intramuscular injection. According to Kalytera, the injection is fairly easy and the therapy's potency is sustained for prolonged periods following administration. This is in contrast with other NO liquid form products, such as nitroglycerin, which rapidly induces tolerance and loses biological activity after more than one administration. Preclinical data with animal models of PAH showed that treatment with R-107 led to sustained relief, with pulmonary arterial blood pressure dropping by 70% after a single dose of R-107. The decrease was sustained for more than two days (48 hours). R-107 also outperformed similar therapies tested in the same animal models of PAH in terms of potency and duration of response. It is also being developed as a potential treatment for chlorine inhalation lung injury and for COVID-19 associated lung disease. A study showed that NO inhalation has an inhibitory effect on the replication of the severe acute respiratory syndrome coronavirus (SARS CoV), a virus of the same family as SARS-CoV2 virus — the viral strain that causes COVID-19. A Phase 2 trial (NCT04306393) is evaluating whether inhaled NO improves oxygenation in hospitalized patients with very low oxygen levels due to SARS-CoV2. The trial is underway in the U.S., and sponsored by Massachusetts General Hospital. The Salzman Group aims to test R-107 in patients with COVID-19-related acute respiratory distress syndrome who require intubation and mechanical ventilation. The company plans to submit an application for a Biomedical Advanced Research and Development Authority (BARDA) contract to speed the therapy's consideration for approval by the FDA.  Patricia holds her Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She also served as a PhD student research assistant in the Laboratory of Doctor David A. Fidock, Department of Microbiology & Immunology, Columbia University, New York. × Patricia holds her Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She also served as a PhD student research assistant in the Laboratory of Doctor David A. Fidock, Department of Microbiology & Immunology, Columbia University, New York. Latest Posts  |
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