Publications by authors named "Janethe de Oliveira Pena"
Article Synopsis
- * Enhanced exposure-response and pharmacokinetic/pharmacodynamic modeling revealed that higher concentrations of sotatercept improve key clinical outcomes including 6MWD and the likelihood of achieving lower NT-proBNP levels, while maintaining safety measures like hemoglobin levels.
- * Key factors influencing the treatment's response include patient age and iron supplementation, but their overall impact on outcomes was minimal, supporting the selected dosing strategy of 0.7 mg/kg administered subcutaneously every three weeks as effective and safe.
Efficacy and Safety of Sotatercept Across Ranges of Cardiac Index in Patients with Pulmonary Arterial Hypertension: A Pooled Analysis of PULSAR and STELLAR.
J Heart Lung Transplant
December 2024
Background: This analysis compared effects of the activin signaling inhibitor, sotatercept, across pulmonary arterial hypertension (PAH) subgroups stratified by baseline cardiac index (CI).
Methods: Pooled data from PULSAR (N=106; NCT03496207) and STELLAR (N=323; NCT04576988) were analyzed using two different CI thresholds, < and ≥ 2.0 L/min/m or 2.
Article Synopsis
- Sotatercept is a newly FDA-approved biologic for treating pulmonary arterial hypertension (PAH), which shows promising pharmacokinetic (PK) properties based on clinical studies.
- A population pharmacokinetic model was created using data from various studies, analyzing different dosing methods and durations, which included a total of 350 participants.
- The analysis revealed that body weight and a patient's baseline albumin level influence drug clearance and volume but do not significantly affect sotatercept's overall disposition, supporting its use as a subcutaneous treatment for PAH.
Article Synopsis
- - This study evaluated the effects of sotatercept on exercise tolerance, capacity, and right ventricular function in patients with pulmonary arterial hypertension through the SPECTRA clinical trial.
- - Results showed that out of 21 participants, there was a significant increase in peak oxygen uptake after 24 weeks of treatment, along with improvements in exercise hemodynamics and walking distance.
- - The findings suggest that sotatercept could be a promising new treatment option for pulmonary arterial hypertension, potentially reversing heart function decline.
Article Synopsis
- Sotatercept is a new protein being developed to treat pulmonary arterial hypertension (PAH) by inhibiting activin signaling, and it was tested in a phase III clinical trial called STELLAR.
- In the study, 162 participants were treated either with sotatercept or a placebo, and about 25.9% developed antidrug antibodies (ADAs), with a small percentage also showing neutralizing antibodies.
- The presence of ADAs did not significantly impact the drug's effectiveness, safety, or how the body processed the drug, indicating that sotatercept remains a viable treatment option for patients with PAH.
Article Synopsis
- Pulmonary arterial hypertension (PAH) is a serious disease, and the STELLAR trial evaluated the effects of sotatercept combined with background therapy (BGT) compared to placebo with BGT.* -
- A Markov-type model was used to project long-term outcomes, showing that those receiving sotatercept plus BGT gained an average of 11.5 additional years of life expectancy and reduced hospitalizations and transplants compared to BGT alone.* -
- The study concluded that sotatercept significantly improves the life expectancy of PAH patients while decreasing the need for infused prostacyclin and reducing hospital stays, but real-world validation of these results is necessary.*
Article Synopsis
- The STELLAR trial tested the drug sotatercept, showing it improved walking distance and other health measures in patients with pulmonary arterial hypertension (PAH).
- Data from right heart catheterization and echocardiography were analyzed over 24 weeks to assess the drug's effects on heart and lung parameters.
- Results indicated significant reductions in pulmonary artery pressure and resistance, along with improvements in right heart function, indicating sotatercept's potential effectiveness for PAH patients.
Article Synopsis
- * A phase 3 clinical trial tested sotatercept against a placebo in adults with PAH who were already on stable therapy, focusing on improvements in exercise capacity measured by walking distance over a 24-week period, along with several secondary health measures.
- * Results from the trial indicated that patients taking sotatercept had a significant improvement in their 6-minute walk distance compared to those on placebo, showing a median increase of 34.4 meters versus just
Article Synopsis
- In a study on pulmonary arterial hypertension, 24 weeks of sotatercept showed a significant decrease in pulmonary vascular resistance compared to a placebo, prompting further investigation into its long-term effects over 18-24 months.
- The PULSAR study involved a randomized, double-blind, placebo-controlled design, where participants receiving placebo were switched to sotatercept, while others who started on sotatercept continued their treatment to evaluate its safety and efficacy.
- Results indicated that 30.8% of participants experienced serious side effects, but overall, sotatercept demonstrated sustained clinical benefits and safety, with significant improvements noted in both primary and secondary efficacy endpoints.
Article Synopsis
- Pulmonary arterial hypertension (PAH) involves changes in lung blood vessels and poor outcomes, and the novel drug sotatercept aims to fix dysfunctional signaling in this process.* -
- In a 24-week study with 106 participants, those receiving sotatercept showed significant improvements in pulmonary vascular resistance compared to the placebo group, particularly at dosages of 0.3 mg and 0.7 mg.* -
- While sotatercept improved exercise capacity and reduced certain biomarkers, common side effects included lowered platelet count and increased hemoglobin, with one serious adverse event reported.*
Objectives: Riociguat is approved for pulmonary arterial hypertension and has antiproliferative, anti-inflammatory and antifibrotic effects in animal models of tissue fibrosis. We evaluated the efficacy and safety of riociguat in patients with early diffuse cutaneous systemic sclerosis (dcSSc) at high risk of skin fibrosis progression.
Methods: In this randomised, double-blind, placebo-controlled, phase IIb trial, adults with dcSSc of <18 months' duration and a modified Rodnan skin score (mRSS) 10-22 units received riociguat 0.
Patient perception of disease burden in diffuse cutaneous systemic sclerosis.
J Scleroderma Relat Disord
February 2020
Purpose: Systemic sclerosis is a rare multi-organ autoimmune rheumatic disease, resulting in progressive fibrosis of the skin/internal organs. This study aimed to understand the impact of diffuse cutaneous systemic sclerosis symptoms and disease burden from the patient's perspective.
Methods: This was a mixed methodology, market research study involving ethnography, structured interviews, video diaries, and patient tasks.
Objectives: Mortality and worsening of organ function are desirable endpoints for clinical trials in systemic sclerosis (SSc). The aim of this study was to identify factors that allow enrichment of patients with these endpoints, in a population of patients from the European Scleroderma Trials and Research group database.
Methods: Inclusion criteria were diagnosis of diffuse SSc and follow-up over 12±3 months.
Objectives: To determine whether progressive skin fibrosis is associated with visceral organ progression and mortality during follow-up in patients with diffuse cutaneous systemic sclerosis (dcSSc).
Methods: We evaluated patients from the European Scleroderma Trials and Research database with dcSSc, baseline modified Rodnan skin score (mRSS) ≥7, valid mRSS at 12±3 months after baseline and ≥1 annual follow-up visit. Progressive skin fibrosis was defined as an increase in mRSS >5 and ≥25% from baseline to 12±3 months.
Objective: To gain insight into clinical practice regarding referral, early diagnosis and other aspects of the management of patients with dcSSc in Europe and the USA.
Methods: Semi-structured interviews were conducted with 84 rheumatologists (or internal medicine physicians) and 40 dermatologists in different countries (the UK, France, Germany, Italy, Spain and the USA). Physicians were asked to identify key steps in the patient pathway relating to patient presentation, diagnosis and referral, in addition to other treatment and follow-up processes.
Unlabelled: RISE-SSc is a randomized, double-blind, placebo-controlled phase 2 study investigating the efficacy and safety of riociguat in patients with diffuse cutaneous systemic sclerosis (dcSSc). Based on positive results from riociguat trials in patients with pulmonary hypertension and chronic thromboembolic pulmonary hypertension in combination with the known antiproliferative and antifibrotic effects seen in animal models, patients with SSc may benefit from treatment with riociguat. Patients with SSc meeting the ACR/EULAR systemic sclerosis classification criteria with diffuse cutaneous SSc (dcSSc) subset per LeRoy criteria, and a disease duration of less than or equal to 18 months will be randomized to placebo or riociguat 0.
View Article and Find Full Text PDFBackground: The 12-week, phase III Pulmonary Arterial hyperTENsion sGC-stimulator Trial (PATENT)-1 study investigated riociguat in patients with pulmonary arterial hypertension (PAH). Here, we present a prospectively planned analysis of the safety and efficacy of riociguat in the subgroup of patients with PAH associated with connective tissue disease (PAH-CTD).
Methods: Patients with PAH-CTD were further classified post hoc as having PAH associated with systemic sclerosis or PAH-other defined CTD.