Ryan Haumschild, PharmD, MS, MBA

Panelists discuss how maintenance therapy should be tailored based on risk profiles, with standard-risk patients receiving single-agent lenalidomide while high-risk patients may benefit from combination maintenance strategies to achieve more durable responses.

Panelists discuss the promising results of the ReNeu trial for mirdametinib, highlighting its significant tumor shrinkage and improvements in quality of life for patients with NF1-associated plexiform neurofibromas, and how mirdametinib’s favorable adverse effect profile and effectiveness in complex or refractory cases may provide a valuable treatment option in real-world clinical practice.

Panelists discuss how managing patients with comorbidities requires careful evaluation to distinguish true Group 1 PAH features from underlying cardiac or pulmonary disease, with a more measured treatment approach and close monitoring for complications.

Panelists discuss how prostacyclin pathway treatments remain important but challenging due to significant side effects and delivery complexity, with sotatercept potentially reducing reliance on parenteral prostacyclins while maintaining their role in high-risk patients with low cardiac output.

Panelists discuss recent phase 3 trial results of a novel oral agent for idiopathic pulmonary fibrosis (IPF) and progressive pulmonary fibrosis (PPF), highlighting its ability to significantly reduce lung function decline and mortality—even when added to existing antifibrotic therapies—while maintaining a favorable safety and tolerability profile.

Panelists discuss exciting phase 3 trial data on a novel selective phosphodiesterase inhibitor that elevates intracellular cyclic AMP (cAMP) to activate anti-inflammatory and antifibrotic pathways, showing promise in slowing disease progression in idiopathic pulmonary fibrosis (IPF) and progressive pulmonary fibrosis (PPF).

Panelists discuss how minimal residual disease (MRD) status can guide post-transplant treatment decisions, particularly for high-risk patients who don’t achieve MRD negativity and patients considering discontinuation of long-term maintenance therapy.

Panelists discuss key lessons learned from previous NF1-associated plexiform neurofibroma therapies, including the limitations of traditional treatments due to high toxicity and limited efficacy, and how these challenges are driving the development of more targeted therapies like MEK inhibitors, personalized medicine, and combination strategies to improve outcomes and reduce relapse.

Panelists discuss how quadruplet regimens show improved efficacy over triplet regimens without significantly increased safety concerns, with anti-CD38 antibodies being well tolerated and NCCN guidelines now recommending 4-drug regimens for transplant-eligible patients.

Panelists discuss how sotatercept represents a paradigm shift as the first activin signaling inhibitor showing dramatic improvements in heavily pretreated patients, with the Stellar and Zenith trials demonstrating significant reductions in clinical worsening and potential for reverse remodeling.

Panelists discuss how riociguat targets the nitric oxide pathway and is approved for both Group 1 PAH and Group 4 chronic thromboembolic pulmonary hypertension, with specific patient selection criteria based on risk stratification and surgical candidacy.

Panelists discuss several promising new agents for idiopathic pulmonary fibrosis (IPF) and progressive pulmonary fibrosis (PPF), including an LPA1 receptor antagonist showing lung function preservation, integrin inhibitors targeting fibrotic pathways despite safety challenges, and inhaled prostaglandins being evaluated as a well-tolerated add-on therapy in ongoing trials.

Panelists discuss how antifibrotic therapies for idiopathic pulmonary fibrosis (IPF) and progressive pulmonary fibrosis (PPF) slow disease progression despite challenges with cost, tolerability, and limited symptom relief, emphasizing the importance of patient education and aligning treatment goals.

Panelists discuss the critical role of psychosocial support in managing NF1-associated plexiform neurofibromas, emphasizing the need for psychotherapy, counseling, and social work interventions to address anxiety, depression, and social stigma while also highlighting challenges in accessing therapies like selumetinib due to cost, insurance barriers, and health care system disparities.

Panelists discuss how treatment strategies differ between transplant-eligible and transplant-ineligible multiple myeloma patients, with transplant eligibility determined by functional status rather than age alone, and how quadruple therapy regimens are increasingly used for transplant-eligible patients to achieve deep, durable responses.

Panelists discuss how autologous stem cell transplant remains essential in frontline multiple myeloma management despite improved quadruple therapy outcomes, emphasizing that current evidence still supports transplantation for eligible patients rather than deferring it.

Panelists discuss how current therapeutic strengths include 15 approved treatments with different mechanisms of action that can work synergistically, while limitations involve variable delivery methods and complexity, leading to more aggressive upfront combination therapy approaches.

Panelists discuss how treatment individualization depends on patient-specific factors including age, comorbidities, disease severity, and personal preferences, balancing clinical guidelines with shared decision-making to optimize both efficacy and adherence.

Panelists discuss how effective management of idiopathic pulmonary fibrosis (IPF) and progressive pulmonary fibrosis (PPF) relies on early, accurate diagnosis and multidisciplinary collaboration, with a focus on personalized treatment strategies, proactive adverse effect management, and supportive interventions like pulmonary rehabilitation and nutrition to enhance quality of life and improve long-term outcomes.

Panelists discuss how managing idiopathic pulmonary fibrosis (IPF) and progressive pulmonary fibrosis (PPF) requires a comprehensive, patient-centered approach that addresses not only physiological decline but also the significant psychological, social, and functional burdens patients face—highlighting the need for early diagnosis, emotional support, and coordinated care to improve both quality of life and health care outcomes.

Panelists discuss how disease progression monitoring requires integrating multiple data points including symptoms, six-minute walk tests, biomarkers like BNP/NT-proBNP, and imaging studies, with risk stratification tools helping guide treatment decisions while considering individual patient characteristics.

Panelists discuss how early detection barriers include nonspecific symptoms like progressive dyspnea that are often misattributed to more common conditions, leading to delayed diagnosis and the need for improved diagnostic algorithms using biomarkers and imaging.

Panelists discuss the exploration of combination therapies in treating complex or refractory NF1-associated plexiform neurofibromas, including MEK inhibitors combined with PI3K inhibitors, anti-angiogenic agents, and chemotherapies, and review early clinical findings suggesting potential synergistic effects, while highlighting concerns about toxicity and the need for careful patient monitoring.

Panelists discuss key findings supporting the use of MEK inhibitors, such as selumetinib, in treating NF1-associated plexiform neurofibromas, emphasizing its impact on clinical decision-making, particularly for pediatric patients with progressive, symptomatic tumors who are not surgical candidates, and review current guidelines recommending its use to control tumor growth and improve quality of life.

Panelists discuss how progressive pulmonary fibrosis (PPF) and idiopathic pulmonary fibrosis (IPF), though distinct in etiology, share a common trajectory of irreversible lung scarring and functional decline—highlighting the importance of recognizing progressive phenotypes across interstitial lung diseases to guide timely diagnosis, personalized treatment, and improved patient outcomes.

Panelists discuss how progressive pulmonary fibrosis (PPF) and idiopathic pulmonary fibrosis (IPF), though distinct in etiology, share a common trajectory of irreversible lung scarring and functional decline—highlighting the importance of recognizing progressive phenotypes across interstitial lung diseases to guide timely diagnosis, personalized treatment, and improved patient outcomes.

Panelists discuss how MEK inhibitors, such as trametinib and selumetinib, are emerging as first-line treatments for symptomatic NF1-associated plexiform neurofibromas, especially in progressive or inoperable cases, while emphasizing the importance of surgical resection, pain management, physical therapy, and psychosocial support for improving quality of life.

Panelists discuss the current management goals for NF1-associated plexiform neurofibromas, focusing on tumor control, symptom management, and prevention of malignant transformation, while highlighting the evolving future goals of targeted therapies, personalized treatment plans, and enhanced early detection to improve outcomes.

Panelists discuss how pulmonary arterial hypertension diagnosis requires comprehensive evaluation including right heart catheterization to confirm hemodynamic criteria, with normal mean PA pressure being ≤20 mmHg and the need to rule out other causes of pulmonary hypertension.

Panelists discuss how the pathophysiology of pulmonary arterial hypertension involves complex mechanisms across multiple genetic and treatment pathways, with over 20 identified genes and four major therapeutic targets including nitric oxide, endothelin, prostacyclin, and activin signaling inhibition.