Ryan Haumschild, PharmD, MS, MBA

Panelists discuss how the future of PAH treatment looks promising with potential for disease remission through reverse remodeling agents, emphasizing the need for continued research focus on patients with other forms of pulmonary hypertension and those with significant comorbidities.

Panelists discuss how cost-effectiveness analyses of newer therapies like sotatercept show substantial benefits in reducing clinical worsening events and potentially decreasing downstream costs through reduced hospitalizations and transplantations, supporting value-based payment models.

Panelists discuss that improving outcomes for patients with idiopathic pulmonary fibrosis (IPF) and progressive pulmonary fibrosis (PPF) requires multifaceted efforts including developing patient-centered clinical measures, managing comorbidities, leveraging digital health tools for real-time monitoring, and addressing health equity to ensure timely, personalized care that truly reflects patients’ lived experiences.

Panelists discuss the shift toward patient-centered outcomes in idiopathic pulmonary fibrosis and progressive pulmonary fibrosis treatment, emphasizing quality of life and symptom relief alongside lung function, while expressing optimism that emerging therapies may eventually reverse or prevent fibrosis, fundamentally transforming disease management.

Panelists discuss how emerging therapies like CAR T cells and bispecific antibodies may transform frontline treatment by potentially replacing transplant or changing induction regimens, while considering the cost implications and need for sustainable care models.

Panelists discuss how early relapse in standard-risk patients represents a failure of current risk assessment methods and may require advanced sequencing technologies to identify hidden high-risk features that traditional fluorescence in situ hybridization testing misses.

Panelists discuss how mirdametinib's oral administration offers convenience and improves compliance, especially in pediatric patients, and how its ability to stabilize progressive, symptomatic NF1-associated plexiform neurofibromas without the need for invasive procedures fills a critical gap in treatment options while also addressing potential access barriers related to insurance coverage and cost.

Panelists discuss how patient financial burden varies significantly by insurance type and often leads to treatment interruptions, while prior authorization barriers create administrative challenges that could be improved through better collaboration between medical and payer communities.

Panelists discuss how healthcare resource utilization is primarily driven by hospitalizations, medication costs, and outpatient monitoring, with early diagnosis and treatment potentially reducing long-term costs by preventing disease progression and avoiding expensive advanced therapies.

Panelists discuss how emerging, better-tolerated oral therapies for idiopathic pulmonary fibrosis (IPF) and progressive pulmonary fibrosis (PPF) could improve patient adherence and simplify treatment regimens, potentially enabling combination approaches that target multiple disease pathways while reducing polypharmacy and health care costs.

Panelists discuss recent trial data demonstrating that a novel agent not only slows lung function decline but also delays oxygen dependency and improves survival in both idiopathic pulmonary fibrosis (IPF) and a broad progressive pulmonary fibrosis (PPF) population, with consistent safety and tolerability supporting its expanding role in fibrotic lung disease management.

Panelists discuss how autologous stem cell transplant (ASCT) deferral should be approached cautiously with concrete medical reasons, as transplant continues to provide superior progression-free survival and potentially curative outcomes for a subset of patients.

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.