Ryan Haumschild, PharmD, MS, MBA

Panelists discuss how newer immune-based therapies and bispecific antibodies may enable fixed-duration treatment approaches that could eliminate the need for stem cell transplant in older but fit patients, potentially allowing for treatment-free intervals after achieving deep responses.

Panelists discuss how to balance achieving deeper MRD-negative responses against increased toxicity risks in transplant-ineligible patients by personalizing therapy through dose modifications, weekly vs twice-weekly dosing schedules, and careful monitoring while maintaining treatment intensity similar to clinical trials.

Panelists discuss how concurrent comorbidities, such as thyroid disorders and other autoimmune conditions, are commonly seen in patients with alopecia areata (AA) and how they complicate disease management and increase the overall burden on patients.

Panelists discuss how the epidemiology of alopecia areata (AA) varies across different patient populations, with certain forms of the disease being more prevalent in specific groups.

Panelists discuss the drivers and consequences of delayed diagnosis in hidradenitis suppurativa, emphasizing the need for provider education, clinical decision support, and multidisciplinary collaboration to promote earlier recognition and more effective, coordinated care.

Panelists discuss the challenges of timely diagnosis and comprehensive management of hidradenitis suppurativa, emphasizing the need for early recognition, multidisciplinary care, and empathy-centered approaches to address the physical, emotional, and social burden of the disease.

Panelists discuss current guidelines recommending mirdametinib for pediatric patients (2 years and older) with progressive, symptomatic NF1-associated plexiform neurofibromas that are inoperable or difficult to manage surgically, and emphasize the importance of early initiation, regular monitoring, and a multidisciplinary approach to optimize treatment outcomes.

Panelists discuss how NCCN guidelines are expected to incorporate quadruplet-based regimens as reasonable treatment approaches for transplant-ineligible patients, while emphasizing the need for personalized treatment strategies that consider individual patient frailty and high-risk genetics rather than applying uniform approaches across all older patients.

Panelists discuss how the CEPHEUS trial demonstrated that quadruplet therapy (daratumumab, bortezomib, lenalidomide, and dexamethasone) significantly improved minimal residual disease negativity rates compared to triplet therapy in transplant-ineligible multiple myeloma patients, achieving approximately 60% vs 47% 10–5 responses while maintaining manageable safety profiles.

Panelists discuss how biomarkers, including genetic testing and tumor profiling, can help identify patients with RAS/MEK/ERK pathway activation or specific NF1 mutations, enabling more personalized and effective treatment with therapies like mirdametinib or gene therapies for progressive, symptomatic NF1-associated plexiform neurofibromas.

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.