Video Series

A panelist discusses how the highest unmet needs in acute myeloid leukemia include treatments for patients with refractory and relapsed disease, addressing poor outcomes in patients with TP53 mutations and MECOM rearrangements, while emerging trends focus on combination therapies (doublets, triplets, quadruplets), the shift toward more convenient oral therapies, increased emphasis on minimal residual disease negativity as an end point, and expanded transplant eligibility for older patients aged into their mid to late 70s.

Panelists discuss how intrathecal delivery of onasemnogene abeparvovec in the STEER study demonstrates statistically significant motor function improvements in older patients with SMA (ages 2-18 years) with favorable safety profiles, potentially expanding gene therapy access beyond the current age restriction of under 2 years.

Panelists discuss how gaps in SMA care persist despite highly effective treatments, particularly regarding racial and ethnic disparities in research participation and global access challenges due to high costs and infrastructure limitations in developing countries.

Panelists discuss how supporting primary care providers requires moving beyond passive quality measures to peer-to-peer education, transparent performance feedback, multidisciplinary team resources, and creative care delivery models that address the "27-hour day" problem.

Panelists discuss how robust clinical evidence from major studies like the EPIC and OPERA trials demonstrates that progression independent of relapse activity (PIRA) is the primary driver of confirmed disability progression in patients with multiple sclerosis (MS), with clinical parameters over 3 to 6 months being the most meaningful measures of treatment impact.

Panelists emphasize that the approval of menin inhibitors has transformed the treatment landscape for KMT2A-rearranged acute myeloid leukemia (AML) by replacing historically limited and toxic chemotherapy-based strategies with a targeted, guideline-endorsed option that aligns with disease biology—offering renewed hope for both adult and pediatric patients, especially in the relapsed/refractory setting.

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 report that the AUGMENT-101 trial demonstrated meaningful clinical benefits of menin inhibitors in relapsed/refractory KMT2A-rearranged acute myeloid leukemia (AML)—with a 23% complete remission rate and manageable safety profile including differentiation syndrome—highlighting the importance of early recognition, patient education, and supportive care to optimize outcomes as these agents move toward becoming a new standard of care.

Panelists discuss how UPMC's integrated delivery and financing system enables coordinated care through e-consults, proactive outreach to primary care physicians, case management, telemedicine, and streamlined referral processes that reduce friction for patients and providers.

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.

A panelist discusses how personalized ITP care requires open conversations about testing costs, insurance coverage, and treatment accessibility, with clinicians helping patients navigate financial barriers through pharmaceutical assistance programs and clinical trials.

Panelists discuss how progression independent of relapse activity (PIRA) represents a distinct pathological process involving smoldering inflammation and neurodegeneration that drives disability in patients with multiple sclerosis (MS), particularly manifesting around midlife despite being present from disease onset.

A panelist discusses how the COAT trial showed that switching patients with uncontrolled dry eye disease on cyclosporine 0.05% to the 0.09% solution led to significant improvements in corneal staining and symptoms by week 4, offering earlier relief than typically expected and supporting informed patient counseling.

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 spinal muscular atrophy (SMA) treatment will evolve over the next 5 years, likely incorporating improved gene therapy delivery systems, combination therapies, and rehabilitation models, while maintaining individualized approaches for each patient.

Panelists discuss how communication between clinicians, patients, and payers could improve equitable access to spinal muscular atrophy (SMA) treatments, while acknowledging the complex value assessment of high-cost therapies vs improved quality and length of life.

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 how successfully integrating subcutaneous (SubQ) therapies into oncology practice depends on comprehensive education, optimized workflows, transparent patient communication, and ongoing evaluation to ensure improved care quality and operational efficiency.

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 how incorporating subcutaneous (SubQ) formulations into clinical formularies requires careful evaluation of clinical equivalence, workflow fit, and reimbursement logistics, with pharmacy and therapeutics committees balancing patient benefit, operational feasibility, and financial risk to guide adoption.

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 the future of Bruton tyrosine kinase (BTK) inhibitor therapies, considering emerging treatments, regulatory changes, evolving cost dynamics, and the current unmet needs in chronic lymphocytic leukemia (CLL) and mantle cell lymphoma (MCL) care, and how these factors may shape the landscape of treatment options.

Panelists discuss strategies and resources health care providers can offer to help alleviate the financial burden of long-term Bruton tyrosine kinase (BTK) inhibitor therapy for patients with chronic lymphocytic leukemia (CLL) and mantle cell lymphoma (MCL), focusing on support programs, cost-sharing solutions, and patient assistance initiatives.

A panelist discusses recent advancements in bispecific antibody therapies for relapsed/refractory multiple myeloma, emphasizing the promising efficacy and improved safety profile of GPRC5D-targeting agents, the importance of patient-specific factors in therapy selection, and the role of prophylactic measures to manage infection risks, supporting personalized treatment strategies.

Panelists discuss how payer policies and clinical pathways currently influence clinical decision-making for chronic lymphocytic leukemia (CLL) and mantle cell lymphoma (MCL) treatments and explore strategies that managed care organizations can implement to control costs while ensuring patients have access to the most appropriate BTK inhibitor therapy.

Panelists discuss how spinal muscular atrophy is an autosomal recessive genetic disease affecting motor neurons with 3 currently approved disease-modifying therapies that restore SMN protein expression, including 2 requiring ongoing treatment and 1 gene transfer therapy (onasemnogene abeparvovec) approved only for patients under 2 years of age.

Panelists discuss how the pooled 3-year data for delandistrogene moxeparvovec show sustained motor function stabilization and less deterioration compared with natural history, providing confidence in the therapy’s long-term benefits while acknowledging the need for survival data and addressing concerns about potential transgene dilution over time.

Panelists discuss how implementing cardiovascular-kidney-metabolic health requires a team-based approach using the analogy of football, where physicians coach multidisciplinary teams to help patients overcome barriers and achieve health goals.

Panelists highlight that menin inhibitors represent a breakthrough in the treatment of KMT2A-rearranged AML by targeting the disease’s core epigenetic drivers, with early clinical trials like AUGMENT-101 showing promising results in heavily pretreated, high-risk patients and supporting the potential for a new therapeutic standard in this aggressive leukemia subtype.