Clinical

The FDA has removed Risk Evaluation and Mitigation Strategies (REMS) for approved chimeric antigen receptor (CAR) T-cell therapies for hematologic malignancies, aiming to ease provider burden and expand patient access.

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.

In myasthenia gravis, complement plays a big role in what happens to the muscle end of the neuromuscular junction, explains Miriam Freimer, MD, an author on 5 abstracts presented at the recent 15th MGFA International Conference on Myasthenia and Related Disorders.

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.

A post hoc analysis of the phase 3b JUMP trial supports the real-world use of ruxolitinib with anemia supportive care to maintain dosing and clinical outcomes in patients with myelofibrosis, offering a practical strategy that does not compromise efficacy.

Ibrahim Aldoss, MD, discusses the potential of AZD0486 for treating relapsed or refractory B-cell acute lymphoblastic leukemia (ALL) and outlines the SYRUS study objectives.

INCA33989 continues to show strong safety and early signs of disease modification in essential thrombocythemia, according to John Mascarenhas, MD, with next steps focused on optimizing dosing and delivery scheduling.

Mirvetuximab soravtansine continues to show efficacy and safety in the final analysis of the phase 2 PICCOLO study.

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 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 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.

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.

A post-hoc analysis of the phase 3b JUMP trial assessed outcomes in patients with myelofibrosis and baseline anemia who were treated with ruxolitinib alongside anemia-supportive therapies, according to Pankit Vachhani, MD.

Revumenib for relapsed/refractory acute leukemias with KMT2A translocation is cost-neutral for health plans, but traditional methods of analysis may not be accurate for rare diseases, said Ivo Abraham, PhD, RN, of The University of Arizona.

John Mascarenhas, MD, discusses phase 1 data showing that INCA33989 is a well-tolerated therapy for calreticulin–mutant essential thrombocythemia, with early signs of efficacy and potential disease modification.

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.

Primary and secondary end points were demonstrated in a phase 3 trial of subcutaneous marstacimab as treatment for patients with hemophilia A or B.

Iptacopan targets one of the major proteins involved in the enzymes of the alternative pathway of complement, explains Carla Nester, MD, of Stead Family Children's Hospital.

John Mascarenhas, MD, discusses the rationale, mechanism, and design of the phase 1 INCA33989-101 and -102 trials investigating a CAL-R–targeted antibody for patients with essential thrombocythemia.

On-body delivery systems for subcutaneous isatuximab could enable patient self-administration, according to Xavier Leleu, MD, PhD, improving convenience and transforming treatment for relapsed/refractory multiple myeloma.