Clinical

Panelists discuss how patient perspectives on new therapies center around clinically meaningful outcomes and survival while balancing individual risk tolerance, with some patients willing to accept higher risks for potentially transformative treatments in devastating diseases.

Panelists discuss how exciting ongoing research efforts are expanding similar gene therapy technologies to other muscular dystrophies like FSHD and myotonic dystrophy, using strategies to knock down rather than restore gene expression for these autosomal dominant conditions.

A panelist discusses how the ASCERTAIN-V study demonstrated that an all-oral combination of decitabine-cedazuridine plus venetoclax achieved a 47% complete response rate and 15.5-month median overall survival in older, unfit AML patients, representing a potential new standard of care that eliminates the need for intravenous infusions and significantly reduces clinic time burden while serving as a backbone for future oral combination therapies.

Collaborations between academic and community cancer centers enhance access to care, with success in acute myeloid leukemia and precision oncology.

The indirect comparative analysis is the first of its kind to assess the relative efficacy of approved covalent Bruton tyrosine kinase inhibitors (cBTKis) in the absence of head-to-head trials.

Carrie Kitko, MD, explains that axatilimab maintains strong response rates in patients with chronic graft-versus-host disease (GVHD) regardless of previous treatment lines and emphasizes the need to explore combination therapies.

Panelists discuss how unconscious bias and weight stigma create barriers to care, requiring providers to acknowledge their own biases, create welcoming clinical environments with appropriate accommodations, and approach patients with compassion rather than judgment about willpower.

Panelists discuss how effective provider-patient communication about weight requires opening conversations with empathy and collaboration, using phrases like "I'm concerned about your weight" and avoiding stigmatizing language while understanding patient motivations and readiness for change.

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.

Depemokimab significantly reduces asthma exacerbations, benefiting patients regardless of their baseline asthma control levels. A biologics license application for depemokimab is currently being reviewed by the FDA.

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.

The finding that basophil counts may have prognostic significance aligns with a growing body of research into basophils in myeloproliferative neoplasms.

The post hoc analysis of the AGAVE-201 trial found that axatilimab demonstrated consistent response rates in patients with chronic graft-vs-host disease (GVHD), regardless of the number or type of prior therapies.

Both regulatory-approved and pipeline treatments for moderate to severe hidradenitis suppurativa demonstrate comparable efficacy and safety, offering hope for patients often dissatisfied with current treatment options.

The findings contradict previous research suggesting high-risk patients with multiple myeloma were more likely to have a higher percentage of regulatory T cells (Tregs).

July is UV Safety Awareness Month, highlighting the growing risks of UV radiation and encouraging the use of protective measures to reduce sun-related health threats.

AZD0486 demonstrated encouraging safety and dose-dependent efficacy in heavily pretreated adolescent and adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia (ALL), according to early findings from the phase 1/2 SYRUS trial and Ibrahim Aldoss, MD, of City of Hope.

While artificial intelligence (AI) holds promise to improve the diagnosis of melanoma, researchers of a new review outlined challenges with these novel approaches.

The recent increase in prolonged emergency department (ED) stays and boarding times for older adults reveals systemic challenges in US hospital care.

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.