Clinical

Pfizer’s investigational mini-dystrophin gene therapy, fordadistrogene movaparvovec, did not meet its primary end point of improvement in motor function in ambulatory patients with Duchenne muscular dystrophy (DMD).

Medical experts highlight unmet needs in the PNH treatment landscape and considerations for utilizing eculizumab.

Dr Haumschild continues leading a discussion surrounding management of PNH, emphasizing the impact of complement inhibitors.

Determining the roles that various inflammatory markers and pathways play in lower-risk myelodysplastic syndromes (MDS) could lead to therapies that keep disease progression at bay.

This study provides a comprehensive review of both established and emerging prognostic markers in chronic lymphocytic leukemia (CLL), and their role in predicting disease, treatment response, and overall survival.

Study findings show eculizumab is effective and well tolerated in reducing disease burden in adolescents with refractory anti-acetylcholine receptor antibody–positive generalized myasthenia gravis.

Encouraging trends were seen with osimertinib and platinum-pemetrexed chemotherapy in patients with EGFR-mutated advanced non–small cell lung cancer (NSCLC) in the phase 3 FLAURA2 study.

The albumin-bilirubin score is a reliable predictor of treatment response for hepatocellular carcinoma (HCC) after yttrium 90 transarterial radioembolization (TARE).

The treatment regimen not only significantly improved progression-free survival (PFS) for patients with advanced stage classic Hodgkin lymphoma but was also better tolerated compared with a 20-year-old regimen being used.

With its recent approvals around the world to treat newly diagnosed and relapsed/refractory diffuse large B-cell lymphoma (DLBCL), the antibody-drug conjugate polatuzumab vedotin is changing regimens for current and future patients, with manageable adverse effects.

A recent review found that benzene exposure due to work may be linked to the development of colorectal cancer.

The late-breaking oral session at the European Hematology Association (EHA) 2024 Congress featured new data from abstracts submitted after the deadline, including both interventional and foundational science.

Mohamad Mohty, MD, PhD, of Saint-Antoine Hospital and Sorbonne University, highlights how immunotherapies are offering various effective treatment options for patients with multiple myeloma and improving quality of life for relapsed/refractory patients.

Joseph Mikhael, MD, MEd, FRCPC, FACP, of the International Myeloma Foundation, HonorHealth, and TGen, notes that US physicians have more flexibility when treating patients with early relapsed/refractory multiple myeloma, whereas those in Europe must follow stricter protocols.

At a session of the European Hematology Association 2024 Congress, presenters shared updates from trials of treatment strategies for newly diagnosed multiple myeloma (MM).

Researchers have found that specific genetic markers may increase the risk of cardiovascular adverse effects in patients using Bruton’s tyrosine kinase (BTK) inhibitors.

Investigators from Germany conducted a systematic review of different MRI features in soft tissue sarcoma (STS) that are used to infer tumor histological grade.

Ryan Jacobs, MD, of Atrium Health Levine Cancer Institute, explains that the phase 2 CAPTIVATE study's fixed-duration treatment cohort shows long-term progression-free survival for patients with chronic lymphocytic leukemia (CLL) who received the first-line treatment of ibrutinib and venetoclax.

Heidi Crayton, MD, and Maria Lopes, MD, discuss the limitations of using relapse rates as a critical endpoint in trials for patients with non-relapsing secondary progressive multiple sclerosis experiencing disease progression without relapse, explore methods to assess therapeutic benefit in this population, examine the relationship between cost of care and disease severity, and highlight the impact of decreased mobility and cognition on MS patients along with strategies for improvement.

Heidi Crayton, MD, and Maria Lopes, MD, discuss the impact of multiple sclerosis on patients, families, and caregivers, differentiate relapsing forms (relapsing-remitting and primary progressive) from non-relapsing forms (non-relapsing secondary progressive) in clinical practice, and highlight the key pathophysiological features of relapsing multiple sclerosis and the impact of progression independent of relapse activity (PIRA) after an initial demyelinating event.

Bringing the bispecific T-cell engager into the frontline consolidation phase "redefines the standard of care" for patients with B-ALL, an investigator said.

The results of the 3-year follow-up show meaningful improvement in overall survival (OS) and progression-free survival (PFS) for tislelizumab plus chemotherapy compared with placebo plus chemotherapy.

Abstracts presented at the 2024 European Hematology Association Congress deliver results on regimens involving venetoclax for chronic lymphocytic leukemia (CLL).

Results presented at the 2024 European Hematology Association Congress suggest that chimeric antigen receptor (CAR) T-cell therapy can be feasibly and safely delivered in the outpatient setting as treatment for lymphoma.

Adolescent anterior cruciate ligament reconstruction surgery can preserve health and restore function of the knee joint, but the surgical cost has outpaced inflation.

Marta Rodríguez Martínez, PhD, sustainability officer of the European Molecular Biology Laboratory, highlights the primary sources of scientific research waste and how to reduce them.

At an Institute for Value-Based Medicine® (IVBM) event cohosted by The American Journal of Managed Care® and Sutter Health, experts illuminated the current state of cardiometabolic health care and offered a glimpse into the future.

Chesahna Kindred, MD, MBA, FAAD, board-certified dermatologist, Kindred Hair & Skin Center, discusses how diversity in clinical trials are helping bridge the racial health equity gap in psoriasis care.

Data from this new study suggest that tailoring induction therapy for acute myeloid leukemia (AML) based on cytarabine pharmacogenomic 10–single-nucleotide variant score could better treatment for these young patients, particularly those who are Black.

The most common treatment-related adverse events were injection site reactions, the study found.