Clinical

Innovations in rare blood disorders took center stage at the 2024 American Society of Hematology (ASH) Annual Meeting & Exposition, with key research presented on immune thrombocytopenia, hemophilia, and multiple myeloma.

Conventional parameters for risk stratification prognosis for patients with low-risk myelodysplastic neoplasms, especially bone marrow blast percentage, drop their clinical significance once molecular lesions are included in prognostic scores.

Bispecific antibodies show strong initial uptake in community settings for multiple myeloma treatment, particularly among Black patients compared with clinical trials.

By reducing cytokine release syndrome and neurotoxicity, prophylactic tocilizumab could facilitate safe outpatient administration of bispecifics to treat multiple myeloma (MM), explained Robert Rifkin, MD.

A panelist discusses how the FINEARTS-HF trial employed rigorous potassium-monitoring protocols and predefined hyperkalemia thresholds to evaluate finerenone's safety profile in patients with heart failure (HF), revealing important associations between baseline characteristics and hyperkalemia risk while demonstrating the drug's complex effects on potassium homeostasis.

A panelist discusses how glucagon-like peptide-1 receptor (GLP-1) agonists' cardioprotective effects likely stem from multiple physiological mechanisms, including reduced inflammation, improved glucose metabolism, enhanced endothelial function, and potential antiatherosclerotic properties that collectively lower the risk of cardiac events.

A shorter duration of venetoclax treatment for newly diagnosed acute myeloid leukemia (AML) may enhance efficacy and reduce toxicity, according to Amir Ali, PharmD, BCOP.

The panelist discusses Vowst, an FDA-approved live microbiota therapeutic that works by introducing healthy donor-derived gut bacteria to restore microbiome diversity, distinguishing itself from traditional antibiotics in preventing recurrent Clostridioides difficile infections in adults who have completed antibiotic treatment.

The panelist discusses Rebyota, a treatment approved by the FDA for preventing recurrent Clostridioides difficile infections in adults who have completed antibiotic treatment for recurrent C difficile infections.

Implementing an individualized starting dose (ISD) of niraparib (Zejula; GSK) reduced severe hematologic adverse event (AE) management costs by 48% compared with a fixed starting dose (FSD) in US patients with ovarian cancer.

Higher dietary magnesium intake is associated with a reduced risk of frailty in patients with chronic obstructive pulmonary disease (COPD), emphasizing its potential role in improving clinical outcomes.

Single-agent, subcutaneous epcoritamab generated deep responses in patients with relapsed/refractory chronic lymphocytic leukemia (R/R CLL).

The phase 3 AQUILA study showed a 63.1% progression-free rate at 5 years with daratumumab versus 40.8% with active monitoring.

The top 5 most-viewed content from this year's American Society for Preventive Cardiology (ASPC) Congress on Cardiovascular Disease (CVD) Prevention included interviews with Robert Kushner, MD, MS, and Martha Gulati, MD, MS, FACC, FAHA, FASPC, FESC, as well as coverage on multiple areas of cardiovascular medicine.

The SEQUOIA study's 5-year follow-up confirms zanubrutinib's safety profile and low cardiovascular events in treatment-naive chronic lymphocytic leukemia, according to Mazyar Shadman, MD, MPH, of Fred Hutchinson Cancer Center.

The meta-analysis found indications that autologous stem cell transplantation (ASCT) is associated with higher rates of disease-free survival and relapse-free survival, as well as lower rates of relapse compared with chemotherapy after patients achieved their first complete response (CR).

Myasthenia Gravis Foundation of America (MGFA) class correlates with activities of daily living and other clinical characteristics on a group level, but individual variability is significant between patients of the same MGFA class.

Promising topline results from a phase 3B study show the efficacy and quality of life (QOL) improvements of iptacopan (Fabhalta) as a twice-daily oral monotherapy for adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who transitioned from anti-C5 therapies.

Data presented at the 2024 American Heart Association (AHA) Scientific Sessions showed that continuous treatment with acoramidis (Attruby; BridgeBio Pharma) significantly reduced the risk of all-cause mortality (ACM) and cardiovascular-related hospitalizations (CVH) in patients with transthyretin amyloid cardiomyopathy (ATTR-CM).

Top coverage from the European Respiratory Society (ERS) Congress 2024 spanned various topics, including artificial intelligence–powered robots transforming respiratory care and late-breaking findings on an epilepsy drug showing promise for treating obstructive sleep apnea.

In part 2 of our interview, Cesar Davila-Chapa, MD, discusses key findings, limitations, and future research directions from his study on racial disparities in idiopathic pulmonary fibrosis (IPF) outcomes.

While race-aware models showed superior statistical calibration across racial and ethnic groups, their benefits in clinical decision-making were less pronounced than expected.

Gene therapy partially restores visual processing in the geniculostriate pathway of patients with Leber congenital amaurosis type 2 while maintaining compensatory activity in the retinotectal pathway.

The panelist discusses how Rebyota is an FDA-approved microbiome therapeutic that delivers standardized, purified intestinal microbiota through enema administration to restore gut microbial diversity and prevent recurrent Clostridioides difficile infection, differing from conventional fecal microbiota transplantation (FMT) in its regulatory status, standardized manufacturing process, and enhanced safety profile due to extensive screening and purification of donor material.

The panelist discusses how fecal microbiota transplantation (FMT) involves transferring stool from healthy donors to restore gut microbiota in patients with recurrent Clostridioides difficile infection, though safety concerns include potential pathogen transmission and careful donor screening requirements, while OpenBiome’s standardized frozen FMT preparations have helped advance accessibility but should be used according to current FDA guidance for investigational use.