AJMC Contributor

The researchers have detailed their complete findings of subcutaneous (SC) vs intravenous (IV) efgartigimod in generalized myasthenia gravis (gMG) from the phase 3 ADAPT-SC trial and initial findings from the open-label extension of the study, ADAPT-SC+.

In addition to being diagnosed more frequently in older patients, pulmonary arterial hypertension (PAH) is more frequently diagnosed in patients with cardio-pulmonary comorbidities, which poses challenges to the optimal treatment of PAH, explained the researchers.

The researchers suggest certain refinements to the 2022 European LeukemiaNet classification model for patients aged 60 years or older receiving lower intensity treatment (LIT), including the adoption of a mutation score that accounts for certain gene mutations.

The potential value of these biomarkers may help differentiate acetylcholine receptor antibody seropositive (AChR+) myasthenia gravis (MG) from healthy patients.

The European approval comes months after the FDA’s approval of the first-of-its-kind treatment for pulmonary arterial hypertension (PAH).

If integrated into clinical practice, the approach could fill a need for more optimal diagnostic approaches for myasthenia gravis (MG), explained the researchers.

By analyzing the surface proteome of a wide-ranging sample of acute myeloid leukemia (AML) specimens, researchers identified potential antigens and primitive cell markers representative of AML supgroups.

The researchers found inferior outcomes and higher accrued costs associated with alloimmunization in patients with myelodysplastic syndromes (MDS), a disease that relies on transfusion of red blood cells.

Recent years have brought a more targeted approach to treating acute myeloid leukemia (AML) based on accumulating knowledge about the genetic makeup of the heterogenous disease.

A Canadian study showed a high probability that using next-generation sequencing (NGS) for treatment sequencing based on risk stratification is cost-effective.

As the treatment landscape for chronic lymphocytic leukemia (CLL) has moved into an era where targeted treatments are a mainstay of treatment, new opportunities to tailor therapy have emerged.

The findings support previous research suggesting that worse prognosis in patients with del(6q) is not solely a result of genomic instability or presence of multiple genetic alterations.

New data from the single-center, open-label extension EDITA-ON study suggest that early treatment for pulmonary arterial hypertension (PAH) could benefit patients with systemic sclerosis and early pulmonary vascular disease.

A recent study showed that the Myasthenia Gravis (MG) Symptoms PRO scales viably measure core symptoms of the disease.

Beremagene geperpavec-svdt (B-VEC) entered the market in 2023 as the first approved corrective treatment for dystrophic epidermolysis bullosa (DEB), a rare genetic disease affecting the skin and nails that is caused by mutations in the COL7A1 gene.

The biomarker, G-protein-coupled receptor 176 (GPR176), may be a driver in the progression of ovarian cancer and potential target for gene therapy, suggested researchers of a new study.

Researchers collected feedback from 20 clinicians implementing a telehealth serious illness conversation (SIC) with their patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS).

The researchers noted that most genomic analyses have traditionally focused on commonly mutated genes, which can pinpoint mutations occurring most frequently but does not account for the extent to which these mutations impact cancer cell survival and proliferation.

In a recently published systematic review, researchers determined that VEXAS (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) syndrome typically presents in men, with atypical morphology and with systemic symptoms.

In addition to ensuring residency programs provide educational and practical opportunities for addressing skin of color (SOC) hair-related concerns, prioritizing having residents of diverse backgrounds can help close knowledge and comfort gaps, suggest researchers.

Of 7 subtypes identified 10,000 melanoma cells, C4 Melanoma COCO1A was the only to show indications of being more sensitive to natural killer (NK) and T cells.

With an emerging hypothesis that inflammation of the skin prompts intestinal remodeling through various mechanisms, researchers explored the role of inflammatory signals, microbiome alterations, metabolites, and the nervous system.

Early findings from a new preclinical study indicate that dual blockade of aurora-A kinase and PD-L1 signaling can inhibit tumor growth in triple-negative breast cancer (TNBC), treatment for which has historically remained limited.

Notably, adolescents and young adults (AYA) living with type 1 diabetes (T1D) who reported more comfort with self-management tended to have worse glycemic control.

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.

Patients receiving chlorthalidone had lower rates of major cardiovascular (CV) events and noncancer-related deaths vs those receiving hydrochlorothiazide.

Although further research is needed, these new findings suggest that using the 2 intranasal sprays is an effective option for patients with myasthenia gravis (MG) who experience eyelid drooping.

The data come from the observational Cost of Hemophilia in Europe: A Socioeconomic Survey II (CHESS II) study of 288 Spanish patients with hemophilia A and B, which showed certain differences between disease subtypes but overall similar trends in disease impact.

An abstract presented at the 2024 American Society of Clinical Oncology annual meeting showed that patients treated with zanubrutinib or acalabrutinib had more favorable safety and efficacy outcomes.

Researchers say their findings will help inform treatment protocols for this patient population, as questions remain about optimal duration of these treatments following first-line treatment with pyridostigmine.