Hematology

Real-world clinical data from the Adelphi PNH II Disease Specific Programme were reported at ASH 2024.

Danicopan add-on therapy demonstrated more favorable safety in the ALPHA trial than pegcetacoplan did in the PEGASUS trial in a new analysis.

At ASH 2024, Annette von Drygalski, MD, PharmD, described the paradigm shift that has taken place in hemophilia over the past decade.

Data from the PINES trial at ASH 2024 suggest eltrombopag could outperform current standard of care for platelet response in newly diagnosed pediatric ITP.

While the therapy was generally well-tolerated, the PIVOT trial failed its primary endpoint of dose-limiting toxicities.

The therapy uses a novel pyruvate kinase activator mechanism and warrants further investigation in an ongoing phase 3 trial.

Notably, participants without platelet responses also experienced improvements in fatigue, which may be due to the monoclonal antibody’s anti-inflammatory action.

Topline results from APPULSE-PNH show the positive efficacy and safety of twice-daily oral iptacopan in adults with PNH switched from anti-C5 therapies.

Dexamethasone treatment was linked to worse clinical outcomes in patients with sickle cell disease hospitalized for COVID-19.

Anemia in heart failure with preserved ejection fraction was linked to right ventricular dysfunction in an analysis of more than 1000 patients in China.

Insufficient ferric carboxymaltose dosing and timing fail to significantly improve hemoglobin levels, suggesting gaps in perioperative anemia management.

New late-breaking ACG 2024 data supports AIMS65 as a risk factor tool for patients undergoing anticoagulant reversal.

On October 25, 2024, Kind Pharmaceutical announced receipt of FDA Orphan Drug Designation for AND017 in the treatment of sickle cell disease.

Anemia could be a reliable sign of all-cause mortality in patients with heart failure.

A systematic review found cognitive impairment a defining characteristic of SCA, with a notable impact on individuals of all ages.

The anti-TFPI significantly reduced ABR compared to routine prophylaxis treatment in the phase 3 BASIS trial.

Education and employment, social and emotional functioning, and healthcare access are notable factors influencing pain in sickle cell disease.

This retrospective analysis examined the current management of early and late anemia in a representative sample of the kidney transplant population in Spain.

Anemia is frequently observed in primary care patients with HF and is linked to worse prognosis and comorbidities, including cancer.

An elevated social vulnerability index score was linked to increased mortality among patients with SCD in the 5-year CDC WONDER database.

An analysis of NYC’s lead registry data from 2005 to 2019 revealed a potential association between sickle cell disease and childhood lead poisoning.

This month in review highlights vitamin D supplementation in anemia, the prevalence of iron deficiency in US adults, and the efficacy of rpFVIII in treating acquired hemophilia A.

Pfizer has announced the voluntary withdrawal of voxelotor in all markets after a report found the benefits do not outweigh the risks for sickle cell disease.

A large study shows no women were anemic in the first trimester of pregnancy, but more than 80% of the women were iron deficient by the third trimester.

Rates of absolute and functional iron deficiency were high among adults in the US, even in those without anemia, heart failure, or chronic kidney disease.