Sickle Cell

Strunk highlights reduced length of stay and superior outpatient pain management due to palliative care in hospitalized patients.

Our recap of the first half of 2025 highlights 5 regulatory updates, 5 trial announcements, and 3 top perspectives in hematology.

A relatively quiet quarter for hematology, punctuated by both successes and failures in clinical trials and a handful of Orphan Drug and Fast Track designations.

The FDA distributed several designations and many trials successfully met their endpoints during an eventful June for hematology.

Data from the Phase 1/2 BEACON trial indicate the efficacy and safety of this investigative 1-time therapy, particularly in decreasing blood cell sickling.

An ongoing phase 1 study aims to prove the safety and tolerability of VAS-101, as well as its effect on blood flow dynamics, red blood cell sickling, and oxygen dissociation.

A relatively quiet month for hematology, marked by brief ups and downs for trials and new research on anemia and sickle cell disease.

Investigators examined emergency department utilization as a product of age, patient-reported severity of vaso-occlusive episodes, and Social Vulnerability Index score.

A mixture of thalidomide and hydroxyurea outperformed hydroxyurea alone in all primary outcomes.

Patient-reported outcome measures display positive trends after hematopoietic stem cell transplantation for patients with sickle cell disease.

Bernaudin discussed the Drepagreffe-1 and 2 studies and improvements seen over 10 years of follow-up.

Appiah-Kubi also noted that despite elevated HbF levels, very young children with SCA already had anemia and reticulocytosis.

Frangoul discussed highlights from the latest data update from the CLIMB SCD-121 trial of Casgevy.

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.

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

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

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

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

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.

This August 2024 month in review highlights new recommendations from the USPSTF, FDA approval of a treatment for chronic GVHD, and Phase 2 data for severe HDFN.

Thrombotic risk is more likely influenced by disease status than hormonal contraception exposure in women with SCD.