Fetal Hemoglobin Decrease During Voxelotor Treatment.

Voxelotor modifies hemoglobin-oxygen affinity improving anemia and reducing hemolysis in sickle cell patients. However, the impact of Voxelotor on fetal hemoglobin (HbF) levels is unknown. We describe here variations of percentage of HbF measured by high performance liquid chromatography and mean corpuscular fetal Hb in a cohort of sickle cell patients treated with Voxelotor at Henri Mondor Sickle Cell Referral Center.

Clinical burden and healthcare resource utilization associated with managing transfusion-dependent β-thalassemia in France.

Objective: To describe the clinical burden and healthcare resource utilization associated with managing transfusion-dependent β-thalassemia (TDT) in France.

Methods: We used the French National Health Data System (système national des données de santé) to identify eligible patients from January 1, 2012, to March 1, 2019. Inclusion criteria were a diagnosis of β-thalassemia, ≥8 red blood cell (RBC) transfusion episodes per year in ≥2 consecutive years following the diagnosis, and ≥1 year of follow-up data.

Predictors of health-related quality of life in a large cohort of adult patients living with sickle cell disease in France: the DREPAtient study.

Background: Sickle cell disease (SCD) is an inherited autosomal recessive disorder exhibiting a range of symptoms and acute and/or chronic complications that affect the quality of life. This study aimed to assess health-related quality of life (HRQoL) and to identify the associated factors in adult patients with SCD in France.

Methods: DREPAtient is a cross-sectional, multicenter study conducted from June 2020 to April 2021 in France and in certain French overseas territories where SCD is highly prevalent.

Cardiac diastolic maladaptation is associated with the severity of exercise intolerance in sickle cell anemia patients.

This pilot study focusing on Sickle Cell Anemia (SCA) patients offers a comprehensive and integrative evaluation of respiratory, cardiovascular, hemodynamic, and metabolic variables during exercise. Knowing that diastolic dysfunction is frequent in this population, we hypothesize that a lack of cardiac adaptation through exercise might lead to premature increase in blood lactate concentrations in SCA patients, a potential trigger for acute disease complication. SCA patients were prospectively included in PHYSIO-EXDRE study and underwent a comprehensive stress test with a standardized incremental exercise protocol up to 4 mmol L blood lactate concentration (BL4).