Sickle Cell Disease
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Compared with placebo, crizanlizumab has superior safety and comparable efficacy in patients with sickle cell disease.
The study led by Paul George, MD, PhD, also highlights the importance of patient regimen adherence to secure these benefits.
The findings coincide with patient reports of experiencing increased vaso-occlusive episodes during menses.
The Foundation for Sickle Cell Disease Research will evaluate VAS-101 in a phase 1 clinical study.
The platform features incubation technology able to replicate cell microenvironments associated with SCD sickling.
Patients with follow-up of one month or longer saw hemolysis marker improvement and reported no vaso-occlusive crises.
In a phase III trial 90% of evaluable patients were free of severe vaso-occlusive crisis for at least 12 consecutive months.
Reni-cel led to hemoglobin normalization and increased fetal hemoglobin in patients with sickle cell disease.
An expert panel's review finds SCT has often been misattributed as the direct cause of mortality in patients who have SCT.
Preliminary efficacy and safety findings from a multicenter, open-label, phase Ib/II prospective study have been encouraging.
In a study SCT appeared linked to an increased risk of pregnancy-associated VTE, pulmonary embolism, and isolated DVT.
Patients receiving mitapivat showed a statistically significant hemoglobin response rate compared with placebo.
A review article describes current clinical knowledge of the role of inflammation and the activity of immune cells in SCD.
A study's efficacy and safety findings show promise for lovo-cel as a one-time intervention for SCD with history of stroke.
A cohort lifetime study from Hamda Khan, MA, and Jason Hodges, PhD, MA, follows patients from pediatric into adult SCD care.
Dr. Theodore Wun and Dr. Olubusola Oluwole investigated the causes of rising stroke rates among pediatric and adult patients.
A study has assessed cryogenically preserved ovarian tissue samples from women with SCD for any effects from hydroxyurea.
Kristen Howell, PhD, MPH, has investigated how transfer gaps in SCD care affect patients as they transition to adult care.
The REACH trial’s findings suggest a reduced burden upon transfusion resources worldwide is possible.
The gene therapy, which costs $3.1 million, was approved by the FDA in December 2023.
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