The aim of the PRESTA study
The PRESTA study aims to determine whether giving a treatment as soon as a patient arrives at the emergency department with a vaso-occlusive crisis (VOC) can prevent acute chest syndrome (ACS), a serious lung complication of sickle cell disease and one of the condition’s leading causes of death.
How are patients at risk identified?
Doctors use a score called PRESEV, based on simple information collected in the emergency department: the patient’s symptoms, the clinical examination and blood-test results. Developed by our teams and validated in Europe and Africa, the score helps identify patients at high risk of developing acute chest syndrome.
The treatment being assessed
The first treatment assessed in the PRESTA study is tocilizumab, a drug that targets interleukin 6 (IL-6), a protein involved in inflammatory processes. Our teams previously used this treatment in people with sickle cell disease who experienced a vaso-occlusive crisis during the COVID-19 pandemic. Research conducted in PRESEV 1 also showed particularly high IL-6 levels in patients who subsequently developed acute chest syndrome.
If tocilizumab does not show sufficient efficacy, other candidate medicines may be assessed in sequence through a research platform dedicated to treating vaso-occlusive crises. The aim is to identify the most appropriate treatment to prevent complications and alter the course of the disease.
How the study works
Patients may take part in the study if they:
- are adults;
- have sickle cell disease;
- attend the emergency department with a severe pain crisis;
- are at high risk of developing acute chest syndrome.
After giving informed consent, patients receive one dose of the treatment being studied. They are then followed during their hospital stay and for a further three months.
Expected benefits
If the treatment proves effective, it could:
- reduce the number of lung complications;
- shorten hospital stays;
- reduce the need for blood transfusions;
- prevent some admissions to intensive care;
- reduce the risk of death.
Why is this study important?
There is currently no specific preventive treatment that can stop acute chest syndrome developing during a severe vaso-occlusive crisis. PRESTA could improve care from the moment patients arrive at the emergency department and help prevent potentially serious complications.
