Sickle cell disease is a severe monogenic genetic disorder caused by an autosomal recessive mutation of the β-globin gene, leading to production of abnormal hemoglobin (HbS). It primarily affects individuals from Africa or the French overseas territories. In France, approximately 26,000 patients are affected. Improved care has significantly increased life expectancy.
Vaso-occlusive crises (VOC) are the main clinical complication. They result from polymerization of HbS, deforming red blood cells and causing capillary occlusion, tissue hypoxia, intense bone pain, and frequent hospitalizations. In France in 2015, 25,150 hospitalizations were recorded, 61% of which were for VOC.
Iloprost is a prostacyclin (PGI2) analogue with vasodilatory, anti-platelet, anti-inflammatory, and antioxidant properties. It is used to treat severe limb ischemia and Raynaud's phenomenon, administered by IV infusion for 5 to 28 days.
It is well tolerated and has shown efficacy for bone pain related to bone marrow edema. Its rapid and sustained action makes it an interesting candidate for VOC, which are comparable to ischemic-origin pain. To date, only one reported case of iloprost use for a VOC exists, showing rapid and lasting improvement.
This randomized, multicenter, double-blind, placebo-controlled clinical trial aims to evaluate the efficacy of iloprost in patients hospitalized for VOC, with the objective of reducing pain and opioid consumption.
This comprehensive approach could significantly improve VOC management.
Of childbearing potential (defined by the CTCG as a fertile woman, after menarche and until menopause, except in cases of permanent sterility, including hysterectomy, bilateral salpingectomy, or bilateral oophorectomy);
Using a highly effective method of contraception according to CTCG recommendations (combined hormonal contraception [containing estrogens and progestogens] associated with inhibition of ovulation, progestogen-only hormonal contraception associated with inhibition of ovulation, intrauterine device (IUD), intrauterine hormone-releasing system (IUS), bilateral tubal occlusion, vasectomized partner, sexual abstinence) for at least 4 weeks prior to inclusion and throughout the entire duration of systemic exposure to the study treatment. AND
Having a negative urine pregnancy test at inclusion;
Postmenopausal: Menopause, according to CTCG recommendations, is defined as the absence of menstruation for 12 months without any other medical cause. Elevated follicle-stimulating hormone (FSH) levels in the postmenopausal interval can be used to confirm postmenopausal status in women who are not using hormonal contraception or hormone replacement therapy. However, in the absence of 12 months of amenorrhea, a single FSH measurement is insufficient.
pregnancy, breastfeeding
Hypersensitivity to the active substance or to any of the excipients.
Conditions where the risk of bleeding may be increased due to the effects of iloprost on platelets (e.g., active peptic ulcer, trauma, intracranial hemorrhage).
severe coronary disease
Parallel
Iloprost
Drug1 ampoule IV over 6 h daily for 5 days diluted in 250 mL G5%
Placebo
Drug250 mL G5% IV over 6 h daily for 5 days
Opioid consumption
Mean opioid consumption in morphine equivalent mg/day over the 28 days following randomization
Mean pain
Mean pain according to the VAS (Visual Analog Scale) over the first 28 days. The VAS used in this study will be a scale from 0 to 10, with ratings ranging from "no pain" (0) to "the worst pain" (10). If the patient has multiple painful areas, the worst pain should be reported.
Length of hospital
Length of hospital stay capped at 28 days
Acute chest syndrome during hospitalization
Number of priapism episodes during hospitalization
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This trial is not recruiting at the moment. You can still explore other options:
