1 Hospices Civils de Lyon, Department of Internal Medicine, Edouard Herriot Hospital, Lyon, France.
2 Claude Bernard University Lyon 1, Laboratoire Interuniversitaire de Biologie de la Motricité EA7424, Equipe ‘Vascular biology and red blood cell’, Villeurbanne, France.
3 Hospices Civils de Lyon, Hematology Department, Lyon-Sud Hospital, Pierre Bénite, France.
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While hydroxycarbamide (hydroxyurea, HU) has less and fewer indications in malignant hemopathies, it represents the only widely used drug which modifies sickle cell disease pathogenesis. Clinical experience with HU for patients with sickle cell disease has been accumulated over the past 25 years in Western countries. The review of the literature provides increasing support for safety and efficacy in both children and adults for reducing acute vaso-occlusive events including pain episodes and acute chest syndrome. No increased incidence of leukemia and teratogenicity was demonstrated. HU has become the standard-of-care for sickle cell anemia but remains underused. Barriers to its use should be identified and overcome.
|Figure 1. Structure of hydroxycarbamine (hydroxyurea, HU).|
Sickle Cell Disease: Historical Considerations
HU Mechanisms of Action in Sickle Cell Anemia
The Use of HU in Sickle Cell Disease placebo.
|Table 1. Randomized trials comparing HU with placebo.|
|Table 2. Observational studies addressing acute clinical events with HU in sickle cell anemia.|
Distribution of HbF
A Clinical and Economic Problem
Beyond HU Therapy