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Hydroxyurea-derived clinical and biological benefits and safety were retrospectively studied for 123 adult patients from 2 sickle cell disease referral centers during a total follow-up of 654 patient-years and total hydroxyurea exposure of 549 patient-years. Fifty-six adverse events occurred (incidence: 12%/patient-year), with leg ulcers being the most frequent. Adverse events could arise at any time and were usually reversible. No malignancy was observed. Clinical and biological benefits of our cohort were similar to those previously reported. Based on this relatively long retrospective study, the risk/benefit ratio for moderate hydroxyurea doses was satisfactory., (Copyright © 2010 S. Karger AG, Basel.)

Survival of sickle cell disease has improved dramatically. Sickle cell disease is now a chronic adult illness characterized by progressive multiorgan failure, particularly involving the eye, kidney and heart. It remains severe in its repercussions on everyday life, especially due to unpredictable, recurrent, and intense pain. Providing health care to these patients becomes a public health priority because of the increasing number and ageing of patients. Complications of sickle cell disease include acute manifestations such as vaso-oclusive crises, acute chest syndrome, priapism, infections, and chronic complications which lead to severe functional sequela such as renal, pulmonary, bone, retinal damage. The prevention of these complications becomes the prevailing therapeutic approach in sickle cell adult patients. The best treatment of severe acute complications that sometimes threaten life is the prevention. Hydroxyurea is the only treatment directed at the prevention of the complications in adult patients. Concerns about the effects on male fertility should not be a barrier for its prescription because its efficiency has been proven, even for survival. [ABSTRACT FROM AUTHOR]

Résumé La drépanocytose est une maladie monogénique de transmission autosomique récessive. Elle résulte d’une mutation ponctuelle du sixième codon du gène de la bglobine qui entraîne une polymérisation de l’hémoglobine mutée (H βS) en situation désoxygénée. L’HbS est à l’origine de l’hémolyse et des phénomènes vaso-occlusifs. C’est la première maladie génétique en France et dans le monde. Du fait des progrès des traitements et de l’allongement de l’espérance de vie des malades la prévalence de cette maladie est en constante augmentation et elle a été reconnue comme priorité de santé publique par les autorités de santé nationales et internationales. Au cours de cette maladie chronique émaillée de complications aiguës, le biologiste intervient à différentes étapes de la prise en charge du malade (établissement du diagnostic, suivi du traitement, dépistage de certaines complications et diagnostic prénatal). Summary Sickle cell disease (SCD) is a monogenic autosomal recessive disorder. It is the result of a single point mutation in the b-globin gene producing the abnormal Haemoglobin S (HbS). Deoxygenation of SS erythrocytes leads to polymerization of SS hemoglobin causing hemolysis and vaso-occlusion. It is the most common genetic disease in the world. In the last decades, the care of patients with SCD has undergone important advances and life expectancy has risen dramatically. World Health Organization (WHO) and United Nations have recognized SCD as a public health priority. Currently, SCD is a multisystem chronic disease with episodes of acute complications. Biologist is involved in diagnosis, prenatal diagnosis, follow-up, monitoring of treatment, detection of complications. [ABSTRACT FROM AUTHOR]