Περίληψη:
The aim of this prospective study was to evaluate the long-term efficacy
and safety of hydroxyurea (HU) in patients with sickle cell disease
(SCD). Thirty-four patients with sickle cell anemia (hemoglobin S
[HbS]/HbS), 131 with HbS/beta(0)-thal, and 165 with HbS/beta(+)-thal
participated in this trial. HU was administered to 131 patients, whereas
199 patients were conventionally treated. The median follow-up period
was 8 years for HU patients and 5 years for non-HU patients. HU produced
a dramatic reduction in the frequency of severe painful crises,
transfusion requirements, hospital admissions, and incidence of acute
chest syndrome. The probability of 10-year survival was 86% and 65%
for HU and non-HU patients, respectively (P = .001), although HU
patients had more severe forms of SCD. The 10-year probability of
survival for HbS/HbS, HbS/beta (0)-thal, and HbS/IVSI-110 patients was
100%, 87%, and 82%, respectively, for HU patients and 10%, 54%, and
66%, for non-HU patients. The multivariate analysis showed that fetal
hemoglobin values at baseline and percentage change of lactate
dehydrogenase between baseline and 6 months were independently predicted
for survival in the HU group. These results highlight the beneficial
effect of HU, which seems to modify the natural history of SCD and raise
the issue of expanding its use in all SCD patients. (Blood. 2010; 115:
2354-2363)
Συγγραφείς:
Voskaridou, Ersi
Christoulas, Dimitrios
Bilalis, Antonios and
Plata, Eleni
Varvagiannis, Konstantinos
Stamatopoulos, George
and Sinopoulou, Klio
Balassopoulou, Aggeliki
Loukopoulos,
Dimitris
Terpos, Evangelos
