The effect of prolonged administration of hydroxyurea on morbidity and mortality in adult patients with sickle cell syndromes: results of a 17-year, single-center trial (LaSHS).
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.
Voskaridou
Ersi
E
Thalassemia Center, Laikon General Hospital, GR-11526 Athens, Greece. ersi_voskaridou@yahoo.com
Christoulas
Dimitrios
D
Bilalis
Antonios
A
Plata
Eleni
E
Varvagiannis
Konstantinos
K
Stamatopoulos
George
G
Sinopoulou
Klio
K
Balassopoulou
Aggeliki
A
Loukopoulos
Dimitris
D
Terpos
Evangelos
E
D000328:Adult; D000368:Aged; D000755:Anemia, Sickle Cell / Q000188:drug therapy* / Q000235:genetics / Q000401:mortality*; D000986:Antisickling Agents / Q000008:administration & dosage* / Q000009:adverse effects; D001803:Blood Transfusion / Q000706:statistics & numerical data; D005260:Female; D005319:Fetal Hemoglobin / Q000235:genetics; D005500:Follow-Up Studies; D006451:Hemoglobin, Sickle / Q000235:genetics; D006760:Hospitalization / Q000706:statistics & numerical data; D006801:Humans; D006918:Hydroxyurea / Q000008:administration & dosage* / Q000009:adverse effects; D008297:Male; D008875:Middle Aged; D009017:Morbidity; D015999:Multivariate Analysis; D011237:Predictive Value of Tests; D011446:Prospective Studies; D016019:Survival Analysis; D013997:Time Factors; D055815:Young Adult
D000986:Antisickling Agents; D006451:Hemoglobin, Sickle; D005319:Fetal Hemoglobin; D006918:Hydroxyurea