Prime

Type your tag names separated by a space and hit enter

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).

Abstract

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.

Links

  • FREE Publisher Full Text
  • Authors+Show Affiliations

    ,

    Thalassemia Center, Laikon General Hospital, GR-11526 Athens, Greece. ersi_voskaridou@yahoo.com

    , , , , , , , ,

    Source

    Blood 115:12 2010 Mar 25 pg 2354-63

    MeSH

    Adult
    Aged
    Anemia, Sickle Cell
    Antisickling Agents
    Blood Transfusion
    Female
    Fetal Hemoglobin
    Follow-Up Studies
    Hemoglobin, Sickle
    Hospitalization
    Humans
    Hydroxyurea
    Male
    Middle Aged
    Morbidity
    Multivariate Analysis
    Predictive Value of Tests
    Prospective Studies
    Survival Analysis
    Time Factors
    Young Adult

    Pub Type(s)

    Clinical Trial, Phase II
    Journal Article

    Language

    eng

    PubMed ID

    19903897

    Citation

    TY - JOUR T1 - 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). AU - Voskaridou,Ersi, AU - Christoulas,Dimitrios, AU - Bilalis,Antonios, AU - Plata,Eleni, AU - Varvagiannis,Konstantinos, AU - Stamatopoulos,George, AU - Sinopoulou,Klio, AU - Balassopoulou,Aggeliki, AU - Loukopoulos,Dimitris, AU - Terpos,Evangelos, Y1 - 2009/11/10/ PY - 2009/11/12/entrez PY - 2009/11/12/pubmed PY - 2010/4/14/medline SP - 2354 EP - 63 JF - Blood JO - Blood VL - 115 IS - 12 N2 - 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. SN - 1528-0020 UR - https://www.unboundmedicine.com/medline/citation/19903897/full_citation L2 - http://www.bloodjournal.org/cgi/pmidlookup?view=long&pmid=19903897 ER -