|
 Previous Article | Table of Contents | Next Article 
A cautionary note regarding hydroxyurea in sickle cell disease
EP Vichinsky and BH Lubin
Department of Hematology/Oncology, Children's Hospital Oakland Research
Institute, CA 94609.
Hydroxyurea can increase fetal hemoglobin (HbF) and improve the clinical
course of sickle cell disease (SCD) patients. However, several issues of
hydroxyurea therapy remain unresolved, including differences in patients'
drug clearance, predictability of drug response, reversibility of sickle
cell disease-related organ damage by hydroxyurea, and the efficacy of
elevated HbF. We treated two patients with hydroxyurea for periods of 1 to
4 years, monitoring clinical course and laboratory parameters at regular
intervals. The first patient (patient A) had a history of chronic pain and
extensive hospitalizations. The second patient (patient B) had a history of
stroke and refused to continue with chronic transfusion therapy and
chelation. Both patients showed a fivefold to tenfold increase in HbF (5%
to 25%, 3% to 31%). However, patient A developed an acute chest syndrome,
despite an HbF level of 20%. After red blood cell transfusions for hypoxia,
the HbF level decreased to 5%. When hydroxyurea dosage was increased,
pancytopenia developed and was not resolved until 2 months after
hydroxyurea was discontinued; Patient B developed a cerebral hemorrhage on
hydroxyurea; he died shortly thereafter. His HbF level was 21% before
death. We noted an increase in HbF and a general improvement in the two
patients. However, both experienced major SCD-related complications despite
HbF levels over 20%. Our findings also suggest that the progressive
vascular changes associated with SCD are unlikely to be dramatically
affected by increased HbF levels. Because neither the efficacy nor the
toxicity of hydroxyurea have been thoroughly investigated, physicians
should be cautious in prescribing hydroxyurea for patients with SCD before
completion of the National Clinical Trial.
Volume 83,
Issue 4,
pp. 1124-1128,
02/15/1994
Copyright © 1994 by The American Society of Hematology
 CiteULike  Connotea  Del.icio.us  Digg  Reddit  Technorati What's this?
This article has been cited by other articles:
|
|
|
|
 
S. Lanzkron, J. J. Strouse, R. Wilson, M. C. Beach, C. Haywood, H. Park, C. Witkop, E. B. Bass, and J. B. Segal
An Evidence-based Review for the NIH Consensus Development Conference on Hydroxyurea for the Treatment of Adults with Sickle Cell Disease
Ann Intern Med,
May 5, 2008;
(2008)
0000605-200806170-00221.
[Abstract]
[Full Text]
|
|
|
|
|
|
|
F. Bernaudin, G. Socie, M. Kuentz, S. Chevret, M. Duval, Y. Bertrand, J.-P. Vannier, K. Yakouben, I. Thuret, P. Bordigoni, et al.
Long-term results of related myeloablative stem-cell transplantation to cure sickle cell disease
Blood,
October 1, 2007;
110(7):
2749 - 2756.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
D. A. Lee and B. U. Mueller
Long-term Use of Hydroxyurea for Sickle Cell Anemia
JAMA,
August 13, 2003;
290(6):
753 - 754.
[Full Text]
[PDF]
|
|
|
|
|
|
|
K. J. Helton, W. C. Wang, L. W. Wynn, R. B. Khan, and R. G. Steen
The Effect of Hydroxyurea on Vasculopathy in a Child with Sickle Cell Disease
AJNR Am. J. Neuroradiol.,
November 1, 2002;
23(10):
1692 - 1696.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
R. J. Adams, K. Ohene-Frempong, and W. Wang
Sickle Cell and the Brain
Hematology,
January 1, 2001;
2001(1):
31 - 46.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
M de Montalembert, P Begue, F Bernaudin, I Thuret, D Bachir, and M Micheau
Preliminary report of a toxicity study of hydroxyurea in sickle cell disease
Arch. Dis. Child.,
November 1, 1999;
81(5):
437 - 439.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
R. E. Ware, S. A. Zimmerman, and W. H. Schultz
Hydroxyurea as an Alternative to Blood Transfusions for the Prevention of Recurrent Stroke in Children With Sickle Cell Disease
Blood,
November 1, 1999;
94(9):
3022 - 3026.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
G. D. Sher, G. D. Ginder, J. Little, S. Yang, G. J. Dover, and N. F. Olivieri
Extended Therapy with Intravenous Arginine Butyrate in Patients with {beta}-Hemoglobinopathies
N. Engl. J. Med.,
June 15, 1995;
332(24):
1606 - 1610.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
S. Charache, M. L. Terrin, R. D. Moore, G. J. Dover, F. B. Barton, S. V. Eckert, R. P. McMahon, D. R. Bonds, and The Investigators of the Multicenter Study of Hydr
Effect of Hydroxyurea on the Frequency of Painful Crises in Sickle Cell Anemia
N. Engl. J. Med.,
May 18, 1995;
332(20):
1317 - 1322.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
