Fetal hemoglobin in sickle cell anemia: Determinants of response to hydroxyurea

Martin H. Steinberg, Zhi Hong Lu, Franca B. Barton, Michael L. Terrin, Samuel Charache, George J Dover

Research output: Contribution to journalArticle

Abstract

Hydroxyurea (HU) can increase fetal hemoglobin (HbF) in sickle cell anemia (HbSS). To identify determinants of the HbF response, we studied 150 HU-treated patients grouped by quartiles of change in HbF from baseline to 2 years. Half of the HU-assigned patients had long-term increments in HbF. In the top two quartiles, HbF increased to 18.1% and 8.8%. These patients had the highest baseline neutrophil and reticulocyte counts, and largest treatment-associated decrements in these counts. In the lower two quartiles, 2-year HbF levels (4.2% and 3.9%) and blood counts changed little from baseline. In the highest HbF response quartile, myelosuppression developed in less than 6 months, compliance was best, and final doses of HU were 15 to 22.5 mg/kg. All four quartiles had substantial increases of F cells in the first year. This was maintained for 2 years only in the top three quartiles. Leukocyte and reticulocyte counts decreased initially in all quartiles, but drifted back toward baseline levels in the lowest HbF response quartile. Initial HbF level and phenotype of the F-cell production (FCP) locus were not associated with HbF response, but absence of a Central African Republic (CAR) haplotype was. Bone marrow ability to withstand HU treatment may be important for sustained HbF increases during HU treatment of HbSS.

Original languageEnglish (US)
Pages (from-to)1078-1088
Number of pages11
JournalBlood
Volume89
Issue number3
StatePublished - Feb 1 1997
Externally publishedYes

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Fetal Hemoglobin
Hydroxyurea
Sickle Cell Anemia
Reticulocyte Count
Central African Republic
Leukocyte Count
Haplotypes
Compliance
Bone
Neutrophils
Blood
Therapeutics
Bone Marrow
Phenotype

ASJC Scopus subject areas

  • Hematology

Cite this

Steinberg, M. H., Lu, Z. H., Barton, F. B., Terrin, M. L., Charache, S., & Dover, G. J. (1997). Fetal hemoglobin in sickle cell anemia: Determinants of response to hydroxyurea. Blood, 89(3), 1078-1088.

Fetal hemoglobin in sickle cell anemia : Determinants of response to hydroxyurea. / Steinberg, Martin H.; Lu, Zhi Hong; Barton, Franca B.; Terrin, Michael L.; Charache, Samuel; Dover, George J.

In: Blood, Vol. 89, No. 3, 01.02.1997, p. 1078-1088.

Research output: Contribution to journalArticle

Steinberg, MH, Lu, ZH, Barton, FB, Terrin, ML, Charache, S & Dover, GJ 1997, 'Fetal hemoglobin in sickle cell anemia: Determinants of response to hydroxyurea', Blood, vol. 89, no. 3, pp. 1078-1088.
Steinberg MH, Lu ZH, Barton FB, Terrin ML, Charache S, Dover GJ. Fetal hemoglobin in sickle cell anemia: Determinants of response to hydroxyurea. Blood. 1997 Feb 1;89(3):1078-1088.
Steinberg, Martin H. ; Lu, Zhi Hong ; Barton, Franca B. ; Terrin, Michael L. ; Charache, Samuel ; Dover, George J. / Fetal hemoglobin in sickle cell anemia : Determinants of response to hydroxyurea. In: Blood. 1997 ; Vol. 89, No. 3. pp. 1078-1088.
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