Combination erythropoietin-hydroxyurea therapy in sickle cell disease: Experience from the National Institutes of Health and a literature review

Jane A. Little, Vicki R. McGowan, Gregory J. Kato, Kristine S. Partovi, Jordan J. Feld, Irina Maric, Sabrina Martyr, James G. Taylor VI, Roberto F. Machado, Theo Heller, Oswaldo Castro, Mark T. Gladwin

Research output: Contribution to journalArticle

Abstract

Erythropoietin is being used more widely in the management of sickle cell disease (SCD, inclusive of homozygous sickle beta, SS, and compound heterozygous sickle beta thalassemia, Sβ° thal), often in conjunction with hydroxyurea (HU). Herein, we summarize the published experience with erythropoietin use in SCD, in 39 patients (SS, n=30; Sβ° thal, n=9) who were treated between 1990 and 1996; and in 13 patients with sickle syndromes (SS, n=12, compound heterozygous SC disease, n=1) who were treated with erythropoietin or darbepoietin at the National Institutes of Health (NIH) since 2002. The dose range of erythropoietin for SCD in the published series, at a median of >200 U/Kg/dose, is higher than that used in end-stage renal disease. The median duration of erythropoietin therapy was ≥3 months, with minimal reported side-effects. At the NIH, the median age of sickle syndrome patients who received erythropoietin or darbepoietin (both referred to as EPO in the NIH series) was 51 (24 to 70) years; 12/13 patients had sickle-associated pulmonary hypertension. Eleven out of the 13 patients were treated with both HU and EPO for > 4 months (median of 11 months on EPO) without complication. Of the 13 patients, five (all SS) with pulmonary hypertension were given EPO for reticulocytopenia (

Original languageEnglish (US)
Pages (from-to)1076-1083
Number of pages8
JournalHaematologica
Volume91
Issue number8
StatePublished - Aug 2006
Externally publishedYes

Fingerprint

Hydroxyurea
National Institutes of Health (U.S.)
Sickle Cell Anemia
Erythropoietin
Pulmonary Hypertension
Hemoglobin SC Disease
Therapeutics
beta-Thalassemia
Chronic Kidney Failure

Keywords

  • Erythropoietin
  • Hydroxyurea
  • Pulmonary hypertension
  • Sickle cell disease

ASJC Scopus subject areas

  • Hematology

Cite this

Little, J. A., McGowan, V. R., Kato, G. J., Partovi, K. S., Feld, J. J., Maric, I., ... Gladwin, M. T. (2006). Combination erythropoietin-hydroxyurea therapy in sickle cell disease: Experience from the National Institutes of Health and a literature review. Haematologica, 91(8), 1076-1083.

Combination erythropoietin-hydroxyurea therapy in sickle cell disease : Experience from the National Institutes of Health and a literature review. / Little, Jane A.; McGowan, Vicki R.; Kato, Gregory J.; Partovi, Kristine S.; Feld, Jordan J.; Maric, Irina; Martyr, Sabrina; Taylor VI, James G.; Machado, Roberto F.; Heller, Theo; Castro, Oswaldo; Gladwin, Mark T.

In: Haematologica, Vol. 91, No. 8, 08.2006, p. 1076-1083.

Research output: Contribution to journalArticle

Little, JA, McGowan, VR, Kato, GJ, Partovi, KS, Feld, JJ, Maric, I, Martyr, S, Taylor VI, JG, Machado, RF, Heller, T, Castro, O & Gladwin, MT 2006, 'Combination erythropoietin-hydroxyurea therapy in sickle cell disease: Experience from the National Institutes of Health and a literature review', Haematologica, vol. 91, no. 8, pp. 1076-1083.
Little, Jane A. ; McGowan, Vicki R. ; Kato, Gregory J. ; Partovi, Kristine S. ; Feld, Jordan J. ; Maric, Irina ; Martyr, Sabrina ; Taylor VI, James G. ; Machado, Roberto F. ; Heller, Theo ; Castro, Oswaldo ; Gladwin, Mark T. / Combination erythropoietin-hydroxyurea therapy in sickle cell disease : Experience from the National Institutes of Health and a literature review. In: Haematologica. 2006 ; Vol. 91, No. 8. pp. 1076-1083.
@article{629cc0173bee4b54a5d2bb49339b477c,
title = "Combination erythropoietin-hydroxyurea therapy in sickle cell disease: Experience from the National Institutes of Health and a literature review",
abstract = "Erythropoietin is being used more widely in the management of sickle cell disease (SCD, inclusive of homozygous sickle beta, SS, and compound heterozygous sickle beta thalassemia, Sβ° thal), often in conjunction with hydroxyurea (HU). Herein, we summarize the published experience with erythropoietin use in SCD, in 39 patients (SS, n=30; Sβ° thal, n=9) who were treated between 1990 and 1996; and in 13 patients with sickle syndromes (SS, n=12, compound heterozygous SC disease, n=1) who were treated with erythropoietin or darbepoietin at the National Institutes of Health (NIH) since 2002. The dose range of erythropoietin for SCD in the published series, at a median of >200 U/Kg/dose, is higher than that used in end-stage renal disease. The median duration of erythropoietin therapy was ≥3 months, with minimal reported side-effects. At the NIH, the median age of sickle syndrome patients who received erythropoietin or darbepoietin (both referred to as EPO in the NIH series) was 51 (24 to 70) years; 12/13 patients had sickle-associated pulmonary hypertension. Eleven out of the 13 patients were treated with both HU and EPO for > 4 months (median of 11 months on EPO) without complication. Of the 13 patients, five (all SS) with pulmonary hypertension were given EPO for reticulocytopenia (",
keywords = "Erythropoietin, Hydroxyurea, Pulmonary hypertension, Sickle cell disease",
author = "Little, {Jane A.} and McGowan, {Vicki R.} and Kato, {Gregory J.} and Partovi, {Kristine S.} and Feld, {Jordan J.} and Irina Maric and Sabrina Martyr and {Taylor VI}, {James G.} and Machado, {Roberto F.} and Theo Heller and Oswaldo Castro and Gladwin, {Mark T.}",
year = "2006",
month = "8",
language = "English (US)",
volume = "91",
pages = "1076--1083",
journal = "Haematologica",
issn = "0390-6078",
publisher = "Ferrata Storti Foundation",
number = "8",

}

TY - JOUR

T1 - Combination erythropoietin-hydroxyurea therapy in sickle cell disease

T2 - Experience from the National Institutes of Health and a literature review

AU - Little, Jane A.

AU - McGowan, Vicki R.

AU - Kato, Gregory J.

AU - Partovi, Kristine S.

AU - Feld, Jordan J.

AU - Maric, Irina

AU - Martyr, Sabrina

AU - Taylor VI, James G.

AU - Machado, Roberto F.

AU - Heller, Theo

AU - Castro, Oswaldo

AU - Gladwin, Mark T.

PY - 2006/8

Y1 - 2006/8

N2 - Erythropoietin is being used more widely in the management of sickle cell disease (SCD, inclusive of homozygous sickle beta, SS, and compound heterozygous sickle beta thalassemia, Sβ° thal), often in conjunction with hydroxyurea (HU). Herein, we summarize the published experience with erythropoietin use in SCD, in 39 patients (SS, n=30; Sβ° thal, n=9) who were treated between 1990 and 1996; and in 13 patients with sickle syndromes (SS, n=12, compound heterozygous SC disease, n=1) who were treated with erythropoietin or darbepoietin at the National Institutes of Health (NIH) since 2002. The dose range of erythropoietin for SCD in the published series, at a median of >200 U/Kg/dose, is higher than that used in end-stage renal disease. The median duration of erythropoietin therapy was ≥3 months, with minimal reported side-effects. At the NIH, the median age of sickle syndrome patients who received erythropoietin or darbepoietin (both referred to as EPO in the NIH series) was 51 (24 to 70) years; 12/13 patients had sickle-associated pulmonary hypertension. Eleven out of the 13 patients were treated with both HU and EPO for > 4 months (median of 11 months on EPO) without complication. Of the 13 patients, five (all SS) with pulmonary hypertension were given EPO for reticulocytopenia (

AB - Erythropoietin is being used more widely in the management of sickle cell disease (SCD, inclusive of homozygous sickle beta, SS, and compound heterozygous sickle beta thalassemia, Sβ° thal), often in conjunction with hydroxyurea (HU). Herein, we summarize the published experience with erythropoietin use in SCD, in 39 patients (SS, n=30; Sβ° thal, n=9) who were treated between 1990 and 1996; and in 13 patients with sickle syndromes (SS, n=12, compound heterozygous SC disease, n=1) who were treated with erythropoietin or darbepoietin at the National Institutes of Health (NIH) since 2002. The dose range of erythropoietin for SCD in the published series, at a median of >200 U/Kg/dose, is higher than that used in end-stage renal disease. The median duration of erythropoietin therapy was ≥3 months, with minimal reported side-effects. At the NIH, the median age of sickle syndrome patients who received erythropoietin or darbepoietin (both referred to as EPO in the NIH series) was 51 (24 to 70) years; 12/13 patients had sickle-associated pulmonary hypertension. Eleven out of the 13 patients were treated with both HU and EPO for > 4 months (median of 11 months on EPO) without complication. Of the 13 patients, five (all SS) with pulmonary hypertension were given EPO for reticulocytopenia (

KW - Erythropoietin

KW - Hydroxyurea

KW - Pulmonary hypertension

KW - Sickle cell disease

UR - http://www.scopus.com/inward/record.url?scp=33746942243&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=33746942243&partnerID=8YFLogxK

M3 - Article

C2 - 16885048

AN - SCOPUS:33746942243

VL - 91

SP - 1076

EP - 1083

JO - Haematologica

JF - Haematologica

SN - 0390-6078

IS - 8

ER -