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Alpha-thalassaemia and response to hydroxyurea in sickle cell anaemia.
Eur J Haematol. 2014 Apr; 92(4):341-5.EJ

Abstract

BACKGROUND

Hydroxyurea (HU) reduces vaso-occlusive crises (VOC) and other complications of sickle cell anaemia (SCA). Alpha-thalassaemia is a known modifier of SCA. Studies on the efficacy of HU in SCA patients with α-thalassaemia have yielded varying results.

OBJECTIVE

To determine the effect of α-thalassaemia in response to HU therapy in the Multicenter Study of Hydroxyurea (MSH) cohort.

METHODS

We compared the laboratory parameters and VOC incidence in the MSH cohort stratified by the presence or the absence of α-thalassaemia.

RESULTS

Hydroxyurea showed significant (P = 0.001 for all baseline vs. follow-up comparisons) treatment effect on red cell indices irrespective of α-globin gene deletion. The magnitude of the HU-related changes was similar for mean corpuscular volume (MCV) (no α-thalassaemia 13 fl and α-thalassaemia 13 fl) and mean corpuscular haemoglobin (MCH) (no α-thalassaemia 4 pg and α-thalassaemia 4 pg) in both groups. Foetal haemoglobin (HbF) and F-cells also increased significantly with HU treatment in both groups. Total haemoglobin increased after HU treatment in both groups, but the increase was smaller and not statistically significant in patients with α-thalassaemia. In contrast, HU-related reduction in VOCs was more pronounced in patients with α-thalassaemia (VOC incidence rate ratio HU/placebo: 0.63 for α-thalassaemia and 0.54 for no α-thalassaemia (P for interaction 0.003).

CONCLUSION

Hydroxyurea decreases VOCs in SCA patients with and without α-thalassaemia, and the degree of VOC reduction was more pronounced in the patients with alpha-thalassaemia. Despite the lower baseline values, changes in standard laboratory parameters such as MCV and HbF percent remain useful in monitoring HU therapy in the presence of α-thalassaemia.

Links

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Authors+Show Affiliations

Darbari DS
Division of Haematology, Center for Cancer and Blood Disorders, Children's National Medical Center, Washington, DC, USA; Department of Paediatrics, The George Washington University Medical Center, Washington, DC, USA.
Nouraie M
No affiliation info available
Taylor JG
No affiliation info available
Brugnara C
No affiliation info available
Castro O
No affiliation info available
Ballas SK
No affiliation info available

MeSH

AdultAnemia, Sickle CellAntisickling AgentsFemaleGene DeletionHumansHydroxyureaMaleRisk FactorsTime FactorsTreatment OutcomeYoung Adultalpha-Globinsalpha-Thalassemia

Pub Type(s)

Journal Article
Multicenter Study
Research Support, N.I.H., Intramural

Language

eng

PubMed ID

24330217

Citation

Darbari, Deepika S., et al. "Alpha-thalassaemia and Response to Hydroxyurea in Sickle Cell Anaemia." European Journal of Haematology, vol. 92, no. 4, 2014, pp. 341-5.
Darbari DS, Nouraie M, Taylor JG, et al. Alpha-thalassaemia and response to hydroxyurea in sickle cell anaemia. Eur J Haematol. 2014;92(4):341-5.
Darbari, D. S., Nouraie, M., Taylor, J. G., Brugnara, C., Castro, O., & Ballas, S. K. (2014). Alpha-thalassaemia and response to hydroxyurea in sickle cell anaemia. European Journal of Haematology, 92(4), 341-5. https://doi.org/10.1111/ejh.12245
Darbari DS, et al. Alpha-thalassaemia and Response to Hydroxyurea in Sickle Cell Anaemia. Eur J Haematol. 2014;92(4):341-5. PubMed PMID: 24330217.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Alpha-thalassaemia and response to hydroxyurea in sickle cell anaemia. AU - Darbari,Deepika S, AU - Nouraie,Mehdi, AU - Taylor,James G, AU - Brugnara,Carlo, AU - Castro,Oswaldo, AU - Ballas,Samir K, Y1 - 2014/01/30/ PY - 2013/12/06/accepted PY - 2013/12/17/entrez PY - 2013/12/18/pubmed PY - 2014/12/15/medline KW - alpha-thalassaemia KW - hydroxyurea KW - sickle cell anaemia KW - vaso-occlusive pain crisis SP - 341 EP - 5 JF - European journal of haematology JO - Eur J Haematol VL - 92 IS - 4 N2 - BACKGROUND: Hydroxyurea (HU) reduces vaso-occlusive crises (VOC) and other complications of sickle cell anaemia (SCA). Alpha-thalassaemia is a known modifier of SCA. Studies on the efficacy of HU in SCA patients with α-thalassaemia have yielded varying results. OBJECTIVE: To determine the effect of α-thalassaemia in response to HU therapy in the Multicenter Study of Hydroxyurea (MSH) cohort. METHODS: We compared the laboratory parameters and VOC incidence in the MSH cohort stratified by the presence or the absence of α-thalassaemia. RESULTS: Hydroxyurea showed significant (P = 0.001 for all baseline vs. follow-up comparisons) treatment effect on red cell indices irrespective of α-globin gene deletion. The magnitude of the HU-related changes was similar for mean corpuscular volume (MCV) (no α-thalassaemia 13 fl and α-thalassaemia 13 fl) and mean corpuscular haemoglobin (MCH) (no α-thalassaemia 4 pg and α-thalassaemia 4 pg) in both groups. Foetal haemoglobin (HbF) and F-cells also increased significantly with HU treatment in both groups. Total haemoglobin increased after HU treatment in both groups, but the increase was smaller and not statistically significant in patients with α-thalassaemia. In contrast, HU-related reduction in VOCs was more pronounced in patients with α-thalassaemia (VOC incidence rate ratio HU/placebo: 0.63 for α-thalassaemia and 0.54 for no α-thalassaemia (P for interaction 0.003). CONCLUSION: Hydroxyurea decreases VOCs in SCA patients with and without α-thalassaemia, and the degree of VOC reduction was more pronounced in the patients with alpha-thalassaemia. Despite the lower baseline values, changes in standard laboratory parameters such as MCV and HbF percent remain useful in monitoring HU therapy in the presence of α-thalassaemia. SN - 1600-0609 UR - https://www.unboundmedicine.com/medline/citation/24330217/Alpha_thalassaemia_and_response_to_hydroxyurea_in_sickle_cell_anaemia_ DB - PRIME DP - Unbound Medicine ER -