Red blood cells free α-haemoglobin pool: a biomarker to monitor the β-thalassemia intermedia variability. The ALPHAPOOL study

Br J Haematol. 2017 Oct;179(1):142-153. doi: 10.1111/bjh.14800. Epub 2017 Jun 23.

Abstract

The severity of β-thalassaemia (β-thal) intermedia is mainly correlated to the degree of imbalanced α/non α-globin chain synthesis. The phenotypic diversity of β-thal depends on this imbalance and reflects all possible combinations of α- and β-globin genotypes, levels of fetal haemoglobin (HbF) and co-inheritance of other modulating factors. This study aimed to demonstrate the validity of a new surrogate of α/non α-globin biosynthetic ratio by measuring the soluble α-Hb pool in lysed red blood cells. Our results confirm that the α-Hb pool measurement allows a good discrimination between β-thal intermedia patients, controls and α-thal patients (P < 0·003). Receiver operator characteristic analyses revealed an area under the curve of 0·978 for the α-Hb pool measurement at a threshold of 120 ng free α-Hb/mg of total Hb/ml of haemolysate (ppm) with a sensitivity and specificity of 86% and 100%, respectively, to discriminate between β-thal and not β-thal subjects. Significant correlations were observed between the α-Hb pool and biological parameters of β-thal, the most significant association being observed with red cell hexokinase activity. This study indicates that the α-Hb pool could be a new marker for assistance in diagnostic orientation of β-thal intermedia patients and may be clinically useful for monitoring the evolution of the disequilibrium of globin synthesis in response to treatments.

Keywords: alpha-haemoglobin pool; haemoglobinopathies; severity test; thalassaemia intermedia; α/non α-globin biosynthesis.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Biomarkers
  • Case-Control Studies
  • Erythrocytes / metabolism*
  • Female
  • France
  • Genotype
  • Hematologic Tests
  • Humans
  • Male
  • Middle Aged
  • Mutation
  • Reproducibility of Results
  • Sensitivity and Specificity
  • Young Adult
  • alpha-Globins / genetics
  • alpha-Globins / metabolism*
  • alpha-Thalassemia / blood
  • alpha-Thalassemia / genetics
  • beta-Globins / genetics
  • beta-Thalassemia / blood*
  • beta-Thalassemia / diagnosis*
  • beta-Thalassemia / genetics

Substances

  • Biomarkers
  • alpha-Globins
  • beta-Globins