Hydroxyurea therapy in β‐thalassaemia intermedia: improvement in haematological parameters due to enhanced β‐globin synthesis

• DOI: 10.1111/j.1365-2141.1995.tb05584.x
• Publication Date: 2008-03-12T11:27:52Z
• Authors: YI‐TAO ZENG; SHU‐ZHEN HUANG; ZHAO‐RUI REN; ZHI‐HONG LU; FAN‐YI ZENG; ALAN N. SCHECHTER; GRIFFIN P. RODGERS
• Keywords: Haemolysis; Ineffective erythropoiesis; Hemoglobinopathy

Summary. The β‐thalassaemias represent a heterogenous group of diseases resulting from decreased erythroid β‐globin mRNA expression and imbalanced a/β‐globin chain synthesis which are manifest clinically by ineffective erythropoiesis excessive haemolysis. Increasing levels haemoglobin F (HbF) pharmacological agents has been proposed to ameliorate the severity disease improving balance in globin synthesis. Hydroxyurea (HU), as an effective agent with low toxicity for activating 7‐globin gene, shown enhance HbF experimental animals patients sickle cell anaemia. However, previous trials HU β‐thalassaemia ambiguous, small number having increased In recent study effects Chinese j3 ‐thalassaemia we unexpectedly found that two unrelated intermedia demonstrated improvement effectiveness reflected increase concentration (from 4‐1 6‐3 g/dl, patient 1; 6‐5 97 2) red volume 68 104 fl, 85 after period excess 300 d low‐dosage treatment. These effects, however, appear be due increased,3‐globin biosynthesis, because percentage each total Hb increased. This was changes β/a ratio 0′301 0‐581, 0′348 0‐487, minimal biosynthesis. We conclude addition its known stimulating production, hydroxyurea may have more general role augmenting synthesis, including some thalassaemia who maintain capacity express normal chains.