Background
Thalassemia is the most common single gene inherited hematopoietic
disorder[1]. In β-thalassemia, hemoglobin (Hb)
production is insufficient because of mutations or deletions ofHBB gene, causing an imbalance between α-globin and β-globin.
Complete deficiency of β-globin is regarded as β0 and
partial β-globin deficiency is regarded as β+.
According to the requirement of transfusion, β-thalassemia is classified
into non-transfusion-dependent β-thalassemia (NTDT) (transfusions
required under certain conditions such as infection, pregnancy or
surgery) and transfusion-dependent β-thalassemia (TDT) (regular and
lifelong transfusions required)[2]. Patients with
β-hemoglobinopathies with higher fetal hemoglobin (HbF) level present
with milder phenotypes than those with a lower HbF level.
Inducers of HbF, such as hydroxyurea and histone deacetylase inhibitors,
are effective in improving hemoglobin level in some patients with
β-thalassemia[3, 4].Thalidomide is an
immunomodulatory agent with antiangiogenic properties. Clinical studies
have demonstrated that thalidomide exerts promising effects on
ameliorating anemia in patients with myeloproliferative disorders,
sickle cell disease and β-thalassemia[4-7].
Previous studies have also shown that thalidomide is effective in some
adult patients with β-thalassemia[7-9], but still
not recommended to children for scarce evidence of safety and efficacy.
Therefore, we performed this retrospective study to provide more
evidence for the use of thalidomide in children with TDT β-thalassemia.
Furthermore, we evaluated the potential predictors for response to
thalidomide in children with TDT.