Introduction
multi-organ damage, multiple myeloma, acute hemolytic reactions, transfusion-related infectious diseases, non-transfusion-dependent β-thalassemia, Thalassemia, leprosy, blood transfusions, transfusion-dependent, thalassemia, NTDT
MULTIPLE MYELOMA, THALASSEMIA, LEPROSY, THALASSEMIA, EVENTS, IRON OVERLOAD, COMPLICATIONS, HEREDITARY DISEASE
Thalassemia is a common single gene hereditary disease, characterized by insufficient hemoglobin (Hb). Based on the globin gene involved, thalassemia may generally be classified as α-thalassemia or β-thalassemiaβ-thalassemia, on the basis of blood transfusion requirement, is classified into transfusion-dependent β-thalassemia (TDT) and non-transfusion-dependent β-thalassemia (NTDT). Lifelong and regular blood transfusions are required for TDT patients, while NTDT patients require only occasional blood transfusions, if anyAt present, β-thalassemia seriously threatens public health, and the main therapies for patients with β-thalassemia include regular blood transfusion and iron chelation. However, chronic blood transfusion poses a significant risk of iron overload and subsequent multi-organ damage, as well as an increased risk of transfusion-related infectious diseases and acute life-threatening events such as acute hemolytic reactions, and anaphylaxisOwing to the risks and limitations of current treatment options, fetal hemoglobin (HbF) inducers have markedly attracted clinicians’ attention in recent years. A number of studies have demonstrated the widespread use of hydroxyurea (HU) in patients with β-thalassemia because of its HbF inducing propertiesOther therapeutic options in TDT and NTDT patients include DNA methyl-transferase inhibitors and histone deacetylase inhibitors which can also induce HbF production, however, their safety and efficacy is yet to be determinedRecently, drug repositioning, also referred to as drug repurposing or re-tasking has gained recognition as an alternative strategy to discover new pharmacological effects of an existing drug that has well-proven pharmacokinetics and safety in humansThalidomide, a well-known drug, has previously been repositioned two times with success (first in 1998, for complications of leprosy and then in 2006, for multiple myeloma as first line drug)