THIOTEPA

About THIOTEPA

Thiotepa is an alkylating agent with good ability to cross the blood-brain barrier and

has been widely used in the treatment of a variety of lymphohematopoietic diseases and pretreatment for hematopoietic stem cell transplantation. Thiotepa also shows a promising clinical outcome for induction chemotherapy of central nervous system (CNS) lymphoma and prevention and treatment of CNS invasion.

The number of hematopoietic stem cell transplantations in China has been increasing year by year, from fewer than 4,000 cases in 2013 to over 23,000 cases in 2024, among which allogeneic transplants reached 16,000 cases.

ABOUT Β-THALASSEMIA

β-thalassemia refers to an inherited mutation of the beta-globin gene, causing a reduced beta-globin chain of hemoglobin. It includes three main forms: β-thalassemia major (TM), also referred to as “Cooley’s anemia” and “Mediterranean anemia”; β-thalassemia intermedia (TI); and thalassemia minor, called “β-thalassemia carrier,” “β-thalassemia trait,” or “heterozygous β-thalassemia.”

TM is the most severe form of β-thalassemia and is usually brought to medical attention between ages 6 and 24 months. Bone marrow transplant and cord blood transplant offer the only potential cure for β-thalassemia.

The frequency of β-thalassemia mutations varies by regions of the world with the highest prevalence in the Mediterranean, the Middle-East, and Southeast and Central Asia. Approximately 68,000 children are born with various thalassemia syndromes each year. β-thalassemia is highly prevalent, with 80 to 90 million people reported to be carriers across the world (1.5% of the global population).

According to the data from different screening studies, thalassemia is the most commonly seen hereditary hemolytic disease in China. Trends in thalassemia prevalence in mainland China were not steady. The overall prevalence of β-thalassemia is 2.21%.