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azacitidine + n-acetylcysteine
- Development stage
- Unknown
- Lead developer
- First Affiliated Hospital of Soochow University
- Modality
- Small Molecules
- Administration
- Intravenous, Subcutaneous, Oral
01
Overview
This combination therapy consists of **azacitidine**, a hypomethylating agent, and **n-acetylcysteine** (NAC), a potent antioxidant. It is currently being investigated by **The First Affiliated Hospital of Soochow University** for the treatment of prolonged isolated thrombocytopenia (PIT) in patients following allogeneic hematopoietic stem cell transplantation (HSCT). PIT is a serious complication characterized by delayed platelet recovery, which is often refractory to standard treatments like recombinant human thrombopoietin (rhTPO). The rationale for this combination is twofold: azacitidine is thought to promote megakaryocyte differentiation and platelet release through the epigenetic modulation of DNA methylation, while NAC addresses the abnormal oxidative microenvironment in the bone marrow by scavenging reactive oxygen species (ROS), thereby improving the conditions for hematopoiesis.
- Other names
- AZA + NACAzacitidine and N-Acetyl-L-cysteineAzacitidine and NAC
02
Targets
DNMT (DNA methyltransferase)
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