Prolonged isolated thrombocytopenia (PIT) that is refractory to conventional treatments has
remained a critical complication after allogeneic hematopoietic cell transplantation since
decades years ago. Recombinant human thrombopoietin (rhTPO) is the main therapy in clinical
practice, but remains low efficiency for PIT. Demethylating drugs have shown thier potential
in high-risk myelodysplastic syndromes (MDS) and acte myeloid leukemia (AML). In addition,
decitabine has demonstrated its efficacy of over 70% for response rate in treatment for PIT
in early clinical trials with elusive mechanism. Preliminary experiments revealed that PIT
was associated with abnormality of oxidation microenvironment, and N-Acetyl-L-cysteine (NAC)
was the most commonly used antioxidant. Therefore, the investigators have been wondering
whether Azacitidine in combination with NAC could improve PIT post HSCT and explore the
possible mechanism of it.
Phase:
Phase 2
Details
Lead Sponsor:
The First Affiliated Hospital of Soochow University