A Safety and Efficacy Study of NAC in Patients With TA-TMA

Hematopoietic stem cell transplantation (HSCT) has been commonly used as a potentially curative option in the treatment of various hematological malignancies. However, it may end up with serious complications in various systems, including the hemostatic system. HSCT associated thrombotic microangiopathy(TA-TMA) is a heterogeneous, fatal disorder seen within 100 days post-transplant and presents with thrombocytopenia, hemolysis, acute renal failure, mental status changes and involvement of other organs. Since the pathophysiology has not been clarified, there are no established treatments for TA-TMA several agents seem to have successful results. N-Acetylcysteine (NAC) is a small, simple molecule that began as a generic drug almost 40 years ago. It has since been approved by the FDA for many indications. Studies showed NAC reduce plasma VWF multimers and VWF multimeric size without an effect on the bleeding time in vitro and in vivo, thus proposed as a possible supplementary treatment for TTP. The investigators conducted an prospective clinical trial to evaluate the safety and efficiency of NAC in patients with TA-TMA.

Inclusion criteria

• Patients be scheduled to undergo HSCT;
• Not received decitabine 6 month ago;
• Without severe organ damage;
• ECOG 0-2;
• Informed consent were obtained.

Exclusion criteria

• Be sensitive to NAC;
• Bronchial asthma；
• Peptic ulcer；
• Severe organ damage；
• Pregnancy and breastfeeding women；