NAC for Hematopoietic Recovery in SAA

N-acetyl-L-cysteine for Promoting Hematopoietic Recovery in Patients With Severe Aplastic Anemia (SAA) After Haploidentical Transplantation -- a Prospective Single-arm Clinical Study

This is a prospective single-arm clinical study to evaluate the role of NAC among patients with severe aplastic anemia (SAA) can promote hematopoietic recovery after haploidentical transplantation.

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an effective treatment of severe aplastic anemia (SAA). However, poor hematopoietic reconstitution including poor graft function (PGF) and prolonged isolated thrombocytopenia (PT), remains a life-threatening complication after allo-HSCT. Especially with the increasing use of haploidentical allo-HSCT (haplo-HSCT) in the past ten years, PGF and PT have become growing obstacles contributing to high morbidity and mortality after allo-HSCT. A previous clinical prospective cohort study showed that NAC could improve the function of bone marrow endothelial progenitor cells and promote hematopoietic recovery among leukemia patients after haploidentical transplantation. Therefore, we hypothesized that the prophylactic administration of NAC could facilitate the recovery of hematopoietic capacity by improving the bone marrow microenvironment of patients with SAA after haploidentical transplantation.

Inclusion Criteria:

1. Diagnosed with SAA or vSAA
2. Aged 18-50
3. No severe organ injury
4. No uncontrolled active infections
5. Sign informed consent form, have the ability to comply with study and follow-up procedures

Exclusion Criteria:

1. Hypersensitivity to NAC or history of bronchial asthma
2. Life expectancy less than 30 days post-transplantation
3. Uncontrolled infections pre-transplantation
4. Cardiac dysfunction (particularly congestive heart failure, unstable coronary artery disease and serious cardiac ventricular arrhythmias requiring antiarrhythmic therapy)
5. Respiratory failure ( PaO2 ≤60mmHg)
6. Hepatic abnormalities (total bilirubin ≥2 times the upper limit of normal [ULN], alanine aminotransferase or aspartate aminotransferase ≥2 times the ULN)
7. Renal dysfunction (creatinine ≥1.5 times the ULN or creatinine clearance rate < 30 mL/min)
8. ECOG performance status ≥3
9. With any conditions not suitable for the trial (investigators' decision)