NAC prophylaxis after allo-HSCT
Trial question
What is the role of N-acetyl-L-cysteine prophylaxis in patients with allogeneic HSCT?
Study design
Single center
Open label
RCT
Population
Characteristics of study participants
43.0% female
57.0% male
N = 120
120 patients (52 female, 68 male).
Inclusion criteria: high-risk patients with acute leukemia undergoing haploidentical HSCT.
Key exclusion criteria: hypersensitivity to N-acetyl-L-cysteine; bronchial asthma; ejection fraction < 50%, creatinine ≥ 1.5 times the ULN; TBIL or aminotransferase ≥ 2 times the ULN.
Interventions
N=80 N-acetyl-L-cysteine prophylaxis (400 mg TID from 14 days pre-HSCT to after 2 months).
N=40 nonprophylaxis (no N-acetyl-L-cysteine prophylaxis).
Primary outcome
Poor graft function and prolonged isolated thrombocytopenia at 60 days
7.5%
22.5%
22.5 %
16.9 %
11.3 %
5.6 %
0.0 %
N-acetyl-L-cysteine
prophylaxis
Nonprophylaxis
Significant
decrease ▼
NNT = 6
Significant decrease in poor graft function and prolonged isolated thrombocytopenia at 60 days (7.5% vs. 22.5%; HR 0.317, 95% CI 0.11 to 0.89).
Safety outcomes
No significant differences in infections, gastrointestinal adverse events.
Conclusion
In high-risk patients with acute leukemia undergoing haploidentical HSCT, N-acetyl-L-cysteine prophylaxis was superior to nonprophylaxis with respect to poor graft function and prolonged isolated thrombocytopenia at 60 days.
Reference
Yu Wang, Yuan Kong, Hong-Yan Zhao et al. Prophylactic NAC promoted hematopoietic reconstitution by improving endothelial cells after haploidentical HSCT: a phase 3, open-label randomized trial. BMC Med. 2022 Apr 27;20(1):140.
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