6/28/23, 11:22 PM NAC prevention of PGF and PT Pathway Feedback Search Clinical Topics Home Studies NAC prevention of PGF and PT NAC prevention of PGF and PT Disease Disease Acute myeloid leukemia Acute promyelocytic leukemia 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 N = 120 57.0% male 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 22.5 % 22.5 https://web.pathway.md/studies/rec04C3PIOedSxDag 1/2 6/28/23, 11:22 PM NAC prevention of PGF and PT Pathway 5 16.9 % 11.3 % Significant decrease 7.5 5.6 % NNT = 7 0.0 % N-acetyl-L-cysteine prophylaxis Nonprophylaxis 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 were 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. Open reference URL https://web.pathway.md/studies/rec04C3PIOedSxDag 2/2