Can N-acetylcysteine reduce red blood cell transfusion burden in patients with transfusion-dependent β-thalassemia?

• Published in: Pediatric hematology and oncology 2024-05, Vol.41 (4), p.251-259
• Main Authors: Bahoush, Gholamreza, Rahab, Mahdi, Ahmadvand, Parnian
• Format: Article
• Language: English
• Subjects: Acetylcysteine - therapeutic use; beta-Thalassemia; Erythrocyte Transfusion; Hemoglobins - analysis; Humans; Iron Overload - drug therapy; Iron Overload - etiology; Quality of Life
• ISSN: 0888-0018; 1521-0669
• DOI: 10.1080/08880018.2023.2292556

Patients with beta-thalassemia major require lifelong and frequent red blood cell transfusions for survival, impacting their quality of life and life expectancy. This treatment approach poses risks of organ damage, iron overload, and increased transfusion-transmitted diseases. N-acetylcysteine (NAC) has been studied for its potential antioxidant effects on hemoglobin stability, aiming to reduce the burden of red blood cell transfusions. To explore this possibility further, we conducted a quasi-experimental study involving 35 individuals with thalassemia major over six months All subjects were already receiving iron chelators and blood transfusions. They were given a daily oral dose of 10 mg/kg NAC for three months. After three months of treatment with NAC, the serum levels of ferritin and liver enzymes (SGOT and SGPT) did not show significant changes (  = 0.35,  = 0.352, and  = 0.686, respectively). However, the red blood cell transfusion burden was significantly reduced in all patients after NAC therapy (  = 0.029), with no corresponding decrease in serum hemoglobin levels (  = 0.931), indicating maintained hemoglobin concentration despite reduced transfusion volume. The study indicates that NAC can effectively decrease the burden of red blood cell transfusions without significant toxicity in these patients. This finding suggests the potential for NAC as a cost-effective and manageable treatment option for these patients. A larger clinical trial with more robust statistical methods could further confirm these results and pave the way for using NAC as a valuable therapeutic agent for managing beta-thalassemia major patients.