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Sci Rep. 2018 May 29;8(1):8293. doi: 10.1038/s41598-018-26033-z.

The effect of N-acetyl-l-cysteine (NAC) on liver toxicity and clinical outcome after hematopoietic stem cell transplantation.

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ECM, KFC, Department of Laboratory Medicine, Karolinska Institutet, Stockholm, Sweden.
Department of Clinical and Experimental Medicine, Linköping University, Linköping, Sweden.
Center for Allogeneic Stem Cell Transplantation, Karolinska University Hospital, Stockholm, Sweden.
Department of Oncology and Pathology, Karolinska Institutet, Stockholm, Sweden.
College of Medicine, University of Sharjah, Sharjah, UAE.
ECM, KFC, Department of Laboratory Medicine, Karolinska Institutet, Stockholm, Sweden.
Experimental Cancer Medicine, Clinical Research Center, Karolinska University Hospital, Huddinge, Sweden.


Busulphan (Bu) is a myeloablative drug used for conditioning prior to hematopoietic stem cell transplantation. Bu is predominantly metabolized through glutathione conjugation, a reaction that consumes the hepatic glutathione. N-acetyl-l-cysteine (NAC) is a glutathione precursor used in the treatment of acetaminophen hepatotoxicity. NAC does not interfere with the busulphan myeloablative effect. We investigated the effect of NAC concomitant treatment during busulphan conditioning on the liver enzymes as well as the clinical outcome. Prophylactic NAC treatment was given to 54 patients upon the start of busulphan conditioning. These patients were compared with 54 historical matched controls who did not receive NAC treatment. In patients treated with NAC, aspartate transaminase (AST), alanine transaminase (ALT) and alkaline phosphatase (ALP) were significantly (P < 0.05) decreased after conditioning compared to their start values. Within the NAC-group, liver enzymes were normalized in those patients (30%) who had significantly high start values. No significant decrease in enzyme levels was observed in the control group. Furthermore, NAC affected neither Bu kinetics nor clinical outcome (sinusoidal obstruction syndrome incidence, graft-versus-host disease and/or graft failure).


NAC is a potential prophylactic treatment for hepatotoxicity during busulphan conditioning. NAC therapy did not alter busulphan kinetics or affect clinical outcome.

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