Tehran University of Medical Sciences International Journal of Hematology-Oncology and Stem Cell Research 2008-2207 12 2 2018 04 05 77 83 EN Maryam Barkhordar Hematology-Oncology and Stem Cell Research Center, Tehran University of Medical Sciences, Tehran, Iran Mehdi Mohammadi Department of Clinical Pharmacy, Faculty of Pharmacy, Alborz University of Medical Sciences, Karaj, Iran Ahmad Reza Shamshiri Department of Epidemiology and Biostatistics, School of Public Health, Tehran University of Medical Sciences, Tehran, Iran Molouk Hadjibabaie Research Center for Rational Use of Drugs, Tehran University of Medical Sciences, Tehran, Iran. AND Department of Clinical Pharmacy, Faculty of Pharmacy, Tehran University of Medical Sciences, Tehran, Iran Ardeshir Ghavamzadeh Hematology-Oncology and Stem Cell Research Center, Tehran University of Medical Sciences, Tehran, Iran 2017 01 22 2018 01 07 Background: Allogeneic hematopoietic stem cell transplantation has been used widely to treat various types of malignant and non-malignant disorders. Graft-versus-host disease is one of the main complications of this procedure which is associated with considerable mortality and affects quality of life. Despite careful selection of HLA-matched donors and implementing immunosuppressive therapy, the incidence rate of graft-versus-host disease remains high. Macrolide antibiotics are well-known immunomodulatory agents and have been effective as prophylaxis for graft-versus-host disease in preclinical studies. Materials and Methods: Ninety-six adult patients with acute leukemia were recruited into a double-blind, randomized, placebo-controlled trial. All patients were first-time transplant candidates for a full-matched related or unrelated donor. Patients were allocated to receive azithromycin 500 mg daily (n=48) or placebo (n=48) from day -6 to +12. All patients received high-dose chemotherapy, standard immunosuppressive regimen and supportive care according to institutional protocols. Results: The incidence of acute graft-versus-host disease grade III-IV and chronic graft-versus-host disease garde I-III was not significantly different between the two study arms. Oral mucositis grade 1-3 occurred in significantly lower number of patients in the azithromycin group compared with placebo. Conclusion: Based on the results of this study, protective effect of azithromycin on graft-versus-host disease could not be demonstrated.     https://ijhoscr.tums.ac.ir/index.php/ijhoscr/article/view/738 https://ijhoscr.tums.ac.ir/index.php/ijhoscr/article/download/738/630