Myelosuppression after busulfan conditioning in hematopoietic cell transplantation (HCT) may increase the risk of severe oral mucositis (OM) and prolonged oropharynx/esophageal mucositis in patients with hematologic malignancies, according to a study published in Supportive Care in Cancer

Chemotherapy conditioning with busulfan is a critical component prior to HCT, but has been associated with severe central nervous system and gastrointestinal adverse events, particularly mucositis. Nonmyeloablative (NMA) or reduced-intensity conditioning (RIC) with busulfan have been used in efforts to reduce toxicity, but its association with OM is poorly described.

For this retrospective study, authors evaluated the outcomes of 100 patients with hematologic malignancies (eg, leukemia, lymphoma, multiple myeloma) who underwent HCT after myeloablative conditioning (MAC) (n = 66) or NMA/RIC (n = 34) with busulfan. Data regarding OM severity/duration, opioid use, and total parenteral nutrition (TPN) use were collected. 

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Results showed that although the frequency and duration of OM were comparable between the 2 groups, patients who underwent MAC experienced higher OM severity (P<.009) and longer duration of mucositis in the oropharynx/esophagus (P=.022) compared with patients in the NMA/RIC group. 

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The MAC dose of busulfan was identified as a risk factor for OM grade 2 or greater (P=.002), as well as mucositis in the oropharynx/esophagus (P=.035), and an increased use of opioids was associated with a prolonged duration of oropharynx/esophageal mucositis (<.001).

Patients undergoing MAC, however, had a significantly longer overall survival (OS) vs patients undergoing NMA/RIC (P=.017). 

The authors concluded that “myelosuppression in regimens with busulfan are predisposed to a high risk of severe OM and to prolonged mucositis in the oropharynx/esophagus. OS was higher in the MAC group compared with the NMA/RIC group, but mucositis during transplantation was not associated with OS.” 


Eduardo FP, Bezinelli LM, Gobbi M, et al. Retrospective study of the digestive tract mucositis derived from myeloablative and non-myeloablative/reduced-intensity conditionings with busulfan in hematopoietic cell transplantation patient[published online August 14, 2018]. Support Care Cancer. doi: 10.1007/s00520-018-4362-3