Prophylactic cryotherapy for 2 hours prior to high-dose melphalan before autologous hematopoietic stem cell transplantation (HSCT) was as effective as 7 hours of cryotherapy in preventing grade 3 or 4 oral mucositis in patients with multiple myeloma. These results from a randomized, controlled phase 3 trial was published in Bone Marrow Transplantation.
The approach cools the oral mucosa and is considered to be the standard-of-care treatment for the prevention of oral mucositis in patients with multiple myeloma who are receiving high-dose melphalan prior to autologous HSCT. Based on the design of earlier studies on this approach, cryotherapy is typically administration for 7 hours; however, this duration of cryotherapy delivery has been associated with patient discomfort that can negatively affect patient adherence.
In this study, cryotherapy was initiated 30 minutes prior to a single high dose of melphalan in all patients. Patients were randomly assigned 1:1 to another 1.5 hours of cryotherapy or another 6.5 hours of cryotherapy. The primary end point of the study was noninferiority in the incidence of grade 3 or grade 4 oral mucositis according to World Health Organization (WHO) criteria.
Of the 94 patients included in the primary analysis, 48 and 46 patients underwent cryotherapy for 2 and 7 hours, respectively. There were no significant differences in the baseline characteristics of the 2 study arms. Rates of grade 3 or grade 4 oral mucositis were 2.1% and 4.3% in the 2-hour and 7-hour cryotherapy arms, respectively (95% CI, –0.09 to 0.049; P =.98). Interestingly, all patients in the 2-hour arm completed cryotherapy, whereas 7 of those in the 7-hour arm discontinued cryotherapy early.
In addition to concluding that 2-hour cryotherapy is equally as effective as 7-hour cryotherapy in preventing oral mucositis in patients with myeloma receiving high-dose melphalan before autologous HSCT, the authors also noted that “the true probability of moderate-to-severe oral mucositis with cryotherapy should rather be around 5%” and a significant number of patients did not develop mucositis at all.
Johansson JE, Bratel J, Hardling M, Heikki L, Mellqvist UH, Hasséus B. Cryotherapy as prophylaxis against oral mucositis after high-dose melphalan and autologous stem cell transplantation for myeloma: a randomized, open-label, phase 3, non-inferiority trial[published online February 4, 2019]. Bone Marrow Transplant. doi: 10.1038/s41409-019-0468-6