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Cryotherapy as prophylaxis against oral mucositis after high-dose melphalan and autologous stem cell transplantation for myeloma: a randomised, open-label, phase 3, non-inferiority trial

Abstract

The conditioning therapy used in connection with haematopoietic stem cell transplantation (HSCT) can induce painful oral mucositis, which has negative impacts on patient quality of life and survival, as well as on health-care costs. While cooling of the oral mucosa (cryotherapy) is regarded as standard prophylaxis against oral mucositis, the long duration of the treatment affects compliance owing to side effects. In this prospective, randomised trial, 94 patients (62 males/32 females; median age 59 years, range 34–69) with a diagnosis of myeloma who were undergoing autologous HSCT were randomised 1:1 to receive cryotherapy for 7 h (N = 46) or 2 h (N = 48). Oral mucositis was evaluated prospectively. No significant difference was observed with respect to the proportion of patients who showed grades 3 and 4 toxicity according to the WHO scale (2.1 and 4.3% for 2 and 7 h, respectively; 95% CI −0.09 to 0.049; p = 0.98) as between the groups. Two hours of cryotherapy was as effective as 7 h in terms of protecting against severe oral mucositis in connection with autologous HSCT for myeloma. This trial is registered with ClinicalTrials.gov (NCT03704597).

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Acknowledgements

The trial was funded by the Healthcare Committee, Region Västra Götaland (Grant number VGFOUREG-64832). We thank all the patients for their participation in the trial.

Author contributions

J-EJ, who was the chief investigator, and BH, who was the principal investigator, developed the study design. All the authors participated in the planning and performance of the study and in the interpretation of the results. BH, JB, and LH performed the oral assessments. J-EJ identified eligible patients and drafted the manuscript, the final version of which was seen and approved by all contributors.

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Correspondence to Jan-Erik Johansson.

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Johansson, JE., Bratel, J., Hardling, M. et al. Cryotherapy as prophylaxis against oral mucositis after high-dose melphalan and autologous stem cell transplantation for myeloma: a randomised, open-label, phase 3, non-inferiority trial. Bone Marrow Transplant 54, 1482–1488 (2019). https://doi.org/10.1038/s41409-019-0468-6

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