Trial stopped
Although the study was designed to enroll 116 patients to have a power of 80% to detect a 50% reduction in the rate of severe oral mucositis from an anticipated 50% in the placebo arm, a planned interim analysis conducted after approximately half of the target events occurred showed no difference in the rates of severe oral mucositis, and the trial was halted.
A total of 28 patients in the folinic acid group and 24 in the placebo group were available for the analysis.
The rate of grade 3 or 4 mucositis, the primary endpoint, was 46.6% in the folinic acid group, and 45.8% in the placebo group.
Respectively, the median duration of severe oral mucositis was 4 days in each group, days to neutrophil engraftment were a median of 12 and to platelet engraftment were a median of 13 days in each group, rates of febrile neutropenia were 57.1% and 58.3%, rates of bloodstream infections were 10.7% and 16.6%, rates of veno-occlusive disease were 7% and 12.5%, need for TPN occurred in 14.2% vs. 25%, need for opiates occurred in 78.5% vs. 66.6%, and median time to discharge was 18 and 19 days. As noted, none of the differences were statistically significant.
“These unequivocal interim results led to our decision to discontinue the study,” Dr. Yeshrun said.
Arnon Nagler, MD, MSc, from Sheba Medical Center Tel HaShomer at Tel Aviv University in Israel, who was not involved in the study, said in an interview “I think this is a very practical and important study, because we need evidence-based data such as this.”
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Dr. Arnon Nagler
Dr. Nagler, who comoderated the session where the data were presented, noted that the study was limited by the small number of patients and the lack of a subgroup analysis, but emphasized that the findings were important nonetheless.
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Dr. Maria Gilleece
His comoderator, Maria Gilleece, MD, director of the Yorkshire (England) Blood and Marrow Transplant Program, noted in an interview that folinic acid is frequently used in the United Kingdom for patients receiving high-dose methotrexate, ”and certainly, this study suggests that may be inappropriate.”
Rabin Medical Center sponsored the trial. Dr. Yeshrun, Dr. Nagler, and Dr. Gilleece reported no relevant conflicts of interest.
SOURCE: Yeshrun M. et al. TCT 2020. Abstract 61.