Multi-institutional, randomized, controlled trial to assess the efficacy and tolerability of a reusable, self-contained cryotherapy delivery device.

Purpose: Cryotherapy is an effective mucositis intervention for selected chemotherapy regimens, but reliance on ice chips has limited its applicability. Our objective was to assess the efficacy and tolerability of a reusable, self-contained, device as an alternative delivery mode.

Methods: A total of 164 patients randomized 2:1 received the device (Chemo Mouthpiece; CMP]) + best supportive care (BSC) (arm A) or BSC only (control, arm B), across 16 study sites. Inclusion criteria allowed broad tumor diagnosis and chemotherapy regimens. During the first two cycles of treatment, patients completed daily questionnaires in which they graded oral pain (OP) and analgesic use (AU) to control mouth pain. A questionnaire assessed device tolerability at the trial's end.

Results: The majority of patients received chemotherapy for breast cancer (arm A 52.7%, arm B 61.1%). Colorectal cancers were the next most common diagnosis. The percentage of visits in which any OP (> 0) in cycles 1 and 2 was reported was less among device-using individuals (CMP 17.8% vs Control 24.6%; p < 0.001). Whereas 26% of controls (arm B) required analgesics during their two cycles of treatment, the frequency was 11.3% among arm A (p = 0.01). Arm B AU was reported in 7.7% of visits compared to 1.8% of arm A visits (p < 0.001). End-of-study assessments of device tolerability were favorable.

Conclusions: CMP use for 14 days following chemotherapy infusion mitigated chemotherapy-associated OP and AU. The effect was seen over two cycles of treatment with known stomatotoxic agents having both short and long half-lives. The device was well tolerated.

Trial Registration: ClinicalTrials.gov (NCT04595838) Posted August 31, 2020.