Efficacy and safety of Aiyu capsules for chemotherapy-induced leukopenia in cancer patients: A multi-center parallel-group randomized controlled trial

Abstract

Objective

This study aimed to assess the efficacy and safety of Aiyu capsules, a traditional Chinese medicine (TCM) formulation, in preventing and treating chemotherapy-induced leukopenia, as well as to evaluate their effects on patient-reported quality of life. The findings are intended to provide evidence supporting the adjunctive use of Aiyu capsules for the management of chemotherapy-associated bone marrow suppression.

Methods

In this multicenter, randomized controlled trial, 288 patients diagnosed with colorectal cancer, gastric cancer, lymphoma, or ovarian cancer were recruited from 20 centers, including Jiangxi Provincial People's Hospital, the First Affiliated Hospital of Guizhou University of Traditional Chinese Medicine, Shunde Hospital of Guangzhou University of Chinese Medicine, and the First Affiliated Hospital of Guangzhou University of Chinese Medicine, between May 2022 and February 2025. Participants were allocated in a 1:1 ratio to either the Aiyu capsules group or the control group using a stratified block dynamic randomization approach. Patients in the control group (n = 144) received standard chemotherapy and placebo therapy, whereas those in the Aiyu capsules group (n = 144) were administered oral Aiyu capsules beginning three days prior to chemotherapy initiation and continued for 21 consecutive days in conjunction with standard chemotherapy. Hematological parameters were monitored following chemotherapy and during the subsequent treatment cycle. Additional assessments included the utilization rate of granulocyte colony-stimulating factor (G-CSF), quality of life as measured by the Quality of Life Questionnaire-Core 30 (QLQ-C30), TCM syndrome scores, and the incidence and severity of adverse events (AEs) throughout the study period.

Results

Patients receiving Aiyu capsules demonstrated significantly greater improvements in quality of life and TCM syndrome scores compared with those receiving chemotherapy alone. At day 21, QLQ-C30 evaluations showed that the median change from baseline in the total score for items 1-28 was −2.50 in the Aiyu capsules group, compared with 0 in the control group (p < 0.0001), indicating a more pronounced enhancement in overall quality of life. Consistently, the median changes from baseline in global health status and overall quality of life scores were both 1 in the Aiyu capsules group and 0 in the control group (p < 0.0001). With regard to TCM-related outcomes, reductions in fatigue and palpitations at day 21 were significantly greater in the Aiyu capsules group (p < 0.005), and the decrease in total TCM syndrome scores was also more substantial (−2 vs. 0, p = 0.0002). The incidence of severe AEs was lower in the Aiyu capsules group than in the control group. Among all reported AEs, severe AEs accounted for 1.29% in the Aiyu capsules group versus 3.51% in the control group (p < 0.005). Similarly, grade 4 or higher AEs, as defined by Common Terminology Criteria for Adverse Event (CTCAE) criteria, occurred less frequently in the Aiyu capsules group (0.86% vs. 3.51%, p < 0.005).

Conclusion

When administered as an adjunct to standard chemotherapy in patients with colorectal cancer, gastric cancer, lymphoma, or ovarian cancer, Aiyu capsules were associated with meaningful improvements in quality of life and a reduction in the incidence of severe AEs, while maintaining a favorable safety profile.