Abstract
BACKGROUND: Cachexia is a leading cause of death among individuals with advanced cancer, yet effective pharmacological treatments are lacking. In this single-center retrospective study, we aimed to investigate the efficacy and safety of tocilizumab for the treatment of cancer cachexia accompanied by systemic hyperinflammation. METHODS: Data were collected from 20 patients treated with tocilizumab and a control group of 20 patients matched for age, sex, and comorbidities. Both groups received corticosteroids. In the tocilizumab treatment group, patients received a single dose of tocilizumab (8 mg/kg, maximum 800 mg) in combination with corticosteroids. Weight, body mass index, liver metastasis, Eastern Cooperative Oncology Group score, patient-generated subjective global assessments, the Anorexia/Cachexia Subscale of the Functional Assessment of Anorexia/Cachexia Therapy, handgrip strength, neutrophil-to-lymphocyte ratio, and the C-reactive protein, hemoglobin, prealbumin, and albumin levels were recorded in both groups. RESULTS: Tocilizumab treatment favorably influenced the levels of patient biomarkers (p<0.05), ameliorated systemic inflammation, and demonstrated enhanced clinical short-term efficacy compared to the control group, including rates of symptomatic relief (60% vs. 20%, p = 0.024), improvement of serum PAB and ALB (70% vs. 25%, p = 0.004), weight gain >2% (45% vs. 15%, p = 0.038), and improvement of grip strength and 6-m walk speed (p<0.05). Treatment with tocilizumab was generally safe, with no observed increase in infection rates (10% vs. 15%, p = 0.633) or intensive care unit admissions (10% vs. 25%, p = 0.405), and was more favorable for restarting antitumor therapy (70% vs. 35%, p = 0.027). CONCLUSIONS: Tocilizumab, in combination with corticosteroids, is favorable for alleviating cancer cachexia with systemic hyperinflammation, despite the small sample size. Thus, this combination holds great potential as a novel strategy for treating cancer cachexia with systemic hyperinflammation.