Recombinant Human Thrombopoietin Combined With Avatrombopag for Chemoradiotherapy-Induced Thrombocytopenia in Nasopharyngeal Carcinoma: A Prospective, Randomized Controlled Trial

<b>Aims/Background</b> Chemoradiotherapy-induced thrombocytopenia is a frequent dose-limiting toxicity in nasopharyngeal carcinoma (NPC). Thrombopoietin receptor agonists have shown potential in accelerating platelet recovery. This study aimed to evaluate the clinical efficacy of recombinant human thrombopoietin (rhTPO) combined with avatrombopag in treating chemoradiotherapy-induced thrombocytopenia in NPC patients. <b>Methods</b> A total of 42 NPC patients receiving chemoradiotherapy at Zhejiang Cancer Hospital between May 2023 and June 2024 were randomly allocated into an experimental group (n = 21) and a control group (n = 21) using the envelope method. The control group received avatrombopag alone, whereas the experimental group was administered rhTPO plus avatrombopag. Clinical efficacy, platelet parameters, serological indicators, and adverse events were assessed and compared. <b>Results</b> No significant difference was observed between groups in the overall response rate (95.24% vs. 71.43%) (χ² = 2.743, <i>p</i> = 0.098). Before treatment, platelet parameters and serological indicators did not differ significantly between groups (<i>p</i> > 0.05). Following treatment, platelet distribution width (PDW) and mean platelet volume (MPV) were significantly reduced, while plateletcrit (PCT) was significantly elevated in the experimental group compared to the control group (<i>p</i> < 0.05). Additionally, serum levels of thrombopoietin (TPO), signal transducer and activator of transcription 3 (STAT3), and mitogen-activated protein kinase (MAPK) were markedly elevated in the experimental group (<i>p</i> < 0.05). The incidence of adverse events did not differ significantly between groups (47.62% vs. 52.38%, χ² = 0.095, <i>p</i> = 0.758). <b>Conclusion</b> The combination of rhTPO and avatrombopag demonstrates potential clinical efficacy with a favorable safety profile. However, larger randomized controlled trials are warranted to validate these findings. <b>Clinical Trial Registration</b> Chinese Clinical Trial Registry (ChiCTR2400081992).