Objective To observe the clinical efficacy of sirolimus combined with pingyangmycin in the treatment of extensive venous malformation with coagulation dysfunction.

Methods A total of 83 patients with extensive venous malformation and coagulation dysfunction from May 2017 to May 2023 were selected as study subjects. They were randomly divided into monotherapy group (n=45, treated with pingyangmycin) and combination group (n=38, treated with sirolimus combined with pingyangmycin) using a computer-generated random number table method. The changes in lesion volume, Visual Analogue Scale (VAS) score, coagulation function indicatorstissue-type plasminogen activator (t-PA), platelet alpha-granule membrane protein-140 (GMP-140), prothrombin time (PT), fibrinogen (FIB), and changes in score of quality of life were compared between the two groups before and after treatment. The treatment efficacy, adverse reactions, drug resistance rate, and recurrence rate were also compared.

Results After 3 and 6 months oftreatment, the lesion volume in the combination group was smaller than that in the monotherapy group, and the VAS score was lower, with statistically significant differences(P < 0.05). After 3 and 6 months of treatment, the lesion volume in both groups was smaller than that before treatment, and the VAS score was lower (P < 0.05). After 6 months of treatment, the lesion volume in both groups was smaller than that after 3 months of treatment, and the VAS score was lower (P < 0.05). After 3 and 6 months of treatment, the levels of t-PA and GMP-140 in the combination group were lower than those in the monotherapy group, the PT was shorter, and the FIB was higher (P < 0.05). After 3 and 6 months of treatment, the levels of t-PA and GMP-140 in both groups were lower than those before treatment, the PT was shorter, and the FIB was higher (P < 0.05). After 6 months of treatment, the levels of t-PA and GMP-140 in both groups were lower than those after 3 months of treatment, the PT was shorter, and the FIB was higher (P < 0.05). The total treatment efficacy in the combination group was higher than that in the monotherapy group (P < 0.05). After 6 months of treatment, the quality of life scores in both groups were higher than those before treatment, and the score in the combination group was higher than that in the monotherapy group (P < 0.05). The drug resistance rate in the combination group was lower than that in the monotherapy group (P < 0.05). There were no statistically significant differences in the recurrence rate and adverse reactions between the two groups (P>0.05).

Conclusion Sirolimus combined with pingyangmycin can promote the reduction of lesions, alleviate pain, improve treatment efficacy, coagulation function, and quality of life, and reduce the drug resistance rate in patients with extensive venous malformation and coagulation dysfunction, with high safety.