Cost and effectiveness comparison of sirolimus versus standard treatment in Kasabach-Merritt phenomenon: a real-world evidence study in Thailand

The conventional treatment of Kasabach-Merritt Phenomenon (KMP) consists of corticosteroids with vincristine/vinblastine or others. The aim of the study is to compare the first-year direct costs and effectiveness between sirolimus and conventional treatment. A retrospective case-control study of KMP patients was conducted at a mean age of 9 months (1 day to 12 years) between 2000 and 2022 from four tertiary centers in Thailand. The direct costs, hematologic and clinical complete response (HCR, CCR), hospitalization, length of stay, and complications were compared. Of 29 patients, 13 underwent sirolimus (four upfront and nine were refractory to the conventional). The first-year total cost had no statistically significant difference between sirolimus VS conventional treatment (8,852.63 VS 9,083.56 USD: p value: 0.94). The therapeutics achievement was the same in both HCR (244.75 VS 168.94 days; p value: 0.60) and CCR (419.77 VS 399.87 days; p value: 0.90). The subgroup analysis of the first-line sirolimus (n = 4) compared with the conventional (n = 25) showed a more reduced total cost (4,907.84 VS 9,664.05 USD; p value: 0.26) rendered net total cost of -4,756.21 USD per patient (cost saving). A more significant contrast of therapeutic achievement by reduction of both HCR (11.67 VS 224.20 days; p value: 0.36) and CCR (38.50 VS 470.88 days; p value: 0.04) was shown. The sirolimus had no difference in hospitalization, length of stay, and complications. Even though, it was unable to identify significant differences in cost-effectiveness. Sirolimus is suitable for all patients who have diagnosis of KMP either for rescue therapy or first-line treatment.