Corticosteroid-to-sirolimus sequential therapy: a case series of infantile kaposiform hemangioendothelioma associated with Kasabach-Merritt phenomenon
Abstract
Background Kaposiform hemangioendothelioma (KHE) with Kasabach-Merritt phenomenon (KMP) is a rare and life-threatening vascular tumor in infants, characterized by severe thrombocytopenia and consumptive coagulopathy. Although corticosteroids and sirolimus have both been used in treatment, there is no consensus on the optimal regimen, and evidence remains limited regarding sequential therapeutic strategies. This study aimed to explore the efficacy and safety of corticosteroid-to-sirolimus sequential therapy in a retrospective case series. Methods From June 2022 to December 2024, nine infants diagnosed with KHE and KMP at our hospital received corticosteroid-to-sirolimus sequential therapy. Initial treatment included intravenous methylprednisolone (4 mg/kg/day), followed by a switch to oral methylprednisolone (4 mg/kg every other day) in combination with oral sirolimus (0.8 mg/m2 twice daily), with dose adjustments to maintain sirolimus blood concentrations between 10–15 ng/mL. Treatment efficacy was assessed by platelet recovery time, tumor regression (via imaging), and overall response rates according to modified RECIST criteria. Safety outcomes were evaluated by monitoring adverse events graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events version 2.0 (NCI-CTCAE v2.0.). Follow-up lasted 6 months to 2 years. Results All patients achieved a platelet count ≥100×109/L within 3.0±1.7 days after corticosteroid initiation. Four patients…
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Evidence Hub reads this as a case series, done in people. Its evidence level is 5 of 5 (Very low · case / narrative / animal / in vitro / in silico). Case reports, narrative reviews, and animal, cell or computer-model studies give the weakest evidence for human health.
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