Sirolimus for Kaposiform Hemangioendothelioma With Kasabach-Merritt Phenomenon in Two Infants

Abstract

Kaposiform hemangioendothelioma is an aggressive vascular tumor with infiltrative growth that commonly occurs in infancy and is associated with a life-threatening consumptive coagulopathy, as well as Kasabach–Merritt phenomenon. Recently, promising results have shown that sirolimus had been successfully used to treat Kasabach–Merritt phenomenon without significant toxicity. However, the situation the authors encountered in treating infants was not so satisfactory. Here, the authors present 2 patients younger than 3 months with refractory Kaposiform hemangioendothelioma treated with sirolimus and experienced severe pneumonia. The outcomes suggest that it is necessary to keep an eye on any symptoms indicate the infection of respiratory tract and use the antibiotics in time. The 2 cases also remind us of the potential sign that indicate the recurrence of KMP, which refers to firmer lesion with deepen color, especially when it comes with complications.

Sirolimus combined with glucocorticoids in the treatment of Kasabach-Merritt phenomenon in a neonate: A case report

“…[13,14] Sirolimus, an mTOR inhibitor, has achieved better efficacy in the treatment of diseases associated with abnormal PIK3/AKT/ mTOR signaling pathway. [3,8,15] Efficacy of sirolimus in the treatment of KHE and slow-flow malformations achieved, [16][17][18] and the current commonly used dose is 0.8 mg with the trough serum concentration of 10 to 15 ng/mL. [19] However, there are also many studies pointing out that the dose and blood concentration are high, which can easily lead to stomatitis, gastrointestinal reactions, fever, rash, pain and even life-threatening Pneumocystis carinii pneumonia.…”

“…In severe cases, there is a tendency to bleeding and even death due to diffuse intravascular coagulation. [ 3 ] Therefore, early diagnosis and effective treatment are particularly important. [ 4 ] Recently, a child with knee KHE, complicated by KMP and scalp hematoma received sirolimus combined with methylprednisolone for treating KMP.…”

Efficacy of transcatheter arterial chemoembolization combined with sirolimus for treating Kasabach–Merritt phenomenon in infants, a retrospective study

“…Later, multiple individual reports on KMP treatment by sirolimus appeared around the world. Sirolimus achieved very good efficacy in KMP cases who relapsed after surgical resection and showed no response to other drug therapies [ 6 , 8–12 ], proving the safety and efficacy of sirolimus in the treatment of KMP. In 2017, Yi et al.…”

“…Sirolimus is an immunosuppressant, which can lower patients’ immunity, increasing the risk of infection. Furthermore, some patients die of pulmonary infection after taking sirolimus [ 8 ]. Considering the pulmonary toxicity of bleomycin used in treating TACE, the author recommended combined anti-infective therapy.…”

Case report: Kaposi hemangioendothelioma of the right upper limb with the Kasabach–Merritt phenomenon: A potentially lethal diagnostic challenge

“…Our experience suggests that sirolimus monotherapy of 12 months is a reasonable treatment for an infant with KHE with KMP, as it showed a good resolution of the lesion over the right upper limb and coagulopathy. We also monitored the patient for possible adverse effects of sirolimus including bone marrow suppression, metabolic derangements of hypercholesterolemia and hypertriglyceridemia, gastrointestinal side effects, as well as the rare side effect of pneumonia ( 14 , 25 ). The patient tolerated the oral sirolimus well with only transient hypertriglyceridemia.…”