Sirolimus with or without steroid therapy

In a prospective study that assessed the efficacy and safety of sirolimus for the treatment of complicated vascular anomalies, 13 patients with kaposiform hemangioendotheliomas were treated with sirolimus.[31]

In a prospective study that assessed the efficacy and safety of sirolimus for the treatment of complicated vascular anomalies, 13 patients with kaposiform hemangioendotheliomas were treated with sirolimus.[31]

In patients with kaposiform hemangioendotheliomas and Kasabach-Merritt phenomenon, ten of ten patients had partial responses, with normalization of their platelet count and fibrinogen at the end of 6 and 12 courses.

In a prospective study that assessed the efficacy and safety of sirolimus for the treatment of complicated vascular anomalies, 13 patients with kaposiform hemangioendotheliomas were treated with sirolimus.[31]

Of the three patients with kaposiform hemangioendotheliomas without Kasabach-Merritt phenomenon, two patients experienced partial responses by the end of course 12, and the third patient with multifocal bony disease had disease progression.

In a prospective study that assessed the efficacy and safety of sirolimus for the treatment of complicated vascular anomalies, 13 patients with kaposiform hemangioendotheliomas were treated with sirolimus.[31]

Side effects were minimal in this group of young patients, and no patient with a kaposiform hemangioendothelioma required a dose adjustment or was removed from the study because of toxicity of sirolimus.

A retrospective study of sirolimus therapy in patients who had nearly all received previous other treatments reported a complete response rate of 73%. All patients assessed as having Kasabach-Merritt phenomenon had recovery of platelet counts between 1 day and 3 weeks (mean, 1.3 weeks).[34]

A retrospective study of sirolimus therapy in patients who had nearly all received previous other treatments reported a complete response rate of 73%. All patients assessed as having Kasabach-Merritt phenomenon had recovery of platelet counts between 1 day and 3 weeks (mean, 1.3 weeks).[34]

One death occurred in this study from a respiratory infection in a child with a pleural effusion and multifocal disease involving the thoracic cavity. Biopsy was not routinely used in this study to confirm diagnosis, raising the possibility that this child had an unrecognized complex lymphatic anomaly.

A multicenter, retrospective cohort study analyzed 52 Chinese patients with progressive kaposiform hemangioendotheliomas. Thirty-seven patients (71%) had Kasabach-Merritt phenomenon. Those without Kasabach-Merritt phenomenon received sirolimus alone, and 21 of the patients with Kasabach-Merritt phenomenon received a combination of sirolimus and prednisone.[30]

A multicenter, retrospective cohort study analyzed 52 Chinese patients with progressive kaposiform hemangioendotheliomas. Thirty-seven patients (71%) had Kasabach-Merritt phenomenon. Those without Kasabach-Merritt phenomenon received sirolimus alone, and 21 of the patients with Kasabach-Merritt phenomenon received a combination of sirolimus and prednisone.[30]

Overall, 96% of patients at 6 months and 98% of patients at 12 months demonstrated improvement in notable symptoms and/or had improved complications.

A single case report of a child with a kaposiform hemangioendothelioma who developed recurrence of pain and fibrosis years after initial therapy and was treated with sirolimus for 26 months observed the following:[33]

A single case report of a child with a kaposiform hemangioendothelioma who developed recurrence of pain and fibrosis years after initial therapy and was treated with sirolimus for 26 months observed the following:[33]

The patient's contracture and range of motion improved, the lesion shrank, and the child was well 2 years later.

A prospective randomized study of patients with kaposiform hemangioendothelioma associated with Kasabach-Merritt syndrome compared sirolimus monotherapy with sirolimus in combination with prednisolone. The primary outcome was defined as achievement of a durable platelet response (platelet count >100 × 109/L) at week 4.[35][Level of evidence A3]

A prospective randomized study of patients with kaposiform hemangioendothelioma associated with Kasabach-Merritt syndrome compared sirolimus monotherapy with sirolimus in combination with prednisolone. The primary outcome was defined as achievement of a durable platelet response (platelet count >100 × 109/L) at week 4.[35][Level of evidence A3]

At week 4, a durable platelet response was achieved in 35 of 37 patients who received sirolimus and prednisolone, compared with 24 of 36 patients who received sirolimus monotherapy (difference, 27.9%; 95% CI, 10.0%–44.7%).

A prospective randomized study of patients with kaposiform hemangioendothelioma associated with Kasabach-Merritt syndrome compared sirolimus monotherapy with sirolimus in combination with prednisolone. The primary outcome was defined as achievement of a durable platelet response (platelet count >100 × 109/L) at week 4.[35][Level of evidence A3]

Compared with the sirolimus monotherapy group, the combination treatment group showed improvements in measures of durable platelet responses at all points during the initial 3-week treatment period. The durable platelet responses included median platelet counts during weeks 1 to 4, an increased number of patients achieving fibrinogen stabilization at week 4, and objective lesion responses at 12 months.

A prospective randomized study of patients with kaposiform hemangioendothelioma associated with Kasabach-Merritt syndrome compared sirolimus monotherapy with sirolimus in combination with prednisolone. The primary outcome was defined as achievement of a durable platelet response (platelet count >100 × 109/L) at week 4.[35][Level of evidence A3]

The frequencies of total and serious adverse events were similar in both groups.

A prospective randomized study of patients with kaposiform hemangioendothelioma associated with Kasabach-Merritt syndrome compared sirolimus monotherapy with sirolimus in combination with prednisolone. The primary outcome was defined as achievement of a durable platelet response (platelet count >100 × 109/L) at week 4.[35][Level of evidence A3]

Patients who received combination therapy had lower total disease sequelae and fewer blood transfusions.