ABSTRACT
Kaposiform hemangioendothelioma is a locally invasive tumor and we were unable to find any previous reports of multifocal progression. Sirolimus, a mammalian target of rapamycin inhibitor, has been widely used to treat kaposiform hemangioendothelioma. Herein, we report a case of multifocal progressive kaposiform hemangioendothelioma, wherein sirolimus treatment caused severe thrombocytopenia. A 12-year-old East Asian girl presented with indurated dark-purple masses on her back. The patient had received three surgical interventions following the first appearance of the masses in 2012 and subsequent reappearances in 2014 and 2016. Kaposiform hemangioendothelioma was diagnosed based on radiological and pathological findings. Two more masses appeared in the following year. The patient was treated with oral sirolimus (2.5 mg/ m2/day) and developed grade 3 thrombocytopenia 8 days later. The patient was uneventfully relieved 5 days later after the withdrawal of sirolimus and the administration of appropriate medications. This rare case indicated that kaposiform hemangioendothelioma could be progressive with local metastatic characteristics in children. Besides, the severe sirolimus-induced complication highlights the importance of serum drug level monitoring during treatment. Physicians should be extremely cautious while treating kaposiform hemangioendothelioma patients with sirolimus.
Subject(s)
Anemia , Hemangioendothelioma , Kasabach-Merritt Syndrome , Sarcoma, Kaposi , Child , Female , Humans , Anemia/chemically induced , Hemangioendothelioma/diagnosis , Hemangioendothelioma/drug therapy , Kasabach-Merritt Syndrome/diagnosis , Kasabach-Merritt Syndrome/drug therapy , Sarcoma, Kaposi/chemically induced , Sarcoma, Kaposi/diagnosis , Sarcoma, Kaposi/drug therapy , Sirolimus/adverse effectsSubject(s)
Cell Proliferation/physiology , Immunosuppressive Agents/pharmacology , Melanins/metabolism , Melanocytes/metabolism , Sirolimus/pharmacology , Cell Proliferation/drug effects , Cells, Cultured , Dose-Response Relationship, Drug , Humans , Melanocytes/drug effects , Skin Transplantation/methods , TOR Serine-Threonine Kinases/antagonists & inhibitors , TOR Serine-Threonine Kinases/metabolismSubject(s)
Anti-Inflammatory Agents/administration & dosage , Immunosuppressive Agents/administration & dosage , Kasabach-Merritt Syndrome/diagnostic imaging , Kasabach-Merritt Syndrome/drug therapy , Prednisolone/administration & dosage , Sirolimus/administration & dosage , Administration, Oral , Drug Therapy, Combination , Humans , Infant , Male , Treatment OutcomeABSTRACT
BACKGROUND: The skin is one of the most affected organs in tuberous sclerosis complex and angiofibromas are seen in almost 80% of such patients. These benign tumors impose a great psycho-social burden on patients. OBJECTIVE: The aim of the study was to evaluate the effectiveness and tolerability of topical sirolimus for facial angiofibromas in patients with tuberous sclerosis complex. METHODS: This was a prospective, single-blinded, cross-over study which involved twelve patients. We investigated the effect and safety of topical 0.1% sirolimus, which was obtained by crushing sirolimus tablets and mixing it with petrolatum. The patients were asked to apply the cream to one side of their face, and vaseline to the other side. The effect of topical sirolimus was evaluated using the "facial angiofibroma severity index." RESULTS: There was a significant improvement in the redness and extension of the tumors on the sides to which the active ingredient was applied. Some side effects such as itching and irritation occurred in three patients, which were treated with topical hydrocortisone cream. CONCLUSION: Topical sirolimus appears to be a promising, fairly well tolerated treatment for facial angiofibromas in patients with tuberous sclerosis complex. Although its efficacy diminishes with time, repetitive usage is effective.