Multifocal lymphangioendotheliomatosis with thrombocytopenia/cutaneovisceral angiomatosis with thrombocytopenia

Abstract

Multifocal lymphangioendotheliomatosis with thrombocytopenia (MLT)/cutaneovisceral angiomatosis with thrombocytopenia, first reported as a new nosological entity in 2004, is an extremely rare vascular pathology of the neonatal period without hereditary nature. This disease has an extremely poor prognosis. According to the previously published data, this vascular pathology is characterized by involvement of the skin and internal organs in the pathological process. Lesions of the gastrointestinal tract are almost obligatory in this disease. MLT is associated with high mortality, which, according to some authors, can reach more than 50%. Fulminant gastrointestinal bleedings due to specific lesions of the intestinal mucosa, severe thrombocytopenia and consumption coagulopathy, are the leading cause of death in these patients. Currently, there are no standard treatment options for MLT. The data about the efficacy of glucocorticosteroids, vincristine, thalidomide, propranolol, and rapamycin in the treatment of MLT, are controversial. Here we present a case report of a patient with MLT with a classic clinical spectrum of this disease. We first report on the efficacy of a polychemotherapeutic regimen with liposomal doxorubicin in the treatment of this disease: the patient achieved a complete hematological response after 2 months from the treatment start. A durable clinical response was documented after 6 months of treatment. This disease represents challenges, both from a diagnostic and therapeutic point of view. Further studies are required to find the optimal treatment regimens for this condition. The patient's parents have consented to the use of de-identified clinical information and photos of the patient in scientific research and publications.