Multifocal Lymphangioendotheliomatosis With Thrombocytopenia

North, Paula E.; Kahn, Teri A.; Cordisco, Maria; Dadras, Soheil S.; Detmar, Michael; Frieden, Ilona J.

doi:10.1001/archderm.140.5.599

Cited by 110 publications

(74 citation statements)

References 35 publications

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“…Most reported cases display decreased severity of gastrointestinal bleeding after 2 or 3 years of age. 1 Therefore, the clinical improvement in our patients, who received treatment early in life, may not be attributed to the natural history of the disease.…”

Section: Discussionmentioning

confidence: 81%

“…However, neither platelet count within the normal range nor absence of skin lesions is classically reported. 1 Biopsy specimens from patient 3 were negative for D2-40, with focal positivity for LYVE1. Negative staining for LYVE1 does not exclude the diagnosis of MLT because focal positivity might be missed on a small biopsy specimen.…”

Section: Discussionmentioning

confidence: 93%

“…As for MLT, these diseases both exhibit lymphatic differentiation. 1,8 In addition, LYVE1 or D2-40 expression may be associated with a degree of platelet trapping because these proteins are known to bind to platelets and/or be associated with maturation of capillaries.…”

Section: Discussionmentioning

confidence: 99%

“…The various treatment attempts were based on drugs used for MIH with extracutaneous involvement (corticosteroids, 1 vincristine, 6 interferon alfa-2a, 1,6 and propranolol 6 ), but they produced limited success. Bevacizumab is a humanized monoclonal antibody that binds 5 isoforms of human vascular endothelial growth factor, and recent study results for this drug were encouraging in 2 cases of MLT.…”

Section: Discussionmentioning

confidence: 99%

“…The disease is characterized by multifocal cutaneous vascular lesions that are negative for glucose transporter 1 (GLUT-1), and it features thrombocytopenia and extracutaneous disease but mostly severe gastrointestinal involvement. [1][2][3][4][5] The prognosis is grim, with high mortality often linked to gastrointestinal bleeding and unsatisfactory treatment. 6 Because MLT is a recently described and rare condition, 1 all facets of the disease are still unknown, and a treatment strategy remains to be established.…”

mentioning

confidence: 99%

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Multifocal Lymphangioendotheliomatosis With Thrombocytopenia: Clinical Features and Response to Sirolimus

et al. 2015

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International audienceMultifocal lymphangioendotheliomatosis with thrombocytopenia (MLT) is a recently described glucose transporter 1-negative multifocal vascular disorder with significant morbidity and mortality. However, data are lacking on the clinical spectrum, long-term prognosis, and treatment of MLT. It is often confused with multifocal infantile hemangioma, but the conditions must be differentiated for appropriate assessment and therapeutic management. Treatments for MLT have been disappointing, and the treatments classically used for infantile hemangioma are often ineffective. We report 3 newborn cases featuring various clinical and biological phenotypes of MLT: 1 patient had severe brain involvement and died early; another had no thrombocytopenia; and the third had nearly no skin involvement. Histologically, all were negative for glucose transporter 1 and positive for the lymphatic marker lymphatic vessel endothelial hyaluronan receptor 1 or D2-40 (∼38-kDa O-linked transmembrane sialoglycoprotein podoplanin). Two cases with severe gastrointestinal bleeding were treated with sirolimus 0.1 mg/k per day, which was efficient after the first month of treatment. MLT clinically presents in various forms, and when complicated by widespread or severe extracutaneous involvement, initial aggressive therapeutic intervention is justified. The pathogenesis of MLT remains unclear, but lymphatic differentiation is widely acknowledged. Because of its antiangiogenic properties, including anti-lymphangiogenesis, sirolimus offers an adequate and targeted therapeutic approach for ML

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Section: Discussionmentioning

confidence: 81%

Section: Discussionmentioning

confidence: 93%

Section: Discussionmentioning

confidence: 99%