Multifocal Lymphangioendotheliomatosis with Thrombocytopenia: Presentation of Two Cases Treated with Sirolimus

Lanöel, Agustina; Huamani, Adriana Natalia Torres; Feliú, Aurora; Sala, María Josefina; Álvarez, Mariana; Cervini, Andrea Bettina

doi:10.1111/pde.12879

Cited by 13 publications

(23 citation statements)

References 17 publications

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“…Given the rarity of this disease, there is limited data on treatment options. A case series of two patients by Lanöel et al 28 reported successful treatment with sirolimus. Prior to sirolimus, vincristine and steroids have been tried with minimal effect.…”

Section: Multifocal Lymphangiomatosis and Thrombocytopenia Or Cutaneomentioning

confidence: 99%

“…Prior to sirolimus, vincristine and steroids have been tried with minimal effect. [28][29][30] Bevacizumab (Avastin, San Francisco, California, United States), an antibody to vascular endothelial growth factor (VEGF), has been used to successfully eliminate GI bleeding in two case reports. Of note, both patients had side effects from the medication.…”

Section: Multifocal Lymphangiomatosis and Thrombocytopenia Or Cutaneomentioning

confidence: 99%

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Colorectal Vascular Anomalies

McGuire

Dickie

2020

Eur J Pediatr Surg

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Vascular anomalies represent a diverse group of tumors and malformations. Those involving the colon and rectum can greatly impact patients' quality of life. Proper workup to ensure the correct diagnosis can vary and may include endoscopic and radiographic studies. These lesions can also be challenging to treat and often require a multidisciplinary approach to ensure the best possible outcome. Treatment can include medical therapy, sclerotherapy, endoscopic, and operative intervention. Many patients will require multimodal therapy. We discuss the workup and management of vascular lesions of the colon and rectum.

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Section: Multifocal Lymphangiomatosis and Thrombocytopenia Or Cutaneomentioning

confidence: 99%

Section: Multifocal Lymphangiomatosis and Thrombocytopenia Or Cutaneomentioning

confidence: 99%

Colorectal Vascular Anomalies

McGuire

Dickie

2020

Eur J Pediatr Surg

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“…Multifocal lymphangioendotheliomatosis with thrombocytopenia (MLT) is a rare disorder first described by this term in 2004 by North et al 1 It has been described by different names in several other publications. 2 -4 MLT presents with vascular lesions seen on the skin and in the gastrointestinal (GI) tract. Intestinal lesions can result in severe bleeding and mortality in neonates.…”

Section: Introductionmentioning

confidence: 99%

“…There is no definitive treatment known for this condition, although some case reports have demonstrated success with sirolimus, a mammalian target of rapamycin inhibitor. 2 It has potent immunosuppressive, antiangiogenic, and antiproliferative properties. Hence, sirolimus is a treatment option for vascular tumors.…”

Section: Introductionmentioning

confidence: 99%

Novel Route of Sirolimus Administration in a Neonate

Clark

Chandrasekar

Nickleson

2017

Journal of Pharmacy Practice

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A term newborn presented with widespread cutaneous erythematous to bluish lesions since birth. He had extensive lesions in the gastrointestinal tract, brain, retina, heart, and bones. He also developed an intestinal perforation due to erosion of an intestinal lesion. Due to his critical status and clinical presentation, he was initially diagnosed with multifocal lymphangioendotheliomatosis with thrombocytopenia (MLT), and sirolimus treatment was initiated. Sirolimus was given by buccal route in this nonfeeding patient. Therapeutic serum levels were obtained comparable to enteral administration. Buccal mucosa was an effective novel route of sirolimus administration in this patient.

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“…Besides skin and gastrointestinal involvement, multiple other organs may be involved. There are no established treatments, although bevacizumab and sirolimus have been used with some success . The prognosis is poor, with high morbidity and mortality, often linked to gastrointestinal involvement, which may cause life‐threatening bleeding in newborns.…”

mentioning

confidence: 99%

Multifocal lymphangioendotheliomatosis without thrombocytopenia or clinical signs of systemic bleeding

Merino

Almaraz

Larrinoa

et al. 2019

Pediatric Dermatology

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Multifocal lymphangioendotheliomatosis with thrombocytopenia (MLT) is an extremely rare recently described disorder characterized by diffuse congenital skin and gastrointestinal vascular lesions that may be associated with gastrointestinal bleeding and thrombocytopenia. We herein present a case report of multifocal lymphangioendotheliomatosis without thrombocytopenia or extensive extracutaneous involvement (gastrointestinal bleeding). Given the high morbidity and mortality associated with this disease, it is important for clinicians to recognize this disorder in order to select the most appropriate therapeutic approach.

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Multifocal Lymphangioendotheliomatosis with Thrombocytopenia: Presentation of Two Cases Treated with Sirolimus

Cited by 13 publications

References 17 publications

Colorectal Vascular Anomalies

Colorectal Vascular Anomalies

Novel Route of Sirolimus Administration in a Neonate

Multifocal lymphangioendotheliomatosis without thrombocytopenia or clinical signs of systemic bleeding

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