Overview
Multifocal lymphangioendotheliomatosis with thrombocytopenia (MLT), also known as cutaneovisceral angiomatosis with thrombocytopenia, is an extremely rare vascular disorder that is usually present at birth or develops in early infancy. In this condition, abnormal blood vessel growths (vascular lesions) form in multiple areas of the body, including the skin and the gastrointestinal (GI) tract. These lesions are made up of unusual cells that line blood and lymph vessels. A hallmark feature of MLT is a low platelet count (thrombocytopenia), which means the blood does not clot properly. This can lead to serious and sometimes life-threatening bleeding, especially from the GI tract. Babies and young children with MLT typically show red or reddish-brown skin spots or bumps that may bleed easily. Internal bleeding from the stomach or intestines can cause bloody stools, anemia, and the need for blood transfusions. The GI bleeding can be chronic and difficult to manage. Some children also develop bleeding in other organs. There is currently no cure for MLT. Treatment focuses on managing symptoms, particularly controlling bleeding episodes and maintaining adequate blood counts through transfusions. Various medications have been tried, including drugs that target blood vessel growth (such as sirolimus and bevacizumab), corticosteroids, and other therapies, but responses vary from patient to patient. Because the disease is so rare, treatment approaches are still evolving and are often tailored to each individual child's needs.
Also known as:
Key symptoms:
Red or reddish-brown skin spots or bumpsLow platelet count (thrombocytopenia)Gastrointestinal bleeding (bloody stools)Anemia from chronic blood lossEasy bruisingMultiple vascular lesions on the skinInternal bleeding from the stomach or intestinesNeed for frequent blood transfusionsPale skin due to anemiaFatigue and weaknessSkin lesions that may bleed when touched or injuredPoor weight gain or failure to thrive
Clinical phenotype terms (46)— hover any for plain English
Sporadic
Usually appears on its own, not inherited from a parent
Neonatal
Begins at or shortly after birth (first 4 weeks)
FDA & Trial Timeline
10 eventsTanta University — NA
Al-Azhar University — NA
Tanta University — NA
Ain Shams University — PHASE3
Cairo University — NA
Lars Vedel Kessing — PHASE2
Ain Shams University — PHASE3
Zhuan Zhang — NA
Bangladesh Medical University — PHASE2
Helse Fonna — NA
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
No FDA-approved treatments are currently listed for Multifocal lymphangioendotheliomatosis-thrombocytopenia syndrome.
View clinical trials →Clinical Trials
View all trials with filters →No actively recruiting trials found for Multifocal lymphangioendotheliomatosis-thrombocytopenia syndrome at this time.
New trials open frequently. Follow this disease to get notified.
Treatment Centers
8 centersBaylor College of Medicine Rare Disease Center ↗
Baylor College of Medicine
📍 Houston, TX
🏥 NORDStanford Medicine Rare Disease Center ↗
Stanford Medicine
📍 Stanford, CA
🔬 UDNNIH Clinical Center Undiagnosed Diseases Program ↗
National Institutes of Health
📍 Bethesda, MD
🔬 UDNUCLA UDN Clinical Site ↗
UCLA Health
📍 Los Angeles, CA
🔬 UDNBaylor College of Medicine UDN Clinical Site ↗
Baylor College of Medicine
📍 Houston, TX
🔬 UDNHarvard/MGH UDN Clinical Site ↗
Massachusetts General Hospital
📍 Boston, MA
🏥 NORDMayo Clinic Center for Individualized Medicine ↗
Mayo Clinic
📍 Rochester, MN
👤 Mayo Clinic Center for Individualized Medicine
🏥 NORDUCLA Rare Disease Day Program ↗
UCLA Health
📍 Los Angeles, CA
Travel Grants
No travel grants are currently matched to Multifocal lymphangioendotheliomatosis-thrombocytopenia syndrome.
Community
No community posts yet. Be the first to share your experience with Multifocal lymphangioendotheliomatosis-thrombocytopenia syndrome.
Start the conversation →Latest news about Multifocal lymphangioendotheliomatosis-thrombocytopenia syndrome
Disease timeline:
New recruiting trial: Buspirone and Melatonin for Depression Following Traumatic Brain Injury
A new clinical trial is recruiting patients for Multifocal lymphangioendotheliomatosis-thrombocytopenia syndrome
New recruiting trial: Testing Effects of Melatonin on Uterine Contractions in Women
A new clinical trial is recruiting patients for Multifocal lymphangioendotheliomatosis-thrombocytopenia syndrome
New recruiting trial: Effect of Melatonin in Patients With Diabetic Peripheral Neuropathy
A new clinical trial is recruiting patients for Multifocal lymphangioendotheliomatosis-thrombocytopenia syndrome
New recruiting trial: Sleep and Light Intervention (SALI) for Menopausal Mood Dysfunction
A new clinical trial is recruiting patients for Multifocal lymphangioendotheliomatosis-thrombocytopenia syndrome
New recruiting trial: Melatonin for Prevention of Kidney Injury
A new clinical trial is recruiting patients for Multifocal lymphangioendotheliomatosis-thrombocytopenia syndrome
New recruiting trial: Sleep, Diabetic Retinopathy and Melatonin
A new clinical trial is recruiting patients for Multifocal lymphangioendotheliomatosis-thrombocytopenia syndrome
New recruiting trial: The Use of Sublingual Melatonin Premedication in Geriatric Cataract Surgery
A new clinical trial is recruiting patients for Multifocal lymphangioendotheliomatosis-thrombocytopenia syndrome
New recruiting trial: Efficacy of Melatonin, Low-dose Quetiapine, or Placebo in Patients With Psychiatric Disorders and Comorbid Insomnia
A new clinical trial is recruiting patients for Multifocal lymphangioendotheliomatosis-thrombocytopenia syndrome
New recruiting trial: The Role of Melatonin as an Adjuvant Therapy in Childhood Pneumonia
A new clinical trial is recruiting patients for Multifocal lymphangioendotheliomatosis-thrombocytopenia syndrome
New recruiting trial: Comparative Evaluation of Melatonin Versus MTA on Vital Pulp Therapy in Young Permanent First Molars: An in Vivo Study
A new clinical trial is recruiting patients for Multifocal lymphangioendotheliomatosis-thrombocytopenia syndrome
Caregiver Resources
NORD Caregiver Resources
Support, advocacy, and financial assistance for caregivers of rare disease patients.
Mental Health Support
Rare disease caregiving can be isolating. Connect with counseling and peer support.
Family & Caregiver Grants
Financial assistance programs specifically for caregivers of rare disease patients.
Social Security Disability
Learn how rare disease patients may qualify for SSDI/SSI benefits.
Questions for your doctor
Bring these to your next appointment
- Q1.How severe is my child's condition, and what organs are affected?,What treatment options are available, and what are the risks and benefits of each?,How often will my child need blood or platelet transfusions?,What signs of bleeding should I watch for at home, and when should I go to the emergency room?,Are there any clinical trials or new therapies being studied for this condition?,Should we be seen at a specialized vascular anomalies center?,What activities should my child avoid, and how can we support normal development?
Common questions about Multifocal lymphangioendotheliomatosis-thrombocytopenia syndrome
What is Multifocal lymphangioendotheliomatosis-thrombocytopenia syndrome?
Multifocal lymphangioendotheliomatosis with thrombocytopenia (MLT), also known as cutaneovisceral angiomatosis with thrombocytopenia, is an extremely rare vascular disorder that is usually present at birth or develops in early infancy. In this condition, abnormal blood vessel growths (vascular lesions) form in multiple areas of the body, including the skin and the gastrointestinal (GI) tract. These lesions are made up of unusual cells that line blood and lymph vessels. A hallmark feature of MLT is a low platelet count (thrombocytopenia), which means the blood does not clot properly. This can l
How is Multifocal lymphangioendotheliomatosis-thrombocytopenia syndrome inherited?
Multifocal lymphangioendotheliomatosis-thrombocytopenia syndrome follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.
At what age does Multifocal lymphangioendotheliomatosis-thrombocytopenia syndrome typically begin?
Typical onset of Multifocal lymphangioendotheliomatosis-thrombocytopenia syndrome is neonatal. Age of onset can vary across affected individuals.
Which specialists treat Multifocal lymphangioendotheliomatosis-thrombocytopenia syndrome?
11 specialists and care centers treating Multifocal lymphangioendotheliomatosis-thrombocytopenia syndrome are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.