Clinical trial · Observational
Role of Sirolimus in Treatment of Microcystic , Mixed Lymphatic and Vascular Malformations
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
Lympho-vascular malformations result from errors in embryologic vasculogenesis involving capillaries, veins, arteries, lymphatics, or a combination of these. Infantile haemangiomas \& Vascular malformations like : Capillary malformations \& Venous malformations : they increase in size and never regress on their own. \& They are generally present at birth, they enlarge in response to infection, hormonal changes or trauma . Lymphatic malformations can be classified into macrocystic (cyst diameter \>1cm), microcystic (cyst diameter \<1 cm), or mixed , in macrocystic lymphatic malformations, surgery and sclerotherapy are effective . Surgery of microcystic lymphatic malformations remains challenging due to their infiltrative nature \& Sclerotherapy is often impossible. As especially large microcystic and mixed malformations are still a therapeutic challenge, pharmaceutical treatment as sirolimus is used in last years as main line of treatment with great efficacy.
Conditions
Conditions (3)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Combined Vascular Malformation | — | UNRESOLVED | — |
| Microcystic Lymphatic Malformation | Lymphangioma Circumscriptum | ALIAS | 0.90 |
| Vascular Malformations | — | UNRESOLVED | — |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Sirolimus 1Mg Oral Tablet | Drug | Sirolimus | ALIAS |
Design
Arms and outcomes
Arms (2)
- label
- group A
- description
- microcystic \& mixed lymphatic malformation
- interventionNames
- Drug: Sirolimus 1Mg Oral Tablet
- label
- group B
- description
- vascular malformation
- interventionNames
- Drug: Sirolimus 1Mg Oral Tablet
Primary outcomes (1)
- measure
- changes in size of the lesion
- timeFrame
- 1 year
- description
- efficacy of sirolimus in decreasing size of the lesion over time , by clinical exam of size lesion \& measurement of size of leesion by ultrasound \& MRI
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 6 Months
- Maximum age
- 12 Years
Show eligibility criteria text
Inclusion Criteria: * from age 6 months to 12 years , not before 6 months to complete their vaccination program \& maturation of hepatic enzymes . * Patients diagnosed with microcystic \& mixed Lymphatic malformations . * After failure of other lines of treatment as regard propranolol , steroid for infantile haemangiomas \& Vascular malformations and lymphovascular malformations . * After failure of surgical excision \& injection of bleomycin of Lympho-vascular malformations Exclusion Criteria: * Macrocystic Lymphatic malformations \& high flow vascular malformations like arterio-venous malformations . * An active infection that requires systemic treatment during the attack . * Side effects of the drug as ( history of an allergic reaction to sirolimus or patients who develop severe allergic reaction to drug during treatment , hyperlipidemia , leucopenia , etc… ) * Chronic liver or kidney disease or on chronic drug treatment as (steroids, interferon or chemotherapeutic agents) . * An immunodeficiency condition such as a human immunodeficiency viral infection or primary immunodeficiency disease. * Patients who received drug less than 6 months duration .
References
Publications (4)
- BACKGROUNDSadick M, Muller-Wille R, Wildgruber M, Wohlgemuth WA. Vascular Anomalies (Part I): Classification and Diagnostics of Vascular Anomalies. Rofo. 2018 Sep;190(9):825-835. doi: 10.1055/a-0620-8925. Epub 2018 Jun 6. PMID 29874693
- BACKGROUNDVlahovic AM, Vlahovic NS, Haxhija EQ. Sirolimus for the Treatment of a Massive Capillary-Lymphatico-Venous Malformation: A Case Report. Pediatrics. 2015 Aug;136(2):e513-6. doi: 10.1542/peds.2014-3469. Epub 2015 Jul 6. PMID 26148957
- BACKGROUNDMuller-Wille R, Wildgruber M, Sadick M, Wohlgemuth WA. Vascular Anomalies (Part II): Interventional Therapy of Peripheral Vascular Malformations. Rofo. 2018 Feb 7. doi: 10.1055/s-0044-101266. Online ahead of print. PMID 29415296
- BACKGROUNDAdams DM, Wentzel MS. The role of the hematologist/oncologist in the care of patients with vascular anomalies. Pediatr Clin North Am. 2008 Apr;55(2):339-55, viii. doi: 10.1016/j.pcl.2008.01.007. PMID 18381090