Role of Sirolimus in Treatment of Microcystic , Mixed Lymphatic and Vascular Malformations

Recruiting

• Conditions: Combined Vascular Malformation; Vascular Malformations; Microcystic Lymphatic Malformation
• Interventions: Drug: Sirolimus 1Mg Oral Tablet

• Registration Number: NCT06160739
• Lead Sponsor: Sohag University

Brief Summary

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.

Detailed Description

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. Sirolimus is a natural macrolide isolated from a bacteria strain of the Streptomyces genus \& Streptomyces hygroscopicus . It was initially used as an antibiotic and antifungal agent, subsequent studies have revealed impressive cytostatic, antiproliferative, and immunosuppressive properties . Sirolimus not only prevents the growth of abnormal lymphatics but also induces the partial regression of lesions, without apparent effects on normal lymphatics .

Study Timeline

• Study Start: 2023-11-20
• Study First Submitted: 2023-11-29
• Study First Submitted QC: 2023-11-29
• Status Verified: 2023-12
• Study First Posted: 2023-12-07
• Last Update Submitted: 2023-12-08
• Last Update Posted: 2023-12-15
• Primary Completion: 2024-09-01
• Study Completion: 2024-09-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 10

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 .

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
group A	Sirolimus 1Mg Oral Tablet	microcystic \& mixed lymphatic malformation
group B	Sirolimus 1Mg Oral Tablet	vascular malformation

Primary Outcome Measures

Name	Time	Method
changes in size of the lesion	1 year	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

Trial Locations

Locations (1)

Sohag university Hospital; 🇪🇬 Sohag, Egypt