Combined Vascular Malformation, Microcystic Lymphatic Malformation, Vascular Malformations
Conditions
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 .
Interventions
patients with Microcystic , Mixed Lymphatic and Vascular Malformations will be given sirolimus 1 mg oral tab for 3-6 months with follow up of lesion size by clinical exam & ultrasound & MRI to compare lesion size before & after use of the drug with observation of potential side effects & after exclusion of it before use of the drug
Sponsors
Sohag University
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
6 Months to 12 Years
Healthy volunteers
No
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 .
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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 |
Countries
Egypt
Contacts
Primary ContactTarek A Abd elsalam, resident
Backup ContactMohamed yousef, Ass. Prof.
Outcome results
None listed