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

Last updated: December 8, 2023
Sponsor: Sohag University
Overall Status: Active - Recruiting

Phase

N/A

Condition

Birth Defects

Abnormal Blood Vessels (Arteriovenous Malformations)

Holoprosencephaly

Treatment

Sirolimus 1Mg Oral Tablet

Clinical Study ID

NCT06160739
soh-Med-23-11-16MS
  • Ages 6-12
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • from age 6 months to 12 years , not before 6 months to complete their vaccinationprogram & maturation of hepatic enzymes .
  • Patients diagnosed with microcystic & mixed Lymphatic malformations .
  • After failure of other lines of treatment as regard propranolol , steroid forinfantile haemangiomas & Vascular malformations and lymphovascular malformations .
  • After failure of surgical excision & injection of bleomycin of Lympho-vascularmalformations

Exclusion

Exclusion Criteria:

  • Macrocystic Lymphatic malformations & high flow vascular malformations likearterio-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 patientswho develop severe allergic reaction to drug during treatment , hyperlipidemia ,leucopenia , etc… )
  • Chronic liver or kidney disease or on chronic drug treatment as (steroids, interferonor chemotherapeutic agents) .
  • An immunodeficiency condition such as a human immunodeficiency viral infection orprimary immunodeficiency disease.
  • Patients who received drug less than 6 months duration .

Study Design

Total Participants: 10
Treatment Group(s): 1
Primary Treatment: Sirolimus 1Mg Oral Tablet
Phase:
Study Start date:
November 20, 2023
Estimated Completion Date:
September 01, 2024

Study 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 .

Connect with a study center

  • Sohag university Hospital

    Sohag,
    Egypt

    Active - Recruiting

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