Clinical Trials /

Sirolimus in the Treatment for Infantile Hepatic Hemangioendothelioma(IEEH)

NCT04406870

Description:

Infantile hepatic hemangioendothelioma (IHHE) is an infantile hemangioma involving the liver.Since 2008, propranolol has been used for the treatment of hemangioma, and some researchers have also started to report the use of propranolol for the treatment of infantile hepatic hemangioma in children. Sirolimus can be used in patients with vascular malformations such as hemangiomas. IHHE is also an infantile hemangioma involving the liver,thus sirolimus may paly the role in treatment of IHHE.The clinical trial explores the efficacy of sequential treatment of sirolimus to refractory IHHE patients resistant with propranolol, to improve the therapeutic effect, reduce the side effects of traditional treatment methods (hormones, interferon), and reduce the number of operations and interventions and to provide a clinical basis for the application of the new therapeutics model of IHHE of "propranolol + sequential sirolimus treatment".

Related Conditions:
  • Hemangioendothelioma
Recruiting Status:

Not yet recruiting

Phase:

Phase 4

Trial Eligibility

Document

Title

  • Brief Title: Sirolimus in the Treatment for Infantile Hepatic Hemangioendothelioma(IEEH)
  • Official Title: Sirolimus in the Treatment for Propranolol-resistant Infantile Hepatic Hemangioendothelioma

Clinical Trial IDs

  • ORG STUDY ID: PRL-SRL-IHHE
  • NCT ID: NCT04406870

Conditions

  • Hemangioendothelioma of Liver

Interventions

DrugSynonymsArms
Sirolimus Oral ProductTORISEL, rapamycinIntervention
propranololxin de an (chinese name means making me comfortable)Intervention

Purpose

Infantile hepatic hemangioendothelioma (IHHE) is an infantile hemangioma involving the liver.Since 2008, propranolol has been used for the treatment of hemangioma, and some researchers have also started to report the use of propranolol for the treatment of infantile hepatic hemangioma in children. Sirolimus can be used in patients with vascular malformations such as hemangiomas. IHHE is also an infantile hemangioma involving the liver,thus sirolimus may paly the role in treatment of IHHE.The clinical trial explores the efficacy of sequential treatment of sirolimus to refractory IHHE patients resistant with propranolol, to improve the therapeutic effect, reduce the side effects of traditional treatment methods (hormones, interferon), and reduce the number of operations and interventions and to provide a clinical basis for the application of the new therapeutics model of IHHE of "propranolol + sequential sirolimus treatment".

Detailed Description

      According to the World Health Organization(WHO) classification of digestive system tumors in
      the fourth edition in 2010, infantile hepatic hemangioendothelioma (IHHE) is an infantile
      hemangioma involving the liver. It is a benign tumor, which can be subgrouped into focal,
      multifocal, and diffusing in children, as well as combining vascular malformations of the
      skin, brain, digestive tract and other organs.Since 2008, propranolol has been used for the
      treatment of hemangioma, and some researchers have also started to report the use of
      propranolol for the treatment of infantile hepatic hemangioma in children.And more and more
      clinicians unanimously recommend propranolol as first-line medication for the treatment of
      IHHE.However, there are few solutions to propranolol-resistant IHHE. Sirolimus is a
      serine/threonine kinase which plays a pivotal role in cell mortality, angiogenesis and cell
      growth. Sirolimus can be used in patients with vascular malformations such as hemangiomas.
      Children with vascular malformations received 0.1mg/kg of sirolimus orally every day and
      maintained the blood concentration at 8-15ng/ml. The lesions gradually disappear with time
      going on. Therefore, sirolimus can be used as a second-line medicine for refractory
      hemangioma and vascular malformation.Interestingly, the investigator's previous study which
      is retrospective analysis of 30 patients has indicated the effective rate of propranolol
      alone in treating infantile hepatic endothelial tumor (IHHE) was 57.7%, while that of
      sequential treatment combined with sirolimus was 84.6%.So, this clinical trial is going to
      explore the efficacy of sequential treatment of sirolimus to refractory IHHE patients
      resistant with propranolol,to improve the therapeutic effect,furtherly, reduce the side
      effects of traditional treatment methods (hormones, interferon), as well as the number of
      operations and interventions and to provide a clinical guide for the novel therapeutics model
      of propranolol combined with sirolimus for sequential treatment of infantile hepatic
      hemangioendothelioma.
    

Trial Arms

NameTypeDescriptionInterventions
InterventionExperimentalpatients with propranolol-resistant IHHE are given propranolol combined with sirolimus
  • Sirolimus Oral Product
  • propranolol

Eligibility Criteria

        Inclusion Criteria:

          -  Confirmed as IHHE

          -  Age between 1 month and 36 months

          -  Receiving propranolol for 3 months but the tumor size shrinks <30%

          -  With written informed consent

        Exclusion Criteria:

          -  Confirmed as hepatoblastoma

          -  Have accepted surgical resection

          -  Clinical data missing

          -  Patients with Kasabach-Merritt phenomenon

          -  Receiving propranolol for 3 months and the tumor size shrinks >30%
      
Maximum Eligible Age:36 Months
Minimum Eligible Age:1 Month
Eligible Gender:All
Healthy Volunteers:No

Primary Outcome Measures

Measure:Change of tumor size
Time Frame:1 month
Safety Issue:
Description:measuring the tumor size by ultrasound

Secondary Outcome Measures

Measure:Change of PIVKA-II
Time Frame:1 month
Safety Issue:
Description:Using PIVKA-II assay kit (ARCHITECT I2000SR REFURB, Abbott, America).
Measure:Change of alpha-1 fetoprotein (AFP)
Time Frame:1 month
Safety Issue:
Description:Using the AFP assay kit (ARCHITECT AFP, Abbott, America).

Details

Phase:Phase 4
Primary Purpose:Interventional
Overall Status:Not yet recruiting
Lead Sponsor:Shanghai Children's Medical Center

Trial Keywords

  • propranolol-resistant
  • sirolimus
  • infantile hepatic hemangioendothelioma

Last Updated

May 23, 2020