Clinical Trial: Identification of Biomarkers for Patients With Vascular Anomalies

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational

Official Title: Identification of Biomarkers for Patients With Vascular Anomalies

Brief Summary: The study will use participants tissue previously collected and stored in a tissue bank maintained by the Department of Hematology/Oncology as well as tissue banked in the future from participants undergoing prescribed surgical resection of vascular anomalies of interest proposed in this study. The principle investigator and study staff will also obtain blood from these participants.

Detailed Summary: While vascular anomalies are rare diseases, they can be life-threatening and devastating to affected children and their families. Advances in diagnosis, monitoring and therapies will be significantly improved if non-invasive biomarkers that are sensitive and specific can be identified. Identification of VEGF-D as a biomarker in LAM (Lymphangiomyomatosis) patients is an excellent example of how a blood biomarker can impact diagnosis and treatment. Biomarkers may also give insights into disease pathogenesis, as often they are released by cells that contribute to the disease process. The studies in this protocol will characterize and define biomarkers to aid in the diagnosis and treatment of patients with vascular anomalies. The differential diagnosis of some of these entities can be challenging, especially GLA versus KLA and KLA versus KHE. Obtaining a tissue biopsy to help in diagnosis can actually worsen the disease and so identification of specific blood biomarkers is highly desirable. Studies will measure angiogenic factors in serum and plasma samples at baseline and on therapy.
Sponsor: Children's Hospital Medical Center, Cincinnati

Current Primary Outcome:

  • Correlation of Biomarkers with Differential Diagnosis [ Time Frame: An Average of Every 2 Years ]
  • Correlation of Biomarkers with Disease Severity [ Time Frame: An Average of Every 2 Years ]
  • Correlation of Biomarkers with Response to Therapies [ Time Frame: An Average of Every 2 Years ]


Original Primary Outcome: Same as current

Current Secondary Outcome:

Original Secondary Outcome:

Information By: Children's Hospital Medical Center, Cincinnati

Dates:
Date Received: December 15, 2016
Date Started: April 2015
Date Completion:
Last Updated: April 26, 2017
Last Verified: April 2017