Retrospective Case Series of Trans-scleral Cryotherapy for Retinal Hemangioblastoma

Background:

Retinal hemangioblastoma (RH) is a tumor. It grows from the retina in the eye. It can threaten a person s vision. Trans-scleral cryotherapy is used to destroy the tumors and minimize the long-term risks of vision loss. RH is a rare condition, often occurring in people with von Hippel-Lindau disease. There are no clinical trials to study how well the treatment works. Researchers want to study the medical records of people with RH who were treated at the NIH eye clinic to learn more.

Objective:

To analyze clinical data collected over a 20-year span to study consecutive cases of RH managed with trans-scleral cryotherapy at the NIH.

Eligibility:

People who took part in NIH natural history protocols for which cryotherapy of RH was performed as a standard care measure.

Design:

Researchers will collect and study data from participants medical charts. Participants will not be contacted because no new data is needed. Researchers were granted a waiver of informed consent for use of these medical records. To protect patient privacy, participants will be assigned an ID number. Their data will be entered into a spreadsheet in a coded fashion. The key to this code will be kept in a secure file. No patient identifying information will be used in the analysis or the publication....

Study Overview

• Status: Active, not recruiting
• Conditions: Von Hippel-Lindau Disease; Retinal Hemangioblastoma

Detailed Description

Study Description: This study is a retrospective review of medical records in the NIH eye clinic. To our knowledge, there has been no report in the current era dedicated to a description of trans-scleral cryotherapy for retinal hemangioblastomas despite its routine use in this setting. This study aims to explore features and outcomes of these cases to better inform best practices.

Objectives: The objective of this study is to perform a retrospective analysis of consecutive cases of retinal hemangioblastoma managed with trans-scleral cryotherapy at the NIH (single center retrospective case series).

• Study Type: Observational
• Enrollment (Actual): 25

Contacts and Locations

Study Locations

• United States
  • Maryland
    • Bethesda, Maryland, United States, 20892
      • National Eye Institute (NEI)

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Data (medical history and images) was collected under NEI and non-NEI natural history protocols allowing administration of standard care measures to participants.

Description

• INCLUSION CRITERIA:

  1. Participants were enrolled in NEI and non-NEI natural history protocols, but in all cases, cryotherapy was administered as a standard care measure (not as an investigative treatment).
  2. Patients were identified by searching the NIH eye clinic EMR for all of those manifesting one or more retinal hemangioblastomas (with or without associated von Hippel-Lindau disease) treated with cryotherapy.

EXCLUSION CRITERIA:

None

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Other

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
Participants; Participants with retinal hemangioblastoma (RH) managed with trans-scleral cryotherapy at the NIH.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To perform a retrospective analysis of consecutive cases of retinal hemangioblastoma (RH) managed with trans-scleral cryotherapy at the NIH	Some key measures of interest will include treatment outcomes (rate of control of tumors; visual function measures; post-treatment lesion characteristics; sequelae and complications) and association of outcomes with eye and lesion characteristics.	ongoing

Collaborators and Investigators

• Sponsor: National Eye Institute (NEI)
• Investigators: Principal Investigator: Emily Y Chew, M.D., National Eye Institute (NEI)

Study record dates

Study Major Dates

• Study Start (Actual): July 2, 2020
• Primary Completion (Estimated): June 30, 2024
• Study Completion (Estimated): June 30, 2024

Study Registration Dates

• First Submitted: July 3, 2020
• First Submitted That Met QC Criteria: July 3, 2020
• First Posted (Actual): July 7, 2020

Study Record Updates

• Last Update Posted (Estimated): February 13, 2024
• Last Update Submitted That Met QC Criteria: February 10, 2024
• Last Verified: February 9, 2024

More Information

Terms related to this study

• Keywords: Natural History; Vision Loss; Vascular Tumor
• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Nervous System Diseases; Neoplasms by Histologic Type; Neoplasms; Congenital Abnormalities; Genetic Diseases, Inborn; Hemangioma; Neoplasms, Vascular Tissue; Abnormalities, Multiple; Neurocutaneous Syndromes; Ciliopathies; Angiomatosis; Hemangioma, Capillary; Von Hippel-Lindau Disease; Hemangioblastoma
• Other Study ID Numbers: 999920129; 20-EI-N129

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No