Clinical Trial: SPT Screening in Irradiated Hereditary Retinoblastoma Survivors

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

Official Title: Multicenter Implementation of MR Imaging Studies in Irradiated Hereditary Retinoblastoma Survivors for Assessment of the Value of Craniofacial MRI Screening for Early Detection of Second Craniofacial

Brief Summary:

Rationale: Hereditary retinoblastoma survivors have an increased risk to develop second primary tumors (SPT) at a later age (with the highest risk in their teens), especially when they have been irradiated for retinoblastoma. The investigators hypothesize that regular screening with magnetic resonance imaging (MRI) could lead to early detection of SPTs leading to improved survival.

Objective: To evaluate the potential benefit of craniofacial MRI screening for early detection subclinical secondary cancers in patients previously irradiated for hereditary retinoblastoma.

Study design: Prospective multicenter non-invasive screening study. The total study duration will be four years of screening plus five years of follow-up.

Study population: Irradiated hereditary retinoblastoma patients 8-18 years old Main study parameters/endpoints: To evaluate the ability of craniofacial MRI for early detection of SPTs, the investigators will determine the sensitivity and specificity of MRI at detecting SPTs in irradiated hereditary retinoblastoma patients.

Nature and extent of the burden and risks associated with participation, benefit and group relatedness: Included patients will undergo yearly craniofacial MRI for a period of 4 years. They will also be asked to fill out a psychological burden assessment form each visit. A potential risk of screening might be associated anticipatory anxiety, but screening also could be reassuring for patients and their parents; the investigators are not sure which will outweigh. False-positive results from MRI screening could lead to unnecessary further diagnostics leading to possible added anxiety and diagnostics (e.g., biopsies). However, this group of patients have a high risk of developing SPTs, with poo

Detailed Summary:
Sponsor: VU University Medical Center

Current Primary Outcome: Diagnostic accuracy of MRI for the detection of second primary tumors in hereditary irradiated retinoblastoma [ Time Frame: 4 years ]

The primary objective of the study is to evaluate the benefit of early detection of craniofacial second primary tumors with MRI in previously irradiated hereditary retinoblastoma survivors. The main outcome measure for assessing the benefit of screening will be the diagnostic accuracy of MRI for detecting craniofacial SPTs: sensitivity (true-positive and false-negative results) and specificity (true-negative and false-positive results).


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • 5-year survival [ Time Frame: 9 years ]
    A secondary objective is to assess the 5-year survival of second primary tumors (SPT) found by screening, SPTs missed by screening, and the non-SPT patients
  • Descriptive statistics [ Time Frame: 4 years ]
    Recording and monitoring location and characteristics (clinical, radiological, histological, treatment) of patients with SPTs: total number of patients included, median follow-up time + range, number of asymptomatic SPTs detected by screening, number of symptomatic SPTs missed by screening, number of SPTs developed after the end of screening, number of SPTs that were totally resected, location of tumor, type of tumor, size of tumor
  • Feasability assessment [ Time Frame: 4 years ]
    Assessment of the feasibility of such this screening program: (a) percentage of patients (or family) willing to participate; (b) compliance of patients and their families to the screening program
  • Assessing the psychosocial burden or benefit [ Time Frame: 4 years ]
    Once a year during the visits, the patient will be asked to fill out the Hospital Anxiety and Depression Scale (HADS).


Original Secondary Outcome: Same as current

Information By: VU University Medical Center

Dates:
Date Received: December 18, 2014
Date Started: October 2014
Date Completion: October 2023
Last Updated: December 29, 2014
Last Verified: December 2014