Clinical trial NCT02329002: SPT Screening in Irradiated Hereditary Retinoblastoma Survivors
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 Primary Tumors and to Enhance Survival
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Retinoblastoma
Keywords: Retinoblastoma
Second primary tumor
Screening
Magnetic resonance imaging
Lead sponsor: Amsterdam UMC, location VUmc
Sponsor class: OTHER
Collaborators: name: ODAS
First posted: 2014-12-31
Enrollment: 400
Sex: ALL
Minimum age (years): 8
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The potential study population will be irradiated hereditary retinoblastoma survivors in the Netherlands and joining centers from other countries. Currently, there is no screening program for these high-risk patients. The age of patients at highest risk of developing craniofacial SPTs is 8-18 years.

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 poor 5-year survival statistics. Early detection and therefore treatment of earlier stage (smaller) tumors, might therefore increase survival of this patient group.


Eligibility criteria: Inclusion Criteria:

* Patients with a history of hereditary retinoblastoma
* Patients that have been treated with external beam radiotherapy for retinoblastoma
* Age of patients at the first MR scan: 8 years or older (≥8 years old), but only as soon as the MR scan can be performed without sedation or general anesthesia, and until and including 18 years of age (≤18 years old).

Exclusion Criteria:

* MRI related exclusion criteria:

  * Claustrophobia
  * Foreign non MR compatible metal objects in the body
  * Foreign incompatible metal objects in or close to the head
  * Exclusion criterium for additional contrast sequences: Allergic reaction to contrast administration in the past

Primary outcomes:
measure: Diagnostic accuracy of MRI for the detection of second primary tumors in hereditary irradiated retinoblastoma
timeFrame: 4 years
Secondary outcomes:
measure: 5-year survival
timeFrame: 9 years
measure: Descriptive statistics
timeFrame: 4 years
measure: Feasability assessment
timeFrame: 4 years
measure: Assessing the psychosocial burden or benefit
timeFrame: 4 years
Other outcomes:
Locations:
city: Amsterdam
country: Netherlands
facility: VU University Medical Center
state: Noord-Holland
Study officials:
affiliation: Amsterdam UMC, location VUmc
name: Jonas A Castelijns, PhD
role: STUDY_CHAIR
References: