Incidence of Malignancy in Patients with Lysosomal Storage Disorders
Research type
Research Study
Full title
Incidence of Malignancy in Patients with Lysosomal Storage Disorders and their Relatives
IRAS ID
185067
Contact name
Derralynn Hughes
Contact email
Sponsor organisation
Royal Free London
Duration of Study in the UK
20 years, 0 months, 1 days
Research summary
This study aims to explore the incidence of malignancy in individuals with lysosomal storage disorders (LSDs) and their relatives. LSDs are a heterogeneous group of over 50 rare, inherited conditions caused by specific defects in the lysosomal system, the major recycling network in our cells. The most common LSDs are Fabry disease and Gaucher disease. There is some evidence that patients with Gaucher disease have an increased incidence of cancer but very little research has been done to investigate cancer rates in patients with other LSDs.
Participants will be recruited from LSD centres in the UK and will initially be asked to complete a questionnaire which gathers data on background information, personal history of malignancy and family history of malignancy. They will then receive a very short questionnaire each year to collect prospective data on cancer incidence over the next 20 years.
REC name
North East - Tyne & Wear South Research Ethics Committee
REC reference
15/NE/0421
Date of REC Opinion
14 Dec 2015
REC opinion
Favourable Opinion