Zoledronate in the prevention of Paget's (ZiPP): protocol for a randomised trial of genetic testing and targeted zoledronic acid therapy to prevent <i>SQSTM1</i>-mediated Paget's disease of bone.

ABSTRACT:

Introduction

Paget's disease of bone (PDB) is characterised by increased and disorganised bone remodelling affecting one or more skeletal sites. Complications include bone pain, deformity, deafness and pathological fractures. Mutations in sequestosome-1 (SQSTM1) are strongly associated with the development of PDB. Bisphosphonate therapy can improve bone pain in PDB, but there is no evidence that treatment alters the natural history of PDB or prevents complications. The Zoledronate in the Prevention of Paget's disease trial (ZiPP) will determine if prophylactic therapy with the bisphosphonate zoledronic acid (ZA) can delay or prevent the development of PDB in people who carry SQSTM1 mutations.

Methods and analysis

People with a family history of PDB aged >30 yea

SUBMITTER: Cronin O 

PROVIDER: S-EPMC6731944 | biostudies-literature | 2019 Sep

REPOSITORIES: biostudies-literature