Durable and sustained immune tolerance to ERT in Pompe disease with entrenched immune responses.

ABSTRACT:

Background

Enzyme replacement therapy (ERT) has prolonged survival and improved clinical outcomes in patients with infantile Pompe disease (IPD), a rapidly progressive neuromuscular disorder. Yet marked interindividual variability in response to ERT, primarily attributable to the development of antibodies to ERT, remains an ongoing challenge. Immune tolerance to ongoing ERT has yet to be described in the setting of an entrenched immune response.

Methods

Three infantile Pompe patients who developed high and sustained rhGAA IgG antibody titers (HSAT) and received a bortezomib-based immune tolerance induction (ITI) regimen were included in the study and were followed longitudinally to monitor the long-term safety and efficacy. A trial to taper the ITI protocol was attempted to

SUBMITTER: Kazi ZB 

PROVIDER: S-EPMC4970756 | biostudies-literature | 2016 Jul

REPOSITORIES: biostudies-literature