CRIM-negative infantile Pompe disease: characterization of immune responses in patients treated with ERT monotherapy.

ABSTRACT:

Purpose

Enzyme replacement therapy (ERT) with recombinant human acid α-glucosidase (rhGAA) prolongs survival in infantile Pompe disease (IPD). However, the majority of cross-reactive immunologic material (CRIM)-negative (CN) patients have immune responses with significant clinical decline despite continued ERT. We aimed to characterize immune responses in CN patients with IPD receiving ERT monotherapy.

Methods

A chart review identified 20 CN patients with IPD treated with ERT monotherapy for ≥6 months. Patients were stratified by anti-rhGAA antibody titers: high sustained antibody titers (HSAT; ≥51,200) at least twice; low titers (LT; <6,400) throughout treatment; or sustained intermediate titers (SIT; 6,400-25,600).

Results

Despite early initiation of treatment, the

SUBMITTER: Berrier KL 

PROVIDER: S-EPMC4561024 | biostudies-literature | 2015 Nov

REPOSITORIES: biostudies-literature