Long-Term Observation of the Safety and Effectiveness of Enzyme Replacement Therapy in Japanese Patients with Pompe Disease: Results From the Post-marketing Surveillance.

ABSTRACT:

Introduction

Alglucosidase alfa received marketing approval for the treatment of Pompe disease in Japan in 2007. We conducted a post-marketing surveillance study to monitor the long-term safety and efficacy of alglucosidase alfa therapy among Japanese patients with Pompe disease.

Methods

The safety and efficacy outcomes were collected as real-world data for up to 9 years following the initiation of treatment with alglucosidase alfa, without any intervention to treatment strategies. The safety of the drug was assessed in 73 patients in terms of the rate of drug-related adverse events, infusion-associated reactions, and antibody titers. The efficacy was evaluated in 72 patients on the basis of subjective evaluation of their general condition after treatment, pulmonary function

SUBMITTER: Nagura H 

PROVIDER: S-EPMC6858897 | biostudies-literature | 2019 Dec

REPOSITORIES: biostudies-literature