Impact of the COVID-19 pandemic on the standard of care for patients with lysosomal storage diseases: A survey of healthcare professionals in the Fabry, Gaucher, and Hunter Outcome Survey registries

Deborah Elstein,Roberto Giugliani,Joseph Muenzer,Jörn Schenk,Ida V.D Schwartz,Christina Anagnostopoulou

doi:10.1016/j.ymgmr.2021.100788

Abstract

The impact of the COVID-19 pandemic on the standards of care of patients with lysosomal storage diseases and the needs of their healthcare providers were explored using a 12-question survey. Overall, 80/91 respondents (88%) indicated that the pandemic had negatively affected standards of care. With increased reliance on telemedicine, the respondents highlighted the need for a personalized approach to care, direct and frequent communication with patients, and greater involvement of patients and caregivers.

Highlights

Lysosomal storage diseases (LSDs), including Fabry disease, Gaucher disease and mucopolysaccharidosis II (MPS II; Hunter syndrome), are rare metabolic diseases characterized by the accumulation of toxic materials within lysosomes due to a deficiency in the activity of the enzymes responsible for their degradation [1]
The associated changes in clinical practice and in the needs of healthcare professionals (HCPs) in this evolving environment, have not been explored. This survey aimed to explore the impact of the COVID-19 pandemic on the standards of care of patients enrolled in the Fabry, Gaucher, and Hunter Outcome Surveys (FOS, Gaucher Outcome Survey (GOS), and HOS, respectively), along with the needs of HCPs treating patients with LSDs
The results highlighted the concerns of HCPs for their patients, the need for personalized medical care, regular and direct communication with patients, and the increased involvement of guardians and caregivers in disease management for the continued de livery of medical care

Summary

Introduction

Lysosomal storage diseases (LSDs), including Fabry disease, Gaucher disease and mucopolysaccharidosis II (MPS II; Hunter syndrome), are rare metabolic diseases characterized by the accumulation of toxic materials within lysosomes due to a deficiency in the activity of the enzymes responsible for their degradation [1]. The associated changes in clinical practice and in the needs of healthcare professionals (HCPs) in this evolving environment, have not been explored This survey aimed to explore the impact of the COVID-19 pandemic on the standards of care of patients enrolled in the Fabry, Gaucher, and Hunter Outcome Surveys (FOS, GOS, and HOS, respectively), along with the needs of HCPs treating patients with LSDs. The role of the FOS, GOS, and HOS registries is to collect long-term, real-world evidence on the safety and efficacy of treatment and on the natural history of Fabry disease, Gaucher disease and MPS II. These global registries offered an opportunity to survey the opinions of a large group of HCPs from across the world and to explore the changing environment of care for patients with complex and rare metabolic diseases, providing a snapshot of the impact of the pandemic on standards of care

Methods

Results

Conclusion