Invisible burden of COVID-19: enzyme replacement therapy disruptions.
OBJECTIVES
Lysosomal storage diseases (LSD) constitute an important group of metabolic diseases, consisting of approximately 60 disorders. In some types of lysosomal diseases, enzyme replacement therapy (ERT) is administered intravenously in weekly or biweekly doses. Unfortunately, scheduled ERT during COVID-19 was disrupted. We considered the possibility of adverse outcomes caused by the disruption in the treatment of patients with lysosomal storage disorders.


METHODS
During the COVID-19 pandemic, we conducted a questionnaire that was delivered via Internet to assess how this vulnerable patient group was affected by the pandemic in terms of their access to treatment and their disease-related symptoms.


RESULTS
The questionnaire was filled out by 75 patients. There were 35 patients whose treatment dose was missed because of COVID-19. The most common reason for skipping treatment was not wanting to go to the hospital for fear of contracting COVID-19. These 35 patients missed a median of four doses of ERT (range: 1-16 dosages). Twenty-one patients (60%) claimed that they were affected physically by not taking ERT (20 mucopolysaccaridoses, 1 Fabry disease), whereas 14 (40%) did not.


CONCLUSIONS
Interruption of ERT during the COVID-19 pandemic may have significant consequences. It may be beneficial to switch to home treatment or reserve dedicated facilities. With proper planning and management, the treatment disruptions of this particular group can be avoided.
['Kahraman|Ayça Burcu|AB|https://orcid.org/0000-0002-9563-0296', 'Yıldız|Yılmaz|Y|https://orcid.org/0000-0001-9076-1388', 'Çıkı|Kısmet|K|', 'Akar|Halil Tuna|HT|https://orcid.org/0000-0003-1982-8046', 'Erdal|İzzet|İ|', 'Dursun|Ali|A|', 'Tokatlı|Ayşegül|A|', 'Sivri|Hatice Serap|HS|']
D000293:Adolescent D000328:Adult D000086382:COVID-19 D002648:Child D002675:Child, Preschool D017281:Cost of Illness D003430:Cross-Sectional Studies D056947:Enzyme Replacement Therapy D005260:Female D006801:Humans D016464:Lysosomal Storage Diseases D008297:Male D008875:Middle Aged D000086402:SARS-CoV-2 D011795:Surveys and Questionnaires D055815:Young Adult
COVID-19 enzyme replacement therapy disruptions lysosomal storage diseases survey