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Observational Study
. 2021 Apr 5;34(5):539-545.
doi: 10.1515/jpem-2021-0067. Print 2021 May 26.

Invisible burden of COVID-19: enzyme replacement therapy disruptions

Ayça Burcu Kahraman  1 , Yılmaz Yıldız  1 , Kısmet Çıkı  1 , Halil Tuna Akar  1 , İzzet Erdal  1 , Ali Dursun  1 , Ayşegül Tokatlı  1 , Hatice Serap Sivri  1
Affiliations
Free article
Observational Study

Invisible burden of COVID-19: enzyme replacement therapy disruptions

Ayça Burcu Kahraman et al. J Pediatr Endocrinol Metab. .
Free article

Abstract

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.

Keywords: COVID-19; disruptions; enzyme replacement therapy; lysosomal storage diseases; survey.

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