Experts’ views on COVID‐19 vaccination and the impact of the pandemic on patients with Gaucher disease

Abstract

The current outbreak of the novel severe acute respiratory syndrome coronavirus-2 (SARS-CoV-2), causing coronavirus disease 2019 , has become a worldwide pandemic with high morbidity and mortality in individuals with chronic disorders. 1 The pandemic introduced many unanticipated challenges for patients with chronic and rare diseases, such as Gaucher disease (GD). 2 GD is the most common inborn error of metabolism, caused by biallelic glucosylceramidase beta (GBA) variants and affecting the recycling of cellular glycolipids; GD manifests as hepatosplenomegaly, thrombocytopenia, anaemia and bone disease. 3 We performed a cross-sectional study using an online survey regarding attitudes of GD experts towards COVID-19 vaccination for patients with GD, and the impact of the pandemic on their patients. The full methods and results are available in the Supplementary Data.A total of 19 GD experts from 10 countries, collectively treating 1417 patients with GD, responded to the survey, revealing overall support of COVID-19 vaccination for patients with GD. This support is mainly explained by the concern that the pathophysiology of GD may put the patients at higher risk of severe COVID-19, coupled with the absence of contraindications for vaccination in this population, which is consistent with professional guidelines. [4][5][6][7] Specifically, mRNA-based vaccines were favoured by GD experts compared to adenovirus-based vaccines due to their overall efficacy, tolerance and safety profiles, as appears from clinical trials and worldwide experience. 8,9 Concerns regarding the AAV-vectored vaccines may be underestimated amongst our cohort, as the questionnaire was distributed when data on only one such vaccine was available (AZD1222, Oxford-AstraZeneca), and some responses were prior to the regulatory authorisation, thus lacking knowledge on its side-effects.Our present study revealed that of the 82 patients with GD who contracted COVID-19 (5Á8% of the entire cohort), 93Á9% were either asymptomatic or mildly affected, while two had severe/critical infection. It is also possible that these numbers are an underestimation, as asymptomatic or mildly symptomatic patients may have not been tested for COVID-19 or may have not reported on contracting COVID-19 to their GD clinic. To date, four reports have been published on COVID-19 infection in patients with GD, all reporting of asymptomatic/mild infections, and only one of severe disease in a patient with multiple comorbidities. [10][11][12][13] This makes

Management, vaccination status and COVID-19 morbidity of patients with Gaucher disease in Germany during the COVID-19 pandemic

“…In accordance with the present German data international experts recommended or strongly recommended COVID-19 vaccination for GD patients without vaccine contraindications; their recommendation was strong for mRNA vaccines in 90 %, but for vector vaccines only in 42 % [18]. The reasons GD experts gave against vaccination with vector vaccines included confusing data from clinical trials, suspension of such vaccines in some European countries, concerns about thrombotic events, and concerns of overall effectiveness [18]. Furthermore, some experts were concerned about interactions of vector-based vaccinations with future genetic therapies.…”

“…There were no hospitalizations, COVID-related therapies, or deaths [9]. A recent multicentre international survey of 19 GD experts treating a total of 1.417 GD patients reported 82 SARS-CoV-2 infections in GD type 1 patients (5.7 %); 77 of the 85 patients (93.9 %) had no or only mild symptoms [18].…”

“…It is difficult to compare the incidence of SARS-CoV-2 infections for GD patients with the incidence expected for a control population because the testing procedures between various surveys, studies, and registries were different, and the incidence of adequate control populations has not been given in the corresponding studies. A large recent international multicentre survey showed that 5.7 % of GD patients had a SARS-CoV-2 infection between March 2020 and March 2021 (18) while the infection rate in the general population was higher in almost all participating regions and countries. All evidence including the present data from Germany suggests that GD patients at least do not have an increased risk to get a SARS-CoV-2 infection.…”

“…It was also reported that a significant proportion of patients switched from hospital-or office-based ERT to home ERT and some also from intravenous ERT to oral SRT [5]. In a recent multicentre international survey, treatment initiation during the pandemic consisted of SRT in 43 % of patients, and 59 % of patients who changed their ERT regimen switched to SRT [18]. In the present survey, switch from ERT to SRT was somewhat more frequent than the switch from hospital-of office-based ERT to home ERT while both treatment changes were relatively rare when compared to previous reports.…”

COVID-19 and Vaccination Status in Lysosomal Storage Diseases: A Single-Center Experience

“… 24 However, severe COVID‐19 with Gaucher disease has been reported rarely in the literature. 25 - 27 Hamiel et al 28 evaluated 1417 Gaucher cases from 10 countries. Eighty-two patients were infected with COVID-19; of the patients who had the infection, 83% survived mild, 11% were asymptomatic, and 2% as critical illnesses.…”