Impact of COVID-19 pandemic on patients with Fabry disease: An Italian experience

Abstract

We conducted an observational study to assess the impact of COVID-19 emergency on management and outcomes of patients with Fabry disease referring to our Center in Naples, Italy. No patient of the 129 included reported suspected symptoms; 3 isolated themselves in auto-quarantine for flu-like symptoms. All treated patients regularly continued their therapies; 8 missed one infusion: 3 for self-isolation with 2 relatives, and 3 refused to receive nurse at home. All elective procedures were deferred and telemedicine was adopted.

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

“…As might be expected and in line with recent reports [ 3 , [5] , [6] , [7] , [8] , [9] , 11 ], HCPs noted that there had been some interruptions to treatment because of the COVID-19 pandemic; 38.0%, 32.6%, and 15.2% of respondents noted that <1%, 1–10%, or > 10–20% of patients, respectively, had missed infusions. A small proportion of HCPs reported that patients had missed >20–50% or > 50% of infusions (4.3% and 5.4% of respondents, respectively).…”

“…Other factors noted by respondents as free text entries included psychiatric effects, stress and fear, access to vaccination, continued treatment, optimum care, disease surveillance and disease management, and access to school, social, and community services. The concerns noted by HCPs for their patients in this survey appear to align with the results of published patient surveys [ [3] , [4] , [5] , [6] , [7] , [8] , [9] ]. Furthermore, most respondents were concerned that the risks associated with contracting COVID-19 for patients with LSDs may be higher than for the general population; 47.8% of respondents were ‘somewhat concerned’ and 34.8% of respondents were ‘greatly concerned’; however, 17.4% of respondents were ‘not concerned’ that risks may be higher in this patient population.…”

“…Current disease-specific standard of care for the treatment of LSDs includes intravenous enzyme replacement therapy (ERT), substrate reduction therapy, and pharmacological chaperones [ 2 ]. In the context of the coronavirus disease 2019 (COVID-19) pandemic, published patient surveys suggest that COVID-19 has had a negative impact on mental health and access to continued care and treatment among patients with LSDs [ [3] , [4] , [5] , [6] , [7] , [8] , [9] , [10] ]. The associated changes in clinical practice and in the needs of healthcare professionals (HCPs) in this evolving environment, however, have not been explored.…”

The Impact of the COVID-19 Pandemic on the Perception of Health and Treatment-Related Issues among Patients with Phenylketonuria in Poland—The Results of a National Online Survey

“…The results of our study confirm previous findings regarding the need for psychological support to reduce levels of stress and anxiety during a pandemic for patients with rare diseases and their families, as well as for providing remote counselling to the extent that, due to the specificity of the disease, it is possible [27][28][29]. Similar to the findings concerning patients with cystic fibrosis (CF) [29], our patients also reported changes in daily routine during self-isolation; this was mostly unintentional non-adherence to treatment for CF, whereas, in our study, most respondents' self-reported adherence was better, though some reported poor adherence or complete non-adherence to their diet.…”

Gaucher disease in the COVID-19 pandemic environment: The good, the bad and the unknown

“…There was also at least a theoretical concern that patients with Gaucher disease might be especially at risk if early Sars-CoV-2 treatments like hydroxychloroquine, that could disrupt autophagy needed to maintain homeostasis, were administered. In response to this call to action, both patient groups and physician investigators have attempted to educate and to survey the Gaucher patient population to assess the evolving impact of the Sars-CoV-2 disease and pandemic on their care and health [ 3 , 5 , 9 , 10 ]).…”