Transcriptomic analyses suggest that mucopolysaccharidosis patients may be less susceptible to COVID‐19

Pierzynowska, Karolina; Gaffke, Lidia; Węgrzyn, Grzegorz

doi:10.1002/1873-3468.13908

Cited by 14 publications

(13 citation statements)

References 33 publications

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“…Almost all patients with infection reported mild, typical COVID-19 symptoms [ 21 ]. Those observations are in line with reports from other health care units worldwide [ 5 , 9 , 22 , 23 , 24 , 25 ]. Only in three cases, the worsening of primary disease symptoms was observed (two patients with NPC and one NPB: psychomotor agitation or deterioration of motor abilities), despite the continuation of symptomatic treatment during infection.…”

Section: Discussionsupporting

confidence: 92%

COVID-19 Pandemic and Patients with Rare Inherited Metabolic Disorders and Rare Autoinflammatory Diseases—Organizational Challenges from the Point of View of Healthcare Providers

Tobór-Świętek

Sykut-Cegielska²,

Bik-Multanowski

et al. 2021

JCM

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COVID-19 pandemic is an organisational challenge for both healthcare providers and patients. People with rare inherited metabolic disorders (IMD) and rare autoinflammatory diseases (AD) are vulnerable patients whose well-being is deeply connected with regular follow-ups. This study aimed to assess how e one year of coronavirus pandemic has impacted the treatment of patients with IMD and AD in Poland. Surveys were distributed to all healthcare providers that coordinate the treatment of IMD and AD patients. Thirty-two responders (55%) answered the survey. They provide care to 1726 patients with IMD/AD, including 246 patients on dedicated treatment. In 35% of units, the regular appointments were disrupted, primarily because of patient infection. In 18 hospitals, remote visits were implemented, but only 66.6% of patients used this form of consultation. In 14/32 hospitals, administration of the therapy was delayed (median: 17.4 days). Forty-four patients suffered from SARS-COV-2 infection, in majority with mild symptoms. However, four adult patients developed complications, and one died following a SARS-COV-2 infection. Although most hospitals managed to maintain regular visits during the pandemic, more comprehensive implementation of telemedicine and switch to oral therapy or home infusions would be a reasonable solution for the current epidemic situation.

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Section: Discussionsupporting

confidence: 92%

COVID-19 Pandemic and Patients with Rare Inherited Metabolic Disorders and Rare Autoinflammatory Diseases—Organizational Challenges from the Point of View of Healthcare Providers

Tobór-Świętek

Sykut-Cegielska²,

Bik-Multanowski

et al. 2021

JCM

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“…Compared to the general Israeli population, the infection rate among LSDs patients is higher (4.4% based on the Israeli Ministry of Health data) but the mortality and morbidity rate is significantly lower. While this might be the result of the relatively small patient cohort, this finding may actually be supported by a study of transcriptomic analyses performed on MPS patients' cell lines by Pierzynowska et al that showed that MPS patients' cells may have lower susceptibility to infection by SARS-CoV-2 due to specific changes in expression of genes coding for proteins involved in interactions with viral proteins [ 15 ]. Another important point is the vast majority of the described Israeli cohort includes younger patients which could partially explain the mild disease, as occurs also in the general pediatric population in Israel.…”

Section: Discussionmentioning

confidence: 88%

The effects of the COVID-19 pandemic on patients with lysosomal storage disorders in Israel

Kristal

Pode‐Shakked

Hazan

et al. 2021

Orphanet J Rare Dis

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Background Severe acute respiratory syndrome coronavirus 2 (SARS CoV-2) is the causative agent of the current COVID-19 pandemic. Lysosomal storage disorders (LSD) comprise of 70 inherited inborn errors of metabolism. Affected individuals suffer from multi-systemic involvement with variable severity and rate of disease progression between different diseases. Some of the LSDs have established treatments, whether parenteral or oral therapies. The full impact of the COVID-19 pandemic together with the lockdown on the wellbeing and medical management of patients with rare diseases, such as LSDs, is widely unknown. Herein, we describe the effects of the COVID-19 pandemic and its associated mandatory home lockdown on patients with LSDs in Israel. Results We present a prospective multi-center questionnaire study including 48 LSD patients from four medical centers in Israel. The study objective was to assess the impact of the COVID-19 pandemic restrictions on individuals with LSDs in Israel, as reported by their caregivers. Secondary objectives were to assess the morbidity from SARS CoV-2 in LSD patients and the impact of changes in mood and behavior on compliance to treatment and to assess the relationship between changes in mood to changes in cognition and behavior. Thirty one of 38 patients (82%) who received any kind of regular treatment did not miss treatments. Among patients receiving enzyme replacement therapy (ERT) in the in-hospital setting, 5 patients (20%) experienced treatment disruptions. Four patients had tested positive for SARS-Cov-2 virus infection by PCR. Seven out of the 48 patients (14%) described mood changes with cognitive and motor deterioration during the home quarantine. Conclusions We observed high rates of treatment adherence and low morbidity through the COVID-19 pandemic in patients with LSDs in Israel. LSDs patients can be a model for patients with complex chronic diseases requiring routine treatments and surveillance during a pandemic or other disruption of daily routine.

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“…Compared to the general Israeli population, the infection rate among LSDs patients is higher (4.4% based on the Israeli Ministry of Health data) but the mortality and morbidity rate is signi cantly lower. This is actually supported by a study of transcriptomic analyses performed on MPS patients' cell lines by Pierzynowska et al that showed that MPS patients' cells may have lower susceptibility to infection by SARS-CoV-2 due to speci c changes in expression of genes coding for proteins involved in interactions with viral proteins 10 . Another important point is the vast majority of the described Israeli cohort includes younger patients which could partially explain the mild disease, as occurs also in the general pediatric population in Israel.…”

Section: Discussionmentioning

confidence: 77%

The Effects of The COVID-19 Pandemic on Patients With Lysosomal Storage Disorders in Israel

Kristal

Pode‐Shakked

Hazan

et al. 2021

Preprint

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Background- Severe acute respiratory syndrome coronavirus 2 (SARS CoV-2) is the causative agent of the current COVID-19 pandemic. Lysosomal storage disorders (LSD) comprise of 70 inherited inborn errors of metabolism. Affected individuals suffer from multi-systemic involvement with variable severity and rate of disease progression between different diseases. Some of the LSDs have established treatments whether parenteral, or oral therapies. The full impact of the COVID-19 pandemic together with the lockdown on the wellbeing and medical management of patients with rare diseases, such as LSDs, is widely unknown. Herein, we describe the effects of the COVID-19 pandemic and its associated mandatory home lockdown on patients with LSDs in Israel. Results- We present a prospective multi-center questionnaire study including 48 LSD patients from four medical centers in Israel. The study objective was to assess the impact of the COVID-19 pandemic restrictions on individuals with LSDs in Israel, as reported by their caregivers. Secondary objectives were to assess the morbidity from SARS CoV-2 in LSD patients and the impact of changes in mood and behavior on compliance to treatment and to assess the relationship between changes in mood to changes in cognition and behavior. Thirty one of 38 patients (82%) who received any kind of regular treatment did not miss treatments. Among patients receiving enzyme replacement therapy (ERT) in the in-hospital setting, 5 patients (20%) experienced treatment disruptions. Four patients had tested positive for SARS-Cov-2 virus infection by PCR. Seven out of the 48 patients (14%) described mood changes with cognitive and motor deterioration during the home quarantine. Conclusions- We observed high rates of treatment adherence and low morbidity through the COVID-19 pandemic in patients with LSDs in Israel. LSDs patients can be a model for patients with complex chronic diseases requiring routine treatments and surveillance during a pandemic or other disruption of daily routine.

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Transcriptomic analyses suggest that mucopolysaccharidosis patients may be less susceptible to COVID‐19

Cited by 14 publications

References 33 publications

COVID-19 Pandemic and Patients with Rare Inherited Metabolic Disorders and Rare Autoinflammatory Diseases—Organizational Challenges from the Point of View of Healthcare Providers

COVID-19 Pandemic and Patients with Rare Inherited Metabolic Disorders and Rare Autoinflammatory Diseases—Organizational Challenges from the Point of View of Healthcare Providers

The effects of the COVID-19 pandemic on patients with lysosomal storage disorders in Israel

The Effects of The COVID-19 Pandemic on Patients With Lysosomal Storage Disorders in Israel

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