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

Abstract

Background Continuation of standard management of Gaucher disease (GD) has been challenging during the COVID-19 pandemic, resulting in infrequent/missed infusions and follow-up appointments. Little data are available on the consequences of these changes and on the SARS-CoV-2 vaccinations in German GD patients.

Methods A survey with 22 questions about GD management during the pandemic was sent to 19 German Gaucher centres. It was answered by 11/19 centres caring for 257 GD patients (almost ¾ of the German GD population); 245 patients had type 1 and 12 had type 3 GD; 240 were ≥ 18 years old.

Results Monitoring intervals were prolonged in 8/11 centres from a median of 9 to 12 months. Enzyme replacement therapy (ERT) was changed to home ERT in 4 patients and substituted by oral substrate reduction therapy (SRT) in 6 patients. From March 2020 to October 2021, no serious complications of GD were documented. Only 4 SARS-CoV-2 infections were reported (1.6%). Two infections were asymptomatic and two mild; all occurred in adult type 1, non-splenectomized patients on ERT. Vaccination rate in adult GD was 79.5% (95.3% mRNA vaccines). Serious vaccination complications were not reported.

Conclusions The COVID-19 pandemic has lowered the threshold for switching from practice- or hospital-based ERT to home therapy or to SRT. No major GD complication was documented during the pandemic. Infection rate with SARS-CoV-2 in GD may rather be lower than expected, and its severity is mild. Vaccination rates are high in GD patients and vaccination was well tolerated.

Zusammenfassung

Hintergrund Kontrolle und Therapie der Gaucher-Krankheit („Gaucher disease“ [GD]) während der COVID-19-Pandemie waren eine Herausforderung, die mit Veränderungen der Infusions- und Kontrollintervalle einherging. Über die Art dieser Veränderungen, Impfstatus und COVID-19-Morbidität bei Gaucher-Patienten im deutschen Sprachraum liegen kaum Daten vor.

Methode Eine Umfrage mit 22 Fragen zum GD-Management während der Pandemie wurde an 19 deutsche Gaucher-Zentren verschickt. Sie wurde durch 11/19 Zentren beantwortet, die 257 GD-Patienten (etwa ¾ der deutschen GD-Population) betreuen; 245 Patienten hatten GD Typ 1 und 12 Typ 3; 240 Patienten waren ≥ 18 Jahre alt.

Ergebnisse Die Überwachungsintervalle wurden in 8/11 Zentren von einem Median von 9 auf 12 Monate verlängert. Die Enzymersatztherapie (ERT) wurde bei 4 Patienten auf Heiminfusionstherapie umgestellt und bei 6 Patienten durch eine orale Substratreduktionstherapie (SRT) ersetzt. Von März 2020-Oktober 2021 wurden keine schwerwiegenden Komplikationen dokumentiert. Es wurden 4 SARS CoV-2-Infektionen gemeldet (1,6 %). 2 Infektionen verliefen asymptomatisch, 2 wiesen einen milden Verlauf auf; alle traten bei erwachsenen, nicht splenektomierten Patienten vom Typ 1 auf, die mittels ERT behandelt wurden. Die Durchimpfungsrate bei erwachsenen GD-Patienten betrug 79,5 % (95,3 % mRNA-Impfstoffe). Schwerwiegende Impfkomplikationen wurden nicht gemeldet.

Schlussfolgerung Die COVID-19-Pandemie hat die Schwelle für die Umstellung von einer parenteralen praxis- oder krankenhausbasierten ERT auf eine Heimtherapie oder auf eine orale SRT gesenkt. Während der Pandemie wurden keine schwerwiegenden primär GD-assoziierten Komplikationen dokumentiert. Die Infektionsrate mit SARS-CoV 2 bei GD ist niedriger als erwartet und der Schweregrad ist gering. Die Impfraten sind bei GD-Patienten hoch und die Impfungen wurden gut vertragen.

Schlüsselwörter

Morbus Gaucher - COVID-19 - Angeborene Stoffwechselstörung

Keywords

Morbus Gaucher - COVID-19 - Inborn Error of metabolism

Publication History

Received: 22 December 2021

Accepted after revision: 04 April 2022

Article published online:
10 January 2023

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