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. 2021 Feb 1;132(2):S84–S85. doi: 10.1016/j.ymgme.2020.12.201

Impact of SARS-CoV-2 pandemic on the first Spanish national domiciliary enzymatic infusion strategy for lysosomal diseases

Jorge Peris a, María José Esteban b, Jaqueline Robert a, Philip Wikman a, José Miguel Seguí a, Ruth Cañizares a, Pedro Esteve a, Paloma Chazarra a, María Luisa Asensio a, Verónica Martínez a, Azucena Sempere a, David Balaz a, Andrea Riaño a, Lourdes Lajara a, Vicente Giner a
PMCID: PMC7849495

In the absence of clear evidence, all experts agree that home administration (HA) of the enzymatic treatment is the best option for patients with lysosomal diseases. This therapeutic strategy avoids the contact with hospitals, but the home visits of healthcare staff could be an added mechanism for infection of this population at-risk for COVID19. We analyzed the impact that the actual SARS-CoV-2 pandemia has meant on the first Spanish national program of enzymes HA for the treatment of Gaucher disease (GD) with velaglucerase α, Fabry disease (FD) with agalsidase α, and Hunter disease (HD) with idursulfase α. This Spanish program began November 2015, 30 patients from 10 hospitals of 5 (of the 17) regions were included (20 FD, 10 GD). In February 2018, 52 patients were included (28 FD, 19 GD, 5 HD) from 19 hospitals of 9 regions. In February 2020, 49 patients were included (25 FD, 17 GD, 7 HD). During these months all programmed infusions were administrated. In June 2020, when the national quarantine ended, 53 patients were in the program (28 FD, 19 GD, 5 HD), and about 14% of the domiciliary infusions not administrated (from March to May). 74% of the suspensions were due to COVID-related reasons: Fear about infection (mainly among FD), and in 3 cases COVID-suggestive symptoms, microbiologically unconfirmed. The first Spanish national program of enzymes HA in lysosomal diseases shows a very slow but continuous implementation in the last years. Although HA has demonstrated to be secure for these patients, the actual pandemic has not accelerated its implementation although most of the regional governments have modified their regulations in favour of this strategy.


Articles from Molecular Genetics and Metabolism are provided here courtesy of Elsevier

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