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Nijmegen, 05 February 2021
Summary of the view of all ERNs on priorities and contra-indications for COVID-19 vaccinations
2021 will be marked by the vaccination programs against SARS-CoV-2. Currently, various Member
States are setting priorities in the vaccination programs for specific groups of patients, including those
with rare diseases. On the other hand, there are some rare disease patients for whom vaccination can
As European Reference Networks (ERNs) are formed by experts in rare diseases, we asked all ERNs to
provide their opinion on the priorities and contraindications for patients with a rare disease within
their network. The results are summarized in this document and were discussed during the internal
ERN-Coordinator group meeting of January 27th
First of all, we advise that the general recommendations of the EMA regarding new medicines need to
be followed. Unfortunately, the recommendations below will not be complete, and only apply to the
adult population. Moreover, these recommendations are based on the expert opinion as evidence is
lacking for the majority of rare diseases. When evidence is available this is mentioned in the statement.
It is very important that evidence is gathered regarding rare disease patients in the vaccination
program, even better will be to set up clinical trials.
This expert opinion-based document with recommendations for adults only, can be no more than a
start with the ultimate goal to have a complete overview of recommendations within the years to
come, and therefore will need very regular updates. Up to date positions will be available on the
websites of many ERNs.
Rare disease patients with priorities for COVID-19 vaccination:
Related to respiratory problems:
• Patients with a rare disease of the respiratory system, such as alpha-1-antitrypsin-deficiency,
pulmonary hypertension, Interstitial lung diseases, especially in those with severe
manifestation of the disease and with impaired pulmonary function.
• Patients with upper airway obstructions that require respiratory support (CPAP, BiPAP,
tracheostoma etc): specifically, Apert, Crouzon and Pfeiffer syndrome, facial dysostosis (e.g.
Treacher Collins, Nager and Miller syndrome), Robin sequence, laryngeal and tracheal cleft and
• Patients with rare neuromuscular diseases with respiratory compromise due to weak
respiratory muscles and/or deformities of the chest wall or upper airways; and patients with
rare bone diseases affecting chest wall abnormalities consequently causing respiratory
Related to Cardiovascular problems:
• Patients with primary arrhythmia syndrome and a history of symptomatic arrhythmias,
including patients with Brugada syndrome.
• Patient with cardiomyopathy (or congenital heart disease) and risk factors (reduced left
ventricular systolic function, heart failure, NYHA III/IV or pulmonary hypertension).
• Patient who carry a (probable) pathogenic variant in the cardiac sodium channel gene SCN5A.
• Patients with collagenopathies which cause cardiovascular complications.
Related to immunodeficiency or cancer:
• Patients with rare cancer within 2 years after diagnosis.
• Patients with treated haematological malignancies, starting by those who received allogeneic
hematopoietic stem cell transplantation (after engraftment) or patients with comorbidities,
and older patients (>65years).
• Patients on immunosuppressive therapy and advanced chronic kidney disease.
• Patients being planned or after solid organ transplantation or chronic allograft dysfunction.
• Patients with disorders of calcium and phosphate complicated with hypocalcaemia and/or
chronic renal disease and/or immune deficiency.
• Patients with APECED/APS1 or auto-immune poly endocrinopathy type 1.
• Patients with auto-immune blistering diseases (AIBD) as they use high dosages of
corticosteroids and immunosuppressive drugs, which highly increase risk and severity of
infection by COVID-19.
• Patients affected with chronic haemolytic anaemia with splenectomy.
Related to endocrine diseases:
• Patients with primary hyperaldosteronism.
• Patients with Cushing’s syndrome: comorbidities (hypertension, diabetes mellitus) with shown
increased risk for severe course of COVID-19.
• Patients with adrenal insufficiency (all kinds of adrenal C14 Addison’s, CAH, SAI): In general we
do not consider patients with adrenal insufficiency being at higher risk for COVID-19 or at a
particular risk for a severe course of COVID-19. But as patients with adrenal insufficiency are
at a higher risk of mortality in case of infections due to the additional risk of adrenal crises, we
agreed that they should be one step up in the priority list for vaccination and get the respective
• In general, patients with rare thyroid disorders should not prioritized. Exceptions to this advice
are patients which are severely debilitated, such as patients with MCT8 deficiency and severe
forms of RTH-alpha.
Other Rare Disease patients:
• Patients with Rare Eye Diseases as they are at higher risk to be contaminated by the COVID-19
virus in daily life, as they cannot apply barrier gestures.
• Patients with advanced liver cirrhosis.
• Patients affected by amino and organic acids-related disorders (AOA).
• Patients affected by disorder of pyruvate metabolism, Krebs cycle defects, mitochondrial
oxidative phosphorylation disorders, disorders of thiamine transport and metabolism (PMMD).
• Patients affected by carbohydrate, fatty acid oxidation and ketone bodies disorders (C-FAO).
• Patients affected by lysosomal storage disorders (LSD).
• Patients affected with haemoglobin disorders as sickle cell disease or thalassaemia major /
intermedia with comorbidities.
• Patients with systemic forms of epidermolysis bullosa and skin fragility diseases.
• Patients with cutaneous Auto immune Bullous diseases (Pemphigus, Pemphigoid) (Joly P et al.
J Am Acad Dermatol (under publication)).
• Women with incontinentia pigmenti are at risk for severe COVID-19 infection, as 25% of them
have high concentrations of autoantibodies directed against type I interferons, causing a more
severe (resuscitating) form of COVID-19 (Bastard et al. Science. 2020 Oct
• Patients with difficult-to-treat epilepsies are at higher COVID-related risks than the general
population, independently of its underlying aetiology. This opinion is based on the (small) case
series on COVID-19 and epilepsy, showing that people with epilepsy appear to have more often
COVID-19 infections and that outcome is worse (Cabezudo-García et al. Neurology. 2020 Sep
8;95(10):e1417-e1425) and that people with epilepsy suffer more frequently from pneumonia
(standardized mortality ratio 6.6; Neligan et al. Brain. 2011 Feb;134(Pt 2):388-95).
Others related to rare disease patients:
• Parents or guardians of children undergoing intensive chemotherapy and / or stem cell
• Parents or guardians of new-borns with a congenital malformation.
• Parents or guardians of children with autoimmune disorders receiving intensive
• Family members of children with congenital or acquired immunodeficiencies.
• Parents or guardians of children who are hospitalised for a long period of time.
Contra-indications for COVID-19 vaccination:
Almost all ERNs indicated that none of rare diseases included within their ERN have a contra-indication
for COVID-19 vaccination. The exceptions are listed below:
• Patients with rare neuromuscular diseases who may have an option with AAV9 treatments
should avoid AAV virus mediated vaccines.
• In general caution should be taken with patients involved in gene therapy trials.
• Some patients participating in clinical trials may have contraindications dependent on the kind
of clinical trial.
• Please note that COVID-19 vaccines neither are tested for the paediatric age group nor are
vaccinations recommended in the paediatric age group undergoing intensive chemotherapy
or allogeneic haematopoietic stem cell transplantation for reasons of lacking or incomplete
We would like to ask you to consider our expert opinion in setting priorities in the vaccination program
of COVID-19. We will be happy to discuss this summary during one of the meetings of the Health
Security Committee. Although we realise that this document is incomplete, we do wish that it can be
Yours sincerely on behalf of all ERN coordinators,
Prof. Nicoline Hoogerbrugge, MD, PhD
Chair of the group of ERN coordinators and Coordinator of ERN GENTURIS
Prof. Luca Sangiorgi
Coordinator of ERN BOND
Prof. Irene Mathijssen
Coordinator of ERN CRANIO
Prof. Alberto Pereira
Coordinator of Endo-ERN
Prof. Alexis Arzimanoglou
Coordinator of ERN EpiCARE
Prof. Franz Schaefer
Coordinator of ERKNet
Prof. Holm Graessner
Coordinator of ERN-RND
Prof. Rene Wijnen
Coordinator of ERNICA
Prof. Thomas Wagner
Coordinator of ERN-LUNG
Prof. Christine Bodemer
Coordinator of ERN-SKIN
Prof. Jean-Yves Blay
Coordinator of ERN EURACAN
Prof. Pierre Fenaux and prof Béatrice Gulbis
Coordinators of ERN-EuroBloodNet
Prof. Teresinha Evangelista
Coordinator of EURO-NMD
Prof. Hélène Dollfus
Coordinator of ERN-EYE
Prof. Arthur Wilde
Coordinator of ERN GUARD-Heart
Prof. Alain Verloes
Coordinator of ERN ITHACA
Prof. Maurizio Scarpa
Coordinator of MetabERN
Prof. Ruth Ladenstein
Coordinator of ERN PaedCan
Prof. Ansgar Lohse
Coordinator of RARE-LIVER
Prof. Marta Mosca
Coordinator of ERN ReCONNET
Prof. Nico Wulffraat
Coordinator of ERN RITA
Prof. Paloma Jara
Coordinator of ERN TRANSPLANT-CHILD
Prof. Guillaume Jondeau
Coordinator of VASCERN
Prof. Wout Feitz
Coordinator of eUROGEN