Zlamy M., et al. 20222020 Zlamy M, Zöggeler T, Bachmann M, Schirmer M, Lechner C, Michel M, et al. Immunological memory and affinity maturation after vaccination in patients with propionic acidemia. Front Immunol. 2022;13:1–9.
|
Cross-sectional |
Austria |
Propionic acidemia |
10 patients
10 controls
|
MMR
Diphtheria and tetanus
|
Yes |
No |
Yes |
Sirrs S., et al. 2022.3939 Sirrs S. Clinical guidance on COVID-19 vaccines for people with metabolically unstable inborn errors of metabolism background and context. British Columbia, Ministry of Health, Centre for Desease Control; 2022.
|
Guideline |
Canada |
All IEM |
NA |
COVID-19 |
Yes |
Yes |
No |
Zubarioglu T., et al. 20223333 Zubarioglu T, Oral-Cebeci S, Hopurcuoglu D, Uygur E, Ahmadzada S, Uzunyayla-Inci G, et al. Safety of COVID -19 vaccines in children with inborn errors of metabolism in terms of developing metabolic decompensation. J Paediatr Child Health. 2022. https://doi.org/10.1111/jpc.16231. https://doi.org/10.1111/jpc.16231....
|
Retrospective cohort |
Turkey |
Fatty acid disorder/ GSD
LPIN1 deficiency / GA-1/ Propionic acidemia/ Isovaleric acidemia/ Urea cycle disorder/ MSUD and PKU
|
18 patients |
COVID-19 Pfizer and CoronaVac |
Yes |
Yes |
No |
Zaffina S., et al. 2022.6464 Zaffina S, E Piano Mortari, RR di Prinzio, Cappa M, Novelli A, Agolini E, et al. Case report: precision COVID-19 immunization strategy to overcome individual fragility: a case of generalized lipodystrophy type 4. Front Immunol. 2022;13:1–6.
|
Case Report |
Italy |
Generalized LipodystrophyType 4 |
1 patient
Health care workers as controls
|
COVID-19 Pfizer and Janssen |
Yes |
No |
Yes |
Ismailova G, et al. 20223737 Ismailova G, Mackenbach MJ, van den Hout JMP, van der Ploeg AT, Brusse E, Wagenmakers MAEM. Mild disease course of SARS-CoV-2 infections and mild side effects of vaccination in Pompe disease: a cohort description. Orphanet J Rare Dis. 2022;17:102–9.
|
Cross-sectional |
Netherlands |
Glycogen Storage disease type 1 |
169 patients |
COVID-19, Pfizer, Moderna and AstraZeneca |
Yes |
Yes |
No |
Izquierdo-Garcia E., et al. 20225151 Izquierdo-Garcia E, Alvaro Alonso EA, Montero Pastor B, Such Díaz A, Escobar Rodríguez I. Reply letter to the editor concerning the article ‘Safety of Sars-Cov-2 vaccines administration for adult patients with hereditary fructose intolerance. Hum Vaccin Immunother. 2022;18:2051414.
|
Commentary |
Spain |
Hereditary Fructose Intolerance |
NA |
COVID-19 |
Yes |
No |
No |
Hady-Cohen R., et al. 20211717 Hady-Cohen R, Dragoumi P, Barca D, Plecko B, Lerman-Sagie T, Zafeiriou D. Safety and recommendations for vaccinations of children with inborn errors of metabolism. Eur J Paediatr Neurol. 2021;35:93–9.
|
Review |
Greece |
All IEM |
NA |
Vaccines in schedule |
Yes |
Yes |
Yes |
Yilmazbas P., et al. 20211919 Yılmazbaş P, Çakar NE. Evaluation of vaccination status among children with inborn errors of metabolism. Med J Bakırkoy. 2021;17:11–7.
|
Cross-sectional |
Turkey |
All IEM |
99 patients |
Vaccines in schedule |
Yes |
Yes |
No |
Saborido-Fiaño R., et al. 20214141 Saborido-Fiaño R, Martinón-Torres N, Crujeiras-Martinez V, Couce ML, Leis R. Letter to the editor concerning the article ‘Safety of vaccines administration in hereditary fructose intolerance. Hum Vaccin Immunother. 2021;17: 2593–4.
|
Case Report |
Spain |
Hereditary Fructose Intolerance |
1 patient |
Rotavirus |
Yes |
Yes |
No |
Kfouri R. A., et al. 20215252 de Ávila KR, Aranda CS, Levi M, Solé D, Raskin S, Petraglia TC, et al. Imunizações em pacientes com doenças raras – Posicionamento conjunto da Sociedade Brasileira de Imunizações (SBIm), Associação Brasileira de Alergia e Imunologia (ASBAI) e Sociedade Brasileira de Pediatria (SBP). Arq Asma Alerg Imunol. 2021;5:36–50.
|
Review |
Brazil |
All IEM |
NA |
Vaccines in schedule |
Yes |
No |
No |
Tan A., et al. 20212424 Tan A, Stepien KM, Thej S, Narayana K. Carnitine palmitoyltransferase II deficiency and post-COVID vaccination rhabdomyolysis. QJM [Internet]. 2021;114:596–7.
|
Case Report |
UK |
Carnitine palmitoyl transferase II |
1 patient |
COVID-19 AstraZeneca |
Yes |
Yes |
No |
Cheung B., et al. 20212525 Cheung B, Hwang J, Stolarczyk A, Mahlof E, Block R. Case study of hypertriglyceridemia from COVID-19 Pfizer-BioNTech vaccination in a patient with familial hypercholesteremia. Eur Rev Med Pharmacol Sci. 2021;25:5525–8.
|
Case Report |
USA |
Heterozygous familial hypercholesterolemia (HeFH) |
1 patient |
COVID-19 Pfizer |
Yes |
Yes |
No |
Lin JJ., et al. 20212626 Jing LJ, Hui LX, Xia L, Ling FY, Hong XX, Hua LS. A niemann-pick C1 disease child with BCG-itis: a case report and analysis. BMC Pediatr. 2021;21:218–23.
|
Case Report |
China |
Niemann-pick C1 |
1 patient |
BCG |
Yes |
Yes |
No |
Urru SAM., et al. 20215050 Urru SAM, Maines E, Campomori A, Soffiati M. Safety of Sars-Cov-2 vaccines administration for adult patients with hereditary fructose intolerance. Hum Vaccin Immunother. 2021;17: 4112–4.
|
Commentary |
Italy |
Hereditary Fructose Intolerance |
NA |
COVID-19 |
Yes |
Yes |
No |
Saborido-Fiaño R., et al.20214949 Saborido-Fiaño R, Martinón-Torres N, Crujeiras-Martinez V, Couce ML, Leis R. Reply letter to “safety of SARS-Cov-2 vaccines administration for adult patients with hereditary fructose intolerance. Hum Vaccin Immunother. 2021;17:4115–6.
|
Commentary |
Spain |
Hereditary Fructose Intolerance |
NA |
COVID-19 Pfizer and Moderna |
Yes |
No |
No |
Hamiel U., et al. 20216060 Hamiel U, Kurolap A, Cohen IJ, Ruhrman-Shahar N, Hershkovitz T, Niederau C, et al. Experts’ views on COVID-19 vaccination and the impact of the pandemic on patients with Gaucher disease. Br J Haematol. 2021;195:e135–7.
|
Cross-sectional |
Israel |
Gaucher Disease |
19 experts |
COVID-19 |
Yes |
No |
No |
Mao S., et al. 20202727 Mao S, Yang L, Yin X, Yang J, Huang X. Ketoacidotic crisis after vaccination in a girl with beta-ketothiolase deficiency: a case report. Transl Pediatr. 2021;10:459–63.
|
Case Report |
China |
Mitochondrial acetoacetyl-CoA thiolase (T2) |
1 patient |
Inactivated Japanese Encephalitis |
Yes |
Yes |
No |
Fois A., et al. 20206565 Fois A, Boucher-Lafleur AM, Legault JT, Renaud C, Morin C, des Rosiers C, et al. Humoral responses to the measles, mumps and rubella vaccine are impaired in Leigh Syndrome French Canadian patients. PLoS ONE. 2020;15:1–13.
|
Cross-sectional |
Canada |
Leigh Syndrome |
8 patients
8 controls
|
MMR |
Yes |
Yes |
Yes |
Han B., et al.20202828 Han B, Nie W, Sun M, Liu Y, Cao Z. Clinical presentation, molecular analysis and follow-up of patients with mut methylmalonic acidemia in Shandong province. China. Pediatr Neonatology. 2020;61:148–54.
|
Cohort |
China |
Methylmalonic acidemia |
20 patients |
Influenza |
No |
Yes |
No |
Maiorana A., et al. 20204646 Maiorana A, Sabia A, Corsetti T, Dionisi-Vici C. Safety of vaccines administration in hereditary fructose intolerance. Orphanet J Rare Dis. 2020;15:274–81.
|
Review |
Italy |
Hereditary Fructose Intolerance |
NA |
Vaccines in schedule |
Yes |
No |
No |
Naur KM., et al. 20201818 Naur O, Anggraini A, Indraswari B, Wandita S, Wibowo T, Haksari E. Immunisation issues in patient with mucopolysaccharidosis: a case report. Med J Malaysa. 2020;75:51–2.
|
Case Report |
Indonesia |
Mucopolysaccharidosis |
1 patient |
Vaccines in schedule |
Yes |
Yes |
No |
Kruk SK., et al.20196666 Kruk SK, Pacheco SE, Koenig MK, Bergerson JRE, Gordon-Lipkin E, McGuire PJ. Vulnerability of pediatric patients with mitochondrial disease to vaccine-preventable diseases. J Allergy Clin Immunol Pract. 2019;7:2415–8.
|
Cross-sectional |
USA |
Mitochondrial diseases |
26 patients |
Vaccines in schedule |
Yes |
Yes |
Yes |
Chang IJ., et al. 20186363 Chang IJ, He M, Lam CT. Congenital disorders of glycosylation. Ann Transl Med. 2018;6:477.
|
Review |
USA |
Congenital Disorders of glycosylation |
NA |
Vaccines in schedule |
Yes |
No |
No |
Mahajan V., et al. 20182929 Mahajan V, Gupta R. AEFI surveillance – the learning curve continues. Indian Pediatr. 2018;55:707.
|
Case Report |
India |
Glutaric Aciduria type 1 |
1 patient |
Pentavalent |
No |
Yes |
No |
Williams RE., et al. 20175858 Williams RE, Adams HR, Blohm M, Cohen-Pfeffer JL, de los Reyes E, Denecke J, et al. Management Strategies for CLN2 disease. Pediatr Neurol. 2017;69:102–12.
|
Review |
UK |
CLN2 |
NA |
Vaccines in schedule |
Yes |
No |
No |
Parikh S., et al. 20173838 Parikh S, Goldstein A, Karaa A, Koenig MK, Anselm I, Brunel-Guitton C, et al. Patient care standards for primary mitochondrial disease: a consensus statement from the mitochondrial medicine society. Genet Med. 2017;19:1–18.
|
Guidelines |
NA |
Mitochondrial diseases |
NA |
Vaccines in schedule |
Yes |
Yes |
No |
Van Wegberg AMJ., et al.20175353 van Wegberg AMJ, MacDonald A, AhringK, Bélanger-Quintana A, Blau N, Bosch AM, et al. The complete European guidelines on phenylketonuria: diagnosis and treatment. Orphanet J Rare Dis. 2017;12:162–218.
|
Guidelines |
Netherlands |
PKU |
NA |
Vaccines in schedule |
Yes |
No |
No |
Cerutti M., et al. 20151616 Cerutti M, de Lonlay P, Menni F, Parini R, Principi N, Esposito S. Vaccination coverage of patients with inborn errors of metabolism and the attitudes of their parents towards vaccines. Vaccine. 2015;33:6520–4.
|
Cross-sectional |
France |
All IEM |
128 patients
128 controls
|
Vaccines in schedule |
Yes |
Yes |
No |
Kishnan PS., et al. 20145656 Kishnani PS, Austin SL, Abdenur JE, Arn P, Bali DS, Boney A, et al. Diagnosis and management of glycogen storage disease type I: a practice guideline of the American college of medical genetics and genomics. Genet Med. 2014;16:1–29.
|
Guideline |
USA |
Glycogen Storage Disease type 1 |
NA |
Vaccines in schedule |
Yes |
No |
No |
Esposito S., et al.20133535 Esposito S, Salvini F, Menni F, Scala A, Salvatici E, Manzoni F, et al. Preliminary data on immunogenicity, safety and tolerability of trivalent inactivated influenza vaccine in children with inborn errors of metabolism at risk of decompensation. Vaccine. 2013;31:5149–51.
|
Clinical Trial |
Italy |
IEM at high risk for decompensation |
20 patients
20 controls
|
Influenza |
Yes |
Yes |
Yes |
Sutton VR., et al.20125555 Sutton VR, Chapman KA, Gropman AL, MacLeod E, Stagni K, SummarML, et al. Chronic management and health supervision of individuals with propionic acidemia. Mol Genet Metabol. 2012;105:26–33.
|
Guideline |
USA |
Propionic aciduria |
NA |
Vaccines in schedule |
Yes |
No |
No |
Menni F., et al. 20123232 Menni F, Chiarelli G, Sabatini C, Principi N, Esposito S. Vaccination in children with inborn errors of metabolism. Vaccine. 2012;30:7161–4.
|
Review |
Italy |
All IEM |
NA |
Vaccines in schedule |
Yes |
Yes |
No |
Klein NP., et al. 20112121 Klein NP, Aukes L, Lee J, Fireman B, Shapira SK, Slade B, et al. Evaluation of immunization rates and safety among children with inborn errors of metabolism. Pediatrics. 2011;127: e1139–46.
|
Cross-sectional |
USA |
All IEM |
77 patients
1540 controls
|
DTP, IPV, MMR, Hep B, Hib |
Yes |
Yes |
No |
Morgan TM., et al. 20113434 Morgan TM, Schlegel C, Edwards KM, Welch-Burke T, Zhu Y, Sparks R, et al. Vaccines are not associated with metabolic events in children with urea cycle disorders. Pediatrics. 2011;127:e1147–53.
|
Cross-sectional |
USA |
Urea Cycle Disorders |
112 patients |
Vaccines in schedule |
Yes |
Yes |
No |
Kishnani PS., et al. 20105757 Kishnani PS, Austin SL, Arn P, Bali DS, Boney A, Case LE, et al. Glycogen Storage Disease Type III diagnosis and management guidelines. Genet Med. 2010;12:446–63.
|
Guideline |
USA |
Glycogen Storage Disease type 3 |
NA |
Vaccines in schedule |
Yes |
No |
No |
Varghese M., et al. 20094040 Varghese M, Cafferkey M, O’Regan M, Monavari AA, Treacy EP. Should children with inherited metabolic disorders receive varicella vaccination? Arch Dis Chid. 2011;96:99–100.
|
Cross-sectional |
Ireland |
IEM at high risk for decompensation |
122 patients |
Varicella |
Yes |
No |
No |
Wilson K., et al. 20092222 Wilson K, Potter B, Manuel D, Keelan J, Chakraborty P. Revisiting the possibility of serious adverse events from the whole cell pertussis vaccine: were metabolically vulnerable children at risk? Med Hypotheses. 2010;74:150–4.
|
Review |
Canada |
Fatty acid disorders |
NA |
DTwP |
Yes |
No |
No |
Barshop BA., et al. 20083636 Barshop BA, SummarML. Attitudes regarding vaccination among practitioners of clinical biochemical genetics. Mol Genet Metabol. 2008;95:1–2.
|
Cross-sectional |
USA |
All IEM |
NA |
Vaccines in schedule |
Yes |
No |
No |
Brady MT., et al. 20062323 Brady MT. Immunization recommendations for children with metabolic disorders: more data would help. Pediatrics. 2006;118:810–3.
|
Commentary |
USA |
All IEM |
NA |
Vaccines in schedule |
Yes |
Yes |
No |
Kingsley JD., et al. 20065454 Kingsley JD, Varman M, Chatterjee A, Kingsley RA, Roth KS. Immunizations for patients with metabolic disorders. Pediatrics. 2006;118:e460–70.
|
Review |
USA |
All IEM |
NA |
Vaccines in schedule |
Yes |
No |
No |
Yang Y., et al. 20063030 YangY, Sujan S, Sun F, Zhang Y, Jiang Y, Song J, et al. Acute metabolic crisis induced by vaccination in seven Chinese patients. Pediatr Neurol. 2006;35:114–8.
|
Case Series |
China |
Methylmalonic aciduria Glutaric aciduria 1 Leigh disease |
5 patients |
DPT, Hep B, Measles |
Yes |
Yes |
No |
Blank C., et al. 20066767 Blank C, Smith LA, Hammer DA, Fehrenbach M, Delisser HM, Perez E, et al. Recurrent infections and immunological dysfunction in congenital disorder of glycosylation Ia (CDG Ia). J Inherit Metabolic Dis. 2006;29:592.
|
Case Series |
USA |
Congenital disorder of glycosylation 1a |
2 patients |
DPT, Hep B, MMR, Hib, Pneumococcal |
Yes |
No |
Yes |
Alkan A., et al. 20023131 Alkan A, Yakıncı C, Sıgírcı AS, Kutlu R. Glutaric aciduria type I diagnosed after poliovirus immunization: magnetic resonance findings. Pediatr Neurol. 2002;26:405–7.
|
Case report |
Japan |
Glutaric aciduria type 1 |
2 patients |
Poliovirus |
No |
Yes |
No |
Lukkarinen M., et al. 19996868 Lukkarinen M, Parto K, Ruuskanen O, Vainio O, Kakayhty K, Lander', et al. B and Tcell immunity in patients with lysinuric protein intolerance. Clin Exp Immunol. 1999;116:430–4.
|
Cross-sectional |
Finland |
Lysinuric protein intolerance |
15 patients |
Td, Hib, PPVS 23 |
No |
No |
Yes |