Adenotonsillar pathology in mucopolysaccharidoses - lysosomal storage predominates in paracortical CD63+ cells.

Adenoidectomy CD63 CD68 Enzyme-replacement therapy Hematopoietic stem cells transplantation Mucopolysaccharidoses

Journal

Virchows Archiv : an international journal of pathology
ISSN: 1432-2307
Titre abrégé: Virchows Arch
Pays: Germany
ID NLM: 9423843

Informations de publication

Date de publication:
03 Oct 2023
Historique:
received: 01 08 2023
accepted: 17 09 2023
revised: 08 09 2023
medline: 3 10 2023
pubmed: 3 10 2023
entrez: 3 10 2023
Statut: aheadofprint

Résumé

Despite the adenoids are regularly removed in patients with mucopolysaccharidoses (MPS), the underlying tissue and cellular pathologies remain understudied. We characterized an (immuno)histopathologic and ultrastructural phenotype dominated by lysosomal storage changes in a specific subset of adenotonsillar paracortical cells in 8 MPS patients (3 MPS I, 3 MPS II, and 2 MPS IIIA). These abnormal cells were effectively detected by an antibody targeting the lysosomal membrane tetraspanin CD63. Important, CD63+ storage vacuoles in these cells lacked the monocytes/macrophages lysosomal marker CD68. Such a distinct patterning of CD63 and CD68 was not present in a patient with infantile neurovisceral variant of acid sphingomyelinase deficiency. The CD63+ storage pathology was absent in two MPS I patients who either received enzyme-replacement therapy or underwent hematopoietic stem cells transplantation prior the adenoidectomy. Our study demonstrates novel features of lysosomal storage patterning and suggests diagnostic utility of CD63 detection in adenotonsillar lymphoid tissue of MPS patients.

Identifiants

pubmed: 37787787
doi: 10.1007/s00428-023-03662-y
pii: 10.1007/s00428-023-03662-y
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Subventions

Organisme : Ministerstvo Školství, Mládeže a Tělovýchovy
ID : LX22NPO5107
Organisme : Univerzita Karlova v Praze
ID : UNCE/MED/007
Organisme : Univerzita Karlova v Praze
ID : SVV260516
Organisme : Univerzita Karlova v Praze
ID : Cooperatio
Organisme : Ministerstvo Zdravotnictví Ceské Republiky
ID : RVO-VFN 64165/2012

Informations de copyright

© 2023. The Author(s), under exclusive licence to Springer-Verlag GmbH Germany, part of Springer Nature.

Références

Muenzer J (2011) Overview of the mucopolysaccharidoses. Rheumatology (Oxford) 50(Suppl 5):v4-12. https://doi.org/10.1093/rheumatology/ker394
doi: 10.1093/rheumatology/ker394 pubmed: 22210669
Taylor M, Khan S, Stapleton M, Wang J, Chen J, Wynn R, Yabe H, Chinen Y, Boelens JJ, Mason RW, Kubaski F, Horovitz DDG, Barth AL, Serafini M, Bernardo ME, Kobayashi H, Orii KE, Suzuki Y, Orii T, Tomatsu S (2019) Hematopoietic Stem Cell Transplantation for Mucopolysaccharidoses: Past, Present, and Future. Biol Blood Marrow Transplant 25:e226–e246. https://doi.org/10.1016/j.bbmt.2019.02.012
doi: 10.1016/j.bbmt.2019.02.012 pubmed: 30772512 pmcid: 6615945
Murgasova L, Jurovcik M, Jesina P, Malinova V, Bloomfield M, Zeman J, Magner M (2020) Otorhinolaryngological manifestations in 61 patients with mucopolysaccharidosis. Int J Pediatr Otorhinolaryngol 135:110137. https://doi.org/10.1016/j.ijporl.2020.110137
doi: 10.1016/j.ijporl.2020.110137 pubmed: 32502916
Simmons MA, Bruce IA, Penney S, Wraith E, Rothera MP (2005) Otorhinolaryngological manifestations of the mucopolysaccharidoses. Int J Pediatr Otorhinolaryngol 69:589–595. https://doi.org/10.1016/j.ijporl.2005.01.017
doi: 10.1016/j.ijporl.2005.01.017 pubmed: 15850680
Ahmad Z, Kruger K, Lautermann J, Lippert B, Tenenbaum T, Tigges M, Tisch M (2023) Adenoid hypertrophy-diagnosis and treatment: the new S2k guideline. HNO. https://doi.org/10.1007/s00106-023-01299-6
doi: 10.1007/s00106-023-01299-6 pubmed: 37491540 pmcid: 10409824
Fujitani T, Kimura A, Inoue K, Okada S (1985) Pathological and biochemical study in the adenoid of mucopolysaccharidosis II. Int J Pediatr Otorhinolaryngol 10:205–212. https://doi.org/10.1016/s0165-5876(85)80066-5
doi: 10.1016/s0165-5876(85)80066-5 pubmed: 3938449
Gonuldas B, Yilmaz T, Sivri HS, Gucer KS, Kilinc K, Genc GA, Kilic M, Coskun T (2014) Mucopolysaccharidosis: Otolaryngologic findings, obstructive sleep apnea and accumulation of glucosaminoglycans in lymphatic tissue of the upper airway. Int J Pediatr Otorhinolaryngol 78:944–949. https://doi.org/10.1016/j.ijporl.2014.03.021
doi: 10.1016/j.ijporl.2014.03.021 pubmed: 24731921
Keilmann A, Lassig AK, Pollak-Hainz A, Mann WJ, Beck M, Hainz M (2015) Adenoids of patients with mucopolysaccharidoses demonstrate typical alterations. Int J Pediatr Otorhinolaryngol 79:115–118. https://doi.org/10.1016/j.ijporl.2014.11.014
doi: 10.1016/j.ijporl.2014.11.014 pubmed: 25487873
Nayak DR, Balakrishnan R, Adolph S (1998) Endoscopic adenoidectomy in a case of Scheie syndrome (MPS I S). Int J Pediatr Otorhinolaryngol 44:177–181. https://doi.org/10.1016/s0165-5876(98)00054-8
doi: 10.1016/s0165-5876(98)00054-8 pubmed: 9725536
Pal AR, Mercer J, Jones SA, Bruce IA, Bigger BW (2018) Substrate accumulation and extracellular matrix remodelling promote persistent upper airway disease in mucopolysaccharidosis patients on enzyme replacement therapy. Plos One 13:e0203216. https://doi.org/10.1371/journal.pone.0203216
doi: 10.1371/journal.pone.0203216 pubmed: 30226843 pmcid: 6143186
Dostalova G, Hlubocka Z, Lindner J, Hulkova H, Poupetova H, Vlaskova H, Sikora J, Linhart A, Zeman J, Magner M (2018) Late diagnosis of mucopolysaccharidosis type IVB and successful aortic valve replacement in a 60-year-old female patient. Cardiovasc Pathol 35:52–56. https://doi.org/10.1016/j.carpath.2018.04.001
doi: 10.1016/j.carpath.2018.04.001 pubmed: 29800929
Bolar NA, Golzio C, Zivna M, Hayot G, Van Hemelrijk C, Schepers D, Vandeweyer G, Hoischen A, Huyghe JR, Raes A, Matthys E, Sys E, Azou M, Gubler MC, Praet M, Van Camp G, McFadden K, Pediaditakis I, Pristoupilova A, Hodanova K, Vyletal P, Hartmannova H, Stranecky V, Hulkova H, Baresova V, Jedlickova I, Sovova J, Hnizda A, Kidd K, Bleyer AJ, Spong RS, Vande Walle J, Mortier G, Brunner H, Van Laer L, Kmoch S, Katsanis N, Loeys BL (2016) Heterozygous loss-of-function SEC61A1 mutations cause autosomal-dominant tubulo-interstitial and glomerulocystic kidney disease with anemia. Am J Hum Genet 99:174–187. https://doi.org/10.1016/j.ajhg.2016.05.028
doi: 10.1016/j.ajhg.2016.05.028 pubmed: 27392076 pmcid: 5005467
Collin M, Bigley V (2018) Human dendritic cell subsets: an update. Immunology 154:3–20. https://doi.org/10.1111/imm.12888
doi: 10.1111/imm.12888 pubmed: 29313948 pmcid: 5904714
Garces S, Yin CC, Miranda RN, Patel KP, Li S, Xu J, Thakral B, Poppiti RJ, Medina AM, Sriganeshan V, Castellano-Sanchez A, Khoury JD, Garces JC, Medeiros LJ (2020) Clinical, histopathologic, and immunoarchitectural features of dermatopathic lymphadenopathy: an update. Mod Pathol 33:1104–1121. https://doi.org/10.1038/s41379-019-0440-4
doi: 10.1038/s41379-019-0440-4 pubmed: 31896812
Reboun M, Rybova J, Dobrovolny R, Vcelak J, Veselkova T, Storkanova G, Musalkova D, Hrebicek M, Ledvinova J, Magner M, Zeman J, Peskova K, Dvorakova L (2016) X-Chromosome Inactivation Analysis in Different Cell Types and Induced Pluripotent Stem Cells Elucidates the Disease Mechanism in a Rare Case of Mucopolysaccharidosis Type II in a Female. Folia Biol (Praha) 62:82–89
pubmed: 27187040

Auteurs

Lenka Murgasova (L)

Department of Otorhinolaryngology, 3rd Faculty of Medicine, Charles University and University Hospital Kralovske Vinohrady in Prague, Prague, Czech Republic.
Research Unit for Rare Diseases, Department of Paediatrics and Inherited Metabolic Disorders, 1st Faculty of Medicine, Charles University and General University Hospital in Prague, Ke Karlovu 2, 128 00, Prague 2, Czech Republic.

Helena Hulkova (H)

Research Unit for Rare Diseases, Department of Paediatrics and Inherited Metabolic Disorders, 1st Faculty of Medicine, Charles University and General University Hospital in Prague, Ke Karlovu 2, 128 00, Prague 2, Czech Republic.
Institute of Pathology, 1st Faculty of Medicine, Charles University and General University Hospital in Prague, Prague, Czech Republic.

Veronika Baresova (V)

Research Unit for Rare Diseases, Department of Paediatrics and Inherited Metabolic Disorders, 1st Faculty of Medicine, Charles University and General University Hospital in Prague, Ke Karlovu 2, 128 00, Prague 2, Czech Republic.

Michal Jurovcik (M)

Department of ENT, 2nd Faculty of Medicine, Charles University and Motol University Hospital in Prague, Prague, Czech Republic.

Jan Stritesky (J)

Institute of Pathology, 1st Faculty of Medicine, Charles University and General University Hospital in Prague, Prague, Czech Republic.

Katarina Jurickova (K)

Department of Paediatrics, Faculty of Medicine, Comenius University in Bratislava and National Institute of Children's Diseases in Bratislava, Bratislava, Slovakia.
Centre for Inborn Errors of Metabolism, National Institute of Children's Diseases in Bratislava, Bratislava, Slovakia.

Martin Magner (M)

Research Unit for Rare Diseases, Department of Paediatrics and Inherited Metabolic Disorders, 1st Faculty of Medicine, Charles University and General University Hospital in Prague, Ke Karlovu 2, 128 00, Prague 2, Czech Republic.

Jakub Sikora (J)

Research Unit for Rare Diseases, Department of Paediatrics and Inherited Metabolic Disorders, 1st Faculty of Medicine, Charles University and General University Hospital in Prague, Ke Karlovu 2, 128 00, Prague 2, Czech Republic. jakub.sikora@lf1.cuni.cz.
Institute of Pathology, 1st Faculty of Medicine, Charles University and General University Hospital in Prague, Prague, Czech Republic. jakub.sikora@lf1.cuni.cz.

Classifications MeSH