Cantù syndrome: Report of a patient with a novel variant in KCNJ8 and revision of literature.


Journal

American journal of medical genetics. Part A
ISSN: 1552-4833
Titre abrégé: Am J Med Genet A
Pays: United States
ID NLM: 101235741

Informations de publication

Date de publication:
06 2022
Historique:
revised: 10 10 2021
received: 18 12 2020
accepted: 28 01 2022
pubmed: 5 3 2022
medline: 18 5 2022
entrez: 4 3 2022
Statut: ppublish

Résumé

Cantù syndrome (CS) is a rare multisystemic disorder, characterized by congenital hypertrichosis, macrocephaly, facial dysmorphisms, cardiomegaly, vascular, and skeletal anomalies. From the cognitive point of view, most of the patients show a mild speech delay and a few of them present intellectual disability and learning difficulties. To date, most CS-reported cases are caused by heterozygous ABCC9 gene mutations. Only three patients with CS and heterozygous KCNJ8 gene variants have been reported. The authors here present the fourth case of CS with a variant in KCNJ8 in a 6-month-old baby. Diagnosis was reached through Trio-Whole Exome analysis that revealed a de novo missense variant in KCNJ8.

Identifiants

pubmed: 35243770
doi: 10.1002/ajmg.a.62710
doi:

Types de publication

Case Reports Journal Article Review

Langues

eng

Sous-ensembles de citation

IM

Pagination

1661-1666

Informations de copyright

© 2022 Wiley Periodicals LLC.

Références

Accogli, A., Scala, M., Calcagno, A., Castello, R., Torella, A., Musacchia, F., Allegri, A. M. E., Mancardi, M. M., Maghnie, M., Severino, M., Nigro, V., & Capra, V., (2018). Novel CNS malformations and skeletal anomalies in a patient with Beaulieu-boycott-Innes syndrome. American Journal of Medical Genetics Part A, 176(12), 2835-2840. http://dx.doi.org/10.1002/ajmg.a.40534
van Bon, B. W., Gilissen, C., Grange, D. K., Hennekam, R. C., Kayserili, H., Engels, H., Reutter, H., Ostergaard, J. R., Morava, E., Tsiakas, K., Isidor, B., Le Merrer, M., Eser, M., Wieskamp, N., de Vries, P., Steehouwer, M., Veltman, J. A., Robertson, S. P., Brunner, H. G., … Hoischen, A. (2012). Cantú Syndrome Is Caused by Mutations in ABCC9. American Journal of Human Genetics, 90(6), 1094-1101. https://doi.org/10.1016/j.ajhg.2012.04.014
Bienengraeber, M., Olson, T. M., Selivanov, V. A., Kathmann, E. C., O'Cochlain, F., Gao, F., Karger, A. B., Ballew, J. D., Hodgson, D. M., Zingman, L. V., Pang, Y.-P., Alekseev, A. E., & Terzic, A. (2004). ABCC9 mutations identified in human dilated cardiomyopathy disrupt catalytic KATP channel gating. Nature Genetics, 36(4), 382-387. http://dx.doi.org/10.1038/ng1329
Brownstein, C. A., Towne, M. C., Luquette, L. J., Harris, D. J., Marinakis, N. S., Meinecke, P., Kutsche, K., Campeau, P. M., Yu, T. W., Margulies, D. M., Agrawal, P. B., & Beggs, A. H. (2013). Mutation of KCNJ8 in a patient with Cantù syndrome with unique vascular abnormalities - Support the role of K (ATP) channels in this condition. European Journal of Medical Genetics, 56(12), 678-682. https://doi.org/10.1016/j.ejmg.2013.09.009
Chihara, M., Asahina, A., & Itoh, M. (2020). A novel mutation in the KCNJ8 gene encoding the Kir6.1 subunit of an ATP-sensitive potassium channel in a Japanese patient with Cantù syndrome. Journal of the European Academy of Dermatology and Venereology, 34(9), e476-e478. https://doi.org/10.1111/jdv.16384
Cooper, P. E., Reutter, H., Woelfle, J., Engels, H., Grange, D. K., van Haaften, G., van Bon, B. W., Hoischen, A., & Nichols, C. G. (2014). Cantu syndrome resulting from activating mutation in the KCNJ8 gene. Human Mutation, 35(7), 809-813. https://doi.org/10.1002/humu.22555
Grange, D. K., Roessler, H. I., McClenaghan, C., Duran, K., Shields, K., Remedi, M. S., Knoers, N. V. A. M., Lee, J.-M., Kirk, E. P., Scurr, I., Smithson, S. F., Singh, G. K., van Haelst, M. M., Nichols, C. G., & van Haaften, G. (2019). Cantu syndrome: Findings from 74 patients in the international Cantu syndrome registry. Journal of Medical Genetics, 181C, 658-681. https://doi.org/10.1002/ajmg.c.31753
McClenaghan, C., Huang, Y., Yan, Z., Harter, T. M., Halabi, C. M., Chalk, R., Kovacs, A., van Haaften, G., Remedi, M. S., & Nichols, C. G. (2020). Glibenclamide reverses cardiovascular abnormalities of Cantu syndrome driven by KATP channel overactivity. The Journal of Clinical Investigation, 130(3), 1116-1121. https://doi.org/10.1172/JCI130571
Nichols, C. G., Singh, G. K., & Grange, D. K. (2013). K ATP channels and cardiovascular disease suddenly a syndrome. Circulation Research, 112(7), 1059-1072. https://doi.org/10.1161/CIRCRESAHA.112.300514
Robertson, S. P., Kirk, E., Bernier, F., Brereton, J., Turner, A., & Benkier, A. (1999). Congenital hypertrichosis, osteocondrodysplasia and cardiomegaly: Cantù syndrome. American Journal of Human Genetics, 85, 395-402.

Auteurs

Erika Solansh Apuril Velgara (ES)

Department of Pediatric, Mariani Foundation Center for Fragile Child ASST-Lariana, Sant'Anna Hospital, San Fermo della Battaglia (Como), Italy.

Milena Mariani (M)

Department of Pediatric, Mariani Foundation Center for Fragile Child ASST-Lariana, Sant'Anna Hospital, San Fermo della Battaglia (Como), Italy.

Annalaura Torella (A)

Department of Precision Medicine, University "Luigi Vanvitelli", Naples, Italy.
Telethon Institute of Genetics and Medicine (TIGEM), Pozzuoli, Italy.

Francesco Musacchia (F)

Telethon Institute of Genetics and Medicine (TIGEM), Pozzuoli, Italy.

Vincenzo Nigro (V)

Department of Precision Medicine, University "Luigi Vanvitelli", Naples, Italy.
Telethon Institute of Genetics and Medicine (TIGEM), Pozzuoli, Italy.

Angelo Selicorni (A)

Department of Pediatric, Mariani Foundation Center for Fragile Child ASST-Lariana, Sant'Anna Hospital, San Fermo della Battaglia (Como), Italy.

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