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Noonan syndrome with multiple lentigines(NSML)

MedGen UID:
104494
Concept ID:
C0175704
Disease or Syndrome
Synonyms: Cardiomyopathic lentiginosis; Lentigines, Electrocardiographic conduction abnormalities, Ocular hypertelorism, Pulmonic stenosis, Abnormal genitalia, Retardation of growth, Deafness; LEOPARD syndrome; NSML
SNOMED CT: Leopard syndrome lentiginosis (111306001); Multiple lentigines syndrome (111306001); LEOPARD syndrome (111306001)
Modes of inheritance:
Autosomal dominant inheritance
MedGen UID:
141047
Concept ID:
C0443147
Intellectual Product
Source: Orphanet
A mode of inheritance that is observed for traits related to a gene encoded on one of the autosomes (i.e., the human chromosomes 1-22) in which a trait manifests in heterozygotes. In the context of medical genetics, an autosomal dominant disorder is caused when a single copy of the mutant allele is present. Males and females are affected equally, and can both transmit the disorder with a risk of 50% for each child of inheriting the mutant allele.
 
Related genes: RAF1, PTPN11, BRAF
 
Monarch Initiative: MONDO:0007893
OMIM®: 151100
OMIM® Phenotypic series: PS151100
Orphanet: ORPHA500

Disease characteristics

Excerpted from the GeneReview: Noonan Syndrome with Multiple Lentigines
Noonan syndrome with multiple lentigines (NSML) is a condition in which the cardinal features consist of lentigines, hypertrophic cardiomyopathy, short stature, pectus deformity, and dysmorphic facial features including widely spaced eyes and ptosis. Multiple lentigines present as dispersed flat, black-brown macules, mostly on the face, neck, and upper part of the trunk with sparing of the mucosa. In general, lentigines do not appear until age four to five years but then increase to the thousands by puberty. Some individuals with NSML do not exhibit lentigines. Approximately 85% of affected individuals have heart defects, including hypertrophic cardiomyopathy (typically appearing during infancy and sometimes progressive) and pulmonary valve stenosis. Postnatal growth restriction resulting in short stature occurs in fewer than 50% of affected persons, although most affected individuals have a height that is less than the 25th centile for age. Sensorineural hearing deficits, present in approximately 20% of affected individuals, are poorly characterized. Intellectual disability, typically mild, is observed in approximately 30% of persons with NSML. [from GeneReviews]
Authors:
Bruce D Gelb  |  Marco Tartaglia   view full author information

Additional description

From MedlinePlus Genetics
Noonan syndrome with multiple lentigines (formerly called LEOPARD syndrome) is a condition that affects many areas of the body. As the condition name suggests, Noonan syndrome with multiple lentigines is very similar to a condition called Noonan syndrome, and it can be difficult to tell the two disorders apart in early childhood. However, the features of these two conditions differ later in life. The characteristic features of Noonan syndrome with multiple lentigines include brown skin spots called lentigines that are similar to freckles, heart defects, widely spaced eyes (ocular hypertelorism), a sunken chest (pectus excavatum) or protruding chest (pectus carinatum), and short stature. These features vary, however, even among affected individuals in the same family. Not all individuals with Noonan syndrome with multiple lentigines have all the characteristic features of this condition.

The lentigines seen in Noonan syndrome with multiple lentigines typically first appear in mid-childhood, mostly on the face, neck, and upper body. Affected individuals may have thousands of small dark brown skin spots by the time they reach puberty. Unlike freckles, the appearance of lentigines has nothing to do with sun exposure. In addition to lentigines, people with this condition may have lighter brown skin spots called café-au-lait spots. Café-au-lait spots tend to develop before the lentigines, appearing within the first year of life in most affected people.

Of the people with Noonan syndrome with multiple lentigines who have heart defects, about 80 percent have hypertrophic cardiomyopathy, which is a thickening of the heart muscle that forces the heart to work harder to pump blood. The hypertrophic cardiomyopathy most often affects the lower left chamber of the heart (the left ventricle). Up to 20 percent of people with Noonan syndrome with multiple lentigines who have heart problems have a narrowing of the artery from the heart to the lungs (pulmonary stenosis).

People with Noonan syndrome with multiple lentigines can have a distinctive facial appearance. In addition to ocular hypertelorism, affected individuals may have droopy eyelids (ptosis), thick lips, and low-set ears. Affected individuals also usually have an abnormal appearance of the chest; they either have pectus excavatum or pectus carinatum.

At birth, people with Noonan syndrome with multiple lentigines are typically of normal weight and height, but in some, growth slows over time. This slow growth results in affected individuals being shorter than average, although less than half of people with Noonan syndrome with multiple lentigines have significantly short stature.

Other signs and symptoms of Noonan syndrome with multiple lentigines include hearing loss caused by abnormalities in the inner ear (sensorineural deafness), mild intellectual disability, and extra folds of skin on the back of the neck. Affected males often have genital abnormalities, which can include undescended testes (cryptorchidism) and a urethra that opens on the underside of the penis (hypospadias). These abnormalities may reduce the ability to have biological children (decreased fertility). Females with Noonan syndrome with multiple lentigines may have poorly developed ovaries and delayed puberty.

Noonan syndrome with multiple lentigines is one of a group of related conditions collectively known as RASopathies. These conditions all have similar signs and symptoms and are caused by changes in the same cell signaling pathway. In addition to Noonan syndrome with multiple lentigines, the RASopathies include Noonan syndrome, cardiofaciocutaneous syndrome, Costello syndrome, neurofibromatosis type 1, and Legius syndrome.  https://medlineplus.gov/genetics/condition/noonan-syndrome-with-multiple-lentigines

Professional guidelines

PubMed

Yi JS, Perla S, Bennett AM
Cardiovasc Drugs Ther 2023 Dec;37(6):1193-1204. Epub 2022 Feb 14 doi: 10.1007/s10557-022-07324-0. PMID: 35156148
Kavamura MI, Leoni C, Neri G
Am J Med Genet C Semin Med Genet 2022 Dec;190(4):452-458. Epub 2022 Dec 21 doi: 10.1002/ajmg.c.32027. PMID: 36541891
Gelb BD, Yohe ME, Wolf C, Andelfinger G
Am J Med Genet C Semin Med Genet 2022 Dec;190(4):541-560. Epub 2022 Dec 19 doi: 10.1002/ajmg.c.32024. PMID: 36533679Free PMC Article

Recent clinical studies

Etiology

Zenker M
Am J Med Genet C Semin Med Genet 2022 Dec;190(4):414-424. Epub 2022 Nov 25 doi: 10.1002/ajmg.c.32015. PMID: 36428239
Palit A, Inamadar AC
Indian J Dermatol Venereol Leprol 2022 May-Jun;88(4):452-463. doi: 10.25259/IJDVL_799_20. PMID: 35138057
Rosser T
Continuum (Minneap Minn) 2018 Feb;24(1, Child Neurology):96-129. doi: 10.1212/CON.0000000000000562. PMID: 29432239
Aoki Y, Niihori T, Inoue S, Matsubara Y
J Hum Genet 2016 Jan;61(1):33-9. Epub 2015 Oct 8 doi: 10.1038/jhg.2015.114. PMID: 26446362
Rauen KA
Annu Rev Genomics Hum Genet 2013;14:355-69. Epub 2013 Jul 15 doi: 10.1146/annurev-genom-091212-153523. PMID: 23875798Free PMC Article

Diagnosis

Monda E, Prosnitz A, Aiello R, Lioncino M, Norrish G, Caiazza M, Drago F, Beattie M, Tartaglia M, Russo MG, Colan SD, Calcagni G, Gelb BD, Kaski JP, Roberts AE, Limongelli G
Circ Genom Precis Med 2023 Aug;16(4):350-358. Epub 2023 May 18 doi: 10.1161/CIRCGEN.122.003861. PMID: 37199218
Kavamura MI, Leoni C, Neri G
Am J Med Genet C Semin Med Genet 2022 Dec;190(4):452-458. Epub 2022 Dec 21 doi: 10.1002/ajmg.c.32027. PMID: 36541891
Delogu AB, Limongelli G, Versacci P, Adorisio R, Kaski JP, Blandino R, Maiolo S, Monda E, Putotto C, De Rosa G, Chatfield KC, Gelb BD, Calcagni G
Am J Med Genet C Semin Med Genet 2022 Dec;190(4):440-451. Epub 2022 Nov 21 doi: 10.1002/ajmg.c.32014. PMID: 36408797
Palit A, Inamadar AC
Indian J Dermatol Venereol Leprol 2022 May-Jun;88(4):452-463. doi: 10.25259/IJDVL_799_20. PMID: 35138057
Rosser T
Continuum (Minneap Minn) 2018 Feb;24(1, Child Neurology):96-129. doi: 10.1212/CON.0000000000000562. PMID: 29432239

Therapy

Chaput D, Andelfinger G
Can J Cardiol 2024 May;40(5):789-799. Epub 2024 Mar 1 doi: 10.1016/j.cjca.2024.02.020. PMID: 38432396
Rauen KA, Alsaegh A, Ben-Shachar S, Berman Y, Blakeley J, Cordeiro I, Elgersma Y, Evans DG, Fisher MJ, Frayling IM, George J, Huson SM, Kerr B, Khire U, Korf B, Legius E, Messiaen L, van Minkelen R, Nampoothiri S, Ngeow J, Parada LF, Phadke S, Pillai A, Plotkin SR, Puri R, Raji A, Ramesh V, Ratner N, Shankar SP, Sharda S, Tambe A, Vikkula M, Widemann BC, Wolkenstein P, Upadhyaya M
Am J Med Genet A 2019 Jun;179(6):1091-1097. Epub 2019 Mar 25 doi: 10.1002/ajmg.a.61125. PMID: 30908877Free PMC Article
Levin MD, Saitta SC, Gripp KW, Wenger TL, Ganesh J, Kalish JM, Epstein MR, Smith R, Czosek RJ, Ware SM, Goldenberg P, Myers A, Chatfield KC, Gillespie MJ, Zackai EH, Lin AE
Am J Med Genet A 2018 Aug;176(8):1711-1722. Epub 2018 Jul 28 doi: 10.1002/ajmg.a.38854. PMID: 30055033Free PMC Article
Stevenson DA, Schill L, Schoyer L, Andresen BS, Bakker A, Bayrak-Toydemir P, Burkitt-Wright E, Chatfield K, Elefteriou F, Elgersma Y, Fisher MJ, Franz D, Gelb BD, Goriely A, Gripp KW, Hardan AY, Keppler-Noreuil KM, Kerr B, Korf B, Leoni C, McCormick F, Plotkin SR, Rauen KA, Reilly K, Roberts A, Sandler A, Siegel D, Walsh K, Widemann BC
Am J Med Genet A 2016 Aug;170(8):1959-66. Epub 2016 May 7 doi: 10.1002/ajmg.a.37723. PMID: 27155140Free PMC Article
McWilliams GD, SantaCruz K, Hart B, Clericuzio C
Am J Med Genet A 2016 Jan;170A(1):195-201. Epub 2015 Sep 17 doi: 10.1002/ajmg.a.37379. PMID: 26377682

Prognosis

Delogu AB, Limongelli G, Versacci P, Adorisio R, Kaski JP, Blandino R, Maiolo S, Monda E, Putotto C, De Rosa G, Chatfield KC, Gelb BD, Calcagni G
Am J Med Genet C Semin Med Genet 2022 Dec;190(4):440-451. Epub 2022 Nov 21 doi: 10.1002/ajmg.c.32014. PMID: 36408797
Davico C, D'Alessandro R, Borgogno M, Campagna F, Torta F, Ricci F, Amianto F, Vittorini R, Carli D, Mussa A, Vitiello B, Ferrero GB
Eur J Pediatr 2022 Aug;181(8):2919-2926. Epub 2022 May 16 doi: 10.1007/s00431-022-04497-6. PMID: 35575813
Kauffman H, Ahrens-Nicklas RC, Calderon-Anyosa RJC, Ritter AL, Lin KY, Rossano JW, Quartermain MD, Banerjee A
Pediatr Res 2021 Aug;90(2):444-451. Epub 2020 Dec 14 doi: 10.1038/s41390-020-01292-7. PMID: 33318624
Gao X, Huang SS, Qiu SW, Su Y, Wang WQ, Xu HY, Xu JC, Kang DY, Dai P, Yuan YY
J Med Genet 2021 Jul;58(7):465-474. Epub 2020 Jul 31 doi: 10.1136/jmedgenet-2020-106892. PMID: 32737134
Jhang WK, Choi JH, Lee BH, Kim GH, Yoo HW
Pediatr Cardiol 2016 Dec;37(8):1539-1547. Epub 2016 Aug 23 doi: 10.1007/s00246-016-1468-6. PMID: 27554254

Clinical prediction guides

Monda E, Prosnitz A, Aiello R, Lioncino M, Norrish G, Caiazza M, Drago F, Beattie M, Tartaglia M, Russo MG, Colan SD, Calcagni G, Gelb BD, Kaski JP, Roberts AE, Limongelli G
Circ Genom Precis Med 2023 Aug;16(4):350-358. Epub 2023 May 18 doi: 10.1161/CIRCGEN.122.003861. PMID: 37199218
Monda E, Verrillo F, Altobelli I, Lioncino M, Caiazza M, Rubino M, Cirillo A, Fusco A, Esposito A, Di Fraia F, Pacileo R, Gragnano F, Passariello A, Calabrò P, Russo MG, Limongelli G
Int J Cardiol 2022 Mar 1;350:77-82. Epub 2021 Dec 27 doi: 10.1016/j.ijcard.2021.12.043. PMID: 34968628
Kauffman H, Ahrens-Nicklas RC, Calderon-Anyosa RJC, Ritter AL, Lin KY, Rossano JW, Quartermain MD, Banerjee A
Pediatr Res 2021 Aug;90(2):444-451. Epub 2020 Dec 14 doi: 10.1038/s41390-020-01292-7. PMID: 33318624
Wang J, Chandrasekhar V, Abbadessa G, Yu Y, Schwartz B, Kontaridis MI
PLoS One 2017;12(6):e0178905. Epub 2017 Jun 5 doi: 10.1371/journal.pone.0178905. PMID: 28582432Free PMC Article
Conboy E, Dhamija R, Wang M, Xie J, Dyck PJ, Bridges AG, Spinner RJ, Clayton AC, Watson RE, Messiaen L, Babovic-Vuksanovic D
J Med Genet 2016 Feb;53(2):123-6. Epub 2015 Sep 2 doi: 10.1136/jmedgenet-2015-103177. PMID: 26337637

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