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Dwarfism, intellectual disability, and eye abnormality

MedGen UID:
208664
Concept ID:
C0796076
Disease or Syndrome
Synonyms: Dwarfism, mental retardation, and eye abnormality; Mollica syndrome; Mollica-Pavone-Antener syndrome
 
Monarch Initiative: MONDO:0009128
OMIM®: 223540

Definition

Delayed growth and mental development with ocular disorders. [from MCA/MR]

Clinical features

From HPO
Severe short stature
MedGen UID:
3931
Concept ID:
C0013336
Disease or Syndrome
A severe degree of short stature, more than -4 SD from the mean corrected for age and sex.
High myopia
MedGen UID:
78759
Concept ID:
C0271183
Disease or Syndrome
A severe form of myopia with greater than -6.00 diopters.
Hypoplasia of the iris
MedGen UID:
91029
Concept ID:
C0344539
Congenital Abnormality
Congenital underdevelopment of the iris.
Short stature
MedGen UID:
87607
Concept ID:
C0349588
Finding
A height below that which is expected according to age and gender norms. Although there is no universally accepted definition of short stature, many refer to "short stature" as height more than 2 standard deviations below the mean for age and gender (or below the 3rd percentile for age and gender dependent norms).
Nuclear cataract
MedGen UID:
140274
Concept ID:
C0392557
Finding
A nuclear cataract is an opacity or clouding that develops in the lens nucleus. That is, a nuclear cataract is one that is located in the center of the lens. The nucleus tends to darken changing from clear to yellow and sometimes brown.
Intellectual disability
MedGen UID:
811461
Concept ID:
C3714756
Mental or Behavioral Dysfunction
Subnormal intellectual functioning which originates during the developmental period. Intellectual disability, previously referred to as mental retardation, has been defined as an IQ score below 70.
Abnormality of the orbital region
MedGen UID:
871367
Concept ID:
C4025863
Anatomical Abnormality
Microcephaly
MedGen UID:
1644158
Concept ID:
C4551563
Finding
Head circumference below 2 standard deviations below the mean for age and gender.

Recent clinical studies

Etiology

Yabumoto M, Kianmahd J, Singh M, Palafox MF, Wei A, Elliott K, Goodloe DH, Dean SJ, Gooch C, Murray BK, Swartz E, Schrier Vergano SA, Towne MC, Nugent K, Roeder ER, Kresge C, Pletcher BA, Grand K, Graham JM Jr, Gates R, Gomez-Ospina N, Ramanathan S, Clark RD, Glaser K, Benke PJ, Cohen JS, Fatemi A, Mu W, Baranano KW, Madden JA, Gubbels CS, Yu TW, Agrawal PB, Chambers MK, Phornphutkul C, Pugh JA, Tauber KA, Azova S, Smith JR, O'Donnell-Luria A, Medsker H, Srivastava S, Krakow D, Schweitzer DN, Arboleda VA
Mol Genet Genomic Med 2021 Oct;9(10):e1809. Epub 2021 Sep 14 doi: 10.1002/mgg3.1809. PMID: 34519438Free PMC Article
Brea-Fernández A, Dacruz D, Eirís J, Barros F, Carracedo Á
Am J Med Genet A 2019 Feb;179(2):290-294. Epub 2018 Dec 20 doi: 10.1002/ajmg.a.60689. PMID: 30569622
Gannon T, Perveen R, Schlecht H, Ramsden S, Anderson B, Kerr B, Day R, Banka S, Suri M, Berland S, Gabbett M, Ma A, Lyonnet S, Cormier-Daire V, Yilmaz R, Borck G, Wieczorek D, Anderlid BM, Smithson S, Vogt J, Moore-Barton H, Simsek-Kiper PO, Maystadt I, Destrée A, Bucher J, Angle B, Mohammed S, Wakeling E, Price S, Singer A, Sznajer Y, Toutain A, Haye D, Newbury-Ecob R, Fradin M, McGaughran J, Tuysuz B, Tein M, Bouman K, Dabir T, Van den Ende J, Luk HM, Pilz DT, Eason J, Davies S, Reardon W, Garavelli L, Zuffardi O, Devriendt K, Armstrong R, Johnson D, Doco-Fenzy M, Bijlsma E, Unger S, Veenstra-Knol HE, Kohlhase J, Lo IF; DDD study, Smith J, Clayton-Smith J
Eur J Hum Genet 2015 Sep;23(9):1165-70. Epub 2014 Nov 26 doi: 10.1038/ejhg.2014.248. PMID: 25424711Free PMC Article
Szakszon K, Salpietro C, Kakar N, Knegt AC, Oláh É, Dallapiccola B, Borck G
Am J Med Genet A 2013 Apr;161A(4):884-8. Epub 2013 Feb 22 doi: 10.1002/ajmg.a.35848. PMID: 23436491
Brancati F, D'Avanzo MG, Digilio MC, Sarkozy A, Biondi M, De Brasi D, Mingarelli R, Dallapiccola B
Am J Med Genet A 2004 Dec 1;131(2):144-9. doi: 10.1002/ajmg.a.30292. PMID: 15523620

Diagnosis

Stoll C, Alembik Y, Dott B, Roth MP
Eur J Med Genet 2022 Nov;65(11):104612. Epub 2022 Sep 20 doi: 10.1016/j.ejmg.2022.104612. PMID: 36150686
Lonardo F, Lonardo MS, Acquaviva F, Della Monica M, Scarano F, Scarano G
Clin Genet 2019 Feb;95(2):253-261. Epub 2018 Jan 25 doi: 10.1111/cge.13127. PMID: 28857140
Merritt J, Hart JC, LeGrow TL
BMJ Case Rep 2017 Jul 14;2017 doi: 10.1136/bcr-2017-219930. PMID: 28710305Free PMC Article
Gannon T, Perveen R, Schlecht H, Ramsden S, Anderson B, Kerr B, Day R, Banka S, Suri M, Berland S, Gabbett M, Ma A, Lyonnet S, Cormier-Daire V, Yilmaz R, Borck G, Wieczorek D, Anderlid BM, Smithson S, Vogt J, Moore-Barton H, Simsek-Kiper PO, Maystadt I, Destrée A, Bucher J, Angle B, Mohammed S, Wakeling E, Price S, Singer A, Sznajer Y, Toutain A, Haye D, Newbury-Ecob R, Fradin M, McGaughran J, Tuysuz B, Tein M, Bouman K, Dabir T, Van den Ende J, Luk HM, Pilz DT, Eason J, Davies S, Reardon W, Garavelli L, Zuffardi O, Devriendt K, Armstrong R, Johnson D, Doco-Fenzy M, Bijlsma E, Unger S, Veenstra-Knol HE, Kohlhase J, Lo IF; DDD study, Smith J, Clayton-Smith J
Eur J Hum Genet 2015 Sep;23(9):1165-70. Epub 2014 Nov 26 doi: 10.1038/ejhg.2014.248. PMID: 25424711Free PMC Article
Carr RE
Int Ophthalmol Clin 1969 Winter;9(4):971-1003. doi: 10.1097/00004397-196900940-00009. PMID: 4101337

Prognosis

Marangi G, Di Giacomo MC, Lattante S, Orteschi D, Patrizi S, Doronzio PN, Riviello FN, Vaisfeld A, Frangella S, Zollino M
Am J Med Genet A 2018 Feb;176(2):455-459. Epub 2017 Dec 11 doi: 10.1002/ajmg.a.38571. PMID: 29226580
Yilmaz R, Beleza-Meireles A, Price S, Oliveira R, Kubisch C, Clayton-Smith J, Szakszon K, Borck G
Am J Med Genet A 2015 Dec;167A(12):3006-10. Epub 2015 Sep 3 doi: 10.1002/ajmg.a.37343. PMID: 26334766
Gannon T, Perveen R, Schlecht H, Ramsden S, Anderson B, Kerr B, Day R, Banka S, Suri M, Berland S, Gabbett M, Ma A, Lyonnet S, Cormier-Daire V, Yilmaz R, Borck G, Wieczorek D, Anderlid BM, Smithson S, Vogt J, Moore-Barton H, Simsek-Kiper PO, Maystadt I, Destrée A, Bucher J, Angle B, Mohammed S, Wakeling E, Price S, Singer A, Sznajer Y, Toutain A, Haye D, Newbury-Ecob R, Fradin M, McGaughran J, Tuysuz B, Tein M, Bouman K, Dabir T, Van den Ende J, Luk HM, Pilz DT, Eason J, Davies S, Reardon W, Garavelli L, Zuffardi O, Devriendt K, Armstrong R, Johnson D, Doco-Fenzy M, Bijlsma E, Unger S, Veenstra-Knol HE, Kohlhase J, Lo IF; DDD study, Smith J, Clayton-Smith J
Eur J Hum Genet 2015 Sep;23(9):1165-70. Epub 2014 Nov 26 doi: 10.1038/ejhg.2014.248. PMID: 25424711Free PMC Article

Clinical prediction guides

Stoll C, Alembik Y, Dott B, Roth MP
Eur J Med Genet 2022 Nov;65(11):104612. Epub 2022 Sep 20 doi: 10.1016/j.ejmg.2022.104612. PMID: 36150686
Martín-Rivada Á, Pozo-Román J, Güemes M, Ortiz-Cabrera NV, Pérez-Jurado LA, Argente J
Horm Res Paediatr 2020;93(9-10):567-572. Epub 2021 Mar 23 doi: 10.1159/000514280. PMID: 33756487
Marangi G, Di Giacomo MC, Lattante S, Orteschi D, Patrizi S, Doronzio PN, Riviello FN, Vaisfeld A, Frangella S, Zollino M
Am J Med Genet A 2018 Feb;176(2):455-459. Epub 2017 Dec 11 doi: 10.1002/ajmg.a.38571. PMID: 29226580
Gannon T, Perveen R, Schlecht H, Ramsden S, Anderson B, Kerr B, Day R, Banka S, Suri M, Berland S, Gabbett M, Ma A, Lyonnet S, Cormier-Daire V, Yilmaz R, Borck G, Wieczorek D, Anderlid BM, Smithson S, Vogt J, Moore-Barton H, Simsek-Kiper PO, Maystadt I, Destrée A, Bucher J, Angle B, Mohammed S, Wakeling E, Price S, Singer A, Sznajer Y, Toutain A, Haye D, Newbury-Ecob R, Fradin M, McGaughran J, Tuysuz B, Tein M, Bouman K, Dabir T, Van den Ende J, Luk HM, Pilz DT, Eason J, Davies S, Reardon W, Garavelli L, Zuffardi O, Devriendt K, Armstrong R, Johnson D, Doco-Fenzy M, Bijlsma E, Unger S, Veenstra-Knol HE, Kohlhase J, Lo IF; DDD study, Smith J, Clayton-Smith J
Eur J Hum Genet 2015 Sep;23(9):1165-70. Epub 2014 Nov 26 doi: 10.1038/ejhg.2014.248. PMID: 25424711Free PMC Article
O'Driscoll M, Dobyns WB, van Hagen JM, Jeggo PA
Am J Hum Genet 2007 Jul;81(1):77-86. Epub 2007 May 17 doi: 10.1086/518696. PMID: 17564965Free PMC Article

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