Literature DB >> 19353582

Significant overlap and possible identity of macrocephaly capillary malformation and megalencephaly polymicrogyria-polydactyly hydrocephalus syndromes.

Karen W Gripp1, Elizabeth Hopkins, Chana Vinkler, Dorit Lev, Gustavo Malinger, Tally Lerman-Sagie, William B Dobyns.   

Abstract

We report on three patients with macrocephaly and polymicrogyria, and additional anomalies seen in megalencephaly polymicrogyria-polydactyly hydrocephalus (MPPH) and macrocephaly capillary malformation (MCM) syndromes. Based on their characteristic brain malformations they were originally diagnosed with MPPH. In one patient the phenotype evolved during early infancy, and ultimately resulted in a diagnosis of MCM. A second was prenatally diagnosed with MPPH, but postnatally visualized capillary malformations led to a diagnosis of MCM. In a third, the original MPPH diagnosis was reconsidered after a critical review revealed additional subtle findings suggestive of MCM. Characteristic brain malformations are thought to distinguish between MPPH with perisylvian polymicrogyria, and MCM with megalencephaly with Chiari 1 malformation. However, polymicrogyria was reported in a significant number of patients with MCM. Conversely, upon review of imaging studies of patients with MPPH, we noted progressive crowding of the posterior fossa and acquired tonsillar herniation, a process deemed characteristic for MCM. Thus, neither polymicrogyria nor acquired tonsillar herniation are distinguishing features, and occur in both disorders. In addition to brain abnormalities, shared findings include cognitive impairment, coarse facial features and postaxial polydactyly. Facial nevus flammeus and cutis marmorata are most noticeable in infancy, and ligamentous laxity and redundant soft tissue are somewhat subjective findings. While asymmetric overgrowth is considered typical for MCM, it is not universally present. These variable and subtle findings can be identified in patients with MPPH. We propose that MPPH and MCM may not represent distinct entities and that the term MPPH-CM syndrome be used to describe this spectrum.

Entities:  

Mesh:

Year:  2009        PMID: 19353582     DOI: 10.1002/ajmg.a.32732

Source DB:  PubMed          Journal:  Am J Med Genet A        ISSN: 1552-4825            Impact factor:   2.802


  12 in total

Review 1.  High incidence of progressive postnatal cerebellar enlargement in Costello syndrome: brain overgrowth associated with HRAS mutations as the likely cause of structural brain and spinal cord abnormalities.

Authors:  Karen W Gripp; Elizabeth Hopkins; Daniel Doyle; William B Dobyns
Journal:  Am J Med Genet A       Date:  2010-05       Impact factor: 2.802

2.  Unbalanced der(5)t(5;20) translocation associated with megalencephaly, perisylvian polymicrogyria, polydactyly and hydrocephalus.

Authors:  Annemieke J M H Verkerk; Rachel Schot; Laura van Waterschoot; Hannie Douben; Pino J Poddighe; Maarten H Lequin; Linda S de Vries; Paulien Terhal; Johanne M D Hahnemann; Irenaeus F M de Coo; Marie-Claire Y de Wit; Leontien S Wafelman; Livia Garavelli; William B Dobyns; Peter J Van der Spek; Annelies de Klein; Grazia M S Mancini
Journal:  Am J Med Genet A       Date:  2010-06       Impact factor: 2.802

Review 3.  Neurocutaneous vascular syndromes.

Authors:  Katherine B Puttgen; Doris D M Lin
Journal:  Childs Nerv Syst       Date:  2010-06-27       Impact factor: 1.475

4.  Sudden death in a case of megalencephaly capillary malformation associated with a de novo mutation in AKT3.

Authors:  Atsuko Harada; Fuyuki Miya; Hidetsuna Utsunomiya; Mitsuhiro Kato; Takumi Yamanaka; Tatsuhiko Tsunoda; Kenjiro Kosaki; Yonehiro Kanemura; Mami Yamasaki
Journal:  Childs Nerv Syst       Date:  2014-11-22       Impact factor: 1.475

Review 5.  Megalencephaly-capillary malformation syndrome and associated hydrocephalus: treatment options and revision of the literature.

Authors:  M Alamar; S Candela; A Flor-Goikoetxea; H Salvador; A F Martinez-Monseny; J Muchart; J Hinojosa
Journal:  Childs Nerv Syst       Date:  2021-05-28       Impact factor: 1.475

6.  Four-year follow-up of megalencephaly, polymicrogyria, postaxial polydactyly and hydrocephalus (MPPH) syndrome.

Authors:  Tara G Zamora; Kari D Roberts
Journal:  BMJ Case Rep       Date:  2013-10-03

Review 7.  Current concepts of polymicrogyria.

Authors:  A James Barkovich
Journal:  Neuroradiology       Date:  2010-03-03       Impact factor: 2.804

Review 8.  A developmental and genetic classification for midbrain-hindbrain malformations.

Authors:  A James Barkovich; Kathleen J Millen; William B Dobyns
Journal:  Brain       Date:  2009-12       Impact factor: 13.501

9.  De novo germline and postzygotic mutations in AKT3, PIK3R2 and PIK3CA cause a spectrum of related megalencephaly syndromes.

Authors:  Jean-Baptiste Rivière; Ghayda M Mirzaa; Brian J O'Roak; Margaret Beddaoui; Diana Alcantara; Robert L Conway; Judith St-Onge; Jeremy A Schwartzentruber; Karen W Gripp; Sarah M Nikkel; Thea Worthylake; Christopher T Sullivan; Thomas R Ward; Hailly E Butler; Nancy A Kramer; Beate Albrecht; Christine M Armour; Linlea Armstrong; Oana Caluseriu; Cheryl Cytrynbaum; Beth A Drolet; A Micheil Innes; Julie L Lauzon; Angela E Lin; Grazia M S Mancini; Wendy S Meschino; James D Reggin; Anand K Saggar; Tally Lerman-Sagie; Gökhan Uyanik; Rosanna Weksberg; Birgit Zirn; Chandree L Beaulieu; Jacek Majewski; Dennis E Bulman; Mark O'Driscoll; Jay Shendure; John M Graham; Kym M Boycott; William B Dobyns
Journal:  Nat Genet       Date:  2012-06-24       Impact factor: 38.330

10.  Increase in cerebellar volume in Cavalier King Charles Spaniels with Chiari-like malformation and its role in the development of syringomyelia.

Authors:  Thomas A Shaw; Imelda M McGonnell; Colin J Driver; Clare Rusbridge; Holger A Volk
Journal:  PLoS One       Date:  2012-04-10       Impact factor: 3.240

View more

北京卡尤迪生物科技股份有限公司 © 2022-2023.