Latest Abstracts on DWS

Mol Genet Metab. 2003 Sep-Oct;80(1-2):36-53.

Human malformations of the midbrain and hindbrain: review and proposed classification scheme.

Parisi MA, Dobyns WB.

Department of Pediatrics, University of Washington, Seattle, WA 98195, USA. [email protected]

Although a great deal of interest in the genetics and etiology of cerebral, particularly forebrain, malformations has been generated in the past decade, relatively little is known about the basis of congenital malformations of the structures of the posterior fossa, namely the midbrain, cerebellum, pons, and medulla. In this review, we present a classification scheme for malformations of the midbrain and hindbrain based on their embryologic derivation, highlight four of the conditions associated with such abnormalities, and describe the genetics, prognosis, and recurrence risks for each. We describe several disorders in addition to Joubert syndrome with the distinctive radiologic sign known as the "molar tooth sign," comprised of midbrain and hindbrain malformations. We discuss Dandy-Walker malformation, its classical definition, and the surprisingly good outcome in the absence of other brain malformations. We consider the heterogeneous entity of cerebellar vermis hypoplasia and describe the recently identified gene associated with an X-linked form of this condition. Finally, the pontocerebellar hypoplasias are discussed in the context of their generally progressive degenerative and severe course, and the differential diagnosis is emphasized. We anticipate that as imaging technologies improve, differentiation of the various disorders should aid in efforts to identify the causative genes.

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Mol Genet Metab. 2003 Sep-Oct;80(1-2):54-65.

Development and malformations of the cerebellum in mice.

Chizhikov V, Millen KJ.

Department of Human Genetics, University of Chicago, 920 E 58th Street, CLSC 319, Chicago, IL 60637, USA.

The cerebellum is the primary motor coordination center of the CNS and is also involved in cognitive processing and sensory discrimination. Multiple cerebellar malformations have been described in humans, however, their developmental and genetic etiologies currently remain largely unknown. In contrast, there is extensive literature describing cerebellar malformations in the mouse. During the past decade, analysis of both spontaneous and gene-targeted neurological mutant mice has provided significant insight into the molecular and cellular mechanisms that regulate cerebellar development. Cerebellar development occurs in several distinct but interconnected steps. These include the establishment of the cerebellar territory along anterior-posterior and dorsal-ventral axes of the embryo, initial specification of the cerebellar cell types, their subsequent proliferation, differentiation and migration, and, finally, the interconnection of the cerebellar circuitry. Our understanding of the basis of these developmental processes is certain to provide insight into the nature of human cerebellar malformations

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J Neurol. 2003 Sep;250(9):1025-36.
Development and developmental disorders of the human cerebellum.

Ten Donkelaar HJ, Lammens M, Wesseling P, Thijssen HO, Renier WO.

321 Dept. of Neurology, University Medical Centre Nijmegen, 9101, 6500 HB, Nijmegen, The Netherlands, [email protected]

The human cerebellum develops over a long time, extending from the early embryonic period until the first postnatal years. This protracted development makes the cerebellum vulnerable to a broad spectrum of developmental disorders. The development of the cerebellum occurs in four basic steps: 1) characterization of the cerebellar territory at the midbrain-hindbrain boundary; 2) formation of two compartments for cell proliferation: first, the Purkinje cells and the deep cerebellar nuclei arise from the ventricular zone of the metencephalic alar plate; second, granule cell precursors are formed from a second compartment of proliferation, i. e. the upper rhombic lip; 3) inward migration of the granule cells: granule precursor cells form the external granular layer, from which (and continuing into the first postnatal year), granule cells migrate inwards to their definite position in the internal granular layer, and 4) formation of cerebellar circuitry and further differentiation. The precerebellar nuclei, i. e. the pontine nuclei and the inferior olive, arise from the lower rhombic lip. Developmental disorders of the cerebellum are often accompanied by malformations of the precerebellar nuclei. In this review the development of the cerebellum and some of its more frequent developmental disorders, such as the Dandy-Walker and related midline malformations, and the pontocerebellar hypoplasias, are discussed.

PMID: 14504962 [PubMed - in process]

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Childs Nerv Syst. 2003 Aug;19(7-8):484-9. Epub 2003 Jul 16.


Dandy-Walker malformation: prenatal diagnosis and prognosis.

Klein O, Pierre-Kahn A, Boddaert N, Parisot D, Brunelle F.

Service de Neurochirurgie Pediatrique, Hopital Necker Enfants-Malades, 149 rue de Sevres, 75743 Paris Cedex 15, France.

INTRODUCTION: The difficulty in prognosticating the clinical and intellectual outcome of fetuses presenting with a Dandy-Walker malformation (DWM) comes from the great variety of cystic, median, and retrocerebellar malformations that probably have nothing in common and the variability of the definitions given to these lesions. In addition, many of these lesions can mimic each other. A correct diagnosis cannot be made without a good quality MRI including sagittal views of the vermis and T2-weighted images. We limited the diagnosis of DWM to those malformations with all of the following features: 1) a large median posterior fossa cyst widely communicating with the fourth ventricle, 2) a small, rotated, raised cerebellar vermis, 3) an upwardly displaced tentorium, 4) an enlarged posterior fossa, 5) antero-laterally displaced but apparently normal cerebellar hemispheres, 6) a normal brain stem. If any one of the previous criteria were not met, the malformation was considered distinct from DWM. MATERIALS AND METHODS: The charts of 26 patients with DWMs (18 females and 8 males; median age 10.5 years) were reviewed retrospectively. The diagnosis of the malformation was made prenatally in 7 children and postnatally in the 19 others. All the patients had both one MRI including axial and sagittal views of the posterior fossa as well as T1- and T2-weighted sequences, and one neuro-psychological investigation. Syndromic DWMs and Dandy Walker variants were excluded from the study. MRIs were reviewed in a blinded manner looking for brain malformation or damage and studying with particular attention the anatomy of the vermis. Systemic malformations were also recorded. Developmental quotient (DQ) and intellectual quotient (IQ) were said to be normal when equal or greater than 85, and low when below this value. Statistical analysis was performed using a Fisher test to analyze the relationship between intellectual performances, vermis anatomy, ventricular size, brain anatomy, and associated malformations. RESULTS. On scrutiny of sagittal T2 sequences, the vermis, although constantly small, rotated, and pushed towards the tentorium presented as two distinct morphologies, leading us to distinguish two groups of patients. In the first group (n=21), the vermis presented with two fissures, three lobes, and a fastigium as in the normal situation. In this particular group, none of the patients had associated brain malformation and all but 2 were functioning normally. One of the 2 retarded children had a fragile X syndrome. The other had a severe periventricular leukomalacia due to prematurity, which, per se, was sufficient to account for mental delay. In the second group (n=5), the vermis was highly malformed, obviously dysplastic, presenting with only one fissure or no fissure at all. It was constantly associated with major brain anomalies, most often a complete corpus callosum agenesis. All the patients in this group were more or less severely retarded. Vermis anatomy in DWMs was statistically correlated to neurological and intellectual outcome. Is the vermis dysplasia responsible, in itself, for this poor outcome? No answer can be given from this series, because retardation was observed in children who always had both a severely dysplastic vermis and other brain malformations. No other patient-related factor was statistically correlated to the outcome, in particular, hydrocephalus and extracerebral malformations. CONCLUSION: We described two types of DWM. The most frequent is characterized by an isolated and partially agenetic vermis. This malformation is compatible with a normal life. The second type consists of a severely abnormally lobulated vermis and associated brain malformation. This malformation is always accompanied by mental retardation.

PMID: 12879343 [PubMed - in process]

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Neuroradiology. 2003 May;45(5):320-4. Epub 2003 Apr 08.


Intellectual prognosis of the Dandy-Walker malformation in children: the importance of vermian lobulation.

Boddaert N, Klein O, Ferguson N, Sonigo P, Parisot D, Hertz-Pannier L, Baraton J, Emond S, Simon I, Chigot V, Schmit P, Pierre-Kahn A, Brunelle F.

Paediatric Radiology Department, Hopital Necker Enfants-Malades, 149 rue de Sevres, 75015 Paris, France. [email protected]

Half of patients with the Dandy-Walker malformation (DWM) have normal intellectual development. We aimed to identify feature on MRI associated with good intellectual prognosis. We reviewed 20 patients with DWM diagnosed on MRI, mean age 14.6+/-9.9 years. We assessed their intellectual development and related it to the MRI features. We found two groups with a statistically different intellectual outcome. All 14 patients with normal intellectual development had a normal lobulation of the vermis, without supratentorial anomalies. Of the six patients with mental retardation, three had an abnormal vermis, together with dysgenesis of the corpus callosum. In the other three, there were normal vermian anatomy with associated anomalies. Normal lobulation of the vermis, in the absence of any supratentorial anomaly, appears to be a good prognostic factor in DWM. Preservation of cerebrocerebellar pathways and neonatal plasticity could explain the normal intellectual development. These findings might be useful in prenatal diagnosis.

PMID: 12682795 [PubMed - indexed for MEDLINE]

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Acta Neurochir (Wien). 2003 Jan;145(1):63-7.


Intraoperative direct neuroendoscopic observation of the aqueduct in Dandy-Walker malformation.

Kawaguchi T, Jokura H, Kusaka Y, Shirane R, Yoshimoto T.

Department of Neurosurgery, Tohoku University Graduate School of Medicine, Sendai, Japan.

A 3-month-old female infant with Dandy-Walker malformation manifesting as hydrocephalus was treated successfully by only ventriculoperitoneal shunting. A flexible neuroendoscope was used intraoperatively to confirm the patency of the aqueduct, i.e. communication of the ventricular system and the cyst in the posterior fossa. Direct confirmation of the patency of the aqueduct and cyst communication is valuable to select the shunt procedure in the treatment of Dandy-Walker malformation.

PMID: 12545264 [PubMed - indexed for MEDLINE]

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Semin Pediatr Neurol. 2002 Dec;9(4):320-34.


Malformations of the posterior fossa: current perspectives.

Niesen CE.

Division of Pediatric Neurology, Cedars-Sinai Medical Center, Los Angeles, CA 90048, USA.

Posterior fossa malformations are a special group of central nervous system anomalies that present during infancy with hypotonia, developmental delay, microcephaly, or hydrocephalus. Recent discoveries of the genetic and epigenetic factors that control hindbrain ontogenesis explain some of these disturbances in cerebellar development. A comprehensive classification of posterior fossa malformations is proposed with particular attention to Dandy-Walker malformation, Joubert syndrome, and other cerebellar hypoplasias. A rare form of cerebellar hypertrophy which caused repeated obstruction at the foramen magnum is recognized. The importance of the cerebellum in language, cognition, and brain growth is stressed.

Publication Types:

        Review

        Review, Tutorial


PMID: 12523556 [PubMed - indexed for MEDLINE]

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