Literature DB >> 29728875

Natural history and clinical characteristics of 50 patients with Wolfram syndrome.

Gema Esteban Bueno1, Dyanne Ruiz-Castañeda2, Javier Ruiz Martínez3, Manuel Romero Muñoz4, Pedro Carrillo Alascio5.   

Abstract

PURPOSE: To describe clinical characteristics of diabetes mellitus (DM) in a group of patients with Wolfram Syndrome (WS).
METHODS: Descriptive, cross-sectional observational design. The sample consisted of 50 patients diagnosed with WS. Clinical criteria contributing to WS diagnosis were analyzed: diabetes mellitus (DM), optic nerve atrophy (OA), sensorineural deafness, urological and neurological dysfunction, among others. These parameters were assessed according to their presence/absence, age of onset, and various clinical-analytical parameters.
RESULTS: All the patients studied presented DM and OA, with a mean age of onset of 5.4 ± .9 (1-14) years and 9 ± .9 (1-16) years, respectively. The remaining criteria were present with a variable frequency: 77% had diabetes insipidus, 66.7% auditory alterations, 77.8% neurogenic bladder, 61.1% neurological involvement, and 27.8% hypogonadism. A 16.7% of the patients had positive albuminuria (urinary albumin/creatinine ratio > 30 mg/g) and 72.2% had hyporreflexia. There were no significant differences in the age of diagnosis nor of the presence of different pathologies according to sex.
CONCLUSIONS: The early presence of a non-autoimmune insulin dependent DM, should alert us of an "infrequent" diabetes syndrome. Wolfram's presumptive diagnosis could be established if juvenile-onset DM occurs concomitantly with OA, and this visual impairment is not attributable to diabetic retinopathy. Despite the long period of evolution of DM and altered values of HbA1c, the prevalence of microvascular complications in the sample are low.

Entities:  

Keywords:  DIDMOAD; Diabetes mellitus; Neurodegeneration; Optic atrophy; Sensorineural deafness; Wolfram syndrome

Mesh:

Year:  2018        PMID: 29728875     DOI: 10.1007/s12020-018-1608-2

Source DB:  PubMed          Journal:  Endocrine        ISSN: 1355-008X            Impact factor:   3.633


  17 in total

1.  Morbidity and mortality in the Wolfram syndrome.

Authors:  B T Kinsley; M Swift; R H Dumont; R G Swift
Journal:  Diabetes Care       Date:  1995-12       Impact factor: 19.112

Review 2.  Endocrine and metabolic aspects of the Wolfram syndrome.

Authors:  Georgios Boutzios; Sarantis Livadas; Evangelos Marinakis; Nicole Opie; Frangiskos Economou; Evanthia Diamanti-Kandarakis
Journal:  Endocrine       Date:  2011-08       Impact factor: 3.633

Review 3.  The syndrome of diabetes insipidus, diabetes mellitus, optic atrophy, deafness, and other abnormalities (DIDMOAD-syndrome). Two affected sibs and a short review of the literature (98 cases).

Authors:  M Dreyer; H W Rüdiger; K Bujara; C Herberhold; J Kühnau; P Maack; H Bartelheimer
Journal:  Klin Wochenschr       Date:  1982-05-03

4.  Juvenile diabetes mellitus, optic atrophy, hearing loss, diabetes insipidus, atonia of the urinary tract and bladder, and other abnormalities (Wolfram syndrome). A review of 88 cases from the literature with personal observations on 3 new patients.

Authors:  C W Cremers; P G Wijdeveld; A J Pinckers
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5.  Microvascular diabetes complications in Wolfram syndrome (diabetes insipidus, diabetes mellitus, optic atrophy, and deafness [DIDMOAD]): an age- and duration-matched comparison with common type 1 diabetes.

Authors:  Aline Cano; Laurent Molines; René Valéro; Gilbert Simonin; Véronique Paquis-Flucklinger; Bernard Vialettes
Journal:  Diabetes Care       Date:  2007-05-29       Impact factor: 19.112

6.  Genetically programmed selective islet beta-cell loss in diabetic subjects with Wolfram's syndrome.

Authors:  A Karasik; C O'Hara; S Srikanta; M Swift; J S Soeldner; C R Kahn; R D Herskowitz
Journal:  Diabetes Care       Date:  1989-02       Impact factor: 19.112

7.  False diagnosis of type 1 diabetes mellitus and its complications in Wolfram syndrome--is it the reason for the low number of reported cases of this abnormality?

Authors:  Katarzyna Homa; Adam Stefański; Agnieszka Zmysłowska; Piotr Molęda; Marta Ewa Bryśkiewicz; Liliana Majkowska
Journal:  Endokrynol Pol       Date:  2014       Impact factor: 1.582

8.  A homozygous mutation in a novel zinc-finger protein, ERIS, is responsible for Wolfram syndrome 2.

Authors:  Sami Amr; Cindy Heisey; Min Zhang; Xia-Juan Xia; Kathryn H Shows; Kamel Ajlouni; Arti Pandya; Leslie S Satin; Hatem El-Shanti; Rita Shiang
Journal:  Am J Hum Genet       Date:  2007-08-20       Impact factor: 11.025

9.  [DIDMOAD syndrome. Study of 3 families with 5 new cases. Differentiation from classic insulin-dependent diabetes mellitus].

Authors:  P P García-Luna; A Leal Cerro; F Villamil; I Gavilán; E Navarro; I Wichmann; A Núñez-Roldán; R Astorga
Journal:  Med Clin (Barc)       Date:  1985-10-19       Impact factor: 1.725

Review 10.  Wolfram Syndrome: Diagnosis, Management, and Treatment.

Authors:  Fumihiko Urano
Journal:  Curr Diab Rep       Date:  2016-01       Impact factor: 4.810

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  3 in total

1.  Wolfram syndrome: clinical and genetic profiling of a cohort from a tertiary care centre with characterization of the primary gonadal failure.

Authors:  Liza Das; Ashutosh Rai; Ravimohan Mavuduru; Kim Vaiphei; Akhilesh Sharma; Vishali Gupta; Sanjay Kumar Bhadada; Sailesh Lodha; Naresh Panda; Anil Bhansali; Paramjeet Singh; Pinaki Dutta
Journal:  Endocrine       Date:  2020-04-29       Impact factor: 3.633

2.  Genetic Spectrum of Neonatal Diabetes.

Authors:  M Kocova
Journal:  Balkan J Med Genet       Date:  2021-03-23       Impact factor: 0.519

3.  Early Intervention and Lifelong Treatment with GLP1 Receptor Agonist Liraglutide in a Wolfram Syndrome Rat Model with an Emphasis on Visual Neurodegeneration, Sensorineural Hearing Loss and Diabetic Phenotype.

Authors:  Toomas Jagomäe; Kadri Seppa; Riin Reimets; Marko Pastak; Mihkel Plaas; Miriam A Hickey; Kaia Grete Kukker; Lieve Moons; Lies De Groef; Eero Vasar; Allen Kaasik; Anton Terasmaa; Mario Plaas
Journal:  Cells       Date:  2021-11-16       Impact factor: 6.600

  3 in total

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