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  1. Al-Herz W, Zainal M, Nanda A
    Front Immunol, 2021;12:751469.
    PMID: 34659256 DOI: 10.3389/fimmu.2021.751469
    Background and Objectives: Reports on skin manifestations in inborn errors of immunity (IEI) are based on retrospective analysis, small series, or isolated case reports. The present prospective study aimed to determine the spectrum of skin manifestations in children with IEI and their relevance to specific molecular defects.

    Materials and Methods: The data were obtained from the Kuwait National Primary Immunodeficiency Disorders Registry during the period of 2004-2020.

    Results: A total of 313 pediatric cases of IEI, 71% diagnosed at molecular level, were registered with a cumulative follow-up period of 29,734 months. Skin manifestations were seen in 40.3% of the patients, and they were among the presenting manifestations in 33%. Patients with skin manifestations were older at both onset and diagnosis ages of IEI symptoms, but this was statistically significant for the latter only. The diagnosis delay was significantly longer in patients with skin manifestations. There was a statistically significant association between having skin manifestations and IEI category, being more common in patients with complement deficiencies, combined immunodeficiencies, and diseases of immune dysregulation. There was no statistically significant association between having skin manifestations and both gender and survival. Skin infections were the most frequent manifestations followed by eczema and autoimmune associations. Among IEI with more than 10 cases, skin lesions were a consistent finding in dedicator of cytokinesis 8 (DOCK8) deficiency, hyper IgE syndrome, ataxia-telangiectasia, and recombination activation gene (RAG)1 deficiency.

    Conclusions: Skin manifestations are common in IEI patients, and they had significant diagnosis delay and referral to specialists. Improvement of awareness about IEI is needed among pediatricians and dermatologists.

    Matched MeSH terms: Genetic Diseases, Inborn/epidemiology*
  2. Ganapaty S, Koo CW, Lo YC
    Mayo Clin Proc, 2024 Jun;99(6):953-954.
    PMID: 38691071 DOI: 10.1016/j.mayocp.2024.02.001
    Matched MeSH terms: Genetic Diseases, Inborn
  3. Tan EC, Loh M, Chuon D, Lim YP
    Hum Mutat, 2006 Mar;27(3):232-5.
    PMID: 16429432
    There is a need for country/population-specific databases because the existence of population-specific mutations for single gene disorders is well documented, and there is also good evidence for ethnic differences in the frequencies of genetic variations involved in complex disorders. Thus the Singapore Human Mutation/Polymorphism Database (SHMPD) was created to provide clinicians and scientists access to a central genetic database for the Singapore population. The data catalogued in the database include mutations identified in Singapore for Mendelian diseases, and frequencies of polymorphisms that have been investigated in either healthy controls or samples associated with specific phenotypes. Data from journal articles identified by searches in PubMed and other online resources, and via personal communications with researchers were compiled and assembled into a single database. Genes are categorized alphabetically and are also searchable by name and disease. The information provided for each variant of the gene includes the protein encoded, phenotype association, gender, size, and ethnic origin of the sample, as well as the reported genotype and allele frequencies, and direct links to the corresponding abstracts on PubMed. Our database will facilitate molecular diagnosis of Mendelian disorders and improve study designs for complex traits. It will be useful not only for researchers in Singapore, but also for those in countries with similar ethnic backgrounds, such as China, Taiwan, Hong Kong, Indonesia, and Malaysia.
    Matched MeSH terms: Genetic Diseases, Inborn/ethnology*; Genetic Diseases, Inborn/genetics*
  4. Ch'ng LS, Bux SI, Liam CK, Rahman NA, Ho CY
    Korean J Radiol, 2013 Sep-Oct;14(5):859-62.
    PMID: 24043987 DOI: 10.3348/kjr.2013.14.5.859
    Pulmonary alveolar microlithiasis (PAM) is a rare chronic disease with paucity of symptoms in contrast to the imaging findings. We present a case of a 24-year-old Malay man having an incidental abnormal pre-employment chest radiograph of dense micronodular opacities giving the classical "sandstorm" appearance. High-resolution computed tomography of the lungs showed microcalcifications with subpleural cystic changes. Open lung biopsy showed calcospherites within the alveolar spaces. The radiological and histopathological findings were characteristic of PAM.
    Matched MeSH terms: Genetic Diseases, Inborn/diagnosis*; Genetic Diseases, Inborn/surgery
  5. Wong HB
    Med J Malaysia, 1985 Sep;40(3):153-64.
    PMID: 3916209
    The different methods of prenatal diagnosis are discussed with special reference to ultrasound scan, amniocentesis for cell culture with processing for chromosome study, biochemical analysis and DNA recombination analysis. Chorionic villi aspiration and fetoscopy are new methods which will enhance considerably the methods for prenatal diagnosis. With regard to chromosome study of amniotic cells, experience with 623 cases is reviewed. 2.7% demonstrated chromosome anomalies and of these Downs anomaly was the commonest. A large proportion of cases requesting for amniocentesis are Caucasians who represent only 2% of the population in Singapore, but 25% of the 440 requests were from Caucasions. The various problems associated with the different methods for prenatal deafness are discussed.
    Matched MeSH terms: Genetic Diseases, Inborn/diagnosis*
  6. Aghamohammadi A, Rezaei N, Yazdani R, Delavari S, Kutukculer N, Topyildiz E, et al.
    J Clin Immunol, 2021 08;41(6):1339-1351.
    PMID: 34052995 DOI: 10.1007/s10875-021-01053-z
    BACKGROUND: Inborn errors of immunity (IEIs) are a heterogeneous group of genetic defects of immunity, which cause high rates of morbidity and mortality mainly among children due to infectious and non-infectious complications. The IEI burden has been critically underestimated in countries from middle- and low-income regions and the majority of patients with IEI in these regions lack a molecular diagnosis.

    METHODS: We analyzed the clinical, immunologic, and genetic data of IEI patients from 22 countries in the Middle East and North Africa (MENA) region. The data was collected from national registries and diverse databases such as the Asian Pacific Society for Immunodeficiencies (APSID) registry, African Society for Immunodeficiencies (ASID) registry, Jeffrey Modell Foundation (JMF) registry, J Project centers, and International Consortium on Immune Deficiency (ICID) centers.

    RESULTS: We identified 17,120 patients with IEI, among which females represented 39.4%. Parental consanguinity was present in 60.5% of cases and 27.3% of the patients were from families with a confirmed previous family history of IEI. The median age of patients at the onset of disease was 36 months and the median delay in diagnosis was 41 months. The rate of registered IEI patients ranges between 0.02 and 7.58 per 100,000 population, and the lowest rates were in countries with the highest rates of disability-adjusted life years (DALY) and death rates for children. Predominantly antibody deficiencies were the most frequent IEI entities diagnosed in 41.2% of the cohort. Among 5871 patients genetically evaluated, the diagnostic yield was 83% with the majority (65.2%) having autosomal recessive defects. The mortality rate was the highest in patients with non-syndromic combined immunodeficiency (51.7%, median age: 3.5 years) and particularly in patients with mutations in specific genes associated with this phenotype (RFXANK, RAG1, and IL2RG).

    CONCLUSIONS: This comprehensive registry highlights the importance of a detailed investigation of IEI patients in the MENA region. The high yield of genetic diagnosis of IEI in this region has important implications for prevention, prognosis, treatment, and resource allocation.

    Matched MeSH terms: Genetic Diseases, Inborn/epidemiology*
  7. Goleg FA, Kong NC, Sahathevan R
    Int Urol Nephrol, 2014 Aug;46(8):1581-7.
    PMID: 24671275 DOI: 10.1007/s11255-014-0694-1
    PURPOSE: End-stage kidney disease (ESKD) is now a worldwide pandemic. In concert with this, ESKD in Libya has also increased exponentially in recent decades. This review aims to define the magnitude of and risks for this ESKD epidemic among Libyans as there is a dearth of published data on this subject.

    METHODS: A systematic review was conducted by searching PubMed, EMBASE and Google scholar databases to identify all relevant papers published in English from 2003 to 2012, using the following keywords: end stage, terminal, chronic, renal, kidney, risk factors, Arab, North Africa and Libya.

    RESULTS: In 2003, the reported incidence of ESKD and prevalence of dialysis-treated ESKD in Libya were the same at 200 per million population (pmp). In 2007, the prevalence of dialysis-treated ESKD was 350 pmp, but the true incidence of ESKD was not available. The most recent published WHO data in 2012 showed the incidence of dialysis-treated ESKD had risen to 282 pmp and the prevalence of dialysis-treated ESKD had reached 624 pmp. The leading causes of ESKD were diabetic kidney disease (26.5 %), chronic glomerulonephritis (21.1 %), hypertensive nephropathy (14.6 %) and congenital/hereditary disease (12.3 %). The total number of dialysis centers was 40 with 61 nephrologists. Nephrologist/internist to patient ratio was 1:40, and nurse to patient ratio was 1:3.7. Only 135 living-related kidney transplants had been performed between 2004 and 2007. There were no published data on most macroeconomic and renal service factors.

    CONCLUSIONS: ESKD is a major public health problem in Libya with diabetic kidney disease and chronic glomerulonephritis being the leading causes. The most frequent co-morbidities were hypertension, obesity and the metabolic syndrome. In addition to provision of RRT, preventive strategies are also urgently needed for a holistic integrated renal care system.

    Matched MeSH terms: Genetic Diseases, Inborn/complications
  8. Ram S, Siar CH
    Dent Update, 2004 Nov;31(9):557-9.
    PMID: 15612463
    Cannon's disease or white sponge naevus is a relatively rare genetically determined skin disorder. It is inherited as an autosomal dominant trait that displays a high degree of penetrance and expressivity. This article describes cases of Cannon's disease in a mother and her son.
    Matched MeSH terms: Genetic Diseases, Inborn/pathology*
  9. Beighton P
    S. Afr. Med. J., 1976 Jul 10;50(29):1125-8.
    PMID: 959924
    Certain uncommon genetic disorders occur relatively frequently in the various population groups of Southern Africa. Prominent among these are porphyria, colonic polyposis and sclerosteosis in the Afrikaner community, Huntington's chorea in the British, Gaucher's and Tay-Sachs diseases in the Jewish population, glucose-6-phosphate dehydrogenase deficiency (G-6-PD deficiency) and thalassaemia in the Greek community, various skeletal dysplasias in the Black group, lipoid proteinosis and cleidocranial dysostosis in the Cape Coloured population, diabetes mellitus in the Indian community and retinitis pigmentosa in the Tristan da Cunha islanders. In addition, 'private' syndromes have been encountered in virtually every group. Awareness of the ethnic distribution of unusual genetic conditions is of considerable practical importance during the differential diagnosis of obscure disease.
    Matched MeSH terms: Genetic Diseases, Inborn/epidemiology*
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