Is the perinatal lethal form of Gaucher disease more common than classic type 2 Gaucher disease?

被引:0
|
作者
Deborah L Stone
Otto P van Diggelen
Johannis BC de Klerk
Johannes LJ Gaillard
Martinus F Niermeijer
Rob Willemsen
Nahid Tayebi
Ellen Sidransky
机构
[1] Clinical Neuroscience Branch,Departments of Clinical Genetics
[2] Intramural Research Program,Department of Clinical Pathology
[3] National Institute of Mental Health,undefined
[4] National Institutes of Health,undefined
[5] University Hospital,undefined
[6] Erasmus University,undefined
[7] Pediatrics,undefined
[8] University Hospital,undefined
[9] Erasmus University,undefined
[10] St Clara Hospital,undefined
来源
关键词
Gaucher disease; lysosomal storage disorder; hydrops fetalis; collodion baby; mutation analysis;
D O I
暂无
中图分类号
学科分类号
摘要
In recent years there has been increased recognition of a severe perinatal lethal form of Gaucher disease, the inherited deficiency of lysosomal glucocerebrosidase. We previously reported a case of severe type 2 Gaucher disease which was seen in a medical center in Rotterdam and now present three new cases from two other families seen at the same center. Mutational analyses of these cases revealed two novel mutations, H311R and V398F, located in exons 8 and 9, respectively. The identification of four cases of lethal type 2 Gaucher disease in a single center seems to be a function of increased awareness of this phenotype, rather than of geographic clustering. The actual incidence of lethal type 2 Gaucher disease may be underestimated, as many cases may have been misclassified as collodion babies or hydrops of unknown cause.
引用
收藏
页码:505 / 509
页数:4
相关论文
共 50 条
  • [1] Is the perinatal lethal form of Gaucher disease more common than classic type 2 Gaucher disease?
    Stone, DL
    van Diggelen, OP
    de Klerk, JBC
    Gaillard, JLJ
    Niermeijer, MF
    Willemsen, R
    Tayebi, N
    Sidransky, E
    EUROPEAN JOURNAL OF HUMAN GENETICS, 1999, 7 (04) : 505 - 509
  • [2] Phenotypic continuum of type 2 Gaucher's disease: an intermediate phenotype between perinatal-lethal and classic type 2 Gaucher's disease
    Ben Turkia, H.
    Tebib, N.
    Azzouz, H.
    Abdelmoula, M. S.
    Ben Chehida, A.
    Caillaud, C.
    Ben Dridi, M. F.
    JOURNAL OF PERINATOLOGY, 2009, 29 (02) : 170 - 172
  • [3] Phenotypic continuum of type 2 Gaucher's disease: an intermediate phenotype between perinatal-lethal and classic type 2 Gaucher's disease
    H Ben Turkia
    N Tebib
    H Azzouz
    M S Abdelmoula
    A Ben Chehida
    C Caillaud
    M F Ben Dridi
    Journal of Perinatology, 2009, 29 : 170 - 172
  • [4] Perinatal Lethal Gaucher Disease
    Plakkal, Nishad
    Soraisham, Amuchou Singh
    Jirapradittha, Junya
    Pinto-Rojas, Alfredo
    INDIAN JOURNAL OF PEDIATRICS, 2011, 78 (01): : 106 - 108
  • [5] Perinatal Lethal Gaucher Disease
    Nishad Plakkal
    Amuchou Singh Soraisham
    Junya Jirapradittha
    Alfredo Pinto-Rojas
    The Indian Journal of Pediatrics, 2011, 78 : 106 - 108
  • [6] Perinatal-lethal Gaucher disease
    Mignot, C
    Gelot, A
    Bessières, B
    Daffos, F
    Voyer, M
    Menez, F
    Bianco, CF
    Odent, S
    Le Duff, D
    Loget, P
    Fargier, P
    Costil, J
    Josset, P
    Roume, J
    Vanier, MT
    Maire, I
    de Villemeur, TB
    AMERICAN JOURNAL OF MEDICAL GENETICS PART A, 2003, 120A (03) : 338 - 344
  • [7] Perinatal lethal form of Gaucher's disease presenting with hemosiderosis
    Sharma, R
    Hudak, ML
    Perszyk, AA
    Premachandra, BR
    Li, HB
    Monteiro, C
    AMERICAN JOURNAL OF PERINATOLOGY, 2000, 17 (04) : 201 - 206
  • [8] Atypical features in a case of lethal perinatal Gaucher disease
    Frosk, P.
    Phillips, S. M.
    Del Bigio, M. R.
    Chodirker, B. N.
    NEUROPATHOLOGY AND APPLIED NEUROBIOLOGY, 2014, 40 (07) : 946 - 950
  • [9] Perinatal lethal form of Gaucher disease. Clinical and molecular characterization of a Greek case
    Michelakakis, Helen
    Dimitriou, Evangelia
    Moraitou, Marina
    Valari, Mantoula
    Mitsiadi, Vassiliki
    Yatrakou, Erasmia
    Karachristou, Korina
    Cozar, Monica
    Vilageliu, Lluisa
    Grinberg, Daniel
    BLOOD CELLS MOLECULES AND DISEASES, 2010, 44 (02) : 82 - 83
  • [10] Perinatal lethal Gaucher's disease without prenatal complications
    Zay, A.
    Choy, F. Y. M.
    MacLeod, P.
    Tan-Dy, C. R.
    CLINICAL GENETICS, 2008, 73 (02) : 191 - 195