Enzyme replacement therapy and monitoring for children with type 1 Gaucher disease: Consensus recommendations

被引:77
|
作者
Charrow, J
Andersson, HC
Kaplan, P
Kolodny, EH
Mistry, P
Pastores, G
Prakash-Cheng, A
Rosenbloom, BE
Scott, CR
Wappner, RS
Weinreb, NJ
机构
[1] Childrens Mem Hosp, Div Genet, Chicago, IL 60614 USA
[2] Northwestern Univ, Feinberg Sch Med, Dept Pediat, Chicago, IL 60611 USA
[3] Tulane Univ, Sch Med, Dept Pediat, New Orleans, LA 70118 USA
[4] Childrens Hosp Philadelphia, Dept Pediat, Philadelphia, PA 19104 USA
[5] Univ Penn, Philadelphia, PA 19104 USA
[6] NYU, Sch Med, Dept Neurol, New York, NY USA
[7] Yale Univ, Sch Med, Dept Med, New Haven, CT 06510 USA
[8] Mt Sinai Sch Med, Dept Human Genet, New York, NY USA
[9] Cedars Sinai Med Ctr, Dept Hematol & Oncol, Los Angeles, CA 90048 USA
[10] Univ Washington, Sch Med, Dept Pediat, Seattle, WA 98195 USA
[11] James Whitcomb Riley Hosp Children, Dept Pediat, Indianapolis, IN 46202 USA
[12] James Whitcomb Riley Hosp Children, Dept Med & Mol Genet, Indianapolis, IN 46202 USA
[13] Indiana Univ, Indianapolis, IN 46204 USA
[14] Univ Hosp, Dept Hematol, Tamarac, FL USA
来源
JOURNAL OF PEDIATRICS | 2004年 / 144卷 / 01期
关键词
D O I
10.1016/j.jpeds.2003.10.067
中图分类号
R72 [儿科学];
学科分类号
100202 ;
摘要
[No abstract available]
引用
收藏
页码:112 / 120
页数:9
相关论文
共 50 条
  • [41] Scintigraphic monitoring of enzyme replacement therapy in patients with Gaucher's disease.
    Lorberboym, M
    Pastores, G
    Kim, CK
    Hermann, G
    Glajchen, N
    Machac, J
    JOURNAL OF NUCLEAR MEDICINE, 1996, 37 (05) : 1325 - 1325
  • [42] IMAGING FINDINGS OF THE OUTCOME OF ENZYME REPLACEMENT THERAPY IN ROMANIAN PATIENTS WITH TYPE 1 GAUCHER DISEASE
    Denes, C. L.
    Munteanu, D.
    Popita, V.
    Drugan, C.
    Grigorescu-Sido, P.
    CLINICAL THERAPEUTICS, 2009, 31 : S201 - S201
  • [43] Quality of life of 22 patients with type 1 Gaucher disease after enzyme replacement therapy
    李红敏
    China Medical Abstracts(Internal Medicine), 2020, 37 (02) : 71 - 72
  • [44] Re: The budget impact of enzyme replacement therapy in type 1 gaucher disease in the United States
    Rochmann, Camille
    Fournier, Marie
    Hull, Judy
    Perichon, Maria
    Pulikottil-Jacob, Ruth
    JOURNAL OF MEDICAL ECONOMICS, 2023, 26 (01) : 627 - 629
  • [45] Initiation of enzyme replacement therapy for an adult patient with asymptomatic type 1 Gaucher's disease
    Tanaka, N
    Saito, H
    Ito, T
    Momose, K
    Ishida, F
    Hora, K
    Kiyosawa, K
    Ida, H
    INTERNAL MEDICINE, 2001, 40 (08) : 716 - 721
  • [46] The effect of enzyme replacement therapy with imiglucerase on bone mineral density in type 1 Gaucher disease
    Wenstrup, R.
    Kaplan, P.
    Pastores, G.
    Prakash-Cheng, A.
    Zimran, A.
    Hangartner, T.
    CLINICAL THERAPEUTICS, 2007, 29 : S117 - S117
  • [47] CLINICAL AND ECONOMIC ASPECTS OF ENZYME REPLACEMENT THERAPY IN RUSSIAN PATIENTS WITH GAUCHER DISEASE TYPE 1
    Ryazhenov, V. V.
    Gorokhova, S. G.
    Gorokhov, V
    VALUE IN HEALTH, 2018, 21 : S444 - S444
  • [48] Effect of enzyme replacement therapy on bone mineral density in type 1 Gaucher disease.
    Wenstrup, R
    Kacena, K
    Kaplan, P
    Pastores, G
    Prakash-Cheng, A
    Zimran, A
    Hangartner, T
    BLOOD, 2005, 106 (11) : 54B - 54B
  • [49] Velaglucerase alfa, a human recombinant glucocerebrosidase enzyme replacement therapy for type 1 Gaucher disease
    Pastores, Gregory M.
    CURRENT OPINION IN INVESTIGATIONAL DRUGS, 2010, 11 (04) : 472 - 478
  • [50] Enzyme replacement therapy dose-response relationships in patients with type 1 Gaucher disease
    vom Dahl, S.
    Grabowski, G. A.
    Charrow, J.
    Hollak, C. E. M.
    Kacena, K.
    Mistry, P. K.
    Zhang, L.
    Zimran, A.
    HAEMATOLOGICA-THE HEMATOLOGY JOURNAL, 2007, 92 : 274 - 274