Allogeneic Hematopoietic Stem Cell Transplantation for Treatment of Gaucher Disease

被引：11

作者：

Machaczka, Maciej ^{[1
,2
]}

机构：

[1] Karolinska Univ Hosp Huddinge, Hematol Ctr Karolinska, SE-14186 Stockholm, Sweden

[2] Karolinska Inst, Div Hematol, Dept Med, Karolinska Univ Hosp Huddinge, Stockholm, Sweden

来源：

PEDIATRIC HEMATOLOGY AND ONCOLOGY | 2013年 / 30卷 / 05期

关键词：

allogeneic; Gaucher disease; hematopoietic stem cell transplantation; outcome; BONE-MARROW TRANSPLANTATION; GLUCOCEREBROSIDASE GENE; NORRBOTTNIAN TYPE; EXPERIENCE; MUTATION;

D O I：

10.3109/08880018.2013.793757

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

Ito and Barrett recently reviewed the international experiences with allogeneic hematopoietic stem cell transplantation (allo-HSCT) for the treatment of the neuronopathic and non-neuronopathic forms of Gaucher disease (GD). In the present communication, the author adds some updated data on the long-term outcome of allo-HSCT for Swedish GD patients with the mean and median follow-up time of 27 years (the longest follow-up of 30 years) and makes comments on some of the authors' statements found in the article.

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收藏

页码：459 / 461

页数：3

相关论文

共 10 条

[1]

DAHL N, 1990, AM J HUM GENET, V47, P275

[2] GAUCHER DISEASE - NORRBOTTNIAN TYPE .1. GENERAL CLINICAL DESCRIPTION [J].

DREBORG, S ;

ERIKSON, A ;

HAGBERG, B .

EUROPEAN JOURNAL OF PEDIATRICS, 1980, 133 (02) :107-118

[3]

HOBBS JR, 1987, LANCET, V1, P1111

[4] Gaucher disease:: Mutation and polymorphism spectrum in the glucocerebrosidase gene (GBA) [J].

Hruska, Kathleen S. ;

LaMarca, Mary E. ;

Scott, C. Ronald ;

Sidransky, Ellen .

HUMAN MUTATION, 2008, 29 (05) :567-583

[5] Gauchers Disease-A Reappraisal of Hematopoietic Stem Cell Transplantation [J].

Ito, Sawa ;

Barrett, A. John .

PEDIATRIC HEMATOLOGY AND ONCOLOGY, 2013, 30 (02) :61-70

[6] Substrate reduction therapy with miglustat for type 1 Gaucher disease: A retrospective analysis from a single institution [J].

Machaczka, Maciej ;

Hast, Robert ;

Dahlman, Ingrid ;

Lerner, Richard ;

Klimkowska, Monika ;

Engvall, Martin ;

Hagglund, Hans .

UPSALA JOURNAL OF MEDICAL SCIENCES, 2012, 117 (01) :28-34

[7] LONG-TERM FOLLOW-UP OF THE 1ST SUCCESSFUL BONE-MARROW TRANSPLANTATION IN GAUCHER DISEASE [J].

RINGDEN, O ;

GROTH, CG ;

ERIKSON, A ;

BACKMAN, L ;

GRANQVIST, S ;

MANSSON, JE ;

SVENNERHOLM, L .

TRANSPLANTATION, 1988, 46 (01) :66-70

[8] Allogeneic hematopoietic stem cell transplantation for inherited disorders:: Experience in a single center [J].

Ringdén, O ;

Remberger, M ;

Svahn, BM ;

Barkholt, L ;

Mattsson, J ;

Aschan, J ;

Le Blanc, K ;

Gustafsson, B ;

Hassan, Z ;

Omazic, B ;

Svenberg, P ;

Solders, G ;

von Döbeln, U ;

Winiarski, J ;

Ljungman, P ;

Malm, G .

TRANSPLANTATION, 2006, 81 (05) :718-725

[9] 10 YEARS EXPERIENCE OF BONE-MARROW TRANSPLANTATION FOR GAUCHER DISEASE [J].

RINGDEN, O ;

GROTH, CG ;

ERIKSON, A ;

GRANQVIST, S ;

MANSSON, JE ;

SPARRELID, E .

TRANSPLANTATION, 1995, 59 (06) :864-870

[10] How I treat Gaucher disease [J].

Zimran, Ari .

BLOOD, 2011, 118 (06) :1463-1471