Hematopoietic stem cell transplantation in childhood inherited bone marrow failure syndrome

被引：60

作者：

Gluckman, E. ^{[1
]}

Wagner, J. E. ^{[2
]}

机构：

[1] Univ Minnesota, Dept Hematol, Eurocord Hop, Minneapolis, MN USA

[2] Univ Minnesota, Blood & Marrow Transplant Program, Minneapolis, MN USA

来源：

BONE MARROW TRANSPLANTATION | 2008年 / 41卷 / 02期

关键词：

aplastic anemia; hematopoietic stem cell transplant; Fanconi anemia;

D O I：

10.1038/sj.bmt.1705960

中图分类号：

Q6 [生物物理学];

学科分类号：

071011 ;

摘要：

Aplastic anemia is a rare disease in children that is most commonly idiopathic and less often a hereditary disorder. Hereditary bone marrow failure (BMF) syndromes, however, should be considered both in children and in adults before any attempt at treatment. Precise diagnosis is important because it will modify prognostic treatment options and the results of bone marrow transplantation. In this review, we will report recent results of treatment of Fanconi anemia and other hereditary BMF syndromes.

引用

页码：127 / 132

页数：6

共 17 条

[1] Malignancies after marrow transplantation for aplastic anemia and Fanconi anemia: A joint Seattle and Paris analysis of results in 700 patients [J].

Deeg, HJ ;

Socie, G ;

Schoch, G ;

HenryAmar, M ;

Witherspoon, RP ;

Devergie, A ;

Sullivan, KM ;

Gluckman, E ;

Storb, R .

BLOOD, 1996, 87 (01) :386-392

[2] Shwachman-Diamond syndrome [J].

Dror, Y ;

Freedman, MH .

BRITISH JOURNAL OF HAEMATOLOGY, 2002, 118 (03) :701-713

[3] Results of unrelated cord blood transplant in Fanconi anemia patients: Risk factor analysis for engraftment and survival [J].

Gluckman, Eliane ;

Rocha, Vanderson ;

Lonescu, Irina ;

Bierings, Marc ;

Harris, Richard E. ;

Wagner, John ;

Kurtzberg, Joanne ;

Champagne, Martin A. ;

Bonfim, Carmem ;

Bittencourt, Marco ;

Darbyshire, Philip ;

Fernandez, Manuel-Nicolas ;

Locatelli, Franco ;

Pasquini, Ricardo .

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 2007, 13 (09) :1073-1082

[4] Successful hernatopoietic stem cell transplantation for Fanconi anemia from an unaffected HLA-genotype-identical sibling selected using preimplantation genetic diagnosis [J].

Grewal, SS ;

Kahn, JP ;

MacMillan, ML ;

Ramsay, NKC ;

Wagner, JE .

BLOOD, 2004, 103 (03) :1147-1151

[5] Successful bone marrow transplantation in a patient with DNA ligase IV deficiency and bone marrow failure [J].

Gruhn, Bernd ;

Seidel, Joerg ;

Zintl, Felix ;

Varon, Raymonda ;

Toennies, Holger ;

Neitzel, Heidemarie ;

Bechtold, Astrid ;

Hoehn, Holger ;

Schindler, Detlev .

ORPHANET JOURNAL OF RARE DISEASES, 2007, 2 (1)

[6] Acute graft-versus-host disease in patients with Fanconi anemia or acquired aplastic anemia undergoing bone marrow transplantation from HLA-identical sibling donors:: risk factors and influence on outcome [J].

Guardiola, P ;

Socié, G ;

Li, XX ;

Ribaud, P ;

Devergie, A ;

Espérou, H ;

Richard, P ;

Traineau, R ;

Janin, A ;

Gluckman, E .

BLOOD, 2004, 103 (01) :73-77

[7]

Guardiola P, 2000, BLOOD, V95, P422

[8] Nijmegen breakage syndrome diagnosed as Fanconi anaemia [J].

New, HV ;

Cale, CM ;

Tischkowitz, M ;

Jones, A ;

Telfer, P ;

Veys, P ;

D'Andrea, A ;

Mathew, CG ;

Hann, I .

PEDIATRIC BLOOD & CANCER, 2005, 44 (05) :494-499

[9] Unusual complications after bone marrow transplantation for dyskeratosis congenita [J].

Rocha, V ;

Devergie, A ;

Socié, G ;

Ribaud, P ;

Espérou, H ;

Parquet, N ;

Gluckman, E .

BRITISH JOURNAL OF HAEMATOLOGY, 1998, 103 (01) :243-248

[10] Cancer incidence in persons with Fanconi anemia [J].

Rosenberg, PS ;

Greene, MH ;

Alter, BP .

BLOOD, 2003, 101 (03) :822-826

← 1 2 →