Treatment of Fanconi anemia patients using fludarabine and low-dose TBI, followed by unrelated donor hematopoietic cell transplantation.

Abstract:

:A nonmyeloablative conditioning regimen consisting of fludarabine (FLU) and 2 Gy TBI has been used extensively and with substantial engraftment success without promoting excessive nonrelapse mortality in medically infirm patients requiring hematopoietic cell transplantation. In this paper, we studied this same low-toxicity regimen as a means of promoting engraftment of unrelated donor hematopoietic cell transplantation in patients with Fanconi anemia (FA). All patients tolerated the regimen well with no mucositis or other severe toxicities. Of six patients transplanted, five achieved stable mixed or full donor chimerism. Acute and chronic GVHD occurred in four and three patients, respectively. Three patients are alive and well at a median of 45.9 (range, 20.9-68.1) months after transplant. In summary, this FLU-based regimen facilitates stable engraftment of unrelated PBSCs, but is associated with significant chronic GVHD.

journal_name

Bone Marrow Transplant

authors

Thakar MS,Kurre P,Storb R,Kletzel M,Frangoul H,Pulsipher MA,Leisenring W,Flowers ME,Sandmaier BM,Woolfrey A,Kiem HP

doi

10.1038/bmt.2010.154

subject

Has Abstract

pub_date

2011-04-01 00:00:00

pages

539-44

issue

4

eissn

0268-3369

issn

1476-5365

pii

bmt2010154

journal_volume

46

pub_type

临床试验,杂志文章,多中心研究
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    doi:10.1038/bmt.2010.32

    authors: Locke FL,Artz A,Rich E,Zhang Y,van Besien K,Stock W

    更新日期:2010-12-01 00:00:00

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    更新日期:2006-05-01 00:00:00

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    pub_type: 杂志文章

    doi:

    authors: Jacobs HC,Falkenburg JH

    更新日期:1998-07-01 00:00:00

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    pub_type: 杂志文章,评审

    doi:10.1038/bmt.2009.214

    authors: Freytes CO,Lazarus HM

    更新日期:2009-11-01 00:00:00

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    authors: Ozyurek E,Cowan MJ,Koerper MA,Baxter-Lowe LA,Dvorak CC,Horn BN

    更新日期:2008-07-01 00:00:00

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    更新日期:2000-10-01 00:00:00

  • Pathology of the liver in mucopolysaccharidosis: light and electron microscopic assessment before and after bone marrow transplantation.

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    pub_type: 杂志文章

    doi:

    authors: Resnick JM,Krivit W,Snover DC,Kersey JH,Ramsay NK,Blazar BR,Whitley CB

    更新日期:1992-09-01 00:00:00

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    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2009.215

    authors: Thompson JS,Chu Y,Glass JF,Brown SA

    更新日期:2010-04-01 00:00:00

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    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1700995

    authors: Kingreen D,Nitsche A,Beyer J,Siegert W

    更新日期:1997-12-01 00:00:00

  • Matched-related allogeneic stem cell transplantation in Saudi patients with Fanconi anemia: 10 year's experience.

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    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2008.114

    authors: Ayas M,Al-Jefri A,Al-Seraihi A,Elkum N,Al-Mahr M,El-Solh H

    更新日期:2008-08-01 00:00:00

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    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1701158

    authors: Jackson SR,Tweeddale MG,Barnett MJ,Spinelli JJ,Sutherland HJ,Reece DE,Klingemann HG,Nantel SH,Fung HC,Toze CL,Phillips GL,Shepherd JD

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    pub_type: 杂志文章

    doi:10.1038/bmt.2012.267

    authors: Christopoulos P,Schmoor C,Waterhouse M,Marks R,Wäsch R,Bertz H,Finke J

    更新日期:2013-07-01 00:00:00

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    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1704172

    authors: Tanner ML,Hoh CK,Bashey A,Holman P,Sun C,Broome HE,Lane T,Ball ED,Carrier E

    更新日期:2003-09-01 00:00:00

  • European results of matched unrelated donor bone marrow transplantation for chronic myeloid leukemia. Impact of HLA class II matching. Chronic Leukemia Working Party of the European Group for Blood and Marrow Transplantation.

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    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1700844

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    更新日期:1997-07-01 00:00:00

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    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章

    doi:

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    pub_type: 杂志文章

    doi:

    authors: Lapidot T,Singer TS,Reisner Y

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    pub_type: 杂志文章

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    更新日期:2018-09-01 00:00:00

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    更新日期:2006-02-01 00:00:00

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    pub_type: 杂志文章

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    更新日期:2001-12-01 00:00:00

  • Superior survival of blood and marrow stem cell recipients given maternal grafts over recipients given paternal grafts.

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    pub_type: 临床试验,杂志文章,随机对照试验

    doi:

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