Fludarabine-based stem cell transplantation protocol for Fanconi's anaemia in myelodysplastic transformation

M. McCloy; A. Almeida; P. Daly; T. Vulliamy; I. A.G. Roberts; I. Dokal

doi:10.1046/j.1365-2141.2001.02585.x

Fludarabine-based stem cell transplantation protocol for Fanconi's anaemia in myelodysplastic transformation

M. McCloy^*, A. Almeida, P. Daly, T. Vulliamy, I. A.G. Roberts, I. Dokal

^*Corresponding author for this work

Research output: Contribution to journal › Article › peer-review

21 Citations (Scopus)

Abstract

Allogeneic stem cell transplantation (SCT) represents the treatment of choice for severe bone marrow (BM) failure in patients with Fanconi's anaemia (FA). However, for FA patients developing leukaemic or myelodysplastic transformation, the results of SCT are much less encouraging. We present a 1 7-year-old girl with myelodysplastic transformation of FA (refractory anaemia with excess blasts) and oculocutaneous albinism, who was treated by sibling SCT using conditioning with fludarabine, cyclophosphamide (CY) and anti-lymphocyte globulin (ALG). She had rapid engraftment with no toxicity and no graft-versus-host disease (GVHD). Twenty-two months after SCT, she had 100% donor chimaerism on Southern blot analysis.

Original language	English
Pages (from-to)	427-429
Number of pages	3
Journal	British Journal of Haematology
Volume	112
Issue number	2
DOIs	https://doi.org/10.1046/j.1365-2141.2001.02585.x
Publication status	Published - 2001
Externally published	Yes

Keywords

Fanconi's anaemia
Fludarabine
Myelodysplasia
Stem cell transplantation

Access to Document

10.1046/j.1365-2141.2001.02585.xLicence: Other

Cite this

@article{6de0e543e0264b2e949c11ed947b6934,

title = "Fludarabine-based stem cell transplantation protocol for Fanconi's anaemia in myelodysplastic transformation",

abstract = "Allogeneic stem cell transplantation (SCT) represents the treatment of choice for severe bone marrow (BM) failure in patients with Fanconi's anaemia (FA). However, for FA patients developing leukaemic or myelodysplastic transformation, the results of SCT are much less encouraging. We present a 1 7-year-old girl with myelodysplastic transformation of FA (refractory anaemia with excess blasts) and oculocutaneous albinism, who was treated by sibling SCT using conditioning with fludarabine, cyclophosphamide (CY) and anti-lymphocyte globulin (ALG). She had rapid engraftment with no toxicity and no graft-versus-host disease (GVHD). Twenty-two months after SCT, she had 100% donor chimaerism on Southern blot analysis.",

keywords = "Fanconi's anaemia, Fludarabine, Myelodysplasia, Stem cell transplantation",

author = "M. McCloy and A. Almeida and P. Daly and T. Vulliamy and Roberts, {I. A.G.} and I. Dokal",

year = "2001",

doi = "10.1046/j.1365-2141.2001.02585.x",

language = "English",

volume = "112",

pages = "427--429",

journal = "British Journal of Haematology",

issn = "0007-1048",

publisher = "John Wiley and Sons Inc.",

number = "2",

}

TY - JOUR

T1 - Fludarabine-based stem cell transplantation protocol for Fanconi's anaemia in myelodysplastic transformation

AU - McCloy, M.

AU - Almeida, A.

AU - Daly, P.

AU - Vulliamy, T.

AU - Roberts, I. A.G.

AU - Dokal, I.

PY - 2001

Y1 - 2001

N2 - Allogeneic stem cell transplantation (SCT) represents the treatment of choice for severe bone marrow (BM) failure in patients with Fanconi's anaemia (FA). However, for FA patients developing leukaemic or myelodysplastic transformation, the results of SCT are much less encouraging. We present a 1 7-year-old girl with myelodysplastic transformation of FA (refractory anaemia with excess blasts) and oculocutaneous albinism, who was treated by sibling SCT using conditioning with fludarabine, cyclophosphamide (CY) and anti-lymphocyte globulin (ALG). She had rapid engraftment with no toxicity and no graft-versus-host disease (GVHD). Twenty-two months after SCT, she had 100% donor chimaerism on Southern blot analysis.

AB - Allogeneic stem cell transplantation (SCT) represents the treatment of choice for severe bone marrow (BM) failure in patients with Fanconi's anaemia (FA). However, for FA patients developing leukaemic or myelodysplastic transformation, the results of SCT are much less encouraging. We present a 1 7-year-old girl with myelodysplastic transformation of FA (refractory anaemia with excess blasts) and oculocutaneous albinism, who was treated by sibling SCT using conditioning with fludarabine, cyclophosphamide (CY) and anti-lymphocyte globulin (ALG). She had rapid engraftment with no toxicity and no graft-versus-host disease (GVHD). Twenty-two months after SCT, she had 100% donor chimaerism on Southern blot analysis.

KW - Fanconi's anaemia

KW - Fludarabine

KW - Myelodysplasia

KW - Stem cell transplantation

UR - http://www.scopus.com/inward/record.url?scp=0035127488&partnerID=8YFLogxK

U2 - 10.1046/j.1365-2141.2001.02585.x

DO - 10.1046/j.1365-2141.2001.02585.x

M3 - Article

C2 - 11167843

AN - SCOPUS:0035127488

SN - 0007-1048

VL - 112

SP - 427

EP - 429

JO - British Journal of Haematology

JF - British Journal of Haematology

IS - 2

ER -

Fludarabine-based stem cell transplantation protocol for Fanconi's anaemia in myelodysplastic transformation

Abstract

Keywords

Access to Document

Other files and links

Fingerprint

Cite this