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

M. S. Thakar; P. Kurre; R. Storb; M. Kletzel; H. Frangoul; M. A. Pulsipher; W. Leisenring; M. E.D. Flowers; B. M. Sandmaier; A. Woolfrey; H. P. Kiem

doi:10.1038/bmt.2010.154

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

M. S. Thakar, P. Kurre, R. Storb, M. Kletzel, H. Frangoul, M. A. Pulsipher, W. Leisenring, M. E.D. Flowers, B. M. Sandmaier, A. Woolfrey, H. P. Kiem^*

^*Corresponding author for this work

Pediatrics

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

16 Scopus citations

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.

Original language	English (US)
Pages (from-to)	539-544
Number of pages	6
Journal	Bone Marrow Transplantation
Volume	46
Issue number	4
DOIs	https://doi.org/10.1038/bmt.2010.154
State	Published - Apr 2011

Funding

We thank the patients and families enrolled in this study. We also thank our in-patient and outpatient clinical transplant teams for the substantial care of our patients, as well as the research nurses and data managers for coordination of this trial. We appreciate the help of Helen Crawford, Bonnie Larson and Sue Carbonneau for paper preparation. MST received salary support from the Fanconi Anemia Research Fund Inc. This trial was also supported in part by NIH/NHLBI HL36444 and CA15704.

Keywords

Fanconi anemia
HLA-matched unrelated donor transplant
fludarabine

ASJC Scopus subject areas

Transplantation
Hematology

Access to Document

10.1038/bmt.2010.154

Cite this

Thakar, M. S., Kurre, P., Storb, R., Kletzel, M., Frangoul, H., Pulsipher, M. A., Leisenring, W., Flowers, M. E. D., Sandmaier, B. M., Woolfrey, A., & Kiem, H. P. (2011). Treatment of Fanconi anemia patients using fludarabine and low-dose TBI, followed by unrelated donor hematopoietic cell transplantation. Bone Marrow Transplantation, 46(4), 539-544. https://doi.org/10.1038/bmt.2010.154

@article{2a932d69050849fab0619b2d0880c71d,

title = "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.",

keywords = "Fanconi anemia, HLA-matched unrelated donor transplant, fludarabine",

author = "Thakar, {M. S.} and P. Kurre and R. Storb and M. Kletzel and H. Frangoul and Pulsipher, {M. A.} and W. Leisenring and Flowers, {M. E.D.} and Sandmaier, {B. M.} and A. Woolfrey and Kiem, {H. P.}",

note = "Funding Information: We thank the patients and families enrolled in this study. We also thank our in-patient and outpatient clinical transplant teams for the substantial care of our patients, as well as the research nurses and data managers for coordination of this trial. We appreciate the help of Helen Crawford, Bonnie Larson and Sue Carbonneau for paper preparation. MST received salary support from the Fanconi Anemia Research Fund Inc. This trial was also supported in part by NIH/NHLBI HL36444 and CA15704.",

year = "2011",

month = apr,

doi = "10.1038/bmt.2010.154",

language = "English (US)",

volume = "46",

pages = "539--544",

journal = "Bone Marrow Transplantation",

issn = "0268-3369",

publisher = "Springer Nature",

number = "4",

}

Thakar, MS, Kurre, P, Storb, R, Kletzel, M, Frangoul, H, Pulsipher, MA, Leisenring, W, Flowers, MED, Sandmaier, BM, Woolfrey, A & Kiem, HP 2011, 'Treatment of Fanconi anemia patients using fludarabine and low-dose TBI, followed by unrelated donor hematopoietic cell transplantation', Bone Marrow Transplantation, vol. 46, no. 4, pp. 539-544. https://doi.org/10.1038/bmt.2010.154

TY - JOUR

T1 - Treatment of Fanconi anemia patients using fludarabine and low-dose TBI, followed by unrelated donor hematopoietic cell transplantation

AU - Thakar, M. S.

AU - Kurre, P.

AU - Storb, R.

AU - Kletzel, M.

AU - Frangoul, H.

AU - Pulsipher, M. A.

AU - Leisenring, W.

AU - Flowers, M. E.D.

AU - Sandmaier, B. M.

AU - Woolfrey, A.

AU - Kiem, H. P.

N1 - Funding Information: We thank the patients and families enrolled in this study. We also thank our in-patient and outpatient clinical transplant teams for the substantial care of our patients, as well as the research nurses and data managers for coordination of this trial. We appreciate the help of Helen Crawford, Bonnie Larson and Sue Carbonneau for paper preparation. MST received salary support from the Fanconi Anemia Research Fund Inc. This trial was also supported in part by NIH/NHLBI HL36444 and CA15704.

PY - 2011/4

Y1 - 2011/4

N2 - 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.

AB - 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.

KW - Fanconi anemia

KW - HLA-matched unrelated donor transplant

KW - fludarabine

UR - http://www.scopus.com/inward/record.url?scp=79953708240&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=79953708240&partnerID=8YFLogxK

U2 - 10.1038/bmt.2010.154

DO - 10.1038/bmt.2010.154

M3 - Article

C2 - 20581880

AN - SCOPUS:79953708240

SN - 0268-3369

VL - 46

SP - 539

EP - 544

JO - Bone Marrow Transplantation

JF - Bone Marrow Transplantation

IS - 4

ER -

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

Abstract

Funding

Keywords

ASJC Scopus subject areas

Access to Document

Other files and links

Fingerprint

Cite this