Low‐dose cyclophosphamide conditioning for haematopoietic cell transplantation from HLA‐matched related donors in patients with Fanconi anaemia

Summary Allogeneic haematopoietic cell transplantation (HCT) is effective therapy for Fanconi anaemia (FA). FA patients do not tolerate conditioning with 200 mg/kg of cyclophosphamide (Cy), typically used in aplastic anaemia. We previously published results of studies in which Cy doses were graduall...

Full description

Saved in:
Bibliographic Details
Published in:British journal of haematology 2005-07, Vol.130 (1), p.99-106
Main Authors: Zanis‐Neto, J., Flowers, M. E. D., Medeiros, C. R., Bitencourt, M. A., Bonfim, C. M., Setúbal, D. C., Funke, V., Sanders, J., Deeg, H. J., Kiem, H. P., Martin, P., Leisenring, W., Storb, R., Pasquini, R.
Format: Article
Language:English
Subjects:
Adolescent
Adult
Biological and medical sciences
Child
Child, Preschool
Chromosome fragility (bloom syndrome, ataxia telangiectasia, fanconi anemia, x-linked mental retardation...)
cyclophosphamide
Cyclophosphamide - administration & dosage
Drug Administration Schedule
Fanconi anaemia
Fanconi Anemia - drug therapy
Fanconi Anemia - immunology
Fanconi Anemia - surgery
Female
Graft vs Host Disease
haematopoietic cell transplantation
Hematologic and hematopoietic diseases
Hematology
Hematopoietic Stem Cell Transplantation
HLA‐matched related donors
Humans
Immunosuppressive Agents - administration & dosage
Infant
Infant, Newborn
Male
Medical genetics
Medical sciences
methotrexate
Middle Aged
Statistics, Nonparametric
Survival Analysis
Transplantation Conditioning - methods
Transplantation, Homologous
Citations: Items that this one cites
Items that cite this one
Online Access:Get full text
Tags: Add Tag
No Tags, Be the first to tag this record!
Description
Summary:Summary Allogeneic haematopoietic cell transplantation (HCT) is effective therapy for Fanconi anaemia (FA). FA patients do not tolerate conditioning with 200 mg/kg of cyclophosphamide (Cy), typically used in aplastic anaemia. We previously published results of studies in which Cy doses were gradually reduced from 200 to 100 mg/kg. Here we update results of the initial studies and report data on 30 new patients conditioned with Cy either at 80 mg/kg (n = 7) or at 60 mg/kg (n = 23), given over 4 days before HCT from human leucocyte antigen‐matched related donors. Methotrexate and cyclosporine were given for graft‐versus‐host disease (GVHD) prophylaxis. All seven patients given Cy at 80 mg/kg and 21 of 23 given Cy at 60 mg/kg had sustained engraftment, while two patients, both with clonal cytogenetics abnormalities, experienced graft failure. Grades 2–3 acute GVHD rates were 57% and 14% for patients given the higher and lower Cy doses, respectively (P = 0·001). Four patients given Cy at 80 mg/kg and 22 given Cy at 60 mg/kg were alive at a median of 47 (44–58) months and 16 (3–52) months, respectively. Cy at 60 mg/kg has acceptable toxicities, low rates of GVHD, and is sufficient for engraftment of related grafts in most FA patients.
ISSN:0007-1048
1365-2141
DOI:10.1111/j.1365-2141.2005.05549.x