Transplant results in adults with Fanconi anaemia

EBMT SAA WP

Research output: Contribution to journalArticle

Abstract

The outcomes of adult patients transplanted for Fanconi anaemia (FA) have not been well described. We retrospectively analysed 199 adult patients with FA transplanted between 1991 and 2014. Patients were a median of 16 years of age when diagnosed with FA, and underwent transplantation at a median age of 23 years. Time between diagnosis and transplant was shortest (median 2 years) in those patients who had a human leucocyte antigen identical sibling donor. Fifty four percent of patients had bone marrow (BM) failure at transplantation and 46% had clonal disease (34% myelodysplasia, 12% acute leukaemia). BM was the main stem cell source, the conditioning regimen included cyclophosphamide in 96% of cases and fludarabine in 64%. Engraftment occurred in 82% (95% confidence interval [CI] 76-87%), acute graft-versus-host disease (GvHD) grade II-IV in 22% (95% CI 16-28%) and the incidence of chronic GvHD at 96 months was 26% (95% CI 20-33). Non-relapse mortality at 96 months was 56% with an overall survival of 34%, which improved with more recent transplants. Median follow-up was 58 months. Patients transplanted after 2000 had improved survival (84% at 36 months), using BM from an identical sibling and fludarabine in the conditioning regimen. Factors associated with improved outcome in multivariate analysis were use of fludarabine and an identical sibling or matched non-sibling donor. Main causes of death were infection (37%), GvHD (24%) and organ failure (12%). The presence of clonal disease at transplant did not significant impact on survival. Secondary malignancies were reported in 15 of 131 evaluable patients.

Original languageEnglish
Pages (from-to)100-109
Number of pages10
JournalBritish Journal of Haematology
Volume180
Issue number1
DOIs
Publication statusPublished - Jan 2018

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Fanconi Anemia
Transplants
Graft vs Host Disease
Siblings
Bone Marrow
Confidence Intervals
Survival
Transplantation
Tissue Donors
HLA Antigens
Cyclophosphamide
Cause of Death
Leukemia
Stem Cells
Multivariate Analysis
Mortality
Incidence
Infection

Keywords

  • Adolescent
  • Adult
  • Cause of Death
  • Fanconi Anemia/diagnosis
  • Graft vs Host Disease/etiology
  • Hematopoietic Stem Cell Transplantation/adverse effects
  • Humans
  • Middle Aged
  • Neoplasms, Second Primary/etiology
  • Prognosis
  • Retrospective Studies
  • Risk Factors
  • Survival Analysis
  • Tissue Donors
  • Transplantation Conditioning
  • Transplantation, Homologous
  • Treatment Outcome
  • Young Adult

Cite this

Transplant results in adults with Fanconi anaemia. / EBMT SAA WP.

In: British Journal of Haematology, Vol. 180, No. 1, 01.2018, p. 100-109.

Research output: Contribution to journalArticle

EBMT SAA WP. / Transplant results in adults with Fanconi anaemia. In: British Journal of Haematology. 2018 ; Vol. 180, No. 1. pp. 100-109.
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abstract = "The outcomes of adult patients transplanted for Fanconi anaemia (FA) have not been well described. We retrospectively analysed 199 adult patients with FA transplanted between 1991 and 2014. Patients were a median of 16 years of age when diagnosed with FA, and underwent transplantation at a median age of 23 years. Time between diagnosis and transplant was shortest (median 2 years) in those patients who had a human leucocyte antigen identical sibling donor. Fifty four percent of patients had bone marrow (BM) failure at transplantation and 46{\%} had clonal disease (34{\%} myelodysplasia, 12{\%} acute leukaemia). BM was the main stem cell source, the conditioning regimen included cyclophosphamide in 96{\%} of cases and fludarabine in 64{\%}. Engraftment occurred in 82{\%} (95{\%} confidence interval [CI] 76-87{\%}), acute graft-versus-host disease (GvHD) grade II-IV in 22{\%} (95{\%} CI 16-28{\%}) and the incidence of chronic GvHD at 96 months was 26{\%} (95{\%} CI 20-33). Non-relapse mortality at 96 months was 56{\%} with an overall survival of 34{\%}, which improved with more recent transplants. Median follow-up was 58 months. Patients transplanted after 2000 had improved survival (84{\%} at 36 months), using BM from an identical sibling and fludarabine in the conditioning regimen. Factors associated with improved outcome in multivariate analysis were use of fludarabine and an identical sibling or matched non-sibling donor. Main causes of death were infection (37{\%}), GvHD (24{\%}) and organ failure (12{\%}). The presence of clonal disease at transplant did not significant impact on survival. Secondary malignancies were reported in 15 of 131 evaluable patients.",
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N2 - The outcomes of adult patients transplanted for Fanconi anaemia (FA) have not been well described. We retrospectively analysed 199 adult patients with FA transplanted between 1991 and 2014. Patients were a median of 16 years of age when diagnosed with FA, and underwent transplantation at a median age of 23 years. Time between diagnosis and transplant was shortest (median 2 years) in those patients who had a human leucocyte antigen identical sibling donor. Fifty four percent of patients had bone marrow (BM) failure at transplantation and 46% had clonal disease (34% myelodysplasia, 12% acute leukaemia). BM was the main stem cell source, the conditioning regimen included cyclophosphamide in 96% of cases and fludarabine in 64%. Engraftment occurred in 82% (95% confidence interval [CI] 76-87%), acute graft-versus-host disease (GvHD) grade II-IV in 22% (95% CI 16-28%) and the incidence of chronic GvHD at 96 months was 26% (95% CI 20-33). Non-relapse mortality at 96 months was 56% with an overall survival of 34%, which improved with more recent transplants. Median follow-up was 58 months. Patients transplanted after 2000 had improved survival (84% at 36 months), using BM from an identical sibling and fludarabine in the conditioning regimen. Factors associated with improved outcome in multivariate analysis were use of fludarabine and an identical sibling or matched non-sibling donor. Main causes of death were infection (37%), GvHD (24%) and organ failure (12%). The presence of clonal disease at transplant did not significant impact on survival. Secondary malignancies were reported in 15 of 131 evaluable patients.

AB - The outcomes of adult patients transplanted for Fanconi anaemia (FA) have not been well described. We retrospectively analysed 199 adult patients with FA transplanted between 1991 and 2014. Patients were a median of 16 years of age when diagnosed with FA, and underwent transplantation at a median age of 23 years. Time between diagnosis and transplant was shortest (median 2 years) in those patients who had a human leucocyte antigen identical sibling donor. Fifty four percent of patients had bone marrow (BM) failure at transplantation and 46% had clonal disease (34% myelodysplasia, 12% acute leukaemia). BM was the main stem cell source, the conditioning regimen included cyclophosphamide in 96% of cases and fludarabine in 64%. Engraftment occurred in 82% (95% confidence interval [CI] 76-87%), acute graft-versus-host disease (GvHD) grade II-IV in 22% (95% CI 16-28%) and the incidence of chronic GvHD at 96 months was 26% (95% CI 20-33). Non-relapse mortality at 96 months was 56% with an overall survival of 34%, which improved with more recent transplants. Median follow-up was 58 months. Patients transplanted after 2000 had improved survival (84% at 36 months), using BM from an identical sibling and fludarabine in the conditioning regimen. Factors associated with improved outcome in multivariate analysis were use of fludarabine and an identical sibling or matched non-sibling donor. Main causes of death were infection (37%), GvHD (24%) and organ failure (12%). The presence of clonal disease at transplant did not significant impact on survival. Secondary malignancies were reported in 15 of 131 evaluable patients.

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