Outcomes of mismatched and unrelated donor hematopoietic stem cell transplantation in Fanconi anemia conditioned with chemotherapy only

M. M. Chao, J. S. Kuehl, G. Strauss, H. Hanenberg, D. Schindler, H. Neitzel, C. Niemeyer, I. Baumann, H. von Bernuth, J. Rascon, M. Nagy, M. Zimmermann, C. P. Kratz, W. Ebell

Research output: Contribution to journalArticle

12 Citations (Scopus)

Abstract

Fanconi anemia (FA) is a genomic instability syndrome associated with bone marrow failure, myelodysplastic syndrome (MDS), and/or acute myeloid leukemia (AML) requiring hematopoietic stem cell transplantation (HSCT) to restore normal hematopoiesis. Although low-intensity fludarabine-based preparative regimens without radiation confer excellent outcomes in FA HSCTs with HLA-matched sibling donors, outcomes for FA patients with alternative donors are less encouraging, albeit improving. We present our experience with 17 FA patients who completed mismatched related or unrelated donor HSCT using a non-radiation fludarabine-based preparative regimen at Charité University Medicine Berlin. All patients engrafted; however, one patient had unstable chimerism in the setting of multi-viral infections that necessitated a stem cell boost to revert to full donor chimerism. Forty-seven percent of patients developed grade I acute graft-verus-host disease (aGVHD). No grade II–IV aGVHD or chronic graft-versus-host disease of any severity occurred. At a median follow-up of 30 months, 88 % of patients are alive with normal hematopoiesis. Two patients died of infections 4 months post-transplantation. These results demonstrate that short-term outcomes for FA patients with mismatched and unrelated donor HSCTs can be excellent using chemotherapy only conditioning. Viral reactivation, however, was a major treatment-related complication.

Original languageEnglish (US)
JournalAnnals of Hematology
Volume94
Issue number8
DOIs
StatePublished - Apr 12 2015

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Fanconi Anemia
Unrelated Donors
Hematopoietic Stem Cell Transplantation
Drug Therapy
Chimerism
Hematopoiesis
Tissue Donors
Transplants
Genomic Instability
Myelodysplastic Syndromes
Graft vs Host Disease
Berlin
Virus Diseases
Acute Myeloid Leukemia
Siblings
Chronic Disease
Stem Cells
Transplantation
Medicine
Radiation

Keywords

  • Alternative donor HSCT
  • Fanconi anemia
  • Fludarabine-based conditioning
  • Non-radiation conditioning

ASJC Scopus subject areas

  • Hematology

Cite this

Outcomes of mismatched and unrelated donor hematopoietic stem cell transplantation in Fanconi anemia conditioned with chemotherapy only. / Chao, M. M.; Kuehl, J. S.; Strauss, G.; Hanenberg, H.; Schindler, D.; Neitzel, H.; Niemeyer, C.; Baumann, I.; von Bernuth, H.; Rascon, J.; Nagy, M.; Zimmermann, M.; Kratz, C. P.; Ebell, W.

In: Annals of Hematology, Vol. 94, No. 8, 12.04.2015.

Research output: Contribution to journalArticle

Chao, MM, Kuehl, JS, Strauss, G, Hanenberg, H, Schindler, D, Neitzel, H, Niemeyer, C, Baumann, I, von Bernuth, H, Rascon, J, Nagy, M, Zimmermann, M, Kratz, CP & Ebell, W 2015, 'Outcomes of mismatched and unrelated donor hematopoietic stem cell transplantation in Fanconi anemia conditioned with chemotherapy only', Annals of Hematology, vol. 94, no. 8. https://doi.org/10.1007/s00277-015-2370-7
Chao, M. M. ; Kuehl, J. S. ; Strauss, G. ; Hanenberg, H. ; Schindler, D. ; Neitzel, H. ; Niemeyer, C. ; Baumann, I. ; von Bernuth, H. ; Rascon, J. ; Nagy, M. ; Zimmermann, M. ; Kratz, C. P. ; Ebell, W. / Outcomes of mismatched and unrelated donor hematopoietic stem cell transplantation in Fanconi anemia conditioned with chemotherapy only. In: Annals of Hematology. 2015 ; Vol. 94, No. 8.
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