Summary:
A fludarabine-based protocol (fludarabine (25 mg/m2/day × 6 days), cyclophosphamide (10 mg/kg/day × 2 days) and ATG (ATGAM 10 mg/kg/day × 4 days)) was used in four multiply transfused Fanconi's anemia (FA) patients aged 5–15 years to reduce rejection during allogeneic bone marrow transplantation (BMT). Graft-versus-host disease (GVHD) prophylaxis consisted of cyclosporine and mini methotrexate. The graft source was G-CSF-stimulated bone marrow or peripheral blood stem cells (PBSC) in two patients each. All patients engrafted with median time to ANC>500/mm3 being 14 days (range: 12–17) and unsupported platelet count >20 ,000/mm3 being 13 days (range: 11–18). One patient had secondary graft rejection on day 56 and expired on day 69 due to fungal pneumonia. One patient who developed acute myeloid leukemia on day 56 underwent successful induction with cytosine and daunorubicin followed by peripheral blood stem cell (PBSC) rescue on day 70 and is presently in remission with complete donor chimerism and grade I GVHD. At a median follow-up of 13 months (range: 4–21), three patients (75%) are well with complete donor chimerism. Addition of fludarabine to the conditioning regimen for BMT in FA can provide additional immunosuppression for engraftment without increasing toxicity.
This is a preview of subscription content, access via your institution
Access options
Subscribe to this journal
Receive 12 print issues and online access
$259.00 per year
only $21.58 per issue
Buy this article
- Purchase on Springer Link
- Instant access to full article PDF
Prices may be subject to local taxes which are calculated during checkout
Similar content being viewed by others
References
Kohli-Kumar M, Morris C, DeLaat C et al. Bone marrow transplantation in Fanconi anemia using matched sibling donors. Blood 1994; 84: 2050–2054.
Storb R, Prentice RL, Thomas ED . Marrow transplantation for treatment of aplastic anemia. An analysis of factors associated with graft rejection. N Engl J Med 1977; 296: 61–66.
Kapelushnik J, Or R, Slavin S et al. A fludarabine based protocol for bone marrow transplantation in Fanconi's anemia. Bone Marrow Transplant 1997; 20: 1109–1110.
Aker M, Varadi G, Slavin S et al. Fludarabine based protocol for human umbilical cord blood transplantation in children with Fanconi's anemia. J Pediatr Hematol Oncol 1999; 21: 237–239.
Boulad F, Gillio A, Small TN et al. Stem cell transplantation for the treatment of Fanconi anemia using a fludarabine based cytoreductive regimen and T-cell depleted related HLA mismatched peripheral blood stem cell grafts. Br J Haematol 2000; 111: 1153–1157.
McCloy M, Almeida A, Daly P et al. Fludarabine based stem cell transplantation protocol for Fanconi's anemia in myelodysplastic transformation. Br J Haematol 2001; 112: 427–429.
Rossi G, Giorgiani G, Comoli P et al. Successful T-cell depleted, related haploidentical peripheral blood stem cell transplantation in a patient with Fanconi anemia using a fludarabine based preparative regimen without radiation. Bone Marrow Transplant 2003; 31: 437–440.
Kurre P, Pulsipher M, Woolfrey A et al. Reduced toxicity and prompt engraftment after minimal conditioning of a patient with Fanconi anemia undergoing hematopoietic stem cell transplantation from a HLA-matched unrelated donor. J Pediatr Hematol Oncol 2003; 25: 581–583.
Boyer MW, Gross TG, Loechelt B et al. Low risk of graft versus host disease with transplantation of CD34 selected peripheral blood progenitor cells from alternative donors for Fanconi anemia. J Pediatr Hematol Oncol 2003; 25: 890–895.
Guardiola P, Kurre P, Vlad A et al. Effective graft-versus-host effect after allogeneic stem cell transplantation using reduced-intensity preparative regimens in Fanconi anemia patients with myelodysplastic syndrome or acute myeloid leukemia. Br J Haematol 2003; 122: 806–809.
Author information
Authors and Affiliations
Corresponding author
Rights and permissions
About this article
Cite this article
George, B., Mathews, V., Shaji, R. et al. Fludarabine-based conditioning for allogeneic stem cell transplantation for multiply transfused patients with Fanconi's anemia. Bone Marrow Transplant 35, 341–343 (2005). https://doi.org/10.1038/sj.bmt.1704785
Received:
Accepted:
Published:
Issue Date:
DOI: https://doi.org/10.1038/sj.bmt.1704785
Keywords
This article is cited by
-
Fludarabine and neurotoxicity in engineered T-cell therapy
Gene Therapy (2018)
-
Population pharmacokinetics of fludarabine in patients with aplastic anemia and Fanconi anemia undergoing allogeneic hematopoietic stem cell transplantation
Bone Marrow Transplantation (2017)
-
Successful allo-HSCT with a minimal myeloablative conditioning regimen in an adult patient with Fanconi’s anemia
Bone Marrow Transplantation (2012)
-
Allogeneic stem cell transplantation in Fanconi anemia patients presenting with myelodysplasia and/or clonal abnormality: update on the Saudi experience
Bone Marrow Transplantation (2008)
-
Stem cell transplantation in India
Bone Marrow Transplantation (2008)