Stem cell transplantation for Fanconi’s Anemia – pro

Fanconi anemia, or FA, is a rare, inherited blood disorder that leads to bone marrow failure. A variety of supportive treatments is available but stem cell transplantation is the only curative option (gene transfer therapies are still experimental).  The two year survival rate is around 70%. Alternative conditioning regimens using fludarabine and avoiding irradiation have shown promising results so far. For those patients without matched sibling stem cell source, the prognosis for unrelated donors is around 20–40% survival at two years and there is a higher risk of graft rejection in somatic mosaics.
Stem cell transplantation is generally accepted as medically appropriate.

M D Tischkowitz and S V Hodgson
Fanconi anaemia
J. Med. Genet., January 1, 2003; 40(1): 1 – 10.

M Ayas et al, Stem cell transplantation for patients with Fanconi anemia with low-dose cyclophosphamide and antithymocyte globulins without the use of radiation therapy
Bone Marrow Transplantation (2005) 35, 463–466.

Rackoff WD. Treatment of bone marrow failure. In: Owen J, ed. Fanconi anemia – standards for clinical care. Fanconi Anemia Research Fund Inc, 1902 Jefferson Street, Suite 2, Eugene, Oregon 97405, 1999:9–20.55.

Guardiola P, Socie G, Pasquini R, Dokal I, Ortega JJ, van Weel-Sipman M, Marsh J, Locatelli F, Souillet G, Cahn JY, Ljungman P, Miniero R, Shaw J, Vermylen C, Archimbaud E, Bekassy AN, Krivan G, Di Bartolomeo P, Bacigalupo AL, Gluckman E. Allogeneic stem cell transplantation for Fanconi anaemia. Severe Aplastic Anaemia Working Party of the EBMT and EUFAR. European Group for Blood and Marrow Transplantation. Bone Marrow Transplant1998;21(suppl 2):S24–7.59.

McCloy M, Almeida A, Daly P, Vulliamy T, Roberts IA, Dokal I. Fludarabine-based stem cell transplantation protocol for Fanconi’s anaemia in myelodysplastic transformation. Br J Haematol2001;112:427–9

MacMillan ML, Auerbach AD, Davies SM, Defor TE, Gillio A, Giller R, Harris R, Cairo M, Dusenbery K, Hirsch B, Ramsay NK, Weisdorf DJ, Wagner JE. Haematopoietic cell transplantation in patients with Fanconi anaemia using alternate donors: results of a total body irradiation dose escalation trial. Br J Haematol2000;109:121–9

Participate in our Forums

To ask questions or participate in a discussion, please visit our Forums. You must LOGIN to participate.

Help Us Help Others

You can become a Site Sponsor. Or you may wish to support our work with a Donation.

Focused Articles For You

Lay Portal