Non-Myeloablative Stem Cell Transplants
Bone Marrow Transplantation (2003) 31, 437–440. doi:10.1038/sj.bmt.1703903
Successful T-cell-depleted, related haploidentical peripheral blood stem cell transplantation in a patient with Fanconi anaemia using a fludarabine-based preparative regimen without radiation
G Rossi1, G Giorgiani1, P Comoli1, B Nobili2, L Salvaneschi3, P De Stefano1, R Maccario1 and F Locatelli1
- 1Oncoematologia Pediatrica, IRCCS Policlinico San Matteo, Pavia, Italy
- 2Clinica Pediatrica, I° Policlinico, IIa Università di Napoli, Pavia, Italy
- 3Servizio Trasfusionale e di Immunoematologia, IRCCS Policlinico San Matteo, Pavia, Italy
Correspondence: Dr F Locatelli, Oncoematologia Pediatrica, Università di Pavia, IRCCS Policlinico San Matteo, P.le Golgi 2, I-27100 Pavia, Italy
Received 7 August 2002; Accepted 7 November 2002.
Abstract
Haematopoietic stem cell transplantation (HSCT) represents the treatment of choice for severe bone marrow failure in patients with Fanconi anaemia (FA). When the donor is a compatible relative, the chance of being cured with an allograft is in the order of 70%. However, for FA children lacking an HLA-identical sibling, the results of HSCT from an alternative donor are less satisfactory because of a higher risk of graft rejection, graft-versus-host-disease (GVHD) and regimen-related toxicity. We report on a 12-year-old girl with FA, who was treated by T-cell-depleted (TCD) peripheral blood HSCT from her haploidentical uncle, using a novel fludarabine-based preparative regimen without radiation. She had rapid engraftment with no toxicity and no GVHD. Progressive recovery of both numbers of lymphocyte and of proliferative response to polyclonal activators occurred over time. At 18 months after transplantation, she is well with 100% donor chimerism and has recovered normal immune function.
Keywords:
Fanconi anaemia, haploidentical stem cell transplantation, adoptive immunotherapy, immune recovery
