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Non-Myeloablative Stem Cell Transplants

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

Bone Marrow Transplantation volume 31, pages 437–440 (2003)Cite this article

Summary:

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.

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Acknowledgements

This work was partly supported by grants from AIRC (Associazione Italiana Ricerca sul Cancro), CNR (Consiglio Nazionale delle Ricerche), MURST (Ministero dell’Università e della Ricerca Scientifica e Tecnologica) and IRCCS (Istituto di Ricovero e Cura a Carattere Scientifico) Policlinico S. Matteo to FL.

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Authors and Affiliations

  1. Oncoematologia Pediatrica, IRCCS Policlinico San Matteo, Pavia, Italy

    G Rossi, G Giorgiani, P Comoli, P De Stefano, R Maccario & F Locatelli

  2. Clinica Pediatrica, I° Policlinico, IIa Università di Napoli, Pavia, Italy

    B Nobili

  3. Servizio Trasfusionale e di Immunoematologia, IRCCS Policlinico San Matteo, Pavia, Italy

    L Salvaneschi

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  1. G Rossi
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  8. F Locatelli
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Rossi, G., Giorgiani, G., Comoli, P. et al. 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. Bone Marrow Transplant 31, 437–440 (2003). https://doi.org/10.1038/sj.bmt.1703903

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