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| Outcome of 69 allogeneic stem cell transplantations for Fanconi anemia using HLA-matched unrelated donors: a study on behalf of the European Group for Blood and Marrow Transplantation |
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| Author(s): Guardiola P, Pasquini R, Dokal I, Ortega JJ, van Weel-Sipman M, Marsh JCW, Ball SE, Locatelli F, Vermylen C, Skinner R, Ljungman P, Miniero R, Shaw PJ, Souillet G, Michallet M, Bekassy AN, Krivan G, Di Bartolomeo P, Heilmann C, Zanesco L, Cahn JY, Arcese W, Bacigalupo A, Gluckman E |
| Source: BLOOD Volume: 95 Issue: 2 Pages: 422-429 Published: JAN 15 2000 |
| Times Cited: 87 References: 38 |
| Abstract: Allogeneic stem cell transplantation is the only treatment that can restore a normal hematopoiesis in Fanconi anemia (FA). In this retrospective multicenter study, we analyzed the results of this approach using HLA-matched unrelated bone marrow donors, and tried to identify covariates predicting the outcome of the transplant. From January 1985 to June 1998, 69 FA patients were transplanted with unrelated HLA-matched donors. Patients' characteristics before and after transplant were provided by the European group blood and marrow transplant registry and were analyzed in collaboration with the European Fanconi Anemia Registry. The 3-year probability of survival was 33%. Extensive malformations, a positive recipient cytomegalovirus serology, the use of androgens before transplant, and female donors were associated with a worse outcome. Primary graft failures were observed more frequently when female donors were used, mainly because the grafts contained lower nucleated cell doses per kilogram of recipient body weight compared with grafts coming from male donors. The probability of grade III-IV acute graft-versus-host disease (GVHD) was 34%. Elevated serum alanine/aspartate transaminases before transplantation; limb, urogenital tract, or nephrologic malformations; and non-T cell-depleted grafts were predictors of severe acute GVHD. This study shows the dramatic impact of preexisting congenital malformations on the outcome of FA patients transplanted with HLA-matched unrelated donors. If the use of T-cell depletion has led to a dramatic reduction of acute GVHD incidence, no significant outcome improvement was observed with this approach, mainly because of an increased risk of graft failure. (Blood, 2000;95:422-429) (C) 2000 by The American Society of Hematology. |
| Document Type: Article |
| Language: English |
| Reprint Address: Gluckman, E (reprint author), Hop St Louis, Serv Greffe de Moelle Trefle 3, Dept Hematol, Bone Marrow Transplant Unit, 1 Ave Claude Vellefaux, F-75475 Paris 10, France |
Addresses:
1. Hop St Louis, Serv Greffe de Moelle Trefle 3, Dept Hematol, Bone Marrow Transplant Unit, F-75475 Paris 10, France
2. Hosp Civil, Dept Hematol, Curitiba, Parana Brazil
3. Hammersmith Hosp, Dept Hematol, London, England
4. Hosp Maternal Infantil Vall Hebron, Dept Hematol, Barcelona, Spain
5. Leiden Univ, Med Ctr, Dept Pediat, Leiden, Netherlands
6. St George Hosp, Sch Med, Div Hematol, London, England
7. Policlin San Matteo, Pediat Clin, I-27100 Pavia, Italy
8. Clin Univ St Luc, Dept Hematol, B-1200 Brussels, Belgium
9. Royal Victoria Infirm, Paediat Oncol Unit, Newcastle Upon Tyne NE1 4LP, Tyne & Wear England
10. Huddinge Hosp, Dept Hematol, S-14186 Huddinge, Sweden
11. Osped Regina Margherita, Dept Pediat, Turin, Italy
12. New Childrens Hosp, Oncol Unit, Sydney, NSW Australia
13. Hosp Debrousse, Pediat Immunol Unit, Lyon, France
14. Hosp Edouard Herriot, Bone Marrow Transplantat Unit, Lyon, France
15. Univ Hosp, Dept Pediat, Lund, Sweden
16. St Laszio Hosp, Bone Marrow Transplantat Unit, Budapest, Hungary
17. Osped Civile, Dept Hematol, Pescara, Italy
18. Rigshosp, Pediat Clin 2, DK-2100 Copenhagen, Denmark
19. Clin Oncoematol Pediat, Padua, Italy
20. Ctr Leucemie Infantili, Padua, Italy
21. Hosp Jean Minjoz, Dept Hematol, Besancon, France
22. Univ La Sapienza, GITMO Roma, Rome, Italy
23. Osped San Martino Genova, Dept Hematol, Genoa, Italy |
| Publisher: AMER SOC HEMATOLOGY, 1200 19TH ST, NW, STE 300, WASHINGTON, DC 20036-2422 USA |
| Subject Category: Hematology |
| IDS Number: 272QG |
| ISSN: 0006-4971 |
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