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Blood, 1 April 2004, Vol. 103, No. 7, pp. 2490-2497.
Prepublished online as a Blood First Edition Paper on December 4, 2003; DOI 10.1182/blood-2003-07-2546.

Submitted July 29, 2003
Accepted November 24, 2003
Long-term outcome after bone marrow transplantation for severe aplastic anemia
Lionel Ades, Jean-Yves Mary, Marie Robin, Christele Ferry, Raphael Porcher, Helene Esperou, Patricia Ribaud, Agnes Devergie, Richard Traineau, Eliane Gluckman, and Gerard Socie*
Service d'Hematologie-Greffe de Moelle, Hospital St Louis, Paris, France
INSERM ERM-0321, Hospital St Louis, Paris, France
INSERM ERM-0220, Hospital St Louis, Paris, France
* Corresponding author; email: gsocie{at}chu-stlouis.fr.
From January 1978 to December 2001, 133 patients with severe aplastic anemia underwent non-T-cell depleted allogeneic bone marrow transplantation from an HLA-identical sibling donor, at the hospital Saint Louis using either the association of cyclophosphamide (Cy) and of thoraco-abdominal irradiation (TAI) (n=100) or of Cy and anti-thymocyte globulin (ATG) (n=33), as conditioning regimen. With a 13.6 years follow-up, the 10-year survival estimates was 64%. Four factors were associated with lower survival: older age, use of Cy-TAI, any form of treatment prior transplant [either androgens or immunosuppressive therapy, (IST)] and grade II-IV acute GvHD. TAI was the sole factor associated with the occurrence of acute GvHD. The risk of cancers (20-year cumulative incidence; 10.9%) was associated with older age and with the use of cyclosporine as IST pre-transplant. Cumulative incidences and risk factors of non-malignant late effect including avascular osteonecrosis, late bacterial, viral and fungal infection were also analyzed. Improved results using Cy-ATG as conditioning can lead to more than 80% chance of cure in patients with SAA. However, only avoidance of any IST before transplant in patients with sibling donor are compatible with such excellent results.

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