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Unrelated stem cell transplantation for severe acquired aplastic anemia: improved outcome in the era of high-resolution HLA matching between donor and recipient.
Haematologica. 2007 May; 92(5):589-96.H

Abstract

BACKGROUND AND OBJECTIVES

Severe acquired aplastic anemia (SAA) is a potentially fatal bone marrow failure syndrome occurring mainly in children and young adults. Immunosuppressive regimens and hematopoietic stem cell transplantation (HSCT) are the only two available curative treatments. Patients who lack an HLA-identical sibling donor may receive HSCT from an unrelated donor, a strategy historically associated with high mortality rates. Thus, for patients refractory to immunosuppressive regimens, the decision to transplant stem cells from unrelated donors is weighed against supportive care and often represents a dilemma for physicians. We aimed to determine whether outcome after unrelated HSCT has improved in recent years and, if so, to determine the factors responsible for the improvement.

DESIGN AND METHODS

We analyzed the outcome of 89 patients (median age 17 years, range 0-52) with acquired SAA undergoing HSCT from an unrelated donor between 1989 and 2004. Cases were consecutively reported to the French Registry (SFGM-TC) by 25 centers.

RESULTS

Patients transplanted during two successive time-periods (1989-1998 and 1999-2004) had different 5-year survival probabilities (+/-95% confidence interval): 29%+/-7% and 50%+/-7%, respectively (p<0.01). The main difference between the two cohorts concerned HLA matching between donors and recipients at the allelic level for the ten HLA-A, -B, -C, -DRB1 and -DQB1 antigens, which was more frequent in 1999-2004 than in the former period (p=0.0004). In multivariate analysis, the only two factors affecting survival were HLA allelic matching (p<0.01) and younger age of recipient (17 pounds sterling years, p<0.0001). Survival reached 78%+/-11% at 5 years for the younger, fully HLA-matched patients.

INTERPRETATION AND CONCLUSIONS

Survival after unrelated HSCT for SAA has improved significantly over the past 15 years, mainly due to better HLA matching. Results for young patients who are fully HLA-matched at the allelic level with their donor are comparable to those observed after HSCT from a related donor.

Authors+Show Affiliations

Service d'Hématologie, Hôpital Henri Mondor, Créteil, France. sebastien.maury@hmn.aphp.frNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Comparative Study
Evaluation Study
Journal Article

Language

eng

PubMed ID

17488681

Citation

Maury, Sébastien, et al. "Unrelated Stem Cell Transplantation for Severe Acquired Aplastic Anemia: Improved Outcome in the Era of High-resolution HLA Matching Between Donor and Recipient." Haematologica, vol. 92, no. 5, 2007, pp. 589-96.
Maury S, Balère-Appert ML, Chir Z, et al. Unrelated stem cell transplantation for severe acquired aplastic anemia: improved outcome in the era of high-resolution HLA matching between donor and recipient. Haematologica. 2007;92(5):589-96.
Maury, S., Balère-Appert, M. L., Chir, Z., Boiron, J. M., Galambrun, C., Yakouben, K., Bordigoni, P., Marie-Cardine, A., Milpied, N., Kanold, J., Maillard, N., & Socié, G. (2007). Unrelated stem cell transplantation for severe acquired aplastic anemia: improved outcome in the era of high-resolution HLA matching between donor and recipient. Haematologica, 92(5), 589-96.
Maury S, et al. Unrelated Stem Cell Transplantation for Severe Acquired Aplastic Anemia: Improved Outcome in the Era of High-resolution HLA Matching Between Donor and Recipient. Haematologica. 2007;92(5):589-96. PubMed PMID: 17488681.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Unrelated stem cell transplantation for severe acquired aplastic anemia: improved outcome in the era of high-resolution HLA matching between donor and recipient. AU - Maury,Sébastien, AU - Balère-Appert,Marie-Lorraine, AU - Chir,Zina, AU - Boiron,Jean-Michel, AU - Galambrun,Claire, AU - Yakouben,Karima, AU - Bordigoni,Pierre, AU - Marie-Cardine,Aude, AU - Milpied,Noel, AU - Kanold,Judith, AU - Maillard,Natacha, AU - Socié,Gérard, AU - ,, PY - 2007/5/10/pubmed PY - 2007/7/17/medline PY - 2007/5/10/entrez SP - 589 EP - 96 JF - Haematologica JO - Haematologica VL - 92 IS - 5 N2 - BACKGROUND AND OBJECTIVES: Severe acquired aplastic anemia (SAA) is a potentially fatal bone marrow failure syndrome occurring mainly in children and young adults. Immunosuppressive regimens and hematopoietic stem cell transplantation (HSCT) are the only two available curative treatments. Patients who lack an HLA-identical sibling donor may receive HSCT from an unrelated donor, a strategy historically associated with high mortality rates. Thus, for patients refractory to immunosuppressive regimens, the decision to transplant stem cells from unrelated donors is weighed against supportive care and often represents a dilemma for physicians. We aimed to determine whether outcome after unrelated HSCT has improved in recent years and, if so, to determine the factors responsible for the improvement. DESIGN AND METHODS: We analyzed the outcome of 89 patients (median age 17 years, range 0-52) with acquired SAA undergoing HSCT from an unrelated donor between 1989 and 2004. Cases were consecutively reported to the French Registry (SFGM-TC) by 25 centers. RESULTS: Patients transplanted during two successive time-periods (1989-1998 and 1999-2004) had different 5-year survival probabilities (+/-95% confidence interval): 29%+/-7% and 50%+/-7%, respectively (p<0.01). The main difference between the two cohorts concerned HLA matching between donors and recipients at the allelic level for the ten HLA-A, -B, -C, -DRB1 and -DQB1 antigens, which was more frequent in 1999-2004 than in the former period (p=0.0004). In multivariate analysis, the only two factors affecting survival were HLA allelic matching (p<0.01) and younger age of recipient (17 pounds sterling years, p<0.0001). Survival reached 78%+/-11% at 5 years for the younger, fully HLA-matched patients. INTERPRETATION AND CONCLUSIONS: Survival after unrelated HSCT for SAA has improved significantly over the past 15 years, mainly due to better HLA matching. Results for young patients who are fully HLA-matched at the allelic level with their donor are comparable to those observed after HSCT from a related donor. SN - 1592-8721 UR - https://www.unboundmedicine.com/medline/citation/17488681/Unrelated_stem_cell_transplantation_for_severe_acquired_aplastic_anemia:_improved_outcome_in_the_era_of_high_resolution_HLA_matching_between_donor_and_recipient_ L2 - http://www.haematologica.org/cgi/pmidlookup?view=long&amp;pmid=17488681 DB - PRIME DP - Unbound Medicine ER -