Outcome of patients activating an unrelated donor search for severe acquired aplastic anemia

Sebastien Maury; Marie-Lorraine Balere-Appert; Simona Pollichieni; Rosi Oneto; Ibrahim Yakoub-Agha; Franco Locatelli; Jean-Hugues Dalle; Edoardo Lanino; Alain Fischer; Andrea Pession; Anne Huynh; Walter Barberi; Mohamad Mohty; Antonio Risitano; Noel Milpied; Gerard Socie; Andrea Bacigalupo; Judith Marsh; Jakob R. Passweg; Severe Aplastic Anemia Working Par

doi:10.1002/ajh.23522

Outcome of patients activating an unrelated donor search for severe acquired aplastic anemia

Sebastien Maury^*, Marie-Lorraine Balere-Appert, Simona Pollichieni, Rosi Oneto, Ibrahim Yakoub-Agha, Franco Locatelli, Jean-Hugues Dalle, Edoardo Lanino, Alain Fischer, Andrea Pession, Anne Huynh, Walter Barberi, Mohamad Mohty, Antonio Risitano, Noel Milpied, Gerard Socie, Andrea Bacigalupo, Judith Marsh, Jakob R. Passweg, Severe Aplastic Anemia Working Par

^*Corresponding author for this work

Research output: Contribution to journal › Article › peer-review

11 Citations (Scopus)

Abstract

Patients with severe aplastic anemia (SAA) without a sibling donor receive immunosuppressive treatment (IST) with anti-thymocyte globulin (ATG). In the case of no response to IST, a voluntary unrelated donor (VUD) search is usually started. This study analyzes the outcome of ATG-refractory SAA patients activating a VUD search. Of 179 patients, 68 had at least one HLA-A, –B, and –DR matched donor identified and underwent HSCT while 50 also with a donor were not transplanted because of early death (8), late response to IST (34), transplant refusal (1), or other (7). Conversely, 61 had no matched donor, 13 of those ultimately received a mismatched HSCT. All but one received marrow stem cells. Among patients aged <17 years, those with at least one matched donor had a significant higher 4-year survival as compared to others (79% ± 6% versus 53% ± 10%, P = 0.01). There was also a survival advantage independent of recipient age when the donor search was initiated in the recent 2000–2005 study-period (74% ± 6% versus 47% ± 10%, P < 0.05). In multivariate analysis, the identification of a matched VUD tended to impact favourably on survival in patients with a recent donor search (P = 0.07). This study provides evidence for the use of unrelated donor HSCT in children and adults with IST-refractory SAA.

Original language	English
Pages (from-to)	868-873
Number of pages	6
Journal	American Journal of Hematology
Volume	88
Issue number	10
DOIs	https://doi.org/10.1002/ajh.23522
Publication status	Published - Oct 2013

Keywords

STEM-CELL TRANSPLANTATION
COLONY-STIMULATING FACTOR
ANTI-THYMOCYTE GLOBULIN
IMMUNOSUPPRESSIVE THERAPY
ANTILYMPHOCYTE GLOBULIN
ANTITHYMOCYTE GLOBULIN
MARROW TRANSPLANTATION
ALTERNATIVE-DONOR
CYCLOSPORINE
CHILDREN

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10.1002/ajh.23522

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Maury, S., Balere-Appert, M.-L., Pollichieni, S., Oneto, R., Yakoub-Agha, I., Locatelli, F., Dalle, J.-H., Lanino, E., Fischer, A., Pession, A., Huynh, A., Barberi, W., Mohty, M., Risitano, A., Milpied, N., Socie, G., Bacigalupo, A., Marsh, J., Passweg, J. R., & Severe Aplastic Anemia Working Par (2013). Outcome of patients activating an unrelated donor search for severe acquired aplastic anemia. American Journal of Hematology, 88(10), 868-873. https://doi.org/10.1002/ajh.23522

@article{a8168634854e41f0b42572f531a544d7,

title = "Outcome of patients activating an unrelated donor search for severe acquired aplastic anemia",

abstract = "Patients with severe aplastic anemia (SAA) without a sibling donor receive immunosuppressive treatment (IST) with anti-thymocyte globulin (ATG). In the case of no response to IST, a voluntary unrelated donor (VUD) search is usually started. This study analyzes the outcome of ATG-refractory SAA patients activating a VUD search. Of 179 patients, 68 had at least one HLA-A, –B, and –DR matched donor identified and underwent HSCT while 50 also with a donor were not transplanted because of early death (8), late response to IST (34), transplant refusal (1), or other (7). Conversely, 61 had no matched donor, 13 of those ultimately received a mismatched HSCT. All but one received marrow stem cells. Among patients aged <17 years, those with at least one matched donor had a significant higher 4-year survival as compared to others (79% ± 6% versus 53% ± 10%, P = 0.01). There was also a survival advantage independent of recipient age when the donor search was initiated in the recent 2000–2005 study-period (74% ± 6% versus 47% ± 10%, P < 0.05). In multivariate analysis, the identification of a matched VUD tended to impact favourably on survival in patients with a recent donor search (P = 0.07). This study provides evidence for the use of unrelated donor HSCT in children and adults with IST-refractory SAA.",

keywords = "STEM-CELL TRANSPLANTATION, COLONY-STIMULATING FACTOR, ANTI-THYMOCYTE GLOBULIN, IMMUNOSUPPRESSIVE THERAPY, ANTILYMPHOCYTE GLOBULIN, ANTITHYMOCYTE GLOBULIN, MARROW TRANSPLANTATION, ALTERNATIVE-DONOR, CYCLOSPORINE, CHILDREN",

author = "Sebastien Maury and Marie-Lorraine Balere-Appert and Simona Pollichieni and Rosi Oneto and Ibrahim Yakoub-Agha and Franco Locatelli and Jean-Hugues Dalle and Edoardo Lanino and Alain Fischer and Andrea Pession and Anne Huynh and Walter Barberi and Mohamad Mohty and Antonio Risitano and Noel Milpied and Gerard Socie and Andrea Bacigalupo and Judith Marsh and Passweg, {Jakob R.} and {Severe Aplastic Anemia Working Par}",

year = "2013",

month = oct,

doi = "10.1002/ajh.23522",

language = "English",

volume = "88",

pages = "868--873",

journal = "American Journal of Hematology",

issn = "0361-8609",

publisher = "Wiley-Liss Inc.",

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Maury, S, Balere-Appert, M-L, Pollichieni, S, Oneto, R, Yakoub-Agha, I, Locatelli, F, Dalle, J-H, Lanino, E, Fischer, A, Pession, A, Huynh, A, Barberi, W, Mohty, M, Risitano, A, Milpied, N, Socie, G, Bacigalupo, A, Marsh, J, Passweg, JR & Severe Aplastic Anemia Working Par 2013, 'Outcome of patients activating an unrelated donor search for severe acquired aplastic anemia', American Journal of Hematology, vol. 88, no. 10, pp. 868-873. https://doi.org/10.1002/ajh.23522

TY - JOUR

T1 - Outcome of patients activating an unrelated donor search for severe acquired aplastic anemia

AU - Maury, Sebastien

AU - Balere-Appert, Marie-Lorraine

AU - Pollichieni, Simona

AU - Oneto, Rosi

AU - Yakoub-Agha, Ibrahim

AU - Locatelli, Franco

AU - Dalle, Jean-Hugues

AU - Lanino, Edoardo

AU - Fischer, Alain

AU - Pession, Andrea

AU - Huynh, Anne

AU - Barberi, Walter

AU - Mohty, Mohamad

AU - Risitano, Antonio

AU - Milpied, Noel

AU - Socie, Gerard

AU - Bacigalupo, Andrea

AU - Marsh, Judith

AU - Passweg, Jakob R.

AU - Severe Aplastic Anemia Working Par

PY - 2013/10

Y1 - 2013/10

N2 - Patients with severe aplastic anemia (SAA) without a sibling donor receive immunosuppressive treatment (IST) with anti-thymocyte globulin (ATG). In the case of no response to IST, a voluntary unrelated donor (VUD) search is usually started. This study analyzes the outcome of ATG-refractory SAA patients activating a VUD search. Of 179 patients, 68 had at least one HLA-A, –B, and –DR matched donor identified and underwent HSCT while 50 also with a donor were not transplanted because of early death (8), late response to IST (34), transplant refusal (1), or other (7). Conversely, 61 had no matched donor, 13 of those ultimately received a mismatched HSCT. All but one received marrow stem cells. Among patients aged <17 years, those with at least one matched donor had a significant higher 4-year survival as compared to others (79% ± 6% versus 53% ± 10%, P = 0.01). There was also a survival advantage independent of recipient age when the donor search was initiated in the recent 2000–2005 study-period (74% ± 6% versus 47% ± 10%, P < 0.05). In multivariate analysis, the identification of a matched VUD tended to impact favourably on survival in patients with a recent donor search (P = 0.07). This study provides evidence for the use of unrelated donor HSCT in children and adults with IST-refractory SAA.

AB - Patients with severe aplastic anemia (SAA) without a sibling donor receive immunosuppressive treatment (IST) with anti-thymocyte globulin (ATG). In the case of no response to IST, a voluntary unrelated donor (VUD) search is usually started. This study analyzes the outcome of ATG-refractory SAA patients activating a VUD search. Of 179 patients, 68 had at least one HLA-A, –B, and –DR matched donor identified and underwent HSCT while 50 also with a donor were not transplanted because of early death (8), late response to IST (34), transplant refusal (1), or other (7). Conversely, 61 had no matched donor, 13 of those ultimately received a mismatched HSCT. All but one received marrow stem cells. Among patients aged <17 years, those with at least one matched donor had a significant higher 4-year survival as compared to others (79% ± 6% versus 53% ± 10%, P = 0.01). There was also a survival advantage independent of recipient age when the donor search was initiated in the recent 2000–2005 study-period (74% ± 6% versus 47% ± 10%, P < 0.05). In multivariate analysis, the identification of a matched VUD tended to impact favourably on survival in patients with a recent donor search (P = 0.07). This study provides evidence for the use of unrelated donor HSCT in children and adults with IST-refractory SAA.

KW - STEM-CELL TRANSPLANTATION

KW - COLONY-STIMULATING FACTOR

KW - ANTI-THYMOCYTE GLOBULIN

KW - IMMUNOSUPPRESSIVE THERAPY

KW - ANTILYMPHOCYTE GLOBULIN

KW - ANTITHYMOCYTE GLOBULIN

KW - MARROW TRANSPLANTATION

KW - ALTERNATIVE-DONOR

KW - CYCLOSPORINE

KW - CHILDREN

U2 - 10.1002/ajh.23522

DO - 10.1002/ajh.23522

M3 - Article

SN - 0361-8609

VL - 88

SP - 868

EP - 873

JO - American Journal of Hematology

JF - American Journal of Hematology

IS - 10

ER -

Outcome of patients activating an unrelated donor search for severe acquired aplastic anemia

Abstract

Keywords

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