Nonmyeloablative Peripheral Blood Haploidentical Stem Cell Transplantation for Refractory Severe Aplastic Anemia

Jennifer Clay; Austin G. Kulasekararaj; Victoria Potter; Francesco Grimaldi; Donal McLornan; Kavita Raj; Hugues de Lavallade; Michelle Kenyon; Tony Pagliuca; Ghulam Mufti; Judith C. W. Marsh

doi:10.1016/j.bbmt.2014.06.028

Nonmyeloablative Peripheral Blood Haploidentical Stem Cell Transplantation for Refractory Severe Aplastic Anemia

Jennifer Clay, Austin G. Kulasekararaj, Victoria Potter, Francesco Grimaldi, Donal McLornan, Kavita Raj, Hugues de Lavallade, Michelle Kenyon, Tony Pagliuca, Ghulam Mufti, Judith C. W. Marsh^*

^*Corresponding author for this work

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

105 Citations (Scopus)

Abstract

New transplant approaches are urgently needed for patients with refractory severe aplastic anemia (SAA) who lack a matched sibling or unrelated donor (UD) or who have failed UD or cord blood transplant. Patients with refractory SAA are at risk of later clonal evolution to myelodysplastic syndrome and acute leukemia. We report our pilot findings with haploidentical hematopoietic stem cell transplantation (haploHSCT) using uniform reduced-intensity conditioning with postgraft high-dose cyclophosphamide in 8 patients with refractory SAA or patients who rejected a prior UD or cord blood transplant. Six of 8 patients engrafted. Graft failure was associated with donor-directed HLA antibodies, despite intensive pre-HSCT desensitization with plasma exchange and rituximab. There was only 1 case of grade II skin graft-versus-host disease. We show that haploHSCT can successfully rescue refractory SAA patients who lack donor-directed HLA antibodies but not in the presence of donor-directed HLA antibodies. This novel protocol for haploHSCT for SAA has been adopted by the European Group for Blood and Marrow Transplantation Severe Aplastic Anaemia Working Party for a future noninterventional, observational study to further evaluate its efficacy.

Original language	English
Pages (from-to)	1711-1716
Number of pages	6
Journal	BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION
Volume	20
Issue number	11
DOIs	https://doi.org/10.1016/j.bbmt.2014.06.028
Publication status	Published - Nov 2014

Keywords

HSCT
Haploidentical
Aplastic anemia
Stem cell transplant
Nonmyeloablative
ACUTE MYELOID-LEUKEMIA
BONE-MARROW
POSTTRANSPLANTATION CYCLOPHOSPHAMIDE
HLA ALLOIMMUNIZATION
HEMATOPOIETIC SCT
EUROPEAN GROUP
WORKING PARTY
RISK-FACTORS
TRANSFUSIONS
ASSOCIATION

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Clay, J., Kulasekararaj, A. G., Potter, V., Grimaldi, F., McLornan, D., Raj, K., de Lavallade, H., Kenyon, M., Pagliuca, T., Mufti, G., & Marsh, J. C. W. (2014). Nonmyeloablative Peripheral Blood Haploidentical Stem Cell Transplantation for Refractory Severe Aplastic Anemia. BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 20(11), 1711-1716. https://doi.org/10.1016/j.bbmt.2014.06.028

@article{69b00f568bb842d19e16d0f30f775dc4,

title = "Nonmyeloablative Peripheral Blood Haploidentical Stem Cell Transplantation for Refractory Severe Aplastic Anemia",

abstract = "New transplant approaches are urgently needed for patients with refractory severe aplastic anemia (SAA) who lack a matched sibling or unrelated donor (UD) or who have failed UD or cord blood transplant. Patients with refractory SAA are at risk of later clonal evolution to myelodysplastic syndrome and acute leukemia. We report our pilot findings with haploidentical hematopoietic stem cell transplantation (haploHSCT) using uniform reduced-intensity conditioning with postgraft high-dose cyclophosphamide in 8 patients with refractory SAA or patients who rejected a prior UD or cord blood transplant. Six of 8 patients engrafted. Graft failure was associated with donor-directed HLA antibodies, despite intensive pre-HSCT desensitization with plasma exchange and rituximab. There was only 1 case of grade II skin graft-versus-host disease. We show that haploHSCT can successfully rescue refractory SAA patients who lack donor-directed HLA antibodies but not in the presence of donor-directed HLA antibodies. This novel protocol for haploHSCT for SAA has been adopted by the European Group for Blood and Marrow Transplantation Severe Aplastic Anaemia Working Party for a future noninterventional, observational study to further evaluate its efficacy.",

keywords = "HSCT, Haploidentical, Aplastic anemia, Stem cell transplant, Nonmyeloablative, ACUTE MYELOID-LEUKEMIA, BONE-MARROW, POSTTRANSPLANTATION CYCLOPHOSPHAMIDE, HLA ALLOIMMUNIZATION, HEMATOPOIETIC SCT, EUROPEAN GROUP, WORKING PARTY, RISK-FACTORS, TRANSFUSIONS, ASSOCIATION",

author = "Jennifer Clay and Kulasekararaj, {Austin G.} and Victoria Potter and Francesco Grimaldi and Donal McLornan and Kavita Raj and {de Lavallade}, Hugues and Michelle Kenyon and Tony Pagliuca and Ghulam Mufti and Marsh, {Judith C. W.}",

year = "2014",

month = nov,

doi = "10.1016/j.bbmt.2014.06.028",

language = "English",

volume = "20",

pages = "1711--1716",

journal = "BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION",

issn = "1083-8791",

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number = "11",

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Clay, J, Kulasekararaj, AG, Potter, V, Grimaldi, F, McLornan, D, Raj, K, de Lavallade, H, Kenyon, M, Pagliuca, T , Mufti, G & Marsh, JCW 2014, 'Nonmyeloablative Peripheral Blood Haploidentical Stem Cell Transplantation for Refractory Severe Aplastic Anemia', BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, vol. 20, no. 11, pp. 1711-1716. https://doi.org/10.1016/j.bbmt.2014.06.028

TY - JOUR

T1 - Nonmyeloablative Peripheral Blood Haploidentical Stem Cell Transplantation for Refractory Severe Aplastic Anemia

AU - Clay, Jennifer

AU - Kulasekararaj, Austin G.

AU - Potter, Victoria

AU - Grimaldi, Francesco

AU - McLornan, Donal

AU - Raj, Kavita

AU - de Lavallade, Hugues

AU - Kenyon, Michelle

AU - Pagliuca, Tony

AU - Mufti, Ghulam

AU - Marsh, Judith C. W.

PY - 2014/11

Y1 - 2014/11

N2 - New transplant approaches are urgently needed for patients with refractory severe aplastic anemia (SAA) who lack a matched sibling or unrelated donor (UD) or who have failed UD or cord blood transplant. Patients with refractory SAA are at risk of later clonal evolution to myelodysplastic syndrome and acute leukemia. We report our pilot findings with haploidentical hematopoietic stem cell transplantation (haploHSCT) using uniform reduced-intensity conditioning with postgraft high-dose cyclophosphamide in 8 patients with refractory SAA or patients who rejected a prior UD or cord blood transplant. Six of 8 patients engrafted. Graft failure was associated with donor-directed HLA antibodies, despite intensive pre-HSCT desensitization with plasma exchange and rituximab. There was only 1 case of grade II skin graft-versus-host disease. We show that haploHSCT can successfully rescue refractory SAA patients who lack donor-directed HLA antibodies but not in the presence of donor-directed HLA antibodies. This novel protocol for haploHSCT for SAA has been adopted by the European Group for Blood and Marrow Transplantation Severe Aplastic Anaemia Working Party for a future noninterventional, observational study to further evaluate its efficacy.

AB - New transplant approaches are urgently needed for patients with refractory severe aplastic anemia (SAA) who lack a matched sibling or unrelated donor (UD) or who have failed UD or cord blood transplant. Patients with refractory SAA are at risk of later clonal evolution to myelodysplastic syndrome and acute leukemia. We report our pilot findings with haploidentical hematopoietic stem cell transplantation (haploHSCT) using uniform reduced-intensity conditioning with postgraft high-dose cyclophosphamide in 8 patients with refractory SAA or patients who rejected a prior UD or cord blood transplant. Six of 8 patients engrafted. Graft failure was associated with donor-directed HLA antibodies, despite intensive pre-HSCT desensitization with plasma exchange and rituximab. There was only 1 case of grade II skin graft-versus-host disease. We show that haploHSCT can successfully rescue refractory SAA patients who lack donor-directed HLA antibodies but not in the presence of donor-directed HLA antibodies. This novel protocol for haploHSCT for SAA has been adopted by the European Group for Blood and Marrow Transplantation Severe Aplastic Anaemia Working Party for a future noninterventional, observational study to further evaluate its efficacy.

KW - HSCT

KW - Haploidentical

KW - Aplastic anemia

KW - Stem cell transplant

KW - Nonmyeloablative

KW - ACUTE MYELOID-LEUKEMIA

KW - BONE-MARROW

KW - POSTTRANSPLANTATION CYCLOPHOSPHAMIDE

KW - HLA ALLOIMMUNIZATION

KW - HEMATOPOIETIC SCT

KW - EUROPEAN GROUP

KW - WORKING PARTY

KW - RISK-FACTORS

KW - TRANSFUSIONS

KW - ASSOCIATION

U2 - 10.1016/j.bbmt.2014.06.028

DO - 10.1016/j.bbmt.2014.06.028

M3 - Article

SN - 1083-8791

VL - 20

SP - 1711

EP - 1716

JO - BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

JF - BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

IS - 11

ER -

Nonmyeloablative Peripheral Blood Haploidentical Stem Cell Transplantation for Refractory Severe Aplastic Anemia

Abstract

Keywords

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