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Nonmyeloablative allogeneic hematopoietic stem cell transplantation for GATA2 deficiency.
Biol Blood Marrow Transplant. 2014 Dec; 20(12):1940-8.BB

Abstract

We treated 14 patients with GATA2 deficiency using a nonmyeloablative allogeneic hematopoietic stem cell transplantation regimen. Four patients received peripheral blood stem cells from matched related donors (MRD), 4 patients received peripheral blood stem cells from matched unrelated donors (URD), 4 patients received hematopoietic stem cells from umbilical cord blood donors (UCB), and 2 patients received bone marrow cells from haploidentical related donors. MRD and URD recipients received conditioning with 3 days of fludarabine and 200 cGy total body irradiation (TBI). Haploidentical related donor recipients and UCB recipients received cyclophosphamide and 2 additional days of fludarabine along with 200 cGY TBI. MRD, URD, and UCB recipients received tacrolimus and sirolimus for post-transplantation immunosuppression, whereas haploidentical recipients received high-dose cyclophosphamide followed by tacrolimus and mycophenolate mofetil. Eight patients are alive with reconstitution of the severely deficient monocyte, B cell, and natural killer cell populations and reversal of the clinical phenotype at a median follow-up of 3.5 years. Two patients (1 URD recipient and 1 UCB recipient) rejected the donor graft and 1 MRD recipient relapsed with myelodysplastic syndrome after transplantation. We are currently using a high-dose conditioning regimen with busulfan and fludarabine in patients with GATA2 deficiency to achieve more consistent engraftment and eradication of the malignant myeloid clones.

Authors+Show Affiliations

Division of Hematology and Hematologic Malignancies, Alberta Health Services, Calgary, Alberta, Canada.Laboratory of Clinical Infectious Diseases, National Institute of Allergy and Infectious Diseases, Bethesda, Maryland.Experimental Transplantation and Immunology Branch, National Cancer Institute, Bethesda, Maryland.Laboratory of Clinical Infectious Diseases, National Institute of Allergy and Infectious Diseases, Bethesda, Maryland.Department of Laboratory Medicine, National Institutes of Health Clinical Center, Bethesda, Maryland.Department of Pharmacy, National Institutes of Health Clinical Center, Bethesda, Maryland.Experimental Transplantation and Immunology Branch, National Cancer Institute, Bethesda, Maryland.Experimental Transplantation and Immunology Branch, National Cancer Institute, Bethesda, Maryland.Leidos Biomedical Research, Inc, Frederick, Maryland; Intramural Clinical Management & Operations Branch, National Institute of Allergy and Infectious Diseases, Bethesda, Maryland.Laboratory of Clinical Infectious Diseases, National Institute of Allergy and Infectious Diseases, Bethesda, Maryland.Laboratory of Clinical Infectious Diseases, National Institute of Allergy and Infectious Diseases, Bethesda, Maryland.Experimental Transplantation and Immunology Branch, National Cancer Institute, Bethesda, Maryland. Electronic address: hicksted@mail.nih.gov.

Pub Type(s)

Clinical Trial
Journal Article
Research Support, N.I.H., Extramural
Research Support, N.I.H., Intramural

Language

eng

PubMed ID

25111582

Citation

Grossman, Jennifer, et al. "Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation for GATA2 Deficiency." Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation, vol. 20, no. 12, 2014, pp. 1940-8.
Grossman J, Cuellar-Rodriguez J, Gea-Banacloche J, et al. Nonmyeloablative allogeneic hematopoietic stem cell transplantation for GATA2 deficiency. Biol Blood Marrow Transplant. 2014;20(12):1940-8.
Grossman, J., Cuellar-Rodriguez, J., Gea-Banacloche, J., Zerbe, C., Calvo, K., Hughes, T., Hakim, F., Cole, K., Parta, M., Freeman, A., Holland, S. M., & Hickstein, D. D. (2014). Nonmyeloablative allogeneic hematopoietic stem cell transplantation for GATA2 deficiency. Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation, 20(12), 1940-8. https://doi.org/10.1016/j.bbmt.2014.08.004
Grossman J, et al. Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation for GATA2 Deficiency. Biol Blood Marrow Transplant. 2014;20(12):1940-8. PubMed PMID: 25111582.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Nonmyeloablative allogeneic hematopoietic stem cell transplantation for GATA2 deficiency. AU - Grossman,Jennifer, AU - Cuellar-Rodriguez,Jennifer, AU - Gea-Banacloche,Juan, AU - Zerbe,Christa, AU - Calvo,Katherine, AU - Hughes,Thomas, AU - Hakim,Fran, AU - Cole,Kristen, AU - Parta,Mark, AU - Freeman,Alexandra, AU - Holland,Steven M, AU - Hickstein,Dennis D, Y1 - 2014/08/09/ PY - 2014/06/02/received PY - 2014/08/04/accepted PY - 2014/8/12/entrez PY - 2014/8/12/pubmed PY - 2015/7/30/medline KW - Familial acute myelogenous leukemia KW - GATA2 KW - Hematopoietic stem cell transplantation KW - Myelodysplastic syndrome SP - 1940 EP - 8 JF - Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation JO - Biol Blood Marrow Transplant VL - 20 IS - 12 N2 - We treated 14 patients with GATA2 deficiency using a nonmyeloablative allogeneic hematopoietic stem cell transplantation regimen. Four patients received peripheral blood stem cells from matched related donors (MRD), 4 patients received peripheral blood stem cells from matched unrelated donors (URD), 4 patients received hematopoietic stem cells from umbilical cord blood donors (UCB), and 2 patients received bone marrow cells from haploidentical related donors. MRD and URD recipients received conditioning with 3 days of fludarabine and 200 cGy total body irradiation (TBI). Haploidentical related donor recipients and UCB recipients received cyclophosphamide and 2 additional days of fludarabine along with 200 cGY TBI. MRD, URD, and UCB recipients received tacrolimus and sirolimus for post-transplantation immunosuppression, whereas haploidentical recipients received high-dose cyclophosphamide followed by tacrolimus and mycophenolate mofetil. Eight patients are alive with reconstitution of the severely deficient monocyte, B cell, and natural killer cell populations and reversal of the clinical phenotype at a median follow-up of 3.5 years. Two patients (1 URD recipient and 1 UCB recipient) rejected the donor graft and 1 MRD recipient relapsed with myelodysplastic syndrome after transplantation. We are currently using a high-dose conditioning regimen with busulfan and fludarabine in patients with GATA2 deficiency to achieve more consistent engraftment and eradication of the malignant myeloid clones. SN - 1523-6536 UR - https://www.unboundmedicine.com/medline/citation/25111582/Nonmyeloablative_allogeneic_hematopoietic_stem_cell_transplantation_for_GATA2_deficiency_ L2 - https://linkinghub.elsevier.com/retrieve/pii/S1083-8791(14)00503-5 DB - PRIME DP - Unbound Medicine ER -