Citation
Parta, Mark, et al. "Allogeneic Hematopoietic Stem Cell Transplantation for GATA2 Deficiency Using a Busulfan-Based Regimen." Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation, vol. 24, no. 6, 2018, pp. 1250-1259.
Parta M, Shah NN, Baird K, et al. Allogeneic Hematopoietic Stem Cell Transplantation for GATA2 Deficiency Using a Busulfan-Based Regimen. Biol Blood Marrow Transplant. 2018;24(6):1250-1259.
Parta, M., Shah, N. N., Baird, K., Rafei, H., Calvo, K. R., Hughes, T., Cole, K., Kenyon, M., Schuver, B. B., Cuellar-Rodriguez, J., Zerbe, C. S., Holland, S. M., & Hickstein, D. D. (2018). Allogeneic Hematopoietic Stem Cell Transplantation for GATA2 Deficiency Using a Busulfan-Based Regimen. Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation, 24(6), 1250-1259. https://doi.org/10.1016/j.bbmt.2018.01.030
Parta M, et al. Allogeneic Hematopoietic Stem Cell Transplantation for GATA2 Deficiency Using a Busulfan-Based Regimen. Biol Blood Marrow Transplant. 2018;24(6):1250-1259. PubMed PMID: 29412158.
TY - JOUR
T1 - Allogeneic Hematopoietic Stem Cell Transplantation for GATA2 Deficiency Using a Busulfan-Based Regimen.
AU - Parta,Mark,
AU - Shah,Nirali N,
AU - Baird,Kristin,
AU - Rafei,Hind,
AU - Calvo,Katherine R,
AU - Hughes,Thomas,
AU - Cole,Kristen,
AU - Kenyon,Meg,
AU - Schuver,Bazetta Blacklock,
AU - Cuellar-Rodriguez,Jennifer,
AU - Zerbe,Christa S,
AU - Holland,Steven M,
AU - Hickstein,Dennis D,
Y1 - 2018/02/03/
PY - 2017/11/25/received
PY - 2018/01/29/accepted
PY - 2018/2/8/pubmed
PY - 2019/4/2/medline
PY - 2018/2/8/entrez
KW - GATA2
KW - GVHD
KW - Graft-versus host disease
KW - HSCT
KW - Hematopoietic stem cell transplant
KW - MDS
KW - Myelodysplastic syndrome
SP - 1250
EP - 1259
JF - Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
JO - Biol Blood Marrow Transplant
VL - 24
IS - 6
N2 - Allogeneic hematopoietic stem cell transplantation (HSCT) reverses the bone marrow failure syndrome due to GATA2 deficiency. The intensity of conditioning required to achieve reliable engraftment and prevent relapse remains unclear. Here, we describe the results of a prospective study of HSCT in 22 patients with GATA2 deficiency using a busulfan-based conditioning regimen. The study included 2 matched related donor (MRD) recipients, 13 matched unrelated donor (URD) recipients, and 7 haploidentical related donor (HRD) recipients. MRD and URD recipients received 4 days of busulfan and 4 days of fludarabine. HRD recipients received low-dose cyclophosphamide for 2 days, fludarabine for 5 days, 2 to 3 days of busulfan depending on cytogenetics, and 200 cGy total body irradiation. MRD and URD recipients received tacrolimus and short-course methotrexate for graft-versus-host disease (GVHD) prophylaxis. HRD recipients received high-dose post-transplant cyclophosphamide (PTCy) followed by tacrolimus and mycophenolate mofetil. At a median follow-up of 24 months (range, 9 to 50), 19 of 22 patients were alive with reversal of the disease phenotype and correction of the myelodysplastic syndrome, including eradication of cytogenetic abnormalities. Three patients died: 1 from refractory acute myelogenous leukemia, 1 from GVHD, and 1 from sepsis. There was a 26% incidence of grades III to IV acute GVHD in the MRD and URD groups and no grades III to IV acute GVHD in the HRD cohort. Similarly, there was a 46% incidence of chronic GVHD in the MRD and URD cohorts, whereas only 28% of HRD recipients developed chronic GVHD. Despite excellent overall disease-free survival (86%), GVHD remains a limitation using standard prophylaxis for GVHD. We are currently extending the use of PTCy to the MRD and URD cohorts to reduce GVHD.
SN - 1523-6536
UR - https://www.unboundmedicine.com/medline/citation/29412158/Allogeneic_Hematopoietic_Stem_Cell_Transplantation_for_GATA2_Deficiency_Using_a_Busulfan_Based_Regimen_
L2 - https://linkinghub.elsevier.com/retrieve/pii/S1083-8791(18)30047-8
DB - PRIME
DP - Unbound Medicine
ER -