Nonmyeloablative Allogeneic Hematopoietic Stem-Cell Transplantation for GATA2 Deficiency
Identifieur interne : 002771 ( Main/Exploration ); précédent : 002770; suivant : 002772Nonmyeloablative Allogeneic Hematopoietic Stem-Cell Transplantation for GATA2 Deficiency
Auteurs : Jennifer Grossman [Canada] ; Jennifer Cuellar-Rodriguez [États-Unis] ; Juan Gea-Banacloche [États-Unis] ; Christa Zerbe [États-Unis] ; Katherine Calvo [États-Unis] ; Thomas Hughes [États-Unis] ; Fran Hakim [États-Unis] ; Kristen Cole [États-Unis] ; Mark Parta [États-Unis] ; Alexandra Freeman [États-Unis] ; Steven M. Holland [États-Unis] ; Dennis D. Hickstein [États-Unis]Source :
- Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation [ 1083-8791 ] ; 2014.
Abstract
We treated 14 patients with GATA2 deficiency using a nonmyeloablative allogeneic hematopoietic stem cell transplantation (HSCT) regimen. Four patients received peripheral blood stem cells (PBSC) from matched-related donors (MRD), four patients received PBSC from matched-unrelated donors (URD), four patients received HSC from umbilical cord blood donors (UCB), and two patients received bone marrow cells from haploidentical related donors. MRD and URD recipients received conditioning with three days of fludarabine and 200cGy total body irradiation (TBI). Haploidentical related donor recipients and UCB recipients received cyclophosphamide and two additional days of fludarabine along with the 200 cGY TBI. MRD, URD, and UCB recipients received tacrolimus and sirolimus for post-transplant 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 (NK) cell populations and reversal of the clinical phenotype at a median follow-up of 3.5 years. Two patients rejected the donor graft (one URD and one UCB), and one MRD recipient relapsed with myelodysplastic syndrome (MDS) post-transplant. 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.
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DOI: 10.1016/j.bbmt.2014.08.004
PubMed: 25111582
PubMed Central: 4253545
Affiliations:
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Bethesda</wicri:cityArea></affiliation></author><author><name sortKey="Freeman, Alexandra" sort="Freeman, Alexandra" uniqKey="Freeman A" first="Alexandra" last="Freeman">Alexandra Freeman</name><affiliation wicri:level="2"><nlm:aff id="A2">Laboratory of Clinical Infectious Diseases, National Institute of Allergy and Infectious Diseases, Bethesda, Maryland</nlm:aff><country xml:lang="fr">États-Unis</country><placeName><region type="state">Maryland</region></placeName><wicri:cityArea>Laboratory of Clinical Infectious Diseases, National Institute of Allergy and Infectious Diseases, Bethesda</wicri:cityArea></affiliation></author><author><name sortKey="Holland, Steven M" sort="Holland, Steven M" uniqKey="Holland S" first="Steven M." last="Holland">Steven M. Holland</name><affiliation wicri:level="2"><nlm:aff id="A2">Laboratory of Clinical Infectious Diseases, National Institute of Allergy and Infectious Diseases, Bethesda, Maryland</nlm:aff><country xml:lang="fr">États-Unis</country><placeName><region type="state">Maryland</region></placeName><wicri:cityArea>Laboratory of Clinical Infectious Diseases, National Institute of Allergy and Infectious Diseases, Bethesda</wicri:cityArea></affiliation></author><author><name sortKey="Hickstein, Dennis D" sort="Hickstein, Dennis D" uniqKey="Hickstein D" first="Dennis D." last="Hickstein">Dennis D. Hickstein</name><affiliation wicri:level="2"><nlm:aff id="A3">Experimental Transplantation and Immunology Branch, National Cancer Institute, Bethesda, Maryland</nlm:aff><country xml:lang="fr">États-Unis</country><placeName><region type="state">Maryland</region></placeName><wicri:cityArea>Experimental Transplantation and Immunology Branch, National Cancer Institute, Bethesda</wicri:cityArea></affiliation></author></titleStmt><publicationStmt><idno type="wicri:source">PMC</idno><idno type="pmid">25111582</idno><idno type="pmc">4253545</idno><idno type="url">http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4253545</idno><idno type="RBID">PMC:4253545</idno><idno type="doi">10.1016/j.bbmt.2014.08.004</idno><date when="2014">2014</date><idno type="wicri:Area/Pmc/Corpus">003A39</idno><idno type="wicri:explorRef" wicri:stream="Pmc" wicri:step="Corpus" wicri:corpus="PMC">003A39</idno><idno type="wicri:Area/Pmc/Curation">003A38</idno><idno type="wicri:explorRef" wicri:stream="Pmc" 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Hematologic Malignancies, Alberta Health Services, Calgary, Alberta, Canada</nlm:aff><country xml:lang="fr">Canada</country><wicri:regionArea>Division of Hematology and Hematologic Malignancies, Alberta Health Services, Calgary, Alberta</wicri:regionArea><wicri:noRegion>Alberta</wicri:noRegion></affiliation></author><author><name sortKey="Cuellar Rodriguez, Jennifer" sort="Cuellar Rodriguez, Jennifer" uniqKey="Cuellar Rodriguez J" first="Jennifer" last="Cuellar-Rodriguez">Jennifer Cuellar-Rodriguez</name><affiliation wicri:level="2"><nlm:aff id="A2">Laboratory of Clinical Infectious Diseases, National Institute of Allergy and Infectious Diseases, Bethesda, Maryland</nlm:aff><country xml:lang="fr">États-Unis</country><placeName><region type="state">Maryland</region></placeName><wicri:cityArea>Laboratory of Clinical Infectious Diseases, National Institute of Allergy and Infectious Diseases, Bethesda</wicri:cityArea></affiliation></author><author><name sortKey="Gea Banacloche, Juan" sort="Gea Banacloche, Juan" uniqKey="Gea Banacloche J" first="Juan" last="Gea-Banacloche">Juan Gea-Banacloche</name><affiliation wicri:level="2"><nlm:aff id="A3">Experimental Transplantation and Immunology Branch, National Cancer Institute, Bethesda, Maryland</nlm:aff><country xml:lang="fr">États-Unis</country><placeName><region type="state">Maryland</region></placeName><wicri:cityArea>Experimental Transplantation and Immunology Branch, National Cancer Institute, Bethesda</wicri:cityArea></affiliation></author><author><name sortKey="Zerbe, Christa" sort="Zerbe, Christa" uniqKey="Zerbe C" first="Christa" last="Zerbe">Christa Zerbe</name><affiliation wicri:level="2"><nlm:aff id="A2">Laboratory of Clinical Infectious Diseases, National Institute of Allergy and Infectious Diseases, Bethesda, Maryland</nlm:aff><country xml:lang="fr">États-Unis</country><placeName><region type="state">Maryland</region></placeName><wicri:cityArea>Laboratory of Clinical Infectious Diseases, National Institute of Allergy and Infectious Diseases, Bethesda</wicri:cityArea></affiliation></author><author><name sortKey="Calvo, Katherine" sort="Calvo, Katherine" uniqKey="Calvo K" first="Katherine" last="Calvo">Katherine Calvo</name><affiliation wicri:level="2"><nlm:aff id="A4">Department of Laboratory Medicine, NIH Clinical Center, Bethesda, Maryland</nlm:aff><country xml:lang="fr">États-Unis</country><placeName><region type="state">Maryland</region></placeName><wicri:cityArea>Department of Laboratory Medicine, NIH Clinical Center, Bethesda</wicri:cityArea></affiliation></author><author><name sortKey="Hughes, Thomas" sort="Hughes, Thomas" uniqKey="Hughes T" first="Thomas" last="Hughes">Thomas Hughes</name><affiliation wicri:level="2"><nlm:aff id="A5">Department of Pharmacy, NIH Clinical Center, Bethesda, Maryland</nlm:aff><country xml:lang="fr">États-Unis</country><placeName><region type="state">Maryland</region></placeName><wicri:cityArea>Department of Pharmacy, NIH Clinical Center, Bethesda</wicri:cityArea></affiliation></author><author><name sortKey="Hakim, Fran" sort="Hakim, Fran" uniqKey="Hakim F" first="Fran" last="Hakim">Fran Hakim</name><affiliation wicri:level="2"><nlm:aff id="A3">Experimental Transplantation and Immunology Branch, National Cancer Institute, Bethesda, Maryland</nlm:aff><country xml:lang="fr">États-Unis</country><placeName><region type="state">Maryland</region></placeName><wicri:cityArea>Experimental Transplantation and Immunology Branch, National Cancer Institute, Bethesda</wicri:cityArea></affiliation></author><author><name sortKey="Cole, Kristen" sort="Cole, Kristen" uniqKey="Cole K" first="Kristen" last="Cole">Kristen Cole</name><affiliation wicri:level="2"><nlm:aff id="A3">Experimental Transplantation and Immunology Branch, National Cancer Institute, Bethesda, Maryland</nlm:aff><country xml:lang="fr">États-Unis</country><placeName><region type="state">Maryland</region></placeName><wicri:cityArea>Experimental Transplantation and Immunology Branch, National Cancer Institute, Bethesda</wicri:cityArea></affiliation></author><author><name sortKey="Parta, Mark" sort="Parta, Mark" uniqKey="Parta M" first="Mark" last="Parta">Mark Parta</name><affiliation wicri:level="2"><nlm:aff id="A6">Leidos Biomedical Research, Inc, National Institute of Allergy and Infectious Diseases, Bethesda, Maryland</nlm:aff><country xml:lang="fr">États-Unis</country><placeName><region type="state">Maryland</region></placeName><wicri:cityArea>Leidos Biomedical Research, Inc, National Institute of Allergy and Infectious Diseases, Bethesda</wicri:cityArea></affiliation></author><author><name sortKey="Freeman, Alexandra" sort="Freeman, Alexandra" uniqKey="Freeman A" first="Alexandra" last="Freeman">Alexandra Freeman</name><affiliation wicri:level="2"><nlm:aff id="A2">Laboratory of Clinical Infectious Diseases, National Institute of Allergy and Infectious Diseases, Bethesda, Maryland</nlm:aff><country xml:lang="fr">États-Unis</country><placeName><region type="state">Maryland</region></placeName><wicri:cityArea>Laboratory of Clinical Infectious Diseases, National Institute of Allergy and Infectious Diseases, Bethesda</wicri:cityArea></affiliation></author><author><name sortKey="Holland, Steven M" sort="Holland, Steven M" uniqKey="Holland S" first="Steven M." last="Holland">Steven M. Holland</name><affiliation wicri:level="2"><nlm:aff id="A2">Laboratory of Clinical Infectious Diseases, National Institute of Allergy and Infectious Diseases, Bethesda, Maryland</nlm:aff><country xml:lang="fr">États-Unis</country><placeName><region type="state">Maryland</region></placeName><wicri:cityArea>Laboratory of Clinical Infectious Diseases, National Institute of Allergy and Infectious Diseases, Bethesda</wicri:cityArea></affiliation></author><author><name sortKey="Hickstein, Dennis D" sort="Hickstein, Dennis D" uniqKey="Hickstein D" first="Dennis D." last="Hickstein">Dennis D. Hickstein</name><affiliation wicri:level="2"><nlm:aff id="A3">Experimental Transplantation and Immunology Branch, National Cancer Institute, Bethesda, Maryland</nlm:aff><country xml:lang="fr">États-Unis</country><placeName><region type="state">Maryland</region></placeName><wicri:cityArea>Experimental Transplantation and Immunology Branch, National Cancer Institute, Bethesda</wicri:cityArea></affiliation></author></analytic><series><title level="j">Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation</title><idno type="ISSN">1083-8791</idno><idno type="eISSN">1523-6536</idno><imprint><date when="2014">2014</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en"><p id="P2">We treated 14 patients with GATA2 deficiency using a nonmyeloablative allogeneic hematopoietic stem cell transplantation (HSCT) regimen. Four patients received peripheral blood stem cells (PBSC) from matched-related donors (MRD), four patients received PBSC from matched-unrelated donors (URD), four patients received HSC from umbilical cord blood donors (UCB), and two patients received bone marrow cells from haploidentical related donors. MRD and URD recipients received conditioning with three days of fludarabine and 200cGy total body irradiation (TBI). Haploidentical related donor recipients and UCB recipients received cyclophosphamide and two additional days of fludarabine along with the 200 cGY TBI. MRD, URD, and UCB recipients received tacrolimus and sirolimus for post-transplant 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 (NK) cell populations and reversal of the clinical phenotype at a median follow-up of 3.5 years. Two patients rejected the donor graft (one URD and one UCB), and one MRD recipient relapsed with myelodysplastic syndrome (MDS) post-transplant. 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.</p></div></front></TEI><affiliations><list><country><li>Canada</li><li>États-Unis</li></country><region><li>Maryland</li></region></list><tree><country name="Canada"><noRegion><name sortKey="Grossman, Jennifer" sort="Grossman, Jennifer" uniqKey="Grossman J" first="Jennifer" last="Grossman">Jennifer Grossman</name></noRegion></country><country name="États-Unis"><region name="Maryland"><name sortKey="Cuellar Rodriguez, Jennifer" sort="Cuellar Rodriguez, Jennifer" uniqKey="Cuellar Rodriguez J" first="Jennifer" last="Cuellar-Rodriguez">Jennifer Cuellar-Rodriguez</name></region><name sortKey="Calvo, Katherine" sort="Calvo, Katherine" uniqKey="Calvo K" first="Katherine" last="Calvo">Katherine Calvo</name><name sortKey="Cole, Kristen" sort="Cole, Kristen" uniqKey="Cole K" first="Kristen" last="Cole">Kristen Cole</name><name sortKey="Freeman, Alexandra" sort="Freeman, Alexandra" uniqKey="Freeman A" first="Alexandra" last="Freeman">Alexandra Freeman</name><name sortKey="Gea Banacloche, Juan" sort="Gea Banacloche, Juan" uniqKey="Gea Banacloche J" first="Juan" last="Gea-Banacloche">Juan Gea-Banacloche</name><name sortKey="Hakim, Fran" sort="Hakim, Fran" uniqKey="Hakim F" first="Fran" last="Hakim">Fran Hakim</name><name sortKey="Hickstein, Dennis D" sort="Hickstein, Dennis D" uniqKey="Hickstein D" first="Dennis D." last="Hickstein">Dennis D. Hickstein</name><name sortKey="Holland, Steven M" sort="Holland, Steven M" uniqKey="Holland S" first="Steven M." last="Holland">Steven M. Holland</name><name sortKey="Hughes, Thomas" sort="Hughes, Thomas" uniqKey="Hughes T" first="Thomas" last="Hughes">Thomas Hughes</name><name sortKey="Parta, Mark" sort="Parta, Mark" uniqKey="Parta M" first="Mark" last="Parta">Mark Parta</name><name sortKey="Zerbe, Christa" sort="Zerbe, Christa" uniqKey="Zerbe C" first="Christa" last="Zerbe">Christa Zerbe</name></country></tree></affiliations></record>Pour manipuler ce document sous Unix (Dilib)
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