TY - JOUR
T1 - Myeloablative Transplantation using either Cord Blood or Bone Marrow leads to Immune Recovery, High Long-Term Donor Chimerism and Excellent Survival in Chronic Granulomatous Disease
AU - Tewari, Priti
AU - Martin, Paul L.
AU - Mendizabal, Adam
AU - Parikh, Suhag H.
AU - Page, Kristin M.
AU - Driscoll, Timothy A.
AU - Malech, Harry L.
AU - Kurtzberg, Joanne
AU - Prasad, Vinod K.
N1 - Funding Information:
Financial disclosure: Partial financial support was provided by the National Heart Lung and Blood Institute of the National Institutes of Health.
Funding Information:
Authorship statement: Priti Tewari was involved with conceptualizing the study, collection of clinical data, analysis of the data, writing of the manuscript, and all the coordination efforts; Vinod K. Prasad was involved in conceptualizing the study, analysis of the data, collection of clinical data, editing the manuscript, and direct contribution in the generation of graft data; Joanne Kurtzberg was involved in conceptualizing the study, collection of clinical data, editing the manuscript, and direct contribution in the generation of graft data; Suhag H. Parikh, Timothy A. Driscoll, Kristin M. Page, and Paul L. Martin were involved with collection of clinical data and preparation of the manuscript; Harry L. Malech provided critical input in preparation and editing of the manuscript and is supported by the Intramural Program of the National Institute of Allergy and Infectious Diseases, NIH .
PY - 2012/9
Y1 - 2012/9
N2 - The curative potential of hematopoietic stem cell transplantation in patients with chronic granulomatous disease depends on availability of a suitable donor, successful donor engraftment, and maintenance of long-term donor chimerism. Twelve consecutive children (median age, 59.5 months; range, 8-140 months) with severe chronic granulomatous disease (serious bacterial/fungal infections pretransplantation; median, 3; range, 2-9) received myeloablative hematopoietic stem cell transplantation using sibling bone marrow ([SibBM]; n = 5), unrelated cord blood (UCB; n = 6), and sibling cord blood (n = 1) at our center between 1997 and 2010. SibBM and sibling cord blood were HLA matched at 6/6, whereas UCB were 5/6 (n = 5) or 6/6 (n = 1). Recipients of SibBM were conditioned with busulfan and cyclophosphamide ± anti-thymocyte globulin (ATG), whereas 6 of 7 cord blood recipients received fludarabine/busulfan/cyclophosphamide/ATG. Seven patients received granulocyte-colony stimulating factor-mobilized granulocyte transfusions from directed donors. The first 2 UCB recipients had primary graft failure but successfully underwent retransplantation with UCB. Highest acute graft-versus-host disease was grade III (n = 1). Extensive chronic graft-vs-host disease developed in 3 patients. All patients are alive with median follow-up of 70.5 months (range, 12-167 months) with high donor chimerism (>98%, n = 10; 94%, n = 1; and 92%, n = 1). Myeloablative hematopoietic stem cell transplantation led to correction of neutrophil dysfunction, durable donor chimerism, excellent survival, good quality of life, and low incidence of graft-vs-host disease regardless of graft source.
AB - The curative potential of hematopoietic stem cell transplantation in patients with chronic granulomatous disease depends on availability of a suitable donor, successful donor engraftment, and maintenance of long-term donor chimerism. Twelve consecutive children (median age, 59.5 months; range, 8-140 months) with severe chronic granulomatous disease (serious bacterial/fungal infections pretransplantation; median, 3; range, 2-9) received myeloablative hematopoietic stem cell transplantation using sibling bone marrow ([SibBM]; n = 5), unrelated cord blood (UCB; n = 6), and sibling cord blood (n = 1) at our center between 1997 and 2010. SibBM and sibling cord blood were HLA matched at 6/6, whereas UCB were 5/6 (n = 5) or 6/6 (n = 1). Recipients of SibBM were conditioned with busulfan and cyclophosphamide ± anti-thymocyte globulin (ATG), whereas 6 of 7 cord blood recipients received fludarabine/busulfan/cyclophosphamide/ATG. Seven patients received granulocyte-colony stimulating factor-mobilized granulocyte transfusions from directed donors. The first 2 UCB recipients had primary graft failure but successfully underwent retransplantation with UCB. Highest acute graft-versus-host disease was grade III (n = 1). Extensive chronic graft-vs-host disease developed in 3 patients. All patients are alive with median follow-up of 70.5 months (range, 12-167 months) with high donor chimerism (>98%, n = 10; 94%, n = 1; and 92%, n = 1). Myeloablative hematopoietic stem cell transplantation led to correction of neutrophil dysfunction, durable donor chimerism, excellent survival, good quality of life, and low incidence of graft-vs-host disease regardless of graft source.
KW - Chronic granulomatous disease
KW - Cord blood
KW - Myeloablative
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U2 - 10.1016/j.bbmt.2012.02.002
DO - 10.1016/j.bbmt.2012.02.002
M3 - Article
C2 - 22326631
AN - SCOPUS:84865170098
SN - 1083-8791
VL - 18
SP - 1368
EP - 1377
JO - Biology of Blood and Marrow Transplantation
JF - Biology of Blood and Marrow Transplantation
IS - 9
ER -