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.

Tewari, Priti

Martin, Paul L

Mendizabal, Adam

Parikh, Suhag H

Page, Kristin M

Driscoll, Timothy A

Malech, Harry L

Kurtzberg, Joanne

Prasad, Vinod K

2022-03-23T20:30:16Z

2022-03-23T20:30:16Z

2012-09

2022-03-23T20:30:16Z

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.

S1083-8791(12)00063-8

1083-8791

1523-6536

https://hdl.handle.net/10161/24692

eng

Elsevier BV

Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation

10.1016/j.bbmt.2012.02.002

Granulocytes

Transplantation Chimera

Humans

Granulomatous Disease, Chronic

Graft vs Host Disease

Myeloablative Agonists

Histocompatibility Testing

Disease-Free Survival

Transplantation Conditioning

Bone Marrow Transplantation

Cord Blood Stem Cell Transplantation

Transplantation, Homologous

Drug Administration Schedule

Siblings

Quality of Life

Child

Child, Preschool

Infant

Female

Male

Unrelated Donors

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.

Journal article

Martin, Paul L|0000-0001-8141-5678

Parikh, Suhag H|0000-0002-6066-9852

Page, Kristin M|0000-0001-9670-8828

Kurtzberg, Joanne|0000-0002-3370-0703

1368

1377

9

Duke

School of Medicine

Faculty

Clinical Science Departments

Institutes and Centers

Pathology

Pediatrics

Duke Cancer Institute

Institutes and Provost's Academic Units

Initiatives

Duke Innovation & Entrepreneurship

Pediatrics, Transplant and Cellular Therapy

Published

18