Faculty Collaboration Database

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. Biol Blood Marrow Transplant 2012 Sep;18(9):1368-77

Date

02/14/2012

Pubmed ID

22326631

Pubmed Central ID

PMC3540103

DOI

10.1016/j.bbmt.2012.02.002

Scopus ID

2-s2.0-84865170098 (requires institutional sign-in at Scopus site)   37 Citations

Abstract

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.

Author List

Tewari P, Martin PL, Mendizabal A, Parikh SH, Page KM, Driscoll TA, Malech HL, Kurtzberg J, Prasad VK

Author

Kristin Page MD, MHS, MEd Associate Professor in the Pediatrics department at Medical College of Wisconsin

MESH terms used to index this publication - Major topics in bold

Bone Marrow Transplantation
Child
Child, Preschool
Cord Blood Stem Cell Transplantation
Disease-Free Survival
Drug Administration Schedule
Female
Graft vs Host Disease
Granulocytes
Granulomatous Disease, Chronic
Histocompatibility Testing
Humans
Infant
Male
Myeloablative Agonists
Quality of Life
Siblings
Transplantation Chimera
Transplantation Conditioning
Transplantation, Homologous
Unrelated Donors