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.
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.
Type
Journal articleSubject
GranulocytesTransplantation 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
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https://hdl.handle.net/10161/24692Published Version (Please cite this version)
10.1016/j.bbmt.2012.02.002Publication Info
Tewari, Priti; Martin, Paul L; Mendizabal, Adam; Parikh, Suhag H; Page, Kristin M;
Driscoll, Timothy A; ... Prasad, Vinod K (2012). 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. Biology of blood and marrow transplantation : journal of the American Society for
Blood and Marrow Transplantation, 18(9). pp. 1368-1377. 10.1016/j.bbmt.2012.02.002. Retrieved from https://hdl.handle.net/10161/24692.This is constructed from limited available data and may be imprecise. To cite this
article, please review & use the official citation provided by the journal.
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Show full item recordScholars@Duke
Timothy Alan Driscoll
Assistant Professor of Pediatrics
Dr. Driscoll participates in multi-institutional studies for the treatment of high
risk neuroblastoma patients using high dose chemotherapy with stem cell transplant
and the development of new therapies for high risk neuroblastoma patients.
Joanne Kurtzberg
Jerome S. Harris Distinguished Professor of Pediatrics
Dr. Kurtzberg is an internationally renowned expert in pediatric hematology/oncology,
pediatric blood and marrow transplantation, umbilical cord blood banking and transplantation,
and novel applications of cord blood and birthing tissues in the emerging fields of
cellular therapies and regenerative medicine. Dr. Kurtzberg serves as the Director
of the Marcus Center for Cellular Cures (MC3), Director of the Pediatric Transplant
and Cellular Therapy Program, Director of the Carolina
Paul Langlie Martin
Professor of Pediatrics
For most of my career in Pediatric Hematology/Oncology I have focused on the use of
stem cell transplant for the treatment of pediatric leukemias (ALL, AML, CML and JMML)
and other non-malignant blood disorders, such as sickle cell disease, hemaphagocytic
disorders, Wiskott-Aldrich, aplastic anemia, Diamond-Blackfan Anemia, as well as inherited
metabolic diseases. In addition to focusing on determining the best use of stem cell
transplants for these disorders, I have also been involved in clinic
Kristin Marie Page
Assistant Professor of Pediatrics
Stem cell transplantation and/or cellular therapies can be used to treat a variety
of pediatric diseases including malignancies such as leukemia, lymphoma and myelodysplastic
syndrome in addition to certain non-malignant conditions (such as immune deficiencies,
inherited metabolic diseases, hemoglobinopathies, and bone marrow failure syndromes).
As the Director of the Pediatric Transplant and Cellular Therapy Survivorship Clinic,
my goal is optimize the care of survivors of pediatric stem cell t
Suhag H. Parikh
Associate Professor of Pediatrics
Stem cell transplantation for a variety of disorders - ranging from malignant diseases
such as leukemia, lymphoma and myelodysplastic syndrome to nonmalignant diseases such
as sickle cell disease, thalassemias, aplastic anemia, histiocytosis and leukodystrophies.
My clinical research interest is stem cell transplantation for children with primary
immune deficiency disorders and hemoglobinopathies such as sickle cell anemia,thalassemia
and other non-malignant disorders. In addition,I am intereste
Vinod K. Prasad
Professor of Pediatrics
1. Expanding the role of umbilical cord blood transplants for inherited metabolic
disorders. 2. Impact of histocompatibility and other determinants of alloreactivity
on clinical outcomes of unrelated cord blood transplants. 3. Studies to analyse the
impact of Killer Immunoglobulin receptors on the outcomes of hematopoietic stem cell
transplantation utilizing haploidentical, CD34 selected, familial grafts. 4. Propective
longitudinal study of serial monitoring of adenovirus in
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