ArticleBiology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation2012
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.
Article in Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.
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Who cites it
17 citing papers in PubMed, 54 citations in OpenAlex.
- A prospective study of allogeneic transplantation from unrelated donors for chronic granulomatous disease with target busulfan-based reduced-intensity conditioning.Bone marrow transplantation · 2019Trial
- Treosulfan-based conditioning and hematopoietic cell transplantation for nonmalignant diseases: a prospective multicenter trial.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2014Trial
- Quality of Life and Social and Psychological Outcomes in Adulthood Following Allogeneic HSCT in Childhood for Inborn Errors of Immunity.Journal of clinical immunology · 2022Article
- Article
- Geographic Variability and Pathogen-Specific Considerations in the Diagnosis and Management of Chronic Granulomatous Disease.Pediatric health, medicine and therapeutics · 2020Review
- Cellular Therapies in Chronic Granulomatous Disease.Frontiers in pediatrics · 2020Review
- Hematopoietic Cell Transplantation for Chronic Granulomatous Disease in Japan.Frontiers in immunology · 2020Article
- Role of Allogeneic Hematopoietic Stem Cell Transplant for Chronic Granulomatous Disease (CGD): a Report of the United States Immunodeficiency Network.Journal of clinical immunology · 2019Article
- A Novel Mutation in Chronic Granulomatous Disease: Treating the Family, Not Just the Patient.Frontiers in pediatrics · 2019Article
- Allogeneic Hematopoietic Cell Transplantation for Chronic Granulomatous Disease: Controversies and State of the Art.Journal of the Pediatric Infectious Diseases Society · 2018Review
- A Review of Chronic Granulomatous Disease.Advances in therapy · 2017Review
- Allogeneic Reduced-Intensity Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease: a Single-Center Prospective Trial.Journal of clinical immunology · 2017Article
- Chronic Granulomatous Disease.Journal of clinical and diagnostic research : JCDR · 2015Article
- Stem cell transplantation for primary immunodeficiency diseases: the North American experience.Current opinion in allergy and clinical immunology · 2014Review
- Reactive oxygen species regulate hematopoietic stem cell self-renewal, migration and development, as well as their bone marrow microenvironment.Antioxidants & redox signaling · 2014Review
- Successful HLA haploidentical hematopoietic SCT in chronic granulomatous disease.Bone marrow transplantation · 2014Article
- Gastrointestinal histopathology in chronic granulomatous disease: a study of 87 patients.The American journal of surgical pathology · 2013Article
Corrections and comments
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Authors and funding
9 authors at 4 institutions in 1 country.
Funding
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.
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