A novel reduced-intensity conditioning regimen for unrelated umbilical cord blood transplantation in children with nonmalignant diseases.
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Reduced-intensity conditioning (RIC) regimens have the potential to decrease transplantation-related morbidity and mortality. However, engraftment failure has been prohibitively high after RIC unrelated umbilical cord blood transplantation (UCBT) in chemotherapy-naïve children with nonmalignant diseases (NMD). Twenty-two children with a median age of 2.8 years, many with severe comorbidities and prior viral infections, were enrolled in a novel RIC protocol consisting of hydroxyurea, alemtuzumab, fludarabine, melphalan, and thiotepa followed by single UCBT. Patients underwent transplantation for inherited metabolic disorders (n = 8), primary immunodeficiencies (n = 9), hemoglobinopathies (n = 4) and Diamond Blackfan anemia (n = 1). Most umbilical cord blood (UCB) units were HLA-mismatched with median infused total nucleated cell dose of 7.9 × 10(7)/kg. No serious organ toxicities were attributable to the regimen. The cumulative incidence of neutrophil engraftment was 86.4% (95% confidence interval [CI], 65% to 100%) in a median of 20 days, with the majority sustaining > 95% donor chimerism at 1 year. Cumulative incidence of acute graft-versus-host disease (GVHD) grades II to IV and III to IV by day 180 was 27.3% (95% CI, 8.7% to 45.9%) and 13.6% (95 CI, 0% to 27.6%), respectively. Cumulative incidence of extensive chronic GVHD was 9.1% (95% CI, 0% to 20.8%). The primary causes of death were viral infections (n = 3), acute GVHD (n = 1) and transfusion reaction (n = 1). One-year overall and event-free survivals were 77.3% (95% CI, 53.7% to 89.8%) and 68.2% (95% CI, 44.6% to 83.4%) with 31 months median follow-up. This is the first RIC protocol demonstrating durable UCB engraftment in children with NMD. Future risk-based modifications of this regimen could decrease the incidence of viral infections. (www.clinicaltrials.gov/NCT00744692).
Graft vs Host Disease
Common Variable Immunodeficiency
Cord Blood Stem Cell Transplantation
Published Version (Please cite this version)10.1016/j.bbmt.2013.11.021
Publication InfoParikh, Suhag H; Mendizabal, Adam; Benjamin, Cara L; Komanduri, Krishna V; Antony, Jeyaraj; Petrovic, Aleksandra; ... Szabolcs, Paul (2014). A novel reduced-intensity conditioning regimen for unrelated umbilical cord blood transplantation in children with nonmalignant diseases. Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 20(3). pp. 326-336. 10.1016/j.bbmt.2013.11.021. Retrieved from https://hdl.handle.net/10161/24650.
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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.
Jerome S. Harris Distinguished Professor of Pediatrics
Dr. Kurtzberg conducts both clinical and laboratory-based translational research efforts, all involving various aspects of normal and malignant hematopoiesis. In the laboratory, her early work focused on studies determining the mechanisms that regulate the choice between the various pathways of differentiation available to the pluripotent hematopoietic stem cell. Her laboratory established a CD7+ cell line, DU.528, capable of multilineage differentiation as well as self-renewal, and subse
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
Professor of Biostatistics & Bioinformatics
Primary interests include clinical trials design and the design and analysis of biomarker and imaging studies especially in the areas of GI cancer, lymphoma, melanoma, transplant and cancer immunotherapy.
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
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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; ... (9 authors) (Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2012-09)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 ...
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Umbilical cord blood transplantation for children with thalassemia and sickle cell disease. Ruggeri, Annalisa; Eapen, Mary; Scaravadou, Andromachi; Cairo, Mitchell S; Bhatia, Monica; Kurtzberg, Joanne; Wingard, John R; ... (20 authors) (Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2011-09)We examined the efficacy of unrelated cord blood (CB) transplantation in children with thalassemia (n = 35) and sickle cell disease (n = 16), using data reported to 3 registries. Donor-recipient pairs were matched at HLA-A ...