Durable engraftment and correction of hematological abnormalities in children with congenital amegakaryocytic thrombocytopenia following myeloablative umbilical cord blood transplantation.
Abstract
The use of HSCT is the only potentially curative treatment for CAMT, but access is
limited by the availability of suitable donors. We report five consecutive patients
with CAMT who received MAC and partially HLA-mismatched, UCBT (unrelated, n = 4).
Median times to neutrophil (>500/μL) and platelet (≥20 000 and ≥50 000/μL) engraftment
were 19, 57, and 70 days, respectively. Acute GvHD, grade II, developed in one patient,
who subsequently developed limited chronic GvHD. At median follow-up of 14 yr, all
patients are alive with sustained donor cell engraftment. To our knowledge, this is
the largest single-center series of UCBT for patients with this disease and suggests
that UCBT is a successful curative option for patients with CAMT.
Type
Journal articleSubject
HumansThrombocytopenia
Graft vs Host Disease
Treatment Outcome
Transplantation Conditioning
Cord Blood Stem Cell Transplantation
Follow-Up Studies
Adolescent
Child
Child, Preschool
Infant
Infant, Newborn
Female
Male
Congenital Bone Marrow Failure Syndromes
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https://hdl.handle.net/10161/24646Published Version (Please cite this version)
10.1111/petr.12577Publication Info
Mahadeo, Kris M; Tewari, Priti; Parikh, Suhag H; Driscoll, Timothy A; Page, Kristin;
Martin, Paul L; ... Prasad, Vinod K (2015). Durable engraftment and correction of hematological abnormalities in children with
congenital amegakaryocytic thrombocytopenia following myeloablative umbilical cord
blood transplantation. Pediatric transplantation, 19(7). pp. 753-757. 10.1111/petr.12577. Retrieved from https://hdl.handle.net/10161/24646.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
Kris Mahadeo
Professor of Pediatrics
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
Consulting Professor in the Department 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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