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.

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Citation

Published Version (Please cite this version)

10.1111/petr.12577

Publication Info

Mahadeo, Kris M, Priti Tewari, Suhag H Parikh, Timothy A Driscoll, Kristin Page, Paul L Martin, Joanne Kurtzberg, Vinod K Prasad, et al. (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.

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Scholars@Duke

Mahadeo

Kris Mahadeo

Professor of Pediatrics

As a committed life-long learner, I am awed by the continued advancements in the field of pediatric transplant and cellular therapy (PTCT).   Following completion of my PTCT fellowship training at Duke, I have developed a career dedicated to expanding indications for PTCT and improving associated outcomes.  I have a special interest in the mitigation of endotheliopathies and continue to lead global harmonization efforts related to management of toxicities of therapy.  As the Division Chief of PTCT at Duke, I am committed to the ensuring our patients receive state of the art care with access to innovative research protocols that change the field.   We are committed to training the next generation of physicians in our field with a commitment to global partnerships to improve care for patients across the world.

Driscoll

Timothy Alan Driscoll

Associate 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.

Martin

Paul Langlie Martin

Professor Emeritus 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 clinical research investigating the prevention and treatment of transplant related morbidity, particularly veno-occlusive disease of the liver, infections and diffuse alveolar hemorrhage. As study chair for the Children's Oncology Group protocol 9904, I was involved in the development, implementation and analysis of a large, international frontline study of childhood acute lymphoblastic leukemia. Results from this study show that a significant number of children with certain favorable cytogenetic abnormalities in their leukemic cells and who have a rapid response to their initial chemotherapy can expect to have a >95% chance of cure when treated with relatively low intensity chemotherapy.  

I have concentrated on providing high quality care for high risk leukemia patients who require high intensity therapies, such as stem cell transplant and immunotherapy.  As a member of the Pediatric Transplant and Cellular Therapy Division I provide clinical care for these patients.  As a member of various cooperative groups and local PI for several drug trials, I have worked to provide better care and more specific therapies for the toxicities associated with stem cell transplant.  

I have also collaborated with the Pediatric Immunology Division to provide a life-saving therapy for a small group of patients with thymic dysfunction, which causes severe immunodeficiency.  Our clinical team now provides support during these patients hospital admissions for donor thymus tissue implantation.  We once again achieved a new record for the number of implanted patients during the 2022-2023 academic year.

Prasad

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 allogenic transpants(SMAART)patients.
5. Use of mesenchymal stem cells for the treatment of GVHD


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