A Phase II Study of Umbilical Cord Blood Transplantation
Summary
This study is designed to determine whether Umbilical Cord Transplantation (UCB) can be substituted for adult bone marrow cells in the standard stem cell transplant regimens used at this hospital for subjects who do not have stem cell donors.
Description
Allogeneic stem cell transplantation (SCT) following myeloablative and non-myeloablative conditioning therapy has proven curative treatment for a number of inherited and acquired hematologic disorders. The success of allogeneic transplantation is largely determined by compatibility between donor and recipient, which predicts the risk of fatal graft-versus-host disease (GVHD). Unfortunately, less than one third of patients needing an allogeneic transplant have an available compatible donor in their family. Registries have been established to match patients with compatible volunteer (unrelated) donors, but many patients, and in particular minority patients, still lack stem cell donors.
Umbilical cord blood (UCB) is a rich source of hematopoietic stem cells, which is readily available from the placenta following childbirth. Blood banks have been established in the United States and abroad to collect, process and store UCB for use in allogeneic transplantation. To date, more than 2000 UCB transplants have been performed in adults and children around the world.
Rationale for use of Umbilical Cord Blood in Transplantation
UCB has a number of proven and theoretical advantages as an alternative source of hematopoietic stem cells for transplantation:
1. Placental or umbilical cord blood is an abundantly available source of stem cells, which is currently discarded and can be harvested at no risk to the mother or infant.
2. Important infectious agents, particularly CMV, are much less common in the newborn than adults, and are less likely to contaminate UCB collections.
3. UCB collections, typed, cryopreserved and banked, are available on demand, eliminating delays and uncertainties that now complicate marrow collection from unrelated donors. At present, UCB can be delivered for infusion within days of the initiation of a search. This compares with a median of 3 months from search to delivery of stem cells through the registries of volunteer adult donors.
4. The intensity of graft-versus-host reactivity of fetal lymphocytes appears to be less than that of adult cells and consequently fetal lymphocytes are more tolerant of HLA incompatibility. Published studies have shown that transplantation of UCB matched at 4-5/6 antigens results in a comparable incidence of GVHD to transplantation of unrelated stem cells fully matched at 6/6 antigens.
5. Frozen UCB can be easily shipped, stored at the treating institution, and thawed for use when needed, compared to freshly donated stem cells which have a limited shelf-life of one day or less, necessitating coordination between harvesting surgeons, transportation, and transplantation teams.
This research study has been designed for people who have been diagnosed with a blood tumor, which has not responded to treatment or has recurred, a bone marrow failure state such as aplastic anemia, or one of certain inherited metabolic disorders; and whose doctor feels the best treatment is an allogeneic stem cell transplant (alloSCT) but a related or unrelated adult donor is not available. Instead, a single unit of umbilical cord blood (UCB) will be used as the source of the subject's immune system. This study is designed to determine whether a single unit of UCB can be substituted for adult bone marrow cells in the standard stem cell transplant regimens used at this hospital for subjects who do not have stem cell donors.
Study Design
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Conditions
Leukemia
Intervention
Umbilical Cord Blood Transplantation After Myeloablative Conditioning, Umbilical Cord Blood Transplantation After Reduced-Intensity Conditioning
Location
Tufts Medical Center
Boston
Massachusetts
United States
02111
Status
Recruiting
Source
Tufts Medical Center
Results (where available)
Links
- Source: http://clinicaltrials.gov/show/NCT00676806
- Information obtained from ClinicalTrials.gov on July 15, 2010
Medical and Biotech [MESH] Definitions
Umbilical Veins
Venous vessels in the umbilical cord. They carry oxygenated, nutrient-rich blood from the mother to the FETUS via the PLACENTA. In humans, there is normally one umbilical vein.
Umbilical Arteries
Specialized arterial vessels in the umbilical cord. They carry waste and deoxygenated blood from the FETUS to the mother via the PLACENTA. In humans, there are usually two umbilical arteries but sometimes one.
Fetal Blood
Blood of the fetus. Exchange of nutrients and waste between the fetal and maternal blood occurs via the PLACENTA. The cord blood is blood contained in the umbilical vessels (UMBILICAL CORD) at the time of delivery.
Cord Blood Stem Cell Transplantation
Transplantation of STEM CELLS collected from the fetal blood remaining in the UMBILICAL CORD and the PLACENTA after delivery. Included are the HEMATOPOIETIC STEM CELLS.
Hernia, Umbilical
A HERNIA due to an imperfect closure or weakness of the umbilical ring. It appears as a skin-covered protrusion at the UMBILICUS during crying, coughing, or straining. The hernia generally consists of OMENTUM or SMALL INTESTINE. The vast majority of umbilical hernias are congenital but can be acquired due to severe abdominal distention.
Clinical Trials
Umbilical Cord Blood Transplantation As Treatment Of Adult Patients With Hematologic Disorders
To determine the time to and rate of hematologic engraftment following unrelated umbilical cord blood transplantation in adults with one or two cord blood units using total body irradiatio...
Phase I Study of Umbilical Cord Blood Transplantation Followed by Third Party Thymus Transplantation
The objective of this trial is to assess the toxicity of thymus transplantation following unrelated umbilical cord blood transplantation. Emphasis will be placed on adverse events that ar...
This is a pilot study designed to evaluate the safety and feasibility of performing umbilical cord blood transplants in adults with high-risk hematopoietic malignancies. A novel myeloablat...
The purpose of this research study is to determine the effects of 16, 16 Dimethyl-Prostaglandin E2 (dmPGE2) treatment on umbilical cord blood units to be used in transplantation. dmPGE2 t...
The purpose of this study is to determine if the administration of parathyroid hormone after a sequential dual umbilical cord blood transplant can improve the time to engraftment.
PubMed Articles
Umbilical cord blood-derived mesenchymal cell fate after mouse umbilical cord blood transplantation.
Effectiveness of double umbilical cord blood (dUCB) grafts relative to conventional marrow and mobilized peripheral blood from related and unrelated donors has yet to be established. We studied 536 pa...
Umbilical cord blood: a guide for primary care physicians.
Umbilical cord blood stem cell transplants are used to treat a variety of oncologic, genetic, hematologic, and immunodeficiency disorders. Physicians have an important role in educating, counseling, a...
Milestones in umbilical cord blood transplantation.
Much has been learned about umbilical cord blood (UCB) since the first human cord blood transplant was performed back in 1988. Cord blood banks have been established worldwide for the collection, cryo...
BACKGROUND: Umbilical cord blood transplantation (UCBT) is increasingly used and produces similar results to matched unrelated donor transplantation. METHODS: We performed a retrospective single-cente...
