SALT: Alternative Donor Bone Marrow and Cord Blood Transplantation for High Risk Sickle Cell Disease

16:31 EDT 30th August 2014 | BioPortfolio

Summary

We hope to gain valuable information about the safety, success of engraftment, and rates of complications using alternate donor transplantation for children with severe SCD. Crucial information will be also collected about late effects from alternate donor BMT sickle cell, providing valuable information to clinicians and families making decisions among interventions for children with severe sickle cell disease. If successful, alternate donor transplantation in this setting could pave the way to offering curative treatment to many more patients with severe SCD.

Description

Unfortunately, less than 1/4th of patients with severe SCD will have a matched sibling donor that can serve as a BMT donor. This research protocol proposes to study the safety and usefulness of "alternate donor transplant" (using donors other than matched siblings). We will offer this treatment to children with severe sickle cell disease that do not have a matched sibling BMT donor. Alternative donors can be family members who are slightly less than completely matched, unrelated volunteer donors who are completely matched, and donated banked umbilical cord blood that is completely or nearly completely matched.

Alternative donor transplant has been performed commonly in patients with cancer, and also provides curative therapy for several non-malignant diseases (severe immunodeficiency, marrow failure and metabolic storage diseases). Alternate donor transplants carry higher risks of complications, including graft-versus-host disease, infection, and graft failure. Therefore, we will be selective about which patients are invited to participate, limiting eligibility to those patients that have had a severe SCD related problem (rather than those who are doing well and are likely to have few SCD related problems), but excluding patients who have such severe organ damage that they are more likely to die during transplant, and limiting eligibility to a young age group. A multi-step review algorithm that includes internal, local and external expert review has been constructed to provide a thorough, safe and ethical accrual process. We will treat patients using drugs and methods commonly used in alternate donor transplant for other diseases such as leukemia, and incorporate lessons learned from our previous experience in BMT for sickle cell by modifying supportive care measures. Special attention will be given to evaluation of post-BMT effects in this population, as well as potential reasons for adverse effects (such as graft failure).

We think that Atlanta is a particularly good place to study this kind of transplant for several reasons. One reason is experience: our program has transplanted more children with SCD than any other single institution in North America, with excellent outcomes. Additionally, SCD patients in our area often have been treated on a special red cell transfusion program that limits the number of people donating the blood; we think this is likely to reduce the chance of graft failure.

Study Design

Control: Active Control, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Conditions

Sickle Cell Disease

Intervention

Alternative donor bone marrow and cord blood

Location

Children's Healthcare of Altanta
Atlanta
Georgia
United States
30322

Status

Recruiting

Source

Emory University

Results (where available)

View Results

Links

Clinical Trials [2365 Associated Clinical Trials listed on BioPortfolio]

Stem Cell Transplantation With Identical Donors for Patients With Sickle Cell Disease

This protocol studied the effect of administration of a myeloablative pretransplant preparative regimen followed by an infusion of donor stem cells in children with severe sickle cell dise...

Bone Marrow Transplant From Related Donor for Patients With High Risk Hemoglobinopathies

The major goal of this study is to determine the risks and benefits of bone marrow transplants in patients with severe thalassemia or sickle cell disease. Participation in this project wil...

Sickle Cell Disease Treated By Bone Marrow Transplant

Hematopoietic stem cell transplantation (HSCT) is emerging as a therapeutic alternative for patients with sickle cell disease. Conventional HSCT therapy has been limited to extremely high...

New York Blood Center National Cord Blood Program

Umbilical cord blood is used as a source of hematopoietic stem cells for bone marrow reconstition in patients who would be potential candidates for a bone marrow transplant from an unrelat...

Stem Cell Transplant in Sickle Cell Disease and Thalassemia

Sickle cell disease is a genetic disorder in which a mutation in the beta chain of human hemoglobin results in abnormal blood hemoglobin, causing red blood cells to sickle under stress wit...

PubMed Articles [31984 Associated PubMed Articles listed on BioPortfolio]

Donor-derived 47, XXY in an unrelated cord blood transplant recipient.

A 65-year-old Japanese male with therapy-related myelodysplastic syndrome was admitted for unrelated cord blood transplantation. A cord blood unit from a male donor was obtained from the Japan Cord Bl...

Recovery of CD45(-)/Lin(-)/SSEA-4(+) very small embryonic-like stem cells by cord blood bank standard operating procedures.

Very small embryonic-like (VSEL) stem cells are a rare cell population present in bone marrow, cord blood and other tissues that displays a distinct small cell size and the ability to give rise to cel...

Facial Swelling in a Sickle Cell Patient.

Sickle cell disease (SCD) is characterized as a chronic hemolytic anemia with vaso-occlusive crises that result in multisystem organ damage. Bone marrow is one of the more common sites of these crises...

Haematopoietic stem cell transplantation for severe sickle cell disease in childhood: a single centre experience of 50 patients.

Despite improvements in medical management, sickle cell disease (SCD) remains associated with severe morbidity and decreased survival. Allogeneic haematopoietic stem cell transplantation (HSCT) remain...

Non-myeloablative peripheral blood haploidentical stem cell transplantation for refractory severe aplastic anemia.

New transplant approaches are urgently needed for patients with refractory severe aplastic anemia (SAA) who lack a matched sibling or unrelated donor (UD), or who have failed an UD or cord blood trans...

Medical and Biotech [MESH] Definitions

Transfer of HEMATOPOIETIC STEM CELLS from BONE MARROW or BLOOD between individuals within the same species (TRANSPLANTATION, HOMOLOGOUS) or transfer within the same individual (TRANSPLANTATION, AUTOLOGOUS). Hematopoietic stem cell transplantation has been used as an alternative to BONE MARROW TRANSPLANTATION in the treatment of a variety of neoplasms.

Transplantation of stem cells collected from the peripheral blood. It is a less invasive alternative to direct marrow harvesting of hematopoietic stem cells. Enrichment of stem cells in peripheral blood can be achieved by inducing mobilization of stem cells from the BONE MARROW.

Repetitive withdrawal of small amounts of blood and replacement with donor blood until a large proportion of the blood volume has been exchanged. Used in treatment of fetal erythroblastosis, hepatic coma, sickle cell anemia, disseminated intravascular coagulation, septicemia, burns, thrombotic thrombopenic purpura, and fulminant malaria.

The soft tissue filling the cavities of bones. Bone marrow exists in two types, yellow and red. Yellow marrow is found in the large cavities of large bones and consists mostly of fat cells and a few primitive blood cells. Red marrow is a hematopoietic tissue and is the site of production of erythrocytes and granular leukocytes. Bone marrow is made up of a framework of connective tissue containing branching fibers with the frame being filled with marrow cells.

An abnormal hemoglobin resulting from the substitution of valine for glutamic acid at position 6 of the beta chain of the globin moiety. The heterozygous state results in sickle cell trait, the homozygous in sickle cell anemia.

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