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Cord Blood Transplantation: Current Status and Future Directions

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Umbilical cord blood transplantation (UCBT) is currently an effective therapeutic option for children as well as adults with a range of hematologic disorders.

Written by

Dr. Saranya. P

Medically reviewed by

Dr. Abdul Aziz Khan

Published At May 16, 2024
Reviewed AtMay 16, 2024

Introduction:

Treatments for both malignant and non-malignant illnesses are achieved by hematopoietic cell transplants utilizing stem cells from umbilical cord blood. This stem cell source has demonstrated encouraging results in the successful treatment of hereditary metabolic diseases, cancer, hematologic disorders, and immunologic disorders through transplant operations. These stem cells are readily available, which is a significant benefit over other unrelated donor transplants, particularly in circumstances when waiting might have a negative impact on prognosis.

What Is Cord Blood Transplantation?

Stem cells are extracted from umbilical cord blood (UCB) and used in cord blood transplants. Stem cells are undifferentiated or partially differentiated cells with the ability to specialize into multiple cell types and multiply endlessly to generate a greater quantity of identical stem cells. The blood that remains in the umbilical cord following childbirth is known as umbilical cord blood. Until it is needed for a cord blood transplant, the cord blood can be preserved and frozen.

Compared to other bone marrow transplant possibilities, cord blood has fewer stem cells accessible. However, compared to bone marrow-derived stem cells, those found in cord blood have the capacity to produce more blood cells. Since cord blood cells have not been exposed to pathogens, they are also immunologically unaware. As a result, they are more accepting of a patient's tissue mismatch with the cord cells. Transplant patients without a properly matched donor can utilize them.

What Are the Potential Advantages of Cord Blood Stem Cells?

  • While finding and verifying a bone marrow or peripheral blood donor can take several months, cord blood kept in a cord blood bank has already undergone prescreening, testing, and freezing, making it ready for use.

  • Research has shown that the incidence of GVHD was lower in patients who received cord blood stem cell transplants than in those who received bone marrow or peripheral blood transplants, and in those who did, the consequence was typically milder. A dangerous and occasionally lethal side effect of allogeneic stem cell transplantation is GVHD. In GVHD, the patient's healthy tissue (the host) is attacked by the immune cells of the donor (the graft).

  • Donor cord blood units may offer a supply of stem cells that reflects racial diversity by expanding the collection process to hospitals with a high proportion of babies from a variety of ethnic origins.

  • Compared to stem cells from the peripheral blood or bone marrow of related or unrelated donors, cord blood stem cell transplants have a lower risk of spreading blood-borne infectious illnesses.

  • HLA (human leukocyte antigen) compatibility between the recipient and donor cord blood has a significant impact on the outcome of stem cell transplantation, both related and unrelated. Successful engraftment (when the transplanted stem cells are accepted by the human body and start to make new immune cells and blood cells), the severity of graft-versus-host disease (GVHD), and overall survival are all influenced by HLA matching. A patient's post-transplant success may be enhanced by a close match between the donor cord blood unit and the recipient. Though clinical studies indicate that the match may not need to be as close as that required for bone marrow or peripheral blood transplants, a closely matched cord blood unit is still desirable.

What Are the Various Applications of Cord Blood Transplants?

1. Hematological Conditions:

Individuals with leukemia, lymphoma (a group of lymphocyte-derived blood and lymph cancers), myeloma (a particular kind of blood cancer that arises from bone marrow plasma cells), myeloproliferative disorders, genetic illnesses, and metabolic disorders may benefit from umbilical cord blood transplantation. Reduction in transplant-related mortality and greater use in older patients are the results of decreased intensity regimens and double cord blood transplantation in adults. Unrelated donor cord blood transplantation (CBT) has become a commonly accepted treatment for a wide range of hematologic diseases, including acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), and aplastic anemia. It is a safe and reliable substitute for peripheral blood (PB) or bone marrow (BM) transplants.

2. Neurological Conditions:

The proliferative potential of UCB (umbilical cord blood) stem cells is higher than that of stem cells derived from adult bone marrow harvests. Neurologic disorders such as autism, traumatic brain injury, hypoxia ischemic encephalopathy, and cerebral palsy have all been treated with UCB.

3. Cardiovascular Illnesses:

Clinical trials using mesenchymal stem cells produced from UCB are being conducted for ischemic illness and dilated cardiomyopathy in cardiovascular disease.

How to Choose the Optimal UCB Unit for a Transplant?

The best high-resolution HLA matching, sufficient cell dosage, and acceptable quality are the guiding principles for choosing CBUs. Although there are differences in the specific selection criteria used by transplant centers with experience with UCB, there are common principles that all centers recognize, such as the significance of locating a unit with a highly viable CD34 cell dosage and HLA compatibility for four of eight HLA antigens.

What Is Double Cord Blood Transplants?

A significant rate of graft failure and transplant-related death was attributed to the modest total nucleated and CD34+ cell content in a single unit of umbilical cord blood. Experts developed a novel approach that appears to have significantly improved adult engraftment rates and outcomes by mixing two cord blood units to enhance infused cell doses. Double unit cord blood transplantation, or DUCBT, is now standard treatment at many facilities.

How Are Umbilical Cord Blood Stem Cells Multiplied?

Numerous strategies have been employed to grow HPCs (hematopoietic progenitor cells) and HSCs (hematopoietic stem cells) from UCB, including the use of small compounds, feeder stromal cells, and epigenetic modifiers. The stem cell population is exponentially increased using a regimen comprising various cytokines or small compounds, which affects the engraftment rates and shortens the time frame of extreme neutropenia.

What Are the Future Directions?

From being restricted to hematopoietic cell transplant (HCT) for pediatric patients, the application of UCBT has expanded to include adoptive cellular therapy. The medical field has seen remarkable advancements in stem cell expansion therapy that have helped overcome UCB's limits and dramatically enhanced hematopoietic and immunologic recovery. As a result, patients have been hospitalized for shorter periods of time and have used fewer healthcare resources overall. With the more recent methods, the results of umbilical cord blood transplantation are becoming better. Expanding multi-virus-specific T cells (T lymphocytes), NK (natural killer) cells, and in vivo and ex vivo UCB growth will all be important in this new era of UCB-derived cells. Beyond HCT, the growing application of UCB-derived immune therapy will be crucial in maintaining better treatment results.

Conclusion:

Umbilical cord blood is an excellent source of stem cells for transplantation, much like bone marrow and peripheral blood. Compared to bone marrow (BM) and peripheral blood progenitor cells (PBPCs) as transplant sources, UCBT offers several benefits, such as fewer restrictive HLA-matching standards and the availability of cryopreserved specimens in both public and commercial health banks. Moreover, graft-versus-host disease (GvHD) is less common in cord blood due to the immune cells' immature stage.

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Dr. Abdul Aziz Khan
Dr. Abdul Aziz Khan

Medical oncology

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