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What Makes Cord Blood Transplants Ideal For Pediatric Non-Malignant Disorders!

What Makes Cord Blood Transplants Ideal For Pediatric Non-Malignant Disorders!

Written By Umme Sadiya Qamar - December 29, 2023
Read Time - 4 min read

Bank your baby's stem cells at the time of delivery!

Are you a soon-to-be parent who’s been receiving a lot of this advice, lately? Wondering what the fuss is all about? Then here's something worth considering while making this important decision.

Globally, about 40,000 umbilical cord blood transplants have been performed for various malignant and non-malignant disorders1. In fact, umbilical cord blood (UCB) is used for transplants for a majority of children with inherited metabolic diseases.

Even in children with primary immunodeficiency disorders, bone marrow failure syndromes, and hemoglobinopathies (like sickle cell disease & thalassemia), UCB transplants continue to demonstrate improved outcomes1.

If all this wasn't enough good news, here comes another scientific breakthrough moment for umbilical cord blood transplants.

A recent study evaluated the 2-year post-transplant survival rates of children who underwent UCB transplants for the treatment of non-malignant (non-cancerous) disorders. The results of this evaluation showed an improved survival rate of the patients which was as high as 91%! What’s even more surprising is that these patients did not require serotherapy* during the entire duration of the study2.

But the major outcome of this study puts unmatched umbilical cord blood as a reliable donor source in the absence of a matched sibling donor for the treatment of pediatric non-malignant disorders2.

Curious already? Then, without further ado, let’s dive straight into uncovering the details of this study…

Umbilical Cord Blood - What Makes It An Ideal Donor Source?

The medical fraternity often considers a matched sibling donor as the ‘gold standard’ for transplants3. However, getting a matching sibling donor is not logically possible, always. Also, so far there is no consensus on the best possible donor in the absence of a matched related donor (MRD) for children with non-malignant disorders and immune deficiencies2

The current study at Texas Children’s hospital, therefore, attempts to shine a light on how stem cells from cord blood can fill this gap. To do so, it evaluated the 2-year survival rate of children with non-malignant disorders (Bone Marrow Failure, Hurler, Krabbes etc), who received matched unrelated umbilical cord blood transplantation (UCBT). What’s also important to note is that these transplants did not include the usual serotherapy2.

*Serotherapy is a widely used practice wherein antibody agents like Anti-Thymocyte globulin / Anti-T-Lymphocyte globulin are injected after pediatric hematopoietic stem cell transplantation4. This is done to ensure reduced incidences of the Graft-vs-Host-disease (GvHd), where the donor stem cells perceive the recipient’s body as a foreign and mount  an attack against it5.

However, serotherapy comes with its own challenges as overdosing has been found to increase incidences of infection and relapse, whereas under-dosing can raise chances of GvHd4. Therefore, the exclusion of serotherapy during pediatric umbilical cord blood transplantations only acts as a bonus.

Now, let’s learn more details about the study and its subjects.

What The Study Was About

The study was carried out at Texas Children's Hospital, Baylor College of Medicine. About 55 children who were undergoing UCBT at the hospital's Blood and Marrow Transplantation unit were enrolled into the prospective trial from September 2009 to February 2020.

The patients included 42 males and 13 females between ages 1 month to 9 years, with a median age during transplantation being 5 months.

Their diagnosis was as follows:

Inclusion Criteria

Children who were considered eligible for enrolment into this study were those with congenital or acquired immunological, metabolic or hematological disorder lacking a 10/10 HLA A, B, C, DR, and DQ matched family donor.

These children were then provided with appropriate supportive care. During this time, strict weight management and fluid control was maintained, with patients being limited to <5% weight gain from the time of initial hospitalization.

Following the supportive care, the patients received HLA-matched units for transplantation.


HLA-Matching Criteria

For this study, the enrolled patients received HLA-matched units based on original HLA criteria (low-intermediate resolution molecular at Class I A & B and high resolution at HLA Class II DRB1).

Of the total patients, 35 (64%) received a 5/6 match and 20 (36%) a 6/6 match graft from the umbilical cord blood of an unrelated donor.

The median total nucleated cell dose was 15x10e7 cells /kg (range: 537e10/kg).

The transplantation outcomes were measured for: overall survival, engraftment, engraftment syndrome, GvHD, lung injury, infections, and immune reconstitution. 

Let us check how the Umbilical cord blood transplantation with a matched unrelated donor, fared over two years on the measured parameters.

What The Outcome Suggests

Following the 2-year evaluation, the study revealed outstanding results such as:

  • The overall survival rate of the patients at 2 years was as high as 91%
  • The neutrophil recovery median time  was achieved in 17 days (range: 5-39 days). Neutrophils are a type of white blood cells that help fight infections and thus perform an important function in the human immune system6.
  • The platelet recovery median time was achieved in 37 days (range: 20-92 days).
  • By day 100, the collective incidence rate of acute GvHD (grade II-IV) was 16% (n=9).
  • All patients with viral infections at the time of transplant cleared the infection at a median time of 54 days (range: 44-91 days).
  • All evaluable patients had achieved correction of their metabolic and immune defects.

The overall key points of these results highlight that:

  1. Umbilical Cord Blood Transplantation is a reliable donor source in the absence of a matched sibling for the treatment of pediatric non-malignant disorders.
  2. When treated without serotherapy, Umbilical Cord Blood transplantations could prove to be lifesaving in immune deficiency disorders, even with active infections2

Now, isn’t this something you’d proudly flaunt to all those who opposed your decision to bank your baby’s stem cells?

In A Nutshell

Cord blood stem cell banking has been gaining momentum for a while now. However, it’s a once-in-a-lifetime decision that parents need to make before their baby’s delivery. Parents who chose LifeCell’s Community Banking Program in order to assure their family and baby’s future good health swear by its immense benefits.

For one, this program not only benefits the baby, but also the baby’s siblings, parents, and grandparents, all of whom can have a fair chance of finding unrelated matching cord blood units from the community registry when the need arises.

What’s more? The community banking program also comes with an added advantage of free unlimited stem cell retrievals as well as financial assistance of 20 lakhs towards the treatment costs for parents and children. 

For those expectant parents who are still confused, the results of this study on pediatric UCBT outcomes for non-malignant disorders can be a sufficient-enough convincing factor. So, why wait any longer? Go ahead and ensure you give the best gift to your baby before its arrival - the gift of good health!

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