Should families with a thalassemia child collect cord blood?

By Dr. Lawrence Faulkner. Director Sankalp-People Tree Hospitals Bone Marrow Transplant Unit, Bangalore, India and Medical Coordinator Cure2Children Foundation Florence, Italy. The short answer is no. Why than many families with a child with thalassemia collect and store cord blood? Unfortunately this is a consequence of business-oriented and unscientific practices ignoring evidence-based medical recommendations. Of course here I am referring to private banking storing your newborn child cord blood for a fee and not public banking collecting donated cord blood free of charge as a useful source of transplantable stem cell for unrelated individuals. There is no clear medical indication to the use of your child’s own cord blood and very few transplants have been done for thalassemia using compatible newborn sibling cord blood: In 33 patients given related cord blood transplants published in 2003 on the official journal of the American Sociey of Hematology1, 7 patients (21%) rejected the transplant, a rate which is much higher than that seen after bone marrow transplantation (BMT), generally in the 5% range. Moreover, most of these 7 patients who rejected cord blood did not benefit from a second transplant from the same donor using freshly collected bone marrow. In fact, even though second BMTs can be successful, transplantation should be considered a one-shot opportunity to be offered in the best possible circumstances. This is the primary reason why in a situation like thalassemia where there is no need to rush to BMT, it seems far better to collect fresh bone marrow when the compatible newborn grows up to a weight of at least 8 to 10 kilograms. i.e. around 1 year of age, when the collection can be performed safely and transplant success maximized. Another issue with the use of cord blood is an increased infectious risk related to delayed recovery of white blood cells which will also substantially increase costs and hospital stay. Can stored cord blood be beneficial in combination with fresh bone marrow? In an even smaller experience on 13 patients, the co-administration of cord blood and bone marrow from the same sibling donor seemed to be associated with very low risks of rejection or graft versus host disease (GVHD), a complication potentially making health-related quality of life post-BMT worse than the thalassemia itself2. However, in our experience on a much larger number of patients, the rate of rejection and GVHD is also very low after matched sibling BMT, so that there is no compelling evidence that cord blood may help when added to bone marrow. The potential issues raised by the collection of cord blood itself should also be considered: Unless an elective Cesarian section is planned with its added risks and costs, round-the-clock experienced personnel needs to be available for spontaneous deliveries. Lastly, cord blood collection, assessment and cryopreservation requires high standards of quality assurance which are not always guaranteed in the absence of a formal accreditation process which is not currently in place in India.
Because of these reasons our personal belief, based on available evidence, is that cord blood use for thalassemia transplantation is medically not indicated, largely profit-driven and ethically questionable.
References 1. Locatelli, F. et al. Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease. Blood 101, 2137–2143 (2003). 2. Soni, S. et al. Combined umbilical cord blood and bone marrow from HLA-identical sibling donors for hematopoietic stem cell transplantation in children with hemoglobinopathies. Pediatr. Blood Cancer 61, 1690–1694 (2014).
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