Brief report. Cure of  -thalassaemia major by umbilical cord blood transplantation - a case report of Malaysia's first cord blood transplantation

Lee-Lee, Chan

doi:10.1093/tropej/45.4.243

Cited by 3 publications

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Alternative Donor/Unrelated Donor Transplants for the β-Thalassemia and Sickle Cell Disease

Fitzhugh¹,

Abraham²,

Hsieh³

2017

Advances in Experimental Medicine and Biology

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Considerable progress with respect to donor source has been achieved in allogeneic stem cell transplant for patients with hemoglobin disorders, with matched sibling donors in the 1980s, matched unrelated donors and cord blood sources in the 1990s, and haploidentical donors in the 2000s. Many studies have solidified hematopoietic progenitors from matched sibling marrow, cord blood, or mobilized peripheral blood as the best source-with the lowest graft rejection and graft versus host disease (GvHD), and highest disease-free survival rates. For patients without HLA-matched sibling donors, but who are otherwise eligible for transplant, fully allelic matched unrelated donor (8/8 HLA-A, B, C, DRB1) appears to be the next best option, though an ongoing study in patients with sickle cell disease will provide data that are currently lacking. There are high GvHD rates and low engraftment rates in some of the unrelated cord transplant studies. Haploidentical donors have emerged in the last decade to have less GvHD; however, improvements are needed to increase the engraftment rate. Thus the decision to use unrelated cord blood units or haploidentical donors may depend on the institutional expertise; there is no clear preferred choice over the other. Active research is ongoing in expanding cord blood progenitor cells to overcome the limitation of cell dose, including the options of small molecule inhibitor compounds added to ex vivo culture or co-culture with supportive cell lines. There are inconsistent data from using 7/8 or lower matched unrelated donors. Before routine use of these less matched donor sources, work is needed to improve patient selection, conditioning regimen, GvHD prophylaxis, and/or other strategies.

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Alternative Donor/Unrelated Donor Transplants for the β-Thalassemia and Sickle Cell Disease

Fitzhugh¹,

Abraham²,

Hsieh³

2017

Advances in Experimental Medicine and Biology

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Umbilical cord blood transplants

Gluckman

Locatelli

2000

Current Opinion in Hematology

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With the establishment of cord blood banks, the number of related and unrelated umbilical cord blood transplants is increasing worldwide. Close links have been established with the cord blood banks. Available data showed that umbilical cord blood transplants offer overall results comparable to those obtained with related or unrelated bone marrow transplants. Several differences were found: engraftment with cord blood was delayed, resulting in an increased incidence of early transplant complications, and the incidence of acute and chronic graft-versus-host disease was significantly reduced with cord blood grafts, even in HLA-mismatched transplants and in adults. In patients with leukemia, the rate of relapse appeared to be similar to that documented in bone marrow transplant recipients. These data confirm the potential benefit of using umbilical cord blood hematopoietic stem cells for allogeneic transplants.

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