Cord blood stem cell transplantation for haemoglobinopathies

Abstract

Summary Despite improvements in supportive care, patients with β‐thalassaemia major or sickle cell disease (SCD) may benefit from haematopoietic stem cell transplantation at some point during their lives. Human leucocyte antigen (HLA)‐matched sibling bone marrow donors are not always available and alternative sources of stem cells have been sought, including related and unrelated donor cord blood transplants (CBT). The outcome of CBT from related donors for the treatment of both thalassaemia major and SCD is now approaching that for bone marrow transplantation, with around 90% of patients surviving disease‐free. The main complication is graft rejection, which may be reduced by increasing pretransplant immune suppression. Transplant‐related mortality following HLA‐identical matched related donor CBT is extremely low but is significant in the small series of unrelated and/or mis‐matched donor CBT. The principal limitation to extending the use of CB stem cells for the cure of haemoglobinopathies is the need to better understand the mechanisms of action and optimal conditioning regimens used to secure long‐term engraftment while minimizing morbidity and mortality. Further biological studies and clinical trials are needed to address this aim.

Therapeutic effects of induced pluripotent stem cells in chimeric mice with -thalassemia

“…14,15 Hematopoietic stem cell or cord blood transplantation are also used as a strategy for partially relieving the symptoms. [16][17][18] However, resources for finding matched bone marrow donors are limited. All of the aforementioned therapies are expensive and can be painful procedures.…”

Unrelated cord blood transplantation in pediatric patients: a report from Saudi Arabia

“…[1][2][3][4][5][6][7][8] In UCB transplants, the degree of HLA matching has been closely associated with long-term survival. Thus, it is now a major goal of public cord blood banking to provide a large and diverse inventory of HLA typing to extend transplant access to racial and ethnic minorities.…”

Fetal hemoglobin chemical inducers for treatment of hemoglobinopathies

“…Furthermore, bone marrow transplantation from an HLAidentical familial donor is associated with a 5% mortality rate; this mortality rate is higher (about 20%) for bone marrow transplantation from non-related HLA-matched donors. Cord blood may be used as an alternative source of hematopoietic stem cells for curative treatment of hemoglobinopathies but had the same limitations described for bone marrow (reviewed in [239]). …”