Abstract
Allogeneic haematopoietic stem cell transplantation (HSCT) is indicated in children with high-risk, relapsed or refractory acute lymphoblastic leukaemia (ALL). HLA-matched grafts from cord blood and stem cell repositories have allowed patients without suitable sibling donors to undergo HSCT. However, challenges in procuring matched unrelated donor (MUD) grafts due to high cost, ethnic disparity and time constraints have led to the exponential rise in the use of stem cells from human leukocyte antigen (HLA)-haploidentical family donors. Whilst HLA-haploidentical HSCT (hHSCT) performed in adult patients with acute leukaemia has produced outcomes similar to MUD transplants, experience in children is limited. Over the last 5 years, more data have emerged on hHSCT in the childhood ALL setting, allowing comparisons with matched donor transplants. The feasibility of hHSCT using adult family donors in childhood ALL may also address the ethical issues related to selection of minor siblings in matched sibling donor transplants. Here, we review hHSCT in paediatric recipients with ALL and highlight the emergence of hHSCT as a promising therapeutic option for patients lacking a suitable matched donor. Recent issues related to conditioning regimens, donor selection and graft-vs.-host disease prophylaxis are discussed. We also identify areas for future research to address transplant-related complications and improve post-transplant disease-free survival.
Highlights
Allogeneic haematopoietic stem cell transplantation (HSCT) is used to consolidate remission in patients with genetic subtypes of childhood acute lymphoblastic leukaemia (ALL) at high risk of relapse as well as those with relapsed or refractory disease
In an international study of 180 children with ALL who received haploidentical HSCT (hHSCT)-post-transplant cyclophosphamide (PTCy), a multivariate analysis found that donor selection based on relationship to recipient did not affect non-relapse mortality (NRM); instead, disease status at transplant, age >13 years and use of peripheral blood stem cell grafts were independent factors associated with decreased OS [46]
The exponential increase in the use of hHSCT for haematological malignancies in the last 10 years has allowed more data to emerge from the paediatric ALL population to guide optimal management choices
Summary
Allogeneic haematopoietic stem cell transplantation (HSCT) is used to consolidate remission in patients with genetic subtypes of childhood acute lymphoblastic leukaemia (ALL) at high risk of relapse as well as those with relapsed or refractory disease. In a retrospective study comparing hHSCT-PTCy to HSCT using an MUD or MMUD after RIC in paediatric patients with acute leukaemia, a group in Italy reported similar outcomes with regards to 5-year OS, NRM and relapse incidence between the three groups [39].
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