Hematopoietic stem cell transplantation (HSCT) still constitutes an important therapeutic option for pediatric patients with acute lymphoblastic leukemia (ALL), despite the overall improvements in cure rates and despite the advent of innovative therapies. However, due to the increasing complexity in patients’ stratification and due to the availability of novel treatments, it is important to establish when and how a patient should undergo HSCT. Besides the indication, the choice of donor and the choice of conditioning regimen play a central role. In the last decade, the use of mismatched donors (including haploidentical donors) has dramatically increased due to the availability of innovative approaches to limit graft-versus-host disease and rejection. In terms of conditioning regimens, the FORUM trial has recently established that total body irradiation (TBI) offers significant advantages in terms of outcome. However, as TBI in children is associated with long-term sequelae, it is likely that strategies to improve the outcome of chemotherapy-based conditionings or to reduce radiation burden will be investigated in the next few years. In the decision-making process of HSCT for pediatric ALL patients it is important to consider how the multiple choices regarding donor type, stem cell source, GvHD prophylaxis, and conditioning regimens used impact on post-HSCT immune reconstitution and risk of relapse and therefore on patients’ outcome. In conclusion, the definition of eligibility criteria, timing and modality of HSCT for pediatric ALL patients is a complex and dynamic process, continuously adjusted according to chemotherapy and HSCT results. The availability of novel therapies, in particular immunotherapy, further increases the complexity of this process. It is therefore crucial to build and update algorithms which account for patient characteristics and protocol findings to allocate each patient with ALL to the best treatment strategy tailored to their risk profile.
Nucera, S., Limido, F., Balduzzi, A. (2024). HSCT in the First CR and in the Subsequent CRs: Indications and Conditioning Regimens. In G. Escherich, V. Conter (a cura di), Acute Lymphoblastic Leukemia in Children and Adolescents (pp. 261-276). Springer Science and Business Media Deutschland GmbH [10.1007/978-3-031-71180-0_17].
HSCT in the First CR and in the Subsequent CRs: Indications and Conditioning Regimens
Nucera S.;Limido F.;Balduzzi A.
2024
Abstract
Hematopoietic stem cell transplantation (HSCT) still constitutes an important therapeutic option for pediatric patients with acute lymphoblastic leukemia (ALL), despite the overall improvements in cure rates and despite the advent of innovative therapies. However, due to the increasing complexity in patients’ stratification and due to the availability of novel treatments, it is important to establish when and how a patient should undergo HSCT. Besides the indication, the choice of donor and the choice of conditioning regimen play a central role. In the last decade, the use of mismatched donors (including haploidentical donors) has dramatically increased due to the availability of innovative approaches to limit graft-versus-host disease and rejection. In terms of conditioning regimens, the FORUM trial has recently established that total body irradiation (TBI) offers significant advantages in terms of outcome. However, as TBI in children is associated with long-term sequelae, it is likely that strategies to improve the outcome of chemotherapy-based conditionings or to reduce radiation burden will be investigated in the next few years. In the decision-making process of HSCT for pediatric ALL patients it is important to consider how the multiple choices regarding donor type, stem cell source, GvHD prophylaxis, and conditioning regimens used impact on post-HSCT immune reconstitution and risk of relapse and therefore on patients’ outcome. In conclusion, the definition of eligibility criteria, timing and modality of HSCT for pediatric ALL patients is a complex and dynamic process, continuously adjusted according to chemotherapy and HSCT results. The availability of novel therapies, in particular immunotherapy, further increases the complexity of this process. It is therefore crucial to build and update algorithms which account for patient characteristics and protocol findings to allocate each patient with ALL to the best treatment strategy tailored to their risk profile.
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