For children with acute leukemia (AL), allogeneic hematopoietic stem cell transplantation (HSCT) plays a role in the treatment of some patients, even as frontline chemotherapy options have advanced over the years. Mattia Algeri, MD, of Bambino Gesù Childrens’ Hospital in Rome, Italy, and colleagues recently detailed the current place of allogeneic HSCT in treatment of pediatric AL in a review article published in the Journal of Clinical Medicine.

Some reasons for the continued utility of allogeneic HSCT in treatment of children with AL include improvements in human leukocyte antigen-typing, conditioning regimens, supportive care, and graft-versus-host disease prophylaxis. There are also potential opportunities for improved outcomes with HSCT through the use of precision medicine strategies that may enable improved remission status prior to HSCT, Dr Algeri and colleagues noted in their report.

The emphasis on strategies involving minimal residual disease (MRD)-based therapy additionally holds promise in optimizing outcomes for these patients. MRD status may also have use in risk stratification for patients under consideration for allogeneic HSCT. Posttransplant MRD status additionally has been shown to be a predictor of disease recurrence in patients with acute lymphoblastic leukemia.

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“For several years, donor characteristics have played a relevant role in determining eligibility for HSCT, with alternative donors being accepted only in those cases deemed at the highest risk of leukemia relapse,” Dr Algeri and colleagues wrote. Recently, results of studies have indicated that the use of alternative donors for HSCT have reduced the need to limit HSCT to only those circumstances in which a fully matched donor is available.

Advances in therapy options involving combination chemotherapy and immunotherapy may challenge the continued utility of HSCT in treatment of pediatric AL. However, the authors explained that logistical difficulties could impede comparisons of HSCT with other options through clinical trials.

In the absence of such trials, Dr Algeri and colleagues stated that “determining the appropriate role of HSCT in childhood AL will continue to be a challenging and dynamic process,” while working toward determining which children may benefit more from HSCT than from other therapies.

“In this regard, close cooperation between chemotherapy cooperative study groups and transplant/cell therapy societies, and monitoring of treatment-related late effects with long-term patient follow-up is crucial,” the authors concluded in their report.

Disclosures: Some authors have declared affiliations with or received grant support from the pharmaceutical industry. Please refer to the original study for a full list of disclosures.


Algeri M, Merli P, Locatelli F, Pagliara D. The role of allogeneic hematopoietic stem cell transplantation in pediatric leukemia. J Clin Med. 2021;10(17):3790. doi:10.3390/jcm10173790