The role of tyrosine kinase inhibitors as a post-hematopoietic stem cell transplant continuation therapy for pediatric acute leukemia: a systematic review

Relapse following hematopoietic stem cell transplantation (HSCT) is a leading cause of transplant failure for pediatric acute leukemia patients, emphasizing the need for an effective post-transplant continuation therapy. This systematic review evaluates the role of tyrosine kinase inhibitors (TKIs) as a post-HSCT continuation therapy in pediatric acute leukemia.

A literature search of PubMed, Cochrane, Web of Science, and VHL was conducted according to PRISMA guidelines. Original studies were included if they were relevant to the research question and fit the eligibility criteria.

The initial search identified 765 articles, with 16 studies included after filtration. TKIs were indicated for patients with chromosomal alterations causing constitutively active kinase signaling. Common agents included imatinib, dasatinib, ponatinib, sorafenib, and gilteritinib, and selection was based on disease status and molecular profiling. Duration of continuation ranged from 10 months to 3 years, with dosage dependent on the agent and tolerability. TKIs were generally well-tolerated, with reported side effects including cytopenia, liver dysfunction, and skin conditions. Across the included studies, MRD negativity and sustained molecular remission were reported in selected patients receiving post-HSCT TKI continuation therapy, while relapse was also reported after treatment discontinuation or in resistant disease. However, optimal duration and long-term safety remain unclear in the pediatric population.