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

In 30 seconds

This systematic review assessed tyrosine kinase inhibitors (TKIs) as continuation therapy after hematopoietic stem cell transplantation (HSCT) in children with acute leukemia. Of 765 articles identified, 16 studies met the review criteria. Included studies described use of agents such as imatinib, dasatinib, ponatinib, sorafenib, and gilteritinib in patients with chromosomal alterations driving kinase signaling. Selected patients achieved measurable residual disease (MRD) negativity or sustained molecular remission, but relapse was also reported, including after treatment cessation or with resistant disease. The review concludes that treatment may be appropriate for selected high-risk patients, while optimal duration and long-term safety remain uncertain.

Key findings

• The review included 16 studies from an initial search of 765 articles.
• Included studies reported MRD negativity and sustained molecular remission in selected patients receiving post-HSCT TKI therapy.
• Relapse was reported after TKI discontinuation and in patients with resistant disease.
• Reported adverse effects included cytopenia, liver dysfunction, and skin conditions; optimal duration and long-term safety remain unclear.

Why it matters

Relapse remains a major cause of transplant failure in pediatric acute leukemia. The review suggests that molecularly targeted continuation therapy may help maintain remission in selected patients, while emphasizing that outcomes depend on disease and mutation context. Its findings also highlight the need to balance potential benefit against toxicity and uncertainty about treatment duration.

What to keep in mind

The review reports that optimal treatment duration and long-term safety in children remain unclear, and prospective studies are needed to establish standardized strategies.