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Peer-reviewedCC BY 4.0Cancer Chemotherapy and Pharmacology

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

  • Mahati Avineni1,2
  • Yara K. Habo2
  • Nora K. Habo2
  • Ahmed Adham R. Elsayed2,3,4
  • Marc D. Basson2,3,4
4 affiliations
  1. 1College of Graduate Studies, Northeast Ohio Medical University, Rootstown, OH 44272 USA
  2. 2College of Medicine, Northeast Ohio Medical University, 4209 St, OH-44, Rootstown, OH 44272 USA
  3. 3Department of Surgery, Northeast Ohio Medical University, 4209 St, OH-44, Rootstown, OH 44272 USA
  4. 4Department of Biomedical Sciences, Northeast Ohio Medical University, 4209 St, OH-44, Rootstown, OH 44272 USA
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Abstract

Purpose: 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.

Methods: 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.

Results: 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.

Conclusion: TKIs can serve as a targeted post-HSCT continuation therapy in select pediatric acute leukemia patients, particularly those with high-risk, mutation-driven disease. Molecular profiling and MRD monitoring are central to guiding agent selection, timing, and mutation-directed therapy adjustments. These findings support individualized TKI treatment to reduce relapse risk following HSCT, while highlighting the need for prospective studies to define standardized treatment strategies.

Graphic abstract:

Supplementary Information: The online version contains supplementary material available at https://doi.org/10.1007/s00280-026-04976-9.

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Cite

BibTeXRIS
BibTeX
@article{avineni2026role,
  title = {{The role of tyrosine kinase inhibitors as a post-hematopoietic stem cell transplant continuation therapy for pediatric acute leukemia: a systematic review}},
  author = {Mahati Avineni and Yara K. Habo and Nora K. Habo and Ahmed Adham R. Elsayed and Marc D. Basson},
  journal = {Cancer Chemotherapy and Pharmacology},
  year = {2026},
  month = oct,
  doi = {10.1007/s00280-026-04976-9},
  url = {https://doi.org/10.1007/s00280-026-04976-9},
  note = {CC BY 4.0},
}
RIS
TY  - JOUR
TI  - The role of tyrosine kinase inhibitors as a post-hematopoietic stem cell transplant continuation therapy for pediatric acute leukemia: a systematic review
AU  - Mahati Avineni
AU  - Yara K. Habo
AU  - Nora K. Habo
AU  - Ahmed Adham R. Elsayed
AU  - Marc D. Basson
PY  - 2026
DA  - 2026/10/27
JO  - Cancer Chemotherapy and Pharmacology
DO  - 10.1007/s00280-026-04976-9
UR  - https://doi.org/10.1007/s00280-026-04976-9
AB  - Purpose: 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.  Methods: 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.  Results: 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.  Conclusion: TKIs can serve as a targeted post-HSCT continuation therapy in select pediatric acute leukemia patients, particularly those with high-risk, mutation-driven disease. Molecular profiling and MRD monitoring are central to guiding agent selection, timing, and mutation-directed therapy adjustments. These findings support individualized TKI treatment to reduce relapse risk following HSCT, while highlighting the need for prospective studies to define standardized treatment strategies.  Graphic abstract:  Supplementary Information: The online version contains supplementary material available at https://doi.org/10.1007/s00280-026-04976-9.
SN  - 0344-5704
SN  - 1432-0843
ER  -