Targeted Therapies and Molecular Advances in Pediatric Acute Lymphoblastic Leukemia: Epidemiology, Genetics, and Future Directions
Session
Pharmaceutical and Natural Sciences
Description
Pediatric acute lymphoblastic leukemia (pALL) remains the most common childhood malignancy, comprising approximately 30% of all pediatric cancers and demonstrating remarkable heterogeneity in disease biology and treatment response. Advances in risk stratification, driven by cytogenetic and genomic profiling—including identification of aneuploidy, chromosomal translocations, and key mutations—have enabled substantial improvements in cure rates, now reaching 80-90% in developed settings. This review synthesizes current epidemiological trends, known genetic drivers such as PAX5, IKZF1, and TP53, and the implementation of precision medicine approaches. Special considerations are given to high-risk subgroups (Down syndrome–associated ALL and relapsed/refractory cases), the impact of pharmacogenetics on therapy personalization, and recent developments in targeted agents, immunotherapies, and CAR T-cell platforms. Ongoing hurdles in global access and therapy-related toxicity in vulnerable cohorts are discussed, highlighting future strategies to further optimize supportive care and therapeutic efficacy.
Keywords:
ALL, Gene mutations, pALL, Down syndrome
Proceedings Editor
Edmond Hajrizi
ISBN
978-9951-982-41-2
Location
UBT Lipjan, Kosovo
Start Date
25-10-2025 9:00 AM
End Date
26-10-2025 6:00 PM
DOI
10.33107/ubt-ic.2025.349
Recommended Citation
Aliu, Erda; Temaj, Anjeza; Gashi, Yllka; Selmani, Driton; Bilalli, Sefedin; Temaj, Gazmend; and Nuhi, Nexhibe, "Targeted Therapies and Molecular Advances in Pediatric Acute Lymphoblastic Leukemia: Epidemiology, Genetics, and Future Directions" (2025). UBT International Conference. 11.
https://knowledgecenter.ubt-uni.net/conference/2025UBTIC/PNS/11
Targeted Therapies and Molecular Advances in Pediatric Acute Lymphoblastic Leukemia: Epidemiology, Genetics, and Future Directions
UBT Lipjan, Kosovo
Pediatric acute lymphoblastic leukemia (pALL) remains the most common childhood malignancy, comprising approximately 30% of all pediatric cancers and demonstrating remarkable heterogeneity in disease biology and treatment response. Advances in risk stratification, driven by cytogenetic and genomic profiling—including identification of aneuploidy, chromosomal translocations, and key mutations—have enabled substantial improvements in cure rates, now reaching 80-90% in developed settings. This review synthesizes current epidemiological trends, known genetic drivers such as PAX5, IKZF1, and TP53, and the implementation of precision medicine approaches. Special considerations are given to high-risk subgroups (Down syndrome–associated ALL and relapsed/refractory cases), the impact of pharmacogenetics on therapy personalization, and recent developments in targeted agents, immunotherapies, and CAR T-cell platforms. Ongoing hurdles in global access and therapy-related toxicity in vulnerable cohorts are discussed, highlighting future strategies to further optimize supportive care and therapeutic efficacy.