[Infant acute lymphoblastic leukemia: treatment strategies and new insights]

Area of Science:

• Pediatric Oncology
• Hematology
• Cancer Genetics

Background:

• Infant acute lymphoblastic leukemia (ALL), especially KMT2A-rALL, is a rare and aggressive malignancy.
• Current treatment regimens yield poor long-term event-free survival rates (≤50%) and significant acute/late toxicities.
• There is an urgent need for improved risk stratification and less toxic therapeutic approaches.

Purpose of the Study:

• To review pivotal clinical trials and current treatment strategies for infant ALL.
• To highlight the necessity for international collaboration in establishing evidence-based treatment protocols for rare pediatric leukemias.
• To discuss emerging therapeutic options, including blinatumomab, for KMT2A-rALL.

Main Methods:

• Literature review of key clinical trials and recent advancements in infant ALL treatment.
• Analysis of treatment strategies focusing on risk stratification and toxicity reduction.
• Examination of ongoing international collaborative efforts, including European study groups.

Main Results:

• Infant KMT2A-rALL presents a particularly dismal prognosis, underscoring treatment challenges.
• Intensive chemotherapy protocols are associated with substantial acute and long-term adverse effects.
• International collaboration is crucial for developing effective strategies for rare diseases like infant ALL.

Conclusions:

• Novel treatment strategies with reduced toxicity are imperative for improving outcomes in infant ALL.
• Enhanced patient stratification is essential for tailoring therapy and minimizing treatment-related harm.
• Ongoing international studies, incorporating agents like blinatumomab, represent a promising direction for future infant ALL management.