[Allogeneic hematopoietic stem cell transplantation for pediatric acute leukemia harboring the PICALM-MLLT10 fusion in two cases]

Area of Science:

• Hematology
• Pediatric Oncology
• Molecular Diagnostics

Background:

• PICALM-MLLT10 gene fusion is a rare but significant genetic abnormality in acute leukemia.
• Pediatric acute leukemia presents unique treatment challenges, especially with central nervous system involvement or high-risk disease.
• Venetoclax has emerged as a targeted therapy option in certain hematologic malignancies.

Purpose of the Study:

• To report the clinical outcomes of two pediatric patients with PICALM-MLLT10-positive acute leukemia.
• To evaluate the efficacy of a treatment regimen combining conventional chemotherapy, venetoclax, and allogeneic hematopoietic stem cell transplantation.
• To assess the potential of this combined approach for improving long-term survival in this specific leukemia subtype.

Main Methods:

• Retrospective analysis of clinical data from two pediatric patients.
• Treatment included conventional chemotherapy, venetoclax, and sequential allogeneic hematopoietic stem cell transplantation.
• Monitoring of bone marrow remission and molecular negativity post-treatment.

Main Results:

• Both patients achieved complete bone marrow remission after venetoclax-combined chemotherapy.
• Both patients achieved molecular negativity and successfully underwent allogeneic hematopoietic stem cell transplantation.
• At the latest follow-up, both patients were alive and in good clinical condition.

Conclusions:

• Venetoclax-based chemotherapy followed by allogeneic hematopoietic stem cell transplantation appears to be a promising strategy for pediatric PICALM-MLLT10-positive acute leukemia.
• This approach may enhance long-term survival outcomes in children with this rare leukemia subtype.
• Further investigation with larger cohorts is warranted to confirm these findings.