Haploidentical SCT in children: an update and future perspectives

Area of Science:

• Hematology
• Oncology
• Immunology

Background:

• Haploidentical stem cell transplantation (HSCT) is a vital treatment, expanding donor availability for patients with malignant diseases.
• Graft manipulation techniques are crucial for optimizing HSCT outcomes.

Purpose of the Study:

• To review current outcomes and novel strategies in haploidentical HSCT, with a focus on pediatric malignant diseases.
• To evaluate the efficacy of different graft manipulation methods and supportive care strategies.

Main Methods:

• Review of current literature on haploidentical HSCT, focusing on graft manipulation techniques like CD34(+) selection and T/B cell depletion.
• Analysis of outcomes including graft-versus-host disease (GVHD), engraftment, survival, and treatment-related mortality (TRM).

Main Results:

• T and B cell depletion effectively reduces GVHD and achieves high primary engraftment (83-100%) with sufficient stem cell doses.
• Disease-free survival at 3 years for acute lymphoblastic leukemia (ALL) in remission ranges from 22-48%.
• Reduced-intensity conditioning and proactive viral management improved TRM, primarily due to viral infections.

Conclusions:

• T and B cell depletion is a promising strategy for haploidentical HSCT, improving engraftment and reducing GVHD.
• Optimized conditioning regimens and vigilant infection control are key to reducing TRM.
• Further research is needed to improve outcomes for patients with active disease at transplantation, exploring novel cell therapies and treatments.