Hematopoietic stem cell transplantation for children with primary immunodeficiency diseases: single center experience...

Area of Science:

• Immunology
• Pediatrics
• Hematology

Background:

• Primary immunodeficiencies (PID) are a group of genetic disorders affecting the immune system.
• Hematopoietic stem cell transplantation (HSCT) is a potentially curative treatment for many PIDs.
• Limited data exists on HSCT outcomes for PID patients in developing countries.

Purpose of the Study:

• To evaluate the outcomes of HSCT for children with PID in a developing country setting.
• To identify challenges and specific complications associated with HSCT for PID in this region.

Main Methods:

• Retrospective review of pediatric PID patients who underwent HSCT at King Hussein Cancer Center (KHCC), Jordan.
• Data collected included patient demographics, diagnoses, HSCT type, conditioning regimens, complications, and survival.
• Follow-up duration was a median of 32 months.

Main Results:

• Twenty-eight children with various PIDs (including SCID, CHS, HLH, WAS) received HSCT.
• Overall survival was 72% (21 out of 28 patients alive).
• Complications included BCG reactivation (32%) and graft failure (21%), with graft failure higher in patients without conditioning.

Conclusions:

• HSCT can be performed with good outcomes for PID patients in developing countries.
• Delayed diagnosis/referral and BCG reactivation are significant challenges unique to this setting.
• Optimizing diagnosis and managing BCG reactivation are crucial for improving HSCT success in resource-limited areas.