علاج متلازمة IPEX بالخط الأول

References

• Currently, the primary therapeutic approach for IPEX syndrome is allogeneic hematopoietic stem cell transplantation (HSCT).
• Allogeneic HSCT is the therapy of choice for IPEX syndrome.
• Cells from either human leukocyte antigen (HLA)-identical siblings or other HLA-matched donors are used for HSCT.
• Patients receive HSCT from cord blood, peripheral blood, or bone marrow.
• Only partial donor chimerism was sufficient for complete IPEX syndrome remission.
• HSCT results in disease resolution with a better quality of life, regardless of age, donor source, or conditioning regimen.

DOI: 10.3390/genes12030323