Hematopoietic Stem Cell Transplantation for Primary Immunodeficiency Disorders: Experience from a Referral Center in...

Area of Science:

• Pediatric Hematology-Oncology
• Immunology
• Stem Cell Transplantation

Background:

• Primary immunodeficiency disorders (PIDs) are a group of genetic conditions impairing the immune system.
• Hematopoietic stem cell transplantation (HSCT) is a potentially curative treatment for severe PIDs.
• Long-term outcomes and challenges of HSCT in pediatric PIDs require continuous evaluation.

Purpose of the Study:

• To report the experience of a high-volume center in pediatric HSCT for PIDs over 15 years.
• To analyze survival, morbidity, and mortality associated with HSCT in children with PIDs.
• To identify factors influencing outcomes in this complex patient population.

Main Methods:

• Retrospective medical record review of pediatric patients (<18 years) with PIDs undergoing HSCT.
• Data collection from 2002 to August 2017 at a specialized referral center.
• Analysis of key outcomes including engraftment, survival, disease-free survival, graft-versus-host disease (GvHD), and complications.

Main Results:

• Eighty-five HSCTs for PIDs were performed, with an 80 (94%) engraftment rate and 67% overall survival.
• Individualized conditioning regimens were used. Mixed chimerism occurred in 20% of patients, with 56% remaining disease-free.
• GvHD affected 39.2% of patients, most commonly in chronic granulomatous disease. Infections were the leading cause of mortality, particularly in severe combined immunodeficiency.
• Haploidentical HSCT (29.4% of cases) showed a 70% survival rate.

Conclusions:

• Pediatric HSCT is a viable curative option for PIDs in India.
• Outcomes are significantly influenced by the expertise of pediatric transplant physicians and intensivists.
• Careful patient selection and management of complications are crucial for successful HSCT in PIDs.