<scp>TCR</scp>
                    ‐αβ/
                    <scp>CD</scp>
                    19 Depleted Graft Versus T Replete Graft With Post‐Transplant Cyclophosphamide in Children Undergoing Haploidentical Donor Hematopoietic Stem Cell Transplan

doi:10.1111/petr.70436

DOI: 10.1111/petr.70436 ISSN: 1397-3142

TCR ‐αβ/ CD 19 Depleted Graft Versus T Replete Graft With Post‐Transplant Cyclophosphamide in Children Undergoing Haploidentical Donor Hematopoietic Stem Cell Transplan

Anuraag Reddy Nalla, Kavitha Ganesan, Vijayshree Muthukumar, Minakshi Balwani, Nithya Seshadri, Krithika Krishnakumar, Ramya Uppuluri, Revathi Raj

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ABSTRACT

Background

The study aimed to analyze the outcomes of TCRαβ / CD19 ‐depleted and T‐replete graft with post‐transplant cyclophosphamide ( PTCY ) in haploidentical hematopoietic stem cell transplantation ( HSCT ) in inborn errors of immunity ( IEI ).

Patients and Methods

We performed a retrospective analysis of children from birth to 18 years of age with IEI who underwent haploidentical HSCT between April 2009 and September 2024, with a minimum follow‐up period of 12 months.

Results

A total of 230 children with IEI underwent HSCT in the 15‐year period, where 105 (45%) underwent haploidentical HSCT and were included in the analysis with a follow up period of 0.5 to 170 months. TCR ‐αβ/ CD19 ‐depleted HSCT was done in 65 (62%) children, while T‐replete graft with PTCY was used in 40 (38%) children. There was no significant difference in engraftment rates, acute and chronic graft versus host disease rates between both the cohorts: T‐depleted group—89%, 8.6%, 12% respectively versus T‐replete group—85%, 15%, 17.5% respectively. The 5‐year overall survival was 62% (95% CI : 48%–73%) in the TCR ‐αβ/ CD 19‐depleted cohort in comparison to 54% (95% CI : 48% to 73%) in the PTCY cohort. When the survival was compared in children less than 2 years of age, it was 60% in the TCR ‐αβ/ CD 19 depleted cohort versus 37% in PTCY cohort (p value of 0.001).

Conclusion

Survival was superior in the T depleted cohort as compared to the T replete group, with viral reactivation remaining a persisting challenge. Letermovir prophylaxis and memory cell add‐back would be potential strategies to improve survival.