Changing outcomes of stem cell transplantation in primary immunodeficiencies: Results from a tertiary-care charitable trust hospital in Mumbai.

Abstract

Background:

Hematopoietic stem cell transplantation in primary immunodeficiency disorders has come a long way since the first transplant in 1968. In India, pediatric stem cell transplantation long-term survival outcomes range from 62.5% to 75%, compared to 90% in high-income countries.

Objective:

We present single-center data of primary immunodeficiency transplants with immune-reconstitution evaluation after transplantation from a charitable trust hospital.

Methods:

Retrospective data of children transplanted for primary immunodeficiency disorders from March 2019 to March 2022 in a newly established transplant unit were collected. Data of pretransplant infections and comorbidities, surveillance for carbapenem-resistant Enterobacteriaceae, transplant characteristics, donor source, graft-versus-host disease, posttransplant infections, immune reconstitution, overall survival at 1 year, and immunodeficiency-free survival were collated.

Results:

Twenty-one patients underwent transplantation for primary immunodeficiency disorders. The median age at transplantation was 3 years and 5 months (range, 7 months to 17 years). Seventy-five percent of the cohort had organ involvement, with lung being the most common organ involved, followed by central nervous system. Fifty-two percent of children had peritransplant infections, with most of them recognized at the pretransplant assessment. Among 20 of 21 children with engraftment, 94% had complete chimerism initially, with 33% developing mixed chimerism over time. The median duration of immunosuppression was 3 months after transplantation, and only 1 child required systemic graft-versus-host disease treatment for more than a year. Immune-reconstitution showed good T-cell recovery at 3 months and naive T-cell production at 6 months. There was no regimen-related or sepsis-related mortality. Overall survival of the cohort was 95% at 1-year follow-up. Immunodeficiency-free survival was 86% after a median follow-up of 20 months.

Conclusions:

Immunodeficiency-free and graft-versus-host disease-free survival can be achieved in the majority of children with primary immunodeficiencies using enhanced supportive care and the latest transplantation algorithms.

• Authors: Ambreen Pandrowala, Mukesh Desai, Manisha Madkaikar, Shilpa Kulkarni, Lakshmi Shobhavat, Jayashree Mishra, Shreepal Jain, Parmarth Chandane, Kunal Sehgal, Saroj Chavan, Parag Karkera, Pradnya Bendre, Ameet Thanky, Sudha Rao, Shakuntala Prabhu, Minnie Bodhanwala, Bharat Agarwal, Prashant Hiwarkar
• Journal: The journal of allergy and clinical immunology. Global (J Allergy Clin Immunol Glob) Vol. 2 Issue 3 Pg. 100105 (Aug 2023) ISSN: 2772-8293 [Electronic] United States
• PMID: 37779531 (Publication Type: Journal Article)
• Copyright: © 2023 The Author(s).