Improving Outcomes with Haploidentical Stem Cell Transplantation [HaploSCT] in Children Using Post-transplant
Biju George1, Uday Kulkarni1, Sharon Lionel1
1Department of Haematology, Christian Medical College, Vellore, India.
Insights
Haplo-identical stem cell transplants are a viable option for children lacking a matched sibling donor, offering improved survival rates over time. Further research is needed to reduce graft failure and infections, which negatively impact outcomes.
Area of Science:
- Pediatric Hematology
- Transplant Immunology
- Oncohematology
Background:
- Haplo-identical stem cell transplantation (SCT) with post-transplant cyclophosphamide is an emerging alternative for pediatric patients without a matched sibling donor.
- This approach is increasingly utilized for both malignant and non-malignant pediatric disorders.
Purpose of the Study:
- To evaluate the outcomes of haplo-identical SCT using post-transplant cyclophosphamide in children.
- To identify factors influencing overall survival in this patient population.
Main Methods:
- Retrospective analysis of 138 SCT procedures in 127 children between 2010 and June 2021.
- Utilized both myeloablative and reduced intensity conditioning regimens with peripheral blood stem cells.
- Assessed engraftment, graft-versus-host disease (GVHD), infections, and overall survival (OS).
Main Results:
- Engraftment was achieved in 81.9% of patients, with a 10.2% primary graft failure rate.
- Cumulative incidence of acute and chronic GVHD was 49.5% and 40.7%, respectively.
- Two-year OS was 54.9%, with significantly lower survival in younger children (0-5 years). Survival rates improved over time (2010-2021).
- Bacterial infection, invasive fungal disease, and graft failure were identified as negative prognostic factors for OS.
Conclusions:
- Haplo-identical SCT with post-transplant cyclophosphamide is a feasible option for pediatric patients lacking a matched sibling donor.
- Strategies to mitigate graft failure, infection-related mortality, and GVHD are crucial for improving long-term outcomes.
Abstract:
Haplo-identical stem cell transplant using post-transplant cyclophosphamide is increasingly being used in children without a matched sibling donor. Between 2010 and June 2021, 127 children underwent 138 transplants with a median age of 7.1 years for malignant and non-malignant disorders. Conditioning regimens included both myeloablative and reduced intensity regimens with peripheral blood stem cells as the main graft source. Engraftment occurred in 113 [81.9%] at a median of 16 days [range: 10-32] with primary graft failure in 10.2%. Cumulative incidence of grade II-IV acute graft versus host disease (GVHD) was 49.5% and chronic GVHD in 40.7%. Majority [92.7%] had at least one infection with 31% incidence of bacterial infection, 76% incidence of viral and 16% incidence of fungal infection. The 2-year overall survival (OS) is 54.9 ± 4.6% with a lower survival among young children aged 0-5 years [28.2 ± 6.4%] compared to 5-10 years [71.3 ± 6.8%] and 11-15 years [55.7 ± 8.8%] [p = 0.032]. 2-year OS has gradually improved from 25.0 ± 2.1% for 2010-2013 to 47.5 ± 6.2% for 2014-2017 and 67.1 ± 6.6% for 2018-2021 [p = 0.049]. On multivariate analysis, bacterial infection [p = 0.017], invasive fungal disease [p = 0.002] and graft failure [p = 0.029] negatively impacted overall survival. Haplo-identical SCT with post-transplant cyclophosphamide is a reasonable option for children who do not have a matched sibling donor. Strategies to reduce graft failure, infection related mortality and GVHD needs to be explored.
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