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Published on: March 17, 2020
Prognostic factors and outcomes for pediatric patients receiving an haploidentical relative allogeneic transplant
M A Diaz1, A Pérez-Martínez2, B Herrero3
1Hematopoietic Stem Cell Transplantation Unit, Pediatric Department, Children's University Hospital Niño Jesús, Madrid, Spain.
Insights
Haploidentical stem cell transplants offer a viable treatment for pediatric blood cancers when a matched donor isn't available. This method shows promising results, particularly for patients in early disease stages, with good survival rates.
Area of Science:
- Hematology
- Oncology
- Immunology
Background:
- Allogeneic hematopoietic stem cell transplantation (HSCT) is crucial for pediatric hematological malignancies.
- Finding HLA-identical donors is challenging, necessitating alternative donor sources.
Purpose of the Study:
- To evaluate the efficacy and safety of T-cell-depleted haploidentical HSCT in pediatric patients.
- To explore the impact of donor KIR B haplotype and CD34+ cell dose on outcomes.
Main Methods:
- Seventy-five T-cell-depleted haploidentical HSCTs were performed in 70 pediatric patients with various hematological malignancies.
- Graft failure, engraftment times, infections, nonrelapse mortality, relapse rates, and disease-free survival were analyzed.
Main Results:
- Engraftment occurred in most patients, with median neutrophil and platelet recovery at 13 and 10 days, respectively.
- Nonrelapse mortality was 10±4% by day +100, and disease-free survival was 52±6% at 22 months median follow-up.
- Early disease phase patients showed encouraging results; KIR B haplotype donors correlated with lower relapse rates.
Conclusions:
- T-cell-depleted haploidentical HSCT is a valid and effective option for pediatric patients lacking HLA-identical donors.
- Optimizing graft composition, including high CD34+ cell dose and KIR B haplotype selection, may improve outcomes and reduce relapse.
Abstract:
Haploidentical hematopoietic stem cell transplantation using T-cell-depleted grafts is a valid option for pediatric patients with hematological malignancies in need of an allogeneic transplantation and lacking an HLA-identical donor. Seventy-five transplantations were performed in 70 patients. Thirty-eight patients had ALL, 32 had AML, 3 had advanced myelodysplastic syndromes and 2 juvenile myelomonocytic leukemia; 19 were in first CR, 30 in second CR, 12 in greater than second CR and 14 were considered to be in refractory disease at time of transplantation. Four patients developed graft failure. Among engrafted patients, the median time to neutrophil and platelet recovery was 13 (range 8-20) and 10 days (range 8-70), respectively. In 64 (85%) cases, ⩾1 infections were diagnosed after transplant. The probability of nonrelapse mortality by day +100 after transplantation was 10±4%. With a median follow-up of 22 months, the probability of relapse was 32±6% and disease-free survival was 52±6%. Haploidentical transplantation using CD3/CD19 depletion is associated with encouraging results especially in patients in early phase of disease. Killer-cell Ig-like receptor B haplotype donors confer a rapid natural killer cells expansion early after transplantation, resulting in lower probability of relapse and suggesting a GvL effect apart from graft-versus-host reactions. Donor infusion of high numbers of CD34+ cells is recommended in order to improve T-cell reconstitution.
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