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Haploidentical- versus identical-sibling transplant for high-risk pediatric AML: A multi-center study
Feng-Mei Zheng1, Xi Zhang2, Chun-Fu Li3
1National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Peking University People's Hospital, Peking University Institute of Hematology, Beijing, 100044, P. R. China.
Insights
Haploidentical donor stem cell transplantation (HID-SCT) offers outcomes comparable to human leukocyte antigen-identical sibling donor SCT for high-risk pediatric acute myeloid leukemia. While GVHD rates may be higher, this approach provides a viable alternative for patients needing curative treatment.
Area of Science:
- Hematology
- Pediatric Oncology
- Immunology
Background:
- Human leukocyte antigen-identical sibling donor (ISD)-hematopoietic stem cell transplantation (SCT) is a potentially curative treatment for high-risk pediatric acute myeloid leukemia (AML).
- Haploidentical donor (HID) stem cell transplantation is an alternative when an ISD is unavailable.
- The role of HID-SCT in high-risk pediatric AML requires further clarification.
Purpose of the Study:
- To compare the overall survival (OS) and relapse rates between HID-SCT and ISD-SCT in pediatric patients with high-risk AML.
- To evaluate graft-versus-host disease (GVHD) incidence and non-relapse mortality (NRM) in both groups.
- To analyze subgroup data based on cytogenetic risk and disease status at transplantation.
Main Methods:
- Retrospective analysis of 179 high-risk pediatric AML patients under 18 years old undergoing either ISD-SCT (n=23) or HID-SCT (n=156).
- HID-SCT utilized granulocyte colony-stimulating factor and anti-thymocyte globulin-based regimens.
- Comparison of outcomes including overall survival, relapse rates, GVHD, and NRM.
Main Results:
- Three-year overall survival rates were comparable: 73.0% for ISD-SCT vs. 74.6% for HID-SCT (P=0.689).
- Relapse rates were significantly lower in the HID-SCT group (16.4%) compared to ISD-SCT (39.1%, P=0.027).
- Subgroup analysis showed significantly lower relapse rates (9.2% vs. 50.0%, P=0.001) and higher disease-free survival (DFS) (81.2% vs. 50.0%, P=0.021) with HID-SCT.
Conclusions:
- Unmanipulated HID-SCT achieves comparable DFS and OS outcomes to ISD-SCT for high-risk pediatric AML.
- HID-SCT may be associated with a higher incidence of GVHD, but it is manageable.
- HID-SCT represents a viable and effective alternative for pediatric patients with high-risk AML lacking an HLA-identical sibling donor.
Background:
Human leukocyte antigen-identical sibling donor (ISD)-hematopoietic stem cell transplantation (SCT) is a potentially curative treatment for high-risk pediatric acute myeloid leukemia (AML). A haploidentical donor (HID) is readily available to almost all children. Previous studies have demonstrated that patients with HID-SCT had similar outcomes compared to ISD-SCT for pediatric and adult AML. However, the role of HID-SCT in high-risk pediatric AML is unclear.
Methods:
To compare the overall survival of high-risk AML children who underwent either HID-SCT or ISD-SCT, we analyzed 179 cases of high-risk AML patients under 18 years of age treated with either ISD-SCT (n = 23) or HID-SCT (n = 156). Granulocyte colony-stimulating factor plus anti-thymocyte globulin-based regimens were used for HID-SCT. We also analyzed the subgroup data of AML patients at first complete remission (CR1) before SCT with known cytogenetic risk.
Results:
The numbers of adverse cytogenetic risk recipients were 8 (34.8%) and 13 (18.8%) in the ISD-SCT group and the HID-SCT group, and the number of patients with disease status beyond CR1 were 6 (26.1%) and 14 (20.3%) in the two groups. The cumulative rates of grades II-IV acute graft-versus-host disease (GVHD) were 13.0% in the ISD-SCT group and 34.8% in the HID-SCT group (P = 0.062), with a three-year cumulative rates of chronic GVHD at 14.1% and 34.9%, respectively (P = 0.091). The relapse rate in the ISD-SCT group was significantly higher than that in the HID-SCT group (39.1% vs. 16.4%, P = 0.027); with non-relapse mortality at 0.0% and 10.6% (P = 0.113), respectively. The three-year overall survival rates were 73.0% for the ISD-SCT group and 74.6% for the HID-SCT group (P = 0.689). In subgroup analysis, the three-year relapse rate in the ISD-SCT group was higher than that in the HID-SCT group (50.0% vs. 9.2%, P = 0.001) and the three-year DFS in the ISD-SCT group (50.0%) was lower than that in the HID-SCT group (81.2%) (P = 0.021).
Conclusions:
Unmanipulated HID-SCT achieved DFS and OS outcomes comparable to those of ISD-SCT for high-risk pediatric AML patients with potentially higher rate but manageable GVHD.
