Long‐term follow‐up of haploidentical haematopoietic stem cell transplantation in paediatric patients with high‐risk acute myeloid leukaemia: Report from a single centre

Bai, Lu; Zhang, Zhi‐Xiao; Hu, Guan‐Hua; Cheng, Yi‐Fei; Suo, Pan; Wang, Yu; Yan, Chen‐Hua; Sun, Yu‐Qian; Chen, Yu‐Hong; Chen, Huan; Liu, Kai‐Yan; Xu, Lan‐Ping; Huang, Xiao‐Jun

doi:10.1111/bjh.19086

Cited by 4 publications

(2 citation statements)

References 40 publications

(64 reference statements)

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“…The above rates differed significantly between the two groups of patients, depending on the MRD status before transplantation. Patients who were MRD-negative achieved better OS, EFS, and CIR rates of 80.5%, 73.3%, and 23.8%, respectively, compared to MRD-positive children pre-transplant, for whom the aforementioned rates were 63.4%, 51.4%, and 41.0%, respectively [ 9 ].…”

Section: Malignant Disordersmentioning

confidence: 99%

“…Minimal residual disease (MRD), defined as the lasting presence of leukemia cells after a chemotherapy/radiotherapy regimen, can be used to evaluate the risk of relapse and reaction to a therapy plan [ 8 ]. The assessment of the MRD status before transplantation is a crucial and significant prognostic factor in children and adolescents with hematological malignancies, as patients with negative MRD before haplo-HSCT tend to achieve substantially longer overall survival (OS) and event-free survival (EFS) [ 9 ].…”

Section: Introductionmentioning

confidence: 99%

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Haploidentical HSCT in the Treatment of Pediatric Hematological Disorders

Marszołek,

Leśniak,

Sekunda

et al. 2024

IJMS

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Allogeneic hematopoietic stem cell transplantation has become a treatment option for otherwise non-curative conditions, both malignant and benign, affecting children and adults. Nevertheless, the latest research has been focusing extensively on transplantation from related and unrelated haploidentical donors, suitable for patients requiring emergent hematopoietic stem cell transplantation (HSCT) in the absence of an HLA-matched donor. Haploidentical HSCT (haplo-HSCT) can be an effective treatment for non-malignant pediatric disorders, such as primary immunodeficiencies or hemoglobinopathies, by enabling a much quicker selection of the appropriate donor for virtually all patients, low incidence of graft-versus-host disease (GVHD), and transplant-related mortality (TRM). Moreover, the outcomes of haplo-HSCT among children with hematological malignancies have improved radically. The most demanding tasks for clinicians are minimizing T-cell-mediated alloreactivity as well as early GVHD prevention. As a result, several T-cell depletion approaches, such as ex vivo T-cell depletion (TCD), and T-cell replete approaches, such as a combination of anti-thymocyte globulin (ATG), post-transplantation cyclophosphamide (PTCy), cyclosporine/tacrolimus, mycophenolate mofetil, or methotrexate, have been taken up. As more research is needed to establish the most beneficial form of therapy, haplo-HSCT is currently considered an alternative donor strategy for pediatric and adult patients with complications like viral and bacterial infections, invasive fungal disease, and GVHD.

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Section: Malignant Disordersmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Haploidentical HSCT in the Treatment of Pediatric Hematological Disorders

Marszołek,

Leśniak,

Sekunda

et al. 2024

IJMS

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Risk stratification in the clinical application of minimal residual disease assessment in acute myeloid leukemia

Zhang,

Gu,

Wang

et al. 2024

Cancer

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BackgroundIn acute myeloid leukemia (AML), further investigation is warranted to integrate measurable residual disease (MRD) with genetic characteristics for formulating a dynamic prognostic system for predicting response and selecting appropriate postremission therapeutic strategies.MethodsThe authors incorporated MRD with genetic risk classification and assessed its impact on transplantation decision making within different risk cohorts, comprising 769 patients with newly diagnosed AML across three clinical trials. Only patients who achieved complete remission (CR) within two courses of chemotherapy were selected.ResultsIn the favorable‐risk and intermediate‐risk groups, patients who underwent transplantation according to the protocol experienced significant 3‐year overall survival (OS) benefits compared with those who did not (favorable‐risk group: hazard ratio [HR], 0.38; 95% confidence interval [CI], 0.20–0.73l p = .004; intermediate‐risk group: HR, 0.53; 95% CI, 0.33–0.85; p = .008). In the intermediate‐risk group, early detection of MRD positivity, even after the initial course of chemotherapy, was associated with a significantly elevated cumulative incidence of relapse (47.2% vs. 36.0%; p = .009) and a notable extension of OS with allogeneic hematopoietic stem cell transplantation (HR, 0.47; 95% CI, 0.28–0.79; p = .004). Conversely, patients who achieved MRD negativity at either of the two time points had comparable OS in the favorable‐risk and intermediate‐risk groups, regardless of whether they underwent transplant or not. In the adverse‐risk group, allogeneic hematopoietic stem cell transplantation led to improvements in OS irrespective of MRD status (HR, 0.51; 95% CI, 0.38–0.69; p < .001).ConclusionsEarly clearance of MRD demonstrated significant prognostic value, particularly for patients in the favorable‐risk and intermediate‐risk groups. Positive MRD status after two courses of intensive chemotherapy were associated with a higher relapse rate and inferior OS, necessitating allogeneic hematopoietic stem cell transplantation.

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Desensitization Strategies for Donor-Specific Antibodies in HLA-Mismatched Stem Cell Transplantation Recipients: What We Know and What We Do Not Know

Zhou,

Chen,

Huang

et al. 2024

Oncol Ther

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In human leukocyte antigen (HLA)-mismatched allogeneic stem cell transplantation settings, donor-specific anti-HLA antibodies (DSAs) can independently lead to graft failure, including both primary graft rejection and primary poor graft function. Although several strategies, such as plasma exchange, intravenous immunoglobulin, rituximab, and bortezomib, have been used for DSA desensitization, the effectiveness of desensitization and transplantation outcomes in some patients remain unsatisfactory. In this review, we summarized recent research on the prevalence of anti-HLA antibodies and the underlying mechanism of DSAs in the pathogenesis of graft failure. We mainly focused on desensitization strategies for DSAs, especially novel methods that are being investigated in the preclinical stage and those with promising outcomes after preliminary clinical application.

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Long‐term follow‐up of haploidentical haematopoietic stem cell transplantation in paediatric patients with high‐risk acute myeloid leukaemia: Report from a single centre

Cited by 4 publications

References 40 publications

Haploidentical HSCT in the Treatment of Pediatric Hematological Disorders

Haploidentical HSCT in the Treatment of Pediatric Hematological Disorders

Risk stratification in the clinical application of minimal residual disease assessment in acute myeloid leukemia

Desensitization Strategies for Donor-Specific Antibodies in HLA-Mismatched Stem Cell Transplantation Recipients: What We Know and What We Do Not Know

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