Pediatric acute myeloid leukemia: towards high-quality cure of all patients

Kaspers, Gertjan J. L.; Zwaan, C. Michel

doi:10.3324/haematol.11203

Cited by 136 publications

(111 citation statements)

References 142 publications

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“…[1][2][3] Besides better risk stratification, use of repeated course of intensive consolidation therapy and amelioration of supportive therapy, a remarkable contribution to this improvement has been given by the wide use of hematopoietic SCT (HSCT). [4][5][6][7] In particular, for children achieving first CR1, allogeneic (ALLO) HSCT from an HLA-identical sibling has been shown to be the most effective post-remission therapy for preventing leukemia recurrence.…”

Section: Introductionmentioning

confidence: 99%

Outcome of children with high-risk acute myeloid leukemia given autologous or allogeneic hematopoietic cell transplantation in the aieop AML-2002/01 study.

Locatelli

Masetti

Rondelli

et al. 2014

Bone Marrow Transplant

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on behalf of AIEOP BMT Working GroupWe analyzed the outcome of 243 children with high-risk (HR) AML in first CR1 enrolled in the AIEOP-2002/01 protocol, who were given either allogeneic (ALLO; n = 141) or autologous (AUTO; n = 102) hematopoietic SCT (HSCT), depending on the availability of a HLA-compatible sibling. Infants, patients with AML-M7, or complex karyotype or those with FLT3-ITD, were eligible to be transplanted also from alternative donors. All patients received a myeloablative regimen combining BU, Cyclophosphamide and Melphalan; AUTO-HSCT patients received BM cells in most cases, while in children given ALLO-HSCT stem cell source was BM in 96, peripheral blood in 19 and cord blood in 26. With a median follow-up of 57 months (range 12-130), the probability of disease-free survival (DFS) was 73% and 63% in patients given either ALLO-or AUTO-HSCT, respectively (P = NS). Although the cumulative incidence (CI) of relapse was lower in ALLO-than in AUTO-HSCT recipients (17% vs 28%, respectively; P = 0.043), the CI of TRM was 7% in both groups. Patients transplanted with unrelated donor cord blood had a remarkable 92.3% 8-year DFS probability. Altogether, these data confirm that HSCT is a suitable option for preventing leukemia recurrence in HR children with CR1 AML.

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Section: Introductionmentioning

confidence: 99%

Outcome of children with high-risk acute myeloid leukemia given autologous or allogeneic hematopoietic cell transplantation in the aieop AML-2002/01 study.

Locatelli

Masetti

Rondelli

et al. 2014

Bone Marrow Transplant

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“…16,17 In patients with relapsed AML, depending on cytogenetic profile, age and time of first remission, cytarabine-based salvage chemotherapy is sometimes satisfactory. 15,18,19 However, only a few randomized studies address this issue, which thus remains a gray area. 20,21 In second line setting, favorable, intermediate or high intermediate group are suitable to SWOG 9126 protocol (high dose cytarabine, daunorubicin and cyclosporine).…”

Section: Discussionmentioning

confidence: 99%

“…discussion AML with active disease (relapsed/refractory) remains a challenge for clinicians worldwide, especially in patients previously treated with ASCT. 15 In this setting of patients, prognosis is still poor and this group may benefit from new drug developments and alternative approaches for disease control and delay of a novel relapse. 16,17 In patients with relapsed AML, depending on cytogenetic profile, age and time of first remission, cytarabine-based salvage chemotherapy is sometimes satisfactory.…”

Section: Discussionmentioning

confidence: 99%

Outcomes of allogeneic stem cell transplantation among patients with acute myeloid leukemia presenting active disease: Experience of a single European Comprehensive Cancer Center

De-Mello

Pinho-Vaz

Branca

et al. 2016

Rev. Assoc. Med. Bras.

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OutcOmes Of allOgeneic stem cell transplantatiOn amOng patients with acute myelOid leukemia presenting active disease: experience Of a single eurOpean cOmprehensive cancer center rev assoC med bras 2016; 62 (7) Introduction: Allogeneic hematopoietic stem cell transplantation (ASCT) represents a potentially curative approach for patients with relapsed or refractory acute myeloid leukemia (AML). We report the outcome of relapsed/refractory AML patients treated with ASCT. Method: A retrospective cohort from 1994 to 2013 that included 61 patients with diagnosis of relapsed/refractory AML. Outcomes of interest were transplant--related mortality (TRM), incidence of acute and chronic graft-versus-host disease (GVHD), relapse incidence, progression-free survival (PFS) and overall survival (OS). Statistical significance was set at p<0.05. Results:The median age was 61 years (range 1 to 65). The cumulative incidence of 90 days, 1 year, and 3 years TRM were 60%, 26.7%, and 13.3%, respectively (p<0.001). The incidence of relapse was 21.7% at 1 year, 13% at 3 years, and 8.7% at 5 years. Median OS was estimated to be 8 months ) and median PFS, 3 months (95CI 1.835-4.165). Conclusion:In our cohort, TRM in first years after ASCT remains considerable, but ASCT in this setting seems to be a good choice for AML patients with active disease. However, novel approaches are needed to reduce TRM and relapse in this set of patients.

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“…1 Despite intensive chemotherapy, only 60% of patients with AML are cured. 2 The heterogeneity of AML is reflected by differences in morphology, immunophenotype as well as cytogenetic and molecular abnormalities. Recurrent (cyto)genetic aberrations and response to treatment are important prognostic factors in AML and are therefore used for risk group stratification.…”

Section: Introductionmentioning

confidence: 99%

The heterogeneity of pediatric MLL-rearranged acute myeloid leukemia

et al. 2011

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Translocations involving the mixed-lineage leukemia (MLL) gene, localized at 11q23, comprise 15 to 20% of all pediatric acute myeloid leukemia (AML) cases. This review summarizes current knowledge about the etiology, biology, clinical characteristics and differences in outcome in MLL-rearranged pediatric AML. Furthermore, we discuss the role of cooperating events in MLL-rearranged pediatric AML, and future therapeutic strategies to improve outcome. We conclude that MLL-rearranged pediatric AML is a heterogeneous disease, and prognosis depends on various factors, for example, translocation partner, age, WBC and additional cytogenetic aberrations. The relationship of outcome with specific translocation partners requires that they be searched for in the diagnostic work-up of AML. To achieve further improvements in outcome, unraveling the biology of MLL-rearranged pediatric AML is warranted.

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Pediatric acute myeloid leukemia: towards high-quality cure of all patients

Cited by 136 publications

References 142 publications

Outcome of children with high-risk acute myeloid leukemia given autologous or allogeneic hematopoietic cell transplantation in the aieop AML-2002/01 study.

Outcome of children with high-risk acute myeloid leukemia given autologous or allogeneic hematopoietic cell transplantation in the aieop AML-2002/01 study.

Outcomes of allogeneic stem cell transplantation among patients with acute myeloid leukemia presenting active disease: Experience of a single European Comprehensive Cancer Center

The heterogeneity of pediatric MLL-rearranged acute myeloid leukemia

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