Place of HSCT in treatment of childhood AML

Klingebiel, Thomas; Reinhardt, Dirk; Bader, Peter

doi:10.1038/bmt.2008.276

Cited by 17 publications

(14 citation statements)

References 14 publications

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“…27 Similarly, in the BFM-AML98 study, intent-to-treat analysis revealed no difference in 5-year disease-free survival among 275 high-risk patients with or without an HLA-identical sibling donor (47% vs 41%, P ϭ .4). 14 The CCG1941 study found no advantage of HCT for ALL patients with early relapse (3-year disease-free survival 29% with sibling HCT vs 27% with chemotherapy alone), and the CCG1952 study found no advantage in either early relapse (2-year EFS, 43% for HCT and 38% for chemotherapy) or late relapse (56% for HCT and 62% for chemotherapy). 28, 29 An HLA-identical sibling donor has been considered the gold standard as stem cell source.…”

Section: Discussionmentioning

confidence: 99%

“…For example, patients with core-binding-factor AML or high-risk ALL are now typically treated with chemotherapy alone. 8,14 Therefore, a key question now is whether HCT benefits patients whose leukemia is chemo-resistant to current intensive chemotherapy. One concern regarding the use of HCT in these patients is that its toxicity may be excessive because of preexisting organ dysfunction or infections caused by prior intensive chemotherapy.…”

Section: Introductionmentioning

confidence: 99%

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High success rate of hematopoietic cell transplantation regardless of donor source in children with very high-risk leukemia

Leung

Campana

Yang

et al. 2011

Blood

158

136

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We evaluated 190 children with very highrisk leukemia, who underwent allogeneic hematopoietic cell transplantation in 2 sequential treatment eras, to determine whether those treated with contemporary protocols had a high risk of relapse or toxic death, and whether non-HLA-identical transplantations yielded poor outcomes. For the recent cohorts, the 5-year overall survival rates were 65% for the 37 patients with acute lymphoblastic leukemia and 74% for the 46 with acute myeloid leukemia; these rates compared favorably with those of earlier cohorts (28%, n ‫؍‬ 57; and 34%, n ‫؍‬ 50, respectively). Improvement in the recent cohorts was observed regardless of donor type (sibling, 70% vs 24%; unrelated, 61% vs 37%; and haploidentical, 88% vs 19%), attributable to less infection (hazard ratio [HR] ‫؍‬ 0.12; P ‫؍‬ .005), regimen-related toxicity (HR ‫؍‬ 0.25; P ‫؍‬ .002), and leukemiarelated death (HR ‫؍‬ 0.40; P ‫؍‬ .01). Survival probability was dependent on leukemia status (first remission vs more advanced disease; HR ‫؍‬ 0.63; P ‫؍‬ .03) or minimal residual disease (positive vs negative; HR ‫؍‬ 2.10; P ‫؍‬ .01) at the time of transplantation. We concluded that transplantation has improved over time and should be considered for all children with very high-risk leukemia, regardless of matched donor availability. (Blood. 2011;118(2):223-230)

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Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

High success rate of hematopoietic cell transplantation regardless of donor source in children with very high-risk leukemia

Leung

Campana

Yang

et al. 2011

Blood

158

136

View full text Add to dashboard Cite

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“…The significantly lower relapse risk associated with allo-HSCT compared with postremission chemotherapy has not consistently translated into an improvement in survival. [103][104][105] Moreover, it has been calculated that at least 10 children must be transplanted to avoid 1 relapse. 105 The risk-benefit for patients relies on intensity of prior chemotherapy, risk group allocation, CR status, donor type, predicted transplant-related mortality, and long-term toxicity.…”

Section: Postremission Strategiesmentioning

confidence: 99%

Diagnosis and management of acute myeloid leukemia in children and adolescents: recommendations from an international expert panel

Creutzig

Heuvel‐Eibrink

Gibson

et al. 2012

Blood

480

526

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IntroductionChildhood acute myeloid leukemia (AML) is a rare and heterogeneous disease, with an incidence of 7 cases per million children younger than 15 years. In high-income countries, intensive therapy in conjunction with effective supportive care has increased survival rates to ϳ 70%. In 1990 and 2003, expert working groups made recommendations for diagnosis, outcomes, standardization of response criteria, and reporting standards for AML. 1,2 Recent improvements in identifying the molecular genetics and pathogenesis of AML have been implemented in the new World Health Organization (WHO) classification of AML. 3 These changes, and the definition of new diagnostic and prognostic markers and their associated targeted therapies, have prompted the update of earlier recommendations by an international group, on behalf of the European LeukemiaNet for AML in adults in 2010. 4 Despite broad overlap in the diagnostic and treatment recommendations for AML for children and adults, there are important differences in both the diagnostic criteria and disease management, which merit age-specific recommendations. The absence of published recommendations specific for pediatric AML motivated an international group of pediatric hematologists and oncologists (panel and participating groups see "Appendix") to develop evidence-and expert opinionbased consensus recommendations for the diagnosis and management of AML in children, incorporating emerging information on the biology of the disease. The scope of the review is presented in the "Appendix." Recommendations for specific subgroups are also included. This article discusses diagnostic procedures and initial workup, prognostic factors, response criteria, and management, and in particular focuses on differences between adults and children with AML. For personal use only. on May 12, 2018. by guest www.bloodjournal.org From WHO classification and pediatric AMLThe recent WHO 2008 classification is applicable to both adult and pediatric AML 3,5 and has been summarized by Döhner et al. 4 The classification contains most, but not all, cytogenetic subgroups specific to children. Differences in genetic background between children and adults are given in Table 1 and discussed further in "Cytogenetics."Compared with previous classifications (European Group of Immunologic Characterization of Leukemias [EGIL], WHO 2001), 6 the new WHO classification introduced a stringently defined subclass of acute leukemias of ambiguous lineage (mixed phenotype acute leukemias [MPALs]), mainly on the basis of detailed immunophenotypic criteria (Table 2) or presence of t(9;22)(q34; q11.2)/BCR-ABL1 or t(v;11q23)/MLL rearrangement. 3,5,6 The new classification aims to create uniform subgroups defined by unifying molecular targets, which allow selection of specific treatment. Diagnostic procedures and initial workupThe minimal diagnostic requirements in childhood AML are morphology with cytochemistry, immunophenotyping, karyotyping, FISH, and specific molecular genetics in the bone marrow, which is comparable ...

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“…[19][20][21] Indications for HSCT, donor type and stem cell sources varied over the time. However, in 2013, most cooperative groups indicate allogeneic SCT for patients with highrisk factors from either sibling or alternative donor.…”

Section: Discussionmentioning

confidence: 99%

Impact on long-term OS of conditioning regimen in allogeneic BMT for children with AML in first CR: TBI+CY versus BU+CY: a report from the Société Française de Greffe de Moelle et de Thérapie Cellulaire

Berranger

Cousien

Petit

et al. 2013

Bone Marrow Transplant

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Allogeneic hematopoietic SCT (HSCT) appears to be an efficient tool to cure high-risk AML in first CR but the choice between BUbased or TBI-based conditioning regimens still remains controversial. In order to analyze the impact of conditioning regimen on long-term survival, we conducted a retrospective analysis from French registry data including all consecutive patients under 18 years old (n ¼ 226) from 1980 to 2004 transplanted for AML in CR1 from sibling (n ¼ 142) or matched unrelated donors and given either TBI-1200 cGy and CY 120 mg/kg (TBI-Cy, n ¼ 84) or BU 16 mg/kg and CY 200 mg/kg (BuCy200, n ¼ 142). Patient subgroups were comparable for all criteria except for median age at diagnosis and HSCT and for donor type. Both 5-year OS and disease-free survival (DFS) were significantly better in BuCy200 group (P ¼ 0.02 and 0.005, respectively). In multivariate analysis, both HLA matching and BuCy200 appeared as good prognostic factors for treatment-related mortality and DFS. Grade 2-4 acute GvHD and chronic GvHD rates were statistically higher in TBI-Cy group than in Bu-Cy200 one with a RR at 2 (P ¼ 0.002). In total, Bu-Cy200 conditioning regimen gives better outcome compared with TBI-Cy irrespective of the stem cell source and the donor type.

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Place of HSCT in treatment of childhood AML

Cited by 17 publications

References 14 publications

High success rate of hematopoietic cell transplantation regardless of donor source in children with very high-risk leukemia

High success rate of hematopoietic cell transplantation regardless of donor source in children with very high-risk leukemia

Diagnosis and management of acute myeloid leukemia in children and adolescents: recommendations from an international expert panel

Impact on long-term OS of conditioning regimen in allogeneic BMT for children with AML in first CR: TBI+CY versus BU+CY: a report from the Société Française de Greffe de Moelle et de Thérapie Cellulaire

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