Hematopoietic stem cell transplantation for childhood malignancies of myeloid origin

Shenoy, Shalini; Smith, Franklin O.

doi:10.1038/sj.bmt.1705961

Cited by 17 publications

(17 citation statements)

References 46 publications

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“…[17][18][19] In the present study, 8 of the 13 patients subsequently received allogeneic HSCT, but only 2 of those 8 patients (25%) survived. It is well known that the transplantation-related mortality For personal use only.…”

Section: Discussionmentioning

confidence: 89%

Clinical characteristics and outcome of refractory/relapsed myeloid leukemia in children with Down syndrome

Taga

Saito

Kudo

et al. 2012

Blood

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Myeloid leukemia in Down syndrome (ML-DS) is associated with good response to chemotherapy and favorable prognosis. Because little research has been focused on refractory/relapsed (R/R) cases, we conducted a retrospective analysis for R/R ML-DS. Among ML-DS patients diagnosed between 2000 and 2010 in Japan, 26 relapsed (25 in the BM and 1 in the skin), and 3 refractory patients were enrolled. The male/female ratio was 18/11. The median age at initial diagnosis of ML-DS was 2 years, and the median time to relapse was 8.6 months. Each patient initially had been treated with ML-DS-specific protocols. Thirteen of the 26 patients achieved complete remission with various kinds of reinduction chemotherapies; 2 of 8 survived without further recurrence after receiving allogeneic hematopoietic stem cell transplantation, and 4 of 5 maintained complete remissions with chemotherapy alone. Treatment failures mostly were associated with disease progression rather than treatment-related toxicities. The 3-year OS rate was 25.9% ؎ 8.5%. A longer duration from initial diagnosis to relapse was a significant favorable prognostic factor (P < .0001). We conclude that clinical outcome for patients with R/R ML-DS generally are unfavorable, even in those receiving hematopoietic stem cell transplantation. Novel methods to identify poor prognostic factors for ML-DS are necessary. (Blood. 2012; 120(9):1810-1815)

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Section: Discussionmentioning

confidence: 89%

Clinical characteristics and outcome of refractory/relapsed myeloid leukemia in children with Down syndrome

Taga

Saito

Kudo

et al. 2012

Blood

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“…Currently, the remission rate of pediatric non-promyelocytic AML is about 70-90%, but the survival rate has been reported to be only 45-66% despite intensive post-remission therapy (22). After the great success of treatment in pediatric acute lymphoblastic leukemia (ALL) with risk based therapy, this kind of efforts are also introduced to AML (5, 6).…”

Section: Discussionmentioning

confidence: 99%

High Transcript Level of FLT3 Associated with High Risk of Relapse in Pediatric Acute Myeloid Leukemia

et al. 2010

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Identification of prognostic factors and risk-based post-remission therapy was proposed to improve the outcomes of acute myeloid leukemia (AML) and a mutation of FLT3 has been reported to be a risk factor, especially for pediatric patients. Recently, FLT3 expression level was implicated to have prognostic significance in adults, but little is known for childhood AML. To define the prognostic significance, transcript level of FLT3 was analyzed in 52 pediatric AML patients. The median copy number of FLT3 was 4.6×103 (40-5.9×107 copies)/1.0×106 GAPDH copy, and the relapse free survival of patients with high transcript level of FLT3 (>106 copy number) (0%) was significantly lower than that of the others (53.2%). High transcript level of FLT3 was associated with a markedly high risk of relapse. The development of new therapeutic scheme such as a frontline allogeneic stem cell transplantation or administration of FLT3 inhibitor is needed to improve outcomes.

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“…Immunosuppressive effect was required to ensure engraftment. 4,27,28 Historically, combination of CY (Cy 120 mg/kg) and TBI was the most used conditioning regimen. 25,28,29 Radiation-free regimen had been proven effective for eradication of leukemia and sufficiently immunosuppressive for engraftment in HLA-matched sibling transplantation.…”

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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Hematopoietic stem cell transplantation for childhood malignancies of myeloid origin

Cited by 17 publications

References 46 publications

Clinical characteristics and outcome of refractory/relapsed myeloid leukemia in children with Down syndrome

Clinical characteristics and outcome of refractory/relapsed myeloid leukemia in children with Down syndrome

High Transcript Level of FLT3 Associated with High Risk of Relapse in Pediatric Acute Myeloid Leukemia

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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