scholarly journals Hematopoietic stem cell transplantation for children with acute myeloid leukemia in second remission: A report from the Australasian Bone Marrow Transplant Recipient Registry and the Australian and New Zealand Children's Haematology Oncology Group

2019 ◽  
Vol 66 (8) ◽  
Author(s):  
Adrian Selim ◽  
Frank Alvaro ◽  
Catherine H. Cole ◽  
Chris J. Fraser ◽  
Francoise Mechinaud ◽  
...  
Blood ◽  
2004 ◽  
Vol 104 (11) ◽  
pp. 5131-5131
Author(s):  
Andree-Laure Herr ◽  
Myriam Labopin ◽  
Rosy Reiffers ◽  
Donald Bunjes ◽  
Didier Blaise ◽  
...  

Abstract Hematopoietic stem cell transplantation (HSCT) is a potentially curative treatment for patients with acute myeloid leukemia (AML). AML of the FAB M0 subtype is rare, often associated with a complex karyotype and a poor prognosis. Results of HSCT for this AML subtype have never been reported separately from other subtypes. We did a survey of the results of 274 HSCT in adults with M0 AML in first complete remission (CR1), performed in EBMT centres since January 1990 until 2002. One hundred fifty patients were transplanted with an HLA identical donor (HLA-id), 30 with an HLA-matched unrelated donor (MUD) and 94 received an autologous transplant (auto). The median age was 45 years (16–71), the median interval from diagnosis to HSCT was 4 months for HLA-id, 6 months for MUD and 5 months for auto HSCT. The median follow-up time (range) was 20 months (1–109), 12 (2–53) and 10 months (1–96) for HLA-id, MUD and auto-HSCT respectively. The source of stem cells was peripheral blood stem cells for 67% of cases, and bone marrow for the remaining. The majority of grafts were non-T-cell depleted. Acute GVHD (grade I–IV) occurred in 56% of HLA-id and in 64% of MUD cases. The table shows the outcomes at two years according to the type of transplant. In conclusion, outcomes after HLA identical HSCT and MUD in adult patients with AML FAB subtype M0 in CR1 are encouraging. In comparison to allogeneic transplant cases, LFS is decreased in patients receiving an autologous transplant due to a high relapse incidence, reflecting the probable role of a graft-versus-leukemia effect in this FAB subtype. Results of HSCT in AML M0 CR1 patients Outcomes HLA-id n=150 MUD n= 30 Auto n=94 LFS: leukemia free survival; OS: overall survival; RI: relapse incidence; TRM: treatment-related mortality 2y LFS 50% 45% 33% 2y OS 58% 50% 49% 2y RI 25% 40% 57% 2y TRM 24% 14% 9%


Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 2032-2032
Author(s):  
Takayoshi Tachibana ◽  
Masatsugu Tanaka ◽  
Estuko Yamazaki ◽  
Ayumi Numata ◽  
Hirotaka Takasaki ◽  
...  

Abstract Abstract 2032 Background: Recent studies have suggested that iron overload is one of important predictors for outcome in allogeneic hematopoietic stem cell transplantation (allo-HSCT). We previously reported that a new scoring system by combination with pre-transplant serum ferritin (SF) and disease risk is useful for predicting outcome in patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) who underwent allo-HSCT at Kanagawa Cancer Center (KCC) (Bone Marrow Transplantation 46: 150–151, 2011). To clarify the availability of this scoring system in another cohort, we performed a validation in multicenter study. Patients and methods: We retrospectively collected clinical data of adult patients with AML and MDS who received allo-HSCT between 2000 and 2010 at three institutes. The patients analyzed in the previous study were excluded. Pre-transplant SF value was categorized as low-SF (<1000 ng/ml) and high-SF (≥1000 ng/ml) according to the previous report. First/second complete remission in AML and refractory anemia in MDS were defined as standard disease risk and others were defined as high risk. Pre-transplant SF and disease risk were each assigned a score, and the sum total was tested as prognostic score based on three risk groups: low (score=0), intermediate (score=1) and high (score=2). The log-rank test was used for comparisons of Kaplan-Meier curves. Cox regression model was used for multivariate analyses. Results: A total of 153 patients were analyzed. Their median age was 48 years old (range: 17–65), with 57 males and 96 females. Related or unrelated bone marrow (n=106), related peripheral blood stem cell (n=14) or unrelated cord blood (n=33) were transplanted as stem cell sources. Myeloablative conditioning regimens were used in 112 patients while reduced-intensity regimens in 41. The median level of pre-transplant SF was 1,139 ng/ml (range:22–6,262) in all patients. Standard and high disease risks were 82 and 71 patients, respectively. There were no significant differences of patient characteristics among the three institutes except pre-transplant SF level. The median follow-up period among survivors was 25.9 months (range: 7.1–124). Univariate analysis for 5-year overall survival (OS) demonstrated an inferior outcome in patient with high SF (41% vs. 48%, p=0.025) or high disease risk (21% vs. 61%, p<0.001), compared with low SF or standard disease risk, respectively. Multivariate analysis for 5-year OS demonstrated high SF (HR: 1.794, 95% CI: 1.11–2.91, p=0.018) and high disease risk (HR: 2.08, 95% CI: 1.26–3.41, p=0.004) at transplantation as inferior prognostic predictors. According to the scoring system with pre-transplant SF and disease risk, the 5-year OS of the low (n=36), intermediate (n=82) and high (n=35) risk group was 68%, 42% and 20%, respectively (p<0.001). Conclusions: The usefulness of the simple scoring system with pre-transplant SF and disease risk was validated by the multicenter analysis. This prognostic scoring system might help to stratify patients in clinical studies and choose patients for iron chelating therapy. Disclosures: No relevant conflicts of interest to declare.


Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 2547-2547
Author(s):  
Shinsuke Takagi ◽  
Kazuhiro Masuoka ◽  
Naoyuki Uchida ◽  
Mineo Kurokawa ◽  
Hirohisa Nakamae ◽  
...  

Abstract BACKGROUND/METHODS: Long-term survivors with Philadelphia chromosome-negative myeloproliferative neoplasms (Ph-neg MPNs) can eventually develop acute myeloid leukemia (AML, i.e. blast transformation). Allogeneic hematopoietic stem cell transplantation (allo-HSCT) can be the only curative treatment for such patients. To clarify its outcome, we retrospectively studied the cases with AML transformed from Ph-neg MPNs using the national registry data of JSHCT. The cases transplanted after 2000 were collected. RESULTS: Thirty-nine cases were extracted (male, n=21; female, n=18). Median age was 57 years (range, 22-71). Underlying Ph-neg MPNs included essential thrombocythemia (ET, n=21), primary myelofibrosis (PMF, n=11) and polycythemia vera (PV, n=7). FAB classification was M0 (n=4), M1 (n=4), M2 (n=10), M3 (n=0), M4 (n=1), M5 (n=3), M6 (n=0), M7 (n=1) and unknown (n=16). Median value of WBC at diagnosis of AML was 8300/uL (250-338000). Karyotype at diagnosis of AML was normal (n=6), complex (n=12), others (n=17) and unknown (n=4). Thirty-two cases (82%) were not in remission at the time of allo-HSCT (1st relapse, n=7; primary induction failure, n=18; untreated, n=12). Median duration between diagnosis of AML and allo-HSCT was 134 days (range, 24-369). The donors were related bone marrow or related mobilized peripheral blood stem cell (rBM/rPBSC, n=8), unrelated bone marrow (uBM, n=15) and unrelated umbilical cord blood (uCB, n=16). Myeloablative conditioning regimens (MAC) were used in 15 cases, and the remaining 24 cases were conditioned by reduced-intensity regimens (RIC), according to CIBMTR definition (Giralt S, et al, 2009). Cumulative incidence of neutrophil engraftment was 74.4% at day 60 (patients engrafted, n=29; death before day 60, n=6; relapse before day 60, n=4). At 2 years after transplant, overall survival (OS) was 29.2%. The median duration of follow up of survivors was 1989.5 days (range, 285-3270). In univariate analysis, age (>57 vs. <57, p=0.87), underlying MPNs (PMF vs. ET vs. PV, p=0.16), disease status at allo-HSCT (CR vs. non-CR, p=0.09), conditioning regimen (MAC vs. RIC, p=0.95) and donor selection (rBM/PBSC vs. uBM vs. uCB, p=0.10) had no impact on OS. The only variable that influenced on OS was chromosome at diagnosis of AML (normal vs. others vs. complex, p=0.02). The cumulative incidence of relapse and non-relapse mortality at 3 years was 34.4% and 38.1%, respectively. CONCLUSION: Although the prognosis of AML transformed from Ph-neg MPNs is dismal in general, the study suggested a promising result that there were curable patients with allo-HSCT. Figure 1 Figure 1. Figure 2 Figure 2. Disclosures No relevant conflicts of interest to declare.


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