ALBERT

Description: Background; The number of non-Hodgkin's lymphoma (NHL) patients in the elderly has been increasing due to the long-lived society. It is not rare to treat elderly NHL patients aged over 85 years old. Individualizing the doses of cancer chemotherapy agents and progress in supportive therapy has improved the prognosis for elderly patients with NHL. Prolonged hospitalization elderly patients have adverse effects, which include dementia, difficulty in walking, and depression. Optimal treatment for these patients is unknown. Patients and Methods; In our hospital between April 2012 and December 2014, we treated 19 elderly patients (85 and over 85 years) with NHL as outpatients with VDS 3mg (day 1) and Dexa. (day 1-4) which repeated every 3 weeks for as long as possible. Results; Complete remission was achieved in 5 patients and partial remission in 6; the median duration of survival was 14 months. Adverse effects included leukopenia in 1 patient (

Description: [Study purpose] We have reported that overall results of cord blood transplantation (CBT) were inferior to those of bone marrow transplantation (BMT) from unrelated donor in myelodysplastic syndrome (MDS) including transformed acute myelogenous leukemia (AML/MDS) (ASH 2007). Poor engraftment and higher incidence of relapse in CBT patients were serious problems. We now show here the impact of conditioning regimen and GVHD prophylaxis method on clinical outcomes of CBT, and then show the results when we looked for the appropriate graft selection in terms of human leukocyte antigen (HLA) compatibility and cell dose. [Patients and Methods] Clinical data of 333 patients with MDS including AML/MDS who received unrelated CBT without prior transplant history between 1998 and 2006 in Japan were collected by the Japan Cord Blood Bank Network (JCBBN). The median period of follow-up for survivors (n=148) after transplants was 13 (range, 1–99) months. We analyzed the hematopoietic recovery, incidences of acute and chronic graft-versus-host disease (GVHD), risks of transplant-related mortality (TRM) and relapse, and disease-free survival (DFS) using competing risk regression models. [Results] Both myeloablative conditioning regimen including 8Gy or more dose of total body irradiation (TBI) and GVHD prophylaxis as calcineurin inhibitor (cyclosporin or taclorimus) plus methotrexate (MTX) significantly (p

Description: Background: Cytogenetic abnormalities at diagnosis are recognized as a potent prognostic factor for acute leukemia patients. Among acute myeloid leukemia patients, the prognostic implications of cytogenetic abnormalities have been established for those treated with chemotherapy as well as those undergoing allo-SCT. In the context of Ph-negative ALLpatients, cytogenetic abnormalities at diagnosis clearly stratify the prognosis, whereas it has not been elucidated whether similar prognostic stratification is applicable to allo-SCT recipients. Objective: The aim of this retrospective study was to assess the prognostic impact of cytogenetic abnormalities in adult Ph-negative ALL patients who underwent allo-SCT. Patients and Methods: The study cohort included 373 adult Ph-negative ALL patients aged over 15 years who underwent allo-SCT for the first time between January 2001 and December 2012 at the 23 institutions participating in the Kanto Study Group for Cell Therapy (KSGCT). Patients' clinical data were collected from the KSGCT database. The Institutional Review Board of Gunma University approved the protocol of this study. Karyotypes considered high risk (HR) included t(4;11), t(8;14), low hypodiploidy, and complex (equal or more than five abnormalities), and all other karyotypes were designated standard risk (SR). On this basis, 308 patients (82.6%) were categorized as SR and 65 patients (17.4%) were categorized as HR at diagnosis. Of the 373 patients, 267 underwent allo-SCT in complete remission (CR) (224 in the SR group and 43 in HR group), and 106 in non-CR (84 in the SR group and 22 in HR group). For analysis, the study population was stratified based on disease status at the time of transplant. Almost all patients were conditioned with total body irradiation (TBI)-containing myeloablative conditioning (MAC) regimens prior to transplantation. Overall survival (OS) was defined as the interval from the date of transplantation to the date of death. Non-relapse mortality (NRM) was defined as any death in continuous complete remission (CR). The Fisher's exact test was used for comparison of binary variables. OS and RFS were estimated by the Kaplan-Meier method, and compared using the log-rank test. Cumulative incidences (CI) of relapse and NRM were compared using the stratified Gray test. P 〈 0.05 was considered as statistically significant. Results: [Patients in CR] No significant difference in patient characteristics and transplant procedures was observed between the SR and HR groups. The 5-year OS rates were similar between the SR and HR groups (60.5% vs. 74.1%, respectively; p = 0.225) (Figure 1). Similarly, there were no significant differences in the 5-year CI of relapse and NRM rates between the two groups (relapse: 26.3% vs. 24.8%, respectively; p = 0.498, NRM: 19.6% vs. 10.0%, respectively; p = 0.232). Multivariate analysis for OS identified MAC and TBI-containing regimens, not cytogenetic risk, as significant positive prognostic factors. [Patients in non-CR] No significant difference was observed between the SR and HR groups in terms of patient characteristics or transplant procedures, although there was a female predominance in the HR group. Patients in the SR group had a significantly superior 5-year OS rate compared to the HR group (15.4% vs. 4.5%, respectively; p = 0.022). There was no significant difference in the 5-year CI of relapse between the SR and HR groups (60.3% vs. 50.0%, respectively; p = 0.411), whereas the 5-year CI of NRM in the SR group was significantly lower than that in the HR group (24.8% vs. 45.5%, respectively; p = 0.024). Multivariate analysis revealed cytogenetic risk group as an independent prognostic factor. Conclusion: These findings suggest that adult Ph-negative ALL patients in remission with HR cytogenetic abnormalities have similar transplant outcomes to those in the SR group. Considering the reported equality of the CR rates between the two groups, allo-SCT at an early clinical phase is recommended for HR group patients, reminiscent of Ph-positive ALL patients in the pre-imatinib era. Current transplant procedures do not improve outcomes for patients who are not in remission, especially those with HR cytogenetic abnormalities. Disclosures Usuki: MSD: Other: personal fees, Research Funding; SymBio Pharmaceutical: Other: personal fees, Research Funding; GlaxoSmithKline: Other: personal fees, Research Funding; Shionogi: Other: personal fees; Fujimoto Pharmaceutical: Research Funding; Bristol-Myers Squibb: Other; Takeda Pharmaceutical: Research Funding; Nippon Shinyaku: Other: personal fees, Research Funding; Sanofi: Other: personal fees, Research Funding; Shire: Research Funding; Kyowa Hakko Kirin: Other: personal fees, Research Funding; Otsuka Pharmaceutical: Research Funding; Eisai: Research Funding; Novartis: Other: personal fees, Research Funding; Boehringer Ingelheim: Other: personal fees, Research Funding; Celgene: Other: personal fees, Research Funding; Sumitomo Dainippon Pharma: Other: personal fees, Research Funding; Chugai Pharmaceutical: Other: personal fees; Fuji Film RI Pharma: Other: personal fees; Taiho Pharmaceutical: Other: personal fees, Research Funding; Astellas: Research Funding. Nakaseko:Novartis: Honoraria, Research Funding, Speakers Bureau; BMS: Honoraria, Research Funding, Speakers Bureau; Pfizer: Honoraria, Research Funding, Speakers Bureau; Otsuka: Honoraria, Research Funding.

Description: Introduction: LR11 is a type I membrane protein that plays a key role in the migration of undifferentiated vascular smooth muscle cells, and circulating soluble LR11 (sLR11) has been known as a biomarker for coronary stenosis. We have previously found that LR11 is highly expressed in acute leukemia, diffuse large B cell lymphoma (DLBCL), and follicular lymphoma (FL) cells. Soluble LR11 were detected in the patients' serum, and our retrospective cohort demonstrated that serum sLR11 level is significantly increased at diagnosis and normalized at remission (Sakai et al. Clin Chim Acta. 2012, Ohwada et al. 2011 ASH annual meeting). Furthermore, high serum sLR11 level had a significant association with relapse and inferior progression-free survival (PFS) in patients with FL (Kawaguchi et al. Br J Haematol. 2013). Based on these findings, we have conducted a multicenter prospective observational study to validate the clinical impact of serum sLR11 in patients with newly diagnosed DLBCL. Patients and Methods: Ninety-seven consecutive patients with newly diagnosed DLBCL between 2010 and 2013 in Chiba University Hospital and affiliated hospitals were enrolled. Serum samples were collected at diagnosis and when the patients reached complete remission. Clinical and laboratorial data were collected prospectively. Serum sLR11 levels were measured with enzyme-linked immunosorbent assay. Normal control samples were obtained from 75 healthy adult volunteers who had given informed consent. Results: The patients had a median age of 69 years (range, 18-94). Ninety percent of patients were treated with R-CHOP-based regimen, and 80% of them achieved complete remission (CR). Serum sLR11 levels of DLBCL patients were significantly elevated than those of normal controls (21.2 ±27.6 ng/ml vs. 8.8 ±1.8 ng/ml, p

Description: [Background] Allogeneic hematopoietic stem cell transplantation (HSCT) is a potent treatment to cure in patients with aplastic anemia (AA). However, an optimal pre-transplant conditioning remains unclear. The combination of high-dose cyclophosphamide (CY) and anti-thymocyte globulin (ATG) has been used as an effective conditioning, but cardiotoxity due to high-dose CY has been a major concern especially in patients with iron overload by excessive transfusion. In addition, the appropriate dose and timing of ATG use is another unsolved topic in HSCT for AA. Therefore, we performed a prospective study to assess the safety and efficacy of a conditioning regimen using fludarabine (Flu), reduced-dose CY, and low-dose thymoglobulin in HSCT for AA. [Methods] Patients with severe AA, aged between 16 and 65 years, who have an HLA-matched or 1-locus mismatched, related or unrelated donor were prospectively included. A conditioning regimen consisted of Flu 30mg/m2 for 4 days, CY 25mg/kg for 4 days, and thymoglobulin 1.25mg/kg for 2 days (days -4, and -3). In patients who underwent transplantation from unrelated and/or HLA-mismatched donor, 2 Gy of total body irradiation was added. Cyclosporine for an HLA-matched related donor or tacrolimus for the other donors, together with short-term methotrexate were used as graft-versus-host disease (GVHD) prophylaxis. Granulocyte-colony stimulating factor was used from 1 day after transplantation. Primary outcome measure was an overall survival at 1 year after HSCT. This study was approved by the Institutional Review Board of all the participating institutions. [Results] Twenty-eight patients were enrolled between 2011 and 2017, and their median age was 36 years (range: 18 - 61y). Sixteen patients were male. A median time from diagnosis to transplantation was 1858 days in patients who had previously received immunosuppressive therapy (IST) using ATG (n = 16) and 121 days in those who had not received IST (n = 12). Sixteen patients received graft form related donors including 1 HLA-mismatched donor, and 12 patients did from unrelated donors including 3 mismatched donors. Stem cell source was bone marrow in 27 out of 28 patients. All patients but one, who died early due to infection, achieved neutrophil engraftment at a median of 19 days after HSCT. Mixed chimerism (MC) was observed in 6 patients at day 30, and 3 out of those 6 patients achieved complete donor chimerism by day 90. On the other hand, MC was newly observed in additional 2 patients at day 90. Only one patient experienced secondary engraftment failure, and which developed with complete donor-type chimerism at day 99. No patients developed grade 2-4 acute GVHD. However, the cumulative incidence of chronic GVHD was 39.9 % at 1 year, and one third of them had extensive type. With a median follow-up period of 1727 days for survivors, overall survival rates (OS) were 96.4 % at 1 year and 82.8 % at 5 years (Figure 1). Cytomegalovirus (CMV) antigenemia was detected in 17 patients, but no patients developed CMV disease. A high Epstein-Barr virus (EBV)-DNA load (more than 1×104 copies/mL) was detected in 2 patients at day 60 and 1 patient at day 90. Neither developed EBV-lymphoproliferative disorder (LPD) within a year. However, another patient, in whom high EBV-DNA load was not detected within 90days after HSCT, developed EBV-LPD and died at three and a half years after HSCT. [Conclusion] HSCT for AA using Flu, reduced-dose CY, and low-dose thymoglobulin as a conditioning regimen was safe and effective. Relatively high incidences of MC and chronic GVHD need further improvement. (This study is registered with www.umin.ac.jp as UMIN000006071.) Disclosures Kako: Takeda Pharmaceutical Company Limited.: Honoraria; Takeda Pharmaceutical Company Limited.: Honoraria; Celgene K.K.: Honoraria; Bristol-Myers Squibb: Honoraria; Sumitomo Dainippon Pharma Co., Ltd.: Honoraria; Chugai Pharmaceutical Co., Ltd.: Honoraria; Otsuka Pharmaceutical Co., Ltd.: Honoraria; Ono Pharmaceutical Co., Ltd.: Honoraria; Janssen Pharmaceutical K.K.: Honoraria. Kanda:Tanabe-Mitsubishi: Research Funding; Shionogi: Consultancy, Honoraria, Research Funding; Taisho-Toyama: Research Funding; Pfizer: Research Funding; Taiho: Research Funding; MSD: Research Funding; Sanofi: Research Funding; Novartis: Research Funding; Eisai: Consultancy, Honoraria, Research Funding; Bristol-Myers Squibb: Consultancy, Honoraria; Otsuka: Research Funding; Asahi-Kasei: Research Funding; Takeda: Consultancy, Honoraria, Research Funding; CSL Behring: Research Funding; Ono: Consultancy, Honoraria, Research Funding; Nippon-Shinyaku: Research Funding; Astellas: Consultancy, Honoraria, Research Funding; Kyowa-Hakko Kirin: Consultancy, Honoraria, Research Funding; Dainippon-Sumitomo: Consultancy, Honoraria, Research Funding; Chugai: Consultancy, Honoraria, Research Funding; Takara-bio: Consultancy, Honoraria; Celgene: Consultancy, Honoraria; Mochida: Consultancy, Honoraria; Alexion: Consultancy, Honoraria. Usuki:Ono Pharmaceutical: Speakers Bureau; GlaxoSmithKline K.K.: Research Funding; Janssen Pharmaceutical K.K: Research Funding; Sanofi K.K.: Research Funding; Shire Japan: Research Funding; SymBio Pharmaceuticals Limited.: Research Funding; Celgene Corporation: Research Funding, Speakers Bureau; Daiichi Sankyo: Research Funding; Takeda Pharmaceutical: Speakers Bureau; Boehringer-Ingelheim Japan: Research Funding; Pfizer Japan: Research Funding, Speakers Bureau; Sumitomo Dainippon Pharma: Research Funding, Speakers Bureau; Novartis: Speakers Bureau; Chugai Pharmaceutical: Speakers Bureau; Kyowa Hakko Kirin Co., Ltd.: Research Funding; Otsuka Pharmaceutical Co., Ltd.: Research Funding; Astellas Pharma Inc.: Research Funding; Nippon Shinyaku: Speakers Bureau; Mochida Pharmaceutical: Speakers Bureau; MSD K.K.: Speakers Bureau. Mori:Astella Pharma: Honoraria; Shire Japan: Honoraria; Janssen: Honoraria; SHIONOGI: Honoraria; Taisho Toyama Pharmaceutical Co: Honoraria; Novartis Pharma: Honoraria; Celgene: Honoraria; Japan Blood Products Organization: Honoraria; Pfizer: Honoraria; CHUGAI: Honoraria; MSD: Honoraria; Eisai: Honoraria; Novartis Pharma: Research Funding; Asahi Kasei: Research Funding; MSD: Research Funding; Kyowa Hakko Kirin: Honoraria; Ono: Honoraria.

Description: Abstract 3071 Objective: Double-unit cord blood transplantation (dCBT) has been widely applied for patients with less dose single-unit cord blood (sCB) because of a higher engraftment failure. Although several reports have suggested that dCBT may have impact on increasing GVHD incidence and reducing disease relapse, pre-transplant conditioning and GVHD prophylaxis varied too much to read the results. We therefore conducted a prospective multi-center phase II study assessing safety and efficacy of dCBT by using relatively uniform myeloablative conditioning and GVHD prophylaxis in Japan. Methods: From Apr. 2006 to Jan. 2010, patients younger than 55 years with hematological malignancies who lack any adequate unrelated and related donors and without prior history of transplantation were prospectively enrolled. Thirty-nine centers participated following approval by each institutional review board (Trial identifier: UMIN :C000000359, C-SHOT0507). Patients were eligible when there is no single unit with total nucleated cell (TNC) dose of 〉 2.5 × 107 /kg available, and at least 2 units available that have combined TNC 〉 2.5 × 107 /kg, with one of each 〉 1.5 × 107 /kg and the other 〈 2 × 107 /kg. CB units mismatched at 0–2 antigen in HLA-A, -B, -DR between unit and patient were selected. Preparative regimens were 12 Gy of TBI, Ara-C (12 g/m2) combined with G-CSF, and 120 mg/kg of CY for myeloid diseases, while TBI + CY for lymphoid diseases (S.Takahashi, et al. Blood 109;1322, 2007). GVHD prophylaxis was short term MTX and CsA. Results: Seventy patients were enrolled and 61 patients (AML 29, ALL 17, CML 6, MDS 6, ML 3) received dCBT. Nine patients did not receive dCBT due to disease progression (n=7) and other SCT (n=2). Disease status at dCBT was 27 in standard (CR1, CP1) and 34 in advanced (beyond CR1 or CP1). The median age was 37 years (range; 10–54) and median weight was 70.5kg (50.1–129.8). Median total and CD34+ cell doses (both units combined) at cryopreservation were 3.52 × 107 /kg (2.25 – 4.43) and 1.04 × 105 /kg (0.39 – 2.67), respectively. Median TNCs of larger and smaller unit were 1.90 × 107 /kg (1.47 – 2.48) and 1.60 × 107 /kg (0.74 – 1.97), respectively. Cumulative incidence of neutrophil recovery (NR, 〉0.5 × 109/L) was 67%(53–77) at day28, and 85%(73–92) at day 50. The median time to NR was day 26 (18–50) and median time to transfusion-independent platelet recovery (〉20 × 109/L) was day 53 (32–98). Four died early before day 30 (2 on day8, 1 each on day11 and day 29) and 6 received 2nd transplantation due to engraftment failure (on day30, 33, 37, 42, 50, 54). Among engrafted 51 patients, all 50 patients assessed for chimeric status showed single donor-derived engraftment; 27 were from larger unit and 23 from smaller dose of TNC. Number of TNC of engrafted smaller unit was ranged from 0.74 – 1.96 × 107 /kg, including 6 that were even less than 1.5 × 107 /kg. There is no correlation between unit dominance and the number of TNC, CD34+ cell dose, CFU-GM dose, degrees of HLA/sex/ABO mismatches between units and recipients, and graft viability. Acute GVHD progressed in 33 (65%) of 51 evaluable patients (29% grade II-IV), and chronic GVHD was observed in 18 (36%) of the 50 evaluable patients (18% extensive). Actual EFS, RFS and OS at 1y and 3y was 48%(37–58) and 46%(35–56), 49%(36–61) and 47%(34–59), 57%(44–69) and 54%(40–65), respectively. Median follow-up period of survivors (n=32) are 1226.5days (365–1721). EFS at 1y of standard and advanced status patients was 67%(46–81) and 32%(18–48), respectively (p=0.023). Dose of TNC, CD34 and HLA disparity did not significantly affect on survival. Cumulative incidence of day100-TRM was 26%, and relapse late at 3y was 16.3%. Cause of death were disease progression in 11 and TRM (infection and organ failures) in 18. Discussion: Myeloablative dCBT can be a safe and effective alternative option for those who only have insufficient sCB available (less than 2.5×107/kg of TNC). So far, parameters assessed, such as the engraftment kinetics, survival rate, cumulative incidence of GVHD and relapse in dCBT seem to be comparable to our historical data of sCBT. Determinants of engrafting units after dCBT are still unclear. Disclosures: Off Lavel Use: G-CSF at pre-conditioning for myeloid malignancies is an off-label use in Japan. This study was supported by a Research Grant for Tissue Engineering (H17-014) and a Research Grant for Allergic Disease and Immunology (H20-015) from the Japanese Ministry of Health, Labor and Welfare. Disclosures: Off Label Use: G-CSF at pre-conditioning for myeloid malignancies is an off-label use in Japan. Kato:Research Grant for Tissue Engineering (H17-014) and a Research Grant for Allergic Disease and Immunology (H20-015) from the Japanese Ministry of Health, Labor and Welfare.: Research Funding.

Description: Background: GF after allogeneic SCT is a life-threatening event. To clarify the clinical feature of patients with GF and the feasibility of second SCT for GF, we retrospectively investigated the patients who developed GF after SCT. Patients and Methods: we surveyed adult patients who received SCT in KSGCT between January 1994 and December 2005. Primary and Secondary GF were defined as a failure of absolute neutrophil count (ANC) ≥ 0.5x109/l for at least 3 consecutive days and a decrease of ANC 〈 0.5x109/l for at least 7 days consecutive days after initial engraftment, respectively. Results: Of the 1,963 patients, 60 patients (3.1%) were identified with GF. Their median age was 47 years (range, 16 to 63). There were 52 patients with hematological malignancies and 8 with aplastic anemia. The incidence of GF according to donor source was 9/597 patients (1.5%) in related donor-bone marrow (RBM), 1/359 (0.3%) in related donor-peripheral blood stem cells (RPBSC), 18/839 (2.1%) in unrelated donor-bone marrow (UBM), and 32/168 (19%) in unrelated cord blood (UCB). GF in UCB was significantly higher than that in other donors (P

Description: Abstract 1532 The current standard initial treatment for APL is all-trans retinoic acid (ATRA) and anthracycline with or without citarabine, followed by ATRA maintenance. Although the treatment has greatly improved clinical outcomes of APL, a considerable proportion of patients still undergo disease relapse, requiring salvage therapy with such as arsenic trioxide (ATO). GO, an anti-CD33 monoclonal antibody conjugated with calicheamicin, is also highly effective on APL. Highly homogeneous expression of CD33, potential efficacy of calicheamicin and lower expression of P-glycoprotein on APL cells explain the high potency of GO on APL. We report here on the efficacy of GO monotherapy against relapsed/refractory APL in a post-marketing surveillance study of GO in Japan. All patients who were treated with GO alone were obligatorily registered into a post-marketing surveillance study run by Wyeth Japan, Co. Ltd. under the guidance of the Pharmaceutical and Medical Devices Agency. We obtained permission to access patients' data from each institution and Pfizer Japan, Co. Ltd. The survey items included adverse events, treatment outcome, overall survival (OS) and relapse-free survival (RFS). Clinical and biological characteristics were analyzed in APL and compared by chi-square test or Fisher's exact test for categorical data, and Wilcoxon rank-sum test for continuous data. OS and RFS were estimated by the Kaplan-Meier method in APL, and then compared to those from the simultaneous post-marketing study in acute myeloid leukemia (AML) by the log-rank test. Between 2005 and 2008, 27 relapsed/refractory APL patients were enrolled in this surveillance. Of these, two patients were excluded from analysis because of insufficient data, while 726 AML patients were enrolled and 503 were evaluable. Twelve (48%) and 2 (9%) APL patients who were treated with GO alone achieved CR and CRp, respectively, while 42 (8%) and 41 (8%) of AML achieved CR and CRp, respectively. Remission duration of first CR and number of relapses influenced CR rates, but previous usage of ATO and age did not in APL. OS and RFS at 2 years were 63% and 71% in APL, and 14% and 20% in AML, respectively. These were shown in figures. OS and RFS in APL were better than those in AML (P

Description: [Background] Bendamustine has demonstrated high response rates in non-Hodgkin lymphomas (NHL). However, standard administration schedule frequently shows delayed hematological recovery resulting in the discontinuation of treatment. Here we designed a novel treatment schedule that could be more tolerable and conducted randomized phase II study (UMIN000008702). [Methods] Patients (pts) with relapsed/refractory(R/R) indolent B-cell NHL and mantle cell lymphoma were randomly assigned to standard arm (120mg/m2 on day1 and 2, every 3 weeks) or Benda-14 arm (120 mg/m2 on day1 and 15, every 4 weeks) of bendamustine monotherapy. Each arm was repeated to 6 cycles and the accomplishment rate (AR) of all scheduled treatment was analyzed as primary end point. [Results] A total of 46 pts were enrolled into the study. Baseline characteristics were: median age 64 years (range 46-78); 48% male; 76% follicular lymphoma; 33% ECOG PS ≥1; 50% having one previous regimen. 65% stage III/IV. 24% bone marrow positive. 33% with bulky mass (〉6cm). Using random allocation, twenty two and 24 pts were assigned to standard and Benda-14 arm and the AR of 6 cycles in each arm was 41 and 38%, respectively. The median number of cycles was 4.5 in both arms. Eleven (50%) in standard and 10 (42%) in Benda-14 arm withdrew from protocol due to mainly prolonged hematological toxicities. Three withdrew due to disease progression. Two withdrew due to adverse events (AE). Grade 4 non-hematological AE was observed in one. Overall response rate (ORR) in the standard arm was 77% (95% confidence interval [CI], 59 to 95), including a 50% complete response (CR) compared with 83% (95% CI, 68-99), including a 46% CR in Benda-14 arm.　After a median follow-up time of 16 months, the median event-free survival (EFS) in the standard arm and Benda-14 arm was 14.6 months (95% CI, 8-26) and 15 months (95% CI, 11 - not reached), respectively. There was no significant difference between two arms in AR and in EFS (p=0.431). The overall survival (OS) in both arms was the same of 89% at 15 months. [Conclusions] Although this study did not confirm the superiority of Benda-14 to complete 6 cycles of treatment, Benda-14 arm appears to be equally tolerable and active as the present standard therapy. Benda-14 could be a study arm of next trial that determines better practical strategy for this disease population. Disclosures Igarashi: Zenyaku-Kogyo Inc.: Research Funding. Tsukasaki:Daiichi Sankyo Co., Ltd.: Consultancy; Takeda: Research Funding.

Description: INTRODUCITON: We reported the outcomes of double-unit cord blood transplantation (dCBT) after myeloablative conditioning performed in a prospective multicenter phase II study (C-SHOT0507) (Biol Blood Marrow Transplant 19, 2013: 812-819). However, the benefit of using double unit compared to single unit was not clearly shown. We performed matched control analysis to compare dCBT and single-unit CBT (sCBT), using C-SHOT0507 data of dCBT and registry data of sCBT. METHODS: Between Apr.2006 and Jan.2010, 61 cases of dCBT in C-SHOT0507 phase II clinical study and 932 cases of sCBT were performed in Japan. Because all cases of dCBT perfomed by TBI12Gy containing myeloablative conditioning (CA/Cy/TBI +/- G-CSF for myeloid and Cy/TBI for lymphoid) and uniform GVHD prophylaxis (CSA/MTX), we excluded reduced intensity conditioning and myeloablative conditioning without TBI 12 Gy from the cases of sCBT. Finally 307 sCBT patients who had hematological malignancies (AML170, ALL80, MDS24, CML14 and ML20) were extracted. All the sCBT recipients received uniform preconditioning described above and same GVHD prophylaxis (CSA/MTX), selected as a matched control cohort of dCBT C-SHOT0507 study. All statistical analyses were performed using EZR (Kanda Y, Saitama Medical Center, Jichi Medical University), a graphical user interface for R (The R Foundation for Statistical Computing, version 2.13.0). RESULTS: Median observation period of survivors was 1431 (106-2315) days post-transplant. Backgrounds of dCBT group and matched-control sCBT group were comparable except gender: (dCBT:M/F = 53 /8 versus sCBT:M/F = 154/153). Median age were 37 (10-54) in dCBT and 40 (14-54) in sCBT. Disease risk included standard 27, advanced 34 cases in dCBT, and standard 142, advanced 165 cases in sCBT. sCBT were grouped according to infused TNC number; 148 cases of low TNC (2x107/kg TNC, advantage of adding another CB unit may be quite limited in Japan both scientifically and financially, until meaningful benefit is demonstrated by randomized phase III study such as BMT-CTN 0501. Figure 1 Figure 1. Figure 2 Figure 2. Disclosures No relevant conflicts of interest to declare.