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Blood
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Blood
Article . 2021 . Peer-reviewed
Data sources: Crossref
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Outcomes of Transplant-Eligible Patients with Myelodysplastic Syndrome-Refractory Anemia with Excess Blasts Registered in a Prospective Observational Study: The JALSG-CS11-MDS-SCT

Authors: Noriharu Nakagawa; Ken Ishiyama; Kensuke Usuki; Satoru Takada; Tatsuki Tomikawa; Hiroshi Handa; Yuna Katsuoka; +18 Authors

Outcomes of Transplant-Eligible Patients with Myelodysplastic Syndrome-Refractory Anemia with Excess Blasts Registered in a Prospective Observational Study: The JALSG-CS11-MDS-SCT

Abstract

Abstract Introduction: Allogeneic hematopoietic stem cell transplantation (allo-SCT) is the sole curative therapy for myelodysplastic syndromes (MDS). Several studies have addressed the efficacy of bridging therapy before allo-SCT including hypomethylating agents, combination chemotherapy (e.g. induction chemotherapy regimen for acute myeloid leukemia [AML]), and low-dose chemotherapy. However, these studies were retrospective, so selection bias was unavoidable, and the optimal bridging therapy prior to allo-SCT remains unclear. We analyzed the bridging therapy and outcomes of patients with MDS registered in a prospective observational study, the JALSG-AML/MDS/CMML Clinical Observational Study-11 (CS-11). Methods: We studied 393 patients with MDS-refractory anemia with excess blasts (MDS-EB) ≤70 years old at registration for CS-11 between 2011 and 2016. We collected new data using an online survey software program (Survey Monkey ®) and analyzed these data following their integration into the CS-11 data. This study was approved by the ethical committee of Kanazawa University (No. 2018-227) and all participating institutions. Results: A total of 371 patients (94.4%) had additional data available and who were included in this study. The median age of the patients at registration of CS-11 was 64 (range: 16-70) years, and 279 (75.2%) were male. The patients' cytogenetic subgroups were divided into very good (n=3 [0.8%]), good (n=117 [31.5%]), intermediate (n=60 [16.2%]), poor (n=35 [9.4%]), very poor (n=148 [39.9%]), and missing data (n=8 [2.1%]), according to Revised International Prognostic Scoring System (IPSS-R). A total of 188 patients (50.7%) were considered for allo-SCT at the diagnosis of MDS-EB, and 141 patients (75.0%) underwent allo-SCT, whereas 181 (48.9%) patients were not considered for allo-SCT at their diagnosis. The overall survival (OS) of the patients considered for allo-SCT at their diagnosis was significantly higher than that of the patients who were not considered for allo-SCT (3-year OS, 33.7% vs. 13.9%, P<0.001). The Cox regression analysis identified consideration for allo-SCT at the diagnosis, performance status, and cytogenetic subgroup according to IPSS-R as independent factors that affect the OS. In an intention-to-treat analysis, bridging therapy included azacytidine (n=90 [50%]), combination chemotherapy such as induction chemotherapy regimen for AML (n=33, [18%]), and low-dose chemotherapy (n=12 [7%]), while 32 patients (18%) received allo-SCT without bridging therapy. There was no correlation between the various bridging therapies and the transplantation rates or OS in patients considered for allo-SCT. The reasons for discontinuing allo-SCT included cancellation of transplant request (n=12 [30%]), complications or comorbidity developed during bridging therapy (n=10 [25%]), and uncontrolled MDS (n=7 [17%]). Conclusion: Transplant-eligible patients with MDS-EB should be considered for allo-SCT. Further studies will be required to determine the optimal bridging therapy prior to allo-SCT. Disclosures Usuki: MSD K.K.: Research Funding, Speakers Bureau; Eisai Co., Ltd.: Speakers Bureau; Alexion Pharmaceuticals, Inc.: Research Funding, Speakers Bureau; Pfizer Japan Inc.: Research Funding, Speakers Bureau; Kyowa-Kirin Co., Ltd.: Research Funding, Speakers Bureau; Nippon-Shinyaku Co., Ltd.: Research Funding, Speakers Bureau; Amgen-Astellas Biopharma K.K.: Research Funding; Mundipharma K.K.: Research Funding; Nippon-Boehringer-Ingelheim Co., Ltd.: Research Funding; Takeda Pharmaceutical Co., Ltd.: Research Funding, Speakers Bureau; Celgene K.K.: Research Funding, Speakers Bureau; Janssen Pharmaceutical K.K.: Research Funding; Ono Pharmaceutical Co., Ltd.: Research Funding, Speakers Bureau; Novartis Pharma K.K.: Research Funding, Speakers Bureau; Otsuka Pharmaceutical Co., Ltd.: Research Funding, Speakers Bureau; Sumitomo-Dainippon Pharma Co., Ltd.: Research Funding; Daiichi Sankyo Co., Ltd.: Research Funding, Speakers Bureau; SymBio Pharmaceuticals Ltd.: Research Funding, Speakers Bureau; Gilead Sciences, Inc.: Research Funding; AbbVie GK: Research Funding, Speakers Bureau; Astellas Pharma Inc.: Research Funding, Speakers Bureau; PharmaEssentia Japan KK: Research Funding, Speakers Bureau; Yakult Honsha Co., Ltd.: Research Funding, Speakers Bureau; Bristol-Myers-Squibb K.K.: Research Funding, Speakers Bureau; Apellis Pharmaceuticals, Inc.: Research Funding; Incyte Biosciences Japan G.K.: Research Funding; Chugai Pharmaceutical Co., Ltd.: Research Funding, Speakers Bureau; Sanofi K.K.: Speakers Bureau; Amgen K.K.: Research Funding. Handa: Ono: Honoraria; BMS: Honoraria; Janssen: Honoraria; Daiichi Sankyo: Research Funding; Celgene: Honoraria, Research Funding; Chugai: Research Funding; Kyowa Kirin: Research Funding; Takeda: Honoraria, Research Funding; Shionogi: Research Funding; Sanofi: Honoraria, Research Funding; Abbvie: Honoraria; MSD: Research Funding. Fujisawa: Novartis: Honoraria, Research Funding; Pfizer: Honoraria, Research Funding; Otsuka: Honoraria, Research Funding; Bristol Myers Squibb: Honoraria, Research Funding. Suehiro: Amgen BioPharma: Research Funding; Bayer: Research Funding; Bristol-Myers Squibb: Honoraria; Celgene: Research Funding; Chugai Pharmaceutical: Honoraria, Research Funding; Eisai: Honoraria, Research Funding; Incyte: Research Funding; Kyowa Kirin: Honoraria, Research Funding; Nippon Shinyaku: Honoraria; Novartis: Research Funding; Ono Pharmaceutical: Research Funding; Otsuka Pharmaceutical: Research Funding; Pfizer: Research Funding. Maeda: Bayer Yakuhin, Ltd.: Honoraria; Nippon Shinyaku Co., Ltd.: Honoraria; Novartis Pharmaceuticals: Honoraria, Research Funding; Chugai Pharmaceutical Co., Ltd.: Research Funding; Sumitomo Dainippon Pharma Co., Ltd.: Research Funding; Eisai Co., Ltd.: Research Funding; Astellas Pharma Inc.: Research Funding; Alexion Pharmaceuticals, Inc.: Research Funding. Yamamoto: Bristol-Myers Squibb Company: Honoraria; Chugai Pharmaceutical Co., Ltd.: Honoraria; Eisai Co., Ltd.: Honoraria; Kyowa Kirin Co., Ltd.: Honoraria; NIPPON SINYAKU CO., LTD: Honoraria; Novartis Pharma: Honoraria; ONO PHARMACEUTICAL CO.: Honoraria; Otsuka Pharmaceutical: Honoraria; Pfizer Japan Inc.: Honoraria; Takeda: Honoraria. Kiyoi: Astellas: Honoraria; celgene: Honoraria; Daiichi Sankyo: Honoraria; Dainippon Sumitomo: Honoraria; Eisai: Honoraria; Fijifilm: Honoraria; Kyowa Kirin: Honoraria; Otsuka: Honoraria; Perseus Proteomics: Honoraria; Pfizer: Honoraria; Pfizer: Honoraria; Sanofi: Honoraria; Takeda: Honoraria; Zenyaku Kogyo: Honoraria. Matsumura: Ono: Research Funding; Otsuka: Consultancy, Research Funding, Speakers Bureau; Pfizer: Research Funding, Speakers Bureau; Shionogi: Research Funding; Taiho: Research Funding; Takeda: Research Funding; Sumitomo Dainippon: Research Funding; Nihon Pharmaceutical: Research Funding; Daiichi Sankyo: Research Funding, Speakers Bureau; Japan Blood Products Organization: Research Funding; Mundipharma: Research Funding; AYUMI Pharmaceutical: Research Funding; Eli Lilly Japan: Research Funding; Novartis: Research Funding, Speakers Bureau; Nippon Shinyaku: Research Funding; MSD: Research Funding; Mitsubishi Tanabe: Research Funding; Amgen: Speakers Bureau; Bristol-Myers Squibb: Speakers Bureau; Janssen: Speakers Bureau; Kyowa Kirin: Research Funding; Eisai: Research Funding; Chugai: Research Funding; Astellas: Speakers Bureau; Asahi Kasei: Research Funding; Addvie: Research Funding. Miyazaki: Pfizer: Honoraria; Kyowa-Kirin: Honoraria; Abbvie: Honoraria; Bristol-Myers Squibb: Honoraria; Nippon-Shinyaku: Honoraria; Novartis: Honoraria; Astellas: Honoraria; Sumitomo-Dainippon: Honoraria, Research Funding; Janssen: Honoraria; Eisai: Honoraria; Daiichi-Sankyo: Honoraria; Takeda: Honoraria; Chugai: Honoraria; Sanofi: Honoraria.

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selected citations
These citations are derived from selected sources.
This is an alternative to the "Influence" indicator, which also reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Citations provided by BIP!
popularity
This indicator reflects the "current" impact/attention (the "hype") of an article in the research community at large, based on the underlying citation network.
BIP!Popularity provided by BIP!
influence
This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Influence provided by BIP!
impulse
This indicator reflects the initial momentum of an article directly after its publication, based on the underlying citation network.
BIP!Impulse provided by BIP!
0
Average
Average
Average
bronze