Article
Hematology
Yanming Zhang, Limin Liu, Yejun Si, Miao Miao, Huiying Qiu, Xiaowen Tang, Yue Han, Chengcheng Fu, Zhengming Jin, Suning Chen, Aining Sun, Depei Wu
Summary: In comparing ATG-F and p-ALG as part of the conditioning regimen in HSCT for SAA patients, the study found no significant differences in transplant complications and patient survival rates between the two treatments.
Article
Hematology
Amy E. DeZern, Marianna Zahurak, Heather J. Symons, Kenneth R. Cooke, Carol Ann Huff, Tania Jain, Lode J. Swinnen, Philip H. Imus, Nina D. Wagner-Johnston, Richard F. Ambinder, Mark Levis, Leo Luznik, Javier Bolanos-Meade, Ephraim J. Fuchs, Richard J. Jones, Robert A. Brodsky
Summary: Severe aplastic anemia (SAA) is a marrow failure disorder with high morbidity and mortality. This study found that reduced-intensity conditioning HLA-haploidentical bone marrow transplantation (BMT) with posttransplantation cyclophosphamide (PTCy)-based graft-versus-host (GVHD) prophylaxis resulted in 100% overall survival and minimal GVHD in patients with SAA. This approach not only avoids the adverse effects of immunosuppressive therapy, but also expands access to BMT across all populations.
Article
Hematology
Nobuhiro Hiramoto, Hirohito Yamazaki, Yukinori Nakamura, Naoyuki Uchida, Makoto Murata, Tadakazu Kondo, Satoshi Yoshioka, Tetsuya Eto, Akinori Nishikawa, Takafumi Kimura, Tatsuo Ichinohe, Yoshiko Atsuta, Yasushi Onishi, Ritsuro Suzuki, Takehiko Mori
Summary: A retrospective analysis of 115 adults with idiopathic AA undergoing UCBT using TBI-containing RIC regimens without ATG showed that this approach is suitable for these patients. There were no significant differences in transplantation outcomes between the FM and FC groups.
ANNALS OF HEMATOLOGY
(2022)
Article
Immunology
Yawen Zhang, Yao He, Suli Wang, Jingnan Sun, Jinsong Jia, Yuemin Gong, Guangsheng He, Jianyong Li
Summary: This study retrospectively analyzed the clinical and laboratory characteristics of 124 patients with transfusion-dependent non-severe aplastic anemia (TD-NSAA) and found common complications including iron overload, liver and kidney dysfunction, diabetes mellitus/impaired glucose tolerance, and severe infection. 58% of patients progressed to severe aplastic anemia, and low absolute neutrophil count, severe infection, or iron overload were identified as risk factors for progression. Combination therapy with antithymocyte globulin (ATG) and Cyclosporin A (CsA) showed a higher overall response rate compared to CsA alone, and ATG was a favorable factor for efficacy. Some patients developed secondary clonal hematologic diseases and had a low survival rate.
FRONTIERS IN IMMUNOLOGY
(2023)
Article
Medicine, General & Internal
R. Peffault de Latour, A. Kulasekararaj, S. Iacobelli, S. R. Terwel, R. Cook, M. Griffin, C. J. M. Halkes, C. Recher, F. Barraco, E. Forcade, J-C Vallejo, B. Drexler, J-B Mear, A. E. Smith, E. Angelucci, R. A. P. Raymakers, M. R. de Groot, E. Daguindau, E. Nur, W. Barcellini, N. H. Russell, L. Terriou, A-P Iori, U. La Rocca, A. Sureda, I Sanchez-Ortega, B. Xicoy, I Jarque, J. Cavenagh, F. Sicre de Fontbrune, S. Marotta, T. Munir, J. M. L. Tjon, S. Tavitian, A. Praire, L. Clement, F. Rabian, L. Marano, A. Hill, E. Palmisani, P. Muus, F. Cacace, C. Frieri, M-T Van Lint, J. R. Passweg, J. C. W. Marsh, G. Socie, G. J. Mufti, C. Dufour, A. M. Risitano
Summary: Eltrombopag improves the efficacy of standard immunosuppressive therapy for severe aplastic anemia, leading to higher complete response rates within 3 months.
NEW ENGLAND JOURNAL OF MEDICINE
(2022)
Article
Hematology
Jing Hu, Li Zhang, Xin Zhao, Xu Liu, Liping Jing, Kang Zhou, Yuan Li, Yang Li, Jianping Li, Lei Ye, Guangxin Peng, Huihui Fan, Wenrui Yang, Yang Yang, Youzhen Xiong, Lin Song, Fengkui Zhang
Summary: This study investigated the hematologic responses and long-term overall survival outcomes in severe aplastic anemia (SAA) patients who received rabbit antithymocyte globulin (rATG) and cyclosporine as first-line immunosuppressive therapy. The results showed that 3 mg/kg/day rATG is effective as first-line treatment for SAA.
ANNALS OF HEMATOLOGY
(2022)
Article
Hematology
Yang Lan, Lixian Chang, Meihui Yi, Yuli Cai, Jing Feng, Yuanyuan Ren, Chao Liu, Xiaoyan Chen, Shuchun Wang, Ye Guo, Aoli Zhang, Lipeng Liu, Jingliao Zhang, Xiaofan Zhu
Summary: This retrospective study analyzed the clinical outcomes of 172 children with newly diagnosed SAA treated with rabbit ATG and CsA, and found that mild disease severity was the only predictor for favorable response at 6 months. Factors such as BFU-E levels and time interval before treatment were associated with overall survival rates.
ANNALS OF HEMATOLOGY
(2021)
Article
Immunology
Guangyang Weng, Zhiping Fan, Huiwen Xue, Fen Huang, Na Xu, Hua Jin, Sijian Yu, Zhixin Ye, Jingchao Fan, Li Xuan, Qifa Liu
Summary: The study found that haploidentical donor stem cell transplantation had a lower incidence of bronchiolitis obliterans syndrome (BOS) compared to HLA-matched sibling donor stem cell transplantation. Additionally, the use of antithymocyte globulin (ATG) and mixed grafts may be protective factors in reducing the incidence of BOS.
FRONTIERS IN IMMUNOLOGY
(2022)
Article
Immunology
Yuanfeng Zhang, Xin Chen, Donglin Yang, Aiming Pang, Rongli Zhang, Qiaoling Ma, Weihua Zhai, Yi He, Jialin Wei, Erlie Jiang, Mingzhe Han, Sizhou Feng
Summary: This study conducted a retrospective cohort analysis on patients with severe aplastic anemia (SAA) who underwent allogeneic hematopoietic stem cell transplantation (allo-HSCT) to compare their survival between those with infection and those without infection. The study found that patients with infection before transplantation had inferior survival compared to those without infection, highlighting the importance of prophylaxis and complete control of infectious complications before transplantation among SAA patients.
FRONTIERS IN IMMUNOLOGY
(2022)
Article
Immunology
Sung-Soo Park, Gi June Min, Silvia Park, Sung-Eun Lee, Jae-Ho Yoon, Seung Hwan Shin, Byung-Sik Cho, Ki-Seong Eom, Yoo-Jin Kim, Seok Lee, Chang-Ki Min, Hee-Je Kim, Seok-Goo Cho, Jong Wook Lee
Summary: The study found that there were no significant differences in transplant outcomes among SAA patients receiving SCT from HLA-matched URDs, mismatched URDs, or HAPLO donors. However, selecting 6/8-URDs should be avoided due to inferior survival outcomes.
Article
Hematology
Yoshitaka Zaimoku, Bhavisha A. Patel, Sharon D. Adams, Ruba Shalhoub, Emma M. Groarke, Audrey Ai Chin Lee, Sachiko Kajigaya, Xingmin Feng, Olga Julia Rios, Holly Eager, Lemlem Alemu, Diego Quinones Raffo, Colin O. Wu, Willy A. Flegel, Neal S. Young
Summary: This study found that somatic loss of HLA class I alleles is correlated with clinical presentation and outcomes in immune aplastic anemia patients. Specific HLA genotypes such as HLA-B*14:02, HLA-B*40:02, and HLA-B*07:02 were found to be overrepresented in AA. High-risk clonal evolution, older age, and specific HLA genotypes contribute to a valid prediction model for AA patients.
Review
Pharmacology & Pharmacy
Jinsong Jia, Shasha Wang, Ying Kang, Xiujuan Liu, Lanping Xu
Summary: The meta-analysis conducted in this study showed that porcine antilymphocyte globulin (pALG) is as effective as rabbit antithymocyte globulin (rATG) in treating aplastic anemia (AA) patients, while rATG is associated with a significantly higher incidence rate of infection.
INTERNATIONAL JOURNAL OF CLINICAL PHARMACOLOGY AND THERAPEUTICS
(2023)
Article
Hematology
Tatsuhiro Sakamoto, Naoshi Obara, Yumiko Maruyama, Takayasu Kato, Naoki Kurita, Keiichiro Hattori, Yasuhito Suehara, Hidekazu Nishikii, Yasuhisa Yokoyama, Mamiko Sakata-Yanagimoto, Kensuke Usuki, Shigeru Chiba
Summary: A retrospective evaluation of 19 patients with relapsed or refractory aplastic anemia who received first-line rATG treatment in two centers between 2009 and 2020 revealed that IST2-rATG is effective and safe in treating this disease.
EUROPEAN JOURNAL OF HAEMATOLOGY
(2023)
Article
Biochemistry & Molecular Biology
Rong-Long Chen, Peng Peng Ip, Jy-juinn Shaw, Yun-Hsin Wang, Li-Hua Fan, Yi-Ling Shen, Nithila A. Joseph, Tsen-Erh Chen, Liuh-Yow Chen
Summary: Most cases of AA, including the SAMD9L-inherited subtype, are immune-mediated. The modified PTCy-based regimen presented in this study is efficient and safe for salvage.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2022)
Article
Medicine, General & Internal
Jiayu Wang, Meng Yuan, Guanghua Zhu, Runhui Wu, Chenguang Jia, Bin Wang, Jie Zheng, Jie Ma, Maoquan Qin, Sidan Li
Summary: This study analyzed the immune reconstitution of pediatric patients with aplastic anemia after hematopoietic stem-cell transplantation (HSCT) and found that early rapid immune reconstitution is a good predictor of clinical prognosis in children with AA.
INTERNATIONAL JOURNAL OF MEDICAL SCIENCES
(2022)