Article
Hematology
Xiaodi Ma, Yangyang Zuo, Zhengli Xu, Yuanyuan Zhang, Yifei Cheng, Tingting Han, Pan Suo, Yuqian Sun, Feifei Tang, Fengrong Wang, Chenhua Yan, Yuhong Chen, Yu Wang, Xiaohui Zhang, Kaiyan Liu, Xiaojun Huang, Lanping Xu
Summary: This study retrospectively compared the characteristics and outcomes of patients with hepatitis-associated aplastic anemia (HAAA) and non-hepatitis-associated aplastic anemia (non-HAAA) who underwent haploidentical hematopoietic stem cell transplantation (haplo-HSCT). The results showed no significant differences in neutrophil engraftment time, overall survival rates, and liver event-free survival rates between HAAA and non-HAAA patients. These findings suggest that haplo-HSCT is a feasible treatment option for HAAA when HLA-matched donors are not available, with low risks of transplant-related mortality and complications.
ANNALS OF HEMATOLOGY
(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
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
Oncology
Daijing Nie, Jing Zhang, Fang Wang, Xvxin Li, Lili Liu, Wei Zhang, Panxiang Cao, Xue Chen, Yang Zhang, Jiaqi Chen, Xiaoli Ma, Xiaosu Zhou, Qisheng Wu, Ming Liu, Mingyue Liu, Wenjun Tian, Hongxing Liu
Summary: The study suggests that heterozygous mutations in Fanconi anemia genes can contribute to hematologic disorders, particularly aplastic anemia and leukemia. Different genes have varying frequencies of mutations, with some genes showing associations with specific subgroups of hematologic malignancies.
FRONTIERS OF MEDICINE
(2022)
Article
Oncology
Hongchen Liu, Xiaoli Zheng, Chengtao Zhang, Jiajun Xie, Beibei Gao, Jing Shao, Yan Yang, Hengxiang Wang, Jinsong Yan
Summary: The study demonstrated the efficacy of haploidentical bone marrow transplantation (haplo-BMT) for patients with severe aplastic anemia II (SAA-II), achieving a 3-year overall survival and disease-free survival rate of 86.4% +/- 0.73%. This indicates that haplo-BMT could be considered as an alternative treatment strategy for SAA-II patients lacking HLA-matched donors.
FRONTIERS OF MEDICINE
(2021)
Article
Hematology
Bhavisha A. Patel, Danielle M. Townsley, Phillip Scheinberg
Summary: Severe aplastic anemia, a disease characterized by pancytopenia and a hypocellular marrow, can be treated with immunosuppressive therapy (IST) or hematopoietic stem cell transplant. Adding eltrombopag (EPAG) to the IST regimen improves treatment response rates without increasing the risk of myeloid malignancies. However, relapse remains a challenge, and further optimization of treatment protocols is needed.
SEMINARS IN HEMATOLOGY
(2022)
Article
Hematology
Liangliang Wu, Ming Zhou, Yumiao Li, Xiaowei Chen, Wenjian Mo, Caixia Wang, Shilin Xu, Wei Zhou, Tingfen Deng, Ruiqing Zhou, Shiyi Pan, Shunqing Wang, Yuping Zhang
Summary: Allogeneic hematopoietic stem cell transplantation (allo-HSCT) using bone marrow (BM) combined with peripheral blood stem cells (PBSC) as grafts and a modified post-transplantation cyclophosphamide (PTCy) regimen with an increased dose and backward-adjusted timing of antithymocyte globulin (ATG) is an effective and feasible treatment option for severe aplastic anemia (SAA), with high engraftment rates, low rates of graft failure and graft-versus-host disease (GVHD), and prolonged overall survival (OS) and GVHD-free and failure-free survival (GFFS).
TRANSPLANTATION AND CELLULAR THERAPY
(2023)
Article
Hematology
Yuan-xin Sun, Qi Feng, Shu-wen Wang, Xin Li, Zi Sheng, Jun Peng
Summary: This study found that the levels of HLA-G and ILT2 were significantly increased in the bone marrow of AA patients, inhibiting the proliferation of B cells. Compared with the control group, the number of pro-B plus pre-B cells decreased and the number of mature B cells increased in the bone marrow of AA patients. Inhibiting the HLA-G-ILT2 interaction may help to restore the proliferation of bone marrow B cells in AA patients.
ANNALS OF HEMATOLOGY
(2022)
Article
Hematology
Yun Li, Na Wang, Lin Li, Yang Cao, Jinhuan Xu, Jue Wang, Lifang Huang, Lanlan Wang, Liang Zou, Haiyan Wang, Yi Xiao, Jia Wei, Yicheng Zhang
Summary: Haploidentical transplantation with modified post-transplantation cyclophosphamide (PTCY) has shown promising results for aplastic anemia patients, leading to prolonged survival and reduced complications. The conditioning regimen consisting of various medications was effective in preventing graft versus host disease (GVHD) post-transplantation. Overall, the study demonstrated high rates of successful engraftment and survival among patients undergoing this treatment strategy.
TRANSPLANTATION AND CELLULAR THERAPY
(2021)
Article
Immunology
Xiaodi Ma, Zhengli Xu, Tingting Han, Yuanyuan Zhang, Wei Han, Haixia Fu, Xiaohui Zhang, Fan Lin, Xiaojun Huang, Lanping Xu
Summary: This study modified the Beijing Protocol for haplo-HSCT in SAA patients, achieving successful and stable engraftment while reducing the incidence of severe acute GVHD. In the first batch of 17 patients who received the novel regimen, all patients achieved transplantation without primary graft failure, resulting in a 100% survival rate.
FRONTIERS IN IMMUNOLOGY
(2023)
Article
Cell Biology
Yuanyuan Shao, Weiwei Qi, Xiaomei Zhang, Ningyuan Ran, Chunyan Liu, Rong Fu, Zonghong Shao
Summary: Patients with severe aplastic anemia (SAA) show abnormalities in both their plasma metabolomes and intestinal microbial compositions, which might reflect the molecular mechanisms involved in the defective immunity that characterizes SAA.
FRONTIERS IN CELL AND DEVELOPMENTAL BIOLOGY
(2021)
Article
Gastroenterology & Hepatology
Maxime Gonnot, Florent Neumann, Frederic Huet, Raphaelle Maudinas, Thierry Leblanc, Florence Lacaille
Summary: Hepatitis-associated aplastic anemia (HAAA) accounts for 4% of autoimmune hepatitis in children. This autoimmune disease could be due to a regulation defect in the immune response. Treatment options include hematopoietic stem cell transplantation and medication.
JOURNAL OF PEDIATRIC GASTROENTEROLOGY AND NUTRITION
(2022)
Review
Biochemistry & Molecular Biology
Xinyu Tang, Ruirong Xu, Yan Wang, Kaiqing Chen, Siyuan Cui
Summary: The TERC gene is crucial for telomerase-dependent telomere extension and maintenance. Cell reprogramming can reverse differentiation and enhance pluripotency, and also extend telomere length. This study investigates the role of TERC haploinsufficiency and cell reprogramming in aplastic anemia, aiming to identify diagnostic indicators and therapeutic strategies.
MOLECULAR MEDICINE
(2023)
Article
Immunology
Pengpeng Pan, Congcong Chen, Jian Hong, Yue Gu
Summary: Acquired aplastic anemia (AA) is an autoimmune disease characterized by depleted hematopoietic stem and progenitor cells (HSPCs) and peripheral blood cells. Although immunosuppressive therapy (IST) is an effective treatment, its limitations necessitate further research into the pathogenesis of AA and identification of new drug targets to improve outcomes.
INTERNATIONAL IMMUNOPHARMACOLOGY
(2023)
Article
Pharmacology & Pharmacy
Xuan Gao, Zhu-Li Bian, Xiao-Hong Qiao, Xiao-Wen Qian, Jun Li, Guo-Mei Shen, Hui Miao, Yi Yu, Jian-Hua Meng, Xiao-Hua Zhu, Jun-Ye Jiang, Jun Le, Ling Yu, Hong-Sheng Wang, Xiao-Wen Zhai
Summary: This study developed a population pharmacokinetic model of cyclosporine (CsA) in Chinese pediatric patients with acquired aplastic anemia (AA) and identified body weight and total bilirubin level as important factors affecting the pharmacokinetics of CsA.
FRONTIERS IN PHARMACOLOGY
(2022)
Article
Hematology
Deepak Kumar, Thinh H. Nguyen, Carolyn M. Bennett, Chengyu Prince, Laura Lucas, Sunita Park, Taylor Lawrence, Karin Chappelle, Mariam Ishaq, Edmund K. Waller, Sampath Prahalad, Michael Briones, Shanmuganathan Chandrakasan
Summary: mTOR inhibitors like sirolimus are increasingly used to manage multilineage immune cytopenia (m-IC) in children. However, it is unclear how sirolimus affects the broader immune dysregulation associated with m-IC.
Letter
Hematology
Marta Davidson, Florence Wong, Mostafa Atri, Hassan Sibai, Dawn Maze, Verna Cheung, Jeannie Callum, Eshetu G. Atenafu, Vikas Gupta
AMERICAN JOURNAL OF HEMATOLOGY
(2023)
Editorial Material
Hematology
Mary Eapen
Summary: This article reports on the outcomes of allogeneic hematopoietic stem cell transplantation (allo-HSCT) and conservative management for adult patients with inborn errors of immunity (IEI).
Article
Hematology
Naveen Pemmaraju, Hagop Kantarjian, Kendra Sweet, Eunice Wang, Jayastu Senapati, Nathaniel R. Wilson, Marina Konopleva, Arthur E. Frankel, Vikas Gupta, Ruben Mesa, Matthew Ulrickson, Edward Gorak, Sumeet Bhatia, Tulin Budak-Alpdogan, James Mason, Maria Teresa Garcia-Romero, Norma Lopez-Santiago, Gabriela Cesarman-Maus, Pankit Vachhani, Sangmin Lee, Vijaya Raj Bhatt, William Blum, Roland B. Walter, Dale Bixby, Ivana Gojo, Madeleine Duvic, Raajit K. Rampal, Marcos de Lima, James Foran, Amir T. Fathi, Aric Cameron Hall, Meagan A. Jacoby, Jeffrey Lancet, Gabriel Mannis, Anthony S. Stein, Alice Mims, David Rizzieri, Rebecca Olin, Alexander Perl, Gary Schiller, Paul Shami, Richard M. Stone, Stephen Strickland, Matthew J. Wieduwilt, Naval Daver, Farhad Ravandi, Sumithira Vasu, Monica Guzman, Gail J. Roboz, Joseph Khoury, Muzaffar Qazilbash, Phyu P. Aung, Branko Cuglievan, Yazan Madanat, Mohamed A. Kharfan-Dabaja, Anna Pawlowska, Justin Taylor, Martin Tallman, Prajwal Dhakal, Andrew A. Lane
Summary: Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare hematologic malignancy with frequent cutaneous involvement and potential invasion of other compartments. CD123 is a targetable surface marker in BPDCN, and efforts are being made to explore novel approaches targeting CD123 and other targets. The North American BPDCN Consortium (NABC) was formed to define the current standard of care and identify research questions and future directions in BPDCN.
Article
Hematology
Ram Vasudevan Nampoothiri, Kenny Tang, Andre Schuh, Wilson Lam, Dawn Maze, Fotios V. Michelis, Steven Chan, Vikas Gupta, Dennis Kim, Rajat Kumar, Jeffrey Howard Lipton, Jonas Mattsson, Mark Minden, Aaron Schimmer, Hassan Sibai, Auro Viswabandya, Karen Yee, Tracy Murphy, Arjun D. Law
Summary: Inconclusive cytogenetic analysis may not be an independent prognostic indicator in patients with acute myeloid leukemia (AML). Molecular abnormalities detected through NGS or whole genome sequencing are more likely to be informative in these patients.
EUROPEAN JOURNAL OF HAEMATOLOGY
(2023)
Article
Oncology
Mary Eapen, Ruta Brazauskas, David A. Williams, Mark C. Walters, Andrew St Martin, Benjamin L. Jacobs, Joseph H. Antin, Kira Bona, Sonali Chaudhury, Victoria H. Coleman-Cowger, Nancy L. DiFronzo, Erica B. Esrick, Joshua J. Field, Courtney D. Fitzhugh, Julie Kanter, Neena Kapoor, Donald B. Kohn, Lakshmanan Krishnamurti, Wendy B. London, Michael A. Pulsipher, Sohel Talib, Alexis A. Thompson, Edmund K. Waller, Ted Wun, Mary M. Horowitz
Summary: This study investigates the incidence and risk factors for secondary neoplasm after transplantation for sickle cell disease. The results show that the 10-year incidence of leukemia/MDS was 1.7% and of any secondary neoplasm was 2.4%. Low-intensity regimens were associated with higher risks for leukemia/MDS or any secondary neoplasm compared with more intense regimens.
JOURNAL OF CLINICAL ONCOLOGY
(2023)
Article
Hematology
RuiQi Chen, Eshetu G. Atenafu, Jack Seki, Xing Liu, Steven Chan, Vikas Gupta, Dawn Maze, Andre C. Shuh, Mark D. Minden, Karen Yee, Aaron D. Schimmer, Hassan Sibai
Summary: This study found that PEG-ASP is associated with a higher incidence of VTE compared to L-ASP in patients with ALL, despite prophylactic anticoagulation. Further VTE mitigation strategies are needed for adult patients receiving PEG-ASP.
BRITISH JOURNAL OF HAEMATOLOGY
(2023)
Article
Hematology
Guru Subramanian Guru Murthy, Soyoung Kim, Noel Estrada-Merly, Muhammad Bilal Abid, Mahmoud Aljurf, Amer Assal, Talha Badar, Sherif M. Badawy, Karen Ballen, Amer Beitinjaneh, Jan Cerny, Saurabh Chhabra, Zachariah DeFilipp, Bhagirathbhai Dholaria, MiguelAngel Diaz Perez, Shatha Farhan, Cesar O. Freytes, Robert Peter Gale, Siddhartha Ganguly, Vikas Gupta, Michael R. Grunwald, Nada Hamad, Gerhard C. Hildebrandt, Yoshihiro Inamoto, Tania Jain, Omer Jamy, Mark Juckett, Matt Kalaycio, Maxwell M. Krem, Hillard M. Lazarus, Mark Litzow, Reinhold Munker, Hemant S. Murthy, Sunita Nathan, Taiga Nishihori, Guillermo Orti, Sagar S. Patel, Marjolein van der Poel, David A. Rizzieri, Bipin N. Savani, Sachiko Seo, Melhem Solh, Leo F. Verdonck, Baldeep Wirk, Jean A. Yared, Ryotaro Nakamura, Betul Oran, Bart Scott, Wael Saber
Summary: Allogeneic hematopoietic cell transplantation (allo-HCT) is the only curative treatment for myelofibrosis. However, the optimal conditioning regimen for this procedure is still unknown.
Article
Oncology
John Mascarenhas, Marina Kremyanskaya, Andrea Patriarca, Francesca Palandri, Timothy Devos, Francesco Passamonti, Raajit K. Rampal, Adam J. Mead, Gabriella Hobbs, Joseph M. Scandura, Moshe Talpaz, Nikki Granacher, Tim C. P. Somervaille, Ronald Hoffman, Marielle J. Wondergem, Mohamed E. Salama, Gozde Colak, Jike Cui, Jean-Jacques Kiladjian, Alessandro M. Vannucchi, Srdan Verstovsek, Natalia Curto-Garcia, Claire Harrison, Vikas Gupta
Summary: In patients with myelofibrosis who are naive to JAKi treatment, the rational combination of the BET inhibitor pelabresib and ruxolitinib showed good tolerability and durable improvements in spleen and symptom burden.
JOURNAL OF CLINICAL ONCOLOGY
(2023)
Article
Oncology
Francesca Palandri, Haifa Kathrin Al-Ali, Paola Guglielmelli, Mike W. Zuurman, Rajendra Sarkar, Vikas Gupta
Summary: Bone marrow fibrosis (BMF) is a negative prognostic factor for myelofibrosis (MF). The JUMP trial investigated the safety and efficacy of the JAK1/JAK2 inhibitor ruxolitinib in symptomatic MF patients. This post hoc analysis found that patients with low-grade fibrosis (LGF) had better response rates and survival estimates compared to patients with high-grade fibrosis (HGF). Early initiation of ruxolitinib therapy was associated with increased response rates in all patients.
Article
Oncology
Nora-Medea Messerich, Narasimha Rao Uda, Thomas Volken, Sergio Cogliatti, Thomas Lehmann, Andreas Holbro, Rudolf Benz, Lukas Graf, Vikas Gupta, Wolfram Jochum, Izadora Demmer, Tata Nageswara Rao, Tobias Silzle
Summary: In myelofibrosis, the C-reactive protein (CRP)/albumin ratio (CAR) and the Glasgow Prognostic Score (GPS) add independent prognostic information. Assessing CRP and albumin helps to identify a vulnerable population of MF patients, which eludes current prognostic models, even if the presence of high-risk mutations is considered. Further evaluation of albumin and CRP as prognostic markers in MF is warranted.
Article
Oncology
Anne Tierens, Elizabeth Kagotho, Satoru Shinriki, Andrew Seto, Adam C. Smith, Melanie Care, Dawn Maze, Hassan Sibai, Karen W. Yee, Andre C. Schuh, Dennis Dong Hwan Kim, Vikas Gupta, Mark D. Minden, Hirotaka Matsui, Jose-Mario Capo-Chichi
Summary: Inherited DDX41 mutations cause familial predisposition to hematologic malignancies including acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). However, the genotype-phenotype correlation in DDX41-MDS/AML remains poorly understood.
FRONTIERS IN ONCOLOGY
(2023)
Article
Hematology
Muhammad Bilal Abid, Noel Estrada-Merly, Mei-Jie Zhang, Karen Chen, Christopher Bredeson, David Allan, Mitchell Sabloff, David I. Marks, Mark Litzow, Christopher Hourigan, Partow Kebriaei, Wael Saber
Summary: This retrospective study found that selecting younger unrelated donors can significantly reduce the risk of disease relapse in older ALL patients. However, younger unrelated donors also increase the risk of chronic GVHD and nonrelapse mortality.
TRANSPLANTATION AND CELLULAR THERAPY
(2023)
Meeting Abstract
Oncology
Michael R. Savona, James K. McCloskey, Elizabeth A. Griffiths, Karen W. L. Yee, Amer M. Zeidan, Aref Al-Kali, H. Joachim Deeg, Prapti A. Patel, Mitchell Sabloff, Mary-Margaret Keating, Nancy Zhu, Nashat Gabrail, Salman Fazal, Joseph Maly, Olatoyosi Odenike, Hagop M. Kantarjian, Amy E. DeZern, Casey L. O'Connell, Gail J. Roboz, Lambert Busque, Rena Buckstein, Harshad Amin, Jasleen K. Randhawa, Brian Leber, Kim-Hien Dao, Yuri Sano, Beloo Mirakhur, Winny Chan, Aram Oganesian, Harold Keer, Guillermo Garcia-Manero
CLINICAL LYMPHOMA MYELOMA & LEUKEMIA
(2023)
Meeting Abstract
Oncology
Rajneesh Nath, Stuart Seropian, Hannah Choe, Mark R. Litzow, Camille Abboud, Nebu Koshy, Patrick J. Stiff, Benjamin Tomlinson, Sunil Abhyankar, James M. Foran, Parameswaran Hari, George L. Chen, Zaid S. Al-Kadhimi, Partow Kebriaei, Mitchell Sabloff, Johnnie J. Orozco, Katarzyna Jamieson, Margarida Magalhaes-Silverman, Koen Van Besien, Michael Schuster, Arjun Law, Sameem Abedin, Karilyn Larkin, Scott Rowley, Pashna Munshi, Rachel Cook, Sebastian Mayer, Moshe Yair Levy, Hillard M. Lazarus, Brenda M. Sandmaier, Vijay Reddy, Jennifer Spross, Kathleen McNamara, Elaina Haeuber, Madhuri Vusirikala, Akash Nahar, John M. Pagel, Sergio A. Giralt, Avinash Desai, Boglarka Gyurkocza
CLINICAL LYMPHOMA MYELOMA & LEUKEMIA
(2023)
