Article
Immunology
Meiqing Lei, Yanming Zhang, Wenjing Jiao, Xiaoli Li, Huifen Zhou, Qingyuan Wang, Huiying Qiu, Xiaowen Tang, Yue Han, Chengcheng Fu, Zhengming Jin, Suning Chen, Aining Sun, Miao Miao, Limin Liu, Depei Wu
Summary: This study compares the feasibility and efficacy of haploidentical hematological stem cell transplantation combined with a single unrelated cord blood infusion and haplo-identical HSCT alone in severe aplastic anemia patients. The findings suggest that the coinfusion of a single UCB potentially improves survival of Haplo-HSCT, and an HLA-A allele-matched UCB is the preferred option.
FRONTIERS IN IMMUNOLOGY
(2022)
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
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)
Review
Hematology
Raheel Iftikhar, Qamar un Nisa Chaudhry, Faiz Anwer, Karun Neupane, Abdul Rafae, Syed Kamran Mahmood, Tariq Ghafoor, Nighat Shahbaz, Mehreen Ali Khan, Tariq Azam Khattak, Ghassan Umair Shamshad, Jahanzeb Rehman, Muhammad Farhan, Maryam Khan, Iqraa Ansar, Rabia Ashraf, Judith Marsh, Tariq Mehmood Satti, Parvez Ahmed
Summary: Treatment options for newly diagnosed aplastic anemia patient include upfront allogeneic hematopoietic stem cell transplant (HSCT) or immunosuppressive therapy (IST). Eligibility for upfront HSCT should be assessed based on age, comorbidities, donor availability and response probability to IST. Upfront HSCT is recommended for children and young adults with matched related donor (MRD) and those needing urgent HSCT with matched unrelated donor (MUD).
Article
Immunology
Fan Lin, Tingting Han, Yuanyuan Zhang, Yifei Cheng, Zhengli Xu, Xiaodong Mo, Fengrong Wang, Chenhua Yan, Yuqian Sun, Jingzhi Wang, Feifei Tang, Wei Han, Yuhong Chen, Yu Wang, Xiaohui Zhang, Kaiyan Liu, Xiaojun Huang, Lanping Xu
Summary: Secondary poor graft function (sPGF) after haploidentical hematopoietic stem cell transplantation (HSCT) in patients with acquired aplastic anemia (AA) has a cumulative incidence of 4.62% and is associated with lower 2-year overall survival. Later neutrophil engraftment and a history of refractory cytomegalovirus viremia are risk factors for sPGF.
FRONTIERS IN IMMUNOLOGY
(2022)
Article
Oncology
Fan Lin, Yangyang Zuo, Yuanyuan Zhang, Yifei Cheng, Tingting Han, Xiaodong Mo, Pan Suo, Yuqian Sun, Feifei Tang, Fengrong Wang, Chenhua Yan, Yuhong Chen, Wei Han, Jingzhi Wang, Yu Wang, Xiaohui Zhang, Kaiyan Liu, Xiaojun Huang, Lanping Xu
Summary: Elevated pretransplant serum ferritin levels are associated with increased incidences of higher-grade graft-versus-host disease after haploidentical hematopoietic stem cell transplant in children and adolescents with acquired severe aplastic anemia. However, it does not affect survival rates for these patients.
PEDIATRIC BLOOD & CANCER
(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
Oncology
Juan Chen, Yuanfeng Zhang, Xin Chen, Aiming Pang, Yuanqi Zhao, Li Liu, Runzhi Ma, Jialin Wei, Yi He, Donglin Yang, Rongli Zhang, Weihua Zhai, Qiaoling Ma, Erlie Jiang, Mingzhe Han, Jiaxi Zhou, Sizhou Feng
Summary: A retrospective study found that P-ALG and R-ATG have similar efficacy and safety in patients with acquired aplastic anemia undergoing haplo-HSCT. P-ALG could be an alternative option to R-ATG.
CANCER CELL INTERNATIONAL
(2022)
Article
Immunology
Diego Medina-Valencia, Daniela Cleves, Estefania Beltran, Natalia Builes, Alexis A. Franco, Andres Felipe Escobar-Gonzalez, Manuela Olaya
Summary: Primary hemophagocytic lymphohistiocytosis is a severe and uncommon disease affecting pediatric patients, with genetic abnormalities linked to altered apoptosis and exaggerated inflammatory reactions. This study describes a novel treatment strategy involving haploidentical hematopoietic stem cell transplantation, yielding positive outcomes for all pediatric patients treated. The results suggest this strategy could be a promising alternative in cases where other donor options are limited.
JOURNAL OF CLINICAL IMMUNOLOGY
(2021)
Article
Immunology
Yuehong Zhang, Yuqin Liang, Xu Zhang, Shunqing Wang, Jinpeng Cao, Zongyin Gao, Ling Li, Wenjian Mo
Summary: This study assessed the risk of cytomegalovirus retinitis in severe aplastic anemia patients receiving hematopoietic stem cell transplants. The results showed that pre-transplant platelet refractoriness and alternative donors were significant predictors of cytomegalovirus retinitis. These findings provide important evidence for the development of prevention strategies and preemptive treatment for severe aplastic anemia recipients.
FRONTIERS IN CELLULAR AND INFECTION MICROBIOLOGY
(2022)
Article
Pediatrics
Mohammed Saud Alsaidan, Ohoud Zaid Aljarbou, Waleed Alajroush
Summary: This article reports a rare case of Kaposi sarcoma in a child after hematopoietic stem cell transplantation (HSCT). The patient developed asymptomatic nodular skin lesions, and additional lesions in the liver and oral cavity were confirmed. The patient was treated with Sirolimus and topical timolol, and the lesions completely resolved.
Article
Biochemistry & Molecular Biology
Peng Peng Ip, Li-Hua Fang, Yi-Ling Shen, Kuan-Chiun Tung, Ming-Tsong Lai, Li-Ying Juan, Liuh-Yow Chen, Rong-Long Chen
Summary: Graves' disease, characterized by hyperthyroidism, can be influenced by both genetic and environmental factors. A rare case of Graves' disease in a patient who underwent hematopoietic stem cell transplantation (HSCT) is reported, suggesting that both genetic background and the HSCT environment can contribute to the development of Graves' disease.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2022)
Article
Immunology
Yuanfeng Zhang, Jiali Huo, Li Liu, Yuyan Shen, Juan Chen, Tingting Zhang, Xin Chen, Aiming Pang, Donglin Yang, Rongli Zhang, Qiaoling Ma, Weihua Zhai, Yi He, Jialin Wei, Erlie Jiang, Mingzhe Han, Yizhou Zheng, Sizhou Feng
Summary: This study retrospectively compared the efficacy of different treatment strategies in 387 patients with acquired aplastic anemia (AA). The results showed that haploidentical donor HSCT had higher rates of graft failure and acute chronic GvHD compared with matched sibling donor HSCT and immunosuppressive therapy. HSCT had better blood count recovery compared with IST. In patients aged ≤40 years, MSD-HSCT was superior to HID-HSCT, while HID-HSCT and IST had similar overall survival rates but better failure-free survival rates when followed up for more than 14.5 months.
FRONTIERS IN IMMUNOLOGY
(2022)
Review
Oncology
Yuan Yang, Jiang Ji, Zengwei Tang, Bing Han
Summary: This study compares the efficacy and safety of EPAG+IST and haplo-HSCT in treating severe aplastic anemia. The results show that EPAG+IST has comparable overall response rate and 1-/2-year overall survival, but lower complete response rate compared to haplo-HSCT. Patients treated with haplo-HSCT may experience a higher incidence of GVHD, while those treated with EPAG+IST may have more relapses or clone evolution.
FRONTIERS IN ONCOLOGY
(2021)
Article
Genetics & Heredity
Tonglin Hu, Yanna Zhao, Xiaoling Yu, Ruilan Gao, Liming Yin
Summary: This study used single-cell transcriptomic sequencing to reveal the transcriptomic landscape of hematopoiesis in AA patients, and identified the aberrance of B cells as a factor contributing to hematopoiesis failure. These findings provide valuable insights and potential targets for the treatment of AA.
FRONTIERS IN GENETICS
(2022)
Review
Cell Biology
Mirella Pace, Elia Guadagno, Daniela Russo, Annarita Gencarelli, Annunziata Carlea, Attilio Di Spiezio, Clara Bertuzzi, Massimo Mascolo, Francesco Grimaldi, Luigi Insabato
Summary: This case report presents a rare occurrence of myeloid sarcoma in the breast of a patient with a history of JAK2-mutated essential thrombocythemia. The histology initially resembled lobular carcinoma of the breast, but immunohistochemical evaluation provided a clear diagnosis and prognosis.
Article
Hematology
Yves Chalandon, Giulia Sbianchi, Luuk Gras, Linda Koster, Jane Apperley, Jenny Byrne, Urpu Salmenniemi, Henrik Sengeloev, Mahmoud Aljurf, Grzegorz Helbig, Francesca Kinsella, Goda Choi, Peter Remenyi, John A. Snowden, Marie Robin, Stig Lenhoff, Stephan Mielke, Jakob Passweg, Annoek E. C. Broers, Nicolaus Kroeger, Zeynep Arzu Yegin, Sen Mui Tan, Patrick J. Hayden, Donal P. McLornan, Ibrahim Yakoub-Agha
Summary: With the introduction of TKI, the number of allo-HCT for CP CML patients has decreased significantly. The use of 1, 2, or 3 TKI prior to allo-HCT does not affect the overall survival, progression-free survival, relapse incidence, or non-relapse mortality. However, the disease stage and KPS score significantly influence the survival and progression-free survival, emphasizing the importance of careful candidate selection and performing allo-HCT during CP1 phase.
AMERICAN JOURNAL OF HEMATOLOGY
(2023)
Article
Hematology
Guillermo Orti, Luuk Gras, Nienke Zinger, Maria Chiara Finazzi, Katja Sockel, Marie Robin, Edouard Forcade, Daniele Avenoso, Nicolaus Kroeger, Juergen Finke, Aleksandar Radujkovic, Mathilde Hunault-Berger, Wilfried Schroyens, Tsila Zuckerman, Jean Henri Bourhis, Yves Chalandon, Adrian Bloor, Rik Schots, Liesbeth C. de Wreede, Joana Drozd-Sokolowska, Kavita Raj, Nicola Polverelli, Tomasz Czerw, Juan Carlos Hernandez-Boluda, Donal McLornan, Ibrahim Yakoub-Agha
Summary: Allogeneic hematopoietic cell transplant (allo-HCT) is the only potential route to long-term remission for patients with blast phase transformation of myeloproliferative neoplasm (BP-MPN). A large retrospective registry-based study showed that the 3-year overall survival (OS) of BP-MPN patients undergoing allo-HCT was 36%. Factors associated with lower OS included Karnofsky Performance Score < 90 and active disease at allo-HCT, while more recent allo-HCT was associated with higher OS.
AMERICAN JOURNAL OF HEMATOLOGY
(2023)
Article
Biophysics
Anna Waszczuk-Gajda, Luuk Gras, Liesbeth C. de Wreede, Tiarlan Sirait, Arpad Illes, Zubeyde Nur Ozkurt, John A. Snowden, Mutlu Arat, Claude Eric Bulabois, Judith Niederland, Matjaz Sever, Shankara Paneesha, Victoria Potter, Alain Gadisseur, Thomas Chalopin, Gwendolyn Van Gorkom, Joaquin Martinez Lopez, Tessa Kerre, Joanna Drozd-Sokolowska, Kavita Raj, Patrick J. Hayden, Meral Beksac, Ibrahim Yakoub-Agha, Donal P. McLornan, Stefan Schoenland
Summary: The role of high-dose chemotherapy followed by autologous stem cell transplantation (ASCT) in the treatment of myeloma (MM) patients with severe and/or dialysis-dependent renal impairment remains uncertain. We report on the outcomes of 110 patients (median age 57 years) who had become dialysis-dependent pre-ASCT and who underwent a first ASCT between 1997 and 2017. Age at ASCT and response at ASCT (CR/VGPR/PR vs. MR/SD/progression) were significantly associated with better OS and PFS according to multivariate analyses.
BONE MARROW TRANSPLANTATION
(2023)
Letter
Hematology
Dai Chihara, Luuk Gras, Nienke Zinger, Nicolaus Kroeger, Jiri Mayer, Jakob Passweg, Regis Peffault de Latour, Jenny Byrne, William Krueger, Jan-Paul Bohn, Uwe Platzbecker, Igor Wolfgang Blau, Francesca Bonifazi, Grzegorz Helbig, Andrew McDonald, Martin Mistrik, Mohamad Mohty, Ron Ram, Jaime Sanz, Carlos Vallejo Llamas, Robert J. Kreitman, Patrick J. Hayden, Donal McLornan, Olivier Tournilhac, Michel van Gelder, Ibrahim Yakoub-Agha
Article
Hematology
Patrick Harrington, Richard Dillon, Deepti Radia, Philippe Rousselot, Donal P. McLornan, Mark Ong, Anna Green, Alessandro Verde, Farzana Hussain, Kavita Raj, Shahram Kordasti, Claire Harrison, Hugues de Lavallade
Summary: Dasatinib is a multi-kinase inhibitor that inhibits the SRC kinase LCK, resulting in inhibition of T-cell receptor signaling. It has been found to enhance tumor immunity and selectively deplete regulatory T cells (Tregs), leading to potential immunotherapeutic applications. This study compared the impact of dasatinib with other tyrosine kinase inhibitors (TKI) on chronic myeloid leukemia patients and found that dasatinib inhibits TCR and STAT5 signaling pathways, reduces pro-inflammatory cytokines, and preserves anti-tumor immunity by preventing T-cell exhaustion through TIM-3 signaling. These findings provide insights into the immunomodulatory effects of dasatinib and its potential use in immunotherapies.
Review
Hematology
Nicola Polverelli, Juan Carlos Hernandez-Boluda, Tomasz Czerw, Tiziano Barbui, Mariella D'Adda, Hans Joachim Deeg, Markus Ditschkowski, Claire Harrison, Nicolaus Martin Kroger, Ruben Mesa, Francesco Passamonti, Francesca Palandri, Naveen Pemmaraju, Uday Popat, Damiano Rondelli, Alessandro Maria Vannucchi, Srdan Verstovsek, Marie Robin, Antonio Colecchia, Luigi Grazioli, Enrico Damiani, Domenico Russo, Jessica Brady, David Patch, Slawomir Blamek, Gandhi Laurent Damaj, Patrick Hayden, Donal P. McLornan, Ibrahim Yakoub-Agha
Summary: Splenomegaly is a common complication in myelofibrosis patients and can negatively impact outcomes of allogeneic hematopoietic cell transplantation (HCT). This Position Paper provides a shared position statement on the management of splenomegaly before HCT. The assessment, prevalence, and clinical significance of splenomegaly are discussed, along with the need for therapeutic intervention. Specific scenarios, such as splanchnic vein thrombosis and COVID-19, are also addressed.
LANCET HAEMATOLOGY
(2023)
Review
Hematology
Rhea Suribhatla, Thomas Starkey, Maria C. C. Ionescu, Antonio Pagliuca, Alex Richter, Lennard Y. W. Lee
Summary: Immunocompromised patients, especially those with haematological malignancies, have a higher susceptibility to SARS-CoV-2 infection and severe outcomes. This systematic review and meta-analysis demonstrate that tixagevimab/cilgavimab has a significant clinical effectiveness in reducing the risk of COVID-19 infection, hospitalization, intensive care admission, and COVID-19-specific mortality in immunocompromised individuals.
BRITISH JOURNAL OF HAEMATOLOGY
(2023)
Article
Oncology
Ruben A. Mesa, Stacie Hudgens, Lysbeth Floden, Claire N. Harrison, Jeanne Palmer, Vikas Gupta, Donal P. McLornan, Mary F. McMullin, Jean-Jaques Kiladjian, Lynda Foltz, Uwe Platzbecker, M. Laura Fox, Adam J. Mead, David M. Ross, Stephen T. Oh, Andrew Perkins, Michael F. Leahy, Samineh Deheshi, Rafe Donahue, Barbara J. Klencke, Srdan Verstovsek
Summary: This study evaluated the longitudinal change in total symptom score (TSS) and individual symptom scores in MF patients receiving therapy, and found that momelotinib provided clinically relevant symptom benefits compared to control group.
Article
Hematology
Roni Tamari, Donal P. Mclornan, Kwang Woo Ahn, Noel Estrada-Merly, Juan Carlos Hernandez-Boluda, Sergio Giralt, Jeanne Palmer, Robert Peter Gale, Zachariah Defilipp, David I. Marks, Marjolein van der Poel, Leo F. Verdonck, Minoo Battiwalla, Miguel Angel Diaz, Vikas Gupta, Haris Ali, Mark Robert Litzow, Hillard M. Lazarus, Usama Gergis, Asad Bashey, Jane Liesveld, Shahrukh Hashmi, Jeffrey J. Pu, Amer Beitinjaneh, Christopher Bredeson, David Rizzieri, Bipin N. Savani, Muhammad Bilal Abid, Siddhartha Ganguly, Vaibhav Agrawal, Vera Ulrike Bacher, Baldeep Wirk, Tania Jain, Corey Cutler, Mahmoud Aljurf, Tamila Kindwall-Keller, Mohamed A. Kharfan-Dabaja, Gerhard C. Hildebrandt, Attaphol Pawarode, Melhem M. Solh, Jean A. Yared, Michael R. Grunwald, Sunita Nathan, Taiga Nishihori, Sachiko Seo, Bart L. Scott, Ryotaro Nakamura, Betul Oran, Tomasz Czerw, Ibrahim Yakoub-Agha, Wael Saber
Summary: A prognostic model was developed for myelofibrosis patients undergoing allogeneic hematopoietic cell transplantation. Factors such as patient age, donor compatibility, and hemoglobin levels were found to be associated with mortality risk. The proposed scoring system could accurately predict overall survival and transplant-related mortality in two large cohorts.
Letter
Hematology
Marie Robin, Liesbeth C. de Wreede, Thomas Schroeder, Friedrich Stoelzel, Nicolaus Kroeger, Linda Koster, Uwe Platzbecker, Juergen Finke, Arnold Ganser, Didier Blaise, Fabio Ciceri, Johan Maertens, Helene Labussiere Wallet, Junfeng Wang, Patrice Chevallier, Jakob Passweg, Jan J. Cornelissen, Stephanie Nguyen, Edouard Forcade, Amandine Charbonnier, Francesca Bonifazi, Patrick Hayden, Donal P. McLornan, Ibrahim Yakoub-Agha
Review
Hematology
Dina Mahdi, Jessica Spiers, Alexandros Rampotas, Nicola Polverelli, Donal P. P. McLornan
Summary: Management approaches for accelerated and blast phase myeloproliferative neoplasms remain challenging, and current therapeutic outcomes for non-transplant eligible patients are poor. There is currently no specific predictive signature for the risk and timing of transformation, causing anxiety among patients and clinicians. This review provides an up-to-date understanding of the pathogenesis, discusses current therapies and outcomes, and explores the potential impact of novel agents.
BRITISH JOURNAL OF HAEMATOLOGY
(2023)
Letter
Medicine, Research & Experimental
P. Sriskandarajah, D. P. McLornan, C. Oni, A. J. Wilson, C. Woodley, M. Ciesielska, K. Raj, R. Dillon, M. Ethell, J. Chacko, K. Orchard, D. H. Radia
Summary: Advanced systemic mastocytosis (AdvSM) is a rare, life-limiting mast cell (MC) neoplasm, which is often associated with a hematological neoplasm (AHN) in approximately 70% of patients. Avapritinib, a selective tyrosine kinase inhibitor, has shown potent activity in treating AdvSM and has successfully bridged three patients to allogeneic hematopoietic cell transplant (allo-HCT). Two cases also highlight the risk of clonal evolution within the AHN component and the necessity of close monitoring during targeted therapy.
CURRENT RESEARCH IN TRANSLATIONAL MEDICINE
(2023)
Article
Medicine, General & Internal
Srdan Verstovsek, Aaron T. Gerds, Alessandro M. Vannucchi, Haifa Kathrin Al-Ali, David Lavie, Andrew T. Kuykendall, Sebastian Grosicki, Alessandra Iurlo, Yeow Tee Goh, Mihaela C. Lazaroiu, Miklos Egyed, Maria Laura Fox, Donal McLornan, Andrew Perkins, Sung -Soo Yoon, Vikas Gupta, Jean -Jacques Kiladjian, Nikki Granacher, Sung-Eun Lee, Luminita Ocroteala, Francesco Passamonti, Claire N. Harrison, Barbara J. Klencke, Sunhee Ro, Rafe Donahue, Jun Kawashima, Ruben Mesa
Summary: This study aimed to compare the clinical effects of momelotinib and danazol in patients with intermediate or high-risk myelofibrosis. The results showed that momelotinib can significantly improve myelofibrosis-associated symptoms, anemia measures, and spleen response compared to danazol, with favorable safety.
Article
Hematology
Donal P. McLornan, Anna L. Godfrey, Anna Green, Rebecca Frewin, Siamak Arami, Jessica Brady, Nauman M. Butt, Catherine Cargo, Joanne Ewing, Sebastian Francis, Mamta Garg, Claire Harrison, Andrew Innes, Alesia Khan, Steve Knapper, Jonathan Lambert, Adam Mead, Andrew McGregor, Pratap Neelakantan, Bethan Psaila, Tim C. P. Somervaille, Claire Woodley, Jyoti Nangalia, Nicholas C. P. Cross, Mary Frances McMullin
BRITISH JOURNAL OF HAEMATOLOGY
(2023)