Article
Oncology
Jiang Ji, Ziqi Wan, Jing Ruan, Yuan Yang, Qinglin Hu, Zesong Chen, Chen Yang, Miao Chen, Bing Han
Summary: This trial compared the efficacy of eltrombopag (EPAG)+tacrolimus versus EPAG monotherapy in patients with refractory/relapsed acquired aplastic anaemia (AA). The overall response rate was higher in the EPAG+tacrolimus group compared to the EPAG alone group, but there were no significant differences in safety, clonal evolution, and survival between the two groups. A subgroup analysis suggested that EPAG+tacrolimus may have better efficacy in patients under 60 years old.
BLOOD CANCER JOURNAL
(2023)
Correction
Hematology
Jing Ruan, Wei Zuo, Miao Chen, Chen Yang, Bing Han
Summary: The effectiveness of Eltrombopag in patients with relapse/refractory aplastic anemia is reported from a single center in China.
ANNALS OF HEMATOLOGY
(2021)
Article
Oncology
Harry Lesmana, Timothy Jacobs, Michelle Boals, Nathan Gray, Sara Lewis, Juan Ding, Guolian Kang, Melvanique Hale, Mitchell Weiss, Ulrike Reiss, Winfred Wang, Marcin Wlodarski
Summary: This study evaluated the efficacy and safety of standard immunosuppressive therapy and combination therapy with eltrombopag in pediatric severe aplastic anemia patients. The results showed a higher objective response rate in the treatment group with eltrombopag added, and no significant differences in infections between the two therapies.
PEDIATRIC BLOOD & CANCER
(2021)
Article
Medicine, Research & Experimental
Ruixin Li, Ningling Wang, Xingxing Chai, Linhai Yang, Kangkang Liu, Hailong He, Shengyun Lin, Yan Yang, Jinsong Jia, Donghua Zhang, Yuemin Gong, Jinning Shi, Guangsheng He, Jianyong Li
Summary: Eltrombopag can enhance the effectiveness of immunosuppressive therapy in severe aplastic anemia patients. This study investigates the benefits of continuous usage of Eltrombopag beyond 6 months in these patients. The findings show that continuous administration of Eltrombopag improves hematologic response and event-free survival in patients without achieving complete remission at 6 months.
CLINICAL AND EXPERIMENTAL MEDICINE
(2023)
Article
Hematology
Olga Goronkova, Galina Novichkova, Tatiana Salimova, Irina Kalinina, Dina Baidildina, Ulyana Petrova, Kristina Antonova, Maria Sadovskaya, Elena Suntsova, Dmitry Evseev, Victor Matveev, Dmitry Venyov, Lili Khachatryan, Dmitry Litvinov, Alexey Pshonkin, Galina Ovsyannikova, Natalia Kotskaya, Darina Gobadze, Yulia Olshanskaya, Alexander Popov, Elena Raykina, Olga Mironenko, Kirill Voronin, Bazarma Purbueva, Elmira Boichenko, Yulia Dinikina, Evgeniya Guseynova, Dmitry Sherstnev, Elena Kalinina, Sergey Mezentsev, Olga Streneva, Natalia Yudina, Olga Plaksna, Elena Erega, Michael Maschan, Alexey Maschan
Summary: This study compared the efficacy and safety of eltrombopag combined with immunosuppressive therapy and immunosuppressive therapy alone in treatment-naive children with severe and very severe aplastic anemia. The results showed that adding eltrombopag to standard immunosuppressive therapy increased the complete response rate and overall response rate compared to immunosuppressive therapy alone. The greatest benefit from eltrombopag combined with immunosuppressive therapy was observed in patients with severe aplastic anemia, but not in those with very severe aplastic anemia.
Article
Immunology
Ruixin Li, Jiongtao Zhou, Zhengyuan Liu, Xi Chen, Qiqiang Long, Yan Yang, Shengyun Lin, Jinsong Jia, Guangsheng He, JianYong Li
Summary: The addition of eltrombopag to intensive immunosuppressive therapy can restore hematopoiesis in patients with severe aplastic anemia. A retrospective study found that baseline reticulocyte percentage, absolute reticulocyte count, red cell distribution width-coefficient of variation, and absolute lymphocyte count are potential predictors of treatment response at 6 months.
FRONTIERS IN IMMUNOLOGY
(2022)
Article
Pediatrics
Yufei Zhao, Wenrui Yang, Xin Zhao, Xiangrong Hu, Jing Hu, Xu Liu, Jianping Li, Lei Ye, Youzhen Xiong, Yang Yang, Baohang Zhang, Xiaoxia Li, Xiawan Yang, Yimeng Shi, Guangxin Peng, Yuan Li, Huihui Fan, Kang Zhou, Liping Jing, Li Zhang, Fengkui Zhang
Summary: Adding EPAG to standard IST as the first-line treatment for children with acquired SAA improved the rapidity of hematological response and the complete response rate but did not improve the overall response or event-free survival rates.
FRONTIERS IN PEDIATRICS
(2023)
Article
Hematology
Yuanyuan Jin, Ruixin Li, Shengyun Lin, Jinsong Jia, Yan Yang, Donghua Zhang, Guangsheng He, Jiangyong Li
Summary: The addition of low-dose EPAG to IST in Chinese patients with SAA resulted in higher response rates and complete remission rates, without significant adverse events.
ANNALS OF HEMATOLOGY
(2022)
Review
Hematology
Beatrice Drexler, Jakob Passweg
Summary: Eltrombopag as a TPO receptor agonist shows beneficial effects on expanding and maintaining HSCs in the treatment of AA. When used in combination with horse antithymocyte globulin and cyclosporine, it can significantly improve treatment response rates. However, high-dose usage may lead to adverse events and clonal evolution with chromosomal abnormalities.
THERAPEUTIC ADVANCES IN HEMATOLOGY
(2021)
Article
Hematology
Mervat A. M. Youssef, Mohammed H. Ghazaly, Mai A. Abdelfattah
Summary: This study compared the efficacy and safety of CsA monotherapy with eltrombopag (E-PAG) + CsA combined treatment in children with severe aplastic anemia (SAA). The results showed that E-PAG + CsA significantly improved hematological response and overall survival rates, which is of great clinical significance in countries with limited resources.
ANNALS OF HEMATOLOGY
(2023)
Article
Hematology
Zhuxin Zhang, Qinglin Hu, Chen Yang, Miao Chen, Bing Han
Summary: In this retrospective study, the efficacy and safety of Eltrombopag (ELT) and Avatrombopag (AVA) in relapsed/refractory aplastic anemia (AA) patients were compared. The study found that AVA was more suitable for patients with abnormal liver or renal function, and compared to ELT, AVA had a shorter time to response and fewer adverse events.
THERAPEUTIC ADVANCES IN HEMATOLOGY
(2023)
Review
Pediatrics
Maria Maddalena Marrapodi, Annamaria Mascolo, Domenico Roberti, Martina Di Martino, Concetta Rafaniello, Consiglia Riccardi, Francesca Rossi
Summary: Pediatric patients with acquired aplastic anemia (AAA) have hypocellular bone marrow and pancytopenia. Eltrombopag, an oral thrombopoietin receptor agonist, has shown hematologic improvement in adults with severe aplastic anemia (SAA) refractory to immunosuppressive therapy (IST). The use of ELT and IST as a first-line treatment for SAA in children has been approved by the FDA, but its effects remain controversial. A systematic review of recent literature was conducted to evaluate the efficacy and safety of ELT added to IST for pediatric SAA.
FRONTIERS IN PEDIATRICS
(2023)
Article
Oncology
Limin Liu, Meiqing Lei, Rong Fu, Bing Han, Xin Zhao, Rongrong Liu, Yanming Zhang, Wenjing Jiao, Miao Miao, Fengkui Zhang, Liansheng Zhang, Depei Wu
Summary: This study compared the efficacy and safety of matched related donor-hematopoietic stem cell transplantation (MRD-HSCT) with immunosuppressive therapy plus eltrombopag (IST + EPAG) for severe aplastic anemia (SAA) patients. The results showed that MRD-HSCT achieved faster transfusion independence and better outcomes, making it the preferred first-line treatment option for younger patients or those with vSAA.
JOURNAL OF HEMATOLOGY & ONCOLOGY
(2022)
Review
Chemistry, Medicinal
Marta Bortolotti, Loredana Pettine, Anna Zaninoni, Giorgio Alberto Croci, Wilma Barcellini, Bruno Fattizzo
Summary: Thrombopoietin receptor agonists (TPO-RA) are used to treat chronic immune thrombocytopenia and relapsed refractory aplastic anemia. However, their off-label use is becoming more common, especially in cases of aplasia after chemotherapy and stem cell transplant. Recent evidence suggests that TPO-RA have immunomodulating properties in addition to stimulating platelet production. In this case study, eltrombopag was effective in treating prolonged bone marrow aplasia caused by autologous stem cell transplant. The drug also had an effect on inflammatory cytokine levels and bone marrow histology. This review of literature highlights the efficacy and safety of TPO-RA after stem cell transplant and chemotherapy for hematologic conditions.
Article
Oncology
Esther Natalie Oliva, Marta Riva, Pasquale Niscola, Valeria Santini, Massimo Breccia, Valentina Giai, Antonella Poloni, Andrea Patriarca, Elena Crisa, Isabella Capodanno, Prassede Salutari, Gianluigi Reda, Nicola Cascavilla, Dario Ferrero, Attilio Guarini, Giovanni Tripepi, Giuseppe Ianni, Emilio Russo, Andrea Castelli, Bruno Fattizzo, Germana Beltrami, Monica Bocchia, Alfredo Molteni, Pierre Fenaux, Ulrich Germing, Alessandra Ricco, Giuseppe A. Palumbo, Stefana Impera, Nicola Di Renzo, Flavia Rivellini, Francesco Buccisano, Aspasia Stamatoullas-Bastard, Anna Marina Liberati, Anna Candoni, Ilaria Maria Delfino, Maria Teresa Arcadi, Patrizia Cufari, Lorenzo Rizzo, Irene Bova, Maria Grazia D'Errigo, Gina Zini, Roberto Latagliata
Summary: This study demonstrates the efficacy and safety of eltrombopag in treating low-risk MDS patients with severe thrombocytopenia. Eltrombopag significantly increases the duration of platelet response and reduces the incidence of clinically significant bleeding.
JOURNAL OF CLINICAL ONCOLOGY
(2023)
Article
Oncology
Jing Wu, Ying Yuan, Debra A. Long Priel, Danielle Fink, Cody J. Peer, Tristan M. Sissung, Yu-Ting Su, Ying Pang, Guangyang Yu, Madison K. Butler, Tito R. Mendoza, Elizabeth Vera, Salman Ahmad, Christine Bryla, Matthew Lindsley, Ewa Grajkowska, Kelly Mentges, Lisa Boris, Ramya Antony, Nancy Garren, Christine Siegel, Nicole Lollo, Christine Cordova, Orwa Aboud, Brett J. Theeler, Eric M. Burton, Marta Penas-Prado, Heather Leeper, Javier Gonzales, Terri S. Armstrong, Katherine R. Calvo, William D. Figg, Douglas B. Kuhns, John Gallin, Mark R. Gilbert
Summary: This study investigated the toxicity profile and optimal dosing schedule of zotiraciclib with temozolomide in patients with recurrent high-grade astmcytoma. The results showed that the MTD of zotiraciclib was 250 mg and it combined favorably with dose-dense temozolomide. Patients experienced worsening symptom burden during treatment but recovered quickly.
CLINICAL CANCER RESEARCH
(2021)
Article
Rheumatology
Marcela A. Ferrada, Keith A. Sikora, Yiming Luo, Kristina V. Wells, Bhavisha Patel, Emma M. Groarke, Daniela Ospina Cardona, Emily Rominger, Patrycja Hoffmann, Mimi T. Le, Zuoming Deng, Kaitlin A. Quinn, Emily Rose, Wanxia L. Tsai, Gustaf Wigerblad, Wendy Goodspeed, Anne Jones, Lorena Wilson, Oskar Schnappauf, Ryan S. Laird, Jeff Kim, Clint Allen, Arlene Sirajuddin, Marcus Chen, Massimo Gadina, Katherine R. Calvo, Mariana J. Kaplan, Robert A. Colbert, Ivona Aksentijevich, Neal S. Young, Sinisa Savic, Daniel L. Kastner, Amanda K. Ombrello, David B. Beck, Peter C. Grayson
Summary: Somatic mutations in UBA1 have been identified in a subset of patients with relapsing polychondritis (RP), leading to a newly defined syndrome called VEXAS-RP. Patients with VEXAS-RP have distinct clinical and immunologic features, including a male predominance, onset in midlife or later, specific symptoms, and higher mortality compared to typical RP patients. Early identification and intervention are important in managing VEXAS-RP due to its associated higher mortality rate.
ARTHRITIS & RHEUMATOLOGY
(2021)
Article
Hematology
Emma M. Groarke, Neal S. Young, Katherine R. Calvo
Summary: This review discusses the importance of distinguishing between constitutional and immune bone marrow failure, focusing on the evaluation methods for BMF and specific clinical presentations, as well as the interpretation of bone marrow histology, flow cytometry, and karyotyping. Specialized testing in immune and inherited BMF, as well as genetic testing, are also examined in terms of their roles in patient evaluation and result interpretation.
BEST PRACTICE & RESEARCH CLINICAL HAEMATOLOGY
(2021)
Article
Cell Biology
Weixin Wang, Rui Chen, Stephenie Droll, Emily Barber, Layla Saleh, Meghan Corrigan-Cummins, Megan Trick, Vollter Anastas, Nga Voong Hawk, Zhen Zhao, Donald C. Vinh, Amy Hsu, Dennis D. Hickstein, Steven M. Holland, Katherine R. Calvo
Summary: GATA2 deficiency leads to cytopenias and susceptibility to infections. Increased expression of miR-181c and decreased levels of MCL1 were found in GATA2 deficiency cells, possibly contributing to elevated cell death and cytopenia. GATA2 represses miR-181c expression, while knockdown of GATA2 results in increased miR-181c expression.
JOURNAL OF LEUKOCYTE BIOLOGY
(2022)
Review
Biotechnology & Applied Microbiology
Byung-Chul Lee, Richard J. Lozano, Cynthia E. Dunbar
Summary: Hematopoietic stem and progenitor cell (HSPC) gene therapies have advanced to include targeted genome modification techniques such as zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and CRISPRCas technologies. While promising, challenges related to adverse effects on HSPC function and genomic integrity remain. Targeted gene editing platforms have shown potential for therapeutic applications, but further research is needed to address these obstacles before widespread clinical use.
Article
Hematology
Robert R. West, Katherine R. Calvo, Lisa J. Embree, Weixin Wang, Laura M. Tuschong, Thomas R. Bauer, Desiree Tillo, Justin Lack, Stephenie Droll, Amy P. Hsu, Steven M. Holland, Dennis D. Hickstein
Summary: Patients with GATA2 deficiency have germline mutations in the GATA2 transcription factor gene, which predispose them to myeloid malignancies. Somatic mutations in ASXL1 and STAG2 are associated with lower survival probability, while mutations in DNMT3A are found in early stages of the disease. Chromatin-related genes and cohesin genes characterize the progression of GATA2 deficiency.
Article
Hematology
Diana X. Nichols-Vinueza, Mark Parta, Nirali N. Shah, Jennifer M. Cuella-Rodriguez, Thomas R. Bauer, Robert R. West, Amy P. Hsu, Katherine R. Calvo, Seth M. Steinberg, Luigi D. Notarangelo, Steven M. Holland, Dennis D. Hickstein
Summary: In patients with GATA2 deficiency, a busulfan-based HSCT regimen can reverse the hematologic disease phenotype, and the use of post-transplant cyclophosphamide (PT/Cy) reduces the risk of both acute and chronic GVHD.
BRITISH JOURNAL OF HAEMATOLOGY
(2022)
Article
Hematology
David J. Young, Xing Fan, Emma M. Groarke, Bhavisha Patel, Ronan Desmond, Thomas Winkler, Andre Larochelle, Katherine R. Calvo, Neal S. Young, Cynthia E. Dunbar
Summary: EPAG treatment for aplastic anemia and immune thrombocytopenia can result in iron depletion over time. This study found that duration of EPAG exposure correlated with reduction in ferritin levels and showed faster iron clearance compared to historical responders. Baseline ferritin and duration of therapy were associated with risk of iron depletion, but did not affect treatment response or relapse risk.
AMERICAN JOURNAL OF HEMATOLOGY
(2022)
Review
Pathology
Martina Rudelius, Olga K. Weinberg, Charlotte M. Niemeyer, Akiko Shimamura, Katherine R. Calvo
Summary: The classification of hematologic neoplasia is critical for diagnosis, therapy, research, and clinical trials. This review summarizes recent advances in the classification of myeloid and lymphoblastic neoplasia with germline predisposition, providing important genetic and phenotypic information, relevant laboratory testing, and pathological bone marrow features. It also focuses on the updated classification of pediatric myelodysplastic syndrome and juvenile myelomonocytic leukemia, highlighting the genetic definitions and distinguishing features.
Review
Pathology
Nisha Patel, Katherine R. Calvo
Summary: Pathologists play a crucial role in diagnosing germline predisposition to myeloid neoplasia and monitoring disease progression. The diagnostic workup can be challenging if clinical history, laboratory testing, or genetic studies are incomplete. This article illustrates common diagnostic challenges encountered during bone marrow examination of patients with potential germline predisposition to myeloid neoplasia.
AMERICAN JOURNAL OF CLINICAL PATHOLOGY
(2023)
Article
Hematology
Robert R. West, Thomas R. Bauer Jr, Laura M. Tuschong, Lisa J. Embree, Katherine R. Calvo, Desiree Tillo, Joie Davis, Steven M. Holland, Dennis D. Hickstein
Summary: GATA2 mutations are common in patients with GATA2 deficiency, but this study discovered a multigeneration family with the clinical features of GATA2 deficiency but lacking an apparent GATA2 mutation. Whole genome sequencing revealed a unique variant in the GATA2 enhancer that leads to overexpression of GATA2. The study highlights the association between GATA2 overexpression and MonoMAC syndrome.
Article
Medical Laboratory Technology
Ifeyinwa E. Obiorah, Kalpana D. Upadhyaya, Katherine R. Calvo
CLINICS IN LABORATORY MEDICINE
(2023)
Article
Medicine, Research & Experimental
David H. Mcdermott, Daniel Velez, Elena Cho, Edward W. Cowen, John J. Digiovanna, Diana V. Pastrana, Christopher B. Buck, Katherine R. Calvo, Pamela J. Gardner, Sergio D. Rosenzweig, Pamela Stratton, Melissa A. Merideth, H. Jeffrey Kim, Carmen Brewer, James D. Katz, Douglas B. Kuhns, Harry L. Malech, Dean Follmann, Michael P. Fay, Philip M. Murphy
Summary: In a phase III crossover trial, plerixafor was found to be noninferior to G-CSF in maintaining neutrophil counts and superior in maintaining lymphocyte counts in patients with WHIM syndrome. Although not superior in infection severity score, the study supports further investigation of plerixafor as a potential treatment for WHIM syndrome based on wart regression and hematologic improvement.
JOURNAL OF CLINICAL INVESTIGATION
(2023)
Article
Computer Science, Interdisciplinary Applications
Diego A. Espinoza, Ryland D. Mortlock, Samson J. Koelle, Chuanfeng Wu, Cynthia E. Dunbar
Summary: Clonal tracking methods provide insights into cellular output, but the lack of analysis tools has hindered standardized frameworks. The barcodeR package now offers diverse tools for analyzing and visualizing clonal tracking data, proving useful in exploring longitudinal patterns and lineage relationships in various studies.
NATURE COMPUTATIONAL SCIENCE
(2021)