☆ 4.4 Letter

Use of caplacizumab in a child with refractory thrombotic thrombocytopenic purpura

PEDIATRIC BLOOD & CANCER (2019)

期刊

PEDIATRIC BLOOD & CANCER

卷 66, 期 7, 页码 -

出版社

WILEY

DOI: 10.1002/pbc.27737

关键词

ADAMTS13; caplacizumab; refractory TTP; thrombocytopenia; TTP; vWF

类别

Oncology Hematology Pediatrics

向作者/读者索取更多资源

Protocol

Reagent

作者

我是这篇论文的作者

点击您的名字以认领此论文并将其添加到您的个人资料中。

评论

主要评分

4.4

评分不足

次要评分

新颖性

-

重要性

-

科学严谨性

-

评价这篇论文

推荐

Article Medicine, General & Internal

An Interesting Case of Refractory Thrombotic Thrombocytopenic Purpura in the First Trimester of a Twin Pregnancy

Giuseppina Jacob, Amanpreet Dhaliwal, Vijay Chaudhary

Summary: This case report highlights the importance of prompt identification and appropriate management of TTP in early stages of pregnancy. For patients with refractory TTP (rTTP), including plasmapheresis, caplacizumab, and rituximab, are necessary treatment strategies.

CUREUS JOURNAL OF MEDICAL SCIENCE (2023)

添加到收藏夹

Review Medicine, General & Internal

Thrombotic Thrombocytopenic Purpura: Pathophysiology, Diagnosis, and Management

Senthil Sukumar, Bernhard Lammle, Spero R. Cataland

Summary: TTP is a rare but dangerous disorder characterized by hemolysis, thrombocytopenia, and ischemic damage, often caused by ADAMTS13 deficiency. Front-line treatment typically involves plasma exchange and immunosuppression, as well as drugs like rituximab and caplacizumab. Refractory cases may require alternative therapies.

JOURNAL OF CLINICAL MEDICINE (2021)

添加到收藏夹

Article Hematology

Caplacizumab as rescue therapy in refractory TTP involving neurologic features

Minh -Ha Tran, Lisa X. Lee, Yen Cao, Lan Vu, Zahra Pakbaz

Summary: The role of caplacizumab in the routine treatment of immune thrombotic thrombocytopenic purpura (iTTP) remains to be established. A 56-year-old woman was initially misdiagnosed and managed as Immune Thrombocytopenia (ITP), but upon transfer to our center, proper treatment with caplacizumab led to rapid improvement in hematologic and clinical responses of refractory iTTP with neurologic features.

TRANSFUSION AND APHERESIS SCIENCE (2023)

添加到收藏夹

Article Hematology

Refractory immune TTP following Pfizer-BioNTech COVID-19 vaccine successfully salvaged with caplacizumab

Eva Laverdure, Catherine Sperlich, Susan Fox

Summary: This case report describes a young healthy female who developed immune thrombotic thrombocytopenic purpura (iTTP) after receiving the Pfizer Bio-NTech COVID-19 vaccine. Despite initial response to standard treatment, she experienced acute deterioration with neurological and cardiac involvement. However, she had a prompt response to rituximab and caplacizumab and achieved remission.

JOURNAL OF THROMBOSIS AND HAEMOSTASIS (2022)

添加到收藏夹

Article Immunology

Molecular Diagnosis Is Vital to the Accurate Classification and Management of Thrombotic Thrombocytopenic Purpura in Children

Cecile L. Karsenty, Susan E. Kirk, Hannah L. Helber, Jose M. Esquilin, Jenny M. Despotovic, Amanda B. Grimes

Summary: This article reviews thrombotic thrombocytopenic purpura (TTP) and discusses two cases of TTP in adolescents, emphasizing the need to consider late-onset congenital TTP (cTTP). The importance of early confirmation of ADAMTS13 enzyme deficiency and the presence or absence of ADAMTS13 inhibitor is demonstrated in order to initiate appropriate life-saving therapies. Ultimately, molecular testing is crucial for distinguishing between congenital and acquired immune-mediated TTP.

FRONTIERS IN IMMUNOLOGY (2022)

添加到收藏夹

Article Hematology

Daratumumab for immune thrombotic thrombocytopenic purpura

Jana van den Berg, Johanna A. Kremer Hovinga, Claudia Pfleger, Inga Hegemann, Gregor Stehle, Andreas Holbro, Jan-Dirk Studt

Summary: Immune thrombotic thrombocytopenic purpura (iTTP) is a life-threatening disease that can be treated with the anti-CD38 antibody daratumumab to eradicate persistent ADAMTS13 inhibitory autoantibodies and restore normal ADAMTS13 activity.

BLOOD ADVANCES (2022)

添加到收藏夹

Article Hematology

Refractory acquired thrombotic thrombocytopenic purpura in a patient with sickle cell trait successfully treated with caplacizumab

Vibhuti Aggarwal, Zachary Singer, Donna Ledingham, Ibraheem Othman

Summary: The case study illustrates a rare occurrence of acquired TTP and sickle cell trait in a 20-year-old Nigerian male, who achieved successful remission with the use of caplacizumab after relapsing despite initial treatment methods. This case contributes to the literature by highlighting the efficacy of caplacizumab in treating aTTP in patients with sickle cell hemoglobinopathies.

HEMATOLOGY (2021)

添加到收藏夹

Article Hematology

Thrombotic Thrombocytopenic Purpura: From 1972 to 2022 and Beyond

James N. George

Summary: The article narrates the author's personal experience with TTP, tracing the history and development of treatment methods. Through collaborative research and patient data, a deeper understanding of TTP diagnosis and treatment, as well as the differences between hTTP and iTTP, was gained.

SEMINARS IN THROMBOSIS AND HEMOSTASIS (2022)

添加到收藏夹

Article Cell Biology

Novel antiplatelet strategies targeting VWF and GPIb

Nithya Prasannan, Marie Scully

Summary: Von Willebrand factor plays a crucial role in primary hemostasis, and its involvement in TMA and cardiovascular diseases, especially TTP, has been demonstrated. Current management strategies for TTP include plasma exchange and immunosuppressive therapy. Caplacizumab, a novel antiplatelet strategy targeting VWF and GPIb, has shown benefits in normalizing platelet count quickly and reducing thromboembolic events, particularly in iTTP patients.

PLATELETS (2021)

添加到收藏夹

Review Hematology

ADAMTS13 conformations and mechanism of inhibition in immune thrombotic thrombocytopenic purpura

Konstantine Halkidis, X. Long Zheng

Summary: ADAMTS13 is a crucial plasma enzyme for normal hemostasis, and deficiency caused by acquired autoantibodies can lead to a potentially fatal blood disorder. Recent research has shown that ADAMTS13 exists in multiple conformations and can be regulated by antibodies in different ways.

JOURNAL OF THROMBOSIS AND HAEMOSTASIS (2022)

添加到收藏夹

Article Hematology

A multicenter laboratory assessment of a new automated chemiluminescent assay for ADAMTS13 activity

Emmanuel J. Favaloro, Soma Mohammed, Kent Chapman, Priscilla Swanepoel, Diane Zebeljan, Opelo Sefhore, Erica Malan, Joanne Clifford, Agnes Yuen, Dea Donikian, Mayuko Kondo, Elizabeth Duncan, Sunil Abraham, Joanne Beggs, Ritesh Chatrapati, Joanne Perel, Robyn Coleman, Nathan Klose, Danny Hsu, Penelope Motum, Chee Wee Tan, Timothy Brighton, Leonardo Pasalic

Summary: The study evaluated the utility of a new automated test for ADAMTS13 activity, which showed similar results to the comparator ELISA with slight bias. Detection of ADAMTS13 inhibitors was also comparable with slight bias. The HemosIL AcuStar ADAMTS13 Activity assay provides a fast and accurate means for quantitating plasma levels of ADAMTS13, aiding in the identification/exclusion of TTP/ADAMTS13 and potentially other applications.

JOURNAL OF THROMBOSIS AND HAEMOSTASIS (2021)

添加到收藏夹

Article Medicine, General & Internal

Silent Thrombotic Thrombocytopenic Purpura: PLASMIC, Lessons Learned, and Current Management Overview

Alexandra Pisklakova, Joshua Barbir, Jan-Paul Sambataro, Christian Almanzar, Faiza Manji

Summary: Thrombotic thrombocytopenic purpura (TTP) is a rare and life-threatening disease that can be difficult to diagnose due to its wide spectrum of symptoms. Clinical diagnosis is based on various symptoms and treatment depends on clinical suspicion and diagnostic test results (such as ADAMTS13 levels). The recently developed PLASMIC score can help differentiate TMA patients and serve as an adjunct in diagnosing TTP when clinical presentation is unclear.

CUREUS JOURNAL OF MEDICAL SCIENCE (2021)

添加到收藏夹

Article Medicine, General & Internal

Refractory thrombotic thrombocytopenic purpura

K. Herfurth, J. Ruhe, K. Kentouche, A. Guenther, D. Braemer, N. Eckardt, M. Busch, G. Wolf

Summary: This article presents a case of serious relapse of TTP, despite adequately initiated therapy. It emphasizes the necessity of interdisciplinary expertise in the treatment of the disease.

INNERE MEDIZIN (2022)

添加到收藏夹

Review Medical Laboratory Technology

Laboratory investigation and diagnosis of thrombotic thrombocytopenic purpura

Konstantinos Dimopoulos, Armando Tripodi, Jens P. Goetze

Summary: Thrombotic thrombocytopenic purpura (TTP) is a rare and potentially fatal disease. Rapid diagnosis is crucial for patient outcomes, and measurement of ADAMTS13 enzyme activity can establish the diagnosis of TTP with high accuracy. Besides clinical history, appropriate laboratory assessment is essential for diagnosis and treatment of suspected TTP cases.

CRITICAL REVIEWS IN CLINICAL LABORATORY SCIENCES (2023)

添加到收藏夹

Article Hematology

Microlyse: a thrombolytic agent that targets VWF for clearance of microvascular thrombosis

Steven de Maat, Chantal C. Clark, Arjan D. Barendrecht, Simone Smits, Nadine D. van Kleef, Hinde El Otmani, Manon Waning, Marc van Moorsel, Michael Szardenings, Nicolas Delaroque, Kristof Vercruysse, Rolf T. Urbanus, Silvie Sebastian, Peter J. Lenting, Christoph Hagemeyer, Thomas Renne, Karen Vanhoorelbeke, Claudia Tersteeg, Coen Maas

Summary: Thrombotic microangiopathies are characterized by attacks of disseminated microvascular thrombosis. This study developed a fusion protein called Microlyse that can target and activate plasminogen on microthrombi, showing greater efficacy for the treatment of TTP compared to current methods.

BLOOD (2022)

添加到收藏夹

Editorial Material Pediatrics

Haemophagocytic lymphohistiocytosis restricted to the central nervous system

Senthil Velan Bhoopalan, Patrick K. Campbell, Asim K. Bag, Mihaela Onciu, Ashok Srinivasan

ARCHIVES OF DISEASE IN CHILDHOOD (2021)

添加到收藏夹

Article Oncology

Pharmacokinetics of alemtuzumab in pediatric patients undergoing ex vivo T-cell-depleted haploidentical hematopoietic cell transplantation

Senthil Velan Bhoopalan, Shane J. Cross, John C. Panetta, Brandon M. Triplett

CANCER CHEMOTHERAPY AND PHARMACOLOGY (2020)

添加到收藏夹

Article Biochemistry & Molecular Biology

Integrative proteomics reveals principles of dynamic phosphosignaling networks in human erythropoiesis

Oezge Karayel, Peng Xu, Isabell Bludau, Senthil Velan Bhoopalan, Yu Yao, Freitas Colaco Ana Rita, Alberto Santos, Brenda A. Schulman, Arno F. Alpi, Mitchell J. Weiss, Matthias Mann

MOLECULAR SYSTEMS BIOLOGY (2020)

添加到收藏夹

Article Pediatrics

Dysphagia and Epigastric Pain in an Adolescent Boy

Senthil Velan Bhoopalan, Rabea Alhosh

PEDIATRICS IN REVIEW (2021)

添加到收藏夹

Review Oncology

Reduced-intensity conditioning-based hematopoietic cell transplantation for dyskeratosis congenita: Single-center experience and literature review

Senthil Velan Bhoopalan, Marcin Wlodarski, Ulrike Reiss, Brandon Triplett, Akshay Sharma

Summary: Bone marrow failure in dyskeratosis congenita (DKC) is progressive, with allogeneic hematopoietic cell transplantation (HCT) being the only curative treatment. A reduced-intensity conditioning (RIC) regimen using fludarabine has shown to be feasible and safe for DKC patients undergoing HCT, without accelerating pulmonary damage in the short-to-medium term.

PEDIATRIC BLOOD & CANCER (2021)

添加到收藏夹

Meeting Abstract Hematology

A Novel RPS19-Edited Hematopoietic Stem Cell Model of Diamond-Blackfan Anemia for Development of Lentiviral Vector Gene Therapy

Senthil Velan Bhoopalan, Jonathan Yen, Thiyagaraj Mayuranathan, Yu Yao, Kalin Mayberry, Sheng Zhou, Robert Throm, Marcin W. Wlodarski, Mitchell J. Weiss

BLOOD (2021)

添加到收藏夹

Article Cell & Tissue Engineering

Editing human hematopoietic stem cells: advances and challenges

Senthil Velan Bhoopalan, Jonathan S. Yen, Rachel M. Levine, Akshay Sharma

Summary: Genome editing technologies, specifically CRISPR-Cas9, have made significant progress in precise genome alterations for the treatment of inherited hematopoietic disorders. Despite ongoing clinical trials and promising pre-clinical models, challenges still remain in ensuring safety, accessibility, and improving the manufacturing processes of these therapies. This review highlights recent advances and ongoing challenges in genome editing of hematopoietic stem and progenitor cells.

CYTOTHERAPY (2023)

添加到收藏夹

Article Medicine, Research & Experimental

An RPS19-edited model for Diamond-Blackfan anemia reveals TP53-dependent impairment of hematopoietic stem cell activity

Senthil Velan Bhoopalan, Jonathan S. Yen, Thiyagaraj Mayuranathan, Kalin D. Mayberry, Yu Yao, Maria Angeles Lillo Osuna, Yoonjeong Jang, Janaka S. S. Liyanage, Lionel Blanc, Steven R. Ellis, Marcin W. Wlodarski, Mitchell J. Weiss

Summary: Diamond-Blackfan anemia (DBA) is a genetic blood disease caused by heterozygous loss-of-function mutations in ribosomal protein genes, resulting in hypoplastic anemia and later multilineage cytopenias. A tractable experimental model of DBA was created through genome editing of human HSPCs, revealing associated hematopoietic stem cell defects in the disease.

JCI INSIGHT (2023)

添加到收藏夹

Article Cell & Tissue Engineering

International Society for Cell & Gene Therapy Stem Cell Engineering Committee: Cellular therapies for the treatment of graft-versus-host- disease after hematopoietic stem cell transplant

Moises Garcia-Rosa, Allistair Abraham, Alice Bertaina, Senthil Velan Bhoopalan, Carmem Bonfim, Sandra Cohen, Amy DeZern, Chrystal Louis, Joseph Oved, Mara Pavel-Dinu, Duncan Purtill, Annalisa Ruggeri, Athena Russell, Akshay Sharma, Robert Wynn, Jaap Jan Boelens, Susan Prockop

Summary: Allogeneic hematopoietic stem cell transplant is an effective treatment for various blood diseases, but graft-versus-host disease (GVHD) is still a major concern. Current pharmacologic agents used to prevent and treat GVHD also affect the graft-versus-leukemia effect. Cellular therapies such as mesenchymal stromal cells and regulatory T cells show potential in preventing or treating GVHD. Clinical studies are ongoing to explore the role of these therapies in improving GVHD-related outcomes.

CYTOTHERAPY (2023)

添加到收藏夹

Article Cardiac & Cardiovascular Systems

Novel Score to Predict Immunoglobulin Resistance in Kawasaki Disease

Rupesh Kumar Natarajan, Senthil Velan Bhoopalan, Chad Cross, Rita Shah, Abraham Rothman

Summary: This retrospective cohort study evaluated existing scoring systems and developed a new model to predict IVIG resistance in patients with Kawasaki disease (KD). Among the 115 patients included in the study, 30.4% showed resistance to IVIG. The new scoring system (LVSS) demonstrated higher specificity and comparable sensitivity to other existing scoring systems.

PEDIATRIC CARDIOLOGY (2023)

添加到收藏夹

Article Engineering, Biomedical

Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice

Kelcee A. Everette, Gregory A. Newby, Rachel M. Levine, Kalin Mayberry, Yoonjeong Jang, Thiyagaraj Mayuranathan, Nikitha Nimmagadda, Erin Dempsey, Yichao Li, Senthil Velan Bhoopalan, Xiong Liu, Jessie R. Davis, Andrew T. Nelson, Peter J. Chen, Alexander A. Sousa, Yong Cheng, John F. Tisdale, Mitchell J. Weiss, Jonathan S. Yen, David R. Liu

Summary: Prime editing efficiently corrects the sickle-cell allele in patient haematopoietic stem cells, resulting in erythrocytes resistant to hypoxia-induced sickling. This correction was achieved at frequencies of 15%-41% in patients with sickle-cell disease. After transplantation into mice, the prime-edited cells maintained normal gene expression and displayed similar differentiation and maturation as unedited stem cells from healthy donors.

NATURE BIOMEDICAL ENGINEERING (2023)

添加到收藏夹

Article Genetics & Heredity

Potent and uniform fetal hemoglobin induction via base editing

Thiyagaraj Mayuranathan, Gregory A. Newby, Ruopeng Feng, Yu Yao, Kalin D. Mayberry, Cicera R. Lazzarotto, Yichao Li, Rachel M. Levine, Nikitha Nimmagadda, Erin Dempsey, Guolian Kang, Shaina N. Porter, Phillip A. Doerfler, Jingjing Zhang, Yoonjeong Jang, Jingjing Chen, Henry W. Bell, Merlin Crossley, Senthil Velan Bhoopalan, Akshay Sharma, John F. Tisdale, Shondra M. Pruett-Miller, Yong Cheng, Shengdar Q. Tsai, David R. Liu, Mitchell J. Weiss, Jonathan S. Yen

Summary: A comparison of gene editing strategies using sickle cell disease cells and in vivo transplantation reveals that adenine base editing of the -175A>G site in the γ-globin gene promoters yields durable and potent expression. This approach provides insights into γ-globin gene regulation and offers a potential strategy for inducing HbF. The study also highlights the unexpected phenotypic variation caused by Cas9-generated diverse indels, which can be avoided by base editing.

NATURE GENETICS (2023)

添加到收藏夹

Meeting Abstract Hematology

Pharmacokinetics of Alemtuzumab in Pediatric Patients Undergoing T-Cell Depleted Haploidentical Hematopoietic Cell Transplantation (HCT)

Senthil Velan Bhoopalan, Shane Cross, John Carl Panetta, Brandon M. Triplett

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION (2020)

添加到收藏夹

Meeting Abstract Oncology

ANEMIA AND RESPIRATORY DISTRESS IN A TWO-YEAR-OLD BOY

Senthil Velan Bhoopalan, Akshay Sharma

PEDIATRIC BLOOD & CANCER (2019)

添加到收藏夹

暂无数据

© Peeref 2019-2024. All rights reserved.