4.8 Letter

Caplacizumab for Acquired Thrombotic Thrombocytopenic Purpura

期刊

NEW ENGLAND JOURNAL OF MEDICINE
卷 374, 期 25, 页码 2497-2497

出版社

MASSACHUSETTS MEDICAL SOC
DOI: 10.1056/NEJMc1603180

关键词

-

向作者/读者索取更多资源

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

4.8
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

Letter Hematology

Reduced FVIII recovery associated with anti-FVIII PEG antibodies after BNT162b2 SARS-CoV-2 vaccination

Carla Valsecchi, Roberta Gualtierotti, Sara Arcudi, Alessandro Ciavarella, Lucia Schiavone, Cristina Novembrino, Simona Maria Siboni, Pier Mannuccio Mannucci, Flora Peyvandi

BLOOD ADVANCES (2023)

Article Hematology

Recombinant factor VIII Fc fusion protein for first-time immune tolerance induction: final results of the verITI-8 study

Lynn Malec, An Van Damme, Anthony K. C. Chan, Mariya Spasova, Nisha Jain, Charlotte Sensinger, Jennifer Dumont, Stefan Lethagen, Manuel Carcao, Flora Peyvandi

Summary: Inhibitor development remains a major challenge in factor VIII replacement therapy. The verITI-8 study evaluated the use of a recombinant FVIII Fc fusion protein (rFVIIIFc) for immune tolerance induction in males with severe hemophilia A and high-titer inhibitors. The study showed that rFVIIIFc achieved tolerization in a significant number of patients within a short time period, and adverse events were generally well tolerated.
Editorial Material Gastroenterology & Hepatology

Liver-related aspects of gene therapy for haemophilia: Call to action for collaboration between haematologists and hepatologists

Wolfgang Miesbach, Graham R. Foster, Flora Peyvandi

JOURNAL OF HEPATOLOGY (2023)

Review Hematology

Prevalence of selected bleeding and thrombotic events in persons with hemophilia versus the general population: A scoping review

Amy D. Shapiro, Brandon M. Hardesty, Flora Peyvandi, Alfonso Iorio

Summary: Life expectancy for persons with hemophilia has increased due to advances in treatment, but they are now more likely to be affected by conditions associated with aging. This study analyzed the prevalence of bleeding and thrombotic events in hemophilia populations compared to the general population. The results showed consistently higher rates of bleeding events in hemophilia populations, while the prevalence of arterial thrombosis varied.

RESEARCH AND PRACTICE IN THROMBOSIS AND HAEMOSTASIS (2023)

Review Hematology

International Council for Standardization in Haematology recommendations for laboratory measurement of factor VIII and FIX type I inhibitors

Piet Meijer, Flora Peyvandi, Guy Young, Rajiv Pruthi, Silmara de Lima Montalvao, Steve Kitchen

Summary: This guidance document provides recommendations on the measurement of factor VIII (FVIII) and factor IX (FIX) inhibitors, including screening, assay principle, sample and testing requirements, interpretation, quality assurance, interferences, and recent developments. It focuses on standardizing the laboratory measurement of FVIII and FIX type I inhibitors based on published data and expert opinion.

INTERNATIONAL JOURNAL OF LABORATORY HEMATOLOGY (2023)

Article Medicine, General & Internal

Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A

J. Mahlangu, R. Kaczmarek, A. von Drygalski, S. Shapiro, S. -C. Chou, M. C. Ozelo, G. Kenet, F. Peyvandi, M. Wang, B. Madan, N. S. Key, M. Laffan, A. L. Dunn, J. Mason, D. V. Quon, E. Symington, A. D. Leavitt, J. Oldenburg, H. Chambost, M. T. Reding, K. Jayaram, H. Yu, R. Mahajan, K. -M. Chavele, D. B. Reddy, J. Henshaw, T. M. Robinson, W. Y. Wong, S. W. Pipe

Summary: This study demonstrates the long-term effectiveness and safety of valoctocogene roxaparvovec therapy in reducing bleeding events in men with severe hemophilia A. The pharmacokinetic modeling suggests a half-life of 123 weeks for the transgene-derived factor VIII production system. Models also indicate a similar relationship between factor VIII activity and bleeding episodes as observed in individuals with mild-to-moderate hemophilia A using epidemiologic data.

NEW ENGLAND JOURNAL OF MEDICINE (2023)

Editorial Material Medicine, General & Internal

Mild and Moderate Hemophilia A: Neglected Conditions, Still with Unmet Needs

Giancarlo Castaman, Flora Peyvandi, Raimondo De Cristofaro, Berardino Pollio, Dario M. N. Di Minno

JOURNAL OF CLINICAL MEDICINE (2023)

Article Medicine, General & Internal

Nailfold Videocapillaroscopic Alterations as Markers of Microangiopathy in COVID-19 Patients

Roberta E. Gualtierotti, Sharon Fox, Fernanda Da Silva Lameira, Andrea Giachi, Luca Valenti, Maria Orietta Borghi, Pier Luigi Meroni, Massimo Cugno, Flora Peyvandi

Summary: Nailfold videocapillaroscopic alterations were observed in COVID-19 patients, and these alterations were correlated with biomarkers of inflammation and endothelial perturbation. Nailfold histopathology analysis revealed microvascular damage and microthrombi in some cases. These findings suggest the possibility of non-invasively demonstrating microangiopathy in COVID-19.

JOURNAL OF CLINICAL MEDICINE (2023)

Review Biochemistry & Molecular Biology

Laboratory Diagnosis of Antiphospholipid Syndrome in Anticoagulated Patients

Armando Tripodi, Erica Scalambrino, Marigrazia Clerici, Flora Peyvandi

Summary: The laboratory diagnosis of APS requires the measurement of solid-phase antibodies to cardiolipin or β2-Glycoprotein-I and the search for LA. However, the interpretation of results, especially for LA, is difficult in patients on anticoagulation due to the overlap of clotting time prolongations induced by LA and anticoagulants. This article reviews the effect of anticoagulants on APS diagnosis and discusses potential solutions to overcome this issue.

BIOMEDICINES (2023)

Article Hematology

A comparative study in patients with type 2 von Willebrand disease using 4 different platelet-dependent von Willebrand factor assays

Paola Colpani, Luciano Baronciani, Francesca Stufano, Giovanna Cozzi, Marco Boscarino, Maria Teresa Pagliari, Eugenia Biguzzi, Flora Peyvandi

Summary: This study evaluated four different assays to diagnose and detect various von Willebrand disease (VWD) variants. The VWF:GPIbM-automated assay was the most effective in diagnosing type 2B, while the VWF:RCo assays were the most effective in detecting type 2M and 2M/2A variants. The VWF:GPIbM ELISA overestimated the activity of type 2B patients lacking high-molecular-weight multimers (HMWM). Using a VWF activity/VWF:antigen ratio cut-off of 0.70 reduced the number of misdiagnosed patients by half.

RESEARCH AND PRACTICE IN THROMBOSIS AND HAEMOSTASIS (2023)

Article Hematology

Accreditation model of European Haemophilia Centres in the era of novel treatments and gene therapy

Ana Boban, Fariba Baghaei, Fijnvandraat Karin, Robert Klamroth, Wolfgang Miesbach, David Stephensen, Mary Kavanagh, Declan Noone, Miguel Crato, Flora Peyvandi

Summary: This article introduces the background and purpose of international certification of haemophilia centres in Europe, as well as the methods and results of establishing a new accreditation protocol.

HAEMOPHILIA (2023)

Article Medicine, General & Internal

Characterization of Immunogenicity and Safety of COVID-19 mRNA-1273 in HIV-Positive Italian Patients with Hemophilia: A Prospective Single-Center Cohort Study

Chiara Suffritti, Roberta Gualtierotti, Sara Arcudi, Alessandro Ciavarella, Cristina Novembrino, Anna Lecchi, Silvia La Marca, Lidia Padovan, Erica Scalambrino, Marigrazia Clerici, Patrizia Bono, Ferruccio Ceriotti, Antonio Muscatello, Simona Maria Siboni, Flora Peyvandi

Summary: This study investigated the immunogenicity of the mRNA-1273 vaccine in HIV-positive hemophilic patients during the third COVID-19 wave in Italy and examined biomarkers of coagulation and endothelial perturbation after complete vaccination. The results showed that all patients had effective immunization with anti-spike antibodies two weeks after completing the vaccination schedule. Furthermore, the vaccination had no significant effects on coagulation and endothelial perturbation.

JOURNAL OF CLINICAL MEDICINE (2023)

Article Genetics & Heredity

Population-based prevalence and mutational landscape of von Willebrand disease using large-scale genetic databases

Omid Seidizadeh, Andrea Cairo, Luciano Baronciani, Luca Valenti, Flora Peyvandi

Summary: In this study, the global prevalence of Von Willebrand disease (VWD) was estimated by analyzing exome and genome data from a large number of individuals. The results showed a higher than expected prevalence of potential disease alleles and variants associated with VWD.

NPJ GENOMIC MEDICINE (2023)

Article Hematology

Minimum factor VIII levels to prevent joint bleeding in mild hemophilia A

Pasquale Agosti, Simona Maria Siboni, Sara Scardo, Adriana Torri, Roberta Gualtierotti, Flora Peyvandi

Summary: This study aimed to identify the minimum FVIII levels necessary to prevent joint bleeds in patients with hemophilia A. The findings suggest important implications for the design of prophylactic therapies for moderate and severe hemophilia A.

BLOOD ADVANCES (2023)

Letter Hematology

Comment on: A comparative study in type 2 von Willebrand disease patients using four different platelet-dependent von Willebrand factor assays

Paola Colpani, Luciano Baronciani, Giovanna Cozzi, Maria Teresa Pagliari, Eugenia Biguzzi, Flora Peyvandi

RESEARCH AND PRACTICE IN THROMBOSIS AND HAEMOSTASIS (2023)

暂无数据