Review
Hematology
Mikhail V. Ovanesov, Joseph W. Jackson, Basil Golding, Timothy K. Lee
Summary: This article discusses the factors to consider when choosing an assay for potency assignment and postadministration monitoring of new factor products, including the validity of the assay calibrated with the IS, the meaning of potency values in IU, standards of care for patients, clinical relevance between the assigned potency value and recovery value from clinical laboratories, and patient safety.
JOURNAL OF THROMBOSIS AND HAEMOSTASIS
(2021)
Article
Immunology
Glaivy Batsuli, Jasmine Ito, Elizabeth S. York, Courtney Cox, Wallace Baldwin, Surinder Gill, Pete Lollar, Shannon L. Meeks
Summary: This study analyzed the internalization of FVIII complexed with epitope-mapped FVIII-specific IgG monoclonal antibodies by murine bone marrow-derived dendritic cells (BMDCs) in vitro, as well as the antibody development in hemophilia A (FVIII-/-) mice injected with FVIII-IC over time. The results showed that certain FVIII-IC subsets modulate the humoral response to FVIII in an epitope-dependent manner.
FRONTIERS IN IMMUNOLOGY
(2023)
Review
Medicine, General & Internal
Valeryia Pratasava, Vikram N. Sahni, Aishwarya Suresh, Simo Huang, Abhirup Are, Sylvia Hsu, Kiran Motaparthi
Summary: The pemphigoid family of dermatoses is characterized by autoimmune subepidermal blistering, with the most common member being bullous pemphigoid, which typically presents with significant pruritus in the elderly. Mucous membrane pemphigoid is a group of subepidermal blistering dermatoses that favor mucosal membranes and may lead to scarring. Epidermolysis bullosa acquisita is a chronic blistering disorder with skin fragility, sensitivity to trauma, and resistance to treatment. Clinicians may benefit from an overview of the clinical presentation, diagnostic work-up, and therapeutic management of these pemphigoid disorders, with a focus on bullous pemphigoid.
MEDICINA-LITHUANIA
(2021)
Article
Hematology
Anne-Fleur Zwagemaker, Fabienne R. Kloosterman, Samantha C. Gouw, Sara Boyce, Paul Brons, Marjon H. Cnossen, Peter W. Collins, Jeroen Eikenboom, Charles Hay, Rutger C. C. Hengeveld, Shannon Jackson, Caroline A. M. Klopper-Tol, Marieke J. H. A. Kruip, Britta Laros-van Gorkom, Christoph Male, Laurens Nieuwenhuizen, Susan Shapiro, Karin Fijnvandraat, Michiel Copens
Summary: This study investigated the discrepancy between one-stage and chromogenic assays of coagulation factor activity in moderate and mild hemophilia A and B patients. The results showed that only a small number of patients exhibited a discrepancy between the two assays, and this discrepancy was largely determined by laboratory variables.
JOURNAL OF THROMBOSIS AND HAEMOSTASIS
(2023)
Article
Hematology
Viola J. F. Strijbis, Lorenzo G. R. Romano, Ka Lei Cheung, Jeroen Eikenboom, Ying Poi Liu, Andrew C. McCreary, Frank W. G. Leebeek, Mettine H. A. Bos
Summary: This study aimed to evaluate the efficacy of FIX-FIAV in plasma from HA patients. The results showed that FIX-FIAV could increase the FVIII-equivalent activity and coagulation activity in HA patient plasma, mitigating the HA phenotype. Therefore, FIX-FIAV could serve as a potential treatment for HA patients.
JOURNAL OF THROMBOSIS AND HAEMOSTASIS
(2023)
Article
Hematology
Nasim Shahidi Hamedani, Anouk Anna Marie Therese Donners, Matthijs van Luin, Simone Gasper, Heiko Ruehl, Claudia Klein, Thilo Albert, Mohsin EL Amrani, Bernd Poetzsch, Johannes Oldenburg, Jens Mueller
Summary: This study evaluated the impact of plasma FVIII activity on determining emicizumab levels and proposed different strategies for correcting or preanalytical inhibition of FVIII. The results showed that inhibitor-based FVIII neutralization is a feasible strategy for accurately measuring plasma emicizumab levels in the presence of FVIII activity.
JOURNAL OF THROMBOSIS AND HAEMOSTASIS
(2023)
Article
Hematology
Jacob Lund, Kasper Jensen, Laurent Burnier, Mirella Ezban
Summary: This study aimed to investigate the response of Mim8 in thrombin generation assays and its combination with other hemostatic proteins. The results showed that Mim8 has a similar mode of action with FVIII and the concomitant use of FVIII and Mim8 for bleed treatment is safe.
JOURNAL OF THROMBOSIS AND HAEMOSTASIS
(2023)
Article
Hematology
Laura H. Bukkems, Tim Preijers, Max W. F. van Spengler, Frank W. G. Leebeek, Marjon H. Cnossen, Ron A. A. Mathot
Summary: This study used in silico simulations to analyze the pharmacokinetic differences of various extended half-life factor VIII concentrates, with BAY 94-9027 showing the largest increase in AUC and best target attainment compared to standard half-life factor VIII.
THROMBOSIS AND HAEMOSTASIS
(2021)
Article
Biochemistry & Molecular Biology
Marcela Montilla, Isabel Atienza-Navarro, Francisco Jose Garcia-Cozar, Carmen Castro, Francisco Javier Rodriguez-Martorell, Felix A. Ruiz
Summary: Polyphosphate (polyP) plays an important modulatory role in the binding of von Willebrand factor (VWF) and Factor VIII (FVIII) in blood coagulation, and may be used in developing new therapies to activate VWF.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2022)
Article
Hematology
Melanie Demers, Maria M. Aleman, Elena Kistanova, Robert Peters, Joe Salas, Ekta Seth Chhabra
Summary: Efanesoctocog alfa and rFVIII have similar efficacy in promoting clot formation and injury-induced platelet accumulation.
JOURNAL OF THROMBOSIS AND HAEMOSTASIS
(2022)
Review
Hematology
Kenneth C. Childers, Shaun C. Peters, Paul Clint Spiegel
Summary: Advances in structural studies of blood coagulation factor VIII (FVIII) have provided important insights into the biochemical properties of FVIII, particularly the mutations associated with hemophilia A. By analyzing the atomic details of FVIII structures, researchers can design recombinant FVIII with improved circulatory half-life. Recent structural studies of FVIII bound to inhibitory antibodies have also enhanced our understanding of FVIII binding to activated platelet membranes and the formation of the intrinsic tenase complex.
JOURNAL OF THROMBOSIS AND HAEMOSTASIS
(2022)
Article
Biochemistry & Molecular Biology
Junzheng Wu, Hang Zhang, Tong Lian, Yaling Ding, Chunlei Song, Dekuan Li, Liheng Wu, Tao Lei, Hong Liang
Summary: A novel B-domain-deleted recombinant FVIII was developed in this study without the use of animal or human serum-derived components. rFVIII promoted the generation of activated factor X and downstream thrombin, and exhibited ideal binding affinity to human von Willebrand factor. In vitro and in vivo experiments demonstrated the satisfactory efficacy and potency of rFVIII, suggesting its potential as an effective treatment strategy for FVIII deficiency.
BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS
(2023)
Article
Biology
Jeong Pil Han, Dong Woo Song, Jeong Hyeon Lee, Geon Seong Lee, Su Cheong Yeom
Summary: Innovative gene therapy is advancing towards functional restoration, with hemophilia being a representative genetic disorder. A novel mouse model of hemophilia A has been developed in this study, which could be utilized for future gene correction preclinical trials.
Article
Cardiac & Cardiovascular Systems
Brandon Tse, Rosane Nisenbaum, Georgina Floros, Aziz Jiwajee, Jerome Teitel, Michelle Sholzberg
Summary: Continuous factor VIII (FVIII) or factor IX (FIX) infusions are commonly used for patients with hemophilia A (HA) or B (HB) undergoing surgery. This study found differences in initial recovery and subsequent FIX and FVIII levels, as well as clinical outcomes, among HB and HA patients receiving perioperative continuous infusion (CI) of recombinant FVIII and FIX concentrates. HB patients had higher initial bolus dose and longer length of hospital stay, suggesting a need for a higher CI rate for adequate surgical hemostatic coverage.
JOURNAL OF THROMBOSIS AND THROMBOLYSIS
(2023)
Article
Biology
Haim Cohen, Anat Keren-Politansky, Yonatan Crispel, Chen Yanovich, Keren Asayag, Yona Nadir
Summary: In this study, it was found that calcium can prevent the degradation of factor VIII during intermittent motion. Furthermore, supplementing calcium in the drinking water of mice increased factor VIII levels in the blood and striated muscle. The clinical significance of this study is that oral calcium may be considered as a potential supportive therapy for hemophilia patients.
Letter
Dermatology
Ellie Choi, Nisha S. Chandran
INTERNATIONAL JOURNAL OF DERMATOLOGY
(2019)
Article
Dermatology
Je Yin Law, Ellie C. E. Choi, Nisha Suyien Chandran
PEDIATRIC DERMATOLOGY
(2020)
Letter
Dermatology
Li Wei Heng, Nisha S. Chandran
INTERNATIONAL JOURNAL OF DERMATOLOGY
(2021)
Letter
Dermatology
L. Juay, N. S. Chandran
JOURNAL OF THE EUROPEAN ACADEMY OF DERMATOLOGY AND VENEREOLOGY
(2021)
Letter
Dermatology
Antoni Gostynski, Gilles F. H. Diercks, Maria-Jose Escamez, Nisha Suyien Chandran, Raul de Lucas, Adela Garcia-Martin, Marcela Del Rio, Jeroen Bremer, Maria C. Bolling, Alvaro Meana, Sara G. Llames, Enno Schmidt, Ralf Ludwig, Marcel F. Jonkman, Hendri H. Pas, Anna M. G. Pasmooij
JOURNAL OF INVESTIGATIVE DERMATOLOGY
(2022)
Letter
Dermatology
Jiekai Tan, Jingxiang Huang, Nisha Suyien Chandran
ANNALS OF DERMATOLOGY
(2020)