Overview of Patients with Hemophilia in China: Demographics, Diseases, Treatment, and Health Status
(Source: Patient Preference and Adherence)
Source: Patient Preference and Adherence - January 12, 2024 Category: International Medicine & Public Health Tags: Patient Preference and Adherence Source Type: research
Risk of low bone mineral density in patients with haemophilia: a systematic review and meta-analysis
This study aimed to examine the risk of low BMD and changes in BMD i... (Source: Journal of Orthopaedic Surgery and Research)
Source: Journal of Orthopaedic Surgery and Research - January 11, 2024 Category: Orthopaedics Authors: Haojing Zhou, Lei Chen, Hai Su, Guoqian Chen and Peijian Tong Tags: Systematic Review Source Type: research
Factor IX administration in the skin primes inhibitor formation and sensitizes hemophilia B mice to systemic factor IX administration
CONCLUSION: Intradermal FIX administration is highly immunogenic, suggesting that the skin compartment is not amenable to immune tolerance induction or therapeutic delivery of clotting factors.PMID:38193070 | PMC:PMC10772885 | DOI:10.1016/j.rpth.2023.102248 (Source: Thrombosis and Haemostasis)
Source: Thrombosis and Haemostasis - January 9, 2024 Category: Hematology Authors: Alexandra Sherman Thais B Bertolini Sreevani Arisa Roland W Herzog Radoslaw Kaczmarek Source Type: research
Emicizumab use in females with moderate or mild hemophilia A without factor VIII inhibitors who warrant prophylaxis
CONCLUSION: Overall, results were consistent with those reported in the male population enrolled in the HAVEN 6 study, suggesting efficacy and a favorable safety profile for emicizumab in females with non-severe HA warranting prophylaxis.PMID:38193069 | PMC:PMC10772889 | DOI:10.1016/j.rpth.2023.102239 (Source: Thrombosis and Haemostasis)
Source: Thrombosis and Haemostasis - January 9, 2024 Category: Hematology Authors: Cedric Hermans Giuliana Ventriglia Samya Obaji Benjamin M Beckermann Michaela Lehle Olivier Catalani Roseline d'Oiron Laurent Frenzel Source Type: research
Effect of emicizumab-neutralizing antibodies on activated partial thromboplastin time-based clotting time test results in patients treated with emicizumab
CONCLUSION: The anti-emi antibodies were shown to completely neutralize emicizumab, making samples pretreated with anti-emi analyzable with the 1-stage assay.PMID:38193066 | PMC:PMC10772886 | DOI:10.1016/j.rpth.2023.102260 (Source: Thrombosis and Haemostasis)
Source: Thrombosis and Haemostasis - January 9, 2024 Category: Hematology Authors: Cristina Novembrino Massimo Boscolo-Anzoletti Eleonora Galbiati Sho Shinohara Flora Peyvandi Source Type: research
Race, ethnicity, and immune tolerance induction in hemophilia A in the United States
CONCLUSION: Although the role of ITI may evolve with growing use of emicizumab and the introduction of new hemophilia treatment products, understanding characteristics that influence care, particularly race and ethnicity, where physician bias and patient mistrust can occur, will remain relevant and applicable to other complex therapies, including gene therapy.PMID:38193063 | PMC:PMC10772873 | DOI:10.1016/j.rpth.2023.102251 (Source: Thrombosis and Haemostasis)
Source: Thrombosis and Haemostasis - January 9, 2024 Category: Hematology Authors: Christine L Kempton Amanda B Payne Stacey A Fedewa Source Type: research
Posttraumatic stress disorder and posttraumatic stress symptoms among adults with hemophilia A and B
CONCLUSION: Persons with hemophilia are at risk of developing PTSD and posttraumatic stress symptoms. These data support the need for trauma screening, psychosocial services in the bleeding disorders community, and provision of trauma-informed care by providers.PMID:38193061 | PMC:PMC10772881 | DOI:10.1016/j.rpth.2023.102241 (Source: Thrombosis and Haemostasis)
Source: Thrombosis and Haemostasis - January 9, 2024 Category: Hematology Authors: Amanda Stahl Kaitlin Barnett Amy K Wilson Siyang Ren Donna Neuberg Hae Soo Park Aric Parnes Source Type: research
The World Federation of Hemophilia World Bleeding Disorders Registry: insights from the first 10,000 patients
CONCLUSION: Data reveal large treatment and care disparities between socioeconomic groups, showing improvements when prophylaxis is initiated to prevent bleeding. Overall, care provided in low-income countries lags behind high-income countries by up to 40 years. Limitations in the interpretation of data include risk of survival and selection bias.PMID:38193052 | PMC:PMC10772874 | DOI:10.1016/j.rpth.2023.102264 (Source: Thrombosis and Haemostasis)
Source: Thrombosis and Haemostasis - January 9, 2024 Category: Hematology Authors: Donna Coffin Emma Gouider Barbara Konkle Cedric Hermans Catherine Lambert Saliou Diop Emily Ayoub Ellia Tootoonchian Toong Youttananukorn Pamela Dakik Ticiana Pereira Alfonso Iorio Glenn F Pierce World Bleeding Disorders Registry Participating Investigato Source Type: research
Inhibitor development according to concentrate in severe hemophilia: reporting on 1392 Previously Untreated Patients from Europe and Canada
CONCLUSION: While confirming expected rates of inhibitors in PUPs, inhibitor development was lower in pdFVIII than in SHL-rFVIII. Preliminary data suggest variation in inhibitor development among different SHL-rFVIII and EHL-rFVIII concentrates.PMID:38193044 | PMC:PMC10772871 | DOI:10.1016/j.rpth.2023.102265 (Source: Thrombosis and Haemostasis)
Source: Thrombosis and Haemostasis - January 9, 2024 Category: Hematology Authors: Kathelijn Fischer Riitta Lassila Flora Peyvandi Alexander Gatt Rob Hollingsworth Thierry Lambert Radek Kaczmarek Amanda Bettle Nasrin Samji Georges- Étienne Rivard Manuel Carcao Alfonso Iorio Mike Makris EUHASS and CHESS participants Source Type: research
Effect of emicizumab-neutralizing antibodies on activated partial thromboplastin time-based clotting time test results in patients treated with emicizumab
CONCLUSION: The anti-emi antibodies were shown to completely neutralize emicizumab, making samples pretreated with anti-emi analyzable with the 1-stage assay.PMID:38193066 | PMC:PMC10772886 | DOI:10.1016/j.rpth.2023.102260 (Source: Biomed Res)
Source: Biomed Res - January 9, 2024 Category: Research Authors: Cristina Novembrino Massimo Boscolo-Anzoletti Eleonora Galbiati Sho Shinohara Flora Peyvandi Source Type: research
Pleiotropic Effects of Different Exonic Nucleotide Changes at the Same Position Contributing to Hemophilia B Phenotypic Variation
CONCLUSION: Overall, albeit a multi-tools bioinformatics approach suggested the molecular effects of multiple HB variants, the deep investigation of molecular mechanisms revealed insights into the HB phenotype-genotype relationship, enabling accurate classification of HB variants. Importantly, knowledge of molecular mechanisms allowed the development of tailored RNA therapeutics, which can also be translated to other genetic diseases.PMID:38184202 | DOI:10.1016/j.jtha.2023.12.031 (Source: Thrombosis and Haemostasis)
Source: Thrombosis and Haemostasis - January 6, 2024 Category: Hematology Authors: Huayang Zhang Min Xin Liya Lin Changming Chen Dario Balestra Qiulan Ding Source Type: research
Pleiotropic Effects of Different Exonic Nucleotide Changes at the Same Position Contributing to Hemophilia B Phenotypic Variation
CONCLUSION: Overall, albeit a multi-tools bioinformatics approach suggested the molecular effects of multiple HB variants, the deep investigation of molecular mechanisms revealed insights into the HB phenotype-genotype relationship, enabling accurate classification of HB variants. Importantly, knowledge of molecular mechanisms allowed the development of tailored RNA therapeutics, which can also be translated to other genetic diseases.PMID:38184202 | DOI:10.1016/j.jtha.2023.12.031 (Source: Thrombosis and Haemostasis)
Source: Thrombosis and Haemostasis - January 6, 2024 Category: Hematology Authors: Huayang Zhang Min Xin Liya Lin Changming Chen Dario Balestra Qiulan Ding Source Type: research
Subcutaneous marzeptacog alfa (activated) for on-demand treatment of bleeding events in subjects with haemophilia A or B with inhibitors
Clin Pharmacol Ther. 2024 Jan 3. doi: 10.1002/cpt.3172. Online ahead of print.ABSTRACTMarzeptacog alfa (MarzAA) is under development for subcutaneous treatment of episodic bleeds in hemophilia A/B patients and was studied in a Phase 3 trial evaluating MarzAA compared to standard-of-care (SoC) for on-demand use. The work presented here aimed to evaluate MarzAA and SoC treatment of bleeding events on a standardized 4-point efficacy scale (poor, fair, good and excellent). Two continuous-time Markov modeling approaches were explored; a four-state model analyzing all four categories of bleeding improvement and a two-state model...
Source: Clinical Pharmacology and Therapeutics - January 4, 2024 Category: Drugs & Pharmacology Authors: Alan Faraj Joakim Nyberg Grant E Blouse Tom Knudsen Ulrika S H Simonsson Source Type: research
Subcutaneous marzeptacog alfa (activated) for on-demand treatment of bleeding events in subjects with haemophilia A or B with inhibitors
Clin Pharmacol Ther. 2024 Jan 3. doi: 10.1002/cpt.3172. Online ahead of print.ABSTRACTMarzeptacog alfa (MarzAA) is under development for subcutaneous treatment of episodic bleeds in hemophilia A/B patients and was studied in a Phase 3 trial evaluating MarzAA compared to standard-of-care (SoC) for on-demand use. The work presented here aimed to evaluate MarzAA and SoC treatment of bleeding events on a standardized 4-point efficacy scale (poor, fair, good and excellent). Two continuous-time Markov modeling approaches were explored; a four-state model analyzing all four categories of bleeding improvement and a two-state model...
Source: Clinical Pharmacology and Therapeutics - January 4, 2024 Category: Drugs & Pharmacology Authors: Alan Faraj Joakim Nyberg Grant E Blouse Tom Knudsen Ulrika S H Simonsson Source Type: research
