Clinical and economic burden of immune tolerance induction in entire patients with hemophilia A: Insights from a real-world Korean setting
In this study, we aimed to obtain real-world evidence on the clinical and economic aspects and associated burdens experienced by patients with hemophilia A with inhibitors undergoing ITI in Korea. (Source: Thrombosis Research)
Source: Thrombosis Research - April 12, 2024 Category: Hematology Authors: Ah-Young Kim, Hee Jo Baek, Sukhyang Lee, Eunjung Choo, Young Shil Park, Hankil Lee Tags: Full Length Article Source Type: research
Assessment of jaw bone mineral density, resorption rates, and oral health in patients with severe hemophilia: a case-control study
Conclusions: Because of poor oral hygiene, high bone resorption, and low bone mineral density in these patients, clinicians should consider potential bone changes when planning to treat these patients.PMID:38597918 | DOI:10.2340/aos.v83.40337 (Source: Acta Odontologica Scandinavica)
Source: Acta Odontologica Scandinavica - April 10, 2024 Category: Dentistry Authors: G ülin Acar Alper Akta ş Source Type: research
Long-term efficacy and safety of damoctocog alfa pegol prophylaxis in patients with haemophilia A aged 12- & lt;18 years at enrolment into PROTECT VIII
Conclusion Efficacy and safety of damoctocog alfa pegol was confirmed in adolescent patients with haemophilia A, with data for up to 6 years supporting its use as a long-term treatment option in this group as they transition into adulthood.PMID:38599195 | DOI:10.1159/000538702 (Source: Acta Haematologica)
Source: Acta Haematologica - April 10, 2024 Category: Hematology Authors: Mark T Reding Mindy Simpson Jonathan Ducore P ål Andrè Holme Monika Maas Enriquez Maria Elisa Mancuso Source Type: research
Assessment of jaw bone mineral density, resorption rates, and oral health in patients with severe hemophilia: a case-control study
Conclusions: Because of poor oral hygiene, high bone resorption, and low bone mineral density in these patients, clinicians should consider potential bone changes when planning to treat these patients.PMID:38597918 | DOI:10.2340/aos.v83.40337 (Source: Acta Odontologica Scandinavica)
Source: Acta Odontologica Scandinavica - April 10, 2024 Category: Dentistry Authors: G ülin Acar Alper Akta ş Source Type: research
Long-term efficacy and safety of damoctocog alfa pegol prophylaxis in patients with haemophilia A aged 12- & lt;18 years at enrolment into PROTECT VIII
Conclusion Efficacy and safety of damoctocog alfa pegol was confirmed in adolescent patients with haemophilia A, with data for up to 6 years supporting its use as a long-term treatment option in this group as they transition into adulthood.PMID:38599195 | DOI:10.1159/000538702 (Source: Acta Haematologica)
Source: Acta Haematologica - April 10, 2024 Category: Hematology Authors: Mark T Reding Mindy Simpson Jonathan Ducore P ål Andrè Holme Monika Maas Enriquez Maria Elisa Mancuso Source Type: research
Long-term efficacy and safety of damoctocog alfa pegol prophylaxis in patients with haemophilia A aged 12- & lt;18 years at enrolment into PROTECT VIII
Conclusion Efficacy and safety of damoctocog alfa pegol was confirmed in adolescent patients with haemophilia A, with data for up to 6 years supporting its use as a long-term treatment option in this group as they transition into adulthood.PMID:38599195 | DOI:10.1159/000538702 (Source: Acta Haematologica)
Source: Acta Haematologica - April 10, 2024 Category: Hematology Authors: Mark T Reding Mindy Simpson Jonathan Ducore P ål Andrè Holme Monika Maas Enriquez Maria Elisa Mancuso Source Type: research
Long-term efficacy and safety of damoctocog alfa pegol prophylaxis in patients with haemophilia A aged 12- & lt;18 years at enrolment into PROTECT VIII
Conclusion Efficacy and safety of damoctocog alfa pegol was confirmed in adolescent patients with haemophilia A, with data for up to 6 years supporting its use as a long-term treatment option in this group as they transition into adulthood.PMID:38599195 | DOI:10.1159/000538702 (Source: Acta Haematologica)
Source: Acta Haematologica - April 10, 2024 Category: Hematology Authors: Mark T Reding Mindy Simpson Jonathan Ducore P ål Andrè Holme Monika Maas Enriquez Maria Elisa Mancuso Source Type: research
Ultrasound in addition to clinical assessment of acute musculoskeletal complaints in bleeding disorders: impact on patient management
CONCLUSION: Ultrasound in addition to clinical assessment of acute musculoskeletal complaints in people with hemophilia and VWD has an impact on diagnosis (36%) and treatment (39%), which supports the use of ultrasound in acute musculoskeletal complaints in hemophilia and VWD.PMID:38590365 | PMC:PMC10999475 | DOI:10.1016/j.rpth.2024.102372 (Source: Thrombosis and Haemostasis)
Source: Thrombosis and Haemostasis - April 9, 2024 Category: Hematology Authors: Flora Hendrica Pieternella van Leeuwen Wouter Foppen Pim A de Jong Wobke E M van Dijk Johan Blokzijl Kathelijn Fischer Merel A Timmer Source Type: research
The imperative to prevent joint bleeding in everyone living with hemophilia
Res Pract Thromb Haemost. 2024 Mar 15;8(2):102383. doi: 10.1016/j.rpth.2024.102383. eCollection 2024 Feb.NO ABSTRACTPMID:38590364 | PMC:PMC11000100 | DOI:10.1016/j.rpth.2024.102383 (Source: Thrombosis and Haemostasis)
Source: Thrombosis and Haemostasis - April 9, 2024 Category: Hematology Authors: Leonard A Valentino Source Type: research
Predicting inhibitor development using a random peptide phage-display library approach in the SIPPET Cohort
Blood Adv. 2024 Apr 9:bloodadvances.2023011388. doi: 10.1182/bloodadvances.2023011388. Online ahead of print.ABSTRACTInhibitor development is the most severe complication of hemophilia A care, and is associated with increased morbidity and mortality. The aim of this study was to use a novel IgG epitope mapping method to explore the factor VIII (FVIII)-specific epitope profile in the SIPPET cohort population and to develop an epitope-mapping based inhibitor prediction model. The population consisted of 122 previously untreated patients with severe hemophilia A that were followed-up for 50 days of exposure to FVIII or 3 year...
Source: Adv Data - April 9, 2024 Category: Epidemiology Authors: Shermarke Hassan Guido Baselli Luca Mollica Riccardo L Rossi Himani Chand Amal Mohamed El-Beshlawy Mohsen Saleh Elalfy Vijay Ramanan Peyman Eshghi Mehran Karimi Roberta Palla Frits R Rosendaal Flora Peyvandi Source Type: research
Ultrasound in addition to clinical assessment of acute musculoskeletal complaints in bleeding disorders: impact on patient management
CONCLUSION: Ultrasound in addition to clinical assessment of acute musculoskeletal complaints in people with hemophilia and VWD has an impact on diagnosis (36%) and treatment (39%), which supports the use of ultrasound in acute musculoskeletal complaints in hemophilia and VWD.PMID:38590365 | PMC:PMC10999475 | DOI:10.1016/j.rpth.2024.102372 (Source: Thrombosis and Haemostasis)
Source: Thrombosis and Haemostasis - April 9, 2024 Category: Hematology Authors: Flora Hendrica Pieternella van Leeuwen Wouter Foppen Pim A de Jong Wobke E M van Dijk Johan Blokzijl Kathelijn Fischer Merel A Timmer Source Type: research
The imperative to prevent joint bleeding in everyone living with hemophilia
Res Pract Thromb Haemost. 2024 Mar 15;8(2):102383. doi: 10.1016/j.rpth.2024.102383. eCollection 2024 Feb.NO ABSTRACTPMID:38590364 | PMC:PMC11000100 | DOI:10.1016/j.rpth.2024.102383 (Source: Thrombosis and Haemostasis)
Source: Thrombosis and Haemostasis - April 9, 2024 Category: Hematology Authors: Leonard A Valentino Source Type: research
Gene Therapy in Hemophilia A: Achievements, Challenges, and Perspectives
Semin Thromb Hemost DOI: 10.1055/s-0044-1785483Strides in advancements of care of persons with hemophilia include development of long-acting factor replacement therapies, novel substitution and hemostatic rebalancing agents, and most recently approved gene therapy. Several decades of preclinical and clinical trials have led to development of adeno-associated viral (AAV) vector-mediated gene transfer for endogenous production of factor VIII (FVIII) in hemophilia A (HA). Only one gene therapy product for HA (valoctocogene roxaparvovec) has been approved by regulatory authorities. Results of valoctocogene roxaparvovec trial s...
Source: Seminars in Thrombosis and Hemostasis - April 8, 2024 Category: Hematology Authors: Bala, Natasha S. Thornburg, Courtney D. Tags: Review Article Source Type: research
Multicenter evaluation of the haemostatic activity of emicizumab in patients with severe haemophilia A
CONCLUSION: Using a modified TGA, this study showed that patients who experienced breakthrough bleeds while on emicizumab had a lower thrombin generating capacity compared to others with good clinical response to emicizumab.PMID:38583717 | DOI:10.1016/j.jtha.2024.03.022 (Source: Thrombosis and Haemostasis)
Source: Thrombosis and Haemostasis - April 7, 2024 Category: Hematology Authors: Laurie Josset Alexandre Leuci Maissaa Janbain Ana ëlle De-Wreede Stephanie Desage Anne Lienhart Val érie Bin Doroth ée Lambert Xavier Delavenne Yesim Dargaud Source Type: research
Multicenter evaluation of the haemostatic activity of emicizumab in patients with severe haemophilia A
CONCLUSION: Using a modified TGA, this study showed that patients who experienced breakthrough bleeds while on emicizumab had a lower thrombin generating capacity compared to others with good clinical response to emicizumab.PMID:38583717 | DOI:10.1016/j.jtha.2024.03.022 (Source: Thrombosis and Haemostasis)
Source: Thrombosis and Haemostasis - April 7, 2024 Category: Hematology Authors: Laurie Josset Alexandre Leuci Maissaa Janbain Ana ëlle De-Wreede Stephanie Desage Anne Lienhart Val érie Bin Doroth ée Lambert Xavier Delavenne Yesim Dargaud Source Type: research
