Article
Hematology
Guy Young, Michael U. Callaghan, Vinod Balasa, Amit Soni, Sanjay Ahuja, Jonathan C. Roberts, Mindy L. Simpson, Hande Kizilocak, Andrew Frick, Ali G. Mokdad, Shan Xing, Jorge Caicedo
Summary: This retrospective study examined the clinical outcomes before and after pharmacokinetic-guided prophylaxis in persons with hemophilia. The results showed that the majority of patients experienced a decrease in bleeding rates or maintained zero bleeding rates after pharmacokinetic-guided treatment. Non-switchers did not have significant changes in the cost of therapy, while switchers experienced increased costs due to higher prices of extended half-life products.
Article
Hematology
Kun Huang, Di Ai, Gang Li, Yingzi Zhen, Yan Wang, Ningning Zhang, Aihua Huo, Guoqing Liu, Zhenping Chen, Runhui Wu
Summary: This study demonstrates the effectiveness of an individualised prophylaxis protocol in reducing bleeding rate and improving joint health in boys with severe haemophilia A. The findings highlight the importance of personalised prophylaxis in managing the condition.
Article
Hematology
Fitri Primacakti, Teny T. Sari, Djajadiman Gatot, Hikari A. Sjakti, Novie A. Chozie
Summary: This study investigated the pharmacokinetic profile of children with severe hemophilia A receiving low-dose prophylaxis of coagulation factor VIII (FVIII). The study found inter-individual differences in the PK parameters and suggested different dosing intervals for FVIII prophylaxis based on individual PK profiles to achieve optimal bleeding prevention.
Review
Hematology
Pier Mannuccio Mannucci, Paolo Angelo Cortesi, Matteo Nicola Dario Di Minno, Mario Sano, Lorenzo Giovanni Mantovani, Giovanni Di Minno
Summary: The study evaluated several different treatment products for hemophilia A, finding that while EHL-rFVIII products performed well in reducing injection frequency and maintaining clinical efficacy, there were differences in rFVIII consumption, which may result in different economic impacts.
Article
Oncology
Marguerite Lockhart, Brigitte Tardy-Poncet, Aurelie Montmartin, Pauline Noyel, Sandrine Thouvenin, Claire Berger
Summary: For patients with severe hemophilia A receiving emicizumab treatment, it is important to adjust the dosage of bypassing agents based on thrombin-generation assay and collegial consultation to prevent bleeding risk before surgery.
PEDIATRIC BLOOD & CANCER
(2021)
Article
Hematology
Feng Xue, Xielan Zhao, Jing Sun, Xiaojing Zeng, Fenge Yang, Ming Xu, Ziqiang Yu, Weiying Gu, Ying Feng, Wenqian Li, Changcheng Zheng, Hui Bi, Liangzhi Xie, Wenlin Gai, Renchi Yang
Summary: After a 24-week study, SCT800 demonstrated comparable repeat PK profiles. The total ABR was 2.82, with 43.8% of patients experiencing no bleeding events. The majority of bleeding episodes (89.4%) were controlled with 1-2 injections of SCT800, and the treatment success rate was 92.6%. The incidence of treatment-related adverse events was 53.4%, with a drug-related AE incidence of 4.1%.
Review
Hematology
Madison J. Petkovic, Huyen A. Tran, Peter R. Ebeling, Ayse Zengin
Summary: Patients with haemophilia are at high risk of developing osteoporosis, falls, and fractures. However, current guidelines for managing these conditions are inadequate and further research is needed to improve prevention and management strategies.
Article
Hematology
Carmen H. Coxon, Xinren Yu, James Beavis, Laura Diaz-Saez, Andrew Riches-Duit, Chris Ball, Scott L. Diamond, Sanj Raut
Summary: The study aims to develop and characterize a panel of patient-derived monoclonal antibodies to better understand drug immunogenicity and explore ways to overcome it. By using recombinant antibodies, a rapid, sensitive, flexible and reproducible ex vivo assay was developed, which replicates inhibitor patient blood using blood from healthy volunteers. These materials can serve as experimental exemplars or controls for rational, hypothesis-driven research and assay development related to FVIII immunogenicity and FVIII-related products.
BRITISH JOURNAL OF HAEMATOLOGY
(2021)
Article
Cell Biology
Kui Huang, Shuyu Cai, Ting Fu, Qiang Zhu, Lin Liu, Zhihao Yao, Pengcheng Rao, Xiaorong Lan, Qing Li, Jingang Xiao
Summary: It was found that the expression of Wnt10b RNA in OP-ASCs was significantly lower than that in ASCs. The upregulation of Wnt10b can enhance the osteogenic ability of OP-ASCs and promote bone defect healing.
CELL PROLIFERATION
(2023)
Article
Hematology
Vuokko Nummi, Anna-Elina Lehtinen, Alfonso Iorio, Timea Szanto, Riitta Lassila
Summary: This study compared the clinical and laboratory outcomes between SHL and EHL FVIII prophylaxis in haemophilia A patients. The results showed that switching to EHL led to decreased infusion frequency and factor consumption, and excellent clinical efficacy.
Article
Pharmacology & Pharmacy
Laura H. Bukkems, Lars L. F. G. Valke, Wideke Barteling, Britta A. P. Laros-van Gorkom, Nicole M. A. Blijlevens, Marjon H. Cnossen, Waander L. van Heerde, Saskia E. M. Schols, Ron A. A. Mathot
Summary: The study aimed to develop a population PK/PD model based on FVIII activity levels and NHA patterns, in order to assess the relationship between individual hemostatic capacity and dosing methods in hemophilia A patients.
BRITISH JOURNAL OF CLINICAL PHARMACOLOGY
(2022)
Article
Medicine, General & Internal
Erik Berntorp, Kathelijn Fischer, Daniel P. Hart, Maria Elisa Mancuso, David Stephensen, Amy D. Shapiro, Victor Blanchette
Summary: Haemophilia A and B are rare congenital disorders caused by deficiency of clotting factors, leading to symptoms such as bleeding and joint disease. With advancements in treatment, individuals with haemophilia can now expect almost normal life expectancy and quality of life, although the development of inhibitory antibodies remains a major challenge for some patients. Gene therapy for haemophilia is progressing and may soon become a reality.
NATURE REVIEWS DISEASE PRIMERS
(2021)
Article
Hematology
Jan Astermark, Tyler W. Buckner, Laurent Frenzel, Anthony J. Hatswell, Xiaojun You, Hai Liu, Erin Goodman, Sandra Santos, Charles Hawes, David Hinds, Robert Klamroth
Summary: This study indirectly compared the bleeding rates between valoctocogene roxaparvovec and emicizumab, two treatments for haemophilia A. The results showed that valoctocogene roxaparvovec had lower bleeding rates and higher proportions of participants with no joint bleeds compared to emicizumab. Emicizumab was also more effective than FVIII prophylaxis regimens.
Article
Hematology
Carla Daffunchio, Gustavo Galatro, Virginia Faurlin, Daniela Neme, Horacio Caviglia
Summary: Prophylaxis is the gold standard treatment for children with haemophilia. However, joint damage can still occur, suggesting the presence of subclinical bleeding. Early detection of joint damage is crucial to prevent the development of arthropathy.
THROMBOSIS RESEARCH
(2023)
Article
Hematology
Ragnhild J. Maseide, Erik Berntorp, Vuokko Nummi, Riitta Lassila, Geir E. Tjonnfjord, Pal A. Holme
Summary: Global coagulation assays and thromboelastometry can differentiate patients with moderate hemophilia based on joint health scores, annual factor consumption, and history of orthopedic surgery. These assays may help in predicting the bleeding phenotype in patients with moderate hemophilia.
Article
Orthopedics
Theodore S. Wolfson, Brian Mannino, Brett D. Owens, Brian R. Waterman, Michael J. Alaia
Summary: Bone tunnel-related complications are common in revision ACLR surgeries, and issues such as tunnel positioning, enlargement, containment, and hardware interference can impact the outcomes. However, there is a lack of a systematic approach to address tunnel-related issues. This review aims to highlight the current literature and present an updated approach to evaluate and manage these complications.
AMERICAN JOURNAL OF SPORTS MEDICINE
(2023)
Review
Orthopedics
David A. Bloom, Daniel J. Kaplan, Edward Mojica, Eric J. Strauss, Guillem Gonzalez-Lomas, Kirk A. Campbell, Michael J. Alaia, Laith M. Jazrawi
Summary: This article describes the various methods for determining the minimal clinically important difference (MCID) and discusses their respective strengths and limitations. The article emphasizes the importance of standardization in establishing MCIDs for orthopaedic patient-reported outcome measures and the need for better study design in this area.
AMERICAN JOURNAL OF SPORTS MEDICINE
(2023)
Article
Primary Health Care
Edward S. Mojica, Charles C. Lin, Noah Kirschner, Paola F. Ortega, Eoghan T. Hurley, Kirk A. Campbell, Michael J. Alaia, Laith M. Jazrawi
Summary: This study evaluated the satisfaction and effectiveness of platelet-rich plasma injection in patients with knee osteoarthritis. The results showed that patients were neutral in terms of satisfaction, with only half of them considering the injection worth the cost and willing to undergo it again. Younger age and post-injection pain relief were associated with a higher desire for further injection, but pain relief did not correlate with the perception of worthiness in terms of cost.
PHYSICIAN AND SPORTSMEDICINE
(2023)
Article
Orthopedics
Edward S. Mojica, Andrew S. Bi, Kinjal Vasavada, Jay Moran, Scott Buzin, Joseph Kahan, Erin F. Alaia, Laith M. Jazrawi, Michael J. Medvecky, Michael J. Alaia
Summary: This study compared the outcomes of patients with multi-ligamentous knee injuries (MLKIs) with and without patellar tendon ruptures using propensity-score matching. The results showed that patients with concomitant patellar tendon ruptures had worse functional outcomes compared to those without.
KNEE SURGERY SPORTS TRAUMATOLOGY ARTHROSCOPY
(2023)
Article
Physics, Multidisciplinary
Sergei M. Kuzenko, Michael Ponds, Emmanouil S. N. Raptakis
Summary: In this paper, a new method of defining conformal higher-spin theory in arbitrary backgrounds is provided, and it is extended to the case of N=1 supersymmetry. This method has manifest background conformal symmetry and background N=1 superconformal invariance.
FORTSCHRITTE DER PHYSIK-PROGRESS OF PHYSICS
(2023)
Article
Orthopedics
Andrew S. S. Bi, Mohammad T. Azam, James J. Butler, Michael J. Alaia, Laith M. Jazrawi, Guillem Gonzalez-Lomas, John G. Kennedy
Summary: This study investigated the rates of acute Achilles tendon ruptures at a single academic institute during the COVID-19 pandemic, and found an increased injury rate in 2021, particularly in the NFL. These findings provide prognostic information for counseling patients and athletes on returning to physical activity.
KNEE SURGERY SPORTS TRAUMATOLOGY ARTHROSCOPY
(2023)
Article
Hematology
Rolf Ljung, Anthony K. C. Chan, Heidi Glosli, Olubunmi Afonja, Bastian Becker, Despina Tseneklidou-Stoeter, Maria Elisa Mancuso, Sonata Saulyte-Trakymiene, Gili Kenet
Summary: This study evaluated the efficacy and safety of BAY 81-8973 in previously untreated/minimally treated patients with hemophilia A. It was found that BAY 81-8973 was effective for bleed prevention and treatment, but the occurrence of inhibitors was relatively high. Immune tolerance induction treatment was effective in some patients.
THROMBOSIS AND HAEMOSTASIS
(2023)
Article
Hematology
Kristina Kihlberg, Fariba Baghaei, Maria Bruzelius, Eva Funding, Pal Andre Holme, Riitta Lassila, Vuokko Nummi, Susanna Ranta, Nadine Gretenkort Andersson, Erik Berntorp, Jan Astermark
Summary: This study aimed to assess the health-related quality of life in persons with haemophilia B (PwHB) compared to haemophilia A (PwHA), and to evaluate the impact of joint health on their quality of life. The results showed that both PwHB and PwHA reported impaired quality of life, and joint health had a significant negative impact on their quality of life.
Article
Hematology
Alexandros Arvanitakis, Pal Andre Holme, Erik Berntorp, Jan Astermark
Summary: The timing and type of prophylaxis, as well as F8 genotype, can influence treatment outcomes in adults with severe haemophilia A. Delayed prophylaxis with intermediate dose intensity reduces bleeding but increases arthropathy and decreases HRQoL compared to higher intensity primary prophylaxis. Non-null F8 genotype may allow lower factor consumption with similar joint health and bleeding rates.
Article
Operations Research & Management Science
Marin Bougeret, Artur Pessoa, Michael Poss
Summary: We study robust single machine scheduling problems. First, we prove that with uncertain processing times, minimizing the number of tardy jobs is NP-hard. Second, we show that the weighted variant of the problem has the same complexity as the nominal counterpart whenever only the weights are uncertain. Last, we provide approximation algorithms for the problems minimizing the weighted sum of completion times. Noticeably, our algorithms extend to more general robust combinatorial optimization problems with cost uncertainty, such as max-cut.
OPERATIONS RESEARCH LETTERS
(2023)
Meeting Abstract
Hematology
Lisa Boggio, Maria Elisa Mancuso, Suchitra Acharya, Santiago Bonanad, Meera Chitlur, Yesim Dargaud, Osman Khan, Philip Maes, Wolfgang Miesbach, Danielle Nance, Amina Rafique, Daniel Bonzo, Christopher Macie, Thomas Wilkinson, Miguel Escobar
AMERICAN JOURNAL OF HEMATOLOGY
(2023)
Meeting Abstract
Hematology
Donna Coffin, Barbara Konkle, Mayss Naccache, Cary Clark, Lindsey George, Alfonso Iorio, Wolfgang Miesbach, Declan Noone, Flora Peyvandi, Steve Pipe, Michael Recht, Mark Skinner, Leonard A. Valentino, Johnny N. Mahlangu, Glenn Pierce
AMERICAN JOURNAL OF HEMATOLOGY
(2023)
Meeting Abstract
Hematology
Esteban Gomez, Adam Giermasz, Giancarlo Castaman, Nigel S. Key, Susan Lattimore, Frank W. G. Leebeek, Wolfgang A. Miesbach, Michael Recht, Annette von Drygalski, David Cooper, Yanyan Li, Blanca Salazar, Steven W. Pipe
AMERICAN JOURNAL OF HEMATOLOGY
(2023)
Article
Orthopedics
Charles Wang, Jonathan L. Glashow, Michael J. Alaia
BULLETIN OF THE HOSPITAL FOR JOINT DISEASES
(2023)
Article
Orthopedics
Daniel J. Kaplan, Michael J. Alaia, Eric J. Strauss, Laith M. Jazrawi
Summary: The anterior cruciate ligament (ACL) is the main restraint to tibial internal rotation, supported by secondary stabilizers. Injuries to both primary and secondary rotational stabilizers can cause anterolateral rotatory instability. ACL reconstruction (ACLR) alone does not fully restore native kinematics in combined injuries. Concomitant anterolateral ligament reconstruction (ALLR) and lateral extra-articular tenodesis (LET) techniques have shown to improve rotational control, with LET being slightly more powerful.
BULLETIN OF THE HOSPITAL FOR JOINT DISEASES
(2023)