Search

Your search keyword '"Oldenburg, Johannes"' showing total 1,952 results
Start Over Author "Oldenburg, Johannes"
1,952 results on '"Oldenburg, Johannes"'

4. Prediction of the chance of successful immune tolerance induction in persons with severe hemophilia A and inhibitors: a clinical prediction model

Author

Oomen, Ilja, Abdi, Amal, Camelo, Ricardo M., Callado, Fábia M.R.A., Carvalho, Luany E.M., Calcaterra, Ilenia L., Carcao, Manuel, Castaman, Giancarlo, Eikenboom, Jeroen C.J., Fischer, Kathelijn, Franco, Vivian K.B., Heymans, Martijn W., Leebeek, Frank W.G., Lillicrap, David, Lorenzato, Cláudia S., Mancuso, Maria Elisa, Matino, Davide, Di Minno, Matteo N.D., Mohseny, Alex B., Oldenburg, Johannes, Rezende, Suely Meireles, Rivard, Georges-Etienne, Rydz, Natalia, Schols, Saskia E.M., Voorberg, Jan, Fijnvandraat, Karin, and Gouw, Samantha C.

Published
2024
Full Text
View/download PDF

9. The association between unemployment and treatment among adults with hemophilia

Author

Pabinger, Ingrid, Hermans, Cedric, Roseline d’Oiron, Klamroth, Robert, Oldenburg, Johannes, Marquardt, Natascha, Staritz, Peter, Katsarou, Olga, Martinowitz, Uri, Lubetsky, Aharon, Kenet, Gili, Tagliaferri, Annarita, Mancuso, Maria Elisa, Schutgens, Roger, HolmE, Pål André, Windyga, Jerzy, Zupan, Irena, Yuste, Victor Jimenez, Nunez, Ramiro, de Moerloose, Philippe, Berntorp, Erik, Astermark, Jan, Tait, Campbell, Dolan, Gerry, Qvigstad, Christian, Sørensen, Lars Q., Tjønnfjord, Geir E., and Holme, Pål André

Published
2024
Full Text
View/download PDF

11. Three-year outcomes of valoctocogene roxaparvovec gene therapy for hemophilia A

Author

Madan, Bella, Ozelo, Margareth C., Raheja, Priyanka, Symington, Emily, Quon, Doris V., Leavitt, Andrew D., Pipe, Steven W., Lowe, Gillian, Kenet, Gili, Reding, Mark T., Mason, Jane, Wang, Michael, von Drygalski, Annette, Klamroth, Robert, Shapiro, Susan, Chambost, Hervé, Dunn, Amy L., Oldenburg, Johannes, Chou, Sheng-Chieh, Peyvandi, Flora, Millar, Carolyn M., Osmond, Dane, Yu, Hua, Dashiell-Aje, Ebony, Robinson, Tara M., and Mahlangu, Johnny

Published
2024
Full Text
View/download PDF

12. Global Seroprevalence of Pre-existing Immunity Against AAV5 and Other AAV Serotypes in People with Hemophilia A

Author

Klamroth, Robert, Hayes, Gregory, Andreeva, Tatiana, Gregg, Keith, Suzuki, Takashi, Mitha, Ismail Haroon, Hardesty, Brandon, Shima, Midori, Pollock, Toni, Slev, Patricia, Oldenburg, Johannes, Ozelo, Margareth C, Stieltjes, Natalie, Castet, Sabine-Marie, Mahlangu, Johnny, Peyvandi, Flora, Kazmi, Rashid, Schved, Jean-François, Leavitt, Andrew D, Callaghan, Michael, Pan-Petesch, Brigitte, Quon, Doris V, Andrews, Jayson, Trinh, Alex, Li, Mingjin, and Wong, Yen

Subjects
Medical Biotechnology, Biomedical and Clinical Sciences, Genetics, Prevention, Antibodies, Neutralizing, Antibodies, Viral, Dependovirus, Genetic Vectors, Hemophilia A, Humans, Prospective Studies, Seroepidemiologic Studies, Serogroup, gene therapy, hemophilia A, adeno-associated virus, antibody, seropositivity, Clinical Sciences, Biotechnology, Medical biotechnology
Abstract

Adeno-associated virus (AAV)-mediated gene therapy may provide durable protection from bleeding events and reduce treatment burden for people with hemophilia A (HA). However, pre-existing immunity against AAV may limit transduction efficiency and hence treatment success. Global data on the prevalence of AAV serotypes are limited. In this global, prospective, noninterventional study, we determined the prevalence of pre-existing immunity against AAV2, AAV5, AAV6, AAV8, and AAVrh10 among people ≥12 years of age with HA and residual FVIII levels ≤2 IU/dL. Antibodies against each serotype were detected using validated, electrochemiluminescent-based enzyme-linked immunosorbent assays. To evaluate changes in antibody titers over time, 20% of participants were retested at 3 and 6 months. In total, 546 participants with HA were enrolled at 19 sites in 9 countries. Mean (standard deviation) age at enrollment was 36.0 (14.87) years, including 12.5% younger than 18 years, and 20.0% 50 years of age and older. On day 1, global seroprevalence was 58.5% for AAV2, 34.8% for AAV5, 48.7% for AAV6, 45.6% for AAV8, and 46.0% for AAVrh10. Considerable geographic variability was observed in the prevalence of pre-existing antibodies against each serotype, but AAV5 consistently had the lowest seroprevalence across the countries studied. AAV5 seropositivity rates were 51.8% in South Africa (n = 56), 46.2% in Russia (n = 91), 40% in Italy (n = 20), 37.2% in France (n = 86), 26.8% in the United States (n = 71), 26.9% in Brazil (n = 26), 28.1% in Germany (n = 89), 29.8% in Japan (n = 84), and 5.9% in the United Kingdom (n = 17). For all serotypes, seropositivity tended to increase with age. Serostatus and antibody titer were generally stable over the 6-month sampling period. As clinical trials of AAV-mediated gene therapies progress, data on the natural prevalence of antibodies against various AAV serotypes may become increasingly important.

Published
2022

15. Inhibitor development according to concentrate after 50 exposure days in severe hemophilia: data from the European HAemophilia Safety Surveillance (EUHASS)

Author

Ay, Cihan, Male, Christoph, Hermans, Cedric, Verhamme, Peter, Lissitchkov, Toshko, Antoniades, Marios, Penka, Miroslav, Blatny, Jan, Komrska, Vladimir, Poulsen, Lone Hvitfeldt, Kampmann, Peter, Lehtinen, Anna-Elina, Susen, Sophie, Dargaud, Yesim, Biron, Christine, D'Oiron, Roseline, Harroche, Annie, Klamroth, Robert, Oldenburg, Johannes, Buehrlen, Martina, Miesbach, Wolfgang, Langer, Florian, Spannag, Patrick, Oliveri, Martin, Platokouki, Helen, Nomikou, Efrosyni, Katsarou, Olga, Garypidou, Vasileia, Economou, Marina, Nemes, Laszlo, Nolan, Beatrice, O'Connell, Niamh, Paolo, Radossi, Castaman, Giancarlo, Peyvandi, Flora, Rocino, Angiola, Zanon, Ezio, Tagliaferri, Annarita, Agnelli, Giancarlo, De Crisotofaro, Raimondo, Schinco, Piercarla, Tosetto, Alberto, Lejniece, Sandra, Gailiute, Neringa, Gatt, Alexander, Mäkelburg, Anja, Laros-van Gorkom, Britta, Brons, Paul, Leebeek, Frank W.G., Schutgens, Roger, Windyga, Jerzy, Catarino, Cristina, Aires, Anabela, Fraga, Cristina, Morais, Sara, Araújo, Fernando, Serban, Margit, Davydkin, Igor, Batorova, Angelika, Anzej Doma, Sasa, Martinez, Laura Segura, Palomo Bravo, Angeles, Ortega, Immaculada Soto, Bonanad, Santiago, Baghaei, Fariba, Astermark, Jan, Holmström, Margareta, Fontana, Pierre, Schmugge, Markus, Zulfikar, Bulent, Kavakli, Kaan, Khan, Mohammed, Benson, Gary, Lester, Will, Andrew, Page, Catherine, Bagot, Pinto, Fernando, Salta, Styliani, Farrelly, Cathy, Matthias, Mary, Laffan, Mike, Thynn Thynn, Yee, Mcdonald, Vickie, Austin, Steve, Bella, Madan, Hay, Charles, Grainger, John, Talks, Kate, Shapiro, Susie, Maclean, Rhona, Payne, Jeanette, Fischer, Kathelijn, Lassila, Riitta, Gouw, Samantha C., Hollingsworth, Rob, Lambert, Thierry, Kaczmarek, Radek, Carbonero, Diana, and Makris, Mike

Published
2024
Full Text
View/download PDF

23. Determinants of bleeding before and during immune tolerance in 222 boys with severe hemophilia A and inhibitors >5 BU

Author

Alvarèz Román, MT, Benitez Hidalgo, O, Blatny, J, Bührlen, M, Carvalho, M, Castaman, G, Chambost, H, Rosa Cid, A, Escuriola-Ettingshausen, C, Fischer, K, Van Geet, C, Gretenkort Andersson, N, Kartal-Kaess, M, Knudsen, H, Königs, C, Koskenvuo, M, Male, C, Stamm Mikkelsen, T, Molinari, A, Motwani, J, Nolan, B, d’Oiron, R, Oldenburg, J, Olivieri, M, Oudot, C, Pergantou, H, Pinto, F, Ranta, S, Zápotocká, E, Kenet, G, Carcao, M, Rivard, G, Fischer, Kathelijn, Kenet, Gili, Kurnik, Karin, Carcao, Manuel, Oldenburg, Johannes, Stamm-Mikkelsen, Torben, Cid Haro, Ana Rosa, Koskenvuo, Minna, Blatny, Jan, and Königs, Christoph

Published
2024
Full Text
View/download PDF

28. Emicizumab prophylaxis in patients with acquired haemophilia A (GTH-AHA-EMI): an open-label, single-arm, multicentre, phase 2 study

Author

Tiede, Andreas, Hart, Christina, Knöbl, Paul, Greil, Richard, Oldenburg, Johannes, Sachs, Ulrich J, Miesbach, Wolfgang, Pfrepper, Christian, Trautmann-Grill, Karolin, Holstein, Katharina, Pilch, Jan, Möhnle, Patrick, Schindler, Christoph, Weigt, Carmen, Schipp, Dorothea, May, Marcus, Dobbelstein, Christiane, Pelzer, Fabius J, Werwitzke, Sonja, and Klamroth, Robert

Published
2023
Full Text
View/download PDF

29. German Patient Blood Management Network: effectiveness and safety analysis in 1.2 million patients

Author

Baumhove, Olaf, Leeuw van Weenen, Samuel de, Velten, Markus, Wittmann, Maria, Neumann, Claudia, Kirfel, Andrea, Straßberger-Nerschbach, Nadine, Ehrentraut, Heidi, Grigutsch, Daniel, Guttenthaler, Vera, Puskarevic, Alma, Mohssen, Ghaith, Oldenburg, Johannes, Görtzen, Jan, Narita, Diana, Barbara, Lighvani, Michael Huber, Josef, Choorapoikayil, Suma, Hof, Lotta, Isik, Sabine, Neef, Vanessa, Piekarski, Florian, Schmitt, Elke, Zacharowski, Kai, Walther, Thomas, Keller, Harald, Schnitzbauer, Andreas, Schmitz-Rixen, Thomas, Oikonomou, Kyriakos, Steffen, Bjoern, Zeuzem, Stefan, Czabanka, Marcus, Chun, Felix, Marzi, Ingo, Stöver, Timo, Ghanaati, Shahram, Louwen, Frank, Mueller, Markus M., Geisen, Christoph, Seyfried, Erhard, Herrmann, Eva, Bayer, Alexandra, Weigt, Henry, Lange, Björn, Raadts, Ansgar, Haas, Christoph, Duemmler, Johannes, Lorenzen, Ulf, Pagel, Matthias, Puehler, Thomas, Pochhammer, Julius, Klueter, Tim, Ahmeti, Hajrullah, Bauerschlag, Dirk, Wieker, Henning, Rusch, René, Meybohm, Patrick, Old, Oliver, Müller, Markus M., Geisen, Christof, Seifried, Erhard, de Leeuw van Weenen, Samuel, Friederich, Patrick, Bräutigam, Brigitte, Friedrich, Jens, Gruenewald, Matthias, Elke, Gunnar, Molter, Gerd P., Schwendner, Klaus, Steinbicker, Andrea U., Jenke, Dana J., Thoma, Josef, and Weber, Viola

Published
2023
Full Text
View/download PDF

33. von Willebrand factor neutralizing and non-neutralizing alloantibodies in 213 subjects with type 3 von Willebrand disease enrolled in 3WINTERS-IPS

Author

Pagliari, Maria Teresa, Budde, Ulrich, Baronciani, Luciano, Eshghi, Peyman, Ahmadinejad, Minoo, Badiee, Zahra, Baghaipour, Mohammad-Reza, Benítez Hidalgo, Olga, Biguzzi, Eugenia, Bodó, Imre, Castaman, Giancarlo, Goudemand, Jenny, Karimi, Mehran, Keikhaei, Bijan, Lassila, Riitta, Leebeek, Frank W.G., Lopez Fernandez, Maria Fernanda, Marino, Renato, Oldenburg, Johannes, Peake, Ian, Santoro, Cristina, Schneppenheim, Reinhard, Tiede, Andreas, Toogeh, Gholamreza, Tosetto, Alberto, Trossaert, Marc, Yadegari, Hamideh, Zetterberg, Eva M.K., Mannucci, Pier Mannuccio, Federici, Augusto B., Eikenboom, Jeroen, and Peyvandi, Flora

Published
2023
Full Text
View/download PDF

34. Emicizumab in people with moderate or mild haemophilia A (HAVEN 6): a multicentre, open-label, single-arm, phase 3 study

Author

Négrier, Claude, Mahlangu, Johnny, Lehle, Michaela, Chowdary, Pratima, Catalani, Olivier, Bernardi, Ronald J, Jiménez-Yuste, Víctor, Beckermann, Benjamin M, Schmitt, Christophe, Ventriglia, Giuliana, Windyga, Jerzy, d'Oiron, Roseline, Moorehead, Paul, Koparkar, Sunita, Teodoro, Vanda, Shapiro, Amy D, Oldenburg, Johannes, and Hermans, Cedric

Published
2023
Full Text
View/download PDF

36. Surgical outcomes in people with hemophilia A taking emicizumab prophylaxis: experience from the HAVEN 1-4 studies

Author

Kruse-Jarres, Rebecca, Peyvandi, Flora, Oldenburg, Johannes, Chang, Tiffany, Chebon, Sammy, Doral, Michelle Y., Croteau, Stacy E., Lambert, Thierry, Kempton, Christine L., Pipe, Steven W., Ko, Richard H., Trzaskoma, Benjamin, Dhalluin, Christophe, Bienz, Nives Selak, Niggli, Markus, Lehle, Michaela, Paz-Priel, Ido, Young, Guy, and Jiménez-Yuste, Víctor

Published
2022
Full Text
View/download PDF

38. Von Willebrand factor propeptide and pathophysiological mechanisms in European and Iranian patients with type 3 von Willebrand disease enrolled in the 3WINTERS‐IPS study

Author

Pagliari, Maria Teresa, Rosendaal, Frits R., Ahmadinejad, Minoo, Badiee, Zahra, Baghaipour, Mohammad‐Reza, Baronciani, Luciano, Benítez Hidalgo, Olga, Bodó, Imre, Budde, Ulrich, Castaman, Giancarlo, Eshghi, Peyman, Goudemand, Jenny, Karimi, Mehran, Keikhaei, Bijan, Lassila, Riitta, Leebeek, Frank W.G., Lopez Fernandez, Maria Fernanda, Mannucci, Pier Mannuccio, Marino, Renato, Oldenburg, Johannes, Peake, Ian, Santoro, Cristina, Schneppenheim, Reinhard, Tiede, Andreas, Toogeh, Gholamreza, Tosetto, Alberto, Trossaert, Marc, Yadegari, Hamideh, Zetterberg, Eva M.K., Peyvandi, Flora, Federici, Augusto B., and Eikenboom, Jeroen

Published
2022
Full Text
View/download PDF

40. Superior Prophylactic Effectiveness of a Recombinant FVIIIFc Over Standard Half‐Life FVIII in Hemophilia A: A‐SURE Study.

Author

Oldenburg, Johannes, Hay, Charles, Peyvandi, Flora, Tagliaferri, Annarita, Holme, Pål Andrè, Álvarez‐Román, María Teresa, Biron‐Andréani, Christine, Malmström, Håkan, Bystrická, Linda, and Lethagen, Stefan

Subjects
*BLOOD coagulation factor VIII, *HEMOPHILIACS, *CHIMERIC proteins, *HEMOPHILIA, *INJECTIONS
Abstract

ABSTRACT Objectives Methods Results Conclusions The 24‐month, prospective, non‐interventional, European multicenter A‐SURE study evaluated the real‐world effectiveness of prophylaxis using an extended half‐life recombinant factor VIII (FVIII) Fc fusion protein, efmoroctocog alfa (hereinafter rFVIIIFc), compared with prophylaxis using standard half‐life (SHL) FVIII products in patients with hemophilia A.Primary endpoints were annualized bleeding rate (ABR), annualized injection frequency, and annualized factor consumption. A comparative study design unique for an observational hemophilia study was implemented to reduce potential confounding in effectiveness estimates, wherein each patient prescribed rFVIIIFc was matched with one receiving SHL FVIII. Propensity scores were used for adjustment in statistical analyses.Outcomes for all primary endpoints were significantly better in the rFVIIIFc group (n = 184) compared with the SHL FVIII group (n = 170): mean ABR 1.5 versus 2.3 (difference of −0.8; p = 0.0147); mean annualized injection frequency 114.4 versus 169.2 (difference of −54.8; p < 0.0001); and mean annualized factor consumption 243 024.2 versus 288 718.6 International Units (difference of 45 694.5; p = 0.0003). rFVIIIFc was well tolerated, with no inhibitor development.rFVIIIFc has superior prophylactic effectiveness versus SHL FVIII, providing higher bleed protection with fewer injections and lower factor consumption. [ABSTRACT FROM AUTHOR]

Published
2024
Full Text
View/download PDF

45. Treatment‐related risk factors for inhibitor development in non‐severe hemophilia A after 50 cumulative exposure days: A case‐control study

Author

Abdi, Amal, Eckhardt, Corien L., van Velzen, Alice S., Vuong, Caroline, Coppens, Michiel, Castaman, Giancarlo, Hart, Dan P., Hermans, Cedric, Laros‐van Gorkom, Britta, Leebeek, Frank W.G., Mancuso, Maria Elisa, Mazzucconi, Maria G., McRae, Simon, Oldenburg, Johannes, Male, Christoph, van der Bom, Johanna G., Fijnvandraat, Karin, and Gouw, Samantha C.

Published
2021
Full Text
View/download PDF

47. Determinants of bleeding before and during immune tolerance in 222 boys with severe hemophilia A and inhibitors >5 BU

Author

Poli Van Creveldkliniek Medisch, Child Health, Circulatory Health, Fischer, Kathelijn, Kenet, Gili, Kurnik, Karin, Carcao, Manuel, Oldenburg, Johannes, Stamm-Mikkelsen, Torben, Haro, Ana Rosa Cid, Koskenvuo, Minna, Blatny, Jan, Königs, Christoph, Poli Van Creveldkliniek Medisch, Child Health, Circulatory Health, Fischer, Kathelijn, Kenet, Gili, Kurnik, Karin, Carcao, Manuel, Oldenburg, Johannes, Stamm-Mikkelsen, Torben, Haro, Ana Rosa Cid, Koskenvuo, Minna, Blatny, Jan, and Königs, Christoph

Published
2024

48. Decreased Protein C Pathway Activity in COVID-19 Compared to Non-COVID Sepsis: An Observational and Comparative Cohort Study.

Author

Rühl, Heiko, Bode, Christian, Becher, Tobias, Eckert, Sebastian, Mohsen, Ghaith, McRae, Hannah L., Müller, Jens, Reda, Sara, Loßnitzer, Dirk, Oldenburg, Johannes, Putensen, Christian, and Pötzsch, Bernd

Subjects
COVID-19 pandemic, COVID-19, PROTEIN C, THROMBIN, SEPSIS
Abstract

Sepsis-associated coagulopathy increases risk of mortality. Impairment of the anticoagulant protein C (PC) pathway may contribute to the thrombotic phenotype in coronavirus disease 2019 (COVID-19) sepsis. This study assessed the functionality of this pathway in COVID-19 and non-COVID sepsis by measuring its key enzymes, thrombin and activated PC (APC). The study population included 30 patients with COVID-19, 47 patients with non-COVID sepsis, and 40 healthy controls. In healthy controls, coagulation activation and subsequent APC formation was induced by 15 µg/kg recombinant activated factor VII one hour before blood sampling. APC and thrombin in plasma were measured using oligonucleotide-based enzyme capture assays. The indirect thrombin markers prothrombin-fragment 1+2 (F1+2) and thrombin-antithrombin complex (TAT) were also measured. Compared with stimulated healthy controls, median thrombin, F1+2, and TAT levels were higher in patients with COVID-19 (up to 6-fold, p < 2 × 10−6) and non-COVID sepsis (up to 4.7-fold, p < 0.010). APC levels were 2.4-fold higher in patients with COVID-19 (7.44 pmol/L, p = 0.011) and 3.4-fold higher in non-COVID sepsis patients (10.45 pmol/L, p = 2 × 10−4) than in controls (3.08 pmol/L). Thrombin markers and APC showed correlation in both COVID-19 (r = 0.364–0.661) and non-COVID sepsis patients (r = 0.535–0.711). After adjustment for PC levels, median APC/thrombin, APC/F1+2, and APC/TAT ratios were 2-fold (p = 0.036), 6-fold (p = 3 × 10−7) and 3-fold (p = 8 × 10−4) lower in the COVID-19 group than in the non-COVID sepsis group, and the latter two were also lower in the COVID-19 group than in stimulated healthy controls. In conclusion, it was found that a comparatively lower anticoagulant APC response in COVID-19 patients as compared to non-COVID sepsis patients, potentially linked to endothelial dysfunction, contributes to the prothrombotic phenotype of COVID-19 sepsis. [ABSTRACT FROM AUTHOR]

Published
2024
Full Text
View/download PDF

49. Comprehensive laboratory assessment of lonoctocog alfa versus octocog alfa in severe haemophilia A.

Author

Müller, Jens, Albert, Thilo, Klein, Claudia, Horneff, Silvia, Rühl, Heiko, Pötzsch, Bernd, Goldmann, Georg, Marquardt, Natascha, and Oldenburg, Johannes

Subjects
BLOOD coagulation factor VIII, BLOOD coagulation, HEMOPHILIACS, CHROMOGENIC compounds, THROMBIN, THROMBIN receptors
Abstract

Introduction: Lonoctocog alfa is a single‐chain factor VIII (FVIII) molecule with high binding affinity to von‐Willebrand‐factor. While it is well known that its plasma activity is underestimated by one‐stage clotting assays (OSCA), there is a lack of knowledge on the post‐infusion performance of lonoctocog alfa in global coagulation assays or its potential impact on the haemostatic balance in vivo. Aim: To characterize lonoctocog alfa versus octocog alfa in pre‐ and post‐infusion samples obtained from patients undergoing repeated investigation of incremental recovery (IR). Methods: Eighteen patients with severe haemophilia A (lonoctocog alfa: 10, octocog alfa: 8) were included. A panel of factor‐specific and global coagulation assays was applied, comprising a FVIII OSCA, two FVIII chromogenic substrate assays (CSA), rotational thrombelastography and thrombin generation (TG). Potential activation of coagulation was assessed by measuring plasma thrombin markers and levels of activated protein C. Results: Comparable IRs were found for lonoctocog alfa and octocog alfa (2.36 [IU/dL]/[IU/kg] vs. 2.55 [IU/dL]/[IU/kg], respectively). Lonoctocog alfa activities were found to be underestimated by the FVIII OSCA while also the two FVIII CSAs showed statistically significant assay discrepancies on lonoctocog alfa. Effects of both FVIII products on rotational thrombelastography were less distinct than those on TG parameters. No elevated pre‐ or significantly shifting post‐infusion plasma levels of coagulation biomarkers were detected. Conclusion: Lonoctocog alfa and octocog alfa showed comparable recovery and safety in vivo as well as similar impacts on TG in vitro. Observed assay discrepancies on lonoctocog alfa demonstrated variability of results also between different FVIII CSAs. [ABSTRACT FROM AUTHOR]

Published
2024
Full Text
View/download PDF

50. Moving towards Normalization of haemostasis and health equity: Evolving treatment goals for haemophilia A.

Author

Holme, Pål André, Blatný, Jan, Chowdary, Pratima, Lassila, Riitta, O'Connell, Niamh, Hermans, Cédric, Álvarez Román, María Teresa, Négrier, Claude, Coppola, Antonio, and Oldenburg, Johannes

Subjects
HEMOPHILIA treatment, JOINTS (Anatomy), QUALITY of life, LIFE expectancy, HEALTH equity
Abstract

Background: Treatment options for people with haemophilia are evolving at a rapid pace and a range of prophylactic treatment options using various technologies are currently available, each with their own distinct safety and efficacy profile. Treatment goals: The access to replacement therapy and prophylaxis has driven a dramatic reduction in mortality and resultant increase in life expectancy. Beyond this, the abolition of bleeds and preservation of joint health represent the expected, but rarely attained, goals of haemophilia treatment and care. These outcomes also do not address the complexity of health‐related quality of life impacted by haemophilia and its treatment. Conclusion: Capitalizing on the major potential of therapeutic innovations, 'Normalization' of haemostasis, as a concept, should include the aspiration of enabling individuals to live as normal a life as possible, free from haemophilia‐imposed limitations. To achieve this—being supported by the data reviewed in this manuscript—the concept of haemostatic and life Normalization needs to be explored and debated within the wider multidisciplinary teams and haemophilia community. [ABSTRACT FROM AUTHOR]

Published
2024
Full Text
View/download PDF

Catalog

Books, media, physical & digital resources
See catalog results