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224 results on '"Escuriola-Ettingshausen C"'

1. Bleeding phenotype according to factor level in 825 children with nonsevere hemophilia: data from the PedNet cohort

Author

Alvarèz Román, M.T., Benitez Hidalgo, O., Blatny, J., Bührlen, M., Carvalho, M., Chambost, H., Rosa Cid, A., Oudot, C., Escuriola-Ettingshausen, C., Fischer, K., Van Geet, C., Glosli, H., Gretenkort Andersson, N., Ljung, R., Königs, C., Koskenvuo, M., Male, C., Stamm Mikkelsen, T., Molinari, A., Motwani, J., Nolan, B., d’Oiron, R., Oldenburg, J., Olivieri, M., Pergantou, H., Pinto, F., Ranta, S., Kartal-Kaess, M., Zápotocká, E., Kenet, G., Carcao, M., Rivard, G., de Kovel, Marloes S., Escuriola-Ettingshausen, Carmen, Königs, Christoph, Ranta, Susanna, and Fischer, Kathelijn

Published
2024
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2. 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
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3. Bleeding phenotype according to factor level in 825 children with non-severe hemophilia; data from the PedNet cohort

Author

de Kovel, Marloes S., primary, Escuriola-Ettingshausen, Carmen, additional, Königs, Christoph, additional, Ranta, Susanna, additional, Fischer, Kathelijn, additional, Alvarèz Román, M.T., additional, Hidalgo, O Benitez, additional, Blatny, J., additional, Bührlen, M., additional, Carvalho, M., additional, Chambost, H., additional, Rosa Cid, A., additional, Oudot, C., additional, Escuriola-Ettingshausen, C., additional, Fischer, K., additional, Van GeetH Glosli, C., additional, Andersson, N Gretenkort, additional, Ljung, R., additional, Königs, C., additional, Koskenvuo, M., additional, Male, C., additional, Stamm Mikkelsen, T., additional, Molinari, A., additional, Motwani, J., additional, Nolan, B., additional, d’Oiron, R., additional, Oldenburg, J., additional, Olivieri, M., additional, Pergantou, H., additional, Pinto, F., additional, Ranta, S., additional, Kartal-Kaess, M., additional, Zápotocká, E., additional, Kenet, G., additional, Carcao, M., additional, and Rivard, G., additional

Published
2024
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6. PB0680 Clinical Characteristics, Therapy and Outcome of Children with Hemophilia B and Inhibitors: a PedNet Study

Author

Barg, A., primary, De Kovel, M., additional, Pergantou, H., additional, D'Oiron, R., additional, Nolan, B., additional, Escuriola-Ettingshausen, C., additional, Fischer, K., additional, Gretenkort Andersson, N., additional, Glosli, H., additional, Alvarez-Román, M., additional, Motwani, J., additional, Kenet, G., additional, and Male, C., additional

Published
2023
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7. OC 43.1 A Survey on Clinical Praxis in Initiating Emicizumab Prophylaxis in Previously Untreated Patients in the PedNet Centers

Author

Ranta, S., primary, Motwani, J., additional, Blatny, J., additional, Bührlen, M., additional, Carcao, M., additional, Chambost, H., additional, Escuriola-Ettingshausen, C., additional, Fischer, K., additional, Kartal-Kaess, M., additional, Kenet, G., additional, Male, C., additional, Nolan, B., additional, D’Oiron, R., additional, Olivieri, M., additional, Gretenkort Andersson, N., additional, and Koenigs, C., additional

Published
2023
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12. Population Pharmacokinetic Modeling Of On-Demand And Surgical Use Of Nonacog Beta Pegol (N9-GP) And rFIXFc Based Upon The paradigm 7 Comparative Pharmacokinetic Study

Author

Simpson ML, Kulkarni R, Escuriola Ettingshausen C, Medom Meldgaard R, Cooper DL, and Klamroth R

Subjects
n9-gp, rfixfc, population pk modeling, paradigm 7 trial, Diseases of the blood and blood-forming organs, RC633-647.5
Abstract

Mindy L Simpson,1 Roshni Kulkarni,2 Carmen Escuriola Ettingshausen,3 Rikke Medom Meldgaard,4 David L Cooper,5 Robert Klamroth6 1Pediatric Hematology/Oncology, Rush University Medical Center, Chicago, IL, USA; 2Department of Pediatrics and Human Development, Michigan State University, East Lansing, MI, USA; 3HZRM – Haemophilia Centre Rhein-Main, Frankfurt-Mörfelden, Mörfelden-Walldorf, Germany; 4Novo Nordisk A/S, Søborg, Denmark; 5Novo Nordisk Inc, Plainsboro, NJ, USA; 6Department for Internal Medicine, Vascular Medicine and Haemostaseology, Vivantes Klinikum, Berlin, GermanyCorrespondence: Mindy L SimpsonPediatric Hematology/Oncology, Rush University Medical Center, 1653 W. Congress Parkway, Chicago, IL 60612, USATel +1 312 563 3995Email Mindy_L_Simpson@rush.eduAim/objective: Understanding pharmacokinetic (PK) differences between standard and extended half-life (EHL) products is important, particularly for factor IX (FIX), where differences are more significant than for factor VIII. Two single-dose PK trials showed N9-GP achieves higher FIX levels and greater area-under-the-curve than pdFIX, rFIX, and rFIXFc through higher recovery and longer terminal half-life. In paradigm 7, N9-GP demonstrated consistently favorable PK characteristics compared with rFIXFc. Collins et al explored population PK differences between N9-GP and pdFIX/rFIX based upon paradigm 1 data. This analysis uses population PK models based upon the paradigm 7 trial.Methods: 15 patients (21–65 years) with hemophilia B received single 50-IU/kg doses of N9-GP and rFIXFc ≥21 days apart. A population PK model developed from single-dose PK profiles simulated plasma FIX activity following dosing for surgery and on-demand treatment of bleeds. Simulations explored doses and frequencies required to sustain target World Federation of Hemophilia (WFH) factor activity levels.Results: PK profiles of N9-GP and rFIXFc were described by one- and three-compartment models, respectively. Simulations predicted significantly reduced dosing frequency and consumption for N9-GP than rFIXFc. For severe bleeds, a single N9-GP dose (80 IU/kg) is sufficient to maintain WFH-recommended FIX levels, whereas multiple rFIXFc doses are required. For surgery, redosing in the first week with N9-GP is modeled at day 6 vs rFIXFc dosing at 6, 30, 54, 78, and 126 hrs. For life-threatening bleeds, N9-GP is required at days 0, 3, 6, 13, and 18 vs rFIXFc redosing after 6 hrs with 10 additional doses at 24-, 48-, and 72 hr intervals.Conclusion: PK modeling approaches based upon direct comparative studies offer insights into PK differences between EHL FIX products. Model simulations show N9-GP may allow on-demand treatment and perioperative management with 55–75% fewer injections and 65–74% lower overall factor concentrate consumption than rFIXFc.Keywords: N9-GP, rFIXFc, population PK modeling, paradigm 7 trial

Published
2019

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

Author

Fischer, Kathelijn, Kenet, Gili, Kurnik, Karin, Carcao, Manuel, Oldenburg, Johannes, Stamm-Mikkelsen, Torben, Cid Haro, Ana Rosa, Koskenvuo, Minna, Blatny, Jan, Königs, Christoph, 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, and Rivard, G

Abstract

•In 222 boys with severe hemophilia A and inhibitors of >5 BU, bleeding was reduced from 6.1 to 4.4 per year during ITI.•Before ITI, bleeding was independent of inhibitor titer; during ITI, bleeding increased with higher inhibitor titer and nondaily ITI.

Published
2024
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16. Encore - emicizumab prophylaxis for the treatment of infants with severe hemophilia A without Factor VIII inhibitors: results from the interim analysis of the HAVEN 7 study

Author

Escuriola-Ettingshausen, C, additional, Pipe, W S, additional, Collins, P, additional, Dhalluin, C, additional, Kenet, G, additional, Schmitt, C, additional, Buri, M, additional, Jimenez, V Yuste, additional, Peyvandi, F, additional, Young, G, additional, Oldenburg, J, additional, Mancuso, M E, additional, Kiialainen, A, additional, Chang, T, additional, Lehle, M, additional, and Fijnvandraat, K, additional

Published
2023
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17. EMIIL - Non-interventional study to investigate the effectiveness of Emicizumab under real-world conditions in pediatric, adolescent, adult and elderly patients with hemophilia A (PwHA) with and without FVIII inhibitors: an interim analysis

Author

Oldenburg, J, additional, Escuriola-Ettingshausen, C, additional, Fontana, P, additional, Wenning, S, additional, Pfrepper, C, additional, Heidersdorf, B, additional, Udvardi, P, additional, and Albisetti, M, additional

Published
2023
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19. Inhibitor development and mortality in non‐severe hemophilia A

Author

Eckhardt, C.L., Loomans, J.I., van Velzen, A.S., Peters, M., Mauser‐Bunschoten, E.P., Schwaab, R., Mazzucconi, M.G., Tagliaferri, A., Siegmund, B., Reitter‐Pfoertner, S.E., van der Bom, J.G., Fijnvandraat, K., Kamphuisen, P.W., Peerlinck, K., Oldenburg, J., Santagostino, E., Astermark, J., Eckhardt, C.L, van Velzen, A.S, Streefkerk, N., Loomans, J.L., van Eijkelenburg, A., Jansen, A.J., Kruijt, C.C., van Tienoven, B., van Baar, A.C.G., Corten, I.W., Meijer, K., Nijziel, M.R., Dors, N., Hamulyak, K., Beckers, E., Brons, P.P., Laros‐van Gorkom, B.A.P., van Heerde, W.L., Leebeek, F., Kruip, M., Cnossen, M.H., Mauser‐Bunschoten, E., Fischer, K., Smiers, F.J., Hermans, C., Klamroth, R., Escuriola‐Ettingshausen, C., Königs, C., Petrini, P., Holmström, M., Mäkipernaa, A., Male, C., Pabinger, I., Keenan, R.D., Liesner, R., Khair, K., Yee, T.T., Hart, D.P., Rangarajan, S., Mitchell, M., Thompson, G., Haya, S., Moret, A., Cid, A.R., Jimenez‐Yuste, V., Mancuso, M.E., Mazzuconni, M.G., Santoro, C., Morfini, M., Castaman, G., Schinco, P., Rivolta, G.F., Platokouki, H., and McRae, S.

Published
2015
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33. Hereditärer Faktor-VII-Mangel

Author

Lenz, E., Klarmann, D., Escuriola-Ettingshausen, C., Martinez-Saguer, I., Becker, S., Kreuz, W., Scharrer, Inge, editor, and Schramm, Wolfgang, editor

Published
1996
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37. First prospective report on immune tolerance in poor risk haemophilia A inhibitor patients with a single factor VIII/von Willebrand factor concentrate in an observational immune tolerance induction study

Author

Kreuz, W., Escuriola Ettingshausen, C., Vdovin, V., Zozulya, N., Plyushch, O., Svirin, P., Andreeva, T., Bubanská, E., Campos, M., Benedik-Dolničar, M., Jiménez-Yuste, V., Kitanovski, L., Klukowska, A., Momot, A., Osmulskaya, N., Prieto, M., Šalek, S. Z., Velasco, F., Pavlova, A., Oldenburg, J., Knaub, S., Jansen, M., Belyanskaya, L., and Walter, O.

Published
2016
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49. The changing face of immune tolerance induction in haemophilia A with the advent of emicizumab

Author

Carcao, M, Escuriola-Ettingshausen, C, Santagostino, E, Oldenburg, J, Liesner, R, Nolan, B, Batorova, A, Haya, S, Young, G, and Future Immunotolerance Treatment

Subjects
immune tolerance induction, inhibitor, emicizumab, factor VIII, haemophilia A, bypassing agents
Abstract

Introduction As a result of the new treatment paradigm that the haemophilia community will face with the availability of novel (non-factor) therapies, an updated consensus on ITI recommendations and inhibitor management strategies is needed. Aim The Future of Immunotolerance Treatment (FIT) group was established to contemplate, determine and recommend the best management options for patients with haemophilia A and inhibitors. Discussion and Conclusions Despite the considerable success of emicizumab in the management of inhibitor patients, the FIT group still sees the importance of eradicating inhibitors. However, the availability of emicizumab and other non-factor therapies in the future might impact greatly on how ITI is undertaken. Theoretically, concomitant use of emicizumab and FVIII might allow emicizumab to effectively prevent bleeding with lower dose ITI regimens. This might allow for the greater adoption of low-dose/low-frequency FVIII ITI regimens, which may result in a reduced need for central venous access devices while still maintaining a reasonable likelihood of ITI success. The FIT group proposes a new management algorithm for current ITI (without emicizumab) and a hypothetical new approach with the availability of emicizumab. As there are no published data regarding the concomitant use of emicizumab and FVIII for ITI, the FIT Expert group encourages the undertaking of properly conducted prospective studies to explore these approaches further.

Published
2019

50. Long-Term Outcomes with Subcutaneous C1-Inhibitor Replacement Therapy for Prevention of Hereditary Angioedema Attacks

Author

Zuraw, B, Katelaris, C, Sussman, G, Keith, PK, Yang, W, Hebert, J, Hanzlikova, J, Staubach-Renz, P, Martinez-Saguer, I, Magerl, M, Aygoren-Pursun, E, Farkas, H, Reshef, A, Kivity, S, Neri, S, Crisan, I, Caballero, T, Baeza, ML, Hernandez, MD, Li, H, Lumry, W, Bernstein, JA, Hussain, I, Anderson, J, Schwartz, LB, Jacobs, J, Manning, M, Levy, D, Riedl, M, Christiansen, S, Zuraw, BL, Cicardi, M, Craig, T, Longhurst, H, Grattan, C, Bork, K, Kreuz, W, Jacobs, I, Pragst, I, Herget, S, Dalton, S, Clement, C, Mycroft, S, Pawaskar, D, Zhang, Y, Machnig, T, Waldhauser, H, Feuersenger, H, Lawo, JP, Lang, D, Hoernlein, S, Mildenberger, M, Foeller, K, Ellis, F, Wood, D, Walsh, M, Qu, QL, Mueller-Stark, K, Feussner, A, Kalina, U, Ma, X, Rigsby, S, Scheffler, E, Fischer, JF, Kolb, C, Katelaris, CH, Frankum, B, Keat, K, Philip, B, Lee, JA, Urriola, N, Lee, MW, Sussman, GL, Levi, G, Gould, W, Ferrie, P, Rosenberg, E, Waserman, S, O'Quinn, J, Gagnon, R, Vachova, M, Stauerbach-Renz, P, Weber, A, Zimmer, S, Gilfert, T, Lang, B, Escuriola-Ettingshausen, C, Maurer, M, Metz, M, Schoepke, N, Altrichter, S, Hawro, T, Schwabe, D, Graff, J, Behrens, F, Kohm, M, Andarawewa, S, Temesszentandrasi, G, Kohalmi, VK, Kidon, M, Kadar, L, Benor, S, Bonanni, E, Wu, M, Zanichelli, A, Mansi, M, Rizzotto, A, Giardino, F, Fidone, F, Varga, M, Iftene, M, Badiu-Tisa, ID, Cabanas, R, Pedrosa, M, Rivero-Paparoni, D, Gomez-Traseira, C, Alvez, A, Phillips, E, Prieto, A, Zubeldia, J, Ibanez, E, Almero, R, Buckland, M, Grigoriadou, S, Manson, A, Yeatman, N, Laffan, J, Nasr, I, Ghurye, R, Rehman, T, Schaeffer, C, Ghaffari, G, Kelbel, T, Reddy, V, Buyantseva, L, Mende, C, Jose, J, Novchicht, T, Li, HH, Scarupa, M, Economides, A, White, M, Kaliner, M, Ward, C, Shaikh, S, Johnson, T, Kosh, L, Dauphin, P, Baker, J, Persons, S, Newman, A, Noonan, MJ, Lumry, WR, Poarch, KP, Tucker, J, Aguilar, D, Noth, D, Bernstein, J, Bernstein, D, Evans, S, Crawford, M, McGuckin, SD, McCollum, JR, Bradley, B, Wagner, C, Cartwright, A, Bonner, J, Soong, W, Sikora, M, Lemke, M, Luthin, P, Youngblood, B, DeBerry, E, Gilbert, E, Zhao, W, Ward, B, Alvarez, A, Kumar, S, Akl, E, Curl, J, Silva, K, Mostofi, T, Schultz, N, Manning, ME, Davis, A, Nelson, J, Levy, DS, Christiansen, SC, and COMPACT Investigators

Subjects
Hereditary angioedema, HAEGARDA, Long-term, Prophylaxis, Subcutaneous, Safety, C1-esterase inhibitor
Abstract

BACKGROUND: For the prevention of attacks of hereditary angioedema (HAE), the efficacy and safety of subcutaneous human C1-esterase inhibitor (C1-INH [SC]; HAEGARDA, CSL Behring) was established in the 16-week Clinical Study for Optimal Management of Preventing Angioedema with Low-Volume Subcutaneous C1-Inhibitor Replacement Therapy (COMPACT). OBJECTIVE: To assess the long-term safety, occurrence of angioedema attacks, and use of rescue medication with C1-INH(SC). METHODS: Open-label, randomized, parallel-arm extension of COMPACT across 11 countries. Patients with frequent angioedema attacks, either study treatment-naive or who had completed COMPACT, were randomly assigned (1:1) to 40 IU/ kg or 60 IU/kg C1-INH(SC) twice per week, with conditional uptitration to optimize prophylaxis (ClinicalTrials.gov registration no. NCT02316353). RESULTS: A total of 126 patients with a monthly attack rate of 4.3 in 3 months before entry in COMPACT were enrolled and treated for a mean of 1.5 years; 44 patients (34.9%) had more than 2 years of exposure. Mean steady-state C1-INH functional activity increased to 66.6% with 60 IU/kg. Incidence of adverse events was low and similar in both dose groups (11.3 and 8.5 events per patient-year for 40 IU/kg and 60 IU/kg, respectively). For 40 IU/kg and 60 IU/kg, median annualized attack rates were 1.3 and 1.0, respectively, and median rescue medication use was 0.2 and 0.0 times per year, respectively. Of 23 patients receiving 60 IU/kg for more than 2 years, 19 (83%) were attack-free during months 25 to 30 of treatment. CONCLUSIONS: In patients with frequent HAE attacks, long-term replacement therapy with C1-INH(SC) is safe and exhibits a substantial and sustained prophylactic effect, with the vast majority of patients becoming free from debilitating disease symptoms. (C) 2019 The Authors. Published by Elsevier Inc. on behalf of the American Academy of Allergy, Asthma & Immunology.

Published
2019

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