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1دورية أكاديمية
المؤلفون: Abou-Ismail, Mouhamed Yazan, Zhang, Chong, Presson, Angela P., Chaturvedi, Shruti, Antun, Ana G., Farland, Andrew M., Woods, Ryan, Metjian, Ara, Park, Yara A., de Ridder, Gustaaf, Gibson, Briana, Kasthuri, Raj S., Liles, Darla K., Akwaa, Frank, Clover, Todd, Kreuziger, Lisa Baumann, Sridharan, Meera, Go, Ronald S., McCrae, Keith R., Upreti, Harsh Vardhan, Gangaraju, Radhika, Kocher, Nicole K., Zheng, X. Long, Raval, Jay S., Masias, Camila, Cataland, Spero R., Johnson, Andrew D., Davis, Elizabeth, Evans, Michael D., Mazepa, Marshall, Lim, Ming Y.
المساهمون: National Heart, Lung, and Blood Institute, National Center for Advancing Translational Sciences, National Institutes of Health
المصدر: Research and Practice in Thrombosis and Haemostasis ; volume 8, issue 3, page 102388 ; ISSN 2475-0379
مصطلحات موضوعية: Hematology
الإتاحة: https://doi.org/10.1016/j.rpth.2024.102388Test
https://api.elsevier.com/content/article/PII:S2475037924000773?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:S2475037924000773?httpAccept=text/plainTest -
2دورية
المؤلفون: Metjian, Ara, Abrams, Charles S
المصدر: Biologics (Online), 2009; v.3:p.499-513
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3دورية أكاديمية
المؤلفون: Tarantino, Michael D., Hardesty, Brandon, Metjian, Ara, Ortel, Thomas L., Chen, Jie, Badejo, Kayode, Ma, Alice, Cuker, Adam, Rajasekhar, Anita, Friedman, Kenneth D., Janbain, Maissaa
المصدر: Haemophilia ; volume 29, issue 5, page 1259-1268 ; ISSN 1351-8216 1365-2516
الوصف: Introduction Recombinant porcine factor VIII (rpFVIII, susoctocog alfa) is indicated for the treatment of bleeding episodes in adults with acquired haemophilia A (AHA). Aim To provide long‐term real‐world safety and effectiveness data for rpFVIII in the management of AHA bleeding episodes. Methods US PASS (NCT02610127) was a multicentre, uncontrolled, open‐label, post‐marketing safety surveillance study conducted in adults with AHA. Data were collected retrospectively or prospectively for 180 days after rpFVIII treatment. The primary outcome was the incidence of treatment‐related serious adverse events (SAEs). Secondary outcomes included haemostatic effectiveness of rpFVIII and rpFVIII utilization. Results Fifty‐three patients were enrolled from December 2015 to June 2019 (prospective, n = 30; retrospective, n = 23). Six patients experienced seven treatment‐related SAEs (incidence 12.0%). The most common treatment‐related SAE was FVIII inhibition (inhibiting antibodies to rpFVIII; incidence 8.0%, 95% CI: 2.2–19.2). Five patients reported seven thromboembolic events; one was an SAE and possibly related to rpFVIII. Of bleeding events treated with rpFVIII, 80.3% (57/71) of bleeds resolved with rpFVIII. The median (range) dose of rpFVIII per infusion was 50 (10–300) units/kg, with a median (range) of 6.0 (1–140) infusions and a median (range) time from bleed onset to bleed resolution of 14.0 (2.0–132.7) days. Conclusion In this real‐world study of rpFVIII for AHA, no new safety signals were identified compared with previous clinical trial findings. Eighty percent of bleeds resolved with rpFVIII treatment.
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4دورية أكاديمية
المؤلفون: KREMER HOVINGA, Johanna, DE LA RUBIA, Javier, PAVENSKI, Katerina, METJIAN, Ara, KNÖBL, Paul, PEYVANDI, Flora, CATALAND, Spero, COPPO, Paul, KHAN, Umer, MENAPACE, Laurel A., PAULA MARQUES, Ana, GUNAWARDENA, Sriya, SCULLY, Marie
المصدر: Hematology, Transfusion and Cell Therapy ; volume 45, page S16 ; ISSN 2531-1379
مصطلحات موضوعية: Hematology, Immunology and Allergy
الإتاحة: https://doi.org/10.1016/j.htct.2023.09.028Test
https://api.elsevier.com/content/article/PII:S2531137923002043?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:S2531137923002043?httpAccept=text/plainTest -
5دورية أكاديمية
المؤلفون: Scully, Marie, de la Rubia, Javier, Pavenski, Katerina, Metjian, Ara, Knöbl, Paul, Peyvandi, Flora, Cataland, Spero, Coppo, Paul, Kremer Hovinga, Johanna A, Minkue Mi Edou, Jessica, De Passos Sousa, Rui, Callewaert, Filip, Gunawardena, Sriya, Lin, Julie
المصدر: Scully, Marie; de la Rubia, Javier; Pavenski, Katerina; Metjian, Ara; Knöbl, Paul; Peyvandi, Flora; Cataland, Spero; Coppo, Paul; Kremer Hovinga, Johanna A; Minkue Mi Edou, Jessica; De Passos Sousa, Rui; Callewaert, Filip; Gunawardena, Sriya; Lin, Julie (2022). Long-term follow-up of patients treated with caplacizumab and safety and efficacy of repeat caplacizumab use: Post-HERCULES study. Journal of thrombosis and haemostasis, 20(12), pp. 2810-2822. Wiley-Blackwell 10.1111/jth.15892
مصطلحات موضوعية: 610 Medicine & health, 340 Law
الوصف: INTRODUCTION Caplacizumab demonstrated efficacy and safety in patients with immune-mediated thrombotic thrombocytopenic purpura (iTTP) in the Phase 3 HERCULES trial. However, data on long-term outcomes following caplacizumab treatment are limited. OBJECTIVES The post-HERCULES trial (NCT02878603) evaluated long-term outcomes of patients with iTTP treated with caplacizumab in HERCULES and safety and efficacy of repeated caplacizumab use. PATIENTS/METHODS Over 3 years' follow-up, patients could receive open-label caplacizumab with therapeutic plasma exchange (TPE) and immunosuppressive therapy (IST) in case of recurrence. Adverse events (AEs) were assessed during the overall study period (intention-to-observe [ITO] population) and during recurrences (recurrence population). TTP-related events (TTP-related death, recurrence, major thromboembolic events) were assessed in the efficacy ITO population (patients without recurrence during HERCULES or before post-HERCULES). RESULTS Among 104 enrolled patients, incidences of AEs and serious AEs were similar between patients who had received caplacizumab+TPE+IST during HERCULES (n=75) and those treated with placebo+TPE+IST (placebo; n=29). TTP-related events occurred in 8% of patients (4/49) randomized to caplacizumab during HERCULES versus 38% (11/29) randomized to placebo. Nineteen patients had ≥1 recurrence; 13 of these were treated with caplacizumab. First recurrence episode was resolved or resolving for all patients treated with caplacizumab, including 9 patients with repeat caplacizumab use. All second recurrences (6/6) were resolved. Safety profile of caplacizumab for treatment of recurrence was consistent with HERCULES; most bleeding events were non-serious. No major cases of organ dysfunction were observed. CONCLUSIONS Long-term follow-up supports the safety and efficacy of caplacizumab for iTTP and its repeated use for recurrences.
وصف الملف: application/pdf
العلاقة: https://boris.unibe.ch/173249Test/
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6دورية أكاديمية
المؤلفون: Pektaş, Özgür, Scully, Marie, Rubia, Javier de la, Pavenski, Katerina, Metjian, Ara, Knöbl, Paul, Peyvandi, Flora, Cataland, Spero, Coppo, Paul, Hovinga, Johanna A. Kremer, Edou, Jessica Minkue Mi, Sousa, Rui de Passos, Gunawardena, Sriya, Lin, Julie
المصدر: Hematology, Transfusion and Cell Therapy ; volume 44, page S33-S34 ; ISSN 2531-1379
مصطلحات موضوعية: Hematology, Immunology and Allergy
الإتاحة: https://doi.org/10.1016/j.htct.2022.09.1250Test
https://api.elsevier.com/content/article/PII:S2531137922013748?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:S2531137922013748?httpAccept=text/plainTest -
7دورية
المؤلفون: Abou-Ismail, Mouhamed Yazan, Zhang, Chong, Presson, Angela P., Chaturvedi, Shruti, Antun, Ana G., Farland, Andrew M., Woods, Ryan, Metjian, Ara, Park, Yara A., de Ridder, Gustaaf, Gibson, Briana, Kasthuri, Raj S., Liles, Darla K., Akwaa, Frank, Clover, Todd, Baumann Kreuziger, Lisa, Sridharan, Meera, Go, Ronald S., McCrae, Keith R., Upreti, Harsh Vardhan, Gangaraju, Radhika, Kocher, Nicole K., Zheng, X. Long, Raval, Jay S., Masias, Camila, Cataland, Spero R., Johnson, Andrew D., Davis, Elizabeth, Evans, Michael D., Mazepa, Marshall, Lim, Ming Y.
المصدر: Blood Advances; February 2024, Vol. 8 Issue: 3 p620-623, 4p
مستخلص: [Display omitted]
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8دورية أكاديمية
المؤلفون: Josephson, Cassandra D., Goldstein, Stuart, Askenazi, David, Cohn, Claudia S., Spinella, Philip C., Metjian, Ara, Fasano, Ross M., Music‐Aplenc, Lejla
المساهمون: Octapharma
المصدر: Transfusion ; volume 62, issue 2, page 396-405 ; ISSN 0041-1132 1537-2995
الوصف: Background This study investigated the real‐world safety and tolerability of solvent/detergent‐treated (S/D) plasma for pediatric patients requiring therapeutic plasma exchange (TPE). Study design and methods LAS‐213 was a multicenter, open‐label, interventional, phase 4 study. Patients (≥2 to ≤20 years) receiving TPE therapy were eligible. A total plasma volume of 40–60 ml/kg was recommended, with an infusion rate not exceeding 0.020–0.025 citrate/kg body weight/min (<1 ml/kg body weight/min). The primary endpoint was assessment of safety, monitoring the following: serious adverse events (SAEs), adverse drug reactions (ADRs), thrombotic events (TEs), thromboembolic events (TEEs), and specific laboratory tests. Results In total, 41 children (2 to <12 years [ n = 15]; 12 to <17 years [ n = 13]; ≥17 years [ n = 13]) underwent 102 TPEs with a total of 135,137 ml of S/D plasma exchanged. Each patient group received between 1 and 6 TPEs (mean: 2.5 TPEs). Actual dose administered per TPE was 4–72 ml/kg (mean: 28.6 ml/kg), with a mean total volume of 1324.9 ml (range: 113–4000 ml). Overall safety was excellent for 96/102 (94.0%) TPEs. Six TPEs had a “moderate” safety profile for four patients experiencing eight ADRs. Of these, seven were mild in intensity and one (pyrexia) was moderate, all resolving by study end. Mild citrate toxicity ( n = 2) was the most common ADR. One SAE was reported but was unrelated to the study drug. No TEs, TEEs, or changes in laboratory safety parameters were reported. Conclusion S/D plasma was well tolerated and demonstrated favorable safety, supporting the use of S/D plasma for TPE in pediatrics.
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9دورية أكاديمية
المؤلفون: Peyvandi, Flora, Cataland, Spero, Scully, Marie, Coppo, Paul, Knoebl, Paul, Hovinga, Johanna A. Kremer, Metjian, Ara, de la Rubia, Javier, Pavenski, Katerina, Edou, Jessica Minkue Mi, Callewaert, Filip, De Winter, Hilde
المصدر: Hematology, Transfusion and Cell Therapy ; volume 43, page S46-S47 ; ISSN 2531-1379
مصطلحات موضوعية: Hematology, Immunology and Allergy
الإتاحة: https://doi.org/10.1016/j.htct.2021.10.1047Test
https://api.elsevier.com/content/article/PII:S2531137921011949?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:S2531137921011949?httpAccept=text/plainTest -
10دورية أكاديمية
المؤلفون: Knoebl, Paul, Cataland, Spero, Peyvandi, Flora, Coppo, Paul, Scully, Marie, Kremer Hovinga, Johanna A., Metjian, Ara, de la Rubia, Javier, Pavenski, Katerina, Minkue Mi Edou, Jessica, De Winter, Hilde, Callewaert, Filip
المصدر: Knoebl, Paul; Cataland, Spero; Peyvandi, Flora; Coppo, Paul; Scully, Marie; Kremer Hovinga, Johanna A.; Metjian, Ara; de la Rubia, Javier; Pavenski, Katerina; Minkue Mi Edou, Jessica; De Winter, Hilde; Callewaert, Filip (2020). Efficacy and safety of open-label caplacizumab in patients with exacerbations of acquired thrombotic thrombocytopenic purpura in the HERCULES study. Journal of thrombosis and haemostasis, 18(2), pp. 479-484. Wiley-Blackwell 10.1111/jth.14679
مصطلحات موضوعية: 610 Medicine & health
الوصف: BACKGROUND Acquired thrombotic thrombocytopenic purpura (aTTP) is a rare, life-threatening autoimmune thrombotic microangiopathy. Caplacizumab, an anti-von Willebrand Factor Nanobody® , is effective for treating aTTP episodes and is well tolerated. OBJECTIVES AND METHODS In the phase 3 HERCULES trial (NCT02553317), patients with aTTP received double-blind caplacizumab or placebo during daily therapeutic plasma exchange (TPE) and for ≥30 days thereafter. Patients who experienced an exacerbation while on blinded study drug treatment switched to receive open-label caplacizumab plus re-initiation of daily TPE. Exacerbations were defined as recurrence of disease occurring within 30 days after cessation of daily TPE. RESULTS Thirty-one patients (placebo, n = 28; caplacizumab, n = 3) had an exacerbation during double-blind treatment. Twenty-eight patients switched to open-label caplacizumab (placebo, n = 26; caplacizumab, n = 2); the three others discontinued upon exacerbation. Median time to platelet count response (≥150 x 109 /L) was 3.49 days upon receiving caplacizumab. There were no deaths. During open-label treatment, further exacerbation or a major thromboembolic event (vena cava thrombosis) was experienced by one patient (3.6%) each. Consistent with the double-blind phase, the most frequent treatment-emergent adverse events were catheter site hemorrhage (28.6%), headache (21.4%), and epistaxis (17.9%). CONCLUSIONS These results suggest that caplacizumab was efficacious and well tolerated in patients with aTTP who experienced a disease exacerbation during double-blind treatment in HERCULES.
وصف الملف: application/pdf
العلاقة: https://boris.unibe.ch/134991Test/
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