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Page 1
Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A.
Ozelo MC, Mahlangu J, Pasi KJ, Giermasz A, Leavitt AD, Laffan M, Symington E, Quon DV, Wang JD, Peerlinck K, Pipe SW, Madan B, Key NS, Pierce GF, O'Mahony B, Kaczmarek R, Henshaw J, Lawal A, Jayaram K, Huang M, Yang X, Wong WY, Kim B; GENEr8-1 Trial Group. Ozelo MC, et al. N Engl J Med. 2022 Mar 17;386(11):1013-1025. doi: 10.1056/NEJMoa2113708. N Engl J Med. 2022. PMID: 35294811 Clinical Trial.
Phase 3 Trial of Concizumab in Hemophilia with Inhibitors.
Matsushita T, Shapiro A, Abraham A, Angchaisuksiri P, Castaman G, Cepo K, d'Oiron R, Frei-Jones M, Goh AS, Haaning J, Hald Jacobsen S, Mahlangu J, Mathias M, Nogami K, Skovgaard Rasmussen J, Stasyshyn O, Tran H, Vilchevska K, Villarreal Martinez L, Windyga J, You CW, Zozulya N, Zulfikar B, Jiménez-Yuste V; explorer7 Investigators. Matsushita T, et al. N Engl J Med. 2023 Aug 31;389(9):783-794. doi: 10.1056/NEJMoa2216455. N Engl J Med. 2023. PMID: 37646676 Clinical Trial.
Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A.
Mahlangu J, Kaczmarek R, von Drygalski A, Shapiro S, Chou SC, Ozelo MC, Kenet G, Peyvandi F, Wang M, Madan B, Key NS, Laffan M, Dunn AL, Mason J, Quon DV, Symington E, Leavitt AD, Oldenburg J, Chambost H, Reding MT, Jayaram K, Yu H, Mahajan R, Chavele KM, Reddy DB, Henshaw J, Robinson TM, Wong WY, Pipe SW; GENEr8-1 Trial Group. Mahlangu J, et al. N Engl J Med. 2023 Feb 23;388(8):694-705. doi: 10.1056/NEJMoa2211075. N Engl J Med. 2023. PMID: 36812433 Clinical Trial.
Health-related quality of life following valoctocogene roxaparvovec gene therapy for severe hemophilia A in the phase 3 trial GENEr8-1.
O'Mahony B, Dunn AL, Leavitt AD, Peyvandi F, Ozelo MC, Mahlangu J, Peerlinck K, Wang JD, Lowe GC, Tan CW, Giermasz A, Tran H, Khoo TL, Cockrell E, Pepperell D, Chambost H, López Fernández MF, Kazmi R, Majerus E, Skinner MW, Klamroth R, Quinn J, Yu H, Wong WY, Robinson TM, Pipe SW. O'Mahony B, et al. Among authors: pepperell d. J Thromb Haemost. 2023 Dec;21(12):3450-3462. doi: 10.1016/j.jtha.2023.08.032. Epub 2023 Sep 6. J Thromb Haemost. 2023. PMID: 37678546 Clinical Trial.
Immunoglobulin replacement vs prophylactic antibiotics for hypogammaglobulinemia secondary to hematological malignancy.
McQuilten ZK, Weinkove R, Thao LTP, Crispin P, Degelia A, Dendle C, Gilbertson M, Johnston A, Keegan A, Pepperell D, Pullon H, Reynolds J, van Tonder T, Trotman J, Waters N, Wellard C, Weston H, Morrissey CO, Wood EM. McQuilten ZK, et al. Among authors: pepperell d. Blood Adv. 2024 Apr 9;8(7):1787-1795. doi: 10.1182/bloodadvances.2023011231. Blood Adv. 2024. PMID: 38592710 Free PMC article. Clinical Trial.
Efficacy and safety of avatrombopag in combination with immunosuppressive therapy in treatment-naïve and relapsed/refractory severe aplastic anaemia: protocol for the DIAAMOND-Ava-FIRST and DIAAMOND-Ava-NEXT Bayesian Optimal Phase II trials.
McQuilten Z, Heritier S, Fox L, Fox V, Young L, Blombery P, Cunningham I, Curnow J, Higgins A, Hiwase DK, Filshie R, Firkin F, Lacaze P, Mason K, Mills AK, Pepperell D, Patil S, Stevenson W, Szer J, Waters N, Wilson K, Ting S, Wood E. McQuilten Z, et al. Among authors: pepperell d. BMJ Open. 2024 Jan 18;14(1):e076246. doi: 10.1136/bmjopen-2023-076246. BMJ Open. 2024. PMID: 38238183 Free PMC article.
Immune thrombocytopenia following vaccination during the COVID-19 pandemic.
Choi PY, Hsu D, Tran HA, Tan CW, Enjeti A, Chen VMY, Chong BH, Curnow J, Pepperell D, Bird R. Choi PY, et al. Among authors: pepperell d. Haematologica. 2022 May 1;107(5):1193-1196. doi: 10.3324/haematol.2021.279442. Haematologica. 2022. PMID: 34435486 Free PMC article. No abstract available.
Economic evaluation: immunoglobulin vs prophylactic antibiotics in hypogammaglobulinemia and hematological malignancies.
Carrillo de Albornoz S, Higgins AM, Petrie D, Irving A, Fanning L, Weinkove R, Crispin P, Dendle C, Gilbertson M, Johnston A, Keegan A, Pepperell D, Pullon H, Reynolds J, van Tonder T, Trotman J, Waters N, Wellard C, Weston H, Morrissey CO, Wood EM, McQuilten ZK. Carrillo de Albornoz S, et al. Among authors: pepperell d. Blood Adv. 2024 May 14;8(9):2259-2267. doi: 10.1182/bloodadvances.2023012047. Blood Adv. 2024. PMID: 38484199 Free PMC article.
Assessment of immunological anti-platelet factor 4 antibodies for vaccine-induced thrombotic thrombocytopenia (VITT) in a large Australian cohort: A multicenter study comprising 1284 patients.
Favaloro EJ, Clifford J, Leitinger E, Parker M, Sung P, Chunilal S, Tran H, Kershaw G, Fu S, Passam F, Ahuja M, Ho SJ, Duncan E, Yacoub O, Tan CW, Kaminskis L, Modica N, Pepperell D, Ballard L, Clarke L, Lee CSM, Gardiner EE, Young-Ill Choi P, Tohidi-Esfahani I, Bird R, Brighton T, Chen VM. Favaloro EJ, et al. Among authors: pepperell d. J Thromb Haemost. 2022 Dec;20(12):2896-2908. doi: 10.1111/jth.15881. Epub 2022 Sep 29. J Thromb Haemost. 2022. PMID: 36107495 Free PMC article.
17 results