Post-Marketing Surveillance (Use-result Surveillance) of OBIZUR Injection [Susoctocog Alpha (Porcine Antihemophilic Factor VIII, Recombinant)] for the Approved Indications in South Korea

Acquired hemophilia A (AHA) is a rare bleeding condition which prevents blood clotting. Acquired means that people are not born with this condition or have a family history of blood clotting conditions. People living with AHA can have sudden and severe bleeding. They also have longer bleeding compared to people without AHA.
The main aim of the study is to learn how safe OBIZUR is in adults with AHA.
Other aims are to see how effective OBIZUR is to control bleeding and how treatment is used in a routine clinical practice setting.
The treatment of the participants will be determined by the treating physicians.
During the study, data already existing in the participants' medical record and new data will be collected.

Start Date31 Dec 2024

Sponsor / Collaborator

Takeda Pharmaceutical Co., Ltd.

JPRN-jRCT1031230480 / SuspendedNot ApplicableIIT

Multi-center clinical study to describe the relationship between anti-emicizumab antibodies and plasma emicizumab concentration and various coagulation tests

Start Date25 Nov 2023

Sponsor / Collaborator

Chugai Pharmaceutical Co., Ltd.

NCT05849740 / RecruitingPhase 4IIT

A Multicenter, Single-arm Exploratory Study of CD38 (Daratumumab) Monoclonal Antibody (Daratumumab) Combined Corticosteroid in the Treatment of Acquired Hemophilia A (AHA)

To evaluate the time of response, sustained remission rate and relapse rate of CD38 monoclonal antibody (Daratumumab) combined corticosteroid in the treatment of AHA. To evaluate the safety of CD38 monoclonal antibody in the treatment of AHA.

Start Date22 May 2023

Sponsor / Collaborator-

100 Clinical Results associated with Factor 8 Deficiency, Acquired

100 Translational Medicine associated with Factor 8 Deficiency, Acquired

0 Patents (Medical) associated with Factor 8 Deficiency, Acquired

896

Literatures (Medical) associated with Factor 8 Deficiency, Acquired

01 Nov 2024·European Journal of Haematology

Retroperitoneal and lower extremities muscle bleeding in acquired haemophilia A (AHA): Risk factors and implications in disability and survival

Article

Author: Pardos‐Gea, José ; Benítez, Olga

AbstractAimTo assess risk factors of retroperitoneal and lower extremity musculoskeletal bleed in acquired haemophilia (AHA) and perform an objective assessment of disability and influence on survival.MethodsWe included 49 patients with AHA from November 2017 to May 2023. The occurrence of any retroperitoneal or/and lower extremities bleeding manifestation was investigated. On clinical follow‐up, we search for compressive femoral neuropathy and quadriceps amyotrophy. The lower extremity functional scale (LEFS) was carried out one year after the last bleeding event in all AHA patients.ResultsA 61.2% of patients in our AHA cohort presented with any retroperitoneal and/or lower extremities musculoskeletal manifestation. Those patients had higher percentage of major bleeding EACH2/ISTH criteria (90% vs. 57%, p = .01), needs of blood transfusions (86% vs. 57% of patients, p = .03), and haemostatic by‐pass products (90% vs. 63%, p = .02). Hypertension (HR 2.6, 95% CI 1.1–5.9, p = .02), presence of autoimmune disease (HR 13, 95% CI 1.7–99, p = .01), and inhibitor level > 20 BU (HR 2.6 95% CI 1.0–6.8, p = .04) significantly predicted retroperitoneal/lower extremities clinical manifestations. Most frequent sequelae were quad atrophy (30.6%) and femoral nerve palsy (20.4%). Quad atrophy and LEFS scores under 50 were associated with increased mortality (HR 3, 95% CI 1.1–8.6 and HR 12, 95% CI 3.3–45, respectively).ConclusionAHA with retroperitoneal/lower extremities bleeding involvement is of greater severity and shows high disability and worst survival outcomes. Quadriceps atrophy and LEFS scale scoring under 50 predicted mortality in our AHA patients.

01 Nov 2024·Current Protein & Peptide Science

Emicizumab as a Promising Form of Therapy for Type A Hemophilia - A Review of Current Knowledge from Clinical Trials

Review

Author: Smakosz, Aleksander K. ; Zhao, Lin-Yong ; Kałuzińska-Kołat, Żaneta ; Kołat, Damian ; Grabowska, Katarzyna ; Grzelak, Michalina ; Gielecińska, Adrianna ; Płuciennik, Elżbieta ; Pasieka, Zbigniew ; Kciuk, Mateusz

:Hemophilia is a plasma bleeding disorder characterized by a deficiency of certain blood clotting factors. The most common forms of this disease, i.e., type A and type B, affect approximately 400,000 people worldwide. Without appropriate treatment ensuring the proper coagulation cascade, this disease may lead to serious disability. Minimizing patient discomfort is possible via replacement therapy, consisting of the substitution of a missing coagulation factor via intravenous administration. Frequent medication and the risk related to factor inhibitors are significant disadvantages, necessitating the improvement of current therapies or the development of novel ones. This review examines the humanized bispecific antibody Emicizumab which ensures hemostasis by mimicking the action of the coagulation factor VIII, a deficiency of which causes type A hemophilia. The paper outlines the topic and then summarizes available clinical trials on Emicizumab in type A hemophilia. Several interventional clinical trials have found Emicizumab to be effective in decreasing bleeding episodes and raising patient satisfaction among various hemophilia A populations. Current Emicizumab-related trials are forecast to be completed between 2024 and 2030, and in addition to congenital hemophilia A, the trials cover acquired hemophilia A and patients playing sports. Providing a more comprehensive understanding of Emicizumab may revolutionize the management of hemophilia type A and improve quality of life. Conclusively, Emicizumab is a gentler therapy owing to subcutaneous delivery and fewer injections, which reduces injection-site reactions and makes therapy less burdensome, ultimately decreasing hospital visits and indirect costs.

01 Oct 2024·International Journal of Hematology

Efficacy and safety of recombinant porcine factor VIII in Japanese patients with acquired hemophilia A

Article

Author: Mizuta, Yuki ; Sakaida, Emiko ; Nogami, Keiji ; Kitagawa, Tadayuki ; Kikuchi, Takahide ; Matsushita, Tadashi ; Ogawa, Yoshiyuki ; Seki, Yoshinobu ; Miyaguchi, Yasuo ; Takemura, Kazuhiko

AbstractAcquired hemophilia A (AHA) is a rare bleeding disorder caused by autoantibodies inhibiting human factor VIII (hFVIII). This phase II/III open-label study evaluated the safety and efficacy of recombinant porcine factor VIII (rpFVIII, susoctocog alfa) in adults with AHA and severe bleeding episodes in Japan (NCT04580407). The initial rpFVIII dose was 200 U/kg, with subsequent doses based on clinical measures including plasma FVIII activity. The primary efficacy endpoint was the proportion of severe bleeding episodes with a positive response to rpFVIII therapy 24 h after treatment initiation. Five patients were eligible for, and completed, rpFVIII treatment (age group: 60s–80s; median hFVIII inhibitor: 52 BU/mL; porcine FVIII [pFVIII] inhibitor: 3/5 patients). The median (range) total dose/patient was 548.4 (198–1803) U/kg with a median 3.0 infusions/patient. All patients responded positively to rpFVIII therapy at 24 h regardless of baseline pFVIII inhibitor status. rpFVIII treatment was well tolerated with no adverse events of special interest such as thromboembolic events or de novo pFVIII inhibitors. This study supports the use of rpFVIII as a novel therapy in the clinical management of patients with AHA in Japan. rpFVIII was approved for treating bleeding episodes in adults with AHA in Japan in 2024.

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