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TSUBASA Study: An Observational Study of Emicizumab to Evaluate Physical Activity, Bleeding Events, Quality of Daily Life, and Safety in People with Hemophilia A without FVIII Inhibitors

1Nara Medical University, Department of Pediatrics, Nara, Japan, 2Hiroshima University Hospital, Division of Transfusion Medicine/Hemophilia Treatment Center, Hiroshima, Japan, 3Hyogo College of Medicine, Division of Hematology, Department of Respiratory Medicine and Internal Medicine, Hyogo, Japan, 4Ogikubo Hospital, Department of Blood Coagulation, Tokyo, Japan, 5St. Marianna University School of Medicine, Department of Pediatrics, Kanagawa, Japan, 6Nagoya University Hospital, Department of Transfusion Medicine, Aichi, Japan, 7Institute of Medical Science, University of Tokyo, Center for Translational Research/Division of Advanced Medicine Promotion, Tokyo, Japan, 8Chugai Pharmaceutical Co., Ltd, Tokyo, Japan, 9Tokyo Medical University, Department of Laboratory Medicine, Tokyo, Japan

Abstract Number: PO146

Meeting: ISTH 2021 Congress

Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical

Background: Several clinical studies conducted worldwide have shown that Emicizumab is effective and tolerable in people with hemophilia A (PwHA) regardless of its use in PwHA with/without factor VIII (FVIII) inhibitors. Although physical activity is recommended for PwHA, little information is available on the relationship of bleeding to physical activity in PwHA on Emicizumab. Additionally, the experience with administering Emicizumab to infant patients is limited; thus, more safety data should be accumulated in the real-world setting.

Aims: To evaluate the relationship between exercise status and bleeding, the quality and content of daily life, and the safety of Emicizumab in PwHA without FVIII inhibitors.

Methods: We were enrolling PwHA without FVIII inhibitors scheduled to begin prophylactic treatment with Emicizumab. During 97 weeks after the enrollment, the status of bleeding in patients treated with Emicizumab that are attributable to exercise, the quality and content of daily life and physical activity data extracted from electronic patient-reported outcomes systems and wearable activity tracker will be evaluated by acquiring patient-reported exercise status.

 Study design

Results: Until October 2020, 65 patients (including <2 years old: 8, 2-5 years old: 4, 6-17 years old: 10, ≥18 years old: 43) were enrolled. Regarding the severity, 56 patients were severe and 8 patients were moderate, 1 patient were unknown. And 30 patients, 168 exercise events were reported from ≥6 years old patients evaluated for the exercise status. At this interim analysis, patient characteristics, status of patient exercise and bleeding, and safety information are reported.

Conclusions: This study will clarify the effects of Emicizumab on the lives of hemophilia patients and provide important information for selecting treatments for PwHA without FVIII inhibitors. Acquiring information on demographically diverse patients (especially infant patients) in clinical settings provide particularly valuable data for starting or continuing Emicizumab treatment.

To cite this abstract in AMA style:

Nogami K, Fujii T, Sawada A, Nagao A, Nagae C, Suzuki N, Nojima M, Nosaka D, Nakamura T, Yoshida S, Amano K. TSUBASA Study: An Observational Study of Emicizumab to Evaluate Physical Activity, Bleeding Events, Quality of Daily Life, and Safety in People with Hemophilia A without FVIII Inhibitors [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/tsubasa-study-an-observational-study-of-emicizumab-to-evaluate-physical-activity-bleeding-events-quality-of-daily-life-and-safety-in-people-with-hemophilia-a-without-fviii-inhibitors/. Accessed November 29, 2023.

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ISTH Congress Abstracts - https://abstracts.isth.org/abstract/tsubasa-study-an-observational-study-of-emicizumab-to-evaluate-physical-activity-bleeding-events-quality-of-daily-life-and-safety-in-people-with-hemophilia-a-without-fviii-inhibitors/