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This non-interventional study will prospectively collect detailed, high-quality documentation of bleeds, HRQoL, and safety in patients with hemophilia A with or without FVIII inhibitors treated according to local routine clinical practice (receiving FVIII replacement or bypassing agents as either episodic or prophylactic treatment). Actual patients will be enrolled from routine clinical practice in this observational study.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort A: Adults and Adolescents with FVIII Inhibitors | Adults and adolescents with hemophilia A of any severity with the presence of FVIII inhibitors will be observed. |
| |
| Cohort B: Children with FVIII Inhibitors | Children with hemophilia A of any severity with the presence of FVIII inhibitors will be observed. | ||
| Cohort C: Adults and Adolescents without FVIII Inhibitors | Adults and adolescents with severe hemophilia A without the presence of FVIII inhibitors will be observed. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Bypassing Agents | Drug | Episodic or prophylactic treatment with the use of bypassing agents must be documented for at least the last 6 months prior to the study. During the study, treatment for bleeds will be documented. The choice of coagulation product is at the discretion of the investigator according to local practice standards in this non-interventional study and there are no specific protocol-defined interventions. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Bleeds | Approximately 6 months (from Baseline until study completion) |
| Measure | Description | Time Frame |
|---|---|---|
| European Quality of Life-5 Dimensions (EQ-5D-5L) Questionnaire Score Among Adult and Adolescent Patients | Approximately 6 months (every 4 weeks from Baseline until study completion and on days that bleeds are reported) | |
| Hemophilia A-Specific Quality of Life (Haem-A-QoL) Questionnaire Score Among Adult Patients |
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Inclusion Criteria:
Exclusion Criteria:
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Patients with hemophilia A with or without inhibitors against FVIII under standard-of-care treatment are to be enrolled.
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| Name | Affiliation | Role |
|---|---|---|
| Clinical Trials | Hoffmann-La Roche | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Santa Monica Oncology Center | Santa Monica | California | 90403 | United States | ||
| University of Colorado Denver, Children's Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 32776489 | Derived | Oldenburg J, Shima M, Kruse-Jarres R, Santagostino E, Mahlangu J, Lehle M, Selak Bienz N, Chebon S, Asikanius E, Trask P, Mancuso ME, Jimenez-Yuste V, von Mackensen S, Levy GG. Outcomes in children with hemophilia A with inhibitors: Results from a noninterventional study. Pediatr Blood Cancer. 2020 Oct;67(10):e28474. doi: 10.1002/pbc.28474. Epub 2020 Aug 9. |
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| FVIII Replacement | Drug | Episodic or prophylactic treatment with the use of FVIII replacement must be documented for at least the last 6 months prior to the study. During the study, treatment for bleeds will be documented. The choice of coagulation product is at the discretion of the investigator according to local practice standards in this non-interventional study and there are no specific protocol-defined interventions. |
|
| Approximately 6 months (every 4 weeks from Baseline until study completion) |
| Hemophilia-Specific Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Score | Approximately 6 months (every 4 weeks from Baseline until study completion) |
| Aurora |
| Colorado |
| 80045 |
| United States |
| Georgetown Uni Medical Center; Lombardi Cancer Center | Washington D.C. | District of Columbia | 20007 | United States |
| Winship Cancer Institute | Atlanta | Georgia | 30322 | United States |
| Tulane Uni Health Sciences Center | New Orleans | Louisiana | 70112-2699 | United States |
| Children's Hospital of Michigan; Pediatrics | Detroit | Michigan | 48201 | United States |
| Cornell Univ Medical College; Hematology-Oncolog | New York | New York | 10021 | United States |
| Oregon Health & Science Uni ; Dept of Pediatrics | Portland | Oregon | 97201 | United States |
| University of Pittsburgh | Pittsburgh | Pennsylvania | 15213 | United States |
| Bloodworks Northwest (formerly Puget Sound Blood Center); Hemophilia | Seattle | Washington | 98104 | United States |
| Royal Prince Alfred Hospital; Haematology | Camperdown | New South Wales | 2050 | Australia |
| The Alfred Hospital, Melbourne; Thrombosis and Haemostasis Unit | Melbourne | Victoria | 3004 | Australia |
| Peking Union Medical College Hospital | Beijing | 100730 | China |
| Nanfang Hospital, Southern Medical University | Guangzhou | 510515 | China |
| Tianjin Institute of Hematology & Blood Diseases Hospital | Tianjin | 300020 | China |
| ICIC | San José | 1000 | Costa Rica |
| Universitätsklinikum Bonn (AöR); Inst. für Experimentelle Hämatologie u. Transfusionsmedizin (IHT) | Bonn | 53127 | Germany |
| IRCCS Ca' Granda Ospedale Maggiore Policlinico; Centro Emofilia e Trombosi "Angelo Bianchi e Bonomi" | Milan | Lombardy | 20122 | Italy |
| AOU Careggi; SOD Malattie Emorragiche | Florence | Tuscany | 50134 | Italy |
| Nagoya University Hospital | Aichi | 466-8560 | Japan |
| Hyogo College of Medicine Hospital | Hyōgo | 663-8501 | Japan |
| St. Marianna University School of Medicine Hospital | Kanagawa | 216-8511 | Japan |
| Hospital of the University of Occupational and Environmental Health,Japan | Kitakyushu-shi | 807-8556 | Japan |
| Nara Medical University Hospital | Nara | 634-8521 | Japan |
| Tokyo Medical University Hospital | Tokyo | 160-0023 | Japan |
| Uniwersyteckie Centrum Kliniczne; Klinika Hematologii i Transplantologii | Gdansk | 80-952 | Poland |
| SPSK Nr1 Klinika Hematoo&Transpl.Szpiku | Lublin | 20-081 | Poland |
| ALVAMED Lekarskie Gabinety Specjalistyczne | Poznan | 61-828 | Poland |
| Instytut Hematologii i Transfuzjologii; Klinika Zaburzeń Hemostazy i Chorób Wewnętrznych | Warsaw | 02-776 | Poland |
| Charlotte Maxeke Johannesburg Hospital; Haemophilia Comprehensive Care Center | Johannesburg | 2193 | South Africa |
| Severance Hospital | Seoul | 03722 | South Korea |
| Hospital Universitario la Paz; Servicio de Hematologia | Madrid | 28046 | Spain |
| Hospital Universitario Virgen del Rocio; Servicio de Hematologia | Seville | 41013 | Spain |
| Hospital Universitario la Fe; Servicio de Hematologia | Valencia | 46026 | Spain |
| National Taiwan Uni Hospital | Taipei | 100 | Taiwan |
| ID | Term |
|---|---|
| D006467 | Hemophilia A |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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