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| Name | Class |
|---|---|
| Baxalta Innovations GmbH, now part of Shire | INDUSTRY |
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The purpose of the study is to document the natural history of hemophilia A disease and long-term outcomes in terms of effectiveness, safety and quality of life in participants receiving Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method (rAHF-PFM) or Antihemophilic Factor (Recombinant) - Pegylated (rAHF-PEG) in routine clinical practice
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| rAHF-PFM | Participants treated with rAHF-PFM alone |
| |
| rAHF-PEG | Participants treated with rAHF-PEG alone |
| |
| rAHF-PFM then rAHF-PEG | Participants treated with rAHF-PFM and subsequently switched to rAHF-PEG |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ADVATE | Biological | Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method |
|
| Measure | Description | Time Frame |
|---|---|---|
| Joint Health Outcomes - Assessed by Physical Exam Using Only the Pain, Bleeding, and Physical Exam Parameters of the Gilbert Scale | The World Federation of Hemophilia developed a musculoskeletal evaluation system, commonly referred to as the Gilbert test, to measure hemophilia joint health status.The Gilbert test needs to be performed in the absence of acute bleed, acute pain, and acute inflammation into the evaluated joint. Four parameters are used in each Gilbert test: pain (score: 0-3), bleeding (score: 0-3), physical exam (score: 0-12), and X-ray evaluation (score: 0-13) Scores of 0, represent no pain, no bleeding, no physical exam issues, and/or no x-ray issues. Higher scores for each of these categories represents worsening conditions. | Up to approximately 12 years |
| Measure | Description | Time Frame |
|---|---|---|
| Annualized Bleed Rate, All Joints | The annualized bleed rate for all joints will be calculated per participant and summarized over the set of available participants with a minimum observation period of 90 days per treatment regimen. | Annual/Interval visits:- Up to 8 years if rAHF-PFM alone- Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years- Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit |
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Inclusion Criteria:
Exclusion Criteria:
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The study population will include male and female participants of any race and age who have a diagnosis of hemophilia A (Factor VIII (FVIII) =5%). Participants must have been prescribed rAHF-PFM or rAHF-PEG for the management of hemophilia A by the treating physician prior to the decision to enroll in the study.
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Royal Prince Alfred Hospital | Camperdown | New South Wales | 2050 | Australia | ||
| South Metropolitan Health Service trading as Fiona Stanley Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 38371314 | Derived | Ozelo MC, Hermans C, Carcao M, Guillet B, Gu J, Guerra R, Tang L, Khair K. The effectiveness and safety of octocog alfa in patients with hemophilia A: up to 7-year follow-up of the real-world AHEAD international study. Ther Adv Hematol. 2024 Feb 15;15:20406207231218624. doi: 10.1177/20406207231218624. eCollection 2024. |
| Label | URL |
|---|---|
| To obtain more information on the study, click here/on this link | View source |
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Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
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IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
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| ADYNOVI | Biological | Antihemophilic Factor (Recombinant) Pegylated |
|
|
| Annualized Bleed Rate, All Bleeds | The annualized bleed rate for all bleeds will be calculated per participant and summarized over the set of available participants.with a minimum observation period of 90 days per treatment regimen. | Annual/Interval visits:- Up to 8 years if rAHF-PFM alone- Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years- Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit |
| Annualized bleed rate, pre-existing target joints at baseline | The annualized bleed rate for pre-existing target joints at baseline will be calculated per participant and summarized over the set of available participants with a minimum observation period of 90 days per treatment regimen. | Screening visit; Annual/Interval visits:- Up to 8 years if rAHF-PFM alone- Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years- Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit |
| Incidence of New Target Joints | The incidence of new target joints will be calculated as the total number of new target joints in all participants divided by the total number of observation days. | Screening visit; Annual/Interval visits:- Up to 8 years if rAHF-PFM alone- Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years- Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit |
| Status of joint health by X-ray by Pettersson scale | The status of joint health by X-ray by Pettersson score will be summarized for each observational year. | Screening visit; Annual/Interval visits:- Up to 8 years if rAHF-PFM alone- Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years- Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit. |
| Status of Joint Health by Magnetic Resonance Imaging (MRI) Scoring System- Using The Lund Scoring System (LSS) | LSS score format= A(e:s:h). Sum of values for Subchondral Cyst (score: 1-6), irregularity/erosion of Subchondral Cortex (score: 1-4), and Chondral Destruction (score: 1-6) gives value for the A component of score. e, s, h components represent effusion/hemarthrosis, hypertrophic synovial, & hemosiderin deposition (score: 0-4 for each). Max. score is 16(4:4:4). Subchondral Cyst:
Subchondral Cortex
Chondral Destruction
Effusion/hemarthrosis (e): Hypertrophic synovial (s): Hemosiderin (h): (0-4 for each):
| Screening visit; Annual/Interval visits:- Up to 8 years if rAHF-PFM alone- Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years- Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit |
| Status of joint health using the Hemophilia Joint Health Score (HJHS) | The International Prophylaxis Study Group (IPSG) developed a scoring system for musculoskeletal evaluation, the HJHS, optimized for use in children with no or minimal joint disease. The HJHS includes the following parameters: swelling, duration of swelling, muscle atrophy, joint pain, crepitus on motion, flexion loss, extension loss, strength and global gait. | Screening visit; Annual/Interval visits:- Up to 8 years if rAHF-PFM alone- Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years- Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit |
| Overall effectiveness assessment for prophylaxis therapy |
| Annual/Interval visits:- Up to 8 years if rAHF-PFM alone;- Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years;- Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit |
| Compliance with the dosing prescribed and its relationship with effectiveness | Evaluation of patients´ compliance to prescribed prophylactic treatment will be performed by the treating physician. Compliance will be categorized according to a 4-point table (Highly compliant, Fairly compliant, Moderately compliant, Poorly compliant) | Annual/Interval visits:- Up to 8 years if rAHF-PFM alone;- Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years;- Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit |
| Overall effectiveness assessment for on-demand treatment |
| Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit |
| Global effectiveness assessment for on-demand treatment |
| Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit |
| Number of rAHF-PFM or rAHF-PEG units required for bleed cessation | Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method (rAHF-PFM) Antihemophilic Factor (Recombinant) - Pegylated (rAHF-PEG) | Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit |
| Number of rAHF-PFM or rAHF-PEG infusions required for bleed cessation | Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method (rAHF-PFM) Antihemophilic Factor (Recombinant) - Pegylated (rAHF-PEG) | Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit |
| Incidence of target joint intervention, including surgery, radiosynovectomy, and chemosynovectomy | Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit |
| Incidence of pseudo tumor development | Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visit |
| Quality of Life: HAL questionnaire - for adult patients | The the lHAL measures activities involving the upper extremities, basic activities involving ower extremities and complex activities involving the lower extremities as well as an overall physical activity score for adults. | Enrollment visit; Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visit |
| Quality of Life: SF-12v2 questionnaire - for adult patients | The SF-12v2 measures generic health-related quality of life for adults. | Enrollment visit; Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visit |
| Quality of Life: EQ-5D questionnaire - for adult patients | The EQ-5D measures health utility in adult participants. | Enrollment visit;Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visit |
| Quality of Life: PedHAL questionnaire - for pediatric patients | The PedHAL measures activities involving the upper extremities, basic activities involving the lower extremities and complex activities involving the lower extremities as well as an overall physical activity score for children. For participants 4-13 years of age: - PedHAL (parent version) For participants 14-17 years of age: - PedHAL (child version) | Enrollment visit; Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visit |
| Quality of Life: SF-10 questionnaire - for pediatric patients | The SF-10 measures generic health-related quality of life for children and is parent-completed. | Enrollment visit; Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visit |
| Quality of Life: EQ-5D (14 and up) questionnaire - for pediatric patients | The EQ-5D measures health utility in subjects aged 14 and up. | Enrollment visit; Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visit |
| Chronic pain associated with hemophilia, as measured over a period of 4 weeks on an annual basis, using the visual analog scale (VAS) | The VAS assesses the pain using a scale of 0 (no pain) to 10 (unbearable pain). During screening visit and on an annual basis, the investigators shall ask participants to rate the average level of chronic pain associated with hemophilia over the period of 4 weeks prior to visit date using the VAS. | Enrollment visit; Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visit |
| Acute pain associated with hemophilia, as measured with individual bleeding episodes, using the visual analog scale (VAS) | The VAS assesses the pain using a scale of 0 (no pain) to 10 (unbearable pain). Participants will be asked to provide ratings on level of acute pain associated with each bleeding episode using the VAS. The VAS scores will be recorded in the participant diary. | Enrollment visit; Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visit |
| Number of days lost from school or work due to bleeding episodes | Enrollment visit; Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visit |
| Modalities of switching from a standard FVIII product to rAHF-PEG - 1 | Difference in number of weekly prophylactic infusions between previous regimen and rAHF-PEG | Enrollment visit; Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visit |
| Modalities of switching from a standard FVIII product to rAHF-PEG - 2 | Difference in number of weekly doses between previous regimen and rAHFPEG | Enrollment visit; Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visit |
| Incidence of Inhibitors in Previously Treated Patients (PTPs) with Factor VIII (FVIII) Levels Lesser than (<)1%, Lesser Than or Equal to (<=) 2%, and <= 5% without history of inhibitor | Throughout the study period of up to approximately: 8 years (rAHF-PFM alone), or 12 years (rAHF-PEG alone or after receiving rAHF-PFM) |
| Incidence of Inhibitors in Previously Treated Patients (PTPs) with Factor VIII (FVIII) Levels <1%, <=2%, and <= 5% with history of inhibitor | Throughout the study period of up to approximately: 8 years (rAHF-PFM alone), or 12 years (rAHF-PEG alone or after receiving rAHF-PFM) |
| Incidence of Inhibitors in Previously Untreated Patient (PUPs) and Minimally Treated Patients (MTPs) with Factor VIII (FVIII) Levels <1%, <=2%, and <= 5% | Throughout the study period of up to approximately: 8 years (rAHF-PFM alone), or 12 years (rAHF-PEG alone or after receiving rAHF-PFM) |
| Incidence of therapy-related serious adverse events | Throughout the study period of up to approximately: 8 years (rAHF-PFM alone), or 12 years (rAHF-PEG alone or after receiving rAHF-PFM) |
| Incidence of therapy-related non-serious adverse events | Throughout the study period of up to approximately: 8 years (rAHF-PFM alone), or 12 years (rAHF-PEG alone or after receiving rAHF-PFM) |
| Incidence of inhibitors after switching to rAHF-PEG | Incidence of inhibitors after switching to rAHF-PEG in the same subgroups of patients | Throughout the study period of up to approximately: 8 years (rAHF-PFM alone), or 12 years (rAHF-PEG alone or after receiving rAHF-PFM) |
| Murdoch |
| Western Australia |
| 6050 |
| Australia |
| Landes Frauen und Kinderklinik Linz (LFKK Linz) | Linz | 4017 | Austria |
| Kepler Universitätsklinikum Klinik für Kinder-und Jugendheilkunde | Linz | 4020 | Austria |
| Medizinische Universitaet Wien | Vienna | 1090 | Austria |
| Cliniques Universitaires Saint-Luc | Brussels | B-1200 | Belgium |
| Hemoce - Ce | Fortaleza | Ceará | 60431086 | Brazil |
| Hemocentro do Espírito Santo | Vitória | Espírito Santo | 29040-090 | Brazil |
| Hemepar - Pr | Curitiba | Paraná | 80045-145 | Brazil |
| Hemorgs - Rs | Porto Alegre | Parthenon | 90650-000 | Brazil |
| Fundação HEMOPA | Belém | Pará | 66033-000 | Brazil |
| Hemocentro da UNICAMP | Campinas | São Paulo | 13083-970 | Brazil |
| Hemocentro Ribeirão Preto - SP | Ribeirão Preto | São Paulo | 14051140 | Brazil |
| Hemorio - Rj | Rio de Janeiro | 20211030 | Brazil |
| Faculdade de Medicina da Universidade de São Paulo | São Paulo | 5403000 | Brazil |
| Stollery Children's Hospital, University of Alberta | Edmonton | Alberta | T6G 2B7 | Canada |
| The Moncton City Hospital | Moncton | New Brunswick | E1C 6Z8 | Canada |
| Children's Hospital of Eastern Ontario | Ottawa | Ontario | K1H 8L1 | Canada |
| St-Michael's Hospital | Toronto | Ontario | M5B 1W8 | Canada |
| Sick Kids Hospital | Toronto | Ontario | M5G 1X8 | Canada |
| Royal University Hospital | Saskatoon | Saskatchewan | S7N 0W8 | Canada |
| Shenzhen Children's Hospital | Shenzhen | Futian | China |
| Nanfang Hospital Affiliated to Nanfang Medical University | Guangzhou | Guangdong | China |
| Institute of Hematology, Blood Disease Hospital, PUMC&CAMS | Tianjin | Heping | China |
| Tongji Hospital, Tongji Medical College of Huazhong University of Science & Technology | Wuhan | Hubei | China |
| The Affiliated Hospital of Xuzhou Medical University | Xuzhou | Jiangsu | 221006 | China |
| The Blood Center of Shandong Province | Jinan | Shandong | China |
| Beijing Children's Hospital Affiliated to Capital University of Medical Sciences | Beijing | 100045 | China |
| Nanjing Drum Tower Hospital The Affiliated Hospital of Nanjing University Medical School | Nanjing | China |
| Shenzhen Second People's Hospital | Shenzhen | 518037 | China |
| IPS FUSA SAS Centro Integral de Coagulacion | Atlántico | Colombia |
| Integral Solutions SD SAS | Bogotá | Colombia |
| Fundación Oftalmológica de Santander FOSCAL | Floridablanca | 681004 | Colombia |
| Fakultní nemocnice Brno | Brno | 613 00 | Czechia |
| Fakultní nemocnice Ostrava, Oddělení dětské hematologie a hematoonkologie | Ostrava | 70852 | Czechia |
| Fakultní nemocnice Ostrava | Ostrava | 70852 | Czechia |
| Fakultní nemocnice v Motole | Prague | 150 06 | Czechia |
| Rigshospitalet | Copenhagen | 2100 | Denmark |
| "Hôpital Morvan CHRU de Brest" | Brest | 29200 | France |
| CHU Côte de Nacre - CRTH | Caen | 14033 | France |
| Centre Hospitalier Générale - CTH | Chambéry | 73011 | France |
| CTRH - CHU Bocage | Dijon | 21079 | France |
| Hôpital André Mignot | Le Chesnay | 78157 | France |
| Hôpital de la Mère et de l'Enfant - CHU de LIMOGES | Limoges | 87043 | France |
| CHRU Hôtel Dieu - CRTH | Nantes | 44093 | France |
| Hôpital Cochin | Paris | 75014 | France |
| CHU de Reims Hôpital Maison Blanche - CRTH | Reims | 51092 | France |
| Centre Régional de Traitement de l'Hémophilie et des Maladies hémorragiques. CHU de Rennes - Hôpital Pontchaillou | Rennes | 35033 | France |
| CHRU Charles Nicolle | Rouen | 76031 | France |
| CIC | Saint-Priest-en-Jarez | 42270 | France |
| CHRU Purpan CRTH - Pavillon Sénac | Toulouse | 31059 | France |
| Aghia Sofia Children's Hospital | Goudi | Athens | 11527 | Greece |
| Laikon General Hospital | Goudi | Athens | 11527 | Greece |
| General Hospital of Thessaloniki "Ippokratio" | Thessaloniki | 546 42 | Greece |
| Heim Pál Gyermekkórház | Budapest | H-1089 | Hungary |
| Debreceni Egyetem Klinikai Kozpont | Debrecen | H-4032 | Hungary |
| Mohács City Hospital | Mohács | H-7700 | Hungary |
| Jósa András County Hospital | Nyíregyháza | H-4400 | Hungary |
| Markusovszky Hospital | Szombathely | H-9700 | Hungary |
| Azienda Ospedaliera Policlinico Consorziale Di Bari | Bari | 70124 | Italy |
| Policlinico S Orsola Malpighi | Bologna | 40138 | Italy |
| Azienda Ospedaliera Universitaria Vittorio Emanuele, Ferrarotto, S. Bambino | Catania | 95120 | Italy |
| Ospedale Pugliese -Ciaccio | Catanzaro | 88100 | Italy |
| Az. Osp. Univ. Careggi | Florence | 50134 | Italy |
| Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico | Milan | 20122 | Italy |
| Azienda Ospedaliera Universitaria Federico II | Naples | 80131 | Italy |
| Azienda Ospedaliera di Padova Clinica Medica II | Padova | 35100 | Italy |
| AOUP P. Giaccone | Palermo | 90133 | Italy |
| Ospedale di Macerata | Province of Macerata | 62100 | Italy |
| Azienda Ospedaliera Bianchi Melacrino Morelli | Reggio Calabria | 89123 | Italy |
| Università degli studi di Roma "La Sapienza" | Roma | 00161 | Italy |
| Ospedale Pediatrico Bambino Gesù | Roma | 00165 | Italy |
| Policlinico Universitario Gemelli | Roma | 00168 | Italy |
| Centro Emofilia e Coagulopatie Rare | Scorrano | 73025 | Italy |
| Ospedale Molinette | Torino | 10126 | Italy |
| Rikshospitalet | Oslo | N-0027 | Norway |
| Rikshospitalet, Oslo University Hospital | Oslo | N-0424 | Norway |
| Szpital Uniwersytecki nr 1 im. dr Andrzeja Jurasza | Bydgoszcz | 85-094 | Poland |
| Samodzielny Publiczny Szpital Kliniczny nr 1 | Gdansk | 80-952 | Poland |
| Samodzielny Publiczny Dzieciecy Szpital Kliniczny | Warsaw | 02-091 | Poland |
| Uniwersytecki Szpital Kliniczny we Wrocławiu | Wroclaw | 50-556 | Poland |
| Hospital do Divino Espírito Santo | Ponta Delgada | Azores | 9500-370 | Portugal |
| Centro Hospitalar e Universitade de Coimbra | Coimbra | 3000-602 | Portugal |
| Centro Hospitalar Lisboa Norte Hospital de Sta. Maria | Lisbon | 1649-035 | Portugal |
| Centro Hospitalar de São João, E.P.E. | Porto | 4200-319 | Portugal |
| SBHI Chelyabinsk Regional Children's Clinical Hospital | Chelyabinsk | Russia |
| Federal State Budget Institution "Hematology Research Center" of Ministry of Healthcare of Russian Federation | Moscow | 125167 | Russia |
| SBHI of the Republic of Kareliya Republican Hospital n.a. V.A. Baranov | Petrozavodsk | Russia |
| State Budget Healthcare Institution of Saint-Petersburg "City polyclinic #37" | Saint Petersburg | 191186 | Russia |
| Clinics of FSBEI High Education Samara SMU of MoH of Russia | Samara | 443079 | Russia |
| University Medical Centre Ljubljana | Ljubljana | SI-1000 | Slovenia |
| Hospital Teresa Herrera-Materno Infantil | A Coruña | 15006 | Spain |
| Hospital Hospital Sant Joan de Déu | Barcelona | 08035 | Spain |
| Hospital Universitari Vall d'Hebron (HUVH) | Barcelona | 08035 | Spain |
| Hospital Universitario Son Espases | Palma de Mallorca | 07120 | Spain |
| Sahlgrenska University Hospital | Gothenburg | SE 41345 | Sweden |
| Kliniska studier i Sverige - Forum Söder | Malmö | SE-205 02 | Sweden |
| Karolinska Universitetssjukhuset Solna | Stockholm | 171 76 | Sweden |
| Universitätsspital Basel | Basel | CH-4031 | Switzerland |
| Inselspital Bern | Bern | 3010 | Switzerland |
| Centre Hospitalier Universitaire Vaudois | Lausanne | 1011 | Switzerland |
| Luzerner Kantonsspital | Lucerne | CH-6000 | Switzerland |
| Kantonsspital St. Gallen | Sankt Gallen | 9007 | Switzerland |
| FMH Kinder und Jugendmedizin, im Wabern-Zentrum | Wabern | CH-3084 | Switzerland |
| Kinderspital Zürich | Zurich | CH-8032 | Switzerland |
| Universitätsspital Zürich | Zurich | CH-8091 | Switzerland |
| Hampshire Hospitals NHS Foundation Trust, Basingstoke and North Hampshire Hospital | Basingstoke | Hampshire | RG24 9NA | United Kingdom |
| Cambridge University Hospital NHS Foundation Trust, Addenbrooke's Hospital | Cambridge | CB2 0QQ | United Kingdom |
| East Kent Hospitals University Foundation Trust, Kent & Canterbury Hospital | Canterbury | CT1 3NG | United Kingdom |
| Great Ormond Street Hospital for Children NHS Trust | London | WC1N 3JH | United Kingdom |
| 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 |
Not provided
Not provided
| ID | Term |
|---|---|
| D005169 | Factor VIII |
| C078147 | F8 protein, human |
| C000609799 | BAX 855 |
| ID | Term |
|---|---|
| D001779 | Blood Coagulation Factors |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D011498 | Protein Precursors |
| D001685 | Biological Factors |
Not provided
Not provided