EAHAD Workgroup very rare coagulation factor deficiencies - Survey Healthcare professionals Question Title * 1. What is your profession? Internist Hematologist Pediatric hematologist (Specialized) nurse Physician assistant Laboratory specialist Scientist (PhD student, post doc, primary scientist) Other (please specify) Question Title * 2. In which type of centre do you work? Comprehensive Hemophilia Care Centre / Hemophilia Treatment Centre / Teaching Centre / Tertiary Care Centre Community hospital Private practice Other (please specify) Question Title * 3. How many years have you been involved in the management of bleeding disorders? <5 years 5-10 years 11-20 years >20 years Question Title * 4. What is your gender? Female Male Non-binary Question Title * 5. Which is your country of residence? Afghanistan Albania Algeria Andorra Angola Antigua and Barbuda Argentina Armenia Australia Austria Azerbaijan Bahamas Bahrain Bangladesh Barbados Belarus Belgium Belize Benin Bhutan Bolivia (Plurinational State of) Bosnia and Herzegovina Botswana Brazil Brunei Darussalam Bulgaria Burkina Faso Burundi Cabo Verde Cambodia Cameroon Canada Central African Republic Chad Chile China Colombia Comoros Congo Costa Rica Côte D'Ivore Croatia Cuba Cyprus Czech Republic Democratic People's Republic of Korea Democratic Republic of Congo Denmark Djibouti Dominican Republic Ecuador Egypt El Salvador Equatorial Guinea Eritrea Estonia Fiji Finland France Gabon Gambia Georgia Germany Ghana Greece Grenada Guatemala Guinea Guinea Bissau Guyana Haiti Holy See Honduras Hungary Iceland India Indonesia Iran (Islamic Republic of) Iraq Ireland Israel Italy Jamaica Japan Jordan Kazakhstan Kenya Kiribati Kuwait Kyrgyzstan Lao People's Democratic Republic Latvia Lebanon Lesotho Liberia Libya Liechtenstein Lithuania Luxembourg Madagascar Malawi Malaysia Maldives Mali Malta Marshall Islands Mauritania Mauritius Mexico Micronesia (Federated States of) Monaco Mongolia Montenegro Morocco Mozambique Myanmar Namibia Nauru Nepal Netherlands New Zealand Nicaragua Niger Nigeria Norway Oman Pakistan Palau Panama Papua New Guinea Paraguay Peru Philippines Poland Portugal Qatar Republic of Korea Republic of Moldova Romania Russian Federation Rwanda Saint Kitts and Nevis Saint Lucia Saint Vincent and the Grenadines Samoa San Marino Sao Tome and Principe Saudi Arabia Senegal Serbia Seychelles Sierra Leone Singapore Slovakia Slovenia Solomon Islands Somalia South Africa South Sudan Spain Sri Lanka State of Palestine Sudan Suriname Swaziland Sweden Switzerland Syrian Arab Republic Tajikistan Thailand The former Yugoslav Republic of Macedonia Timor-Leste Togo Tonga Trinidad and Tobago Tunisia Turkey Turkmenistan Tuvalu Uganda Ukraine United Arab Emirates United Kingdom United Republic of Tanzania United States of America Uruguay Uzbekistan Vanuatu Venezuela (Bolivarian Republic of) Vietnam Yemen Zambia Zimbabwe Question Title * 6. Do you screen for very rare coagulation factor deficiencies in the diagnostic work up of patients with a bleeding tendency? Definition of very rare coagulation factor deficiency is as follows: a/hypofibrinogenemia, Dysfibrinogenemia, FII deficiency, FV deficiency, FV & FVIII deficiency, FVII & FX deficiency, FVII deficiency, FX deficiency, FXI deficiency, FXIII deficiency and Hereditary combined deficiency of vitamin K-dependent clotting factors. Yes, always No Other (please specify) Question Title * 7. Do you have lab facilities with appropriate assays to screen for VRCFD in your hospital? Yes No Other (please specify) Question Title * 8. Do you perform genetic analysis to confirm the underlying genetic pathophysiological variant in the VRCFD from question 7? Yes No Other (please specify) Question Title * 9. Which patients with a very rare coagulation factor deficiency (VRCFD) receive a general treatment plan? Only severe VRCFD patients with a factor activity level <10%, irrespective of bleeding tendency and surgical procedures Severe and mild VRCFD patients with a factor activity <20%, irrespective of bleeding tendency and surgical procedures Severe and mild VRCFD patients with a factor activity level <50%, irrespective of bleeding tendency and surgical procedures Severe and mild VRCFD patients with a factor activity level <50% and an increased bleeding tendency Severe and mild VRCFD patients with a bleeding tendency irrespective of the factor activity level Severe VRCFD with a factor activity level <10% who are undergoing an invasive procedure Severe and mild VRCFD patients with a factor activity level <20% who are undergoing an invasive procedure Severe and mild VRCFD patients with a factor activity level <50% who are undergoing an invasive procedure Severe and mild VRCFD patients with a bleeding tendency irrespective of the factor activity level, who are undergoing an invasive procedure Other (please specify) Question Title * 10. Which patients with a very rare coagulation factor deficiency (VRCFD) receive a peri-surgical treatment plan? Only severe VRCFD patients with a factor activity level <10%, irrespective of bleeding tendency and surgical procedures Severe and mild VRCFD patients with a factor activity <20%, irrespective of bleeding tendency and surgical procedures Severe and mild VRCFD patients with a factor activity level <50%, irrespective of bleeding tendency and surgical procedures Severe and mild VRCFD patients with a factor activity level <50% and an increased bleeding tendency Severe and mild VRCFD patients with a bleeding tendency irrespective of the factor activity level Severe VRCFD with a factor activity level <10% who are undergoing an invasive procedure Severe and mild VRCFD patients with a factor activity level <20% who are undergoing an invasive procedure Severe and mild VRCFD patients with a factor activity level <50% who are undergoing an invasive procedure Severe and mild VRCFD patients with a bleeding tendency irrespective of the factor activity level, who are undergoing an invasive procedure Question Title * 11. Do you give these patients also a special emergency card with their diagnosis in combination with a general emergency treatment plan and the HCT emergency number? Yes No In selected cases Question Title * 12. Do you provide follow up visits for these patients in your centre? Yes No In selected cases Question Title * 13. How many children do you treat in your centre for the below cases? Please provide a rough estimation, exact numbers are not expected. Please, note if you treat adults, you can complete with a 0. Afibrinogenemia/hypofibrinogenemia Factor II deficiency Factor V deficiency Factor V & FVIII deficiency Factor VII deficiency Factor VII & X deficiency Factor X deficiency Factor XI deficiency Factor XIII deficiency Hereditary combined deficiency of vitamin K-dependent clotting factors Question Title * 14. How many patients are treated in your HTC with regular prophylactic therapy, with Afibrinogenemia/hypofibrinogenemia Factor II deficiency Factor V deficiency Factor V & VIII deficiency Factor VII deficiency Factor VII & X deficiency Factor X deficiency Factor XI deficiency Factor XIII deficiency Hereditary combined deficiency of vitamin K-dependent clotting factors Question Title * 15. Which hemostastic products do you give patients with a Tranexamic acid Fibrinogen concentrate Fresh Frozen Plasma / omniplasma / pooled plasma Cryoprecipitate PCC (prothrombin complex concentrate) Platelet transfusion Recombinant FVIIa Plasma-derived FVII Plasma derived FXI concentrate Plasma-derived factor XIII concentrate Recombinant factor XIII concentrate Afibrinogenemia/hypofibrinogemia Afibrinogenemia/hypofibrinogemia Tranexamic acid Afibrinogenemia/hypofibrinogemia Fibrinogen concentrate Afibrinogenemia/hypofibrinogemia Fresh Frozen Plasma / omniplasma / pooled plasma Afibrinogenemia/hypofibrinogemia Cryoprecipitate Afibrinogenemia/hypofibrinogemia PCC (prothrombin complex concentrate) Afibrinogenemia/hypofibrinogemia Platelet transfusion Afibrinogenemia/hypofibrinogemia Recombinant FVIIa Afibrinogenemia/hypofibrinogemia Plasma-derived FVII Afibrinogenemia/hypofibrinogemia Plasma derived FXI concentrate Afibrinogenemia/hypofibrinogemia Plasma-derived factor XIII concentrate Afibrinogenemia/hypofibrinogemia Recombinant factor XIII concentrate Dysfibrinogenemia (increased bleeding tendency) Dysfibrinogenemia (increased bleeding tendency) Tranexamic acid Dysfibrinogenemia (increased bleeding tendency) Fibrinogen concentrate Dysfibrinogenemia (increased bleeding tendency) Fresh Frozen Plasma / omniplasma / pooled plasma Dysfibrinogenemia (increased bleeding tendency) Cryoprecipitate Dysfibrinogenemia (increased bleeding tendency) PCC (prothrombin complex concentrate) Dysfibrinogenemia (increased bleeding tendency) Platelet transfusion Dysfibrinogenemia (increased bleeding tendency) Recombinant FVIIa Dysfibrinogenemia (increased bleeding tendency) Plasma-derived FVII Dysfibrinogenemia (increased bleeding tendency) Plasma derived FXI concentrate Dysfibrinogenemia (increased bleeding tendency) Plasma-derived factor XIII concentrate Dysfibrinogenemia (increased bleeding tendency) Recombinant factor XIII concentrate FII deficiency FII deficiency Tranexamic acid FII deficiency Fibrinogen concentrate FII deficiency Fresh Frozen Plasma / omniplasma / pooled plasma FII deficiency Cryoprecipitate FII deficiency PCC (prothrombin complex concentrate) FII deficiency Platelet transfusion FII deficiency Recombinant FVIIa FII deficiency Plasma-derived FVII FII deficiency Plasma derived FXI concentrate FII deficiency Plasma-derived factor XIII concentrate FII deficiency Recombinant factor XIII concentrate FV deficiency FV deficiency Tranexamic acid FV deficiency Fibrinogen concentrate FV deficiency Fresh Frozen Plasma / omniplasma / pooled plasma FV deficiency Cryoprecipitate FV deficiency PCC (prothrombin complex concentrate) FV deficiency Platelet transfusion FV deficiency Recombinant FVIIa FV deficiency Plasma-derived FVII FV deficiency Plasma derived FXI concentrate FV deficiency Plasma-derived factor XIII concentrate FV deficiency Recombinant factor XIII concentrate FVII deficiency FVII deficiency Tranexamic acid FVII deficiency Fibrinogen concentrate FVII deficiency Fresh Frozen Plasma / omniplasma / pooled plasma FVII deficiency Cryoprecipitate FVII deficiency PCC (prothrombin complex concentrate) FVII deficiency Platelet transfusion FVII deficiency Recombinant FVIIa FVII deficiency Plasma-derived FVII FVII deficiency Plasma derived FXI concentrate FVII deficiency Plasma-derived factor XIII concentrate FVII deficiency Recombinant factor XIII concentrate FXI deficiency FXI deficiency Tranexamic acid FXI deficiency Fibrinogen concentrate FXI deficiency Fresh Frozen Plasma / omniplasma / pooled plasma FXI deficiency Cryoprecipitate FXI deficiency PCC (prothrombin complex concentrate) FXI deficiency Platelet transfusion FXI deficiency Recombinant FVIIa FXI deficiency Plasma-derived FVII FXI deficiency Plasma derived FXI concentrate FXI deficiency Plasma-derived factor XIII concentrate FXI deficiency Recombinant factor XIII concentrate FXIII deficiency FXIII deficiency Tranexamic acid FXIII deficiency Fibrinogen concentrate FXIII deficiency Fresh Frozen Plasma / omniplasma / pooled plasma FXIII deficiency Cryoprecipitate FXIII deficiency PCC (prothrombin complex concentrate) FXIII deficiency Platelet transfusion FXIII deficiency Recombinant FVIIa FXIII deficiency Plasma-derived FVII FXIII deficiency Plasma derived FXI concentrate FXIII deficiency Plasma-derived factor XIII concentrate FXIII deficiency Recombinant factor XIII concentrate Other (please specify) Question Title * 16. How often do you see VRCFD patients in the outward patient clinic? Multiple options are possible. Dependent on severity of factor deficiency Dependent of bleeding tendency Once a year Every other year Every 5 years Other (please specify) Question Title * 17. Do you pursue family screening after diagnosing a child with a severe VRCFD (factor activity level <10%)? Yes No Not applicable Question Title * 18. Do you pursue family screening after diagnosing an adult with a severe VRCFD (factor activity level <10%)? Yes No Question Title * 19. Do you pursue family screening after diagnosing a child with a mild VRCFD (Factor activity level >10-50%) Yes No Other (please specify) Question Title * 20. Do you pursue family screening after diagnosing an adult with a mild VRCFD (Factor activity level >10-50%) Yes No Other (please specify) Question Title * 21. Which family members are invited for screening for a VRCFD? Only first degree family members Only first degree family members with an increased bleeding tendency First and second degree family members First and second degree family members with an increased bleeding tendency Other (please specify) Question Title * 22. Are there any ongoing clinical trials (observational, interventional) for VRCFD patients? No Yes, please specify Question Title * 23. Do you prioritize clinical trials for VRCFD patients? Yes No Not applicable Next
