Research question: Whether there are the changes in quality of life in patients with haemophilia A after switching from SHL FVIII prophylaxis to efmoroctocog alfa prophylaxis? A 12-month prospective open-label, single-arm multicentre study. Evaluation of parameters will be carried out on the backdrop of patient treatment in the settings of routine medical practice. No medical examinations/ procedures/ treatment(s) on the top of regular medical practice are planned, except fixed time of examinations.
Quality of life is among the key factors when medical decision about management of haemophilia is taken. There are no scientific data on the HRQoL in patients after switching from Standard Half Life (SHL) FVIII prophylaxis to efmoroctocog alfa (Extended Half Life, EHL) prophylaxis in Russia. It will be a 12-month prospective open-label, single-arm multicentre study. Evaluation of parameters will be carried out on the backdrop of patient treatment in the settings of routine medical practice. No medical examinations/ procedures/ treatment(s) on the top of regular medical practice are planned, except fixed time of examinations. Providing the centers/ patients with the study drug (efmoroctocog alfa) may be considered as interventional component, whereas all administrations should be in accordance with Instruction on Medical Use approved by Russian Ministry of Health. Research question: Whether there are the changes in quality of life in patients with haemophilia A after switching from SHL FVIII prophylaxis to efmoroctocog alfa prophylaxis? No hypothesis is formulated for this exploratory study. The obtained data will be used to assess real world data on quality of life and clinical outcomes in patients with haemophilia A after switching from SHL FVIII prophylaxis to efmoroctocog alfa prophylaxis in routine medical practice in Russia
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
PREVENTION
Masking
NONE
Enrollment
Efmoroctocog alfa should be administered according to Summary of Product Characteristics (SmPC)/ Instruction on Medical Use approved by Russian Ministry of Health ЛП-006034 (13.01.2020).
League of Clinical Research (LeagueCRR)
Moscow, Russia
Health-related quality of life
Haemophilia Adult Quality of Life Questionnaire (Haem-A-QoL) - for patients of 18-25 y.o.; total score, domain scores. or Haemophilia-specific Quality of Life assessment for children and adolescents Short Form (Haemo-QoL SF) - for patients 12-17 y.o.; total score, domain scores.
Time frame: Month 12
Spontaneous bleedings
Number from previous visit
Time frame: Month 1 - Month 3 - Month 6 - Month 12
Spontaneous bleedings - localisation
Localisation of spontaneous bleedings: joints, muscles, subcutaneous/ submucosal, abdominal/ gastrointestinal, retroperitoneal, genitourinary, CNS, pharyngeal, other.
Time frame: Month 1 - Month 3 - Month 6 - Month 12
Post-traumatic bleedings
Number from previous visit
Time frame: Month 1 - Month 3 - Month 6 - Month 12
Post-traumatic bleedings - localisation
Localisation of post-traumatic bleedings: joints, muscles, subcutaneous/ submucosal, abdominal/ gastrointestinal, retroperitoneal, genitourinary, CNS, pharyngeal, other.
Time frame: Month 1 - Month 3 - Month 6 - Month 12
Administration of efmorococtog alfa for correction of bleedings
Dose of efmorococtog alfa: IU.
Time frame: Month 1 - Month 3 - Month 6 - Month 12
Surgery/ Invasive Procedures: major
Number from previous visit.
Time frame: Month 1 - Month 3 - Month 6 - Month 12
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30
Surgery/ Invasive Procedures: minor
Number from previous visit.
Time frame: Month 1 - Month 3 - Month 6 - Month 12
Administration of efmorococtog alfa for correction of bleedings for Surgery/ Invasive Procedures
Dose of efmorococtog alfa: IU.
Time frame: Month 1 - Month 3 - Month 6 - Month 12
Hospitalisations
Number from previous visit.
Time frame: Month 1 - Month 3 - Month 6 - Month 12
Hospitalisations: duration
Total duration of all hospitalisations: days.
Time frame: Month 1 - Month 3 - Month 6 - Month 12
Hemophilia Joint Health Score
Hemophilia Joint Health Score 2.1 for knee, elbow, ankle (left, right): score.
Time frame: Day 0 - Month 6 - Month 12
Target Joints
Target joints: knee, elbow, ankle, hip, shoulder, carpal, other (left, right): yes/ no.
Time frame: Day 0 - Month 6 - Month 12
Ultrasound Scoring
Ultrasound scoring (HEAD-US): score.
Time frame: Day 0 - Month 6 - Month 12
Factor VIII Activity
Factor VIII Activity (before injection; 30 min after injection), clotting assays (one stage): IU/dL.
Time frame: Day 0 - Month 1 - Month 3 - Month 6 - Month 12
Factor VIII Inhibitor Activity
Factor VIII Inhibitor Activity: Nijmegen-Bethesda unit/mL.
Time frame: Day 0 - Month 1 - Month 3 - Month 6 - Month 12
Administration of efmoroctocog alfa
Administration of efmoroctocog alfa, frequency: no. of injections per week.
Time frame: Day 0 - Month 1 - Month 3 - Month 6 - Month 12
Administration of efmoroctocog alfa: factor consumption
Administration of efmoroctocog alfa, factor consumption: IU per week, IU/kg per week (calculation).
Time frame: Day 0 - Month 1 - Month 3 - Month 6 - Month 12
Previous treatment/ prophylaxis of haemophilia
Generic drug name.
Time frame: Day 0 - Month 1 - Month 3 - Month 6 - Month 12