Health Related Quality of Life of Youth and Young Adults With Haemophilia A

Study Overview
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Brief Summary
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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.

Detailed Description
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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

Official Title
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Health Related Quality of Life of Youth and Young Adults With Haemophilia A Treated With Efmoroctocog Alfa in Russia

Conditions
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Hemophilia A

Intervention / Treatment
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* Drug: Efmoroctocog Alfa Injection [Eloctate]


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Signed consent form. Adults, adolescents and children (12-25 years old) with haemophilia A with ABR ≥ 2 on at least one-year SHL FVIII prophylaxis. Exclusion Criteria: Patients who have had hypersensitivity reactions to efmoroctocog alfa or other constituents of the product. History of Factor VIII inhibitors. Patients who have other haemostatic disorders. Patients participating in interventional studies.

Ages Eligible for Study
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Minimum Age: 12 Years
Maximum Age: 25 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Prevention
Intervention Model: Single Group Assignment
Interventional Model Description: A 12-month prospective open-label, single-arm multicentre study.
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Patients receiving Efmoroctocog alfa<br>For long term prophylaxis, the recommended starting dose is 50 IU of factor VIII per kg body weight at intervals of 3 to 5 days. The dose may be adjusted based on patient response in the range of 25 to 65 IU/kg. In some cases, especially in younger patients, shorter dosage intervals or higher doses may be necessary. | Drug: Efmoroctocog Alfa Injection [Eloctate]<br>* 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).<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | Month 12 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Spontaneous bleedings | Number from previous visit | 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. | Month 1 - Month 3 - Month 6 - Month 12 | 
| Post-traumatic bleedings | Number from previous visit | 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. | Month 1 - Month 3 - Month 6 - Month 12 | 
| Administration of efmorococtog alfa for correction of bleedings | Dose of efmorococtog alfa: IU. | Month 1 - Month 3 - Month 6 - Month 12 | 
| Surgery/ Invasive Procedures: major | Number from previous visit. | Month 1 - Month 3 - Month 6 - Month 12 | 
| Surgery/ Invasive Procedures: minor | Number from previous visit. | 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. | Month 1 - Month 3 - Month 6 - Month 12 | 
| Hospitalisations | Number from previous visit. | Month 1 - Month 3 - Month 6 - Month 12 | 
| Hospitalisations: duration | Total duration of all hospitalisations: days. | 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. | Day 0 - Month 6 - Month 12 | 
| Target Joints | Target joints: knee, elbow, ankle, hip, shoulder, carpal, other (left, right): yes/ no. | Day 0 - Month 6 - Month 12 | 
| Ultrasound Scoring | Ultrasound scoring (HEAD-US): score. | Day 0 - Month 6 - Month 12 | 
| Factor VIII Activity | Factor VIII Activity (before injection; 30 min after injection), clotting assays (one stage): IU/dL. | Day 0 - Month 1 - Month 3 - Month 6 - Month 12 | 
| Factor VIII Inhibitor Activity | Factor VIII Inhibitor Activity: Nijmegen-Bethesda unit/mL. | Day 0 - Month 1 - Month 3 - Month 6 - Month 12 | 
| Administration of efmoroctocog alfa | Administration of efmoroctocog alfa, frequency: no. of injections per week. | 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). | Day 0 - Month 1 - Month 3 - Month 6 - Month 12 | 
| Previous treatment/ prophylaxis of haemophilia | Generic drug name. | Day 0 - Month 1 - Month 3 - Month 6 - Month 12 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Haemophilia A, Health-related Quality of Life, Efmoroctocog alfa