Effects of Emicizumab Treatment in Patients With Hemophilia A

The pain

Mc Gill Short Form will used to determine the type and severity of the pain. A short form of the McGill Pain Questionnaire (SF-MPQ) has been developed. The main component of the SF-MPQ consists of 15 descriptors (11 sensory; 4 affective) which are rated on an intensity scale as 0 = none, 1 = mild, 2 = moderate or 3 = severe.

Hemophilia Joint Health Score (HJHS)

Hemophilia Joint H is a scoring system that targets the evaluation of three major joints (elbow, knee, and ankle). Consists of a total of 9 questions: 8 questions assessing joints and 1 question providing a global gait score. The hemophilia joint health score is calculated by assessing joint swelling, the longevity of the swelling, the presence of atrophy in the surrounding soft tissue, crepitus during active movement, loss of flexion or extension, pain, muscle strength, and the total global gait score. Hemophilia Joint Health Score values range from 0 to 124, with higher scores indicating poorer joint health.

Lower extremity functionality and mobility- 5-Time Sit-to-Go Test

The 5-time sit-to-stand test is scored based on the time (in seconds to the nearest decimal place) in which a patient can move from a sitting position to a standing position and back to a sitting position five times. At the beginning of the test, the test participant is instructed to sit in a chair with their back straight. They are also instructed to fold their arms across their chest. The test participant should then be instructed to sit and stand five times as quickly as possible. The shorter the time taken to complete the test, the better the test result.

Upper extremity functionality

The Questionnaire for Arm, Shoulder, and Hand Disabilities (Q-DASH) will be used to assess individuals' upper extremity functions. This questionnaire is a self-administered measurement tool with validated validity and reliability in Turkish. The questionnaire, consisting of 30 questions, assesses the individual's ability to perform functional activities (21 items), pain (5 items), and psychosocial aspects of the disease (4 items). The total score ranges from 0 to 100, with a higher score indicating better outcomes.

Fear of movement

The Tampa Kinesiophobia Scale will be used to assess patients' fear of movement. The questionnaire, which will be used to measure individuals' perspectives on movement and their fear aspects, consists of 17 questions with total scores ranging from 17 to 68. Tampa scores greater than 37 indicate a high degree of kinesiophobia.

Functional independence levels

The scoring system, categorized according to the International Classification of Functioning, Disability, and Health (ICF), was developed by considering activities that may be affected in the daily lives of hemophilia patients. Being a performance-based scale, it can be used in different languages. The scale consists of eight questions covering the subparameters of self-care, transfer, and locomotion. The self-care subparameter includes eating and hygiene, bathing and dressing; the transfer subparameter includes chair and squatting; and the locomotion subparameter includes walking, climbing and descending stairs (12-14 steps), and running. Activities are scored on a scale of 1 to 4, depending on the degree of independence. The minimum score is 8, and the maximum is 32. A high score indicates an individual's functional independence.

Children's Quality of Life

The Children's Quality of Life Inventory (PedsQL) will be used to assess patients' quality of life. The scale consists of both child and parent assessment forms and assesses four subscales: physical functioning, social functioning, school life, and emotional well-being. The scale, consisting of 5-point Likert-type questions, measures the child's quality of life based on their life experiences over the past week. Scores from each subscale range from 0 to 100. Higher scores indicate better quality of life, while lower scores indicate problems in the relevant area. The parent and child forms can be evaluated separately to obtain results from different perspectives.