An observational study in Hemophilia, Hemophilia A, Severe and Hemophilia B, sponsored by Hasan Kalyoncu University. Status unknown at 1 site in Turkey (Türkiye). Open to male participants aged 4 Years to 17 Years. Per ClinicalTrials.gov, last updated 2023-06-05.
Sponsored by Hasan Kalyoncu University · Observational
This study aimed to establish the validity and reliability of the Turkish version of the Pediatric Haemophilia Activities List (short - PedHALshort) questionnaire.
Hemophilia is a blood disease characterized by a bleeding disorder that develops due to the deficiency of clotting factor VIII (Hamophilia A) or coagulation factor IX (Hamophilia B) proteins that provide clotting in the blood. In the hereditary disease showing X-linked recessive inheritance, female individuals are carriers and male individuals show a clinical picture. Although the clinical picture changes depending on the duration and frequency of bleeding, the severity of the disease is classified as severe, moderate and mild. Musculoskeletal problems are the most common complications in patients with severe and moderate hemophilia. Hemarthroses due to bleeding in the joint (80%) and hematomas due to intramuscular bleeding (20%) cause joint degeneration and muscle atrophy. Recurrent hemarthroses occur when there is not enough clotting factor in the setting, often before the previous hemarthrosis has resolved. This creates a vicious cycle of bleeding-inflammation-rebleeding known as the "target joint". The resulting vicious circle causes permanent damage to bone and cartilage structures and causes "arthropathy". The knee is the joint region where hemarthrosis and arthropathy are most common, followed by the elbow and ankle joints. Since these joints in the musculoskeletal system contain more synovial tissue than the others, they are more exposed to trauma and load and are injured. As a result of all these, pain, joint limitations and muscle strength losses occur. All these clinical findings lead to a decrease in functional independence in activities of daily living with the restriction of physical activity in patients.
In the literature, there are questionnaires and measurement methods in which many functional evaluations are made for hemophilia patients. However, most of them have not been validated and reliable in Turkish. For this purpose, our study aimed to establish the validity and reliability of the Turkish version of the Pediatric Haemophilia Activities List (PedHALshort) questionnaire.
885 studies on the registry are indexed under Hemophilia A; 139 are open to participants now.
This study's planned enrollment of 50 is below the median of 80 across 322 observational studies indexed under Hemophilia A.
Browse Hemophilia A studies →Hasan Kalyoncu University is the lead sponsor of 165 studies on the registry; 32 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Pediatric group hemophilia A and hemophilia B children living in Gaziantep constitute the study population.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
- Children with neurological signs that limit activities of daily living
The PedHAL questionnaire will be administered to children aged 4-17 years with a diagnosis of hemophilia twice, one week apart. For construct validity assessment, the questionnaire will be compared with the Hemophilia Functional Independence Score (HJHS).
Paediatric Haemophilia Activities List
PedHAL contains 22 items in 7 areas: sitting/kneeling/standing, functions of legs, functions of arms, use of transportation, personal care, housework and leisure activities, and sports. It consists of a child version (8-17 years old) and a parent version (4-17 years old). Items are scored for each item on a 6-point Likert scale ("impossible", "always", "usually", "sometimes", "almost never", "never"), "not applicable (N/A)". . The score is converted to a normalized score from 0 to 100, the higher score representing better functional status. If more than half of the items are missing or scored as "not applicable" (N/A), a valid total score will not be calculated.
Time frame: through of the study, average 6 months
FISH
The scoring system, which is categorized according to the international classification of functionality, disability and health (ICF), has been prepared by considering the activities that hemophilia patients may be affected by in their daily lives. Being a performance-based scale allows it to be used in different languages. The scale consists of 8 questions in total, including the sub-parameters of self-care, transfer and locomotion. Self care subparameter; eating-hygiene, bathing and dressing, transfer sub-parameter; chair and squat, locomotion subparameter; It consists of walking, stair climbing (12-14 steps) and running activities. Scoring of the activities is between 1 and 4 according to the degree of independence. The minimum score that can be obtained from the scale is 8, and the maximum score is 32. A high score indicates the functional independence of the individual.
Time frame: through of the study, average 6 months
Plan to share: No
Start is estimated for May 2023; primary completion for Aug 2023, 3 months later.
Of 77 non-industry observational studies under Hemophilia A, half reached primary completion within 33 months of starting (the median), counting those still running; a quarter within 15 and three quarters within 96. This study's planned 3 months is below that.
Counting every sponsor, half of 135 studies reached it within 45 months; a quarter within 25 and three quarters within 75.
How these figures are worked out, and which studies are left out of them.
This study is status unknown, as verified in Jun 2023. You cannot join it, but the record below documents what was studied.
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