HemophiliaNCT05889754
Reliability and Validity of the Turkish Version of the PedHAL
An observational study of Hemophilia and Hemophilia A, Severe, run by Hasan Kalyoncu University at 1 site in Turkey (Türkiye).
Trial summary
This study aimed to establish the validity and reliability of the Turkish version of the Pediatric Haemophilia Activities List (short - PedHALshort) questionnaire. Read more about this study
- Who is it for?
- Ages 4 to 17 years, men only
- Conditions the study is for
- Hemophilia, Hemophilia A, Severe, Hemophilia B
- How many people
- 50 participants
- Where
- 1 siteTurkey (Türkiye)
- Study dates
- 30 May 2023to 30 Dec 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 to 17 years
- Men only
- No healthy volunteers
- Child
Study population: Pediatric group hemophilia A and hemophilia B children living in Gaziantep constitute the study population.
Who may be able to join
- Having been diagnosed with Hemophilia by a physician (Factor VIII-IX)
- Ages 4-17 years old
- Receiving regular prophylactic treatment
- Children who volunteered to participate in the study and whose family consent was given
Who cannot join
- Children with neurological signs that limit activities of daily living
Trial locations and contacts
1 site in 1 country.
Turkey (Türkiye)1 site
Hasan Kalyoncu University
Gaziantep, Şahinbey, 27100
About this study
Official title: The Validity and Reliability of the Turkish Version of the "Pediatric Haemophilia Activities List - Short - PedHALshort" Questionnaire
This study aimed to establish the validity and reliability of the Turkish version of the Pediatric Haemophilia Activities List (short - PedHALshort) questionnaire.
Read the detailed description
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.
- Conditions
- Hemophilia, Hemophilia A, Severe, Hemophilia B
- Keywords
- Functional independence, Activities of daily living, Hemophilia, Factor VIII, Factor IX
- Medical subject headings
- Hemophilia A, Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Hemophilia Group | 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). |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: through of the study, average 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jun 2023
- Study start30 May 2023 (estimated)
- Primary completion30 Aug 2023 (estimated)
- Study completion30 Dec 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (7)Papers the sponsor has linked to this study.
- Other trials for HemophiliaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hasan Kalyoncu University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Tuğba GÖNEN, Hasan Kalyoncu University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05889754
- Sponsor's protocol ID
- 2023/53
- Status last verified
- Jun 2023
- First submitted
- 22 May 2023
- Last updated
- 5 Jun 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Souza JC, Simoes HG, Campbell CS, Pontes FL, Boullosa DA, Prestes J. Haemophilia and exercise. Int J Sports Med. 2012 Feb;33(2):83-8. doi: 10.1055/s-0031-1286292. Epub 2011 Nov 17.
PubMed 22095329Wagner B, Kruger S, Hilberg T, Ay C, Hasenoehrl T, Huber DF, Crevenna R. The effect of resistance exercise on strength and safety outcome for people with haemophilia: A systematic review. Haemophilia. 2020 Mar;26(2):200-215. doi: 10.1111/hae.13938. Epub 2020 Feb 24.
PubMed 32091659Schafer GS, Valderramas S, Gomes AR, Budib MB, Wolff AL, Ramos AA. Physical exercise, pain and musculoskeletal function in patients with haemophilia: a systematic review. Haemophilia. 2016 May;22(3):e119-29. doi: 10.1111/hae.12909. Epub 2016 Apr 14.
PubMed 27075748De la Corte-Rodriguez H, Rodriguez-Merchan EC. The role of physical medicine and rehabilitation in haemophiliac patients. Blood Coagul Fibrinolysis. 2013 Jan;24(1):1-9. doi: 10.1097/MBC.0b013e32835a72f3.
PubMed 23103725Young NL, Bradley CS, Blanchette V, Wakefield CD, Barnard D, Wu JK, McCusker PJ. Development of a health-related quality of life measure for boys with haemophilia: the Canadian Haemophilia Outcomes--Kids Life Assessment Tool (CHO-KLAT). Haemophilia. 2004 Mar;10 Suppl 1:34-43. doi: 10.1111/j.1355-0691.2004.00877.x.
PubMed 14987247Timmer MA, Gouw SC, Feldman BM, Zwagemaker A, de Kleijn P, Pisters MF, Schutgens REG, Blanchette V, Srivastava A, David JA, Fischer K, van der Net J. Measuring activities and participation in persons with haemophilia: A systematic review of commonly used instruments. Haemophilia. 2018 Mar;24(2):e33-e49. doi: 10.1111/hae.13367. Epub 2017 Nov 27.
PubMed 29178149Shrout PE, Fleiss JL. Intraclass correlations: uses in assessing rater reliability. Psychol Bull. 1979 Mar;86(2):420-8. doi: 10.1037//0033-2909.86.2.420.
PubMed 18839484
Study at a glance
From the registry record, last updated by the sponsor on 5 Jun 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Validity and Reliability of the Turkish Version of the "Pediatric Haemophilia Activities List - Short - PedHALshort" Questionnaire
- ClinicalTrials.gov ID
- NCT05889754
- Acronym / sponsor ID
- 2023/53
- Conditions
- Hemophilia, Hemophilia A, Severe, Hemophilia B
- Keywords
- Functional independence, Activities of daily living, Hemophilia, Factor VIII, Factor IX
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Hasan Kalyoncu UniversityUniversity, hospital or other
- Study start
- 30 May 2023 (estimated)
- Primary completion
- 30 Aug 2023 (estimated)
- Study completion
- 30 Dec 2023 (estimated)
- First posted
- 5 Jun 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study aimed to establish the validity and reliability of the Turkish version of the Pediatric Haemophilia Activities List (short - PedHALshort) questionnaire.
Detailed description
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Hemophilia Group | – | –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). |
Who may qualify
- Ages
- 4 to 17 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Pediatric group hemophilia A and hemophilia B children living in Gaziantep constitute the study population.
Key inclusion criteria (4 listed)
- Having been diagnosed with Hemophilia by a physician (Factor VIII-IX)
- Ages 4-17 years old
- Receiving regular prophylactic treatment
- Children who volunteered to participate in the study and whose family consent was given
Key exclusion criteria (1 listed)
- Children with neurological signs that limit activities of daily living
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Paediatric Haemophilia Activities List
Time frame: through of the study, average 6 months
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.
FISH
Time frame: through of the study, average 6 months
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.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Jun 2023.
- Background: PubMed 22095329Souza JC, Simoes HG, Campbell CS, Pontes FL, Boullosa DA, Prestes J. Haemophilia and exercise. Int J Sports Med. 2012 Feb;33(2):83-8. doi: 10.1055/s-0031-1286292. Epub 2011 Nov 17.
- Background: PubMed 32091659Wagner B, Kruger S, Hilberg T, Ay C, Hasenoehrl T, Huber DF, Crevenna R. The effect of resistance exercise on strength and safety outcome for people with haemophilia: A systematic review. Haemophilia. 2020 Mar;26(2):200-215. doi: 10.1111/hae.13938. Epub 2020 Feb 24.
- Background: PubMed 27075748Schafer GS, Valderramas S, Gomes AR, Budib MB, Wolff AL, Ramos AA. Physical exercise, pain and musculoskeletal function in patients with haemophilia: a systematic review. Haemophilia. 2016 May;22(3):e119-29. doi: 10.1111/hae.12909. Epub 2016 Apr 14.
- Background: PubMed 23103725De la Corte-Rodriguez H, Rodriguez-Merchan EC. The role of physical medicine and rehabilitation in haemophiliac patients. Blood Coagul Fibrinolysis. 2013 Jan;24(1):1-9. doi: 10.1097/MBC.0b013e32835a72f3.
- Background: PubMed 14987247Young NL, Bradley CS, Blanchette V, Wakefield CD, Barnard D, Wu JK, McCusker PJ. Development of a health-related quality of life measure for boys with haemophilia: the Canadian Haemophilia Outcomes--Kids Life Assessment Tool (CHO-KLAT). Haemophilia. 2004 Mar;10 Suppl 1:34-43. doi: 10.1111/j.1355-0691.2004.00877.x.
- Background: PubMed 29178149Timmer MA, Gouw SC, Feldman BM, Zwagemaker A, de Kleijn P, Pisters MF, Schutgens REG, Blanchette V, Srivastava A, David JA, Fischer K, van der Net J. Measuring activities and participation in persons with haemophilia: A systematic review of commonly used instruments. Haemophilia. 2018 Mar;24(2):e33-e49. doi: 10.1111/hae.13367. Epub 2017 Nov 27.
- Background: PubMed 18839484Shrout PE, Fleiss JL. Intraclass correlations: uses in assessing rater reliability. Psychol Bull. 1979 Mar;86(2):420-8. doi: 10.1037//0033-2909.86.2.420.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia and Hemophilia A, Severe, run by Hasan Kalyoncu University at 1 site in Turkey (Türkiye).
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 5 Jun 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Turkey (Türkiye). Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Tuğba GÖNEN, Asst. Prof. (05050905846, tugba.badat@hku.edu.tr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Paediatric Haemophilia Activities List (time frame: through of the study, average 6 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05889754, which also lists 7 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Jun 2023.