Reliability and Validity of the Turkish Version of the PedHAL

Hemophilia B Clinical Trial

Official title:

The Validity and Reliability of the Turkish Version of the "Pediatric Haemophilia Activities List - Short - PedHALshort" Questionnaire

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT05889754
• Other study ID #: 2023/53
• Status: Not yet recruiting
• Start date: May 30, 2023
• Est. completion date: December 30, 2023

Study information

• Verified date: June 2023
• Source: Hasan Kalyoncu University
• Contact: Tugba GÖNEN, Asst. Prof.
• Phone: 05050905846
• Email: tugba.badat[@]hku.edu.tr
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

This study aimed to establish the validity and reliability of the Turkish version of the Pediatric Haemophilia Activities List (short - PedHALshort) questionnaire.

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 50
• Est. completion date: December 30, 2023
• Est. primary completion date: August 30, 2023
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 4 Years to 17 Years

Eligibility

Inclusion Criteria:

• 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

Exclusion Criteria:

• Children with neurological signs that limit activities of daily living

Related Conditions & MeSH terms

• Hemophilia
• Hemophilia A
• Hemophilia A, Severe
• Hemophilia B

Locations

Country	Name	City	State
Turkey	Hasan Kalyoncu University	Gaziantep	Sahinbey

Sponsors (1)

Lead Sponsor	Collaborator
Hasan Kalyoncu University

Country where clinical trial is conducted

Turkey, 

References & Publications (7)

De 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. — View Citation

Schafer 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. — View Citation

Shrout 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. — View Citation

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. — View Citation

Timmer 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. — View Citation

Wagner 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. — View Citation

Young 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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	through of the study, average 6 months
Primary	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.	through of the study, average 6 months