Comparison of 30-Second Sit-to-Stand Test Performance Between Individuals With Hemophilia and Healthy Peers

Официальное название

Comparison of 30-Second Sit-to-Stand Test Performance Between Individuals With Hemophilia and Healthy Peers: A Cross-Sectional Observational Study

Обзор

Hemophilia is an inherited bleeding disorder characterized by recurrent musculoskeletal bleeding, particularly into joints, leading to impaired physical function and reduced functional performance. The 30-Second Sit-to-Stand Test (30STS) is a simple, reliable, and valid measure of lower extremity functional performance; however, evidence regarding its performance in individuals with hemophilia remains limited. This cross-sectional observational study aims to compare 30STS performance between adults with hemophilia and age-matched healthy controls and to investigate the relationship between 30STS performance, joint health, and activity level in individuals with hemophilia. Fifty-eight participants (29 individuals with hemophilia and 29 healthy controls) aged 18-35 years will be recruited. Participants will complete the 30STS. Individuals with hemophilia will additionally undergo joint health assessment using the Hemophilia Joint Health Score (HJHS) and activity evaluation using the Hemophilia Activities List (HAL).

Подробное описание

Hemophilia is an inherited bleeding disorder caused by deficiency of coagulation factor VIII (hemophilia A) or factor IX (hemophilia B), resulting in recurrent bleeding episodes. Repeated musculoskeletal bleeding, particularly hemarthrosis, may lead to progressive joint damage, reduced physical activity, muscle weakness, and impaired functional performance. Therefore, objective assessment of physical function is important for monitoring functional limitations and planning appropriate rehabilitation strategies in individuals with hemophilia.

The 30-Second Sit-to-Stand Test (30STS) is a simple, practical, and reliable performance-based assessment used to evaluate lower extremity strength, endurance, and functional mobility. Although the test is widely used in different populations, evidence regarding 30STS performance in individuals with hemophilia is limited. Comparing functional performance between individuals with hemophilia and healthy peers may provide valuable information regarding the functional impact of hemophilia and may contribute to clinical assessment approaches.

The aim of this cross-sectional observational study is to compare 30STS performance between individuals with hemophilia and age-matched healthy controls. Additionally, the study aims to investigate the relationship between 30STS performance, joint health status, and activity level in individuals with hemophilia.

A total of 58 participants aged 18-35 years will be included in the study, consisting of 29 individuals diagnosed with hemophilia A or hemophilia B and 29 age-matched healthy controls. All participants will perform the 30STS. Demographic characteristics, including age, height, and body weight, will be recorded. For participants with hemophilia, additional clinical information including hemophilia type, disease severity, bleeding history, and prophylaxis status will be collected.

Joint health status of individuals with hemophilia will be evaluated using the Hemophilia Joint Health Score (HJHS), and functional activity level will be assessed using the Hemophilia Activities List (HAL). The results of the 30STS will be compared between groups, and associations between functional performance, joint health, and activity level will be analyzed within the hemophilia group.

The findings of this study may contribute to a better understanding of lower extremity functional performance in individuals with hemophilia and may provide clinically relevant information for functional evaluation and rehabilitation planning

Первичные конечные точки

• 30-Second Sit-to-Stand Test Performance [Срок оценки: Baseline assessment (single assessment visit)]

Вторичные конечные точки (2)

• Joint Health Status Assessed by Hemophilia Joint Health Score (HJHS) [Срок оценки: Baseline assessment (single assessment visit)]
• Hemophilia Activities List (HAL) [Срок оценки: Baseline assessment (single assessment visit)]

Критерии участия

Критерии включения

Hemophilia Group:

• Diagnosis of hemophilia A or hemophilia B
• Age between 18 and 35 years
• Ability to walk independently
• Willingness to participate in the study voluntarily

Healthy Control Group:

• Age between 18 and 35 years
• No history of hemophilia or other bleeding disorders
• No history of lower extremity surgery or serious lower extremity injury within the previous 6 months
• No neurological, rheumatological, or severe orthopedic disorders
• Willingness to participate in the study voluntarily

Критерии исключения

Hemophilia Group:

\- Acute joint bleeding episode within the previous 3 months

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Да

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

Turkey (Türkiye) · 1 центр

• Bahcesehir university — Istanbul

Публикации

• van Genderen FR, van Meeteren NL, van der Bom JG, Heijnen L, de Kleijn P, van den Berg HM, Helders PJ. Functional consequences of haemophilia in adults: the development of the Haemophilia Activities List. Haemophilia. 2004 Sep;10(5):565-71. doi: 10.1111/j.1365-2516.2004.01016.x. PMID 15357785
• Kuijlaars IAR, van der Net J, Feldman BM, Aspdahl M, Bladen M, de Boer W, Cuesta-Barriuso R, Matlary RED, Funk SM, Hilliard P, John JA, Kempton CL, de Kleijn P, Manco-Johnson M, Petrini P, Poonnoose P, St-Louis J, Thomas S, Timmer MA, Trakymiene SS, van Vlimmeren L, Fischer K. Evaluating international Haemophilia Joint Health Score (HJHS) results combined with expert opinion: Options for a shorter PMID 33058441
• McKay MJ, Baldwin JN, Ferreira P, Simic M, Vanicek N, Burns J; 1000 Norms Project Consortium. Reference values for developing responsive functional outcome measures across the lifespan. Neurology. 2017 Apr 18;88(16):1512-1519. doi: 10.1212/WNL.0000000000003847. Epub 2017 Mar 22. PMID 28330961
• Mannucci PM. Hemophilia therapy: the future has begun. Haematologica. 2020 Mar;105(3):545-553. doi: 10.3324/haematol.2019.232132. Epub 2020 Feb 14. PMID 32060150
• Stonebraker JS, Bolton-Maggs PH, Michael Soucie J, Walker I, Brooker M. A study of variations in the reported haemophilia B prevalence around the world. Haemophilia. 2012 May;18(3):e91-4. doi: 10.1111/j.1365-2516.2011.02588.x. Epub 2011 Jun 7. PMID 21649801
• Mannucci PM. Hemophilia: treatment options in the twenty-first century. J Thromb Haemost. 2003 Jul;1(7):1349-55. doi: 10.1046/j.1538-7836.2003.00262.x. PMID 12871268
• Peyvandi F, Bolton-Maggs PH, Batorova A, De Moerloose P. Rare bleeding disorders. Haemophilia. 2012 Jul;18 Suppl 4:148-53. doi: 10.1111/j.1365-2516.2012.02841.x. PMID 22726099

Идентификаторы

NCT: NCT07736976 · E-78097791-020-5767

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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