Holdsworth Catherine, Bladen Melanie, Harbidge Hannah, Drechsler Wendy, Mahaffey Ryan, Perez-Alenda Sofia, Sayers Fionnuala, Strike Karen, Timmer Merel, Stephensen David
University Hospitals Dorset, Bournemouth UK/Southern Haemophilia Network, Poole, UK.
Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
Haemophilia. 2025 Sep;31(5):840-864. doi: 10.1111/hae.70081. Epub 2025 Jul 20.
Currently, physical health assessments in persons with haemophilia focus on bleed-related events and after-effects. The aim of the systematic review was to review and apply standardised criteria to evaluate reliability, responsiveness and construct validity of performance-based instruments evaluating physical capability in persons with haemophilia.
Medline, CINAHL, Embase, EMCARE, and Cochrane (inception-March 2024) were searched using COSMIN filters for 7 performance-based tests in haemophilia, supplemented by manual searches. Reliability, responsiveness and construct validity of the six-minute walk test (6MWT), timed up and go test (TUG), timed up and down stairs (TUDS), 30-second sit-to-stand (30-STS), single leg stance (SLS), tandem stance (TS) and single hop for distance (SH) were evaluated.
The search yielded 88 abstracts; 25 studies remained after full-text screening, covering 5 of 7 performance-based instruments: 6MWT, TUG, TUDS, SLS, and 30-STS. No performance-based test was evaluated for all properties across all ages. Only TUG in adults and older adults and 6MWT in children and adolescents has been tested for all properties. No test received a high grading. Low and very low grades were given mostly for indeterminate results, small or single studies and lack of a similar construct of comparator. The 6MWT in all age groups was the only performance-based test graded moderate, and this was for responsiveness.
With increasing use of performance-based methods of physical function capacity, evaluating measurement properties is a priority. Until evidence is generated, we can only advocate the 6MWT to monitor responsiveness in adult persons with haemophilia affected with marked arthropathy.
Understanding Physical Health in People with Haemophilia Currently, when we check the physical health of people with haemophilia, we mostly look at problems caused by bleeding. But we wanted to see if there are better ways to measure how well people with haemophilia can move and do daily activities. What We Did We looked through a lot of medical studies (up to March 2024) to find information on 7 specific physical tests. These tests measure things like: How far someone can walk in six minutes (6-minute walk test or 6MWT) How long it takes to stand up, walk a short distance, and sit down (Timed Up and Go test or TUG) How long it takes to go up and down stairs (timed up and down stairs or TUDS) How many times someone can stand up from a chair in 30 seconds (30-second sit-to-stand or 30-STS) How long someone can stand on one leg (single leg stance or SLS) How long someone can stand with one foot directly in front of the other (Tandem Stance or TS) How far someone can hop on one leg (single hop for distance or SH) We wanted to see how reliable (consistent), responsive (can detect changes), and valid (measures what it's supposed to) these tests were for people with haemophilia. What We Found We found 25 studies that looked at 5 of the 7 tests (6MWT, TUG, TUDS, SLS, and 30-STS). Here's what stood out: No single test was good for everything and for all ages. Only the TUG test (for adults) and the 6MWT (for children) had been fully studied for all aspects (reliability, responsiveness, and validity). None of the tests were rated as highly effective overall. Most got low ratings because the results weren't clear, studies were small, or there wasn't enough good information to compare them to. The 6MWT was the only test that received a 'moderate' rating, but only for its ability to show changes in all age groups. What This Means It's becoming more common to use these kinds of physical tests to understand how well people function. So, it's very important to know if these tests work well for people with haemophilia. For now, based on the evidence, we can only suggest using the 6-minute walk test (6MWT) to track how much someone's physical ability changes over time, especially for adults with haemophilia who have severe joint problems. We need more research to find better tests.
目前，血友病患者的身体健康评估主要集中在出血相关事件及其后遗症上。本系统评价的目的是回顾并应用标准化标准，以评估用于评估血友病患者身体能力的基于表现的工具的可靠性、反应性和结构效度。
使用COSMIN筛选器在Medline、CINAHL、Embase、EMCARE和Cochrane（截至2024年3月）中检索血友病患者的7项基于表现的测试，并辅以手工检索。评估了六分钟步行试验（6MWT）、计时起立行走试验（TUG）、上下楼梯计时试验（TUDS）、30秒坐立试验（30-STS）、单腿站立试验（SLS）、串联站立试验（TS）和单腿跳远距离试验（SH）的可靠性、反应性和结构效度。
检索得到88篇摘要；经过全文筛选后，保留了25项研究，涵盖了7项基于表现的工具中的5项：6MWT、TUG、TUDS、SLS和30-STS。没有一项基于表现的测试在所有年龄段都针对所有属性进行了评估。只有成人和老年人的TUG试验以及儿童和青少年的6MWT试验针对所有属性进行了测试。没有一项测试获得高分。低等级和极低等级主要是由于结果不确定、研究规模小或单一以及缺乏类似结构的对照。所有年龄组的6MWT是唯一一项基于表现的测试，评级为中等，这是针对反应性而言。
随着基于表现的身体功能能力评估方法的使用越来越多，评估测量属性成为当务之急。在有证据之前，我们只能提倡使用6MWT来监测患有明显关节病的成年血友病患者的反应性。
了解血友病患者的身体健康状况目前，当我们检查血友病患者的身体健康时，我们主要关注出血引起的问题。但我们想看看是否有更好的方法来衡量血友病患者的运动能力和日常活动能力。我们所做的我们查阅了大量医学研究（截至2024年3月），以获取有关7项特定身体测试的信息。这些测试测量的内容包括：一个人在六分钟内可以走多远（六分钟步行试验或6MWT）站起来、走一小段距离然后坐下需要多长时间（计时起立行走试验或TUG）上下楼梯需要多长时间（上下楼梯计时试验或TUDS）一个人在30秒内从椅子上站起来的次数（30秒坐立试验或30-STS）一个人单腿站立的时间（单腿站立试验或SLS）一个人双脚前后直接站立的时间（串联站立试验或TS）一个人单腿跳远距离（单腿跳远距离试验或SH）我们想看看这些测试对血友病患者的可靠性（一致性）、反应性（能否检测到变化）和有效性（测量其应测量的内容）如何。我们的发现我们发现了25项研究，涉及7项测试中的5项（6MWT、TUG、TUDS、SLS和30-STS）。突出的情况如下：没有一项测试对所有情况和所有年龄都适用。只有TUG试验（针对成年人）和6MWT试验（针对儿童）在所有方面（可靠性、反应性和有效性）都进行了充分研究。没有一项测试总体上被评为高效。大多数测试得到低评级是因为结果不明确、研究规模小或没有足够的良好信息进行比较。6MWT是唯一一项获得“中等”评级的测试，但仅因其在所有年龄组中显示变化的能力。这意味着什么使用这些类型的身体测试来了解人们的功能状况变得越来越普遍。因此，了解这些测试对血友病患者是否有效非常重要。目前，基于现有证据，我们只能建议使用六分钟步行试验（6MWT）来跟踪某人的身体能力随时间的变化情况，特别是对于患有严重关节问题的成年血友病患者。我们需要更多研究来找到更好的测试方法。
