Zhang Ningning, Carcao Manuel, Ignas Danial M, Feldman Brian M, Hilliard Pamela, Moineddin Rahim, Stain Ann Marie, Babyn Paul, Blanchette Victor S, Doria Andrea S
Department of Radiology, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing 100045, China.
Department of Paediatrics, Division of Haematology/Oncology, University of Toronto, The Hospital for Sick Children, Toronto, ON M5G 1X8, Canada.
J Clin Med. 2025 Jul 7;14(13):4792. doi: 10.3390/jcm14134792.
The variety of magnetic resonance imaging (MRI) scales available to measure soft tissue and osteochondral changes in joints of persons with hemophilia poses challenges in evaluating published clinical/research studies. To evaluate the value of four MRI scales [(i) the 17-point International Prophylaxis Study Group [IPSG] additive scale; (ii) and (iii) the compatible IPSG progressive (P) and additive (A) scales; and (iv) the Denver progressive scale] to assess joint change in boys with hemophilia participating in a prospective two-year prophylaxis study. Boys with severe hemophilia A (ages, 7-16 years) followed at the Hospital for Sick Children, Toronto, Canada had MRI evaluations of six index joints (ankles, knees, elbows) at study entry and exit. Musculoskeletal (MSK) outcomes included in the study were the Colorado Child Physical Examination (PE) scale; the Pettersson (X-ray) scale; and the aforementioned 4 MRI scales. Very strong (r ≥ 0.80) correlations were observed between the IPSG 17-point, the IPSG progressive (P) and the Denver MRI scales, and moderate (r = 0.40-0.59) to strong (r = 0.60-0.79) correlations for the IPSG 17 point and the IPSG additive (A) MRI scales. Very weak (r = 0.20-0.39) or no correlations were observed between soft tissue MRI scores and the swelling item of the Child PE scale. All four MRI scales demonstrated relative comparability of their construct validities for assessing mild/moderate hemophilic arthropathy. The 17-point IPSG additive scale is recommended as a reference standard in future long-term studies of young boys with hemophilia receiving factor and non-factor-based preventive therapies.
可用于测量血友病患者关节软组织和骨软骨变化的多种磁共振成像（MRI）量表，给评估已发表的临床/研究带来了挑战。为评估四种MRI量表的价值：（i）17分国际预防研究组（IPSG）累加量表；（ii）和（iii）与之兼容的IPSG进展性（P）量表和累加性（A）量表；以及（iv）丹佛进展性量表，以评估参与一项前瞻性两年预防研究的血友病男孩的关节变化。在加拿大多伦多病童医院接受随访的重度甲型血友病男孩（年龄7至16岁）在研究开始和结束时对六个指标关节（踝关节、膝关节、肘关节）进行了MRI评估。该研究纳入的肌肉骨骼（MSK）结果包括科罗拉多儿童体格检查（PE）量表；彼得森（X线）量表；以及上述四种MRI量表。IPSG 17分、IPSG进展性（P）量表和丹佛MRI量表之间观察到非常强的相关性（r≥0.80），IPSG 17分与IPSG累加性（A）MRI量表之间的相关性为中等（r = 0.40 - 0.59）至强（r = 0.60 - 0.79）。软组织MRI评分与儿童PE量表的肿胀项目之间观察到非常弱的相关性（r = 0.20 - 0.39）或无相关性。所有四种MRI量表在评估轻度/中度血友病性关节病时均显示出其结构效度的相对可比性。建议将17分IPSG累加量表作为未来对接受基于因子和非因子预防治疗的血友病幼童进行长期研究的参考标准。
