A cumulative analogue joint involvement scale (CAJIS) for fibrodysplasia ossificans progressiva (FOP)

被引：45
作者：
Kaplan, Frederick S. [1 ,2 ,3 ]
Al Mukaddam, Mona [2 ,3 ,5 ]
Pignolo, Robert J. [4 ]
机构：
[1] Univ Penn, Perelman Sch Med, Dept Orthopaed Surg, Philadelphia, PA 19104 USA
[2] Univ Penn, Perelman Sch Med, Dept Med, Philadelphia, PA 19104 USA
[3] Univ Penn, Perelman Sch Med, Ctr Res FOP & Related Disorders, Philadelphia, PA 19104 USA
[4] Mayo Clin, Sch Med, Dept Med, Rochester, MN 55905 USA
[5] Penn Med Univ City, Div Endocrinol Diabet & Metab, Ctr Res FOP & Related Disorders, 3737 Market St,3rd Floor, Philadelphia, PA USA
来源：
BONE | 2017年 / 101卷
关键词：
Fibrodysplasia ossificans progressiva; Heterotopic ossification; Bone morphogenetic protein signaling; ACVR1; Activities of daily living; Ambulation; HETEROTOPIC OSSIFICATION; NATURAL-HISTORY;
D O I：
10.1016/j.bone.2017.04.015
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
Background: Fibrodysplasia ossificans progressiva (FOP) is a catastrophic genetic disorder of progressive heterotopic ossification (HO). Assessment of functional mobility in FOP will be essential to support clinical trials of investigational agents. Results: Of necessity, we developed a simple, rapidly-administered, cumulative analogue joint involvement scale (CAJIS) for FOP based on assessments in 144 individuals worldwide with classic FOP. Conclusions: CAJIS scores correlated with patient age, activities of daily living, and ambulatory function with excellent inter-rater variability. We show here that the CAJIS score provides an accurate and reproducible snapshot of total body and regional mobility burden in FOP. (C) 2017 Elsevier Inc. All rights reserved.
引用
收藏
页码：123 / 128
页数：6
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