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

作者：Kaplan Frederick S^*; Al Mukaddam Mona; Pignolo Robert J^*

来源：Bone, 2017, 101: 123-128.

DOI：10.1016/j.bone.2017.04.015

• Fibrodysplasia ossificans progressiva
• Heterotopic ossification
• Bone morphogenetic protein signaling
• ACVR1
• Activities of daily living
• Ambulation

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.

• 出版日期2017-8