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FAbry STabilization indEX (FASTEX): an innovative tool for the assessment of clinical stabilization in Fabry disease

View Article: PubMed Central - PubMed

ABSTRACT

Two disease severity scoring systems, the Mainz Severity Score Index (MSSI) and Fabry Disease Severity Scoring System (DS3), have been validated for quantifying the disease burden of Fabry disease. We aimed to develop a dynamic mathematical model [the FASTEX (FAbry STabilization indEX)] to assess the clinical stability. A multidisciplinary panel of experts in Fabry disease first defined a novel score of severity [raw score (RS)] based on three domains with a small number items in each domain (nervous system domain: pain, cerebrovascular events; renal domain: proteinuria, glomerular filtration rate; cardiac domain: echocardiography parameters, electrocardiograph parameters and New York Heart Association class) and evaluated the clinical stability over time. The RS was tested in 28 patients (15 males, 13 females) with the classic form of Fabry disease. There was good statistical correlation between the newly established RS and a weighted score (WS), with DS3 and MSSI (R2 = 0.914, 0.949, 0.910 and 0.938, respectively). In order to refine the RS further, a WS, which was expressed as a percentage value, was calculated. This was based on the relative clinical significance of each item within the domain with the panel agreeing on the attribution of a different weight of clinical damage to a specific organ system. To test the variation of the clinical burden over time, the RS was repeated after 1 year. The panel agreed on a cut-off of a 20% change from baseline as the clinical WS to define clinical stability. The FASTEX model showed good correlation with the clinical assessment and with clinical variation over time in all patients.

No MeSH data available.


(A) Percentage severity of single domains and total severity score. (B) Total percentages of severity for single domains and total severity score at first and second visit calculated by applying the algorithm.
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SFW082F2: (A) Percentage severity of single domains and total severity score. (B) Total percentages of severity for single domains and total severity score at first and second visit calculated by applying the algorithm.

Mentions: All combinations of scores for each domain system (nervous, renal and cardiac) calculated by applying the algorithm specified above are provided in the Supplementary data. These values represent all possible combinations of percentages for organ damage subdivided for each domain (Figures 2 and 3).Fig. 2.


FAbry STabilization indEX (FASTEX): an innovative tool for the assessment of clinical stabilization in Fabry disease
(A) Percentage severity of single domains and total severity score. (B) Total percentages of severity for single domains and total severity score at first and second visit calculated by applying the algorithm.
© Copyright Policy - creative-commons
Related In: Results  -  Collection

License
Show All Figures
getmorefigures.php?uid=PMC5036909&req=5

SFW082F2: (A) Percentage severity of single domains and total severity score. (B) Total percentages of severity for single domains and total severity score at first and second visit calculated by applying the algorithm.
Mentions: All combinations of scores for each domain system (nervous, renal and cardiac) calculated by applying the algorithm specified above are provided in the Supplementary data. These values represent all possible combinations of percentages for organ damage subdivided for each domain (Figures 2 and 3).Fig. 2.

View Article: PubMed Central - PubMed

ABSTRACT

Two disease severity scoring systems, the Mainz Severity Score Index (MSSI) and Fabry Disease Severity Scoring System (DS3), have been validated for quantifying the disease burden of Fabry disease. We aimed to develop a dynamic mathematical model [the FASTEX (FAbry STabilization indEX)] to assess the clinical stability. A multidisciplinary panel of experts in Fabry disease first defined a novel score of severity [raw score (RS)] based on three domains with a small number items in each domain (nervous system domain: pain, cerebrovascular events; renal domain: proteinuria, glomerular filtration rate; cardiac domain: echocardiography parameters, electrocardiograph parameters and New York Heart Association class) and evaluated the clinical stability over time. The RS was tested in 28 patients (15 males, 13 females) with the classic form of Fabry disease. There was good statistical correlation between the newly established RS and a weighted score (WS), with DS3 and MSSI (R2 = 0.914, 0.949, 0.910 and 0.938, respectively). In order to refine the RS further, a WS, which was expressed as a percentage value, was calculated. This was based on the relative clinical significance of each item within the domain with the panel agreeing on the attribution of a different weight of clinical damage to a specific organ system. To test the variation of the clinical burden over time, the RS was repeated after 1 year. The panel agreed on a cut-off of a 20% change from baseline as the clinical WS to define clinical stability. The FASTEX model showed good correlation with the clinical assessment and with clinical variation over time in all patients.

No MeSH data available.