Dr Bill Bartlett Joint Clinical Director. Diagnostics Group, Biochemical Medicine, Ninewells Hospital & Medical School, NHS Tayside, Scotland, UK. Bill.Bartlett@nhs.net www.biologicalvariation.com Analytical variance (CVA ). Within Subject biological variance (CVI ). Between Subject biological variance (CVG ). s2Total = s2Analytical + s2Individual + s2Gro CVTotal = CVA + CVl + CVG Biological Variation Serum Creatinine: Average within subject (CVI) = 4.1% Gowans & Fraser. Ann Clin Biochem 1988:25:259-263 Setting of analytical goals (CVgoal). Quality specifications for : total allowable error (TEA) Bias (BA ) Evaluating the significance of change in serial results (RCV). Assessing the utility of reference intervals (Index of Individuality). Assessing number of specimens required to estimate homeostatic set points. Choice of specimen type. Timing of specimens. eGFR > 60 in a 30 year old white female: Changing renal function? These fundamental data have many applications that under-pin our practice! Are These Not Reference Data? Do we have confidence in the data and understand their limitations? Rodin’s Thinker Burrell Collection Glasgow Are current published biological variation data fit for purpose? Are these data valid and robust? Confidence in method of their production and analysis. Contemporaneously valid. Can I apply them to my practice? Population Demographic Diseased v Well Method Time Frame What are the implications of error Grasbeck & Saris 1969 Introduced the term “reference value”: The mode of generation of such values is known with respect to: Selection of subjects Assessment of state of health Population characteristics, age, sex, Specimen collection and storage Analytical technique and performance characteristics Data handling techniques. 40 years of data The Literature • Do the data travel through time • Method developments Quality Commutable Translated into databases • Enough reported detail. • Good Design? • Population demographics. • Healthy? • Diseased? • Excellent Resources • Granular enough? • Data archetype required? • 319 Constituents: • 90 entries based on 1 Paper 66 quantities 34 diseases with 45 references. “For the majority of quantities studied CVI of same order as diseased. “ Disease specific RCVs may be necessary in some cases. Effect of variability in variability not quantitatively studied. “Heterogeneity in study designs and methods compiled” Data Quality? Experimental Design Data Analysis Assay Characteristics What is the uncertainty? What are the quality standards for BV Data? Standard for Production • Experimental Design • Data Analysis Standard for Reporting • Enable Critical Appraisal • Enable Commutability Standard for Transmission • Data Archetype? • Commutability & Valid Application “Our hope is that the comparability of such data might be provided by use of a common study design and analysis of data” Fraser & Harris 1989 Crit Rev in Clin Lab Sci. 1989;27(5)409-437 www.biologicalvariation.com Generation and Application of data on Biological Variation in Clinical Chemistry: Fraser CG, Harris EK. Crit Rev Clin Lab Sci 1989:27,(5), 409-435. Optimal Conditions Precision Purpose of study Experimental Design Characterisation of the methods Data analysis Confidence limits 1. 2. Define the purpose for which they are to be used. Only meaningful and transferable if defined for the population or individual in terms of: Inclusion and exclusion criteria Intake of food & drugs Physiological and environmental conditions Specimen collection criteria Performance characteristics of the analytical method The statistical methods used for estimation of the limits www.biologicalvariation.com www.biologicalvariation.com/Tools.html CVI 4% to 103% with central tertile 28% to 48% 40 studies with confounding factors: Time period over which samples were collected Study design Type of sample and concentration range studied Population studied and state of health Preanalytical factors Poorly described statistical methods Braga et al Clinica Chimica Acta 2010;411:1606-1610. Highlights the need for this approach “Nine recruited studies were limited by choice of analytic methodology, population selection, protocol application and statistical analysis” Issues: Heterogeneity in experimental model Length of study inappropriate (3 days to 6 months) Methods with differing specificities Statistical methods not specified These data have associated metadata that should remain associated with them to enable appropriate application. Enable comutability Analogous to a reference value. Concept of Archetypes may be relevant Population Demographic Method BV Data Published Reference PUBMED Measurement SNOMED-LOINC Disease SNOMED ISSUES Non-complex v complex molecules. Relativity of normality. Improved assay specificity. HbA1c PTH Creatinine Data in chronic stable disease “often can be considered constant over time and geography” “Same order of magnitude in disease and health” Within Subject Variation (CVI,%) for Serum Sodium and Urea No. of subjects 11 11 62 11 10 14 111 37 274 15 9 15 16 Time Sex status Na+ Urea 0.5 h 8h 1d 2 weeks 4 weeks 8 weeks 15 weeks 22 weeks 6 months 40 weeks 2d 6 weeks 8 weeks m m H H H H H H H H H H RF HP DM 0.6 0.5 0.6 0.7 0.9 0.5 0.6 0.5 0.5 0.7 0.8 0.8 0.8 2.2 6.0 4.8 12.3 14.3 11.3 15.7 11.1 11.2 13.9 6.5 14.5 13.0 Fraser 2001 m m F m m - F m PTH Assays through time 1970’s • C-Terminal RIA 1980’s • Development of IRMA assays 1990’s • Nichols institute Ruled the world • Range of other intact assays with antibodies against a variety of epitopes 2004 • Bioactive PTH Assays with n-terminal specific antibodies If clearance of fragments is not identical in all patients and non diseased patients the apparent biological variation will vary and be assay specific. Assay specificity is an important BV qualifier Historical data may not be always applicable. Study Year Subjects (M:F) State of Health Frequency of Sampling Number of Samples Method 1 1985 10(6:4) Healthy 7D 11 - 27 IE 2 1989 8(?) Diabetic 3-4 D 6 Endosmosis 3 1993 73(?) Diabetic 1M &3M 4 Affin Chrom 4 1994 29 (?) Diabetic 3 M & 12 M ? HPLC IE 5 1998 12(7:5) Healthy 15 D 10 HPLC IE 6 2000 11(0:11) Healthy 7D 5 HPLC IE 7 2000 47(?) Diabetic 6M 4-7 Imm Turbid 8 2002 45 (45:0) Diabetic 7D 12 HPLC Affin 9 2010 38(24:14)a Diabetic 1Y 5 HPLC IE H = Healthy Stud y CVI 1H 1.8 D = Diabetic Analytical Desirable Bias CVG Goal TEA(%) Target 0.9 10. 8 22.6 10 3D 4.2 & 7.1 2.1 & 3.5 13.0 & 22.5 3 & 10 4D 2.4 1.2 7.4 1 5H 1.9 6H 7D <0.7 7.9,5.4, 3.3 3.9 < 0.35 3.8,2.7, 1.8 8D 1.7b 0.8 9D 4.8 4.8 3.3 1.4 2.8 1 7.3 0.8 5.8 4.8 2D 6.8 3.6 RCV N for Homeostatic Setting point 1.8 5.7 0.8 2.9 24.3,16.7, 11.8 12,6,3 14.9 4 1 Jaffe methods Enzymatic methods HPLC ID-MS – reference method Review of the sedimentation process which is caused in normal urine by picric acid and a new reaction of creatinine By M. Jaffe (Submitted to the editor on 26th June 1886) Many points of reference. International Standards State of Health CVI Number of Subjects Length of Studies (days) Number Samples/Sub Healthy Median? 4.3 CRF 5.3 17 21 8 Type 1 DM 5.9 27 56 8 Impaired renal function 6.9 9 2 11 Type 1 DM 6.5 11 56 8 Post renal transplant 11.5 41 90 8 Acute MI 13.4 20 4 19.5 CKD children 13.0 54 540 9 Ricos et al Ann Clin Biochem 2007;44: 343-352 Quantity Units Group Mean CVI CVG Index of Individuality Serum Creatinine µmol/L Male (7) 83.9 3.4 6.8 0.54 Fraser µmol/L Female (8) 71.4 4.9 11.8 0.41 Fraser µmol/L* Whole (15) 77.9 4.1 14.1 0.29 Fraser µmol/L ? ? 5.3 14.2 0.4 BioV Site 4.7 14.4 0.33 Reinhard et al µmol/L** N= 20 77 Male (7) Female(13) * Jaffe ** Enzymatic CVI = 5.3 % CVG = 14.2% CVA =2.7% M G F Route Forward? www.biological variation.com • • Need to assess on a case by case basis. Questions around uncertainty. • What are the implications for their application? • Can the impact of uncertainty be quantified and reduced where necessary. Accepted standard needed for their production. • Critical appraisal checklist required to enable veracity of existing and new publications to be established. • Archetype for transmission. Questions to be addressed by the EFCC biological Variation Working group • Kinoull Hill, Perth Scotland. © Ruth Bartlett