BMR - RBM ANNUAL DATA REPORT CYSTIC FIBROSIS BELGIUM FINAL VERSION (2006)
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1 BMR - RBM Belgisch Mucoviscidose Register Registre Belge de la Mucoviscidose J. Wytsmanstraat 14 J. Wytsmanstraat Brussel 1050 Bruxelles Tel : 02/ Fax : 02/ ANNUAL DATA REPORT CYSTIC FIBROSIS BELGIUM FINAL VERSION (2006). Suggested citation: Belgisch Mucoviscidose Register - Registre Belge de la Mucoviscidose, 2006; Annual Data Report, Belgium, Updated October 2008
2 Coordinator H. Jansen WIV-ISP Collaborating Scientist M. Thomas WIV-ISP Scientific Committee P. Lebecque Cliniques Univ. St. Luc, Bruxelles C. Knoop Hôpital Erasme, Bruxelles K. Desager UZ Antwerpen, Antwerpen A. Malfroot UZ Brussel, Brussel S. Vandaele UZ Gent, Gent K. De Boeck UZ Leuven, Leuven Participants to the Registry J.P. Sacré CHR de la Citadelle, Liège F. Pierart Clinique de l'espérance, Montegnée P. Lebecque Cliniques Univ. St. Luc, Bruxelles C. Knoop Hôpital Erasme, Bruxelles G. Casimir HUDERF, Bruxelles I. Stappaerts St. Vincentiusziekenhuis, Antwerpen L. Van Schil St. Vincentiusziekenhuis, Antwerpen K. Desager UZ Antwerpen, Antwerpen A. Malfroot UZ Brussel, Brussel F. De Baets UZ Gent, Gent G. Joos UZ Gent, Gent S. Van Daele UZ Gent, Gent K. De Boeck UZ Leuven, Leuven L. Dupont UZ Leuven, Leuven M. Moens UZ Leuven, Leuven and their co-workers WIV-ISP team members V. Van Casteren Programme responsible M. Thomas Project responsible H. Jansen Coordinator BMR-RBM S. S. Wanyama Biostatistician M-F. Lafontaine Informatician, data management Y. Pirson Administrative We would like to acknowledge and thank Claude Sevens (former coordinator) for starting the BMR-RBM. BMR - RBM Annual Data Report
3 TABLE OF CONTENTS GENERAL DATA... 7 AGE ON DECEMBER 31, DISTRICT OF BIRTH DISTRICT OF RESIDENCE CF DIAGNOSIS SUGGESTED BY...13 AGE AT DIAGNOSIS AGE AT DIAGNOSIS FOR NEW 2006 CASES DNA ANALYSIS Mutation...17 Δf508 and other pairs...18 Homozygotes...18 BMI Z-SCORES BY GENDER USING CACHERA REFERENCES * BMI Z-SCORES BY GENDER USING FLEMISH GROWTH VALUES HEIGHT Z-SCORES BY GENDER USING FLEMISH GROWTH VALUES WEIGHT Z-SCORES BY GENDER USING FLEMISH GROWTH VALUES HEIGHT AND LENGTH Z-SCORES BY GENDER USING FLEMISH GROWTH VALUES GRAPHICAL COMPARISONS OF Z SCORES BY GENDER.. 25 BMI, HEIGHT & WEIGHT PERCENTILES BMI Z-SCORE PERCENTILES USING CACHERA REFERENCE BY AGE GROUP BMI Z-SCORE PERCENTILES USING THE FLEMISH GROWTH VALUES BY AGE GROUP HEIGHT Z-SCORE PERCENTILES USING THE FLEMISH GROWTH VALUES BY AGE GROUP HEIGHT AND LENGTH Z-SCORE PERCENTILES BY AGE GROUP WEIGHT Z-SCORE PERCENTILES USING THE FLEMISH GROWTH VALUES BY AGE GROUP LUNG FUNCTION MEAN % PREDICTED FEV1 USING KNUDSON EQUATIONS MEAN % PREDICTED FEV1 USING KNUDSON EQUATIONS BY AGE MEAN % PREDICTED FEV1 USING KNUDSON EQUATIONS BY AGE AND SEX MEAN % PREDICTED FEV1 USING KNUDSON EQUATIONS BY AGE GROUPS MEAN % PREDICTED FEV1 USING COMBINED WANG - HANKINSON EQUATIONS MEAN % PREDICTED FEV1 USING WANG - HANKINSON EQUATIONS BY AGE MEAN % PREDICTED FEV1 USING WANG - HANKINSON EQUATIONS BY AGE AND SEX MEAN % PREDICTED FEV1 USING WANG - HANKINSON EQUATIONS BY AGE GROUPS BMR - RBM Annual Data Report
4 .Table of contents MEAN % PREDICTED FVC USING KNUDSON EQUATIONS MEAN % PREDICTED FVC USING KNUDSON EQUATIONS BY AGE MEAN % PREDICTED FVC USING KNUDSON EQUATIONS BY AGE AND SEX MEAN % PREDICTED FVC USING KNUDSON EQUATIONS BY AGE GROUPS MEAN % PREDICTED FVC USING WANG - HANKINSON EQUATIONS MEAN % PREDICTED FVC USING WANG - HANKINSON EQUATIONS BY AGE MEAN % PREDICTED FVC USING WANG - HANKINSON EQUATIONS BY AGE AND SEX MEAN % PREDICTED FVC USING WANG - HANKINSON EQUATIONS BY AGE GROUPS RESPIRATORY CULTURE RESULTS POINT PREVALENCE OF PATHOGENS ISOLATED RELATED TO AGE COMPLICATIONS IN COMPLICATIONS RATES BY AGE RESPIRATORY GASTRO-INTESTINAL MISCELLANEOUS TOTAL NUMBER OF VISITS TO CF CARE CENTERS TOTAL NUMBER OF DAYS OF HOSPITALIZATION IN CF CARE CENTERS...69 HOSPITALIZATION BY AGE GROUPS THERAPY Antibiotic Therapy (Ab)...71 Antibiotic Therapy IV...71 Use of Oxygen Therapy...71 Oxygen Therapy Type...71 Use of inhalation Therapy...72 Number of inhalation drugs...72 Inhalation type Administered...72 Anti-inflammatory Aid type...72 Pancreatic enzymes...73 Fat soluble vitamins (ADEK)...73 Ursodeoxycholic acid...73 Parenteral feeding...73 Enteral feeding...74 Enteral feeding type...74 Insulin therapy* 74 Physiotherapy...74 BMR - RBM Annual Data Report
5 .Table of contents IV ANTIBIOTICS (AB) BY AGE GROUPS TRANSPLANT INFORMATION Transplant Status...76 Year and type of transplant...76 AGE AT DEATH Age at death...77 Primary cause of death...79 Primary cause of death by year...79 SOCIAL ALLOWANCES SPECIFIC BENEFITS...80 MISSING DATA BMR - RBM Annual Data Report
6 LIST OF FIGURES Figure 1: Age on Dec 31, Figure 2: Age on Dec 31, 2006 by gender Figure 3: Age at Diagnosis Figure 4: Comparisons of Z-Scores by gender Figure 5: Cachera BMI Z-Score percentiles by age Figure 6: Flemish BMI Z-Score percentiles by age Figure 7: Flemish Height Z-Score percentiles by age Figure 8: Flemish Height and Length Z-Score percentiles by age Figure 9: Flemish Weight Z-Score percentiles by age Figure 10: % predicted FEV1 using Knudson equations Figure 11: % predicted FEV1 using Knudson equations for children Figure 12: % predicted FEV1 using Knudson equations for adults Figure 13: % predicted FEV1 using Knudson equations by age Figure 14: % predicted FEV1 using Knudson equations by age and gender Figure 15: % predicted FEV1 severity groups using Knudson equations Figure 16: % predicted FEV1 using Wang Hankinson equations Figure 17: % predicted FEV1 using Wang Hankinson equations for children Figure 18: % predicted FEV1 using Wang Hankinson equations for adults Figure 19: % predicted FEV1 using Wang Hankinson equations by age Figure 20: % predicted FEV1 using Wang Hankinson equations by age and gender Figure 21: % predicted FEV1 severity groups using Wang - Hankinson equations Figure 22: % predicted FVC using Knudson equations Figure 23: % predicted FVC using Knudson equations for children Figure 24: % predicted FVC using Knudson equations for adults Figure 25: % predicted FVC using Knudson equations by age Figure 26: % predicted FVC using Knudson equations by age and gender Figure 27: % predicted FVC severity groups using Knudson equations Figure 28: % predicted FVC using Wang Hankinson equations Figure 29: % predicted FVC using Wang Hankinson equations for children Figure 30: % predicted FVC using Wang Hankinson equations for adults Figure 31: % predicted FVC using Wang Hankinson equations by age Figure 32: % predicted FVC using Wang Hankinson equations by age and gender Figure 33: % predicted FVC severity groups using Wang - Hankinson equations Figure 34 (a): Point prevalence related to age Figure 34 (b): Point prevalence related to age Figure 35 (a): Respiratory complications by age Figure 35 (b): Gastro-intestinal complications by age Figure 35 (c): Miscellaneous complications by age Figure 36: Number of hospitalization days Figure 37: Hospitalization days versus age Figure 38: Mean IV days at home and in hospital Figure 39: Age at death (all notified cases) Figure 40: Number of reported deaths by year BMR - RBM Annual Data Report
7 GENERAL DATA number of CF patients 1036 number of CF patients with complete registration (registered deceased patients 2006 included) 1026 number of CF patients without observation (postulated alive with no observation in 2006 and/or not registered as deceased) 10 new diagnosis in median age (years) 17.0 age range (years) males (%) 51.4 adults 18 years (%) 48.4 median age at diagnosis (months) 7.2 age range at diagnosis (years) number of transplants performed in deaths reported occurring in median age at death (years) 36 age range at death (years) BMR - RBM Annual Data Report
8 AGE ON DECEMBER 31, 2006 age (years) males females all patients n cum n cum % n cum n cum % n cum n cum % 2 Months Months Months Months Months Months Months Months BMR - RBM Annual Data Report
9 Age on December 31, 2006 age (years) males females all patients n cum n cum % n cum n cum % n cum n cum % total The overall median age at 31, December 2006 is 17.0 years. The median age is 16.3 and 17.5 for male and female patients respectively. BMR - RBM Annual Data Report
10 Age (years) Males Females n patients Figure 1: Age on Dec 31, Cumulative % Males Females All Patients 20 0 < Age (Years) Figure 2: Age on Dec 31, 2006 by gender BMR - RBM Annual Data Report
11 DISTRICT OF BIRTH district n % district n % Aalst Roeselare Antwerpen Soignies Arlon St-Niklaas Ath Thuin Bastogne Tielt Brugge Tongeren Brussel-Bruxelles Tournai Charleroi Turnhout Dendermonde Verviers Diksmuide Veurne Dinant Virton Eeklo Gent Halle-Vilvoorde Hasselt subtotal 842 Huy foreigners 17 Ieper missing 177 Kortrijk total 1036 Leuven Liège Maaseik Marche-en-Famenne Mechelen Moeskroen-Mouscron Mons Namur Neufchâteau Nivelles Oostende Oudenaarde BMR - RBM Annual Data Report
12 DISTRICT OF RESIDENCE district n % district n % Aalst Roeselare Antwerpen Soignies Ath St-Niklaas Bastogne Thuin Brugge Tielt Brussel-Bruxelles Tongeren Charleroi Tournai Dendermonde Turnhout Diksmuide Verviers Dinant Veurne Eeklo Virton Gent Waremme Halle-Vilvoorde Hasselt Huy subtotal 996 Ieper foreigners 10 Kortrijk missing 30 Leuven total 1036 Liège Maaseik Marche-en-Famenne Mechelen Moeskroen-Mouscron Mons Namur Neufchâteau Nivelles Oostende Oudenaarde Philippeville 5 0.5
13 CF DIAGNOSIS SUGGESTED BY all data new cases 2006 n % n % Persistent respiratory problems Failure to thrive Chronic diarrhea/streatorrhea/malabsorption Meconium ileus Neonatal screening test Family history Acute respiratory symptoms Diagnosis other Nasal polyposis / chronic sinusitis Rectal prolapse Intestinal obstruction (other than meconium ileus) Prenatal diagnosis Dehydratation / electrolyte imbalance Infertility Neonatal jaundice *Missing (n patients) 95 2 *Missing are patients who did not have information on any of the above reasons given Reasons for diagnosis are not mutually exclusive There were 52 new CF diagnoses in 2006 BMR - RBM Annual Data Report
14 AGE AT DIAGNOSIS (CHILDREN) diagnosis Age (years) males females all patients n cum n cum % n cum n cum % n cum n cum % < 1 Month Month Months Months Months Months Months Months Months Months Months Months BMR - RBM Annual Data Report
15 Age at diagnosis (Adults) diagnosis Age (years) males females all patients n cum n cum % n cum n cum % n cum n cum % missing total The median age at diagnosis for the entire data for 2006 is 7.2 months. The median age at diagnosis is 6.9 months for males and 7.8 months for females. 34 patients had no known diagnosis age BMR - RBM Annual Data Report
16 n patients Cumulative % < 1 Month 1-3 months 4-6 months 7-11 months 1 year 2 years 3 years 4 years 5 years 6 years 7 years 8 years 9 years 10 years Age (years) 11 years 12 years 13 years 14 years years years years years > 35 years Figure 3: Age at Diagnosis The median age at diagnosis is 7.2 months, and at the age of 18 years 92.7% of the patients are diagnosed. AGE AT DIAGNOSIS FOR NEW 2006 CASES diagnosis age males females all patients (years) n cum n cum % n cum n cum % n cum n cum % < 1 Month Month Months Months Months Months Months total The median age at diagnosis for the new cases is 18.0 months; ( 39.4 months for males and 9.1 months for females) BMR - RBM Annual Data Report
17 DNA ANALYSIS Genotyped n % Executed Not Executed Not available subtotal 1010 missing 26 total 1036 Mutation n % Mutation n % Δf delT G542X insT N1303K G->T G->A K A->G delGG R117H delA S1251N G->A G->A delAG R553X E656X W1282X G458V A455E G551D R1162X L165S [delta]i L227R AA->G Q1313X L927P R1070W kbC->T R347P delTT S1255P E60X Y1092X W401X poly-t tract variations others delC not identified D1152H result unknown G970R subtotal 1968 R334W missing G->A total 2072 G85E Y913C *The most common mutation is Δf508 with a prevalence of 69.5%. BMR - RBM Annual Data Report
18 Δf508 and other pairs Mutation pair n % cumulative % Δf Δf Δf OTHER Δf NI OTHER---OTHER OTHER---NI NI---NI subtotal 963 missing 73 total 1036 * NI = Not Identified The mutation formally known as 'Poly-T tract variations' was treated as Not Identified Homozygotes Homozygote n % Δf N1303K G542X R1162X delA G->A delAG delTT G->T G551D L227R [delta]i total BMR - RBM Annual Data Report
19 BMI Z-SCORES BY GENDER USING CACHERA REFERENCES * All data from patients with a transplant were excluded from the BMI analysis. age (years) males females n mean (std.dev) n mean (std.dev) Total < (1.04) (0.96) (1.20) (0.86) (0.82) (1.29) (0.86) (0.84) (1.07) (1.60) (1.08) (1.08) (1.21) (1.17) (1.00) (1.13) (1.11) (1.14) (1.41) (1.14) (1.25) (1.44) (1.21) (1.13) (1.13) (1.10) (1.10) (1.12) (1.10) (1.69) (1.19) (0.81) (1.35) (1.26) (1.06) (0.80) (1.33) (1.14) (1.65) (1.42) (1.19) (1.27) (1.66) (1.22) (0.97) (0.43) (0.99) (0.78) (1.28) (1.15) (1.68) (0.94) (1.14) (0.99) (1.33) (0.86) (1.15) (1.03) (0.74) (0.67) 14 *BMI Z-score not estimable for females of age > 56.0 years. *Rolland-Cachera MF, Cole TJ, Sempé M, Tichet J, Rossignol C, Charraud A. Body mass index variations: centiles from birth to 87 years. Eur J Clin Nutr 1991; 45: *Rolland-Cachera MF: personal communication *Cole TJ. The LMS method for constructing normalised growth standards. Eur J Clin Nutr 1990; 44: BMR - RBM Annual Data Report
20 . BMI Z-scores By Gender using Cachera references age (years) males females n mean (std.dev) n mean (std.dev) Total (1.30) (0.63) (1.40) (0.27) (0.92) (0.72) (1.33) (0.10) (0.78) (0.87) (1.81) (1.07) (.) (1.29) (0.12) (1.79) (2.15) (1.71) (1.00) (1.37) (1.13) (.) (0.35) (0.84) (0.54) (1.83) (.) (.) (1.14) (.) (0.76) (.) (.) (0.36) (.) (.) (.) (0.14) (.) (1.07) 3 subtotal transplants missing total All transplant patients are omitted from this analysis. 922 patients were used. *BMI Z-score not estimable for females of age > 56.0 years. *Rolland-Cachera MF, Cole TJ, Sempé M, Tichet J, Rossignol C, Charraud A. Body mass index variations: centiles from birth to 87 years. Eur J Clin Nutr 1991; 45: *Rolland-Cachera MF: personal communication *Cole TJ. The LMS method for constructing normalised growth standards. Eur J Clin Nutr 1990; 44: BMR - RBM Annual Data Report
21 BMI Z-SCORES BY GENDER USING FLEMISH GROWTH VALUES The Flemish Growth Charts 2-20 years are based on a representative sample of 7920 Flemish boys and 8176 Flemish girls, examined between , and are appropriate for the evaluation of the normality of growth of children who have at least one parent of Flemish origin, and with possibly the other parent from Wallonia, or from; The Netherlands, Germany, Luxemburg, France, England, Denmark, Ireland, Norway, Sweden, Austria, and are based on measurements made by the same techniques and with identical measuring devices using standardized equipment and methodology. For more details and instructions, refer to In this analysis, BMI, Height, Weight and Height and Length reference values from the above study will be used. age (years) males females n mean (std.dev) n mean (std.dev) Total (0.79) (1.65) (0.88) (0.88) (1.04) (1.57) (1.09) (0.94) (1.02) (1.08) (0.85) (0.98) (0.92) (0.84) (1.35) (1.04) (1.01) (1.20) (0.98) (0.92) (1.04) (0.93) (1.05) (1.02) (1.00) (1.60) (1.16) (0.78) (1.28) (1.22) (1.08) (0.73) (1.49) (1.12) (1.89) (1.33) (1.63) (1.13) 6 subtotal transplants missing total Analysis only available for ages years *H/ EP/2-20 Vrije Universiteit Brussel, Antropogenetica & Katholieke Universiteit Leuven, Jeugdgezondheidszorg vub, kul 2004 BMR - RBM Annual Data Report
22 HEIGHT Z-SCORES BY GENDER USING FLEMISH GROWTH VALUES All data from patients with a transplant were excluded from the Height analysis. age (years) males females n mean (std.dev) n mean (std.dev) Total (0.75) (1.17) (0.70) (0.61) (0.85) (1.01) (1.17) (1.05) (0.91) (0.99) (1.09) (1.24) (1.00) (0.93) (1.14) (1.30) (1.08) (0.94) (0.95) (1.30) (1.12) (0.91) (0.98) (1.29) (1.30) (1.27) (2.11) (0.97) (1.45) (1.63) (0.79) (1.04) (1.41) (0.93) (1.53) (0.97) (1.49) (1.36) 6 subtotal transplants missing total Analysis only available for ages years BMR - RBM Annual Data Report
23 WEIGHT Z-SCORES BY GENDER USING FLEMISH GROWTH VALUES All data from patients with a transplant were excluded from the Weight analysis. age (years) males females n mean (std.dev) n mean (std.dev) Total < (1.67) (0.97) (1.15) (1.05) (0.93) (1.22) (0.81) (0.44) (1.06) (1.29) (1.23) (0.73) (0.86) (1.07) (0.95) (1.04) (0.93) (0.76) (1.22) (1.00) (1.01) (0.94) (0.84) (0.99) (0.93) (0.96) (0.97) (1.45) (1.23) (1.91) (2.40) (1.07) (1.73) (1.63) (1.23) (1.00) (2.12) (1.24) (2.79) (1.47) (2.12) (1.73) 6 subtotal transplants missing total Analysis only available for ages years BMR - RBM Annual Data Report
24 HEIGHT AND LENGTH Z-SCORES BY GENDER USING FLEMISH GROWTH VALUES All data from patients with a transplant were excluded from the Height & Length analysis. age (years) males females n mean (std.dev) n mean (std.dev) Total < (1.22) (0.87) (0.92) (1.24) (0.76) (1.15) (0.70) (0.61) (0.85) (1.01) (1.17) (1.05) (0.91) (0.99) (1.09) (1.24) (1.00) (0.93) (1.14) (1.30) (1.08) (0.94) (0.95) (1.30) (1.12) (0.91) (0.98) (1.29) (1.30) (1.27) (2.11) (0.97) (1.45) (1.63) (0.79) (1.04) (1.41) (0.93) (1.53) (0.97) (1.49) (1.36) 6 subtotal transplants missing total Analysis only available for ages years BMR - RBM Annual Data Report
25 GRAPHICAL COMPARISONS OF Z SCORES BY GENDER Males Females Males Females Cachera BMI Z-Score Mean + 2(SD) Mean - 2(SD) Cachera BMI Z-Score Mean + 2(SD) Mean - 2(SD) Flemish BMI Z-Score Mean + 2(SD) Mean - 2(SD) Flemish BMI Z-Score Mean + 2(SD) Mean - 2(SD) Age (years) Age (years) Age (years) Age (years) Cachera BMI Z-Score Flemish BMI Z-Score Males Females Males Females Height Z-Score Mean + 2(SD) Mean - 2(SD) Height Z-Score Mean + 2(SD) Mean - 2(SD) Height & Length Z-Score Mean + 2(SD) Mean - 2(SD) Height & Length Z-Score Mean + 2(SD) Mean - 2(SD) Age (years) Age (years) Age (years) Age (years) Flemish Height Z-Score Flemish Height & Length Z-Score Males Females Weight Z-Score Mean + 2(SD) Mean - 2(SD) Weight Z-Score Mean + 2(SD) Mean - 2(SD) Age (years) Age (years) Flemish Weight Z-Score BMR - RBM Annual Data Report Figure 4: Comparisons of Z-Scores by gender
26 BMI, HEIGHT & WEIGHT PERCENTILES BMI Z-SCORE PERCENTILES USING CACHERA REFERENCE BY AGE GROUP age groups Percentiles n % n % n % n % n % n % n % n % < p p p p p p p subtotal transplants missing total Cachera BMI percentiles are based on ages years for Males(56.0 Females) A total of 922 patients were used. Cachera BMI percentiles by age 100% 90% Percentage 80% 70% 60% 50% 40% 30% 20% 10% > = p90 p75-90 p50-75 p25-50 p10-25 p5-10 < p5 0% > = 45 Age groups Figure 5: Cachera BMI Z-Score percentiles by age BMR - RBM Annual Data Report
27 BMI Z-SCORE PERCENTILES USING THE FLEMISH GROWTH VALUES BY AGE GROUP age groups Percentiles n % n % n % n % < p p p p p p p subtotal transplants missing total Flemish BMI percentiles are based on ages years A total of 527 patients were used. Flemish BMI percentiles by age 100% 90% Percentage 80% 70% 60% 50% 40% 30% 20% 10% > = p90 p75-90 p50-75 p25-50 p10-25 p5-10 < p5 0% Age groups Figure 6: Flemish BMI Z-Score percentiles by age BMR - RBM Annual Data Report
28 HEIGHT Z-SCORE PERCENTILES USING THE FLEMISH GROWTH VALUES BY AGE GROUP age groups Percentiles n % n % n % n % < p p p p p p p subtotal transplants missing total Height percentiles are based on ages years A total of 527 patients were used. Flemish Height percentiles by age 100% 90% Percentage 80% 70% 60% 50% 40% 30% 20% 10% > = p90 p75-90 p50-75 p25-50 p10-25 p5-10 < p5 0% Age groups Figure 7: Flemish Height Z-Score percentiles by age BMR - RBM Annual Data Report
29 HEIGHT AND LENGTH Z-SCORE PERCENTILES BY AGE GROUP age groups Percentiles n % n % n % n % n % < p p p p p p p subtotal transplants missing total Height for Length percentiles are based on ages years A total of 579 patients were used. Flemish Height & Length percentiles by age 100% 90% Percentage 80% 70% 60% 50% 40% 30% 20% 10% > = p90 p75-90 p50-75 p25-50 p10-25 p5-10 < p5 0% Age groups Figure 8: Flemish Height and Length Z-Score percentiles by age BMR - RBM Annual Data Report
30 WEIGHT Z-SCORE PERCENTILES USING THE FLEMISH GROWTH VALUES BY AGE GROUP age groups Percentiles n % n % n % n % n % < p p p p p p p subtotal transplants missing total Weight percentiles are based on ages years A total of 582 patients were used. Flemish Weight percentiles by age 100% 90% Percentage 80% 70% 60% 50% 40% 30% 20% 10% > = p90 p75-90 p50-75 p25-50 p10-25 p5-10 < p5 0% Age groups Figure 9: Flemish Weight Z-Score percentiles by age BMR - RBM Annual Data Report
31 LUNG FUNCTION All data from patients less than 6 years and those with a transplant were excluded from the lung function analysis. MEAN % PREDICTED FEV1 USING KNUDSON EQUATIONS Knudson FEV1 categories % Predicted FEV1 Children (6-17) Adults (18 + ) Total n % n % n % 0 9.9% % % % % % % % % % % subtotal transplants < 6 years missing total * Knudson RJ, Lebowitz MD, Holberg CJ, Burrows B. Changes in the Normal Maximal Expiratory Flow-Volume Curve with Growth and Aging. Am Rev Resp Dis 1983; 127: BMR - RBM Annual Data Report
32 % % % % % % % % % % 100 % of patients % Predicted FEV1 62.4% have mean % predicted values above 70%. Figure 10: % predicted FEV1 using Knudson equations % of patients % % % % % % % % % % 100 % Predicted FEV1 Figure 11: % predicted FEV1 using Knudson equations for children 82.0% of the children (6 17 years) have mean % predicted values above 70% % of patients % % % % % % % % % % 100 % Predicted FEV1 Figure 12: % predicted FEV1 using Knudson equations for adults 45.5% of the adults (18 years and above) have mean % predicted values above 70%. BMR - RBM Annual Data Report
33 MEAN % PREDICTED FEV1 USING KNUDSON EQUATIONS BY AGE age (years) n FEV1% predicted mean (std.dev) age (years) n FEV1% predicted mean (std.dev) (19.5) (18.4) (14.9) (13.0) (18.8) (15.1) (16.2) (24.7) (26.1) (18.9) (25.3) (24.3) (23.4) (23.5) (22.3) (25.4) (17.7) (25.5) (18.3) (22.2) (28.2) (22.5) (20.4) (28.3) (25.5) (28.7) (20.1) (21.6) (18.4) (16.5) (35.4) (22.2) (30.3) (26.8) (25.6) (18.0) (18.0) (41.9) (29.0) (4.5) (4.8) (.) (.) (28.1) (.) (31.0) (11.5) subtotal 727 transplants 82 < 6 years 161 missing 56 total (25.0) The overall mean % predicted FEV1 using the Knudson reference equations is 75.9% (SD = 25.6). BMR - RBM Annual Data Report
34 MEAN % PREDICTED FEV1 USING KNUDSON EQUATIONS BY AGE AND SEX age (years) FEV1% males FEV1% females n mean (std.dev) n mean (std.dev) Total (17.3) (24.3) (18.0) (14.7) (15.9) (12.8) (13.4) (13.1) (17.4) (20.2) (11.7) (16.8) (12.2) (21.3) (23.6) (26.4) (17.4) (31.3) (16.4) (22.1) (27.3) (23.7) (26.1) (23.7) (23.1) (23.7) (22.9) (24.4) (19.7) (23.7) (18.7) (34.9) (15.0) (21.2) (22.0) (28.4) (19.1) (17.4) (25.7) (20.2) (34.5) (22.1) (31.2) (15.2) (21.4) (20.7) (29.1) (27.0) (27.2) (19.2) (35.4) (12.1) (30.3) (18.1) (20.9) (14.9) (25.0) (17.5) 12 BMR - RBM Annual Data Report
35 .. MEAN % PREDICTED FEV1 USING KNUDSON EQUATIONS BY AGE AND SEX age (years) FEV1% males FEV1% females n mean (std.dev) n mean (std.dev) Total (4.5) (15.3) (.) (23.3) (3.4) (50.0) (29.9) (16.0) (30.3) (24.3) (33.6) (.) (18.4) (10.5) (35.0) (7.4) (.) (.) (.) (.) (27.1) (.) (.) (4.8) (.) (.) (28.1) (.) (31.0) (11.5) 2 subtotal transplants < 6 years missing total There are 727 patients used for this analysis. The overall mean % predicted FEV1 for males is 77.3% (SD=25.1) and for females 74.3% (SD=26.0). BMR - RBM Annual Data Report
36 Mean % Predicted FVE Age (years) Figure 13: % predicted FEV1 using Knudson equations by age Mean % Predicted FVE Males Females Age (years) Figure 14: % predicted FEV1 using Knudson equations by age and gender BMR - RBM Annual Data Report
37 MEAN % PREDICTED FEV1 USING KNUDSON EQUATIONS BY AGE GROUPS Children (6-17) years Adults ( 18 ) years Total Severity Assessment n % n % n % Normal : 90% predicted Mild : 70% - 89% predicted Moderate : 40% - 69% predicted Severe : 40% predicted subtotal transplants < 6 years missing total % of patients Normal (90% +) Mild (70% - 89%) Moderate (40% - 69%) Children Adults Age group Total Severe (< 40%) Figure 15: % predicted FEV1 severity groups using Knudson equations Based on the Knudson equations, 11.7% of the patients have severe lung problems, 33.0% have normal lung function. BMR - RBM Annual Data Report
38 MEAN % PREDICTED FEV1 USING COMBINED WANG - HANKINSON EQUATIONS For the purposes of this analysis, equations from Wang et. all; based on a study on the pulmonary function of children between 6 and 18 years and Hankinson et. all, with participants between 8 and 80 years, were combined to provide a gap free prediction of the relevant FVC and FEV1 values. Wang s equations were used for children, between 6 and 17 years, while Hankinson s were used for predictions for the adults, 18 years and above. Wang-Hankinson FEV1 categories % Predicted FEV1 Children (6-17) Adults (18 + ) Total n % n % n % 0 9.9% % % % % % % % % % % subtotal transplants < 6 years missing total *Hankinson, J. L., Odencrantz J. R., and Fedan. K. B Spirometry reference values from a sample of the general U.S. population. Am. J. Respir. Crit. Care. Med. 159: **Wang, X., Dockery W. J., Wypil. D., Fay. M. E., and Ferris. B. G Pulmonary function between 6 and 18 years of age. Paediatr. Pulmono. 15: BMR - RBM Annual Data Report
39 % % % % % % % % % % 100 % of patients % Predicted FEV1 Figure 16: % predicted FEV1 using Wang Hankinson equations 61.0% of the patients have mean % predicted FEV1 above 70% % of patients % % % % % % % % % % 100 % Predicted FEV1 Figure 17: % predicted FEV1 using Wang Hankinson equations for children 82.2% of the children (6 17 years) have mean % predicted FEV1 above 70% % of patients % % % % % % % % % % 100 % Predicted FEV1 Figure 18: % predicted FEV1 using Wang Hankinson equations for adults 42.2% of the adults (18 years and above) have mean % predicted FEV1 above 70%. BMR - RBM Annual Data Report
40 MEAN % PREDICTED FEV1 USING WANG - HANKINSON EQUATIONS BY AGE age (years) n FEV1% predicted mean (std.dev) age (years) n FEV1% predicted mean (std.dev) (16.8) (17.5) (15.1) (13.1) (19.4) (15.5) (17.3) (25.0) (26.8) (19.0) (24.6) (23.7) (22.4) (23.1) (20.9) (24.2) (17.3) (24.6) (17.8) (21.8) (26.9) (22.5) (20.1) (27.5) (24.7) (28.2) (18.3) (19.8) (15.4) (15.1) (33.4) (22.4) (28.7) (25.9) (26.3) (16.5) (17.2) (38.2) (26.5) (1.3) (0.7) (.) (.) (26.3) (.) (28.6) (10.1) subtotal 727 transplants 82 < 6 years 161 missing 56 total (24.1) The overall mean % predicted FEV1 using the Wang - Hankinson reference equations is 74.8% (SD= 25.8). BMR - RBM Annual Data Report
41 MEAN % PREDICTED FEV1 USING WANG - HANKINSON EQUATIONS BY AGE AND SEX age (years) FEV1% males FEV1% females n mean (std.dev) n mean (std.dev) Total (15.1) (19.5) (18.3) (13.4) (16.2) (12.5) (14.3) (11.6) (18.1) (20.7) (12.1) (17.2) (13.9) (22.0) (25.3) (25.4) (18.0) (30.7) (16.6) (21.8) (26.6) (23.1) (25.1) (23.5) (22.3) (22.7) (21.9) (24.1) (18.1) (23.0) (17.6) (33.6) (14.5) (20.7) (22.0) (26.8) (19.1) (16.7) (26.1) (19.2) (32.6) (21.4) (31.8) (14.3) (21.4) (19.2) (30.0) (24.9) (27.1) (17.9) (34.8) (11.2) (29.6) (17.1) (18.7) (13.6) (23.0) (16.6) 12 BMR - RBM Annual Data Report
42 ..MEAN % PREDICTED FEV1 USING WANG - HANKINSON EQUATIONS BY AGE AND SEX age (years) FEV1% males FEV1% females n mean (std.dev) n mean (std.dev) Total (5.3) (14.1) (.) (21.2) (3.9) (47.2) (30.6) (14.9) (28.7) (25.4) (30.9) (.) (17.3) (9.5) (32.8) (4.5) (.) (.) (.) (.) (24.8) (.) (.) (0.7) (.) (.) (26.3) (.) (28.6) (10.1) 2 subtotal transplants < 6 years missing total There are 727 patients used. The overall mean % predicted FEV1 using the Wang - Hankinson reference equation is 76.9% (SD=25.7) and 72.4% (SD=25.7) respectively for males and females. BMR - RBM Annual Data Report
43 Mean % Predicted FVE Age (years) Figure 19: % predicted FEV1 using Wang Hankinson equations by age Mean % Predicted FVE Males Females Age (years) Figure 20: % predicted FEV1 using Wang Hankinson equations by age and gender BMR - RBM Annual Data Report
44 MEAN % PREDICTED FEV1 USING WANG - HANKINSON EQUATIONS BY AGE GROUPS Children (6-17) years Adults ( 18 ) years Total Severity Assessment n % n % n % Normal : 90% predicted Mild : 70% - 89% predicted Moderate : 40% - 69% predicted Severe : 40% predicted subtotal transplants < 6 years missing total % of patients Normal (90% +) Mild (70% - 89%) Moderate (40% - 69%) Children Adults Age group Total Severe (< 40%) Figure 21: % predicted FEV1 severity groups using Wang - Hankinson equations Using the Wang - Hankinson equations, 12.2% of the patients have severe lung problems, 32.5% have normal lung function. BMR - RBM Annual Data Report
45 MEAN % PREDICTED FVC USING KNUDSON EQUATIONS Knudson FVC categories % Predicted FVC Children (6-17) Adults (18 + ) Total n % n % n % 0 9.9% % % % % % % % % % % subtotal transplants < 6 years missing total * Knudson RJ, Lebowitz MD, Holberg CJ, Burrows B. Changes in the Normal Maximal Expiratory Flow-Volume Curve with Growth and Aging. Am Rev Resp Dis 1983; 127: BMR - RBM Annual Data Report
46 45 40 % of patients % % % % % % % % % % 100 % Predicted FVC Figure 22: % predicted FVC using Knudson equations 81.9% of the patients have mean % predicted FVC above 70%. % of patients % % % % % % % % % % 100 % Predicted FVC Figure 23: % predicted FVC using Knudson equations for children 90.9% of the children (6 17 years) have mean % predicted values above 70%. % of patients % % % % % % % % % % 100 % Predicted FVC Figure 24: % predicted FVC using Knudson equations for adults 74.2% of the adults (18 years and above) have mean % predicted values above 70%. BMR - RBM Annual Data Report
47 MEAN % PREDICTED FVC USING KNUDSON EQUATIONS BY AGE age (years) n FVC% predicted mean (std.dev) age (years) n FVC% predicted mean (std.dev) (19.9) (15.1) (11.4) (19.3) (18.7) (14.1) (11.7) (22.4) (22.5) (16.8) (19.9) (20.6) (19.9) (23.1) (19.1) (20.1) (17.3) (18.5) (16.1) (19.5) (22.0) (24.8) (17.5) (23.4) (23.2) (21.7) (18.1) (15.1) (32.6) (23.2) (28.0) (24.6) (25.1) (19.5) (11.1) (14.9) (19.8) (1.1) (3.4) (.) (.) (34.3) (.) (28.2) (42.3) subtotal 730 transplants 77 < 6 years 161 missing 53 total (21.0) (17.7) (15.1) The overall mean % FVC predicted using the Knudson equations is 87.8% (SD=21.1). BMR - RBM Annual Data Report
48 MEAN % PREDICTED FVC USING KNUDSON EQUATIONS BY AGE AND SEX age (years) FVC % males FVC % females n mean (std.dev) n mean (std.dev) Total (15.8) (27.2) (13.2) (15.6) (12.2) (9.4) (14.0) (23.3) (17.1) (17.7) (10.7) (15.6) (10.0) (14.0) (19.7) (25.3) (16.0) (27.3) (17.1) (17.1) (21.9) (18.4) (21.9) (19.7) (19.4) (21.0) (21.4) (24.1) (11.6) (21.2) (15.1) (27.1) (12.0) (22.5) (15.1) (20.9) (16.8) (15.9) (17.9) (20.5) (24.2) (20.8) (27.5) (23.9) (15.9) (22.4) (22.0) (22.5) (23.9) (23.1) (25.9) (8.7) (20.4) (10.5) (13.9) (17.0) (23.8) (20.9) 12 BMR - RBM Annual Data Report
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