CYSTIC FIBROSIS IN AUSTRALIA 2008

Transcription

1 CYSTIC FIBROSIS IN AUSTRALIA th Annual Report from the Australian Cystic Fibrosis Data Registry

2 Cystic Fibrosis Australia 2010 This work is copyright. Apart from any use as permitted under the Copyright Act 1968, no part may be reproduced by any process without prior written permission from Cystic Fibrosis Australia. Requests and inquiries concerning reproduction and rights should be addressed to Chief Executive Officer, Cystic Fibrosis Australia, PO Box 254, North Ryde NSW 1670 Published by Cystic Fibrosis Australia 21 Manning Road North Ryde NSW 2113 Australia Website: ISSN ii

3 Preface I am pleased to present the eleventh annual report from the Australian Cystic Fibrosis Data Registry. Its release within 15 months of the reference year is a welcome sign that improvement in timeliness of reporting has been maintained. CFA believes that its aim of releasing the Registry s annual report within 12 months of a reference year will be achieved later this year for 2009 data. I extend my very sincere thanks to those in CF treatment centres across Australia who continue to support the Registry through their hard work. A number of technical improvements have been made to the Data Registry this year. Following feedback from users at CF specialist centres, individual patient reports available to contributing centres about their own patients have been improved. Several centres are now using this Registry service as an integral part of their preparation for clinics. Also in response to user demand, centres can now authorise transfer of clinical details, including original diagnostic information stored in the Registry, in association with a patient s move between centres. This new arrangement in no way replaces communication of patient information between clinicians, but has been implemented by CFA to save data entry time at the receiving centre and to remove the risk of error when re-entering information that has already been provided to the Registry by another centre. Once again I thank the members of the ACFDR Advisory Committee for their assistance with these initiatives and for their continued input to registry development. With a lot of work on upgrading and refining the database now behind us, it can be expected that the focus of the Advisory Committee and the Registry s data managers will turn more and more towards information outputs, analyses and engagement with research, for the benefit of CF specialist centres and their patients. In this regard I am expecting that opportunities for international collaboration in CF data analysis and research will move forward strongly this year after the launch by the US Cystic Fibrosis Foundation of version 2 of its patient data registry, PortCF. Australian Registry managers have maintained close contact with their counterparts in the US, and elsewhere, to promote the development of compatible registry data, for benefits such as international benchmarking and comparisons that can greatly enhance the value of each national registry s resources. Terry Stewart Chief Executive Officer Cystic Fibrosis Australia March 2010 iii

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5 Contents Acknowledgments ACFDR trend indicators viii x Chapter 1. People with cystic fibrosis Overview Age Distribution Siblings with cystic fibrosis Adults: marital status, education and activity 5 Chapter 2. Diagnosis Overview Age at diagnosis Presentation and diagnosis Genotyping Pancreatic insufficiency 11 Chapter 3. Health and functioning Respiratory infections Other medical complications Lung function Height and weight 22 Chapter 4. Treatment of cystic fibrosis Visits to clinics Therapy for cystic fibrosis patients Hospital treatment Non-transplant surgery 37 Chapter 5. Organ transplants Patients assessed for transplant in Transplants during Chapter 6. Mortality Deaths recorded in the Australasian CF Data Registry Cause of death 39 Appendix 1 Technical notes 40 v

6 List of tables Table A1. Demographic indicators Table A2. Lung function indicators (FEV1 % predicted) Table A3. Nutrition indicators x xii xii Table 1.1. Age and sex of registrants 3 Table 1.2. Adult status by State/Territory of residence 4 Table 1.3. Sex distribution by State/Territory of residence 4 Table 1.4. Patients who have siblings with cystic fibrosis 5 Table 1.5. Marital status of adults 5 Table 1.6. Number of children 6 Table 1.7. Educational attainment of adults 6 Table 1.8. Activity status of adults 6 Table 2.1. Age at diagnosis 8 Table 2.2. Mode of presentation, by year of diagnosis 9 Table 2.3. Whether patient has been genotyped, by year of diagnosis 9 Table 2.4. Sweat electrolyte levels, by year of diagnosis 10 Table 2.5. Genotype 10 Table 2.6. Pancreatic insufficiency 11 Table 3.1. Number of sputum and BAL/bronchoscopy cultures 12 Table 3.2 Pseudomonas infection by age group 13 Table 3.3. Other respiratory culture by age group 14 Table 3.4. Medical complications 15 Table 3.5. FEV1 per cent predicted children and adolescents 17 Table 3.6. FEV1 per cent predicted adults 18 Table 3.7. Lung Function impairment children and adolescents 19 Table 3.8. Lung Function impairment adults 20 Table 3.9. Child and adolescent height, weight and BMI percentiles 22 Table Child and adolescent height, weight & BMI percentile dist n 24 Table Height and weight z-score dist n, children and adolescents 25 Table Height, weight and BMI z-scores, children and adolescents 26 Table Adult BMI distribution 28 Table 4.1. Outpatient visits to CF clinics 29 Table 4.2. Whether had home IV antibiotic therapy by age group 30 Table 4.3. Antibiotic therapy by age group 30 Table 4.4. Oral antibiotic therapy by age group 31 Table 4.5 Inhaled antibiotics by age group 32 Table 4.6 Other therapy by type 32 Table 4.7. Nutritional supplements by age group 33 Table 4.8. Oxygen therapy 34 vi

7 Table 4.9 Non-invasive ventilation 34 Table 4.10 Hospitalisation, all causes 35 Table 4.11 Hospitalisation related to cystic fibrosis, respiratory causes 36 Table 4.12 Total time in hospital for persons hospitalised 36 Table 4.13 Non-transplant surgery 37 Table 5.1. Patients assessed for transplant 38 Table 5.2. Patients receiving lung transplants 38 Table 6.1. Deaths, by age and sex 39 Table 6.2. Cause of death 39 List of charts Proportion who are adult Median age at diagnosis Median age at death Mean FEV1 per cent predicted Mean child and adolescent BMI percentile Mean adult BMI xi xi xi xii xii xii Figure 1.1. Age distribution by sex 2 Figure 2.1. Infant diagnosis by months of age 8 Figure 3.1. FEV1 per cent predicted children and adolescents 17 Figure 3.2. Lung function (FEV1 per cent predicted) by age 18 Figure 3.3. FEV1 per cent predicted adults 19 Figure 3.4. Lung function impairment children and adolescents 20 Figure 3.5. Lung function impairment adults 21 Figure 3.6. Mean percentile height by age children and adolescents 23 Figure 3.7. Mean percentile weight by age children and adolescents 23 Figure 3.8 Mean percentile BMI by age children and adolescents 24 Figure 3.9 Mean height z-score by age of children and adolescents 27 Figure Mean weight z-score by age of children and adolescents 27 Figure Per cent distribution of adult BMI 28 Figure 4.1. Hospitalisations 35 vii

8 Acknowledgements Many thanks go to the Australasian Cystic Fibrosis Data Registry Advisory Committee, whose members have shown a commitment to making the Data Registry a useful tool for improving health and standards in the CF community. Members are: Dr Scott Bell The Prince Charles Hospital, Brisbane QLD Assoc. Prof. Peter Bye Royal Prince Alfred Hospital, Camperdown NSW Dr Peter Cooper The Children s Hospital, Westmead NSW Dr James Martin Women s and Children s Hospital, Adelaide SA Dr Phil Robinson Royal Children s Hospital. Melbourne VIC Dr Gerard Ryan Sir Charles Gairdner Hospital, Perth WA Ms Zez Stankovic National Data Registry Coordinator, CFA Mr Terry Stewart CEO, Cystic Fibrosis Australia (Chair) Dr Adam Jaffe Sydney Children s Hospital Further acknowledgement must go to the following people and organisations: Ms Zez Stankovic, whose work as part-time National Data Registry Coordinator facilitates the collection of data through the data recorders in CF Centres Ms Ann-Maree Bosch for logistical arrangements and minutes of proceedings of the Advisory Committee Listening Post Pty Ltd, incorporating Prowess Development Pty Ltd for database design and hosting services Mr Geoff Sims and Mr Robert Lipp of Geoff Sims Consulting Pty Ltd for database management and reporting. Thanks also to the following organisations for their generous financial support: Solvay Pharmaceuticals Roche Products AstraZeneca The participants in Cystic Fibrosis The Great Escape car rally LJ Hooker Cystic Fibrosis National Sponsor for Research viii

9 Participating Centres The Data Registry relies on the tireless work of people in the following CF Centres who enter data and handle edit queries for quality control of the annual collection of data: New South Wales Sydney Children s Hospital Royal Prince Alfred Hospital, Sydney The Children s Hospital, Westmead Westmead Hospital Adults Gosford Hospital John Hunter Hospital, Newcastle Victoria Royal Children s Hospital, Melbourne The Alfred Hospital, Melbourne Monash Medical Centre, Clayton Queensland Royal Children s Hospital, Brisbane The Prince Charles Hospital, Brisbane Mater Hospital Children, Brisbane Mater Hospital Adults, Brisbane Southport Hospital Gold Coast South Australia Royal Adelaide Hospital Women s and Children s Hospital, Adelaide Western Australia Princess Margaret Hospital for Children, Perth Sir Charles Gairdner Hospital, Perth Tasmania Royal Hobart Hospital Launceston General Hospital CF Clinic, Burnie ix

10 ACFDR trend indicators Introduction Indicators presented in this section have been re-compiled from a consolidated database that is now held in a consistent format in the Data Registry s online system. Additional records or data entries may have been added to the database since data were first published for earlier. In addition, updated editing and other processes have been applied to all of data. For these reasons indicators for earlier than 2007 may differ from earlier reports. Demographic indicators Table A1: ACFDR Demographic indicators Indicator Registrants at 31 December (number): Males Females Persons Mean age (): Males Females Persons Median age (): Males Females Persons Proportion adult (%) New diagnoses: Number Median age (months) Deaths Number Mean age () Median age () x

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12 Lung function Table A2: ACFDR Lung function indicators (mean FEV1 per cent predicted) 1998 to 2008 Indicator All ages: Males Females Age 6 to 11 Males Females Age 12 to 17 : Males Females Age 18 to 29 : Males Females Age 30 and over: Males Females All adults (18 +): Males Females Nutrition Table A3: ACFDR Nutrition indicators 1998 to 2008 Indicator Mean BMI percentiles: 2 to 5 - males to 5 - females to 11 - males to 11 - females to 17 - males to 17 - females Mean BMI values: 18 to 29 - males to 29 - females males females All adult (30+) - males All adult (30+) - females xii

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15 1 People with cystic fibrosis 1.1 Overview At 31 December 2008 the Australian Cystic Fibrosis Data Registry (ACFDR) held records of 2,843 people with cystic fibrosis. The average age of the Registry population was 18.7 at 31 December This is up from 18.2 at the end of 2007 and 17.8 in The proportion of the registry population that is adult (18 and over) increased to 47.2 percent by the end of The proportion of adults recorded in the registry was 26.4 per cent in 1998, the year that the Registry first reported. Within the Registry population, the median age at 31 December 2008 was 17.0, an increase from 16.4 at the end of The median age for males (17.5 ) remained higher than that for females (16.6 ). The number of new diagnoses of CF notified to the Registry for 2008 was 73, including 62 diagnosed at age less than one year. This is more than the 69 overall and 57 infant diagnoses reported in The number of deaths reported to Registry was 24 in 2008, similar to the numbers reported over the past 5. The median age at death was, 31 in 2008, lower than 31 in 2007, but still indicating an upward trend over the since the Registry began, when it stood at around 20. The coverage of demographic information about persons with CF has improved again in 2008, as indicated by growth of over 200 in the overall number of registrants. This is almost 4 times the increase that can be attributed to an excess of new diagnoses (73) over deaths (19) during the year. Although individuals who do not attend specialist CF treatment centres would are not included in the statistics in this report, the number of such persons is not large. 1

16 1.2 Age distribution Figure 1.1 shows the age distribution of Australian residents recorded in the CF Data Registry at 31 December Details are in Table 1.1. Figure 1.1: ACFDR 2008: Age distribution by sex 2

17 Table 1.1: ACFDR 2008: Age and sex of registrants at 31 December 2008 Age Males Females Persons Per cent male Total 1,492 1,351 2,

18 The proportion of adults in the Registry as a whole was 47.2 per cent at 31 December Two jurisdictions, Victoria and Western Australia, have adult proportions of just over 50 per cent. Lower proportions in the Australian Capital Territory and the Northern Territory are a reflection of coverage issues and smaller numbers. Table 1.2: ACFDR 31 December 2008: Adult status by State/Territory of residence State or Territory of residence Child/adolescent Adult Total Per cent adult New South Wales Victoria Queensland Western Australia South Australia Tasmania Australian Capital Territory Northern Territory Overseas Total 1,501 1,342 2, At 31 December 2008, males made up 53% and females 47% of the Australian Registry population. This has remained a consistent proportion since establishment of the Registry in The proportion who are male is higher amongst the adult population (54%) than the child and adolescent population (51%). Table 1.3: ACFDR 31 December 2008: Sex distribution by State/Territory of residence Place of residence Males Females Persons Per cent male New South Wales Victoria Queensland Western Australia South Australia Tasmania Australian Capital Territory Northern Territory Overseas Total 1,492 1,351 2,

19 1.3 Siblings with cystic fibrosis Nearly one in 6 persons with cystic fibrosis reported having siblings who also have the disease. (Table 1.4) Table 1.4 ACFDR 31 December 2008: Patients who have siblings with cystic fibrosis State or Territory of residence: Yes No Unknown Not reported Total Per cent with CF sibling(a) New South Wales Victoria Queensland Western Australia South Australia Tasmania Australian Capital Territory Northern Territory Overseas Total (a) excludes not reported from total 1.4 Adults: marital status, education and activity Management of cystic fibrosis allows an increasing proportion of persons with the disease to continue with normal activities into their adult life, despite the degenerative effects of the disease. While reporting of current personal information is incomplete, gaps in coverage largely reflect lack of information from a small number of treatment centres. Information compiled below is therefore likely to be reasonably representative of the adult population with cystic fibrosis. Table 1.5 shows that around 35% of adult patients for whom marital status is known were in a formal or informal marriage relationship. Table 1.5: ACFDR 31 December 2008: Marital status of adults Males Females Marital status Number Per cent Number Per cent Married (includes de facto) Not married Unknown (includes not recorded) Total

20 Table 1.6 reports the number of adult CF patients who have children. Over one in 6 adult CF patients are recorded as having children. Table 1.6: ACFDR 31 December 2008: Number of children Sex of CF patient CF patients with: Males Females No children child children or more children 2 7 Unknown/not reported Total Many people with cystic fibrosis continue with education beyond school level, with 10% of adult CF patients for whom educational attainment was reported having university qualifications and a further 14% having completed other study beyond school. Table 1.7: ACFDR 31 December 2008: Educational attainment of adults Number Per cent Junior Secondary (Year 10) Senior Secondary (Year 12) Tertiary Certificate or Diploma University Degree Left school prior to Year Not applicable (a) Total reported Unknown/not reported (incl. as % of total below) Total 1, (a) currently studying Over two thirds of the Registry s adult population were in either full-time or part-time paid employment during Table 1.8: ACFDR 31 December 2008: Activity status of adults Number Per cent Employed, full time paid Employed, part time paid Voluntary work only Unemployed Pensioner Others not in labour force (a) Total reported Unknown/not reported (incl. as % of total below) Total (a) includes homemakers, students 6

21 2 Diagnosis 2.1 Overview In the reference year 2008, 73 newly diagnosed cases of cystic fibrosis were included in the Australasian Cystic Fibrosis Data Registry. This is 20 less than the (revised) number of new cases in Most diagnoses were made during infancy, that is before the first birthday (see Table 2.1). Presentation and diagnostic information was recorded for 93% of the CF patient population in Information was available for at least two thirds of patients in all centres with missing diagnostic information concentrated in a small number of treatment centres. How is cystic fibrosis diagnosed? The majority of Australian cystic fibrosis diagnoses occur within or soon after the neonatal period. Neonatal screening programs test a blood sample taken from all newborn babies at around 3 days. Where immuno-reactive trypsin (IRT), a product of the pancreas, is found in the blood at a level above the 99 th percentile, genetic testing is carried out to screen for the most common gene mutation or mutations associated with cystic fibrosis. Where two copies of a CF gene mutation are found, cystic fibrosis is diagnosed. Where one copy is found, a sweat test for elevated electrolyte levels is undertaken to establish diagnosis. If no copies of a CF gene mutation are found, it is considered unlikely that the child has inherited cystic fibrosis. However, only the most common CF gene mutations from a very large range of mostly rare possibilities are screened. Thus a child may present with other symptoms later. These could be respiratory distress, pancreatic insufficiency, rectal prolapse or failure to thrive. Some may present much later with symptoms indicating infertility. Some other early signs may aid diagnosis. The most common of these is meconium ileus, or failure to pass meconium from the intestines within 48 hours of birth. Where there is already a CF sibling, diagnosis may be established by antenatal testing of fetal DNA obtained at amniocentesis. 2.2 Age at diagnosis Figure 2.1 shows the large proportion of infant diagnoses that are completed in the first three months, the result of neonatal screening programs that operate in all States and Territories of Australia. Figure 2.1 shows how the current year infant diagnosis pattern differs from that for infant diagnoses in all diagnosis represented by persons in the registry. 7

22 Figure 2.1: ACFDR 31 December 2008: Infant diagnosis by months of age (per cent distribution) Australian CF centres reported 6 new cases diagnosed in early childhood (1 to 4 ), four aged 5 to 9 and one new diagnosis for an adult aged over 35 (Table 2.1). Table 2.1: ACFDR 31 December 2008: Age at diagnosis All Number All Per cent 0 months month months months months months months Not known (a) Total ((a) Not known have been excluded when calculating percentages. 8

23 2.3 Presentation and diagnosis Nearly three quarters of new cases in 2008 included neonatal screening as a mode of presentation. Next most common was meconium ileus (18%), with respiratory symptoms and gastrointestinal symptoms each appearing in 7 per cent of cases (Table 2.2). Table 2.2 ACFDR 31 December 2008: Mode of presentation (a) by year of diagnosis All All Number Per cent Neonatal screening 1, Respiratory symptoms Gastrointestinal symptoms Meconium ileus CF sibling Minor manifestations Pre-natal diagnosis Infertility Other Not known(b) Total 2, (a) More than one mode of presentation can be recorded for a patient so numbers in this section add to more than the total number of registrants and percentage columns add to more than (b) Not known have been excluded when calculating percentages. One new case of cystic fibrosis identified in 2008 was not genotyped. Table 2.3: ACFDR 31 December 2008: Whether patient genotyped, by year of diagnosis All All Number Per cent Genotyped 2, Not genotyped Unknown/Not recorded Total 2, Five patients diagnosed in 2008 recorded both sodium and chloride levels below diagnostic thresholds of 60 mmol/l. All of these were genotyped. 9

24 Table 2.4: ACFDR 31 December 2008: Sweat electrolyte levels, by year of diagnosis All All Number Per cent Sodium > 60mmol/l Sodium <= 60mmol/l Not tested / not recorded 1, Total 2, Chloride > 60 mmol/l Chloride <= 60 mmol/l Not tested / not recorded 1, Total 2, Genotyping The genetic mutation F508del has been identified as at least one of the paired genes responsible for the inheritance of cystic fibrosis in 94% of patients who have been genotyped. Just over half (51.4%) are reported as homozygous for F508del. Table 2.5: ACFDR 31 December 2008: Genotype Mutation 1 F508del G551D R117H G542X R553X Other Total Number Mutation 2: F508del 1102 G551D R117H G542X R553X Other Unknown / missing Total Per cent Mutation 2: F508del 51.4 G551D R117H G542X R553X Other Unknown / missing Total

25 2.5 Pancreatic insufficiency Table 2.6: ACFDR 2008: Pancreatic insufficiency Males Females Persons Males Females Persons Number Per cent Total 1,101 1,017 2, The overall proportion of patients who are pancreatic insufficient is nearly 82 percent, with no difference evident between the sexes. 11

26 3 Health and functioning Information in this chapter covers respiratory infections, medical complications, lung function and nutritional measures. For a number of centres microbiology results from respiratory samples, lung function and height and weight measures were taken from data that are now being supplied to the registry for each test or measurement. 3.1 Respiratory infections Table 3.1 ACFDR 2008: Number of sputum and BAL/bronchoscopy cultures Sputum cultures: Per cent of patients tested (a) 35 + None or more Total All ages BAL/bronchoscopy: None or more Total Number of patients Patients tested (a) ,784 Culture not done ,021 Total reported ,805 Not reported Total patients ,843 (a) By any method of obtaining culture. Table 3.1 shows the distribution of CF patients according to the number of both sputum and BAL/bronchoscopy samples examined during The latter method is used mainly on smaller children. Taking sputum samples 12

27 alone, 69 per cent of patients tested had at least two sputum samples in It can also be seen that cultures were not reported for around 36% of patients. The most commonly identified organisms in respiratory specimens are various species and forms of Pseudomonas (see Table 3.2). It can be seen that 53 per cent of patients tested produced positive Pseudomonas cultures, with the mucoid form of Pseudomonas aeruginosa showing in 38 per cent. Its presence is greater in adult patients. The table shows proportions of between 70 and 85 per cent of adult CF patients produced samples indicating the mucoid form of Pseudomonas aeruginosa in Table 3.2: ACFDR 2008: Pseudomonas infection by age group Pseudomonas aeruginosa: Number All ages Mucoid Rough/non-mucoid Not differentiated Any Ps aeruginosa Pseudomonas other species Patients tested ,784 Culture not done ,021 Total reported ,805 Not reported Total patients ,843 Pseudomonas aeruginosa: Per cent Mucoid Rough/non-mucoid Not differentiated Any Ps aeruginosa Pseudomonas other species Patients tested Note: Patient may have had more than one Pseudomonas infection. Totals and percentages add to more than While prevalence of Pseudomonas organisms is lower in children than in adults, though increasing with rising age, young children are more likely than adult patients to produce cultures showing presence of Staphylococcus aureus. About half of all child patients had this bacterial infection. Haemophilus influenzae is also evident in relatively high proportions of child patients, even the youngest. This organism was present in a quarter of all children in the 0 to 4 age group and 23 per cent in the 5 to 9 age group, but it is less prevalent at older ages. The 0 to 4 age group also had the highest proportions (13 per cent) with positive cultures of the bacteria Escherichia coli. 13

28 Table 3.3: ACFDR 2008: Other respiratory culture by age group Bacteria: Number Total Staphylococcus aureus Haemophilus influenzae Burkholderia cepacia (Ps cepacia) Stenotrophomonas maltophilia Escherichia coli MRSA (a) Alcaligenes xylosoxidans Serratia marcescens Klebsiella (any species) Non-tuberculous mycobacterium Fungi: Candida Aspergillus (any species) Scediosporium (any species) Other organisms not listed above Normal flora only No growth/sterile culture Patients tested ,784 Culture not done ,021 Total reported ,805 Not reported Total patients ,843 Per cent of patients tested Bacteria: Staphylococcus aureus Haemophilus influenzae Burkholderia cepacia (Ps cepacia) Stenotrophomonas maltophilia Escherichia coli MRSA (a) Alcaligenes xylosoxidans Serratia marcescens Klebsiella (any species) Non-tuberculous mycobacterium Fungi: Candida Aspergillus (any species) Scediosporium (any species) Other organisms not listed above Normal flora only No growth/sterile culture (a) Methicillin-resistant Staphylococcus aureus (b) Note: Patients may have multiple infections during the year. Totals and percentages add to more than

29 3.2 Other medical complications Complications data were subject to about 30 per cent under-reporting in 2008, with several centres reporting little information. The numbers in the following table show how the prevalence of medical complications increases with age in CF patients. For instance, over one third of adult patients aged 25 or over suffer gastro-oesophageal reflux, one in four have chronic insulin-dependent diabetes and over one third have osteoporosis or osteopenia. The proportion for whom none of the selected complications shown in Table 3.4 has been reported is high for very young children, nearly 85 per cent, but declines to under 30% per cent in older CF patients. Table 3.4 ACFDR 2008: Medical complications Pulmonary: Number Total Major haemoptysis Massive haemoptysis Therapeutic bronchial artery embolisation Pneumothorax Any pulmonary above Gastro-intestinal: Gastro-oesophageal reflux proven at endoscopy Abnormal liver function test Cirrhosis or portal hypertension Pancreatitis Any Gastro-intestinal above Endocrine: Chronic insulin-dependent diabetes Intermittent insulin-dependent diabetes Other glucose abnormality Any Endocrine above Osteo: Osteoporosis Osteopenia Fracture this year Any Osteo above Other: Cancer None of the above Total reported Unknown or not stated Total patients

30 Table 3.4 (cont.)acfdr 2008: Medical complications Pulmonary: Per cent Major haemoptysis Massive haemoptysis Therapeutic bronchial artery embolisation Pneumothorax Any pulmonary above Gastro-intestinal: Gastro-oesophageal reflux proven at endoscopy Abnormal liver function test Cirrhosis or portal hypertension Pancreatitis Any Gastro-intestinal above Endocrine: Chronic insulin-dependent diabetes Intermittent insulin-dependent diabetes Other glucose abnormality Any Endocrine above Osteo: Osteoporosis Osteopenia Fracture this year Any Osteo above Other: Cancer None of the above Total reported Note: Patient may have had more than one complication. Percentages add to more than Total 16

31 3.3 Lung function Table 3.5: ACFDR 2008: FEV 1 per cent predicted - children and adolescents FEV 1 % predicted: Number Per cent Cumulative % Number Per cent Cumulative % Males Females <10% % % % % % % % % % >=100% Total measured Note: Per cent predicted calculations included only patients aged 6 and over for whom lung function test results were available. The usual measure of lung function is forced expiratory volume in one second (FEV1). Per cent predicted FEV1 values, based on normal values observed in studies of healthy subjects, decline along with disease development. The FEV1 values reported in these tables are as at the date of best FEV1 per cent predicted for the year. Two major adult centres reported data for only one measurement date. Lung function was reported for 77 per cent of adult patients and 88 per cent of children/adolescents aged 6 and over. Figure 3.1: ACFDR 2008: Distribution of FEV 1 per cent predicted - children and adolescents aged 6 to 17 17

32 Predicted values for lung function are based on research by Wang et al (1993) and Hankinson et al (1999) see Technical Notes. Figure 3.2 demonstrates that child CF lung function measured as FEV1 falls below predicted normal levels with increasing age. From Table 3.5, 11 per cent of males and 13 per cent of females aged from 6 to 17 have FEV1 values that are below 70% of predicted values. Figure 3.2: ACFDR 2008: Lung function (FEV 1 per cent predicted) by age Figure 3.2 shows that lung function is around 80% of predicted values for older adolescents and around 60% for adults aged 30 and over. Around 56 per cent of male adults and 51 per cent of female adults have lung function values below 70% of predicted (Table 3.6). Table 3.6 ACFDR 2008: FEV 1 per cent predicted - adults Number Per cent Cumulative % Number Per cent Cumulative % Males Females FEV 1 % predicted: <10% % % % % % % % % % >=100% Total measured

33 The decline in lung function with age can be seen also in comparison of Figures 3.1 (children and adolescents) and 3.3 (adults). For adult patients the modal point for the distribution against predicted FEV1 values is in a lower range than is seen for children and adolescents, for both male and female patients. Figure 3.3: ACFDR 2008: Distribution of FEV 1 per cent predicted - adults An alternate view of lung function is provided in the following tables and charts, in which the distributions are summarised into categories of normal lung function (90% of predicted FEV1 and above), mild impairment (below 90% but not below 70%), moderate (below 70% but not below 40%) and severe (below 40%). A similar proportion of male children (61%) and females (59%) have lung function within the normal range (Table 3.7). This is also evident in Figure 3.4 on the following page, where age patterns of lung function are very similar for males and females in Table 3.7: ACFDR 2008: Lung function impairment by age and sex - children and adolescents Severe Moderate Mild Normal Total Severe Moderate Mild Normal Total Age group/sex: Number Per cent Total measured Males Females

34 Figure 3.4: ACFDR 2008: Lung function impairment - children and adolescents Table 3.8 shows generally greater proportions of patients with severe lung function impairment in successive older age groups. The proportion of adult male patients with severe lung function impairment (15%) is greater than the proportion of female patients in this severity category (11%) but both are one percentage point below proportions reported in Table 3.8: ACFDR 2008: Lung function impairment by age and sex - adults Severe Moderate Mild Normal Total Severe Moderate Mild Normal Total Number Per cent Age/sex group: Total measured Males Females

35 Figure 3.5: ACFDR 2008: Lung function impairment - adults 21

36 3.4 Height and weight Height and weight measures are selected from the date of best BMI percentile for 2008 (children and adolescents) or the date of best BMI (adults). For two major adult hospitals, data for one measurement date only were supplied to the Registry. Data were reported for 88 percent of children/adolescents aged 2 to 17 and over 80 per cent of adults. Distributions shown in tables and charts exclude patients for whom data were not reported. Persons known to have had a lung transplant before the measurement date were excluded. Table 3.9: ACFDR 2008: Child and adolescent height, weight and BMI - mean percentiles by age and sex Height Weight BMI Males Females

37 Mean height percentiles for boys aged up to 5 and for girls aged 3 and 4 were above the 50th percentile. At older ages both boys and girls height percentiles were generally lower, although girls in some teenage averaged close the 50th percentile in 2008 Figure 3.6: ACFDR 2008: Mean percentile height by age children and adolescents Weights averaging above or around the 50th percentile were evident in both boys and girls under 10. Boys in the teenage generally averaged weights around the 40th percentile, but girls in some teenage recorded 2008 averages at or above the 50th percentile. BMI percentiles (Figure 3.8 over the page) show a more consistent pattern of lower values at higher ages. Figure 3.7: ACFDR 2008: Mean percentile weight by age children and adolescents 23

38 Figure 3.8: ACFDR 2008: Mean percentile BMI by age children and adolescents Table 3.10: ACFDR 2008: Child and adolescent height, weight and BMI - percentile distribution by sex Height Weight BMI Number Per cent Number Per cent Number Per cent Males < 3rd rd th th th th th th th th th th th th th th th >= 97th Males measured Females < 3rd rd th th th th th th th th th th th th th th th >= 97th Females measured

39 Alternative presentations of child and adolescent height and weight distributions, by percentile and z-scores are presented in Tables 3.10 and In terms of percentile distributions, 61 per cent of children and adolescents overall were below the 50th percentile for height. Corresponding figures for weight and BMI were 51 per cent and 43 per cent. None of these figures showed any variation between males and females. Table 3.11: ACFDR 2008: Height, weight and BMI z-score distribution for children and adolescents Height Weight BMI Number Per cent Number Per cent Number Per cent Males Less than From -3 to < From -2 to < From -1 to < From 0 to < From 1 to < From 2 to < Greater or = Males measured Females Less than From -3 to < From -2 to < From -1 to < From 0 to < From 1 to < From 2 to < Greater or = Females measured

40 Table 3.12: ACFDR 2008: Height, weight and BMI z-scores for children and adolescents Age: Height Weight Weight Mean Standard Mean Standard Number z-score error Number z-score error Number Males Mean z-score Standard error Females

41 Figure 3.9: ACFDR 2008: Mean height z-score by age of children and adolescents Figure 3.10: ACFDR 2008: Mean weight z-score by age of children and adolescents Adult Body Mass Index scores are shown in Figure 3.11 and Table similar proportions of males (57%) and females (58%) had a BMI in the range 20 to less than 25. A higher proportion of females than males have BMI scores below 20. One in four adult males had a BMI above

42 Figure 3.11: ACFDR 2008: Per cent distribution of adult BMI Table 3.13: ACFDR 2008 Adult BMI distribution BMI range Less than 18 From 18 to <20 From 20 to <25 25 and over Total Males: number Adults measured Females: number Adults measured Males: per cent Adults measured Females: per cent Adults measured

43 4 Treatment of cystic fibrosis This Chapter describes the treatments and therapies recorded for patients in the Australian Cystic Fibrosis Registry. 4.1 Visits to clinics Table 4.1: ACFDR 2008: Outpatient visits to CF clinics Children and Adolescents Adults CF patients making: Number Per cent Number Per cent No visits visit (a) visits visits visits visits visits visits visits visits visits visits or more visits Total reported 1, Visits not reported Total persons with CF (a) See text below Table 4.1 demonstrates that some patients make a large number of visits to specialist clinics over a year. The average number of visits for children and adolescents was 4.8 and for adults 5.8. The median number of visits to clinics was 4 for children and adolescents and 5 for adults in Only one clinic visit per patient was reported by one adult centre where it is likely that multiple visits occurred. Clinic visits have not been included in Table 4.1 for this centre. 29

44 4.2 Therapy for cystic fibrosis patients Hospital in the home arrangements, under which patients remain under the hospital s care, but are living at home, are providing the opportunity for cystic fibrosis patients to reduce the time they would otherwise be required to spend in hospital for intravenous antibiotic therapy. Centres failed to report any therapy information for around 21% of patients with several centres not providing any information for nearly all of their patients. Table 4.2 shows that the Data Registry recorded 288 cystic fibrosis patients who had at least one course of intravenous antibiotic therapy administered at home during Around two thirds of these had courses administered both in hospital and at home. Table 4.2: ACFDR 2008: Whether had home IV antibiotic therapy by age group 0 to 4 5 to 9 10 to to to to to Courses at home: Number of patients All ages or more Total Courses at both hospital and home or more Total Almost all cystic fibrosis patients use antibiotic therapy in some form. Table 4.3 shows the proportion to be 95 per cent of patients for whom information was reported. Therapy use information was reported for 73 per cent of patients in Table 4.3: ACFDR 2008: Antibiotic therapy by age group 0 to 4 5 to 9 10 to to to 24 Antibiotic use: Number 25 to to Yes ,967 No Total reported ,082 Not reported Total patients ,843 Per cent Yes No All ages Total reported

45 Oral antibiotics were used by 91 per cent of patients who were reported as using antibiotics. Of these, 41 per cent had used oral antibiotics continuously rather than as needed. The age groups with the highest proportions of continuous oral antibiotics users were young children and adolescents. Within both of these groups, around 40 per cent of oral antibiotics users took them on a continuous basis (Table 4.4). Table 4.4: ACFDR 2008: Oral antibiotic therapy by age group 0 to 4 5 to 9 10 to to to 24 Oral antibiotics use: Per cent 25 to to Yes No Unknown (a) Total antibiotics users Number Total antibiotics users ,967 Mode of use: Per cent As needed (PRN) All ages Continuous Total oral antibiotics users(b) Number Total oral antibiotics users ,890 (a) includes not recorded (b) More than one mode of use can be recored so numbers add to more than As well, mode of use was not recorded for all patients where oral antibiotics were reported. Inhaled antibiotics were used by 53% of CF patients during 2008, with those in the 0 to 4 age group being lowest at 26% (Table 4.5). Amongst inhaled antibiotics users, 25% used on a continuous basis. 31

46 Table 4.5: ACFDR 2008: Inhaled antibiotics by age group 0 to 4 5 to 9 10 to to to 24 Inhaled antibiotics Per cent 25 to to All ages Yes No Unknown (a) Total antibiotics users Number Total antibiotics users ,967 Mode of use As needed (PRN) Continuous Total inhaled antibiotics users(b) Number Total inhaled antibiotics users ,100 (a) includes not recorded (b) More than one mode of use can be recorded so numbers add to more than As well, mode of use was not recorded for all patients where oral antibiotics were reported. Almost all CF patients use a range of other therapies to manage conditions other than infections, and many take nutritional supplements. Therapies used by the highest proportions of patients include pancreatic enzymes (87% of children/adolescents and 72% of adults), vitamin supplements (79% and 67% respectively and bronchodilators (35% and 56%). Table 4.6: ACFDR 2008: Other therapy by type Other therapies used: Child/adolescent Adult Number Per cent Number Per cent Yes No Total reported Not reported Total patients Pulmozyme Pancreatic enzymes Vitamin supplements Bronchodilators Corticosteroids inhaled Corticosteroids oral Insulin Macrolides Salt tablets Antihypercalcaemics Gastric acid secretion reducers Other Total reported Note: individuals may use more than one type of therapy; percentages by type of therapy add to more than

47 One quarter of CF patients also use nutritional supplements. The highest proportion, around one third of patients, occurred in the age group 20 to 34. Table 4.7: ACFDR 2008: Nutritional supplements by age group 0 to 4 5 to 9 10 to to to to 30 Number 30 to All ages Oral (prescribed) Nasogastric TPN Gastrostomy tube/button Total using nutritional supplements Not using nutritional supplements Total reported Not reported Total patients Per cent Oral (prescribed) Nasogastgric TPN Gastrostomy tube/button Total using nutritional supplements Not using nutritional supplements Total reported Note: Individuals may use more than one type Tables 4.8 and 4.9 show numbers of patients reported to have commenced or continued long term oxygen therapy or non-invasive ventilation during Numbers commencing are similar to 2007, but the large number of persons for which no information on this topic was reported (29%) could be expected to contain others using oxygen or non-invasive ventilation therapy. 33

48 Table 4.8: ACFDR 2008: Oxygen therapy Child/adolescent Adult All Ages Number Per cent Number Per cent Number Per cent Long-term oxygen therapy: Commenced during Commenced before No 1, , Unknown Total reported 1, , Not reported Total patients 1,501 1,342 2,843 Table 4.9: ACFDR 2008: Non-invasive ventilation Child/adolescent Adult All Ages Number Per cent Number Per cent Number Per cent Commenced during Commenced before No 1, , Unknown Total reported 1, , Not reported Total patients 1,501 1,342 2,843 34

49 4.3 Hospital treatment Not all CF patients experience hospitalisation during a year. The manner of collection of the data does not allow a distinction to be drawn between no hospitalisation and non-response. A total of 1154 patients, or 44 per cent of patients whose centres provided hospitalisation data to the Data Registry, recorded at least one hospitalisation. Table 4.10: ACFDR 2008: Hospitalisation, all causes Number of hospitalisations: Persons aged Per cent of persons in age group More than Total Number of persons in age group Total All ages Figure 4.1: ACFDR 2008: Hospitalisations 35