1. Dr Jim Littlewood CFWW/CFE Conference CRETE 2005 Standards of care for patients with cystic fibrosis: a European Consensus

2. Concerns of UK families and patients CF Trust questionnaire 2000 Concerns of UK families and patients CF Trust questionnaire 2000 What standard of care should they expect? What standard of care should they expect? Facilities & services that should be available? Person responsible for their care? Person responsible for their care? ‘Shared-care’ arrangements often unclear Local Clinics lacked support of CF Specialist and ‘team’ Local Clinics lacked support of CF Specialist and ‘team’ Poor communication between Specialist Centre & local CF Clinic Specialist adult services inadequate Specialist adult services inadequate Cross-infection issues

3. Artimino, Italy March 2001 ECFS Consensus Conference Artimino March 2004

4. European Cystic Fibrosis Society Initiative by Gerd Döring and the European CF Society Organised by Christine Dubois Organised by Christine Dubois 36 Representatives from many countries To agree a consensus on Standards of Care for people with CF To agree a consensus on Standards of Care for people with CF

5. Format of the meeting In Artimino at the 16 th century La Ferdinanda Medicean Villa In Artimino at the 16 th century La Ferdinanda Medicean Villa 6 round table groups each considered four questions Answers presented to the whole Consensus meeting Answers presented to the whole Consensus meeting Discussion and eventual agreement by Consensus Group

6. We discussed standards of care in the cellars of Artimino – while ‘accompanying persons’ had other interests!

8. A European Consensus To “define standards for the routine evaluation, monitoring and treatment of patients with CF in Europe” To “define standards for the routine evaluation, monitoring and treatment of patients with CF in Europe” Basis for audit of care Provide data for comparison

9. The Consensus Group were unanimous on the need for CF Centre care CF a complex disease requiring an holistic approach CF a complex disease requiring an holistic approach Center care by a team of health care professionals is essential for optimal patient management and outcome Specialist care in dedicated CF Centres is associated with improved survival and QOL Specialist care in dedicated CF Centres is associated with improved survival and QOL Frequent clinical evaluations by physicians and other health workers specifically trained in CF care and early interventions

10. Specialist CF Centres MINIMUM OF 50 PATIENTS Specialist CF staff In a major teaching hospital In a major teaching hospital Treat all CF problems 24 hour access for patients 24 hour access for patients Various specialist facilities - contacts with other experts Liaison with a transplant centre Liaison with a transplant centre Centre treated patients have better prognosis

11. The CF Team

12. The CF Centre Director The leader of a team The leader of a team Main interest CF Expert on CF care Expert on CF care Patients’ advocate Audits performance Audits performance Monitors outcomes Establish network of CF expertise in other hospital departments Establish network of CF expertise in other hospital departments Secure adequate facilities in which to work Communication, education, research Communication, education, research

13. Another CF Specialist Senior, permanent doctor Senior, permanent doctor 30% of time on CF Expert in modern CF care Expert in modern CF care Interest in Pulmonology or Gastroenterology Expert cover when Director is away Expert cover when Director is away

14. CF Nurse Specialist Almost full-time CF Almost full-time CF Wide knowledge of modern CF care Advocate for every patient Advocate for every patient Support and advice for families and patients Patient and family liaison with Center Patient and family liaison with Center Education and research Major role organising home IV antibiotics Major role organising home IV antibiotics

15. Physiotherapist Regular supervision and treatment in the clinic and ward Individualised, reasonable, optimal effective regimen Individualised, reasonable, optimal effective regimen Advice on airway clearance Inhalation therapy – technique and tolerance Inhalation therapy – technique and tolerance Physical education Routine spirometry Routine spirometry hide

16. Dietitian/Nutritionist Ensure provision of appropriate nutrition from infants to adults Ensure provision of appropriate nutrition from infants to adults Dietary assessments Use of pancreatic enzymes Use of pancreatic enzymes Adequate vitamin supplements Advice in both the clinic and ward Advice in both the clinic and ward Intestinal absorption Enteral feeding Enteral feeding CF-related diabetes

17. Psychologist & Social Worker Comprehensive assessment of emotional state Comprehensive assessment of emotional state Annual Review (?) Inclusion in context of CF team Inclusion in context of CF team Transition Transplantation Transplantation Helping team with routine care and at crisis times Families emotional and practical needs Families emotional and practical needs Extra support at various times Access to local services and benefits Complements and works with psychologist

18. CF Pharmacist Monitor and advise on taking complex drug regimens in most effective and acceptable way Monitor and advise on taking complex drug regimens in most effective and acceptable way Inhaled drugs IV antibiotics IV antibiotics Oral drugs Patient education Patient education Drug interactions Flucloxacillin, Ciproxin Flucloxacillin, Ciproxin Nebulised antibiotic Nebulised antibiotic 4 courses I.V. antibiotics/yr 4 courses I.V. antibiotics/yr Bronchodilator Bronchodilator Inhaled steriods Inhaled steriods Pulmozyme Pulmozyme Gastrostomy Gastrostomy Enteral feeds Enteral feeds Vitamins Vitamins Enzymes Enzymes UDCA & taurine UDCA & taurine Insulin Insulin

19. Microbiologist & Laboratory Recognition of typical (usual) CF bacteria Recognition of typical (usual) CF bacteria Confirmation of B. cepacia etc. at CF Laboratory Confirmation of B. cepacia etc. at CF Laboratory Mycobacteria other than M. tuberculosis Mycobacteria other than M. tuberculosis Typing of bacteria Typing of bacteria Different behaviour of antibiotics in CF Different behaviour of antibiotics in CF Antibody levels Antibody levels Aspergillus cultures and antibodies Aspergillus cultures and antibodies Minimum diagnostic service

20. Weekly CF Centre Meeting

21. CF Centre requires 24-HOUR ACCESS FOR PATIENTS 24-HOUR ACCESS FOR PATIENTS Reliable sweat tests Venous access expertise (TIVAD) Venous access expertise (TIVAD) Written guidelines for complications Bronchoscopy service Bronchoscopy service X-rays & Scanners Lung function testing Lung function testing Nasogastric & gastrostomy service Gene mutation testing Gene mutation testing

22. Other Functions of a CF Centre Provision of specialised procedures, diagnostic & laboratory facilities Provision of specialised procedures, diagnostic & laboratory facilities Clinical research, CF meetings & publication Arrangements for transition to adult care Arrangements for transition to adult care Liaison with transplant centre CF education, monitoring, data collection CF education, monitoring, data collection Patient involvement in service Psychological support - CF related Psychological support - CF related

23. Outpatient clinic Every 1-3 months Every 1-3 months Designated CF sessions Designated CF sessions Adequate clinic rooms Adequate clinic rooms CF doctor & CF Nurse CF doctor & CF Nurse Others staff accessible Others staff accessible Weight, Height (& Skull) Weight, Height (& Skull) Throat swab or sputum Throat swab or sputum Respiratory function tests Respiratory function tests History & Examination History & Examination Review treatment Review treatment Discussion Discussion

24. A high standard of hygiene by all staff A high standard of hygiene by all staff Separate clinics for patients with and without chronic P. aeruginosa Separate day for B. cepacia complex and MRSA Separate day for B. cepacia complex and MRSA “Day case” facilities Organise home IV antibiotics Organise home IV antibiotics Separate offices for staff 24 hour access by phone or direct contact 24 hour access by phone or direct contact Outpatient clinic Patients should be segregated according to their microbiological status

25. Inpatient facilities required 3-5 beds per 50 patients – immediate admission when needed 3-5 beds per 50 patients – immediate admission when needed Single rooms, en suite facilities, Parents’ accommodation Parents’ accommodation No patient mixing anywhere Clear infection control policy Clear infection control policy B.cepacia & MRSA in separate cubicles on separate ward Education and recreation facilities Education and recreation facilities Not all patients appreciate segregation!!! segregation!!! Isolate unless recent infection status known

26. Inpatients Physiotherapist - physical treatment, monitoring overnight O 2 saturation Physiotherapist - physical treatment, monitoring overnight O 2 saturation Weekly sputum cultures & Spirometry Skill with venous access & TIVADs Skill with venous access & TIVADs Exercise facilities Clear treatment PROTOCOLS for IV antibiotics and common complications Clear treatment PROTOCOLS for IV antibiotics and common complications

27. Inpatients Review by Pharmacist, Dietitian and Social worker Review by Pharmacist, Dietitian and Social worker CF related diabetes Treatment of DIOS Treatment of DIOS Expertise to start home IV, enteral feeding, & oxygen Review of all aspects care Review of all aspects care Clear treatment plan Summary within 10 days Summary within 10 days

28. Home care – Nurse, Dr & Family Home IV – CF Nurse Home IV – CF Nurse Flushing TIVADs Close supervision of home treatment is essential Close supervision of home treatment is essential Physiotherapy Communication with GP Communication with GP Instruction and support in the home Enteral and gastrostomy feeding, Enteral and gastrostomy feeding, Diabetes management All CF Centres should offer home IV service

29. Shared care for CF at a local “CF Clinic” 20+ patients at local Clinic 20+ patients at local Clinic For children - not adults Special CF clinic sessions Special CF clinic sessions Physiotherapist, dietitian & nurse with a CF interest Shared care arranged as- Shared care arranged as- - Alternate visits to CF Clinic and Specialist CF Centre - Periodic visits to Local CF Clinic by CF Specialist +/- team from centre - Annual Review by staff at the Specialist CF Centre Day-to-day responsibility of Day-to-day responsibility of local consultant local consultant c CF Centre “should not be allowed to result in suboptimal care”.

30. Transition to adult care “Seamless process” “Seamless process” Flexible 16-19 years Close paediatric & adult liaison Close paediatric & adult liaison Ideally common treatment protocols and infection control policies Various patterns of transfer. Various patterns of transfer. Joint adolescent clinic sessions with staff from both units works well

31. The ‘Annual Review’ “Success of treatment will depend on a complete assessment of the patient and then continuing attempts to obtain normal bodily function” “Success of treatment will depend on a complete assessment of the patient and then continuing attempts to obtain normal bodily function” Crozier 1974 Crozier 1974 Detailed history of events over the past year Detailed history of events over the past year Full physical examination Wt & Ht plotted on growth chart Wt & Ht plotted on growth chart Assessment of knowledge of Cystic Fibrosis Check vaccination status Check vaccination status Annual Review should be performed at a CF centre

32. Annual Review Physiotherapist Dietitian Annual Review Physiotherapist Dietitian Assess methods, competence and frequency Assess methods, competence and frequency Spirometry Use of bronchodilator, rhDNase & antibiotics Use of bronchodilator, rhDNase & antibiotics Bronchodilator reversibility testing Review / arrange service of nebuliser Review / arrange service of nebuliser Exercise testing (?) Nutritional review Nutritional review Current diet Wt profile and changes over time Wt profile and changes over time Adequacy and knowledge of pancreatic enzyme therapy Energy supplements and enteral feeds Energy supplements and enteral feeds Vitamins supplements CF related diabetes CF related diabetes

33. Annual Review Laboratory & X-rays Sputum for Non-TB mycobacteria Aspergillus RAST precipitins, IgE, IgA, IgM, IgG, other inflammatory markers Pseudomonas antibodies Chest X-ray Full blood count, urea and electrolytes, liver function tests, vitamins ADE (K), clotting, Full blood count, urea and electrolytes, liver function tests, vitamins ADE (K), clotting, Faecal elastase 1 Faecal elastase 1 Fat absorption – micro’ Fat absorption – micro’ Glucose tolerance test (>10 yrs) Glucose tolerance test (>10 yrs) Liver ultrasound considered (?) Liver ultrasound considered (?) DEXA scan for bone density from 10 yrs (?) DEXA scan for bone density from 10 yrs (?)

34. Annual Review – Results, Decisions & Discussions Director personally reviews clinical & chest X-ray scores Director personally reviews clinical & chest X-ray scores Usually discusses with appropriate team members and plans therapy Discusses findings and suggestions with patient or parents Discusses findings and suggestions with patient or parents Director writes full letter and report to referring consultant, patient’s doctor & copy to patient or parents Data entered into CF Centre and National Databases Data entered into CF Centre and National Databases Fix next year’s appointment!

35. For new patients at their first Annual Review Repeat sweat test Repeat sweat test Genotype if not already done Confirm pancreatic insufficiency with EL1 (even if on enzymes) Confirm pancreatic insufficiency with EL1 (even if on enzymes) Provide CF Centre’s literature Meet members of the CF Team Meet members of the CF Team Mistakes in diagnosis and unnecessary enzyme treatment must be identified

36. Neonatal screening Good methods now well tested Good methods now well tested Major proven nutritional and respiratory advantages Major proven nutritional and respiratory advantages Early CF centre referral and treatment essential Early CF centre referral and treatment essential Genetic advice Genetic advice All newborn infants should be screened for CF

37. Neonatal CF screening

38. Full explanation with a parent/relative present Full explanation with a parent/relative present Written information Meet nurse specialist and others of the CF team Meet nurse specialist and others of the CF team Admit ill infants Start regular treatment – antibiotics, enzymes, vitamins, physiotherapy Start regular treatment – antibiotics, enzymes, vitamins, physiotherapy Regular follow up with staff contact numbers All adults treated at specialist CF centre All adults treated at specialist CF centre “Shared care” for some children Family doctor & health visitor informed Family doctor & health visitor informed Patient informed of the national CF organisation Genetic advice for family Genetic advice for family Sweat test all siblings After neonatal screening/diagnosis

39. Hospital care for adults with CF Complex, changing, developing treatment Complex, changing, developing treatment CF age-related new problems – CFRD, bones, fertility Need staff with wide experience of CF Need staff with wide experience of CF Advice for dealing with diagnosis in adult life Transplant issues Transplant issues Advice on atypical CF – individualised but carefully regular monitoring All adults with CF should attend a Specialist CF Centre attend a Specialist CF Centre

40. Specific problems for discussion or referral Prolonged exacerbations Deterioration RFTS not responding to R/. Deterioration RFTS not responding to R/. Pneumothorax Haemoptysis (>20ml) Haemoptysis (>20ml) ABPA Significant atelectasis Significant atelectasis Respiratory/cardiac failure Meconium ileus Meconium ileus Acute surgery Elective major surgery Elective major surgery GI bleeding Liver/gall bladder complications. Jaundice Liver/gall bladder complications. Jaundice Insertion of TIVADs For gastrostomy For gastrostomy Unexplained wt loss or FTT Glucose intolerance Glucose intolerance Vasculitis, Arthropathy Pregnancy, fertility issues Pregnancy, fertility issues Serious psychological problems Transplant assessment Transplant assessment Osteoporosis Renal disease Renal disease CF related ENT problems

41. Role for many other specialists Gastroenterology Gastroenterology Surgeons – Transplant and others Hepatology Hepatology Diabetes Bone disease Bone disease ENT surgeon Obstetrics & Gynaecology Obstetrics & Gynaecology Fertility Genetic counselling Genetic counselling X-rays & imaging

42. Staff member (per 50 patients) Specialist Paediatric Centre Specialist Adult Centre Consultant 10.5 Consultant 20.2-0.3 Staff Grade0.40.6 CF Sp.Registrar0.5 CF Nurse1-1.51.0 -1.5 Physio0.5 – 1.01.0 Dietitian0.4 Social worker0.4 Psychologist0.4 Pharmacist Secretary 0.3 1.0 0.3 1.0 Data Clerk0.1

43. US CF Foundation’s “FATAL FLAWS” No regular team meetings No regular team meetings Insufficient clinic time to see patients Too many patients per clinic Too many patients per clinic Unreliable sweat test technique Incomplete 24 hr expert CF coverage Incomplete 24 hr expert CF coverage No adult care plan No backup physician No backup physician Less than 50 patients Less than 50 patients

44. There is room for improvement in CF care Wide variation between the “best” and the “rest” Respiratory function (FEV1) differs by 10% Median age of death varies from 23.2 to 28.2 yrs Improving “rest” to the “best” would increase life expectancy by 7 years These differences now receiving much attention National CF registries are essential to show these differences Best Rest

45. Successful CF doctors Lead and support a caring expert CF Team Well informed & have wide experience Focused and aggressive Think hard and often about their patients Never satisfied with present treatment Inventive – tries new approaches Push patients with treatment – kindly! Capacity to learn and adapt rapidly Involve expertise of other departments and colleagues in a large hospital Successfully enlist support of hospital managers Do research with staff and publish results Do research with staff and publish results [Inspired by “The Bell Curve”. Gawande G, New Yorker, Dec 6th 2004] >50 PATIENTS ESSENTIAL >50 PATIENTS ESSENTIAL

46. Standards of care for patients with cystic fibrosis: a European Consensus Standards of care for patients with cystic fibrosis: a European Consensus Kerem, Conway, Elborn, Heijerman et al for the Consensus Committee J of Cystic Fibrosis 2005;4:7 To definefor the routine evaluation, monitoring and treatment of patients with CF in Europe To define standards for the routine evaluation, monitoring and treatment of patients with CF in Europe A basis forCF care A basis for auditing CF care Provides data for between CF Centres Provides data for comparison between CF Centres

47. Standards of care for patients with cystic fibrosis: a European Consensus Standards of care for patients with cystic fibrosis: a European Consensus Kerem, Conway, Elborn, Heijerman et al for the Consensus Committee J. of Cystic Fibrosis 2005;4:7-26 “A Roadmap to Best Care” for “A Roadmap to Best Care” for people with Cystic Fibrosis people with Cystic Fibrosis Littlewood. Editorial. J of Cystic Fibrosis 2005;4:1-5