CARE-NMD results. Jan Kirschner

Transcription

1 CARE-NMD results Jan Kirschner TREAT-NMD Alliance Meeting Newcastle 30 Oct -1 Nov 2013

2 Care recommendations for DMD Consensus process 2

3 Care recommendations for DMD Consensus process Scientific publication 3

4 Care recommendations for DMD Consensus process Scientific publication Family guide 4

5 Care recommendations for DMD Consensus process Scientific publication Family guide?? Care providers Patients/Families? 5

6 Current treatment situation Although guidelines are published many patients do not receive recommended treatment for many reasons Treating health professionals are not trained adequately No access to reference centers No resources for treatment according to recommendation Quality of life and life expectancy varies significantly Few data available concerning health services for DMD 6

7 TREAT-NMD infrastructure Patient registries for DMD in more than 40 countries DMD Registries 7

8 TREAT-NMD infrastructure Patient registries for DMD in more than 40 countries Care and Trial Site Registry with data on more than 250 neuromuscular centres with a worldwide distribution DMD Registries 8

9 TREAT-NMD infrastructure Patient registries for DMD in more than 40 countries Care and Trial Site Registry with data on more than 250 neuromuscular centres with a worldwide distribution Partners in most European countries DMD Registries 9

10 Objectives of CARE-NMD Evaluate current care practice across Europe Identify reasons for non-compliance with recommendations Lack of knowledge/training Professional attitude Financial reasons Social/ethical reasons Assess impact on quality of life of patients with DMD Dissemination and Training Establish a reference network of care centres 10

11 CARE-NMD project EU Health Programme (DG Public Health) Promote health including the reduction of health inequalities Generate and disseminate health information and knowledge CARE-NMD receives funding for 3 yrs (from May 2010) Total budget of 1.6 Mill. (EU-contribution: 0.9 Mill.) 11

12 CARE-NMD partners University Medical Center Freiburg, Germany University of Newcastle, United Kingdom Sofia Medical University, Bulgaria University Hospital Brno, Czech Republic Hungarian Institute of Environmental Health, Hungary Medical University of Warsaw, Poland The National Danish Rehabilitation Centre for Neuromuscular Diseases, Denmark 12

13 CARE-NMD partners University Medical Center Freiburg, Germany University of Newcastle, United Kingdom Total of 1,677 DMD patients Sofia Medical University, Bulgaria University Hospital Brno, Czech Republic Hungarian Institute of Environmental Health, Hungary Medical University of Warsaw, Poland The National Danish Rehabilitation Centre for Neuromuscular Diseases, Denmark 13

14 Partners (associated and collaborating) 14

15 Reference network of care centres Questionnaires to all care centres in participating countries 24 questions on key care settings and common practice Online questionnaire added to existing CTSR data Questions covering different aspects of DMD care centres Many questions based on EU criteria for RD reference centres Members of multidisciplinary team Involvement in clinical research and networks Training activities Specific question about common practice for DMD patients Reimbursement and other hurdles to apply care recommendations Identify at least one DMD care centre per 5 Mill. inhabitants 15

16 Dissemination and training Translation and distribution of family guide in all national languages (now available in 21 languages) Lectures at conferences and patient advocacy meetings, mailings to schools for special needs, TV spot and radio interview, publications in scientific journals Training workshops in particpating countries according to individual needs planned for 2012 Patient workshops planned towards the end of the project to discuss results and possible ways to improve care for DMD patients/families 16

17 Dissemination and training 17

18 Dissemination and training 18

19 Dissemination and training 19

20 Evaluate Care Practice and QoL Questionnaires to all registered patients 42 questions on key aspects of daily life and medical care Online and paper version in all 7 languages Current functional ability and disease stage Education and participation in society Outcome indicators (e.g. age at loss of ambulation, days in hospital) Process indicators (e.g. frequency of cardiac/pulmonary assessment) Assess impact on Quality of Life Standardized quality of life questionnaires Child and parent versions for different age groups PedQL, KIDScreen, SF36, WHO-QoL Bref 20

21 Patient survey: Topics Personal data Age, education, occupation, household income Participation in society, patient advocacy groups Functional status, activities of daily life, assistive devices Medical care Neuromuscular center, frequency of assessment, hospital admissions Diagnosis, Counselling about DMD Use of steroids Cardiac and respiratory treatment Satisfaction with medical treatment 21

22 Age at diagnosis Diagnostic tools Steroid treatment for ambulant and non-ambulant Management of side effects Physiotherapy Stretching Assistive technology Scoliosis surgery Quality of Life Psychosocial and educational support Pulmonary assessment and ventilatory support Cardiac assessment and treatment 22 Lancet Neurology 2010

23 Patient/Family questionnaire GER HU CR DK UK BG PL Total Registered patients Total responses Response Rate 78% 81% 47% 60% 54% 55% 58% 64% Final response rates 23

24 Patient survey: Cohort The mean patient age was 13.1 yrs (range ) 17% of patients were aged >18 years 46 % of patients were ambulatory, 52 % were not 24

25 Patient survey: Cohort 50 Percentage patients >18 yrs (17% of total population) UK DE BG HU PL CZ DK 25

26 Patient survey: Cohort Patients (%) per stage of disease 45% 26% 14% 4% 7% Presymptomatic Early-ambulatory Late-ambulatory Early non-ambul. Late non - amb. 26

27 Key findings Loss of ambulation has been delayed through current care practice including steroid treatment Most but not all patients are offered steroid treatment The number of non-ambulatory and adult patients is constantly increasing Adult patients receive less physiotherapy and are often not seen at a neuromuscular centre Patients seen at neuromuscular centers receive more information and treatment according to recommendations 27

28 Thank you! Kick-off meeting Luxembourg