Assessing quality of life in sarkoma trials

Transcription

1 Pan-European Clinical Trials under current EU regulations: A training course for data managers, study nurses, and junior clinical investigators London, January 24/25, 2008 Assessing quality of life in sarkoma trials Gabriele Calaminus, MD QoL working group Dept. of Paediatric Oncology, Haematology, Münster, Germany 1

2 Frequency of sarcoma 8,3 6,6 4,5 11,1 % Sarcomas 12,2 6 3,4 4,5 21,4 33,1 soft tissue sarcomas bone tumours renal tumours Germ cell tumours other diagnosis Leukaemias CNS tumours Lymphomas Sympathetic nervous system tumours * Kaatsch et al. :Annual Report 2005 German Childhood Cancer Registry 2 6

3 Treating children with cancer Survival is the goal 3

4 Development of Survival Rates Years Survival Rates [%] Year of diagnosis Osteosarcoma Ewing s Sarcoma Rhabdomyosarcoma Morbus Hodgkin Wilms Tumor Acute lymphocytic leukemia Non-Hodgkin-Lymphoma Malignant germ-cell tumours Neuroblastoma Brain tumors Acute myelogenous leukemia 4

5 The Erice Statement

6 What is Quality of Life? the individual perspective in respect to aims, expectations, possibilities influenced by cultural environment. it contains but is not limited to social, emotional and physical capacities of the child / adolescent and his family it has to be viewed from the perspective of the child / adolescent and the family and should include developmental changes Bradlyn,

7 Child outward appearance (nice hair) physical activity (to run, climb, play...) friends Adolescent friends, body image, outward appearance, self dependency, professional training Young adult Autonomy (income, to live independent) professional training, job steady partnership 7

8 Factors influencing QoL age range prognostic variability education Health- /social system treatment diversity duration intensity mutilation compliance Psychosocial network patient / family Quality of life 8 2

9 Measuring QoL Why... How Analysis Interpretation Timing Logistics... 9

10 Measuring QoL: Why? 10

11 QoL data provide additional information to compare treatment strategies Additional information to survival data detection of changes in psychosocial functioning value of physical and psychosocial rehabilitation value of palliative care 11

12 Measuring QoL: How? 12

13 Interviews Limitations: data security, staff, time consuming self / proxy report Questionaires Limitations: different view on QoL and problems self reporting 8 years Limitations: age, ability to understand things 13

14 Analysis Evaluation of scores for different domains and correlation to Norm Groups - Age and sex Significant differences (negative/positive) Over time (E1 E2.. E3 same treatment At one time point between treatment arms 14 2

15 Examples 15

16 Quality of Life in schoolchildren n= % Prozente negativ 60 Autonomiy E K p < 0.05 C 40 Body image p < 0.05 FR J J J. FAM 16

17 Longitudinal comparision of QoL data E2 E3 E4 Week 10 ± 2 post surgery Week 72 ± 4 Week 144 ± 4 E2 E3 E4 E1 Week 10 ± 2 post surgery Week 72 ± 4 Week 144 ± 4 Week 5 E2 E3 E4 Week 10 ± 2 post surgery Week 72 ± 4 Week 144 ± 4 E2 E3 E4 Week 10 ± 2 post surgery Week 72 ± 4 Week 144 ± 4 17

18 Cross sectional comparision of QoL data E2 E3 E4 Week 10 ± 2 post surgery Week 72 ± 4 Week 144 ± 4 E2 E3 E4 E1 Week 10 ± 2 post surgery Week 72 ± 4 Week 144 ± 4 Week 5 E2 E3 E4 Week 10 ± 2 post surgery Week 72 ± 4 Week 144 ± 4 E2 E3 E4 Week 10 ± 2 post surgery Week 72 ± 4 Week 144 ± 4 18

19 Interpretation Correlation to Norm Groups Age and sex Correlation to treatment Surgery, chemo, RT Correlation to somatic data toxicity, functional abilities 19 2

20 Timing Intervals between evaluation > 6 weeks Time window not to short Instruments easy assessable Reminder system effective 20 2

21 Logistics 21 2

22 Flow of QoL information / data in Germany E1 / E2 COSS Stuttgart data synchronisation announcement of registration QoL-center Düsseldorf Deutsches Krebsregister Mainz Attending clinic/physician Patient / Parents E1 and E2 obligatory reminder / QoLbooklets were posted to but returned by patient/parents directly 22

23 Sarcoma What do we know about QoL? 23

24 3/16 (20%) of hospitals account for 60% of Ewing and osteosarcoma patients schooling/vocational training is of high importance in terms of successful long-term rehabilitation. This enables accessible employment, security and the ability to build up own family structures No difference for amputees and non-amputees 24 2

25 3/16 (20%) of hospitals account for 60% of Ewing and Osteosarcoma patients At assessment median age was 15 years, Mean scores were 60, 81, 76, 74, 70, 87 for general health, physical functioning, pain, mental health, self- esteem, family activity Lower results were observed for mental health in girls, for physical functioning, and self-esteem in patients with endoprosthesis, for family activity and pain in patients who had relapsed. 25 2

26 3/16 (20%) of hospitals account for 60% of Published studies patients The main impact on Qol has the ability to participate in normal life roles: get and maintain employment have a familiy and care for it participate in recreational activites 26 2

27 Clinical Outcome and QoL? 27

28 Examples 28

29 Craniopharngeoma impact of RT after incomplete resection 29

30 +/- RT do not implicate a difference in QoL in patients with incompletely resected craniopharngeoma > 2 years after the end of treatment % Median SE E KB C K Fam Fr Normgroup, n=550 - RT n=16 + RT n=21 0 (11/04) 30

31 obesity in Craniopharngeoma 2 years after Dx 31

32 Obese Craniopharyngeoma pts rate their QoL more negative than non-obese pts % Median SE E KB C K Fam Fr BMI > 3 SD BMI < 2 SD p <

33 Cross sectional / longitudinal QoL study (prospective, multicenter) Example: COSS/EURAMOS 1 Investigation of Quality of Life (QoL) in young patients with Osteosarkoma (as one secondary end of the trial) Trial opening in Germany 04/

34 COSS/EURAMOS 1 problems of interest 1. How do young patients with Osteosarkoma appraise their QoL? 2. Is there a difference in QoL between good and poor responders? (cross sectional comparison) 3. Does the additional application of interferon-alpha affect the QoL in patients with good prognosis? 4. Is the QoL affected in patients with poor responding and more intensive therapy? 5. Does the QoL change during and after therapy? (longitudinal comparison) 34

35 Further areas of interest Influence of disease age treatment on QoL surgery irradiation 35

36 Quality of Survival! professional rehabilitation functional outcome Risks histo/site therapy late effects Genetics Sarcoma patient Coping subjective well-being Intact social network Access to insurrance and health care 36

37 QoL working group (Münster) Gabriele Calaminus, MD Pediatrist Head QoL working group Andreas Wiener Pediatric Psychologist Study Coordinator Carmen Teske Data Manager Contact address: Dr. G. Calaminus University Children's Hospital Dept. of Paed.Oncology, Haematology University Hospital Albert-Schweitzerstr.33 D Münster Germany phone:

38 Thank you for your interest 38