WAA registry data of LDL-apheresis and concepts of how to enable a comparison with a control group

1. WAA registry data of LDL-apheresis and concepts of how to enable a comparison with a control group Dr. med. Heinrich Prophet Dialysegemeinschaft Nord, Apheresezentrum Rostock, Deutschland

2. WAA Registry • Why ? • Visualize apheresis activity and bundle efforts in this field. • Recognize indications, techniques, side effects and outcomes. • Compare those modalities nationally and internationally. • Follow apheresis trends over time. • Improve safety and quality of therapy.

3. WAA Registry • Features: • Specific coding of center and patient • No expenses for the collaborating center • Non-competitive evaluation • Single or multi center studies are feasable

4. WAA Registry • Participants (march 08): • 75 centers applied for a login code • 15 centers in 7 countries enter actively • 2013 patients with 12448 procedures have been submitted

5. WAA Registry – LDL apheresis fraction • Participants (august 2008): • 6 centers in 4 countries (Sweden, Lithuania, Czech Republic, Germany) have entered LDL-apheresis procedures • 53 patients with 2740 procedures have been submitted

6. WAA Registry – LDL apheresis fraction • Participants (august 2008):

7. WAA Registry – LDL apheresis fraction • Participants (august 2008):

8. WAA Registry – LDL apheresis fraction • Patients characteristics (august 2008) : • mean age: 50,2 years (10-76) • gender distribution: 31 men : 22 women (58,5:41,5 %)

9. WAA Registry – LDL apheresis fraction • Patients characteristics (august 2008) : • Diseases leading to LDL-apheresis

10. WAA Registry – LDL apheresis fraction • Patients characteristics (august 2008) : • Blood access for chronic LDL-apheresis:

11. WAA Registry – LDL apheresis fraction • Treatment characteristics (august 2008) : • Devices used for direct adsorption/primary separation

12. WAA Registry – LDL apheresis fraction • Treatment characteristics (august 2008) : • Devices used for plasma treatment

13. WAA Registry – LDL apheresis fraction • Treatment characteristics (august 2008) : • Anticoagulation

14. WAA Registry – LDL apheresis fraction • Outcomes (august 2008) : • VAS of functional ability • In only 803 of 2696 therapies submitted (29,8 %)

15. WAA Registry – LDL apheresis fraction • Outcomes (august 2008) : • VAS of quality of life • In only 248 of 2696 therapies submitted (9,2 %)

16. WAA Registry – LDL apheresis fraction • Outcomes (Center Rostock, august 2008) : • All cardiovascular events (MI, PCI, coronary or peripheral bypass) in 29 LDL-apheresis patients of the Rostock Apheresis Center before and after start of apheresis *p<0,001 *

17. WAA Registry – LDL apheresis fraction • Outcomes (Center Rostock, august 2008) : • All cardiovascular events (MI, PCI, coronary or peripheral bypass) in 29 LDL-apheresis patients of the Rostock Apheresis Center before and after start of apheresis n=29

18. WAA Registry – LDL apheresis fraction • Adverse events (august 2008) : • In 2740 sessions only 115 adverse events were stated (4,2 %) n=115

19. WAA Registry – LDL apheresis fraction • Adverse events (august 2008) : • Women show more side effects than men * p=0,014 *

20. WAA Registry – LDL apheresis fraction • Adverse events (august 2008) : • Women drop off more frequent than men * p=0,012 *

21. WAA Registry LDL apheresis – a concept to compare outcomes with a control group • Problems: • Small group of patients with LDL-apheresis • Only few countries with reimbursement • Ethical concerns with an apheresis sham group in this high risk population

22. WAA Registry LDL apheresis – a concept to compare oucomes with a control group • Proposal for a solution: • International control group with patients actually showing the indication for apheresis in countries where therapy is not available • Both groups with optimal conventional therapy • As new study or as historical control (e.g. with established databases)

23. WAA Registry LDL apheresis fraction – Conclusions • Conclusion I: • WAA registry LDL fraction is still relatively small • Yet the submitted data gives information about modalities, quality und results of LDL-apheresis therapy in different countries (so far only Europe)

24. WAA Registry LDL apheresis fraction – Conclusions • Conclusion II: • Number of patients per center differs considerably • Men are more frequent treated than women • Diagnoses leading to apheresis are rather unspecific • Peripheral access is mostly used • Prevailing techniques: DALI and Octo (MDF) • (eg) Citrate is the leading anticoagulant

25. WAA Registry LDL apheresis fraction – Conclusions • Conclusion III: • LDL-apheresis is well tolerated • Women show more side effects and drop off more often than men • Patients show a good quality of life and a rather good functional ability under LDL-apheresis • Cardiovascular events are clearly reduced (Rostock, and personal information from Prague) • Studies are needed to prove this in general • A control group could include maximally but not sufficiently treated patients with no access to LDL-apheresis or historical controls