The Public Presentation does not include any confidential information or findings from this Team Masters Project with BioMarin Pharmaceutical. BioMarin and KGI titled our project as: "Recurrent bleeding in patients with mechanical flow devices: an environmental study."

1. RECURRENT BLEEDING IN PATIENTS WITH
MECHANICAL FLOW DEVICES:
AN ENVIRONMENTAL STUDY
TMP Faculty Presentation, Spring 2016
Courtney Lamb, Cyrus Nguyen, Jill Nicholas,
Hitesh Pathak, Suchitra Ramachandran
1

2. Executive Summary
Project Objectives
• Current therapeutic
approach
• Business opportunity
• Clinical trial concepts
Methods
• Secondary literature
research
• Primary research
• KOL interviews
• Physician Survey
Outcomes
• Target product profile
• Business
assessment
• Clinical trial concepts
Current State
Recurrent gastrointestinal bleeding is recognized as a significant medical problem in
patients following implantation of Left Ventricular Assist Devices (LVADs). Shear stress
applied by LVADs induces Acquired von Willebrand Syndrome. There is no current
therapy indicated to specifically address this type of bleeding.
2

3. CLINICAL LANDSCAPE
3

4. Left VentricularAssist Devices and GI Bleeding
LVADs
• Assist the heart in pumping blood in
advanced heart failure patients
Uses
• Bridge to Transplant (BTT)
• Destination Therapy (DT)
• Bridge to Candidacy (BTC)
• Bridge to Recovery (BR)
Risks and Complications
• Development of clots, pump
thrombosis, damage to heart
valves, discomfort or pain
• Bleeding: Gastrointestinal bleeding
GI Bleeding
• ~22 percent of LVAD patients
develop GI bleeding
• ~60 percent of these patients
developed a recurrent bleed
Possible Causative Factors
• Treatment related
• Chronic anticoagulation
• Device related
• Platelet dysfunction
• Induced shear stress
Bleeding complications can arise after implantation of LVADs due to increased shear
force associated with the pump
https://www.nhlbi.nih.gov/health/health-topics/topics/vad
Loor, G. & Gonzalez Stawinski, G. Best Pract. Res. Clin. Anaesthesiol. 26 (2012), 105–115
Aggarwal, A. Annual Throacic Surgery. (2010)
4
4

5. Changes in vWF structure after LVAD implantation
• Von Willebrand Factor (vWF) in its multimeric structure is essential for
hemostasis
• Increased shear forces due to LVADs induce conformational change, unravel
VWF and increase its protease susceptibility
• Change in vWF structure and the associated decrease in vWF multimers
increases bleeding risk
While LVADs serve to treat one disease, they also cause another. Therefore, a novel
solution is needed to address this significant issue
Franchi, F., et al. Thromb. Res. 2014, 134 (6), 1316–1322. Geisen, U., et al. Eur. J. Cardiothorac. Surg. 2008, 33 (4), 679–684.
5
5

6. Von Willebrand Disease (VWD)
• A group of bleeding disorders characterized by any
deficiency in or defective functioning of the von
Willebrand factor (vWF)
• vWF: glycoprotein that carries factor VIII and stimulates
platelet aggregation
• Congenital VWD: three major types
1. Type 1: characterized by vWF deficiency
2. Type 2: characterized by functional vWF defects
• Further categorized into 4 subgroups
• Type 2A: most common
3. Type 3: characterized by complete vWF absence
• Acquired von Willebrand Syndrome (AVWS) – vWD
developed secondary to other diseases
6

7. Acquired von Willebrand Syndrome (AVWS)
• Genetic and acquired VWD are
characterized by:
• Lack of high molecular weight vWF
multimers due to proteolysis
• A causal link to increased bleeding
risk
Franchi, F., et al. Thromb. Res. 2014, 134 (6), 1316–1322.
Geisen, U., et al. Eur. J. Cardiothorac. Surg. 2008, 33 (4), 679–684.
7
7

8. Diagnosis of AVWS
Doctors treat LVAD patients with a GI bleed under the assumption that they
HAVE AVWS because of the high shear stress
Initial evaluation
(history and physical
examination)
No further evaluation
Initial VWD assays
• VW: Ag
• VWP: Rco
• Factor VIII
Referral for other
appropriate
evaluationReferral for selected specified VWD
studies
• Repeat initial VWD assays if
necessary
• Ration of VWF: RCo to
VWF:Wg
• Multimer distribution
• Collagen binding
• Factor VIII binding
• Platelet VWF studies
• DNA sequencing for VWF gene
Possible referral for other
appropriate evaluation
Positive Negative
Isolated prolonged PTT that
corrects on 1:1 mixing study or no
abnormalities
No test abnormal1 or more tests
abnormal
Other cause identified low
platelets, isolated abnormal
PT, low fibrinogen,
abnormal TT
Laboratory evaluation Initial
hemostasis tests
• CBC and platelet count
• PTT
• PT
• Fibrinogen or TT
(optional)
If bleeding history is strong,
perform initial VWD assays
U.S. Department of Health Services. NIH: National Heart, Lung and Blood Institute. 2015.
Dr. Slaughter, Interview, 2015; Dr. Tiede, Interview, 2015.
8

9. • Lymphoproliferative disorders
• Immunological disorders
Autoantibody degradation of vWF
• Myeloproliferative disorders
Adsorption of vWF to surfaces of transformed platelets
and cells
• LVAD implantation
• Aortic stenosis
• Dysfunctional valve prosthesis
• Endocarditis
Proteolytic cleavage due shear stress induced
unfolding
The multiple mechanisms ofAVWS induced by
comorbidities
Understanding of the various diseases and mechanisms associated with AVWS
is important in understanding the optimal therapy for AVWS patients
Tiede, A. et al. Blood 2011, 117 (25), 6777–6785.
Velik-Salchner, C. et al. J. Cardiothorac. Vasc. Anesth. 2008, 22 (5), 719–724.
9

10. There are three goals in the treatment for GI
bleeding in LVAD patients:
Current therapeutic strategy
Control acute bleeding
Prevent bleeding in high risk situations
Obtain long term remission
1
2
3
10

11. For LVAD patients, long-term treatment options are
limited and primarily based on case studies
Variability in the underlying disorders and mechanisms of AVWS
precludes one standardized course of treatment
Whitlow, C. B. Clin. Colon Rectal Surg. 2010, 23 (1), 31–36.
Cerulli, M. A. Medscape 2015.
Tiede, A., et al. Blood 2011, 117 (25), 6777–6785.
Geisen, U., et al. Eur. J. Cardiothorac. Surg. 2008, 33 (4), 679–684.
Acute Treatments for GI
bleeding
• Hemostatic therapies
administered
endoscopically
• Submucosal injection of
epinephrine
• Bipolar electrocoagulation
via cauterization
• Hemostatic Clips
Treatments for Prevention
and Control of AVWS
• Desmopressin
• VWF-containing Factor VIII
concentrates
• Recombinant Factor VIIa
• Antifibrinolytics
11

12. Physician survey suggests inadequacy of current
treatment options
0
0.5
1
1.5
2
2.5
3
3.5
4
4.5
Not satisfied Neutral Very
satisfied
Satisfaction with Current Treatment Options (n=12)
Hematology Gastroenterology
12

13. The balancing act between anticoagulation and
thrombosis in patients with LVADS
GI Bleeding Event
• Hold anticoagulation/antiplatelet therapy
• GI consult to identify bleeding source
• Endoscopic treatment of bleeding source
Resolution of Bleeding?
Resume anticoagulation
Recurrent Bleed
• Hold anticoagulation until resolution. When
bleeding stops, resume anticoagulation
OR
• Hold anticoagulation indefinitely
Yes No
In managing bleeding in LVAD patients, there is a careful balance between resolving the
bleed and increasing the risk of thrombosis through prolonged holding of anticoagulation
Suarez, J., et al. Am. Heart Assoc. 2011, No. Circ Heart Fail. 2011;4:779-784, 4:779–784.
13

14. 0 2 4 6 8 10 12 14 16 18
None
Marginal
Moderate
Significant
Perception of a Need for a Novel Therapeutic
Cardiology Hematology Gastroenterology
Physician survey supports the conclusion
that there is an unmet clinical need
The “underlying pathophysiology induced by the VAD fosters the on-going
formation of AVMs. So even if some are found and treated, they invariably recur
as the underlying physiologic changes are still present”
Dr. Wild, Gastroenterologist, Duke Medical School
“We don’t want our patients bleeding all the time but we also don’t want to mess up
the pump by keeping them off anticoagulation forever. Is a tough balance.”
Dr. Russell, Cardiologist, Johns Hopkins
“If we had better tolerated, more effective preventive medical therapies, that
would be ideal.”
Dr. Draper, Gastroenterologist, Stanford
14

15. BUSINESS
OPPORTUNITY
15

16. Assumptions from KOL feedback
The majority of physicians are interested in and willing to
prescribe a new medication to stop bleeding due to LVAD
implantation. It is agreed that there is a great medical
need.
Contrary to secondary literature research, KOL interviews
indicate that LVAD implantation is used increasingly as a
destination therapy, rather than a bridge to transplant.
Regardless of improvements in pump technology,
bleeding will continue to be a serious complication for
LVAD patients.
16

17. LVADs increase survival rates
• In most patients without an LVAD, the one-year survival rate is 10%
• Most doctors interviewed have recommended LVAD implantation as a
destination therapy
Because of the increase in survival rates in patients with end stage heart failure due to
LVAD implantation, we can assume that market will continue to increase
Intermacs Quarterly Statistical Report. 2015.
Dr. Tiede, Interview, 2015; Dr. Arabia, Interview, 2015.
0
20
40
60
80
100
0 10 20 30 40 50 60 70
% survival
months after device implantation
Kaplan-meier survival plot
BTT
BTC
DT
17
17

18. LVAD implant incidence and adverse events
.
Table: Adverse event June 2008 to Dec 2014 in first 12 month Post Implant (N=12,030)
Adverse event 2008-2011
(events)
2008-2011
(Rate)
2012-2014
(Events)
2012-2014
(Rate)
P-value
Bleeding 3,932 9.41/100
patients
4,420 7.79/100
patients
<0.001
0
200
400
600
800
1000
1200
1400
2006 2007 2008 2009 2010 2011 2012 2013 2014 2015
Implants per year by device type
BTT
BTC
DT
BR
Even with the improvement in technology of LVADs, bleeding is still a significant risk
Intermacs Quarterly Statistical Report. 2015.
18

19. Survey results support business assumptions regarding
improved pump designs
0 2 4 6 8 10 12 14
None
Marginal
Moderate
Significant
Impact of Improvement in Pump Design
Cardiology Cardiothoracic Surgery
19
19

20. Reasons to believe this is an attractive market
LVADs are highly underutilized
• 3,000 heart transplants are performed each year worldwide
• 15,000 patients are on waiting lists for transplants
Improvement in
technology
Increase in
adoption rate
Larger market
for AVWS
“I know 2 different devices (HeartWare and HeartMate) that are different in the
way blood is pumped through, but the AVWS occurs similarly with both. I am not
aware of an LVAD that doesn’t cause AVWS.”
- Dr.Tiede interview, 2015
20

21. CLINICAL TRIAL
CONCEPTS
21

22. Analysis of current clinical studies regarding LVAD
patients,AVWS, and GI bleeding
Unmet Need
• Pump design:
Moderate impact
• Novel therapy: a
significant
requirement (61%)
• Dissatisfaction
with current AVWS
treatment options
Case
Studies
• 18 trials reviewed
• Trials pertaining to
LVADs and GI
bleeding,
AVWS/VWD, and
general GI
bleeding
• Trial comparison
Challenges
• Total enrollment in
trial
• Defining
primary/secondary
endpoints
• Endpoint
satisfaction
• Bleeding definition
• Trial sites
No clinical trial currently exists that combines both BioMarin’s target
patient population and a new therapeutic strategy
22

23. Research outcomes indicated differences in
current trial parameters
• History of two or more severe GI bleeding episodes associated
with either a drop in hemoglobin of ≥ 2 g/dl or requiring red blood
cell transfusion or treatment with VWD concentrate
• Internal or external bleeding leading to death or prolonged
hospitalization or requiring re-hospitalization
Bleeding
• Treatment success will be defined as a mean efficacy rating score
of < 2.5 for a participant´s bleeding episodes treated with the
investigational product while in a treatment period.
• Cessation of Bleeding [Time Frame: 52 months ]
Primary
outcomes
• Need for transfusion [ Time Frame: 48 hours post administration ]
• von Willebrand study and protein electrophoresis; multimer
analysis
• Proportion of VAD Patients with Gastrointestinal bleeding as
Assessed by HemoQuant Fecal Occult Blood Testing
Secondary
outcomes
https://clinicaltrials.gov
23
23

24. Important considerations in designing trial
Bleeding criteria is useful and relevant to
physicians
Differentiation of post-operative bleeding and
device-related bleeding
• Larger sample size would increase costs and difficulty
Trial sites and patient recruitment
• Could skew trial results
• Increase risk of adverse events: thrombosis
Predictability of development of AVWS
24
24

25. Summary
Unmet Need
•GI bleeding is a
significant
complication for
LVAD patients
•Current therapeutic
approach is
inadequate
•61% of doctors
perceive a significant
need for a new
therapeutic
Business
Opportunity
•A greater number of
LVADs are needed
than are implanted
•Improvements in
pump design
technology
•Market for
therapeutic targeting
AVWS from LVADs is
growing
Clinical Trials
•No current equivalent
clinical trial
•Specifics such as
bleeding criteria and
outcomes were
complied from 18
separate but related
trials
25
Recommendation
The clinical need and market opportunity clearly indicate that this project holds great
potential for BioMarin and should be further developed

26. Acknowledgments
Dr. Andrea Cooper Rajeev Mahimkar Dr. Craig Adams
(Faculty Advisor) (Corporate Liaison) (TMP Director)
26

27. Thank you & Questions?
27

28. APPENDIX A: METHODS
28

29. Timeline of Deliverables
Aug Sep Oct Nov Dec Jan Feb Mar Apr
Clinical Landscape
Literature Research
KOL Interviews
Business Opportunity
Physician Survey
Clinical Trial Concepts
KOL Interviews
29
29

30. Primary Research - KOL Interviews
KOL Interviews
Phone interviews
with 9 KOLs
4 Main specialties
• Gastroenterology (2)
• Cardiothoracic
Surgery (4)
• Cardiology (1)
• Hematology (1)
1 research
scientist – VWF
30
30

31. APPENDIX B: PRESENTATION
SUPPLEMENTS
31

32. Current Treatment Options
Desmopressin
Indication Manage spontaneous or trauma-induced bleeds in patients with
hemophilia A or von Willebrand's disease Type I
Dosage forms Oral
Intranasal
Parenteral (IV)
Half-Life Oral: 1.5-2.5 hours
Intranasal: 3.3-3.5 hours
Injection: Initial = 7.8 minutes, terminal = 0.4-4 hours
Protein binding 50%
Prices Apo-Desmopressin 0.1 mg Tablet = $0.83
Ddavp 0.01% nasal spray = $50.76
Ddavp 0.1 mg/ml Soltuion = $21.26
Ddavp 0.2 mg tablet = $2.98
32

33. Current Treatment Options
VWF-containing Factor
VIII concentrate
Indication For the treatment of hemophilia A, von Willebrand disease
and Factor XIII deficiency
Dosage forms Powder for solution
Half-Life 8.4-19.3 hrs
Clearance 4.1 mL/h•kg [Previously treated pediatric patients]
Prices $1.20 - $1.68/vial
33

34. Current Treatment Options
Recombinant
Factor VIIa
Indication For treatment of hemorrhagic complications in hemophilia A and B
Dosage forms Powder for injection
Volume of
Distribution
121 ± 30 mL/kg [adults]
153 ± 29 mL/kg [children]
280 to 290 mL/kg [congenital Factor VII deficiency]
Clearance 33 – 37 mL/h x kg [healthy]
1375 +/- 396 mL/hr [severe hemophilia A male children]
57.3 +/- 9.5 mL/hr/kg [severe hemophilia A male children]
2767 +/- 385 mL/hr [severe hemophilia A men]
37.6 +/- 13.1 mL/hr/kg [severe hemophilia A men]
Price $1.64/vial
34

35. Current Treatment Options
Anti-
fibrinolytics
Tranexamic Acid Aminoaproic Acide
Indication For use in patients with hemophilia
for short term use (two to eight
days) to reduce or prevent
hemorrhage
For use in the treatment of
excessive postoperative bleeding
Dosage forms Cream
Solution
Tablet
Solution
Syrup
Tablet
Half-life ~3 hours ~2 hours
Volume of
Distribution
9 to 12 L 23.1 ± 6.6 L
Clearance 110 – 116 mL/min 169 mL/min
Price $1.30 - $8.80/unit $2.28 - $7.17/unit
35

36. APPENDIX C: SURVEY
RESPONSES
36

37. Survey Design
- Initial page assesses demographic information and
speciality
- Different survey design based on speciality
- Logic designed to terminate survey if respondent did not
have sufficient knowledge in the area
- 41 respondents so far
- Followed up with 11 respondents to further assess unmet
needs
37
37

38. Arizona, 1
California, 15
Colorado, 3
Florida, 1
Maryland, 5
New York, 1
North Carolina, 12
Pennsylvania, 1
Texas, 2
Q1: In which state is your primary practice located? (n=41)
38

39. 12
19
4
3 3
0-9 years 10-19 years 20-29 years 30-39 years 40+ years
Q2: How long have been practicing medicine? (n=41)
39

40. 0 0
6
40
1
Private practice Group practice Hospital based Academic medicine Other (please specify)
Q3: Which of the following best describes your practice (select all that
apply)? (n=41)
40

41. 16
7
14
3
0
1
Cardiology Cardio-thoracic surgery Gastroenterology Hematology Research Other
Q4: Which of the following best describes your specialty?
(n=41)
41

42. SURVEY RESPONSES
Cardiologists
42

43. 0
2
1
4
0
4
5
Not familiar Moderately
familiar
Very Familiar
Q5: Familiarity with AVWS - Cardiology
(n=16)
0.00 1.00 2.00 3.00 4.00 5.00 6.00
Familiarity with AVWS - Cardiology Average
43

44. 3
2
3
1
7
0-20% 21-40% 41-60% 61-80% >80%
Q6: Approximately what percentage of your patients are diagnosed
with heart failure? (n=16)
44

45. Bridge to Transplant
50%
Destination Therapy
50%
Q8: What is the primary therapeutic purpose for implantation of
LVADs? (n=16)
45

46. 1 1
4
1 1
0
0-1 year 1-2 years 2-3 years 3-4 years 4-5 years >5 years
Q9: How long on average is a patient on an LVAD as
destination therapy? (n=8)
46

47. Yes
87%
No
13%
Q10: Is the LVAD ever replaced when used as destination
therapy? (n=8)
47

48. Device malfunction
86%
Other
14%
Q11: What is the primary reason for the replacement? (n=7)
48

49. 9
5
0 0 0
Heartmate (II, XVE) Heartware Jarvik 2000 Novacor Other
Q12: Which of the following LVAD pumps do you encounter
most often? (n=14)
49

50. 0
1 1
2
1
2
7
<1 in 50 1 in 40 1 in 30 1 in 20 1 in 15 1 in 10 1 in 5
Q13: In what fraction of your patients with LVADs does non-
surgical GI bleeding occur? (n=14)
50

51. 2
1
0 0
3
4 4
Never Rarely Occasionally Always
Q14: Please rank your involvement in the
management of non-surgical GI bleeding in LVAD
patients. (n=14)
0.00 1.00 2.00 3.00 4.00 5.00 6.00
Please rank your involvement in the management of non-surgical
GI bleeding in LVAD patients.
51

52. 0 2 4 6 8 10 12
None
Marginal
Moderate
Significant
Q15: What impact do you think the improvement of pump design
will have, if any, on the problem of GI bleeding? (n=14)
52

53. 0 2 4 6 8 10 12
None
Marginal
Moderate
Significant
Q16: To what extent do you perceive there to be a need to develop a novel therapy
specifically for the management of GI bleeding in LVAD patients with AVWS?
(n=14)
53

54. SURVEY RESPONSES
Cardiothoracic Surgeons
54

55. 0 0 0
1
0
4
2
Not familiar Moderately
familiar
Very Familiar
Q17: Familiarity with AVWS -
Cardiothoracic Surgery (n=7)
0.00 1.00 2.00 3.00 4.00 5.00 6.00 7.00
Please rank your familiarity with Acquired von Willebrand
Syndrome.
55

56. 1
2 2
0
1
0-20% 21-40% 41-60% 61-80% >80%
Q18: Approximately what percentage of your patients are
diagnosed with heart failure? (n=7)
56

57. 2
1 1 1
0
1
0-10% 11-20% 21-30% 31-40% 41-50% >50%
Q19: Approximately what percentage of your patients are
implanted with an LVAD? (n=6)
57

58. Bridge to Transplant
50%
Destination Therapy
50%
Q20: What is the primary therapeutic purpose for implantation of
LVADs? (n=6)
58

59. 0 0 0
1 1 1
0-1 year 1-2 years 2-3 years 3-4 years 4-5 years >5 years
Q21: How long on average is a patient on an LVAD as destination
therapy? (n=3)
59

60. Yes
67%
No
33%
Q22: Is the LVAD ever replaced when used as destination
therapy? (n=3)
60

61. Device malfunction
100%
Q23: What is the primary reason for the replacement? (n=2)
61

62. 3
2
0 0
1
Heartmate (II, XVE) Heartware Jarvik 2000 Novacor Other (please specify)
Q24: Which of the following LVAD pumps do you encounter
most often? (n=6)
62

63. 0 0 0
2
0
2 2
<1 in 50 1 in 40 1 in 30 1 in 20 1 in 15 1 in 10 1 in 5
Q25: In what fraction of your patients with LVADs does non-
surgical GI bleeding occur? (n=6)
63

64. 1
0 0 0
1
3
1
Never Rarely Occasionally Always
Q26: Please rank your involvement in the
management of non-surgical GI bleeding in
LVAD patients. (n=6)
0.00 1.00 2.00 3.00 4.00 5.00 6.00
Please rank your involvement in the management of non-
surgical GI bleeding in LVAD patients.
64

65. 0 1 1 2 2 3 3 4
None
Marginal
Moderate
Significant
Q27: What impact do you think the improvement of pump
design with have, if any, on the problem of GI bleeding? (n=6)
65

66. SURVEY RESPONSES
Hematologists
66

67. 0 0 0 0 0
1
2
Not familiar Moderately
familiar
Very Familiar
Q28: Familiarity with AVWS - Hematology
(n=3)
0.00 1.00 2.00 3.00 4.00 5.00 6.00 7.00 8.00
Please rank your familiarity with Acquired von Willebrand
Syndrome.
67

68. 1
2
0 0 0 0 0
<1 in 50 1 in 40 1 in 30 1 in 20 1 in 15 1 in 10 1 in 5
Q29: What fraction of your patients with GI bleeding have
LVADs? (n=3)
68

69. 0 0 0 0
3
0 0
Never Rarely Occasionally Always
Q30: Please rank your involvement in the
management of non-surgical GI bleeding in
LVAD patients. (n=3)
0.00 1.00 2.00 3.00 4.00 5.00 6.00
Please rank your involvement in the management of non-
surgical GI bleeding in LVAD patients.
69

70. 0
1
0
1
0 0
1
Desmopressin Plasma-derived VWF
concentrate
Recombinant VWF
concentrate
Antifibrinolytics Intravenous
Immunoglobulin
N/A Other (please
specify)
Q31: Which of the following treatments do you use most often
for patients with GI bleeding due to LVADs? (n=3)
Series1
70

71. 0 0 0
2
1
0 0
Not satisfied Neutral Very satisfied
Q32: Please rank your satisfaction with
current treatment options for AVWS. (n=3)
0.00 0.50 1.00 1.50 2.00 2.50 3.00 3.50 4.00 4.50 5.00
Please rank you satisfaction with current treatment
options for AVWS.
71

72. 0
2
0
1
None Marginal Moderate Significant
Q34: To what extent do you perceive there to be a need to develop a novel therapy
specifically for the management of GI bleeding in LVAD patients with AVWS? (n=3)
72

73. SURVEY RESPONSES
Gasteroenterolgists
73

74. 1
0 0
6
1 1
4
Not familiar Moderately
familiar
Very Familiar
Q35: Familiarity with AVWS - Gastroenterology
(n=13)
0.00 1.00 2.00 3.00 4.00 5.00 6.00
Please rank your familiarity with Acquired von Willebrand
Syndrome.
74

75. 1
3
0
1
0
4
0
<1 in 50 1 in 40 1 in 30 1 in 20 1 in 15 1 in 10 1 in 5
Q36: What fraction of your patients with GI bleeding have
LVADs? (n=9)
75

76. 0 0
1
0
4
3
1
Never Rarely Occasionally Always
Q37: Please rank your involvement in the
management of non-surgical GI bleeding in
LVAD patients. (n=9)
0.00 1.00 2.00 3.00 4.00 5.00 6.00
Please rank your involvement in the management of non-
surgical GI bleeding in LVAD patients.
76

77. 1 1
6
0
1
0
Cauterization Hemostatic clips Argon plasma
coagulation
Hemospray N/A Other
Q38: Which of the following treatments do you use most often
for patients with GI bleeding due to LVADs? (n=9)
77

78. 4
1
0
2 2
0 0
Not satisfied Neutral Very
satisfied
Q39: Please rank your satisfaction
with current treatment options for
AVWS. (n=9)
0.00 0.50 1.00 1.50 2.00 2.50 3.00
Please rank your satisfaction with current treatment
options for AVWS.
78

79. 0 1 2 3 4 5 6
None
Marginal
Moderate
Significant
Q41: To what extent do you perceive there to be a need to develop a
novel therapy specifically for the management of GI bleeding in LVAD
patients with AVWS? (n=9))
79

80. SURVEY RESPONSES
Compiled Results
80

81. 0
2
4
6
8
10
12
14
Not familiar Moderately
familiar
Very Familiar
Familiarity with AVWS by Specialty
Cardiology Cardiothoracic Surgery Hematology Gastroenterology
81

82. 0
2
4
6
8
10
12
Never Rarely Occasionally Always
Involvement in Non-surgical GI Bleeding
Management
Cardiology Cardiothoracic Surgery Hematology Gastroenterology
82

83. 0 2 4 6 8 10 12 14
None
Marginal
Moderate
Significant
Impact of Improvement in Pump Design
Cardiology Cardiothoracic Surgery
83

84. 0 2 4 6 8 10 12 14 16 18
None
Marginal
Moderate
Significant
Perception of a Need for a Novel Therapeutic
Cardiology Hematology Gastroenterology
84

85. 0
0.5
1
1.5
2
2.5
3
3.5
4
4.5
Not satisfied Neutral Very satisfied
Satisfaction with Current Treatment Options
Hematology Gastroenterology
85