WFH | Shared Decision Making

Travel
Employment/ education
Injection schedule
Medication

Physical activity
Treatment efficacy
Bleeding risk
Joint pain

Blood-bone infections (HCV/HIV)*
Affected family members and carriers

Daily concerns

Occasional concerns

Little to no concerns

Modified from Krumb & Hermans; “Living with a “hemophilia-free mind” - the new ambition of hemophilia care?”; 2021

Travel
Employment/ education
Injection schedule
Medication

Physical activity
Treatment efficacy
Bleeding risk
Joint pain

Blood-bone infections (HCV/HIV)*
Affected family members and carriers

Daily concerns

Occasional concerns

Little to no concerns

Modified from Krumb & Hermans; “Living with a “hemophilia-free mind” - the new ambition of hemophilia care?”; 2021

Travel
Employment/ education
Injection schedule
Medication

Physical activity
Treatment efficacy
Bleeding risk
Joint pain

Blood-bone infections (HCV/HIV)*
Affected family members and carriers

Daily concerns

Occasional concerns

Little to no concerns

Modified from Krumb & Hermans; “Living with a “hemophilia-free mind” - the new ambition of hemophilia care?”; 2021

Travel
Employment/ education
Injection schedule
Medication

Physical activity
Treatment efficacy
Bleeding risk
Joint pain

Blood-bone infections (HCV/HIV)*
Affected family members and carriers

Daily concerns

Occasional concerns

Little to no concerns

Modified from Krumb & Hermans; “Living with a “hemophilia-free mind” - the new ambition of hemophilia care?”; 2021

Travel
Employment/ education
Injection schedule
Medication

Physical activity
Treatment efficacy
Bleeding risk
Joint pain

Blood-bone infections (HCV/HIV)*
Affected family members and carriers

Daily concerns

Occasional concerns

Little to no concerns

Modified from Krumb & Hermans; “Living with a “hemophilia-free mind” - the new ambition of hemophilia care?”; 2021

Eligibility	Administration
Multiple products that are widely approved	All Ages	An injection into the vein 1-4 times per week		1-2 Times per year and as needed

Eligibility

Administration

Approval Status

Approved Population

Administration & Dosing

Location

Yearly Follow-up Schedule

Multiple products that
are widely approved

All Ages

An injection into the vein 1-4 times per week

1-2

Times per year and as needed

Efficacy		Safety Considerations		Potential Safety Risks
Treated Annual Bleed Rate	Factor Level	Years of Clinical Experience	Adverse Events	Hypersensitivity Reactions	Inhibitors	Thrombotic Events	Elevated Liver Enzymes
1-2	Factor levels increase immediately and decrease rapidly	40+	The most common adverse events are headache, diarrhea, cough, nausea, and joint pain.	Possible	3-5%	Possible

Quality of Life
Psychosocial Burden
Travel Employment/ education Injection schedule Medication Physical activity Treatment efficacy Bleeding risk Joint pain Blood-bone infections (HCV/HIV)* Affected family members and carriers Daily concerns Occasional concerns Little to no concerns Modified from Krumb & Hermans; “Living with a “hemophilia-free mind” - the new ambition of hemophilia care?”; 2021

Eligibility	Administration
Multiple products that are widely approved	All Ages	An injection into the vein 1-4 times per week		1-2 Times per year and as needed

Eligibility

Administration

Approval Status

Approved Population

Administration & Dosing

Location

Yearly Follow-up Schedule

Multiple products that
are widely approved

All Ages

An injection into the vein 1-4 times per week

1-2

Times per year and as needed

Efficacy		Safety Considerations		Potential Safety Risks
Treated Annual Bleed Rate	Factor Level	Years of Clinical Experience	Adverse Events	Hypersensitivity Reactions	Inhibitors	Thrombotic Events	Elevated Liver Enzymes
1-2	Factor levels increase immediately and decrease rapidly	40+	The most common adverse events are headache, diarrhea, cough, nausea, and joint pain.	Possible	3-5%	Possible

Quality of Life
Psychosocial Burden
Travel Employment/ education Injection schedule Medication Physical activity Treatment efficacy Bleeding risk Joint pain Blood-bone infections (HCV/HIV)* Affected family members and carriers Daily concerns Occasional concerns Little to no concerns Modified from Krumb & Hermans; “Living with a “hemophilia-free mind” - the new ambition of hemophilia care?”; 2021

Eligibility	Administration
1 product that is widely approved 1 product in phase 3	All Ages with and without inhibitors	An injection under the skin 1-4 times per month		1-2 Times per year and as needed

Eligibility

Administration

Approval Status

Approved Population

Administration & Dosing

Location

Yearly Follow-up Schedule

1 product that is widely approved

1 product in phase 3

All Ages with and without inhibitors

An injection under the skin 1-4 times per month

1-2

Times per year and as needed

Efficacy	Safety Considerations	Potential Safety Risks
<1.5	Factor levels will not change, but the effect is similar to having mild hemophilia	5+	The most common adverse reactions are injection site reactions, headache, and joint pain.			<1% Occurred in patients receiving at least one dose of aPCC

Efficacy

Safety Considerations

Potential Safety Risks

Treated Annual Bleed Rate

Factor Level

Years of Clinical Experience

Adverse Events

Hypersensitivity Reactions

Inhibitors

Thrombotic Events

Elevated Liver Enzymes

<1.5

Factor levels will not change, but the effect is similar to having mild hemophilia

5+

The most common adverse reactions are injection site reactions, headache, and joint pain.

<1%

Occurred in patients receiving at least one dose of aPCC

Quality of Life
Psychosocial Burden
Travel Employment/ education Injection schedule Medication Physical activity Treatment efficacy Bleeding risk Joint pain Blood-bone infections (HCV/HIV)* Affected family members and carriers Daily concerns Occasional concerns Little to no concerns Modified from Krumb & Hermans; “Living with a “hemophilia-free mind” - the new ambition of hemophilia care?”; 2021

Eligibility	Administration
1 product with limited approval 2 products in phase 3	Adults (12+) with inhibitors	An injection under the skin every 24 hours or 1x per month		2 Every 6 months and as needed

Eligibility

Administration

Approval Status

Approved Population

Administration & Dosing

Location

Yearly Follow-up Schedule

1 product with limited approval

2 products in phase 3

Adults (12+) with inhibitors

An injection under the skin every 24 hours or 1x per month

2

Every 6 months and as needed

Efficacy	Safety Considerations	Potential Safety Risks
<2	Factor levels will not change, but the effect is similar to having mild hemophilia	1+	The most common adverse events are injection site reactions, joint pain, upper respiratory tract infections, elevated prothrombin fragment 1.2, elevated fibrin D-dimer, headache, and fever.	< 3%		<1% Occurred In patients taking additional clotting agents

Efficacy

Safety Considerations

Potential Safety Risks

Treated Annual Bleed Rate

Factor Level

Years of Clinical Experience

Adverse Events

Hypersensitivity Reactions

Inhibitors

Thrombotic Events

Elevated Liver Enzymes

<2

Factor levels will not change, but the effect is similar to having mild hemophilia

1+

The most common adverse events are injection site reactions, joint pain, upper respiratory tract infections, elevated prothrombin fragment 1.2, elevated fibrin D-dimer, headache, and fever.

< 3%

<1%

Occurred In patients taking additional clotting agents

Quality of Life
Psychosocial Burden
Travel Employment/ education Injection schedule Medication Physical activity Treatment efficacy Bleeding risk Joint pain Blood-bone infections (HCV/HIV)* Affected family members and carriers Daily concerns Occasional concerns Little to no concerns Modified from Krumb & Hermans; “Living with a “hemophilia-free mind” - the new ambition of hemophilia care?”; 2021

Eligibility		Administration
Approval Status	Approved Population	Administration & Dosing	Location	Yearly Follow-up Schedule
1 Product with limited approval	Adults with hemophilia A	Gene therapy is a one-time treatment	Specialized Treatment Center	Frequent follow-ups for 2-3 years after treatment

Efficacy	Safety Considerations	Potential Safety Risks
<1	In most cases, factor levels increase to a mild to normal range. Some people do not respond, have a low response or their factor levels decrease with time.	1+	The most common side effects are infusion-related reactions, increased ALT, nausea, headache, above normal factor VIII(8) levels, tiredness, diarrhea, abdominal pain, and vomiting.	~ 5%		Potential for increased thromboembo lic risk	~80% Long-term effects on liver health are unknown

Efficacy

Safety Considerations

Potential Safety Risks

Treated Annual Bleed Rate

Factor Level

Years of Clinical Experience

Adverse Events

Hypersensitivity Reactions

Inhibitors

Thrombotic Events

Elevated Liver Enzymes

<1

In most cases, factor levels increase to a mild to normal range. Some people do not respond, have a low response or their factor levels decrease with time.

1+

The most common side effects are infusion-related reactions, increased ALT, nausea, headache, above normal factor VIII(8) levels, tiredness, diarrhea, abdominal pain, and vomiting.

~ 5%

Potential for increased thromboembo lic risk

~80%

Long-term effects on liver health are unknown

Quality of Life
Psychosocial Burden
Travel Employment/ education Injection schedule Medication Physical activity Treatment efficacy Bleeding risk Joint pain Blood-bone infections (HCV/HIV)* Affected family members and carriers Daily concerns Occasional concerns Little to no concerns Modified from Krumb & Hermans; “Living with a “hemophilia-free mind” - the new ambition of hemophilia care?”; 2021

Welcome

Introduction

Reflection

Education

Summary

COMPARE KEY ATTRIBUTES OF THE MAIN TREATMENT CLASSES FOR HEMOPHILIA B

1-2

1-2

1-2

1-2

1-2

1-2

0-2

0-2

1993

2024

Possible

20-30%

1-2

1-2

<1.5

<1.5

<1%*

2

2

<2

<2

< 3%

< 1%*

<1

<1

~ 5%

~80%

Welcome

Introduction

Reflection

Education

Summary

COMPARE KEY ATTRIBUTES OF THE MAIN TREATMENT CLASSES FOR HEMOPHILIA B

1-2

1-2

1-2

1-2

1-2

1-2

0-2

0-2

1993

2024

Possible

20-30%

1-2

1-2

<1.5

<1.5

<1%*

2

2

<2

<2

< 3%

< 1%*

<1

<1

~ 5%

~80%

SHL Factor Therapy: Quality of Life - Psychosocial Burden

EHL Factor Therapy: Quality of Life - Psychosocial Burden

Bispecific Antibody Therapy: Quality of Life - Psychosocial Burden

Hemostatic Rebalancing Therapy: Quality of Life - Psychosocial Burden

Gene Therapy: Quality of Life - Psychosocial Burden

SHL Factor Therapy

1-2

1-2

40+

Possible

3-5%

Possible

EHL Factor Therapy

1-2

1-2

40+

Possible

3-5%

Possible

Bispecific Antibody Therapy

1-2

<1.5

5+

<1%

Hemostatic Rebalancing Therapy

2

<2

1+

< 3%

<1%

Gene Therapy (Hem A)

<1

1+

~ 5%

~80%

SHL Factor Therapy: Efficacy - Factor Level

EHL Factor Therapy: Efficacy - Factor Level

SHL Factor Therapy: Efficacy - Factor Level

Bispecific Antibody Therapy: Efficacy - Factor Level

Hemostatic Rebalancing Therapy: Efficacy - Factor Level

Gene Therapy: Efficacy - Factor Level

User Instructions