CHS survey of people with hemophilia A and their caregivers

In June and July 2024, the Canadian Hemophilia Society conducted an online survey regarding the lived experience of people with hemophilia A and their caregivers. The purpose of the study was to reflect the patient voice in the CHS submissions to health technology assessment agencies. Canada’s Drug Agency (CDA, formerly CADTH) and Québec’s INESSS are currently evaluating efanesoctocog alfa (called Altuviiio in the U.S.), an ultra-extended half-life factor VIII.

The survey was online, publicized through CHS’ communications tools including social media.

Some of the key findings of the survey.

• One hundred and four (104) people completed the survey, including 33 with a history of inhibitors.
• Fifty-seven (57) reported that they were affected by severe hemophilia A, 14 by moderate hemophilia A, and 33 by mild hemophilia A.
• Fifty-nine (59) reported that the person with hemophilia was on a prophylactic treatment regimen, 30 were receiving on-demand treatment, and 15 reported receiving no treatment.
• Forty-three (43) reported receiving emicizumab (Hemlibra), 26 standard half-life factor VIII (FVIII), 9 extended half-life FVIII, 7 on desmopressin (DDAVP). Three (3) people were treated with tranexamic acid (Cyklokapron). One person had received gene therapy. One person was on a clinical trial for Mim8, a FVIII mimetic similar to emicizumab.

The questions asked in the survey reflected the information requested by CDA and INESSS.

These impacts were mentioned (number of similar responses in parentheses):

PHYSICAL SYMPTOMS
–  Pain, damage and loss of function from arthritic joints; [33 similar comments]
–  Pain from bleeds; [3]
–  Needing invasive medical procedures (infusions); [3]
–  Bleeding from activities; [2]
–  Bleeding episodes; [2]
–  Side effects of HIV treatment; [2]
–  Complications when undergoing surgery; [2]
–  Joint and muscle bleeds;
–  Difficulty sleeping because of pain;
–  Pain when walking, driving, writing, typing, and sleeping;
–  Hard to focus on work because of pain;
–  Complications when taking other medications;
–  Lengthy recovery times (3 months) after bleeding episodes;
–  15 bleeds per year despite prophylaxis;
–  Scarring at infusion sites;
–  Time required for daily infusions;
–  Managing heavy menstrual cycles;
–  Dealing with an inhibitor.

RESTRICTIONS
–  Restriction on physical activities; [12]
–  Restrictions on participation in sports; [10]
–  Loss of mobility, inability to walk very far; [9]
–  Difficulty performing everyday tasks; [4]
–  Restrictions in choice of jobs.

SOCIAL AND PSYCHOLOGICAL IMPACT
–  Having to be ultra cautious; [4]
–  Missing family activities;[3]
–  Social and psychological impacts; [3]
–  Need to constantly monitor young children for bleeds; [2]
–  Frequent periods of immobility leading to depression and anxiety;
–  Mental burden;
–  Having to rely on others when having a bleed;
–  Worry about bleeds;
–  Worry about admission to hospital with no hemophilia expertise;
–  No freedom from worry;
–  Dilemma of if and when to treat with factor;
–  Fear of not being able to control a bleed and get treatment in time;
–  Numerous hospitalizations;
–  Wait times for surgeries;
–  Need to be very mindful of risk of bleeding;
–  People not understanding what you suffer;
–  Uncertainty, stress.

TRAVEL
–  Worry and difficulty to access care when travelling; [3]
–  Fear of and avoidance of travel;
–  Difficulty of traveling with large quantities of factor.

FINANCIAL IMPACT
–  Days lost from work; [3]
–  Missed days from school (when a parent must stay with the child);
–  Needing to stop working.

LOGISTICS
–  The logistics of having factor products at all times.

 

FACTOR VIII PROPHYLAXIS

–  I use factor prophylactically (2-3 times per week); [11]

–  A little difficult for venous access; requires skillfulness and carefulness;

–  Adynovate (extended half-life FVIII) vials three times a week. Works perfectly.

FACTOR VIII ON-DEMAND

–  I manage my hemophilia with FVIII whenever a bleed occurs; [6]

–  Rest and factor infusions when injured;

–  Self-treatment, no difficulties;

–  Only get FVIII for colonoscopy exams and tooth extractions;

–  Very difficult veinous access.

HEMLIBRA (EMICIZUMAB) PROPHYLAXIS

–  Hemlibra injections once per week, no difficulties; (25 similar comments)

–  Hemlibra every 2 weeks; [6]

–  Lab pickup is inconvenient;

–  Emicizumab for prevention of internal bleeding, recombinant FVIII for treatment of breakout bleeds;

–  Physical therapy exercises to strengthen weak/damaged joints;

–  Hemlibra benefits: much more effective at preventing bleeds than FVIII;

–  Hemlibra: I wish I had it as a child, as I feel I would probably be devoid of all the constant pain I have now. It has allowed me freedom of travel;

–  With my current treatment of emicizumab, I am able to perform many more activities without the fear of bleeds, soreness, or pain;

–  I am using Hemlibra and I find it to be an awesome medication. I wish it was available when I was younger. Have not had a bleed yet while using it, for almost 2 years now;

–  We treat with Hemlibra once a week and it has been life-changing. There have been zero bleeds since my son started. He is independent in managing his treatments and the supply volume and accessory supplies have reduced drastically. It is much easier to travel and he can travel independently. Since we started using Hemlibra I haven’t had to take time off work to help him manage a bleed, take him to physiotherapy appointments or visit an ER. We still go to clinic twice a year but we access the health care system 90% less than previously.

GENE THERAPY

–  Gene therapy has been life-altering. The pain he experiences has decreased and, given that he now has over 20% FVIII levels, he can now actually heal wounds, cuts, and scrapes. More importantly, he doesn’t end up with traumatic injury and a bleed every time he steps off the sidewalk wrong or bangs his knee. He hasn’t needed to be treated with intravenous FVIII in 15 months.

DESMOPRESSIN, ANTIFIBRINOLYTICS

–  My biggest issues with treatment have been having to insist to doctors that I do need DDAVP and tranexamic acid. Several times health care providers have brushed my concerns off because my hemophilia is “mild” and that has led to severe consequences health wise;

–  DDAVP by myself, by injection in my stomach fat. Some side effects I’ve experienced are: palpitations, redness of the skin and high fever;

–  I have been tested with desmopressin and had 100% success;

–  When I am injured, I’m instructed to take tranexamic acid tablets to help stop the bleeding. These tablets cost a lot of money.

CARE

–  For the most part, I am able to manage treatments on my own. I get great support from the hemophilia clinic;

–  Set up appointments for prophylaxis treatment before any invasive procedure;

–  If I have concerns, the staff at the clinic are always helpful;

–  I have had hospital stays in last few years. I waited 8 hours in our local emergency with a tongue bleed before seeing a doctor;

–  Continual difficulty in convincing major city hospital emergency medical teams of the need for FVIII treatment or DDAVP during a major bleed;

–  If I have a major bruise, I have to go to the ER to receive DDAVP intravenously. This typically takes 6 or more hours;

–  Any treatment is subject to oversight by the bleeding disorders clinic. They have been awesome coordinating info to staff involved in any procedures;

–  The nearest hemophilia treatment centres are 2 and 4 hours away, which means any time I need treatment I have to present to our local emergency room. The staff here have very little training and experience in treating hemophilia, so there is no consistency in diagnosis or treatment;

–  I feel very fortunate to live in a major city within the province and have firsthand access to physiotherapy and treatment. I have regular follow-ups with the team from the hemophilia centre, including hematologists and physiotherapists;

–  We poke (infuse) twice per week at home. It is difficult if us parents go away, the treatment facility is 40 min away if someone has to take him. We no longer have access to a hematologist on call, so any traumas or issues that need to be seen by the doctor result in long emergency wait times.

INHIBITORS

– To fight the inhibitor, our son’s port was put back in April 2023, it was originally removed Oct 22. Our son loves his port. He hates Hemlibra pokes and has said he would rather poke his own vein than get the Hemlibra poke.

COST, LOGISTICS

–  Living in the interior, blood product is not readily on hand. They have to be ordered from the hemophilia clinic which is usually a day or two to receive.

 

Very satisfied
51

Quite satisfied
38

Not very satisfied
11

Not satisfied at all
4

Total of 104

 

–  Not difficult at all; [60 similar responses]

–  Sometimes difficult (venous access); [8]

–  Very difficult. Long waits in the ER. Have experienced confusion in ER around type and method of treatment when required. I need to go to the HTC or the closest ER of any hospital to get an IV installed; [6]

–  Time consuming; [2]

–  Uncomfortable but manageable;

–  Much simpler with Hemlibra. Instead of doing IVs at home or having to drive into the clinic (hour away);

–  I find that the subcutaneous injection every 2 weeks is quite painful, and the injection site is uncomfortable for several hours after the injection. But it’s better than doing an IV every 2-3 days;

–  We poke our son 3x a week with factor and 2x a month with Hemlibra. It’s a lot for a little guy. Factor pokes are way easier then Hemlibra pokes.

 

Very effective. [50 similar comments]

Very effective but for short term. [2]

Quite effective. [21]

Not very effective. [6]

Effective to prevent bleeding but not to stop.

 

–  Ability to engage in everyday activities (walking, errands, work …) like everybody else; [15 similar comments]

–  Better preventative treatments, higher level of protection; [9]

–   Less pain, better pain management; [8]

–   Reduced frequency of treatment (longer half-life); [8]

–   Easier, less burdensome, less painful administration; [7]

–   Treatment that does not need refrigeration (Hemlibra); [5]

–   Capacity to do riskier activities (e.g. sports); [4]

–   A cure; [4]

–   A once-a-year treatment; [4]

–   An end to IV administration; [3]

–   All of the above; [2]

–   A constant FVIII level of 25%-75% to eliminate risk of bleeding; [2]

–   Longer acting Hemlibra; [2]

–   More available physiotherapy and fitness training; [2]

–   Wider access to DDAVP and tranexamic acid; [2]

–   Fewer restrictions on activities of daily living;

–   Access to products through pharmacies;

–   Gene therapy;

–   No more daily IV treatments;

–   Less time needed to treat;

–   Better care at ERs;

–   Home treatment with a DDAVP inhaler;

–   Less risk of thrombosis from bypassing agent;

–   Preventing long-term joint damage and arthritis;

–   Comprehensive, user-friendly webpage, where patients can access physiotherapy and preventive care videos and resources for daily follow-up;

–   Fewer joint bleeds;

–   Fewer trips to the ER;

–   More compact packaging for medications;

–   Weekly or monthly factor VIII administration;

–   A pill rather than an injection;

–  Treatments under the skin that last a long time.

 

The CHS would like to thank all those who took the time to complete the survey. The information provided is critical to our efforts to advocate for access to novel coagulation therapies.

The full CHS submissions regarding efanesoctocog alfa will appear on the CDA and INESSS websites in the coming months. To access them, please go to:

www.cda-amc.ca and choose “Find Reports”

www.inesss.qc.ca and choose “Produits du système du sang”