The journey toward improving
Quality of life for haemophilia patients

Haemophilia affects approximately 400,000 people worldwide

Approximately 400,000 people worldwide are living with the burden of haemophilia1
Of these, 80%-85% of people suffer with haemophilia A and require replacement Factor VIII to treat or prevent bleeding1
Approximately 15%-20% will develop antibodies (inhibitors) to the factor therapy used to treat or prevent bleeding episodes — one of the most serious complications of haemophilia2
The remainder suffer with haemophilia B and require replacement Factor IX to treat or prevent bleeding1

EVERY BLEED MATTERS

Journey for every haemophilia patient should be toward zero preventable bleeds

Some people with haemophilia are bleeding unnecessarily

  • In some cases, bleeds are treated only when they occur with on-demand therapy rather than prevented with prophylaxis1
Treatment with
on-demand therapy
Bleed prevention
with prophylaxis
Even a few bleeds into the same joint may cause irreversible damage
  • Even a few haemarthroses into the same joint — an annual joint bleeding rate (AJBR) of 2-3 — may cause irreversible, progressive structural changes in the joint3
  • An MRI evaluation of joints in 15 patients found an association between bleeds and arthropathy. Joints with 3 bleeds showed initial signs of haemophilic arthropathy4
Arthropathy is a frequent and serious complication of repeated joint bleeding, resulting in pain, deformity, and disability5
The degree of joint damage is usually associated with the number of joint bleeds
  • Among other factors, the number of joint bleeds was positively associated with increased limitation in the range of joint movement in patients with severe haemophilia6
  • Conversely, a low annual joint bleeding rate has been shown to be associated with good joint health7,8
  • The degree of disability a person with severe haemophilia develops is usually associated with the number of joint bleeds that the individual experiences7
Studies show that patients with a lower AJBR had a better HRQoL.3
Joint bleeding can have a negative impact on HRQoL

Children with an annual bleed rate <3 had significantly better HRQoL scores for some parameters than those who bled more frequently9

A study of adolescent patients in Egypt found a significant correlation between low musculoskeletal function score and high depression score10

Each acute bleeding event can impact mental and physical HRQoL, and frequent bleeding events could have a large cumulative effect on HRQoL11

Joint bleeding can have a negative impact on school and work attendance and academic achievement

Patients with severe haemophilia A receiving prophylaxis had significantly fewer days lost from work or school compared with those receiving on-demand therapy12,13

Children with severe haemophilia who had more school absences had lower scores in mathematics, reading, and total achievement14

Prophylaxis for all

Some patients do not receive adequate prophylaxis, leaving them at risk for bleeds

Routine prophylaxis has the potential to reduce bleeds in patients of all ages15-17

World Federation of Hemophilia
  • Since prophylaxis can prevent bleeding and joint destruction, prophylaxis should be the goal of therapy in order to preserve normal musculoskeletal function1
  • Prophylaxis is cost-effective in the long term, as it eliminates the high cost associated with management of damaged joints and improves HRQoL1
Long-term prophylaxis given from an early age can achieve7,15,16,18

LOW FREQUENCY OF JOINT BLEEDS7,15,19

GOOD JOINT HEALTH15,18

ZERO JOINT SCORES IN SOME PATIENTS WITH SEVERE HAEMOPHILIA7,15,19

Prophylaxis is effective at reducing bleeds15-17,20,21 and at reducing15 or, if started at an early age, preventing joint damage15

People with haemophilia who received routine prophylaxis reported to have a better quality of life than those who receive on-demand therapy17,20,22

Routine prophylaxis has been shown effective at reducing bleeds and preventing joint damage

Graph calculated from Noone et al., 2014

  • Survey to examine the long-term effects of prophylaxis and the continuing benefit of treatment into adulthood17
  • Self-assessed health-related data collected from 124 men from 6 countries17
Adults and children with haemophilia A receiving routine prophylaxis have been shown to achieve significant reduction in bleeding episodes and better joint outcomes compared with those receiving on-demand treatment15-18

Personalised for one

Every patient is unique13

There are many factors to consider when personalising prophylaxis
A range of factors affects response to therapy

There are many factors to consider when individualising prophylaxis13:

The physiology and lifestyle of individuals change over time and has an impact on their needs for Factor VIII23,24

Physical activity26
The level of patient activity can impact the risk of bleeds

One study observed that bleeding incidence reduced by 2% for every 1% increase in clotting factor level2

PK response27
Considerable variation between individuals

Some patients may not receive enough factor to maintain adequate trough levels

Phenotype7,13,28,29
Factor trough level is an important determinant of efficacy of prophylaxis

The target 1% trough level may not be the threshold above which all bleeds are prevented

There is considerable variation in PK between individuals13

In the late 1950s, target Factor VIII trough levels for prophylaxis were set at >1%.27

Recent data suggest that 1% may not be the threshold above which all bleeds are prevented and below which bleeding inevitably occurs for all people with haemophilia A.13

Some physicians recommend the prophylaxis be personalised
A personalised approach is needed because every patient is unique13
  • Early prophylaxis — the evidence-based treatment of choice in children with severe haemophilia to prevent joint damage13,32
  • Prophylactic therapy — can prevent bleeds in some people with haemophilia, creating the possibility for them to pursue the lives they choose13,23,25

References:
1. Srivastava A, Brewer AK, Mauser-Bunschoten EP, et al. Guidelines for the management of hemophilia. Haemophilia. 2013;19(1):e1-47. 2. Centers for Disease Control and Prevention (CDC). Hemophilia: inhibitors. Available at: http://www.cdc.gov/ncbddd/hemophilia/inhibitors.html. 3. Gringeri A, Ewenstein B, Reininger A. The burden of bleeding in haemophilia: is one bleed too many? Haemophilia. 2014;20:1-5. 4. Funk MB, Schmidt H, Becker S, et al. Modified magnetic resonance imaging score compared with orthopaedic and radiological scores for the evaluation of haemophilic arthropathy. Haemophilia. 2002;8:98-103. 5. Valentino LA. Blood-induced joint disease: the pathophysiology of hemophilic arthropathy. J Thromb Haemost. 2010;8:1895-1902. 6. Soucie JM, Cianfrini C, Janco RL, et al. Joint range-of-motion limitations among young males with hemophilia: prevalence and risk factors. Blood. 2004;103:2467-2473. 7. Nilsson IM, Berntorp E, Löfqvist T, Pettersson H. Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B. J Intern Med. 1992;232:25-32. 8. Van den Berg HM, Fischer K, Mauser-Bunschoten EP, et al. Long-term outcome of individualized prophylactic treatment of children with severe haemophilia. Br J Haematol. 2001;112:561-565. 9. Gringeri A, von Mackensen S, Auerswald G, et al. Health status and health-related quality of life of children with haemophilia from six West European countries. Haemophilia. 2004;10 (Suppl 1):26-33. 10. Hassan TH, Badr MA, Fattah NR, Badawy SM. Assessment of musculoskeletal function and mood in haemophilia A adolescents: a cross-sectional study. Haemophilia. 2011;17:683-688. 11. Poon JL, Doctor JN, Nichol MB. Longitudinal changes in health-related quality of life for chronic diseases: an example in Hemophilia A. J Gen Intern Med. 2014;29 Suppl 3:S760-766. 12. Aledort LM, Haschmeyer RH, Pettersson H and the Orthopaedic Outcome Study Group. A longitudinal study of orthopaedic outcomes for severe factor-VIII-deficient haemophiliacs. The Orthopaedic Outcome Study Group. J Intern Med. 1994;236:391-399. 13. Valentino LA. Considerations in individualizing prophylaxis in patients with haemophilia A. Haemophilia. 2014;1-9. 14. Shapiro AD, Donfield SM, Lynn HS, et al and the Academic Achievement in Children with Hemophilia Study Group. Defining the impact of hemophilia: the Academic Achievement in Children with Hemophilia Study. Pediatrics. 2001;108(6):E105. 15. Manco-Johnson MJ, Abshire TC, Shapiro AD, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med. 2007;357(6):535-544. 16. Manco-Johnson MJ, Kempton CL, Reding MT, et al. Randomized, controlled, parallel-group trial of routine prophylaxis vs. on-demand treatment with sucrose-formulated recombinant factor VIII in adults with severe hemophilia A (SPINART). J Thromb Haemost. 2013;11:1119-1127. 17. Noone D, O'Mahony B, Van Dijk K, Prihodova L. A survey of the outcome of prophylaxis, on-demand treatment or combined treatment in 18-35 year old men with severe haemophilia in six countries. Haemophilia. 2013;19:44-50. 18. Liesner RJ, Khair K, Hann IM. The impact of prophylactic treatment on children with severe haemophilia. Br J Haematol. 1996;92:973-978. 19. Gringeri A, Lundin B, von Mackensen S, Mantovani L, Mannucci PM and the ESPRIT study group. A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study). J Thromb Haemost. 2011;9:700-710. 20. Valentino LA, Mamonov V, Hellmann A, et al. A randomized comparison of two prophylaxis regimens and a paired comparison of on-demand and prophylaxis treatments in hemophilia A management [published correction appears in J Thromb Haemost. 2012;10(6):1204]. J Thromb Haemost. 2012;10(3):359-367. 21. Valentino LA, Reyes CM, Ewenstein B, et al. ADVATE prophylaxis: post hoc analysis of joint bleeding episodes. Haemophilia. 2014:20:630-638. 22. Royal S, Schramm W, Berntorp E, et al. Quality-of-life differences between prophylactic and on-demand factor replacement therapy in European haemophilia patients. Haemophilia. 2002;8:44-50. 23. Collins PW. Personalized prophylaxis. Haemophilia. 2012;18 (Suppl 4):131-135. 24. Kearns GL, Abdel-Rahman SM, Alander SW, et al. Developmental pharmacology--drug disposition, action, and therapy in infants and children. N Engl J Med. 2003;349:1157-1167. 25. Berntorp E, Spotts G, Patrone L, Ewenstein BM. Advancing personalized care in hemophilia A: ten years' experience with an advanced category antihemophilic factor prepared using a plasma/albumin-free method. Biologics. 2014 Apr 5;8:115-127. 26. Broderick CR, Herbert RD, Latimer J, et al. Association between physical activity and risk of bleeding in children with hemophilia. JAMA. 2012;308(14):1452-1459. 27. Collins PW, Björkman S, Fischer K, et al. Factor VIII requirement to maintain a target plasma level in the prophylactic treatment of severe hemophilia A: influences of variance in pharmacokinetics and treatment regimens. J Thromb Haemost. 2010;8:269-275. 28. Löfqvist T, Nilsson IM, Berntorp E, Pettersson H. Haemophilia prophylaxis in young patients-a long-term follow-up. J Intern Med. 1997;241:395-400. 29. White GC, et al. Recommendation of the scientific subcommittee on factor VIII and factor IX of the scientific and standardization committee of the International Society on Thrombosis and Haemostasis. Thromb Haemost. 2001;85:560. 30. Kraft J, Blanchette V, Babyn P, et al. Magnetic resonance imaging and joint outcomes in boys with severe hemophilia A treated with tailored primary prophylaxis in Canada. J Thromb Haemost. 2012;10:2494-2502. 31. Hilliard P, Zourikian N, Blanchette V, et al. Musculoskeletal health of subjects with hemophilia A treated with tailored prophylaxis: Canadian Hemophilia Primary Prophylaxis (CHPS) Study. J Thromb Haemost. 2013;11:460-466. 32. Coppola A, Tagliaferri A, Di Capua M, Franchini M. Prophylaxis in Children with Hemophilia: Evidence-Based Achievements, Old and New Challenges. Semin Thromb Hemost. 2012;38:79-94.

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