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Hemophilia Guidelines for All PowerPoint Presentation

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Description : Check out this medical PowerPoint presentation titled "Hemophilia Guidelines for All”.

This medical PowerPoint presentation is about hemophilia, a genetic bleeding disorder in which the blood does not clot normally. There are two main types of hemophilia: hemophilia A, which is caused by a deficiency of clotting factor VIII, and hemophilia B, which is caused by a deficiency of clotting factor IX. Hemophilia guidelines are designed to provide healthcare professionals with recommendations for the diagnosis, treatment, and management of hemophilia.

This presentation gives some general guidelines for the diagnosis and management of hemophilia.

Tags : Hemophilia, genetic bleeding disorder, hemophilia A, hemophilia B, Hematology, Hematology PowerPoint presentation

Published on : May 29, 2023 | Views : 1911 | Downloads : 9

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Slide 1-

Hemophilia Guidelines for All A new ambition of the World Federation of Hemophilia
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WFH Guidelines for the Management of Hemophilia All recommendations are consensus based. Srivastava A et al. Haemophilia. 2020;26(Suppl 6):1–158.
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Chapter 6: Prophylaxis in Hemophilia Manuel Carcao MD, MSc Staff Haematologist Co-Director, Hemophilia Clinic Hospital for Sick Children Professor of Paediatrics University of Toronto Toronto, Ontario, Canada
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Disclosures: Manuel Carcao Research Support from: Bayer, Bioverativ/Sonofi, CSL-Behring, Novo Nordisk, Octapharma, Pfizer and Shire/Takeda Honoraria for speaking/participating in advisory boards from: Bayer, Bioverativ/Sonofi, Biotest, CSL Behring, Grifols, LFB, Novo Nordisk, Octapharma, Pfizer, Roche and Shire/Takeda
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Authors Manuel Carcao H. Marijke van den Berg Emna Gouider Kate Khair Manuel A. Baarslag (PWH) Lisa Bagley (PPWH) Francisco de Paula Careta (PWH) Rolf C. R. Ljung Margaret V. Ragni Elena Santagostino Glenn F. Pierce Alok Srivastava PWH, person with hemophilia, PPWH, parent of person with hemophilia.
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Hemophilia = life-long severe bleeding disorder[without regular factor infusions=prophylaxis]
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Hemophilia = life-long severe bleeding disorder[without regular factor infusions=prophylaxis] Soft tissue Muscle Joint Intracranial
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ppt slide no 8 content not found
Slide 9-

But with prophylaxis They don’t have to experience these things!
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Overall bleeds pediatric Overall bleeds pediatric Joint bleeds adult+pediatric Intermediate dose Intermediate dose Full dose Prophylaxis with Standard T1/2 factor huge in bleeding
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Intermediate dose Intermediate dose Full dose Overall bleeds pediatric Overall bleeds pediatric Joint bleeds adult+pediatric -93% -76% -82% Prophylaxis with Standard T1/2 factor huge in bleeding
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Prophylaxis with Standard T1/2 factor huge improvements in quality of life *Photographs used are with patient consent or are sourced from stock images
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Prophylaxis is standard of care xx Everywhere WFH Guidelines 2020 (Prophylaxis)
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“For patients with severe .. haemophilia A or B, especially children, the WFH recommends regular long-term prophylaxis as the standard of care …. When prophylaxis is not feasible, episodic (on demand) therapy is essential treatment for acute hemorrhages, but will NOT PREVENT long-term joint damage.” Prophylaxis Standard of care-everywhere Chapter 6: Prophylaxis in Hemophilia in Srivastava A et al. WFH Guidelines for the Management of Hemophilia, 3rd ed, Haemophilia, 2020. WFH Guidelines 2020 (Prophylaxis)
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“In countries with significant healthcare constraints, the WFH still advocates for the use of prophylaxis over episodic (on demand) therapy but recognizes that less intensive prophylaxis may be used.” Prophylaxis Standard of care-everywhere WFH Guidelines 2020 (Prophylaxis) Chapter 6: Prophylaxis in Hemophilia in Srivastava A et al. WFH Guidelines for the Management of Hemophilia, 3rd ed, Haemophilia, 2020.
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… The WFH recommends early initiation of prophylaxis … prior to the onset of joint disease and ideally before age 3, … (i.e. primary prophylaxis) Prophylaxis Start early WFH Guidelines 2020 (Prophylaxis) Chapter 6: Prophylaxis in Hemophilia in Srivastava A et al. WFH Guidelines for the Management of Hemophilia, 3rd ed, Haemophilia, 2020.
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“For patients with haemophilia A or B with a severe phenotype (may include patients with moderate haemophilia), the WFH strongly recommends that such patients be on prophylaxis sufficient to prevent bleeds at all times” Prophylaxis Aim to prevent all bleeds WFH Guidelines 2020 (Prophylaxis) Chapter 6: Prophylaxis in Hemophilia in Srivastava A et al. WFH Guidelines for the Management of Hemophilia, 3rd ed, Haemophilia, 2020.
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Overall bleeds pediatric Overall bleeds pediatric Joint bleeds adult+pediatric Prophylaxis with Standard T1/2 factor huge in bleeding Intermediate dose Intermediate dose Full dose -76% -82% -93%
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Overall bleeds pediatric Overall bleeds pediatric Joint bleeds adult+pediatric Prophylaxis with Standard T1/2 factor huge in bleeding Intermediate dose Intermediate dose Full dose
Slide 20-

Overall bleeds pediatric Overall bleeds pediatric Joint bleeds adult+pediatric Prophylaxis with Standard T1/2 factor huge in bleeding Intermediate dose Intermediate dose Full dose Not good enough!
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Overall bleeds pediatric Overall bleeds pediatric Joint bleeds adult+pediatric Prophylaxis with Standard T1/2 factor huge in bleeding Intermediate dose Intermediate dose Full dose Not good enough! Okay but
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Full dose Prophylaxis with SHL-FVIII (182 needles)
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Even 1 needle is hard enough! From venopunctures
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Full dose Prophylaxis with SHL-FVIII – this is very hard (182 needles)
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Missed doses Full dose Prophylaxis with SHL-FVIII – this is very hard
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Full dose Prophylaxis with SHL-FVIII – this is very hard Missed doses
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Missed doses Full dose Prophylaxis with SHL-FVIII – this is very hard Patients need products that allow for less infusions
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(182 needles) What do patients achieve with this? Full dose Prophylaxis with SHL-FVIII – this is very hard
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(182 needles) Factor VIII Trough levels = 1-3% What do patients achieve with this? Full dose Prophylaxis with SHL-FVIII – this is very hard
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Trough Level = 1-3% # of joint bleeds vs baseline FVIII Den Uijl I et al. Hemophilia 2011.
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Trough Level = 1-3% # of joint bleeds vs baseline FVIII Den Uijl I et al. Hemophilia 2011. Patients need higher trough levels
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Prophylaxis: Patients need more IV, intravenous. Or a combination 1) Less infusions 2) Higher factor trough levels 3) Non-IV administered
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Prophylaxis we can now do (and should do) better Chapter 6: Prophylaxis in Hemophilia in Srivastava A et al. WFH Guidelines for the Management of Hemophilia, 3rd ed, Haemophilia, 2020. WFH Guidelines 2020 (Prophylaxis)
Slide 34-

Prophylaxis we can now do (and should do) better Chapter 6: Prophylaxis in Hemophilia in Srivastava A et al. WFH Guidelines for the Management of Hemophilia, 3rd ed, Haemophilia, 2020. WFH Guidelines 2020 (Prophylaxis)
Slide 35-

Prophylaxis We can now do better “Now recognizing that with a 1% trough level, patients remain at risk of bleeding, most clinicians would prefer to target higher trough levels (>3%-5%, or higher).” WFH Guidelines 2020 (Prophylaxis) Chapter 6: Prophylaxis in Hemophilia in Srivastava A et al. WFH Guidelines for the Management of Hemophilia, 3rd ed, Haemophilia, 2020.
Slide 36-

Prophylaxis We can now do better Allow us to achieve better protection than simply a 1% factor level
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Or a combination 1) Less infusions 2) Higher factor trough levels 3) Non-IV administered Benefits of Extended half-life (EHL) CFC IV, intravenous.
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EHL FVIII Current FVIII For the average patient No  in bleeding
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EHL FIX Current FIX For the average patient LESS BLEEDING
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Emicizumab (SQ) prophylaxis >90%  in bleeds (vs on demand) 52 times/yr
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Emicizumab (SQ) prophylaxis >90%  in bleeds (vs on demand) 26 times/yr
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Benefits of Emicizumab Or a combination 1) Less infusions 2) Higher factor trough levels 3) Non-IV administered Sort of! IV, intravenous.
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For patients with severe phenotype hemophilia A WITHOUT INHIBITORS, prophylaxis with emicizumab will prevent hemarthrosis, spontaneous, and breakthrough bleeding. Remark: … long term data on patient outcomes with such an approach … be obtained. Prophylaxis No longer just factor WFH Guidelines 2020 (Prophylaxis) Chapter 6: Prophylaxis in Hemophilia in Srivastava A et al. WFH Guidelines for the Management of Hemophilia, 3rd ed, Haemophilia, 2020.
Slide 44-

What is prophylaxis?
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What is prophylaxis? Blanchette VS, et al. Definitions in hemophilia: communication from the SSC of the ISTH. J Thromb Haemost. 2014;12(11):1935-1939.
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What is prophylaxis? Blanchette VS, et al. Definitions in hemophilia: communication from the SSC of the ISTH. J Thromb Haemost. 2014;12(11):1935-1939. Chapter 6: Prophylaxis in Hemophilia in Srivastava A et al. WFH Guidelines for the Management of Hemophilia, 3rd ed, Haemophilia, 2020.
Slide 47-

Standard of care – everywhere We can now do (and should do) better No longer just factor Key recommendations: prophylaxis WFH Guidelines 2020 (Prophylaxis)
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Can look forward to a better life! Key recommendations: prophylaxis WFH Guidelines 2020 (Prophylaxis)
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Thank you to the Hemophilia Alliance for their support in developing this presentation