Haemophilia A: Management Basics

00:52 Intro

02:35 Structure of Haemophilia A care

05:10 Key aspects of management (On-demand vs prophylaxis)

07:00 Prophylaxis: reduce death rates from ICH and reducing joint bleeds

- Primary prophylaxis: before the 2nd joint bleed

• Severe haemophilia
• Any child spontaneous ICH
• Moderate haemophilia A (1-3 IU/dL)

- Secondary prophylaxis:

• After the 2nd joint bleed
• Limit joint damage and maximize long term function
• ESPRIT trial

- Tertiary prophylaxis:

• If joint disease already established
• Slow progression, reduce pain and improve QOL
• SPINART study

13:10 Phases of treatment in Primary Prophylaxis

- Modify dose during according to needs at that stage of life

- By adulthood, 30% of severe patients can safely stop primary prophylaxis!

15:50 Prophylactic medications

- IV Recombinant FVIII, 1 IU/kg increases by 2 IU/dL (2%)

- Half life: 8-12 hrs 

- Primary Prophylaxis: (needs CVC)

• Aim trough level 1-3 IU/dL
• 25-40 IU/kg approx 3-4x per week
• Titrate clinically which is individual to the patient

- Extended half life...ratio of regular:half-life should be at least 1:1.3

- Cannot START them on this as can cause inhibitor

25:40 Efanesoctocog (EFA) only needs once weekly IV dosing

- Very extended half life

- XTEND 1 and XTEND-KIDS trials

27:52 Emicisimab: Bi-specific Ab, SC, half life 30 days

- Binds FIXa to FX thereby replacing FVIII

- Phenotypically makes patients have mild haemophilia A

- Used for ANY haemophilia with inhibitor OR severe haemophilia without an inhibitor

- HAVEN 3 study

- Breakthrough bleeds ?management challenges at home

- FIBA can cause MAHA (don't use together) 

- Thrombosis risk

- Reduces APTT and interferes with measuring FVIII and inhibitor

45:50 On-demand therapy (inhibitor dependent)

47:50 Joint bleeds

- Moderate bleed: Aim peak of 50- 60 IU/dL

- Severe bleed: Aim peak of 60-80 IU/dL

- Daily dosing

- Assess within 15 mins , treat within 30 mins

- TXA + analgesia, PT + PRICE 

52:20 Other bleeds

- Peak 80-100 IU/dL: Iliopsoas, ICH, GI bleeds, Neck/throat

- Deep cut: aim peak 50 IU/dL

- Keep at peak for 1-3 days then 50% decrease in peak level for the next week

54:10 Case-study: 24M, swollen knee, FVIII 0.3 IU/L

- Patients usually have an emergency plan

- Assume severe if no info.

01:00:15 Case study: 38M, Appendicectomy, FVIII 30 IU/dL

- Hari tricks David, David is tricked

- Give DDAVP because rFVIII can cause an inhibitor

01:03:05 DDAVP (Vasopressin)- IV/SC- 3-5 fold increase of FVIII

- 0.3micrograms/kg

- Releases a pool of FVIII from endothelium *lung*- tachyphylaxis

- Check protocol, side effects and contraindications

1:08:36 Management planning in elective surgery (MDT)

- TXA!

- Calculate dose of Recomb. FVIII

- Check levels at 15mins(pre-op), 4hrs (post-op), next morning

- May need VTEp

01:12:05 Comprehensive Care Centre Annual Review checklist- exam pearl

NB: Target joint- 3 or more bleeds into one joint in a 6 month

'Basics to Brilliance: Haematology Podcast' has been accredited for CPD credit by the Royal College of Pathologists UK.

Medical professionals and clinical scientists holding career-grade positions, who are registered with any of the Royal Colleges for CPD, will be eligible to earn 1 credit for every hour of learning.

Email: basicstobrilliancehaem@gmail.com

Insta: BasicstoBrilliance

X: @basics_2_brill

Send us your feedback!