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Treatment Products in Hemophilia. Nairobi, Kenya. June 24, 2013. Objectives. Identify historical approaches used to treat hemophilia Describe treatment products currently available for use in hemophilia Distinguish classes of factor concentrates

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treatment products in hemophilia
Treatment Products in Hemophilia

Nairobi, Kenya

June 24, 2013


Identify historical approaches used to treat hemophilia

Describe treatment products currently available for use in hemophilia

Distinguish classes of factor concentrates

Discuss donor screening and viral inactivation

List adjuvant therapies for treatment of hemophilia

Explore future therapies

Additional text example

historical treatment of hemophilia
Historical treatment of hemophilia

Injections of adrenaline

Ingestion of such compounds as1:

  • Strychnine − Turpentine
  • Lead − Female hormone
  • Bromide extracts of egg − Peanut flour2


Topical snake venom3

First blood transfusion in 1840 by Dr. Samuel Lane4

  • 1Rosendaal FR, Smit C, BriëtE.Ann Hematol. 1991; 62:5-15.
  • 2Mainwaring D, KeldonS.E. Lancet. 1964; 19:647.
  • 3MacFarlane RG, Barnett, B. Lancet. 1934; ii: 985–987.
  • 4Lane, S. Lancet. 1840; i: 185-188.
cryoprecipitate discovered
Cryoprecipitate Discovered


Discovery of


Judith Graham Pool, MD

File photo courtesy of HANDI, NHF

Pool JG, Shannon AE. N Engl J Med.1965:273:1443-1447.

factor concentrates soon appear
Factor Concentrates Soon Appear

1966: Hyland announces commercial availability of FVIII concentrates

1969: FIX concentrate licensed1

Allowed for greater independence

1. Hoag MS, et al. N EnglJ Med.1969;280(11):581-6

the price of independence
The Price of Independence

1983: Suspicion that HIV threatened the worldwide blood supply

1983:Hemofil-T, first heat-treated FVIII concentrate in the US

1984: Montagnier1 and Gallo2 discover HTLV-3 (HIV)

1984:Efficacy of heat treatment for viral inactivation demonstrated

1984:Recall of blood products initiated

1985: ELISA test used to detect HIV antibodies among blood donors

1985:Safety net:

  • Donor deferral
  • Viral inactivation methods
  • Antibody and NAT testing

1. Barre-Sinoussi F, et al. Science 1983; 220(4599):868-71.

2. Gallo RC, et al. Science 1984; 4;224(4648):500-3.

clotting factor concentrates and other plasma products

Factor replacement therapy

  • Fresh frozen plasma (FFP)
  • Cryoprecipitate
  • Plasma-derived concentrates
    • Recombinant concentrates
wfh recommendation
WFH recommendation

The WFH strongly recommends the use of viral-inactivated plasma-derived or recombinant concentrates in preference to cryoprecipitate or fresh frozen plasma for the treatment of hemophilia

Guidelines for the Management of Hemophilia, 2nd edition, WFH 2012

choice of treatment product
Choice of treatment product

Choice of treatment product is an important decision

Infusion products should be chosen with provider, NMO, and patient/family input

Important issues regarding infusion products:

  • Efficacy
  • Safety
  • Purity
  • Cost
plasma derived products purity
Plasma-derived products: purity

Concentrates on the market vary widely in their purity

High purity

  • Just the clotting factor in the vial exclusive of added stabilizers
  • Activity/protein ratio is very high

Intermediate purity

  • More than just the clotting factor in the vial
  • Activity/protein ratio mid-range

Concentrates of lower purity may give rise to allergic reactions

FVIII concentrates may contain variable amounts of VWF

For treatment of FIX deficiency, a product containing only FIX is more appropriate than PCCs

plasma derived products safety
Plasma-derived products: safety

Viral inactivation is the biggest contributor to safety of treatment products

Heat treatment: effective against enveloped and non-enveloped viruses including (HIV, HAV, HBV and HBC)

Solvent/detergent treatment: effective against non-enveloped viruses such as HIV, HBV, HCV but not HAV)

Some viruses resistant to both types of process (e.g. parvovirus B19)

Products undergo one or two viral inactivation steps; if one, preferably one that is effective against viruses with and without lipid envelopes

recombinant products
Recombinant Products

All recombinant products are high purity

Not made from human plasma

  • 1st generation: Added albumin as stabilizer, human/animal protein exposure during production
  • 2nd generation: Albumin removed as stabilizer, human/animal protein exposure during production
  • 3rd generation: No added human or animal protein during production or in final formulation
fresh frozen plasma
Fresh frozen plasma

Contains all the coagulation factors

Due to concerns about safety and quality of FFP, it is not recommended for treatment of hemophilia, if avoidable

Possible to apply some forms of viral inactivation to packs of FFP but may have impact on coagulation factors

Large volumes of plasma must be transfused, which can lead to a complication called circulatory overload


Prepared by slow thawing of FFP

Contains significant quantities of FVIII, VWF, fibrinogen and FXIII but no FIX

Less safe from viral contamination than factor concentrates; harder to store and administer

Virally-inactivated cryo has been described (S/D cryo)

Preferable to FFP for the treatment of hemophilia A

Cryo cannot be used for treatment of hemophilia B

plasma derived products
plasma-derived products

Blood Donation in South Africa

Whole Blood Donation from voluntary, non-paid blood donors

Hyperimmune Plasma donation from voluntary, non-paid Plasmapheresis donors


<24 hr FFP,

shock frozen to - 30°C

Blood Products:

Cellular Products

Plasma Products:

Fresh Frozen Plasma (FFP) and Hyperimmune FFP

  • Whole Blood
  • Red Cell Concentrate
  • Platelet Concentrate
  • Cryofibrinogen
  • Cryoprecipitate
  • FFP– Therapeutic

Additional text example

NBI, WPBTS and SA Blood Transfusion Services work together to optimise the donor’s gift of life

factor replacement products
Factor Replacement Products
  • FVIII products
    • Advate [r-3rd gen]
    • Xyntha [r-3rd gen]
    • Kogenate FS [r-2nd gen]
    • HelixateFS [r-2nd gen]
    • Recombinate [r-1st gen]
    • Hemophil-M [pd-HP]
    • Monoclate-P [pd-HP]
  • VWF products
    • Humate-P [pd-HP]
    • Alphanate [pd-HP]
    • Wilate [pd-HP]
  • FIX products
    • BeneFIX [r-3rd gen]
    • Mononine [pd-HP]
    • Profilnine [pd-PCC]
    • Bebulin [pd-PCC]
  • Bypassing agents
    • Novo Seven [r-3rd gen]
    • FEIBA [pd-APCC]

MASAC document #151 & 106

other treatment products
Other treatment products


Boosts plasma levels of FVIII and VWF

Does not affect FIX levels

May be treatment of choice for patients with mild or moderate hemophilia A and carriers

Lower cost than plasma products and no risk of viral transmission

Test patient response prior to use


  • IV
  • SQ
  • Intranasal
other treatment products1


Promote clot stability

Useful as adjunctive therapy, particularly for skin and mucosal bleeding, e.g. oral bleeding, epistaxis, menorrhagia

Tranexamic acid available orally, IV, mouthwash

Epsilon aminocaproic acid (EACA) similar but less widely used

Do NOT use in patients with hemophilia B treated with PCCs

other treatment products2
Other treatment products

Hormone therapy (women)



Topical hemostatic agents

Fibrin sealant (fibrin glue)



Vitamin D

future treatment therapies for hemophilia
Future Treatment Therapies for Hemophilia

Longer acting concentrates

Recombinant therapy for VWD

Alternate route therapies

Gene transplantation

Elimination of transfusion-associated infections

Understanding and overcoming inhibitor development

Quality of life issues

  • Elimination of joint morbidity
  • Optimizing the individual’s social and academic performance

Treatment in hemophilia continues to progress

FFP and cryoprecipitate are still used in many parts of the world

Replacement therapies are available in a variety of forms

Choosing a factor concentrate is important to all involved in care

Efficacy, safety and cost remain important considerations when choosing a treatment product

Adjuvant therapies are available to assist in hemophilia treatment

The future of hemophilia treatment appears promising

wfh resources
WFH resources

Guide for the Assessment of Clotting Factor Concentrates

Registry of Clotting Factor Concentrates

FibrinolyticInhibitors in the Management of Bleeding Disorders

Desmopressin (DDAVP) in the Treatment of Bleeding Disorders

Guidelines for the Management of Hemophilia, 2nded