haemophillia333

8/9/2019 Haemophillia333

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By:Pharmacist

Duraid Khalid AL-DabanClinical Pharmacy Division Manager

Pharmacy Dept./Selah aldin H.D./ MOH-Iraq


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Bleeding& Coagulation

7/31/2010 2By: Pharmacist Duraid Khalid Al-Daban


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Clotting Factors

7/31/2010 3By: Pharmacist Duraid Khalid Al-Dab

an


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The Clotting Cascades

HMWK = high molecular weight kininogen.



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7/31/2010 By: Pharmacist Duraid Khalid Al-Daban 7


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Haemophillia Types



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There are two types of Hemophilia, A and B. Both are caused by

deficiencies in the amount of clotting factor in the blood (VIII or

IX). When the blood does not have enough of one of these or is

missing one clotting factor, the bleeding may end very slowly or

may not stop at all. The two types of Hemophilia are linked

together by their similar clinical pictures and their similar

inheritance patterns.

The most dangerous part about having Hemophilia is internal

bleeding. If internal bleeding is left untreated it can lead to

deformity, disability or even death. In a Hemophiliac the

bleeding continues until either it clots long enough for it to heal

or the person will bleed to death.



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History ofHemophilia

The earliest references to hemophilia can be found in

second century. (next page)

The first attempts to treat hemophilia was by replacing the

clotting factory with blood plasma taken from pigs and cows.

In the 1970s scientists found two approaches to the disease

One that was called prophylaxis required injecting doses of

the clotting factor on a regular basis

The second was to inject the factor whenever the bleeding

occurred



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. . . if the first son of a woman iscircumcised and he dies and

the second son is circumcisedand he dies, you must notcircumcise the third son. . . .

Additionally, the sons of thewoman's sister should not becircumcised but the sons of herbrother can be circumcised.

The Talmud (Yebamot 64b)



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Hemophilia is a genetic disease and is passed on by the X chromosome (the chromosome that

carries the clotting factor).

If a boy gets the X chromosome that carries the hemophilia gene he will become a hemophiliac.

If a girl get the gene, she will become the carrier of the gene, not showing symptoms of the disease

though she may have a long or heavy menstrual cycle. The carrier has a 50% chance of passing the

gene on to her children every time she gets pregnant.



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Clinical Severity

Factor level < 2 U/dL

Frequent ,spontaneous bleeding into joints, muscles andinternal organs

Incidence 50% of cases

Se ere

Factor le el = 2-10 U/dL

Some spontaneous bleeds, bleeding after minor trauma

Incidence 3030%% of cases

Moderatelyse ere

Factor le el >10-30 U/dL

Bleeding only after significant trauma, surgery

Incidence 20% of casesMild



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Is this child haemophilic ?

When a baby starts to crawl the parentsmay notice bruises on stomach, chest,buttock, and back.

The baby may also be fussy, notwanting to walk or crawl

Other symptoms include longnosebleeds, excessive bleeding frombiting down on the lips or tongue,excessive bleeding following a toothextraction, excessive bleeding followingsurgery and blood in the urine.



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Life Span in the last 70 years


010

20

30

40

50

60

70

80

Avrage

age

Before 1938

before 1968

1968

1938

1988

1999


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Complications

Severe complications are much more common in severe and

moderate haemophiliacs. Complications may be both directly from the

disease or from its treatment:[

Deep internal bleeding, e.g. deep-muscle bleeding, leading to

swelling, numbness or pain of a limb.

Joint damage, potentially with severe pain and even destruction of the

joint and development ofarthritis.

Transfusion transmitted infection from blood transfusions that are

given as treatment.

Adverse reactions to clotting factor treatment, including the

development of an immune inhibitor which renders factor replacement

less effective.

Intracranial hemorrhage, is a serious medical emergency cause by

the buildup of pressure inside the skull. It can cause disorientation,

nausea, loss of consciousness, brain damage, and death.



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If a patient becomes refractory to replacement coagulation factor

as a result of circulating inhibitors, this may be partially

overcome with recombinant human factor VII (NovoSeven),

which is registered for this indication in many countries.

In early 2008, the USFood and Drug Administration (FDA)

approved Xyntha (Wyeth) anti-haemophilic factor, genetically

engineered from the genes of Chinese hamster ovary cells.

Since 1993 (Dr. Mary Nugent) recombinant factor products(which are typically cultured in Chinese hamster ovary (CHO)

tissue culture cells and involve little, if any human plasma

products) have been available and have been widely used in

wealthier western countries. While recombinant clotting factor

products offer higher purity and safety, they are, like concentrate,extremely expensive, and not generally available in the

developing world. In many cases, factor products of any sort are

difficult to obtain in developing countries.


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Xyntha

XYNTHA [Anti hemophilic Factor

(Recombinant), Plasma/Albumin-

Free] For Intravenous Use, Freeze-

Dried Powder

Initial U.S. Approval: 2008



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Benefix


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