| |
| |
| treatment |
| Due
to the rarity of each factor deficiency, RBDs are relatively
neglected by health care providers, advocacy organizations,
and drug manufacturers. Purified factor concentrates for treatment
of RBDs are not as readily available as they are for haemophilia
A and B. |
| |
| Treatment
of RBDs generally consists of the use of the most purified blood
product available that contains the missing factor. Dosages
and frequency of treatment depend on minimal haemostatic levels
of the deficient factor, plasma half-life and type of bleeding
episode (Table 3). |
| |
| Viral
inactivated plasma, purified concentrates, prothrombin complex
concentrates, cryoprecipitates, or fresh frozen plasma can be
used 1-9-11.
In addition, one recombinant DNA technology drug is available
in Europe but not in USA: recombinant factor VIIa, available
for treating factor VII deficient patients. FV and combined
FV+VIII deficiencies can only be treated with fresh frozen plasma,
because no suitable concentrate form exists. |
| |
| In
choosing the type of treatment consideration of the safety of
the replacement material is of paramount importance. Viral-inactivated
plasma is an important source of replacement, to be recommended
in the majority of these disorders. |
| |
|
Commercial factor replacement concentrates, when available,
are safe but often prohibitively expensive especially for the
countries with the highest frequency of RBDs. Non-virus inactivated
plasma and cryoprecipitates should be avoided if possible. |
| |
| Non-replacement
treatment is often available. Antifibrinolytic amino acids may
be useful either alone or in combination with replacement therapy,
in the management of the less severe forms of mucosal tract
haemorrhages. |
| |
| Epsilon
aminocaproic acid and tranexamic acid can be administered orally
or intravenously. |
| |
| The
continued use of estrogen-progestogen preparations helps to
reduce menstrual blood loss in women with iron deficiency anaemia
due to menorrhagia 9.
|
| |
| The best choice of treatment may vary according to the location, society, customs and economy of the country where the patient is resident. |
|
