Comprehensive Pediatric Care of Rare Bleeding Disorders

Muriel Giansily-Blaizot; Jean-François Schved

Disclosures

Pediatr Health. 2010;4(2):209-217. 

In This Article

Treatment: Which Drug & Which Dose?

Treatments should be divided into two groups: replacement therapy and nonspecific drugs. For all of the disorders studied, nonspecific antihemorrhagic drugs can be helpful for preventing hemorrhages or decreasing their frequency. The most widely used antihemorrhagic drug, which is particularly used in cases of FXI deficiency, is tranexamic acid. However, replacement therapies are required in all cases involving hemorrhagic complications. These involve the use of either a specific concentrate (FXI, FXIII and fibrinogen), when available, or a mixture of different concentrates (either prothrombin complex concentrate [PCC] containing FX, FII, FIX and FVII, or fresh frozen plasma [FFP]). Thus, the treatment requires blood-derived products. Therefore, while viral and pathogen inactivation dramatically reduces the risk of transfusion-transmitted disease, vaccination, when available, is recommended, notably against the hepatitis viruses. The availability of treatment differs according to geographical location; however, the possible choices are as follows:

  • Fibrinogen deficiency: fibrinogen concentrate, cryoprecipitate or FFP;

  • FII deficiency: PCC or FFP;

  • FV deficiency: FFP or platelets;

  • FVII deficiency: recombinant factor VIIa (rFVIIA) and FVII concentrates can be used when available. PCC can be used as an alternative therapy;

  • FX deficiency: freeze-dried human coagulation FIX and FX concentrate is available in some countries. PCC or FFP can also be used;

  • FXI deficiency: use FXI concentrates. When FXI concentrates are not available, FFP can also be used;

  • FXIII deficiency: FXIII concentrate.

There is no standard dose for any of these drugs – the dose infused must be calculated according to the basal level and the level expected after infusion. As such, a pharmacokinetic study in the patient is sometimes required to determine recovery and drug half-life. It should be noted that the expected postinfusion level is not the same for all drugs.

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