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Deficiency

Updated : December 13, 2022





Background

Epidemiology

Anatomy

Pathophysiology

Etiology

Genetics

Prognostic Factors

Clinical History

Physical Examination

Age group

Associated comorbidity

Associated activity

Acuity of presentation

Differential Diagnoses

Laboratory Studies

Imaging Studies

Procedures

Histologic Findings

Staging

Treatment Paradigm

by Stage

by Modality

Chemotherapy

Radiation Therapy

Surgical Interventions

Hormone Therapy

Immunotherapy

Hyperthermia

Photodynamic Therapy

Stem Cell Transplant

Targeted Therapy

Palliative Care

Medication

 

factor X, human 

Factor X Deficiency
Indicated for adults and children with genetic factor X deficiency
:


Perioperative therapy of bleeding with mild, moderate, or severe genetic factor X deficiency, routine prophylaxis to lessen the frequency of bleeding episodes, and on-demand treatment and control of bleeding episodes.
When the bottles are refilled using the sterile water for injection that comes with the kit, the final concentration is 100 IU/mL

Dosage Information
Individual clinical responses should be used to determine the dosage and frequency.
Administer no more than 60 IU/kg per day.
Determine the peak rise in factor X in vivo.
The dosage and duration are determined by the severity of the factor X deficiency, the location and extent of the bleeding, and the patient's clinical status.
Estimate the expected in vivo maximal increase in factor X expressed as IU/dL (or % of normal) using the following formula:
Estimated factor X Increment (IU/dL or % of Normal) = [Total Dose (IU)/Body Weight (kg)] x 2

Calculate factor X peak increase desired in vivo
The following formula may be used to determine the dosage required to produce the desired in vivo peak rise in factor X level:
The difference between the patient's plasma factor X and target levels is the desired factor X increase.
The dosage formula is based on the observed recovery of 2 IU/dL per IU/kg.

Treatment and management of bleeding episodes on demand
When bleeding appears for the first time, provide 25 IU/kg.
Continue doing this every 24 hours until the bleeding stops.

Management of haemorrhage during surgery
Before and after surgery, check each patient's post-infusion plasma factor X levels to ensure hemostatic levels are attained and maintained.
Presurgery: Use the following calculation to determine the dosage required for raising plasma factor X levels to 70-90 IU/dL: Body weight (kg) x Desired factor X Rise (IU/dL) x 0.5 = Required dosage (IU).
Postsurgery: Repeat the dosage as required until the patient is no longer in danger of bleeding, maintaining plasma factor X levels at a minimum of 50 IU/dL.

Prevention of bleeding episodes
25 mg Intravenous twice a week
It is advised that trough blood levels of FX be checked periodically due to inter- and intra-patient variability, particularly during the first several weeks of treatment or after dose adjustments.
Adapt the dosing schedule to the clinical response and minimum trough FX levels of 5 IU/dL.
The peak level of 120 IU/dL is not to be exceeded.



factor XIII A-subunit, recombinant 

Indicated for the regular stoppage of bleeding in people with congenital factor XIII A-subunit deficiency
:


Using a validated test, 35 IU/kg Intravenously once a month will provide the desired trough level of FXIII activity of ≥10%.
The dosage may be adjusted if the prescribed dose (i.e., 35 IU/kg/month) does not produce satisfactory results.



fibrinogen, human 


Indicated for Fibrinogen deficiency
RiaSTAP drug
Dose (mg/kg) = [Target levels (mg/dL) - measured levels (mg/dL)] ÷ 1.7 (mg/dL/mg/kg of body weight) as Intravenously
70 mg/kg intravenously, if the fibrinogen baseline levels are unknown
Target fibrinogen level: 100mg/dl till the hemostasis
Fibryga drug
Dose (mg/kg) = [Target levels (mg/dL) - measured levels (mg/dL)] ÷ 1.8 (mg/dL/mg/kg of body weight) as Intravenously
70 mg/kg intravenously, if the fibrinogen baseline levels are unknown
Target fibrinogen (minor bleeding) level: 100 mg/dL
Target fibrinogen (major bleeding) level: 150 mg/dL
If the fibrinogen level is less than the accepted lower limit (i.e., minor bleeding: 80 mg/dl, major bleeding: 130 mg/dl), administer an additional dose till the targeted levels of hemostasis are achieved



olipudase alfa 


Indicated for Acid Sphingomyelinase Deficiency
Dose based on body weight:
BMI (Body mass index) <30: The dose depends on actual body weight
BMI (Body mass index) >30: The dose depends on adjusted body weight
The calculation for adjusted body weight:
Adjusted weight (K.g) = Actual height (m)2 x 30
Dose build-up:
Dose 1 (day 1 or week 0): 0.1 mg/kg intravenously
Dose 2 (week 2): 0.3 mg/kg intravenously
Dose 3 (week 4): 0.3 mg/kg intravenously
Dose 4 (week 6): 0.6 mg/kg intravenously
Dose 5 (week 8): 0.6 mg/kg intravenously
Dose 6 (week 10): 1 mg/kg intravenously
Dose 7 (week 12): 2 mg/kg intravenously
Dose 8 (week 14): 3 mg/kg intravenously
Note:
The dose build-up phase includes the 1st 3 mg/kg dose
Maintenance dose: 3 mg/kg intravenously every two weeks



tibolone 

Indicated for estrogen deficiency in postmenopausal women
1 tablet orally each day
Start over the treatment after more than 1 year after the last natural bleeding
In case of surgical menopause, start over the treatment immediately to recover estrogen levels



 

factor X, human 

Factor X Deficiency
Indicated for adults and children with genetic factor X deficiency
:


Perioperative therapy of bleeding with mild, moderate, or severe genetic factor X deficiency, routine prophylaxis to lessen the frequency of bleeding episodes, and on-demand treatment and control of bleeding episodes.
When the bottles are refilled using the sterile water for injection that comes with the kit, the final concentration is 100 IU/mL

Dosage Information
The severity of the factor X deficiency, the location and extent of the bleeding, and the patient's clinical status determine the dosage and duration.
Individual clinical responses should be used to determine the dosage and frequency.
Administer no more than 60 IU/kg per day.
Determine the peak rise in factor X in vivo.
<12 years: Estimated Increase of factor X (IU/dL or % of normal) = [Total Dose (IU)/Body Weight (kg)] x 1.7
>12 years: Estimated Increase of factor X (IU/dL or % of normal) = [Total Dose (IU)/Body Weight (kg)] x 2
Calculate factor X peak increase desired in vivo
The following formula may be used to determine the dosage required to produce the desired in vivo peak rise in factor X level:
<12 years: Body Weight (kg) x Desired factor X Rise (IU/dL) x 0.6 = Dose (IU)
>12 years: Body Weight (kg) x Desired factor X Rise (IU/dL) x 0.5 = Dose (IU)
The difference between the patient's plasma factor X and target levels is the desired factor X increase.
The dosage formula is based on the observed recovery of 2 IU/dL per IU/kg.
Treatment and management of bleeding episodes on demand
When bleeding appears for the first time, provide
>12 years: 25 IU/kg intravenous
<12 years: 30 IU/kg intravenous
Continue doing this every 24 hours until the bleeding stops.

Management of haemorrhage during surgery
Before and after surgery, check each patient's post-infusion plasma factor X levels to ensure that hemostatic levels are attained and maintained.
Presurgery: Use the following calculation to determine the dosage required for raising plasma factor X levels to 70-90 IU/dL: Body weight (kg) x Desired factor X Rise (IU/dL) x 0.5 = Required dosage (IU).

Postsurgery: Repeat the dosage as required until the patient is no longer in danger of bleeding, maintaining plasma factor X levels at a minimum of 50 IU/dL.

Prevention of bleeding episodes
>12 years: 25 IU/kg Intravenous twice a week
<12 years: 40 IU/kg Intravenous twice a week
It is advised that trough blood levels of FX be checked periodically due to inter- and intra-patient variability, particularly during the first several weeks of treatment or after dose adjustments.
Adapt the dosing schedule to the clinical response and minimum trough FX levels of 5 IU/dL.
The peak level of 120 IU/dL is not to be exceeded.



factor XIII A-subunit, recombinant 

Indicated for the regular stoppage of bleeding in people with congenital Factor XIII A-subunit deficiency
:


Using a validated test, 35 IU/kg Intravenously once a month will provide the desired trough level of FXIII activity of ≥10%.
The dosage may be adjusted if the prescribed dose (i.e., 35 IU/kg/month) does not produce satisfactory results.



fibrinogen, human 


Indicated for Fibrinogen deficiency
RiaSTAP drug
Dose (mg/kg) = [Target levels (mg/dL) - measured levels (mg/dL)] ÷ 1.7 (mg/dL/mg/kg of body weight) as Intravenously
70 mg/kg intravenously, if the fibrinogen baseline levels are unknown
Target fibrinogen level: 100mg/dl till the hemostasis
Fibryga drug
Age >12 years
Dose (mg/kg) = [Target levels (mg/dL) - measured levels (mg/dL)] ÷ 1.8 (mg/dL/mg/kg of body weight) as Intravenously
Age <12 years
Dose (mg/kg) = [Target levels (mg/dL) - measured levels (mg/dL)] ÷ 1.4 (mg/dL/mg/kg of body weight) as Intravenously
70 mg/kg intravenously, if the fibrinogen baseline levels are unknown
Target fibrinogen (minor bleeding) level: 100 mg/dL
Target fibrinogen (major bleeding) level: 150 mg/dL
If the fibrinogen level is less than the accepted lower limit (i.e., minor bleeding: 80 mg/dl, major bleeding: 130 mg/dl), administer an additional dose till the targeted levels of hemostasis are achieved



olipudase alfa 


Indicated for Acid Sphingomyelinase Deficiency
Dose based on body weight:
BMI (Body mass index) <30: The dose depends on actual body weight
BMI (Body mass index) >30: The dose depends on adjusted body weight
The calculation for adjusted body weight:
Adjusted weight (K.g) = Actual height (m)2 x 30
Dose escalation:
Dose 1 (day 1 or week 0): 0.03 mg/kg intravenously
Dose 2 (week 2): 0.1 mg/kg intravenously
Dose 3 (week 4): 0.3 mg/kg intravenously
Dose 4 (week 6): 0.3 mg/kg intravenously
Dose 5 (week 8): 0.6 mg/kg intravenously
Dose 6 (week 10): 0.6 mg/kg intravenously
Dose 7 (week 12): 1 mg/kg intravenously
Dose 8 (week 14): 2 mg/kg intravenously
Dose 9 (week 16): 3 mg/kg intravenously
Note:
The dose escalation phase includes the 1st 3 mg/kg dose
Maintenance dose: 3 mg/kg intravenously every two weeks



 

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References

Deficiency

Updated : December 13, 2022




factor X, human 

Factor X Deficiency
Indicated for adults and children with genetic factor X deficiency
:


Perioperative therapy of bleeding with mild, moderate, or severe genetic factor X deficiency, routine prophylaxis to lessen the frequency of bleeding episodes, and on-demand treatment and control of bleeding episodes.
When the bottles are refilled using the sterile water for injection that comes with the kit, the final concentration is 100 IU/mL

Dosage Information
Individual clinical responses should be used to determine the dosage and frequency.
Administer no more than 60 IU/kg per day.
Determine the peak rise in factor X in vivo.
The dosage and duration are determined by the severity of the factor X deficiency, the location and extent of the bleeding, and the patient's clinical status.
Estimate the expected in vivo maximal increase in factor X expressed as IU/dL (or % of normal) using the following formula:
Estimated factor X Increment (IU/dL or % of Normal) = [Total Dose (IU)/Body Weight (kg)] x 2

Calculate factor X peak increase desired in vivo
The following formula may be used to determine the dosage required to produce the desired in vivo peak rise in factor X level:
The difference between the patient's plasma factor X and target levels is the desired factor X increase.
The dosage formula is based on the observed recovery of 2 IU/dL per IU/kg.

Treatment and management of bleeding episodes on demand
When bleeding appears for the first time, provide 25 IU/kg.
Continue doing this every 24 hours until the bleeding stops.

Management of haemorrhage during surgery
Before and after surgery, check each patient's post-infusion plasma factor X levels to ensure hemostatic levels are attained and maintained.
Presurgery: Use the following calculation to determine the dosage required for raising plasma factor X levels to 70-90 IU/dL: Body weight (kg) x Desired factor X Rise (IU/dL) x 0.5 = Required dosage (IU).
Postsurgery: Repeat the dosage as required until the patient is no longer in danger of bleeding, maintaining plasma factor X levels at a minimum of 50 IU/dL.

Prevention of bleeding episodes
25 mg Intravenous twice a week
It is advised that trough blood levels of FX be checked periodically due to inter- and intra-patient variability, particularly during the first several weeks of treatment or after dose adjustments.
Adapt the dosing schedule to the clinical response and minimum trough FX levels of 5 IU/dL.
The peak level of 120 IU/dL is not to be exceeded.



factor XIII A-subunit, recombinant 

Indicated for the regular stoppage of bleeding in people with congenital factor XIII A-subunit deficiency
:


Using a validated test, 35 IU/kg Intravenously once a month will provide the desired trough level of FXIII activity of ≥10%.
The dosage may be adjusted if the prescribed dose (i.e., 35 IU/kg/month) does not produce satisfactory results.



fibrinogen, human 


Indicated for Fibrinogen deficiency
RiaSTAP drug
Dose (mg/kg) = [Target levels (mg/dL) - measured levels (mg/dL)] ÷ 1.7 (mg/dL/mg/kg of body weight) as Intravenously
70 mg/kg intravenously, if the fibrinogen baseline levels are unknown
Target fibrinogen level: 100mg/dl till the hemostasis
Fibryga drug
Dose (mg/kg) = [Target levels (mg/dL) - measured levels (mg/dL)] ÷ 1.8 (mg/dL/mg/kg of body weight) as Intravenously
70 mg/kg intravenously, if the fibrinogen baseline levels are unknown
Target fibrinogen (minor bleeding) level: 100 mg/dL
Target fibrinogen (major bleeding) level: 150 mg/dL
If the fibrinogen level is less than the accepted lower limit (i.e., minor bleeding: 80 mg/dl, major bleeding: 130 mg/dl), administer an additional dose till the targeted levels of hemostasis are achieved



olipudase alfa 


Indicated for Acid Sphingomyelinase Deficiency
Dose based on body weight:
BMI (Body mass index) <30: The dose depends on actual body weight
BMI (Body mass index) >30: The dose depends on adjusted body weight
The calculation for adjusted body weight:
Adjusted weight (K.g) = Actual height (m)2 x 30
Dose build-up:
Dose 1 (day 1 or week 0): 0.1 mg/kg intravenously
Dose 2 (week 2): 0.3 mg/kg intravenously
Dose 3 (week 4): 0.3 mg/kg intravenously
Dose 4 (week 6): 0.6 mg/kg intravenously
Dose 5 (week 8): 0.6 mg/kg intravenously
Dose 6 (week 10): 1 mg/kg intravenously
Dose 7 (week 12): 2 mg/kg intravenously
Dose 8 (week 14): 3 mg/kg intravenously
Note:
The dose build-up phase includes the 1st 3 mg/kg dose
Maintenance dose: 3 mg/kg intravenously every two weeks



tibolone 

Indicated for estrogen deficiency in postmenopausal women
1 tablet orally each day
Start over the treatment after more than 1 year after the last natural bleeding
In case of surgical menopause, start over the treatment immediately to recover estrogen levels



factor X, human 

Factor X Deficiency
Indicated for adults and children with genetic factor X deficiency
:


Perioperative therapy of bleeding with mild, moderate, or severe genetic factor X deficiency, routine prophylaxis to lessen the frequency of bleeding episodes, and on-demand treatment and control of bleeding episodes.
When the bottles are refilled using the sterile water for injection that comes with the kit, the final concentration is 100 IU/mL

Dosage Information
The severity of the factor X deficiency, the location and extent of the bleeding, and the patient's clinical status determine the dosage and duration.
Individual clinical responses should be used to determine the dosage and frequency.
Administer no more than 60 IU/kg per day.
Determine the peak rise in factor X in vivo.
<12 years: Estimated Increase of factor X (IU/dL or % of normal) = [Total Dose (IU)/Body Weight (kg)] x 1.7
>12 years: Estimated Increase of factor X (IU/dL or % of normal) = [Total Dose (IU)/Body Weight (kg)] x 2
Calculate factor X peak increase desired in vivo
The following formula may be used to determine the dosage required to produce the desired in vivo peak rise in factor X level:
<12 years: Body Weight (kg) x Desired factor X Rise (IU/dL) x 0.6 = Dose (IU)
>12 years: Body Weight (kg) x Desired factor X Rise (IU/dL) x 0.5 = Dose (IU)
The difference between the patient's plasma factor X and target levels is the desired factor X increase.
The dosage formula is based on the observed recovery of 2 IU/dL per IU/kg.
Treatment and management of bleeding episodes on demand
When bleeding appears for the first time, provide
>12 years: 25 IU/kg intravenous
<12 years: 30 IU/kg intravenous
Continue doing this every 24 hours until the bleeding stops.

Management of haemorrhage during surgery
Before and after surgery, check each patient's post-infusion plasma factor X levels to ensure that hemostatic levels are attained and maintained.
Presurgery: Use the following calculation to determine the dosage required for raising plasma factor X levels to 70-90 IU/dL: Body weight (kg) x Desired factor X Rise (IU/dL) x 0.5 = Required dosage (IU).

Postsurgery: Repeat the dosage as required until the patient is no longer in danger of bleeding, maintaining plasma factor X levels at a minimum of 50 IU/dL.

Prevention of bleeding episodes
>12 years: 25 IU/kg Intravenous twice a week
<12 years: 40 IU/kg Intravenous twice a week
It is advised that trough blood levels of FX be checked periodically due to inter- and intra-patient variability, particularly during the first several weeks of treatment or after dose adjustments.
Adapt the dosing schedule to the clinical response and minimum trough FX levels of 5 IU/dL.
The peak level of 120 IU/dL is not to be exceeded.



factor XIII A-subunit, recombinant 

Indicated for the regular stoppage of bleeding in people with congenital Factor XIII A-subunit deficiency
:


Using a validated test, 35 IU/kg Intravenously once a month will provide the desired trough level of FXIII activity of ≥10%.
The dosage may be adjusted if the prescribed dose (i.e., 35 IU/kg/month) does not produce satisfactory results.



fibrinogen, human 


Indicated for Fibrinogen deficiency
RiaSTAP drug
Dose (mg/kg) = [Target levels (mg/dL) - measured levels (mg/dL)] ÷ 1.7 (mg/dL/mg/kg of body weight) as Intravenously
70 mg/kg intravenously, if the fibrinogen baseline levels are unknown
Target fibrinogen level: 100mg/dl till the hemostasis
Fibryga drug
Age >12 years
Dose (mg/kg) = [Target levels (mg/dL) - measured levels (mg/dL)] ÷ 1.8 (mg/dL/mg/kg of body weight) as Intravenously
Age <12 years
Dose (mg/kg) = [Target levels (mg/dL) - measured levels (mg/dL)] ÷ 1.4 (mg/dL/mg/kg of body weight) as Intravenously
70 mg/kg intravenously, if the fibrinogen baseline levels are unknown
Target fibrinogen (minor bleeding) level: 100 mg/dL
Target fibrinogen (major bleeding) level: 150 mg/dL
If the fibrinogen level is less than the accepted lower limit (i.e., minor bleeding: 80 mg/dl, major bleeding: 130 mg/dl), administer an additional dose till the targeted levels of hemostasis are achieved



olipudase alfa 


Indicated for Acid Sphingomyelinase Deficiency
Dose based on body weight:
BMI (Body mass index) <30: The dose depends on actual body weight
BMI (Body mass index) >30: The dose depends on adjusted body weight
The calculation for adjusted body weight:
Adjusted weight (K.g) = Actual height (m)2 x 30
Dose escalation:
Dose 1 (day 1 or week 0): 0.03 mg/kg intravenously
Dose 2 (week 2): 0.1 mg/kg intravenously
Dose 3 (week 4): 0.3 mg/kg intravenously
Dose 4 (week 6): 0.3 mg/kg intravenously
Dose 5 (week 8): 0.6 mg/kg intravenously
Dose 6 (week 10): 0.6 mg/kg intravenously
Dose 7 (week 12): 1 mg/kg intravenously
Dose 8 (week 14): 2 mg/kg intravenously
Dose 9 (week 16): 3 mg/kg intravenously
Note:
The dose escalation phase includes the 1st 3 mg/kg dose
Maintenance dose: 3 mg/kg intravenously every two weeks



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