myPKFiT® Personalized Dosing Software App for ADVATE® [Antihemophilic Factor (Recombinant)] Patients

Intended Use: myPKFiT is not indicated for the treatment of von Willebrand disease and should not be used for patients who have developed neutralizing antibody to FVIII products. myPKFiT is Rx only. For safe and proper use of the myPKFiT software, please refer to the complete instructions for use in the User Manual.

Please see the full intended use statement for myPKFiT below.

What is personalized PK-guided treatment?

PK-guided treatment utilizes a patient’s pharmacokinetic (PK) profile to create a dosing regimen that is personalized to their lifestyle. PK characteristics may be influenced by patient specific variables such as age and body weight.¹

Patients who may benefit

Personalized PK-guided treatment may benefit patients who^2,3:

Live an active, busy lifestyle
Participate in physical activities
Experience inadequate bleed control

myPKFiT for ADVATE helps enable PK-guided prophylaxis treatment^1,2

myPKFiT is the first and only FDA-approved PK dosing software and patient app designed for ADVATE patients 16 years and older with a body weight of 45 kg or greater with hemophilia A.⁴

simplify

The process of PK
testing and data
analysis^†1,5,6

Personalize

Treatment plans
based on a PK
assessment¹

Educate

Patients using the
software’s reporting
capability^1,2

^†Estimate a PK curve with as few as 2 measurable blood samples, compared to 9-11 as recommended by International Society on Thrombosis and Haemostasis guidelines.

Real-world experience with myPKFiT for ADVATE software^2,3

myPKFiT for ADVATE can be a helpful tool for developing strategies intended to improve adherence, tailor prophylaxis, and provide patient education.
In a retrospective chart view, myPKFiT was used to estimate PK profiles for 27 patients with severe hemophilia A who had not received prior PK assessments
Limitations: this report included a small number of patients, and a study with a larger sample would be needed to confirm these results

A personalized approach to prophylaxis may help patients achieve their goals

Real-world study description⁷

A prospective, observational case series that utilized myPKFiT to estimate PK profiles in a subset of 22 patients recruited from 3 centers in Spain, aged 16-53 years old with severe hemophilia A, without inhibitors, on prophylaxis with ADVATE. Specifically, the study looked at the impact of myPKFiT use for prophylaxis adjustment, annual bleeding rate (ABR), and annual joint bleeding rate (AJBR).

Limitations⁷

Overall effect of myPKFiT on positive results seen in this short-term case series cannot be further assessed, considering the small sample of patients for statistical analysis (n=22).

A study with a larger sample would be needed to confirm these results.

ABR=annualized bleed rate; AJBR=annualized joint bleed rate; IQR=interquartile range.

Advate Selected Important Risk Information

CONTRAINDICATIONS: Patients who have life-threatening hypersensitivity reactions, including anaphylaxis, to mouse or hamster protein or other constituents of the product.

Please see additional detailed important risk information below.

Real-world experiences with myPKFiT

A prospective, observational case series found ABRs and AJBRs were reduced with a personalized prophylaxis regimen (utilizing myPKFiT) vs prior standard prophylaxis schedule in this patient subset.⁷

ABR using myPKFiT

50% reduced ABR after adjustment

(n=11; mean ABR reduction: -2.6 ± 1.4)

18.2 % increased ABR after adjustment

(n=4; mean ABR increase: 1.0 ± 0.0)

AJBR using myPKFiT

54.5% reduced AJBR after adjustment

(n=12; mean AJBR reduction: -2.0 ± 1.2)

9.1% increased AJBR after adjustment

(n=2; mean AJBR increase: 1.0 ± 0.0)

ABR=annualized bleed rate; AJBR=annualized joint bleed rate; IQR=interquartile range.

ADVATE Important Information

Indications

ADVATE is a recombinant antihemophilic factor indicated for use in children and adults with hemophilia A (congenital factor VIII deficiency) for:

Control and prevention of bleeding episodes.
Perioperative management.
Routine prophylaxis to prevent or reduce the frequency of bleeding episodes.

ADVATE is not indicated for the treatment of von Willebrand disease.

DETAILED IMPORTANT RISK INFORMATION

CONTRAINDICATIONS

Patients who have life-threatening hypersensitivity reactions, including anaphylaxis, to mouse or hamster protein or other constituents of the product.

WARNINGS & PRECAUTIONS

Hypersensitivity Reactions

Allergic-type hypersensitivity reactions, including anaphylaxis, have been reported with ADVATE. Symptoms include dizziness, paresthesia, rash, flushing, facial swelling, urticaria, dyspnea, pruritus, and vomiting. Discontinue ADVATE if hypersensitivity symptoms occur and administer appropriate emergency treatment.

Neutralizing Antibodies

Neutralizing antibodies (inhibitors) have been reported following administration of ADVATE predominantly in previously untreated patients (PUPs) and previously minimally treated patients (MTPs). Monitor all patients for the development of factor VIII inhibitors by appropriate clinical observation and laboratory testing. If expected plasma factor VIII activity levels are not attained, or if bleeding is not controlled with an expected dose, perform an assay that measures factor VIII inhibitor concentration.

ADVERSE REACTIONS

Serious adverse reactions seen with ADVATE are hypersensitivity reactions, including anaphylaxis, and the development of high-titer inhibitors necessitating alternative treatments to factor VIII.
The most common adverse reactions observed in clinical trials (>5% of subjects) were pyrexia, headache, cough, nasopharyngitis, arthralgia, vomiting, upper respiratory tract infection, limb injury, nasal congestion, and diarrhea.

Click to read Full Prescribing Information.

MYPKFIT FOR ADVATE INTENDED USE

The myPKFiT software is intended for use by licensed healthcare professionals (HCPs) who are familiar with hemophilia care. The myPKFiT software can be used to generate ADVATE dosage amount and frequency recommendations for routine prophylaxis for an individual patient 16 years of age and older and body weight of 45 kg or greater, using that patient’s age and body weight information and local laboratory FVIII one-stage clotting activity measurements of sparse samples collected from that patient.
A minimum of two sparse sampling points are required at the recommended 3-4 hours (± 30 minutes) and at 24-32 hours (±1 hour) post-infusion.
HCPs will also be able to evaluate various prophylaxis dose regimens tailored to an individual patient's needs and treatment plan.
The software output may be used to guide decisions on appropriate ADVATE dose and infusion intervals to maintain FVIII activity levels at or above a user specified minimum FVIII activity level between 1% to 3% above natural baseline for an individual patient in accordance with the FDA-approved dosing recommendations provided in the ADVATE Prescribing Information (PI).
myPKFiT should only be used to evaluate prophylactic dosing regimens for hemophilia A patients treated with ADVATE, as per the ADVATE Prescribing Information (PI).
myPKFiT for ADVATE is not indicated for the treatment of von Willebrand disease. MyPKFiT for ADVATE should not be used for patients who have developed neutralizing antibody (inhibitor) to FVIII products.

myPKFiT for ADVATE is Rx only. For safe and proper use of the myPKFiT software, please refer to the complete instructions for use in the User Manual.

References References

myPKFiT for healthcare professionals. myPKFiT User Manual. Version 3.3. Shire Inc; 2020.
Álvarez-Román MT, Fernandez-Bello I, de la Corte-Rodriguez H, et al. Experience of tailoring prophylaxis using factor VIII pharmacokinetic parameters estimated with myPKFiT® in patients with severe haemophilia A without inhibitors. Haemophilia. 2017;23(1):e50-e54. doi:10.1111/hae.13141
Valentino LA. Considerations in individualizing prophylaxis in patients with haemophilia A. Haemophilia. 2014;20(5):607-615. doi:10.1111/hae.12438
Takeda data on file.
Björkman S. Limited blood sampling for pharmacokinetic dose tailoring of FVIII in the prophylactic treatment of haemophilia A. Haemophilia. 2010;16(4):597-605. doi:10.1111/j.1365-2516.2009.02191.x
Lee M, Morfini M, Schulman S, Ingerslev J; and the Factor VIII/Factor IX Scientific and Standardization Committee of the International Society on Thrombosis and Haemostasis. The design and analysis of pharmacokinetic studies of coagulation factors. International Society on Thrombosis and Haemostasis Website. https://cdn.ymaws.com/www.isth.org/resource/group/d4a6f49a-f4ec-450f-9e0f-7be9f0c2ab2e/official_communications/fviiipharmaco.pdf. March 21, 2001. Accessed July 20, 2018.
Mingot-Castellano ME, Parra R, Núñez R, Martorell M. Improvement in clinical outcomes and replacement factor VIII use in patients with haemophilia A after factor VIII pharmacokinetic-guided prophylaxis based on Bayesian models with myPKFiT®. Haemophilia. 2018;24(5):e338-e343. doi: 10.1111/hae.13540

What is personalized PK-guided treatment?

myPKFiT for ADVATE helps enable PK-guided prophylaxis treatment1,2

The process of PK testing and data analysis†1,5,6

Treatment plans based on a PK assessment1

Patients using the software’s reporting capability1,2

Real-world experience with myPKFiT for ADVATE software2,3