Hear from a peer about the ADVATE active patient type—video transcript

ADVATE [Antihemophilic Factor (Recombinant)] is a recombinant antihemophilic factor indicated for use in children and adults with hemophilia A for the control and prevention of bleeding episodes, for perioperative management, and for routine prophylaxis to prevent or reduce the frequency of bleeding episodes.

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

ADVATE is contraindicated in patients who have life-threatening hypersensitivity reactions, including anaphylaxis, to mouse or hamster protein or other constituents of the product.

Hi. My name is Dr. Jonathan Roberts, and today, I'm here to talk to you about a particular type of patient with hemophilia A for whom ADVATE is worth considering—active patients.

By active patients, I mean patients who have an active lifestyle. While this could certainly mean participation in appropriate sports, it could also mean a patient whose hobbies keep them moving, such as those who play instruments in a band or like to garden. It could also be a patient whose job has them on their feet all day.

Of course, every patient's activity level and coverage needs will vary depending on the scheduled activities. I want my active patients to be able to keep up with their activities. So, for these patients, my main considerations are proven bleed protection and personalization of treatment. Together, these key treatment considerations can help provide bleed coverage to meet patients’ lifestyle and treatment needs.

One way to personalize treatment is to adjust the patient's treatment regimen to match their factor level and intensity of activity. Research has shown that increased time with a factor VIII activity below 1% is associated with an increased rate of breakthrough bleeding, and higher levels of factor coverage may help prevent bleeds during peak activity levels.

Infusing with factor therapy provides measurable coverage to protect against bleeds and joint bleeds to help active patients participate in their activities.

With more than 15 years of clinical and real-world experience, ADVATE offers bleed protection as well as a variety of options for treatment personalization. For these reasons, I consider ADVATE as a treatment option for my active patients.

Let's take a closer look at the data supporting bleed protection with ADVATE.

A clinical study was performed to assess the relative efficacy of ADVATE use in 2 prophylactic treatment regimens compared with that of on-demand treatment.

In this study, 53 previously treated patients with severe to moderately severe hemophilia underwent 6 months of on-demand treatment, followed by 1 year of either a standard or a pharmacokinetic-tailored (or PK-tailored) prophylaxis regimen.

In this study, ADVATE demonstrated the ability to help prevent bleeding episodes using a prophylaxis regimen with a median ABR and AJBR of 1 during the prophylaxis treatment period.

ABRs for the 2 prophylactic regimens were comparable. A 98% and 97% reduction in bleeds and joint bleeds, respectively, was observed after switching from on-demand or standard to PK-guided prophylaxis. When I consider active patients, the low median ABR and AJBR are compelling.

Before we move on to personalization, let's review some Important Safety Information for ADVATE.

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.

Earlier, we defined the active patient and considered personalization as a key component of treatment for these patients. Factor therapy offers flexibility in both dose and frequency of dosing. And with ADVATE, there is an additional opportunity for personalization. This comes in the form of myPKFiT software—the first and only FDA-approved PK dosing software available for your hemophilia A patients on ADVATE.

The myPKFiT software can be used to help personalize treatment by generating ADVATE dosage amount and frequency recommendations using an individual patient's age and body weight and local laboratory factor VIII one-stage clotting activity measurements.

For ADVATE, myPKFiT is intended to be used with patients aged 16 years or more with a body weight of 45 kilograms or greater. With myPKFiT, PK testing can be performed with a minimum of 2 sparse sampling points—required 3 to 4 hours and 24 to 32 hours post-infusion.

The software may be used to guide decisions on appropriate ADVATE dose and infusion intervals for individual patients in accordance with the dosing recommendations approved by the FDA. myPKFiT is prescription only and should only be used to evaluate prophylactic dosing regimens for patients treated with ADVATE. Is not indicated for the treatment of von Willebrand disease and should not be used for patients who have developed an inhibitor to factor VIII products.

For physicians, myPKFiT software provides the opportunity to immediately assess how a variety of regimens would affect an individual's estimated factor levels by allowing for adjustment of the trough level, which can be set to a target of 1% to 3% above baseline; the dosing interval and time above a specific factor level to help align higher levels with the patient's scheduled activities; the dose; and the time below an adjustable level.

For active patients, myPKFiT can help identify a regimen that is tailored to a patient's needs, taking into account their activities throughout the week, as well as provide estimates of a patient's factor levels at any given time.

Before we wrap up, let's review some Important Safety Information for ADVATE.

Neutralizing antibodies (or inhibitors) have been reported following administration of ADVATE, predominantly in previously untreated patients (or PUPs) and previously minimally treated patients. Monitor patients treated with ADVATE regularly for the development of factor VIII inhibitors; and measure factor VIII inhibitor concentration if expected plasma factor VIII activity levels are not attained, or if bleeding is not controlled with the expected dose.

To summarize, in this video, we have considered a particular subset of patients—active patients. We have discussed some important treatment considerations for these patients, including bleed protection and personalization.

We have also examined the features of ADVATE, including the low median ABR and AJBR in clinical trials, and the opportunity to simplify, personalize, and educate with myPKFiT.

A final important note with regards to ADVATE safety information.

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 (in >5% of subjects) were pyrexia, headache, cough, nasopharyngitis, arthralgia, vomiting, upper respiratory tract infection, limb injury, nasal congestion, and diarrhea.

Please see full ADVATE Prescribing Information.

Thank you for taking the time to consider ADVATE for active patients. For more information on ADVATE and the full Prescribing Information, please visit advatepro.com.

Hear from a peer about the ADVATE high-risk bleed patient type—video transcript

ADVATE [Antihemophilic Factor (Recombinant)] is a recombinant antihemophilic factor indicated for use in children and adults with hemophilia A for the control and prevention of bleeding episodes, or perioperative management, and for routine prophylaxis to prevent or reduce the frequency of bleeding episodes.

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

ADVATE is contraindicated in patients who have life-threatening hypersensitivity reactions, including anaphylaxis, to mouse or hamster protein or other constituents of the product.

Hi. My name is Dr. Mark Reding, and today, I'd like to talk to you about ADVATE.

In particular, I'd like to focus on a specific type of patient with hemophilia A for whom ADVATE is worth considering—patients with a high risk of bleeds.

By that I mean patients that have a high-risk bleeding phenotype which could include a history of severe bleeds and/or history of target joints. Because of their bleeding history, these patients may be previously treated patients (or PTPs).

Based on their bleeding phenotype, concerns for these patients include addressing bleeds and joint bleeds via prophylaxis, understanding the impact of their bleeding history on their quality of life, and assessing options for improvement. And of course, weighing the safety profile of the treatment options.

As a long-established standard half-life product, ADVATE offers bleed protection, over 15 years of clinical and real-world experience, and an established safety profile. For these reasons, I consider ADVATE as a treatment option for my high-risk-of-bleed patients.

Let's review the data supporting the use of ADVATE for prophylaxis.

The clinical study was performed to assess the relative efficacy of ADVATE use in 2 prophylactic treatment regimens compared to that of on-demand treatment.

In this study, 53 previously treated patients with severe to moderately severe hemophilia underwent 6 months of on-demand treatment, followed by 1 year of either a standard or a pharmacokinetically tailored prophylaxis regimen.

In this study, ADVATE demonstrated the ability to help prevent bleeding episodes using a prophylaxis regimen with a median ABR and AJBR of 1 during the prophylaxis treatment period. ABRs for the 2 prophylaxis regimens were comparable.

A 98% and 97% reduction in bleeds and joint bleeds, respectively, was observed after switching from on-demand to standard or PK-guided prophylaxis. When I consider high-risk-of-bleed patients, the low median ABR and AJBR are compelling. It was also found that 42% of patients on prophylaxis with ADVATE experienced zero bleeding episodes.

Before we move on to real-world data, let's review some Important Safety Information for ADVATE.

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.

In addition to the clinical trial, a real-world study, known as the AHEAD study, is being conducted in patients with severe and moderate hemophilia A treated with ADVATE. Interim data from the first 3 years of this 8-year study is currently available for 522 patients.

The primary study objective was to describe joint health in patients receiving ADVATE in a routine clinical practice setting using any treatment regimen. Secondary objectives included assessing long-term outcome data, annualized bleeding rates, and safety.

Because this is a real-world study, it is important to note that there are some limitations, including lack of a standardized treatment protocol and a control arm—with most safety and effectiveness parameters based on participants’ recall or self-reported information.

It’s also important to remember that this is the preliminary analysis, and data on adherence, which could impact outcomes, was not collected. Since these data are from a prospective, noninterventional study, further confirmatory studies are required to draw any conclusions from these data.

In the first 3 years of the AHEAD study, the median annualized bleeding rate in the prophylaxis group ranged from 1.6 to 2.2. In addition, the median annualized joint bleeding rate for the prophylaxis group ranged from 0.9 to 1 in the first 3 years of the study.

The prophylaxis clinical trial for ADVATE also examined health-related quality of life in patients on prophylaxis versus on-demand therapy. After 12 months of prophylaxis with ADVATE, clinically meaningful improvements were observed in physical health-related quality of life, mainly due to a reduction in bodily pain and the impact that can have on performing work or other daily activities.

Clinically meaningful changes were not seen in other physical-functioning categories or general health, nor were they seen in mental health-related score components.

My third treatment consideration for this patient type was the safety profile. The safety of ADVATE in previously treated patients was evaluated from 8 studies in 276 previously treated patients with moderately severe to severe hemophilia A. In this analysis, previously treated patients were defined as those patients with at least 50 exposure days. In the combined analysis, there was a 0.36% incidence of inhibitors, where 1 previously treated patient developed a nonpersistent low-titer inhibitor, and there was another possible case of a low-titer inhibitor.

Let's review some Important Safety Information for ADVATE.

Neutralizing antibodies (or inhibitors) have been reported following administration of ADVATE predominantly in previously untreated patients (or PUPs) and previously minimally treated patients (or MTPs).

Monitor patients treated with ADVATE regularly for the development of factor VIII inhibitors and measure factor VIII inhibitor concentration if expected plasma factor VIII activity levels are not attained, or if bleeding is not controlled with an expected dose.

To summarize, in this video, we have considered a particular subset of patients, those with a high risk of bleeds. We've discussed some important treatment considerations for these patients. We've also examined the ADVATE clinical trial data, including the low median ABR and AJBR, the health-related quality-of-life data, and the established safety profile.

A final important note with regard to ADVATE safety information: 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, which occurred in more than 5% of subjects were pyrexia, headache, cough, nasopharyngitis, arthralgia, vomiting, upper respiratory tract infection, limb injury, nasal congestion, and diarrhea.

Please see full ADVATE Prescribing Information for further details.

Thank you for taking the time to consider ADVATE for your patients at a high risk of bleeds. For more information on ADVATE and the full Prescribing Information, please visit advatepro.com.