Genotyping for Progress in Hemophilia

 
 

frequently asked questions

  • What is the purpose of this initiative? My Life, Our Future (MLOF) is dedicated to improving the lives of people affected by hemophilia through knowledge. Our goals are to enable people with hemophilia and their families to understand more about their hemophilia and to advance scientific research that may improve care and treatment.
  • Who created it?The initiative was founded by leaders in the hemophilia community who are steadfast in their commitment to progress, including the American Thrombosis and Hemostasis Network (ATHN), the National Hemophilia Foundation (NHF), Bloodworks Northwest, and Bioverativ (formerly Biogen).
  • Who can participate? MLOF is currently available to people with hemophilia A and B through participating hemophilia treatment centers (HTCs), as well as confirmed and potential (unconfirmed) carriers. If you are interested in participating, so please check with your HTC to learn more.
  • Can family members and carriers participate in My Life, Our Future at this time? Yes, the program is available to potential (unconfirmed) carriers and confirmed carriers. Specific timing for availability varies by site so check with your HTC to learn more.
  • Who is eligible to participate in the carrier expansion? Eligible participants include:

    • Potential (unconfirmed) carrier, regardless of symptoms
    • Known carrier / factor level <50% / symptomatic (already currently eligible)
    • Known carrier / factor level >50%

    To be eligible to participate in MLOF as a potential (unconfirmed) carrier, you must have a first, second or third degree relative with a confirmed hemophilia A or B diagnosis.

    • First degree relatives include a child, parent, full sibling, and half-sibling by same mother
    • Second-degree relatives include a grandparent, aunt/uncle, grandchild or niece/nephew
    • Third-degree relatives include first cousins and great-grandparents
  • Where is My Life, Our Future available? MLOF is currently available at HTCs across the U.S. Find out if your HTC is participating in the program. Check back regularly — as sites receive institutional review board (IRB) approval, they will be posted.
  • How can I learn about my genotype? If you're interested in learning about your genotype, find out if your HTC is participating in MLOF or ask your healthcare provider for additional information about the program. Once you sign up to participate, your healthcare provider will conduct a blood draw and send the sample to Bloodworks Northwest, a specialized laboratory with expertise in genetic testing and analysis. Your HTC will then receive a report summarizing the genotyping result, which will be shared with you to support your care and treatment.
  • How will my participation support research? You can play an active part in helping the hemophilia community by participating in the research portion of the program; more than 5,000 people have already chosen to so! If you choose to contribute and give informed consent, your de-identified data will be added to the ATHNdataset and coded blood samples added to the My Life, Our Future Research Repository — a collection of samples and data for scientific study.

    The long-term goal of this partnership is to help researchers and clinicians better understand connections between the genetic cause of hemophilia and clinical outcomes. With this enhanced database, scientific research may be accelerated to improve scientific understanding and potentially identify new treatment options.
  • Who determines who can access the My Life, Our Future Research Repository samples for research? An independent, international, multidisciplinary committee managed by ATHN conducts a rigorous review of these applications to access the My Life, Our Future Research Repository. This research review committee includes a molecular pathologist, genetic epidemiologist, patient representative, and hematologists. Bioverativ and/or other industry representatives will not be included on the review committee. Applications will be selected based on scientific merit of their proposals and level of benefit to those with bleeding disorders.
  • Who owns the data and samples contained in the My Life, Our Future Research Repository? Data and samples collected through this program are not owned by any particular party and will never be sold.
  • What is a research repository? A repository is like a bank. It is a collection of samples and data collected through the MLOF program, all stored securely. Like a bank account number, your sample and data will be coded to protect your identity and personal information. Your name and other identifying information are never placed in the My Life, Our Future Research Repository, so confidentiality and privacy are protected. Now that more than 5,000 samples have been collected, scientists and researchers at U.S. academic institutions or companies are able to apply for access to this database to support scientifically sound research projects. Samples and data will continue to be added to the My Life, Our Future Research Repository until enrollment in the program ceases. In addition, through a collaboration with the National Institute of Health, the My Life, Our Future Research Repository samples are undergoing whole-genome analysis.
  • Why would someone volunteer to contribute to a research repository? In addition to learning more about your hemophilia today, you can play an active part in helping the hemophilia community. You can help scientists and researchers better understand hemophilia by contributing to medical research.
  • What questions do you anticipate researchers hope to answer using the My Life, Our Future Research Repository? The long-term goal of MLOF is to help researchers and clinicians better understand connections between the genetic cause of hemophilia and clinical outcomes. With a central database of data and blood samples, research may be accelerated to improve scientific understanding, including:

    ~ Which genetic changes influence joint disease?
    ~ Which variants increase the risk of inhibitor development?
    ~ Why does bleeding severity vary from person to person?
    ~ Which variants may be easier to “edit” (i.e., change or replace)?

    The seven approved research projects from the first MLOF Research Repository application cycle will investigate inhibitor development, bleeding specific to hemophilia A and B, as well as carriers, and F8 clearance.

  • When will results of the first MLOF Research Repository application cycle studies be shared? During the first application cycle, seven research projects were accepted based on scientific merit and level of benefit to those with bleeding disorders. Those projects will investigate inhibitor development, bleeding specific to hemophilia A and B, as well as carriers, and F8 clearance. For more information about progress made as a result of scientists’ access to the MLOF Research Repository, visit http://www.mylifeourfuture.org/research.html. We will continue to update this page with project details and other scientific publications related to the MLOF Research Repository as available.
  • How do I find out more about the My Life, Our Future Research Repository? A healthcare professional at your HTC can explain the process to you and help to answer any questions. You will also receive a consent form that explains your rights as a participant, and includes important details including the purpose, time involved, required testing, risks and potential benefits, and contact information. If you agree and sign the consent form, you are volunteering to take part in the My Life, Our Future Research Repository and any associated studies. After you sign the consent form, you will receive a copy for your records and reference. You can also view the MLOF consent form here.
  • How often will you draw my blood? You will only need to have your blood drawn once for genotyping. If you agree to submit your data to the My Life, Our Future Research Repository as well, you may be asked to contribute additional samples in case researchers use up the entire original sample or if your sample were to become too old to use. Requests for future blood draws will only come from your HTC, as they alone have your name and identifying information. You can always say no if you don't want to give more blood. Giving additional blood is strictly voluntary.
  • How long will it take to participate? Giving blood for genotyping usually takes less than 30 minutes. You will need to read information about the program, decide if you want to sign the ATHN patient authorization form (if you have not already done so), and have your blood drawn. If you agree to submit your data to the My Life, Our Future Research Repository as well, it will take approximately 20 additional minutes to read and sign the consent form, ask questions, and have your blood drawn. You are welcome to take the consent form home and think about it before participating. You can also view the MLOF consent form here.
  • Will it cost anything? Will I get paid? The genotyping test is offered free of charge, however your HTC may ask you to pay for costs associated with the HTC clinic visit, laboratory blood draw, or associated consultations (e.g., genetic counseling). These costs may vary among different centers. If you are interested in participating in MLOF, please speak with your HTC about these potential charges and how to potentially minimize or eliminate them prior to being genotyped. You will not be paid for your participation.
  • Can I change my mind if I say yes? It is voluntary to participate. At any time during the study, you can contact your HTC to request that your sample be withdrawn from the My Life, Our Future Research Repository. To ensure confidentiality, your HTC will provide Bloodworks Northwest with your MLOF identifier to help facilitate the sample removal. Your sample and data in the research repository will then be destroyed. No one will be upset with you if you say no, and you will continue to receive the same quality of care at your HTC.

    However, data submitted to the ATHNdataset and any samples already given to researchers will not be able to be pulled and/or destroyed. Information on this process is included in the informed consent form provided to you before you are genotyped.
  • If I've already been genotyped, can I still participate in My Life, Our Future? Yes, even if you've already been genotyped, we encourage you to participate in MLOF and contribute your data and sample to the My Life, Our Future Research Repository. With this enhanced database, scientific research may be accelerated to improve scientific understanding and potentially identify new treatment options.
  • How can I get more information about My Life, Our Future? We encourage you to learn more via an informational video and to sign up to receive updates from NHF. You may also contact the staff members at HANDI, NHF's information resource center, who are available to answer live requests from Monday through Friday, 9:00 AM to 5:00 PM Eastern Time, at 800.42.HANDI. Requests can also be sent by fax to 212.328.3799 or email to handi@hemophilia.org. Information requests made through HANDI are always kept confidential.
  • Where can I learn more about genotyping? In addition to HANDI, there are many resources available on the benefits of genetic testing:Genetic Alliance: A health advocacy organization committed to transforming health through genetics and promoting an environment of openness centered on the health of individuals, families, and communities.About Health Information Privacy: Background information from the U.S. Department of Health and Human Services on federal laws and regulations designed to protect your privacy.About the Genetic Information Nondiscrimination Act (GINA): Background information from the National Human Genome Research Institute of the National Institutes of Health on a federal law that protects against genetic discrimination in health insurance and employment.