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  • Treatment Breakthroughs
    attach them to the chemical compound polyethylene glycol PEG This process called PEGylation increases the size of the factor protein molecule so that it circulates in the blood longer and is not cleared by the kidneys prematurely Another strategy is to fuse the recombinant factor protein molecule to a partner protein that already has a long half life says Pipe Two naturally occurring partner proteins being fused to the FVIII or FIX molecule are albumin which moves small molecules through the bloodstream and Fc a protein fragment that facilitates binding and recycling of immunoglobulin G IgG Data from early clinical trials on Biogen Idec s recombinant FVIII and FIX Fc fusion products rFVIIIFc and rFIXFc look promising The A LONG study on patients with severe hemophilia A showed a 1 7 fold increase in half life during phase 1 2a clinical trials B LONG studies on patients with severe hemophilia B showed a nearly threefold increase in half life during phase 1 2 trials See Long Strides HemAware Summer 2011 p 14 Adjunctive therapies or drugs that are added to the primary factor product are also being tested in clinical trials Some use molecules that bind to tissue factor pathway inhibitor TFPI preventing it from hindering the action of FXa and thrombin necessary for clot initiation and formation Baxter s BAX513 uses fucoidan a seaweed extract being tested on healthy volunteers without hemophilia If you block the proteins that are slowing down coagulation you can actually restore normal clotting in hemophilic plasma without replacing the missing clotting factor says Pipe For some patients the adjunctive therapy may become the primary therapy reducing the number of infusions needed he says A bonus is that some TFPI antagonists could be taken orally such as the capsule form that delivered fucoidan to trial subjects Compliance with bleeding disorders treatment is always an issue says Bias A drug that works better faster and that you have to take less often can only improve that Innovations for Inhibitors An estimated 25 of patients with severe hemophilia A develop antibodies called inhibitors to the infused factor Currently patients undergo immune tolerance therapy to desensitize their immune systems or take a bypassing agent such as FVIIa The main drawbacks of the recombinant FVIIa product are that its half life is only two hours and it is very expensive Inspiration Biopharmaceuticals is developing a recombinant porcine pig FVIII product for patients with inhibitors You can give a dose and get the measurable level of FVIII That s a distinct advantage when there s a life threatening bleed like a head bleed intracranial hemorrhage or a limb threatening bleed in someone with a compartment syndrome increased pressure in a muscle in an enclosed space Koerper says But because 80 of patients developed antibodies to plasma derived pig factor within five days or after five doses it is possible that a similar scenario might occur with the recombinant product Results of the clinical trials will provide more data but its

    Original URL path: http://www.hemaware.org/print/819 (2016-02-18)
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  • Treatment Breakthroughs | HemAware
    developing a recombinant porcine pig FVIII product for patients with inhibitors You can give a dose and get the measurable level of FVIII That s a distinct advantage when there s a life threatening bleed like a head bleed intracranial hemorrhage or a limb threatening bleed in someone with a compartment syndrome increased pressure in a muscle in an enclosed space Koerper says But because 80 of patients developed antibodies to plasma derived pig factor within five days or after five doses it is possible that a similar scenario might occur with the recombinant product Results of the clinical trials will provide more data but its use will probably be restricted The longer lasting products may have an added benefit for inhibitor patients Some forms of PEGylation strategy and possibly even some of the fusion proteins may result in reduced risk for inhibitors says Pipe Another product now being tested Octapharma s recombinant human cl rhFVIII may reduce the rate of inhibitor development because it uses proteins from human cells not the typical hamster cells Recombinant VWD Product at Last Recombinant products to treat FVIII and FIX were approved in 1992 and 1998 respectively not so for von Willebrand factor VWF It has bothered me for almost 20 years that I couldn t offer a recombinant VWF product to my VWD patients says Koerper That need will be fulfilled once Baxter s recombinant VWF product goes through FDA approval and licensure It will be targeted to patients with type 3 VWD the most severe form and those unresponsive to DDAVP a synthetic hormone used to prevent or stop bleeds Gene Therapy Revisited Researchers can now create precision drugs that treat diseases caused by specific genetic mutations One such drug in phase 2 trials is Ataluren PTC 124 manufactured by PTC Therapeutics Inc It will be used for the approximately 10 15 of patients with hemophilia A and B with a nonsense mutation which halts factor production early Ataluren introduces a molecule that allows the cell to read through the stop signal making more clotting factor It comes in a powder that is mixed in water Something that you can swallow is going to be a huge advantage because there are no needles involved Koerper says See sidebar The Allure of Ataluren in What s Your Genotype HemAware Spring 2010 p 29 Rare Bleeding Disorders on the Radar Patients with rare factor deficiencies know that being one in a million is hardly a cause for celebration People forget that there are other clotting factor deficiencies that in some cases have no treatment says Bias But hope is on the horizon Companies that fractionate or separate plasma are interested in getting as many products out of it as they can says Pipe Developing new markets for new plasma derivatives such as the new FXIII product Corifact approved by the FDA in March 2011 and RiaSTAP a fibrinogen concentrate to treat FI deficiency indicated for patients with congenital fibrinogen deficiency including afibrinogenemia and

    Original URL path: http://www.hemaware.org/story/treatment-breakthroughs?quicktabs_story_page_tabs=0 (2016-02-18)
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  • Banding Together | HemAware
    Fund NHF Judith Graham Pool Research Fellowship The fellowship was established in 2013 In contrast money raised by walks typically goes to chapters which then decide how to use it The Cirellis didn t name the fellowship after their son for recognition They did it so that one day Nicholas can look back and say My parents did something to help me something tangible Like many parents of kids with hemophilia or another bleeding disorder the roots of the Cirellis advocacy efforts stem from a terrifying day in the emergency room Nicholas was five days old and the incision site from his circumcision hadn t stopped bleeding Erin remembers thinking He s going to bleed to death After waiting for hours a pediatrician took one look at Nicholas blood soaked diaper and the staff sprang into action It was like out of a movie says Erin Two nurses ran in and rushed Nicholas to the NICU The baby needed two blood transfusions to restore what he d lost through hemorrhaging This is what led us to believe that we needed to be advocates for Nicholas Erin says If we hadn t it could have had dire consequences That belief underlies everything the Cirellis do for the walk and for hemophilia research Doing the walk helps me regain some control says Erin The Philanthropist Donald Colburn used to say I d love to put myself out of business Colburn co owner with his wife Kathy Ann Keenan of American Homecare Federation Inc AHF had something in common with his customers He had severe hemophilia A His business based in Enfield Connecticut catered exclusively to the bleeding disorders community Unfortunately the Colburns passed away in 2006 But they left a directive to the foundation they d set up in their name They wanted the money they d made through their business to go back to support people who had bleeding disorders and other health problems Through the foundation Donald and Kathy Ann wanted to help people with bleeding disorders thrive in every part of their lives says Jane Cavanaugh Smith a member of the board of directors of the Colburn Keenan Foundation The Colburns had a firm grasp on the major issues facing the bleeding disorders community They understood the financial burden put on families living with an expensive chronic illness They believed strongly in the benefit of summer camp and the importance of a good education says Cavanaugh Smith They wanted to help provide for a good life today and hopefully an even better life in the future That dream included finding a cure The Colburns bequest with an operational budget of about 400 000 is distributed as direct financial grants to individuals and college scholarships for students with hemophilia It also funds organizations that support the needs of people with bleeding disorders and other chronic illnesses The bequest also provides for the medical needs of people with bleeding disorders That s where the JGP Research Fellowship gift comes in The

    Original URL path: http://www.hemaware.org/story/banding-together-hemophilia-research (2016-02-18)
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  • Genotyping for Progress | HemAware
    increase funding for research for bleeding disorders and facilitate genotyping but it was not enacted In 2005 the Centers for Disease Control and Prevention provided access to genotyping to a subset of patients as part of a study of inhibitors but was unable to offer a national genotyping program due to logistical and funding challenges My Life Our Future has been made possible through the collaboration of four leaders in the hemophilia community each of which plays a crucial role in the program ATHN a not for profit organization representing more than 130 hemophilia treatment centers HTCs is ensuring the collection and secure storage of data and preparing HTCs to implement the service NHF is educating patients and the community about the program through its chapter network and resources PSBC a state of the art laboratory and research institution with expertise in hemophilia mutation analysis is conducting the secure genetic testing and analysis Biogen Idec Hemophilia a biotechnology company committed to empowering people with hemophilia is providing funding and scientific expertise for the partnership through at least 2014 Thereafter the project may be extended to provide greater access to those affected by hemophilia In November 2012 MASAC adopted a recommendation that all individuals with hemophilia and their affected family members participate in My Life Our Future My Life Our Future How It Works The goal of My Life Our Future is to genotype as many people in the US affected by hemophilia as possible To ensure a smooth national rollout the program is being piloted in spring 2013 in at least 10 HTCs that will offer free or very low cost genotyping to existing patients Once the results from the pilot sites have been reviewed and any needed adjustments made the genotyping service will be expanded to other HTCs across the country Plans are to eventually expand the service to potential carriers Science Photo Library Glow Images During the pilot phase and after individuals with hemophilia A or B who want to access the genotyping service will only need to visit their participating HTC for a blood test The HTC will send the blood sample to PSBC s specialized hemophilia laboratory for genetic analysis PSBC will then send a clinical report summarizing the test results to the HTC where the test was done The patient s treating physician will then share the screening test results privately with his or her patient This report will be stored in the individual s HTC record and in PSBC s secure laboratory information system The clinical report will not be shared with insurers employers or other parties It will be up to patients whether they want to share the report with others including family members The program will adhere to best practices and all state and federal laws designed to protect the privacy of patients including the Health Insurance Portability and Accountability Act HIPAA and the 2008 Genetic Information Nondiscrimination Act GINA Furthering Scientific Research for Future Generations In addition to receiving results that

    Original URL path: http://www.hemaware.org/story/genotyping-progress (2016-02-18)
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  • Hemophilia Treatment Centers 101 | HemAware
    Some HTCs are portable serving patients via temporary traveling clinics in local hospitals before moving on If you re planning a trip your HTC staff can contact their counterparts in other parts of the country or world to let them know you ll be traveling there and may need assistance Education Emphasis HTCs provide more than just treatment Cohen says One of the major roles of the HTCs is education she explains When children begin prophylaxis treatment for example HTCs will teach parents and caregivers and eventually the children how to infuse themselves so they can treat at home This can save time reduce discomfort and increase convenience during a bleed because patients don t have to travel to an emergency room and wait she adds Steps for Living Treat Responsibly Today for a Healthy Tomorrow The educational role extends beyond the patient and family HTC staff often visit schools explaining to teachers staff and school nurses what hemophilia is what activities a child can engage in and how to recognize an emergency This comprehensive approach to care does more than improve the quality of life for people with bleeding disorders According to studies by the CDC people with hemophilia are 40 less likely to be hospitalized for bleeding complications if they use HTCs rather than receiving care elsewhere see Learn More Those who use HTCs are also 40 less likely to die of hemophilia related complications The comprehensive care approach is so effective that the federal government is considering HTCs as a model for care for diabetes sickle cell anemia and other diseases The new buzzword on Capitol Hill is medical homes something individuals with bleeding disorders have long enjoyed Psychosocial Needs HTCs also address psychosocial needs We re there to make sure the whole person is taken into account all the time says Mary Jane Berry MSW a clinical social worker at the Georgetown University Hemophilia and Thrombosis Center in Washington DC During the comprehensive care clinic appointment Berry schedules a short visit with each family to introduce herself and ask if the family needs help with insurance matters She also screens patients about their coping strategies pain management and any job related marital or other problem If parents are worried about how to manage their insurance copayment for example Berry can connect them to their state s Children s Health Insurance Program If a marriage is in trouble she can provide basic counseling Berry also meets with siblings focusing on such issues as sibling rivalry guilt or caregiving In addition HTC social workers can provide referrals to various helpful agencies in the community Berry also sees patients and families during their routine visits in the clinic for medical care I poke my head in and ask a few questions which breaks the ice Berry says Maybe they don t need anything But if there s a job change or an insurance change or something else comes up then they know my face HTC social workers can also

    Original URL path: http://www.hemaware.org/story/hemophilia-treatment-centers-101 (2016-02-18)
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  • Research Progress Helps Entire Community | HemAware
    dwindling federal research support Although researchers receive money from various government sources federally funded research into bleeding disorders still does not receive sufficient priority based on the impact our conditions have on the federal budget Further basic science research such as that funded by the National Hemophilia Foundation s NHF s Judith Graham Pool JGP Fellowship Award and the Career Development Award CDA drives innovation See Banding Together page 20 Without the support of foundations such as NHF some of our most exciting clinically significant new products might never have moved from the drawing board to the lab and to the pharmacy To evaluate the success of a funding source look no further than the impact of those who have received career funding from NHF Seven current hemophilia treatment center HTC physicians have been awarded the JGP Fellowship Award Another 17 awardees have contributed to gene therapy trials and 14 have contributed to novel factor therapies The CDA has been equally successful in engaging researchers Five current HTC physicians have received support 12 have contributed to gene therapy trials and four have contributed to novel factor therapies This collaboration has already had huge payoffs And together we can reach our common goal the cure Michael Recht MD PhD is director of the Hemophilia Center at Oregon Health Science University Doernbecher Children s Hospital in Portland He is regional director for the federally funded Mountain States Hemophilia Network View the discussion thread Life Stages Infants Children Teens Young Adults Adults Aging Research Treatment Hemophilia A B von Willebrand Disease Rare Bleeding Disorders HIV Hepatitis C Surgery Transplants Researcher Profiles Health Wellness Fitness Nutrition Taking Charge Pain Management Parenting Family Parenting Family Hemaware Jr Women Women s Bleeding Disorders Carriers Watchdog Insurance Blood Safety Advocacy Community HemAware Blogs HemAware Videos HemAware Photos

    Original URL path: http://www.hemaware.org/story/research-progress-helps-entire-community (2016-02-18)
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  • Send page by email | HemAware
    Fitness Nutrition Taking Charge Pain Management Parenting Family Parenting Family Hemaware Jr Women Women s Bleeding Disorders Carriers Watchdog Insurance Blood Safety Advocacy Community HemAware Blogs HemAware Videos HemAware Photos Chapters in Action Global Spotlight Community Voices En Español Need info Ask NHF Contact HANDI NHF s resource center for additional information on bleeding disorders Home Glossary Site Map Donate Advertise Privacy Policy Subscribe Contact Us Life Stages Infants Children

    Original URL path: http://www.hemaware.org/printmail/885 (2016-02-18)
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  • The Carrier Barrier
    symptoms bruising nosebleeds heavy menstrual periods menorrhagia and prolonged bleeding after trauma or dental or surgical procedures can be missed or dismissed in girls and women Danielle and Heather Schwager 25 twins from Strongsville Ohio had symptoms during childhood We were kids who grew up playing outside all the time and we were always bruised says Danielle The twins mother Vickie a neonatal nurse practitioner periodically questioned the pediatrician wondering if her daughters had hemophilia B like her father He kept telling me No females don t have hemophilia Vickie says Five years later Danielle bled for 10 days after having four teeth pulled Her oral surgeon and orthodontist sensed something amiss and referred the family to the hemophilia treatment center HTC in Cleveland Factor level testing confirmed Vickie s hunch My factor level was about 8 or 9 when I was diagnosed with mild hemophilia B says Danielle Heather s was about 40 Those diagnoses took more than a decade During NHF s Annual Meeting in Chicago in November 2011 Koerper and Vickie conducted an educational session for women with mild hemophilia Many women recounted similar experiences These women know they re bleeding too much Koerper says But their doctors disregarded their concerns saying You got a big bruise because you banged your leg Problems result when a surgery is performed without a treatment plan They ll have their gallbladder out and then they have bleeding complications says Koerper Davenport warned her new hematologist who was inexperienced in treating women with bleeding disorders that her factor level might soar from the stress she was feeling before a hysterectomy but then would plummet afterward Because her factor level skyrocketed to 80 the hematologist took no precautions Davenport woke up in the recovery room hearing her doctor tell the staff I don t know what s causing her bleeding She doesn t have hemophilia When they call you a symptomatic carrier and that s your label you tend not to be treated seriously Davenport says For her that resulted in post op bleeding For others it might mean joint damage and arthritis from untreated bleeds or serious complications following childbirth Emotional and Behavioral Toll The emotional and behavioral consequences for women with mild hemophilia who perceived errors in their medical care were documented in a 2011 study in Haemophilia by Nisa Renault and researchers at Dalhousie University in Halifax Nova Scotia Canada The 11 women interviewed cited 264 negative emotional responses including anger doubt and mistrust A doctor s disbelief can lead to a patient s disillusionment It is frustrating to try to explain this to physicians who I would expect to know something about bleeding disorders says Vickie Davenport was devastated when her doctor denied that she had hemophilia after her hysterectomy It was very hard on me to doubt the fact that maybe I believed this for 35 years and it just wasn t right she says The pain went deep as Davenport lost a comforting family connection That

    Original URL path: http://www.hemaware.org/print/885 (2016-02-18)
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