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9. Real-World Amount of Clotting Factor Concentrates Dispensed and Annual Medical Expenditures for Japanese Patients with Hemophilia B

Author

Fukutake,Katsuyuki, Togo,Kanae, Xu,Linghua, Markson,Leona, Alvir,José Maria, Winburn,Ian, Karumori,Toshiyuki, Fukutake,Katsuyuki, Togo,Kanae, Xu,Linghua, Markson,Leona, Alvir,José Maria, Winburn,Ian, and Karumori,Toshiyuki

Abstract

Katsuyuki Fukutake,1,2 Kanae Togo,3 Linghua Xu,3 Leona E Markson,4 José Maria Jimenez Alvir,5 Ian Winburn,6 Toshiyuki Karumori3 1Laboratory Medicine, Tokyo Medical University, Shinjuku, Tokyo, Japan; 2Department of Blood Coagulation Diseases, Ogikubo Hospital, Suginami, Tokyo, Japan; 3Pfizer Japan Inc, Shibuya-ku, Tokyo, Japan; 4Pfizer Inc, Collegeville, PA, USA; 5Pfizer Inc, New York, NY, USA; 6Pfizer Ltd, Surrey, UKCorrespondence: Toshiyuki Karumori, Pfizer Japan Inc, Shinjuku Bunka Quint Bldg, 3-22-7, Yoyogi, Shibuya-ku, Tokyo, Japan, Tel +81-80-4152-4243, Email Toshiyuki.karumori@pfizer.comIntroduction: Until extended half-life (EHL) factor IX (FIX) concentrates became available in Japan in 2010, patients with hemophilia B received intravenous FIX replacement therapy with standard half-life (SHL) FIX concentrates.Purpose: To investigate the amount of factor dispensed and the associated medical expenditures for the treatment of hemophilia B in the real-world clinical setting in Japan.Methods: This retrospective study comprised patients with hemophilia B (N=197) who had filled prescriptions for FIX concentrates reported in Japan’s Medical Data Vision database from 2015 to 2019. Patients were included if they had 2 or more prescriptions for the same FIX concentrates within the first 6 months of the study period and the interval between prescriptions was at least 2 weeks.Results: Since 2015, there was a decrease in the proportion of patients using SHL FIX concentrates and a corresponding increase in international units of dispensed EHL FIX concentrates. Median annualized dispensed dosages (IU/kg body weight) of EHL FIX concentrates were lower than for SHL concentrates for outpatient use only. Annual total health care expenditures per patient and annual expenditures for prescribed FIX concentrates increased each year during the study period. Following a switch from an SHL to an EHL concentrate, the median amount of prescribed FIX concentrate decreased slightly, al

Published
2023

10. Real-World Amount of Clotting Factor Concentrates Dispensed and Annual Medical Expenditures for Japanese Patients with Hemophilia B.

Author

Fukutake, Katsuyuki, Togo, Kanae, Xu, Linghua, Markson, Leona E, Alvir, José Maria Jimenez, Winburn, Ian, and Karumori, Toshiyuki

Subjects
HEMOPHILIACS, JAPANESE people, BLOOD coagulation factor IX, MEDICAL care costs, BLOOD coagulation factors
Abstract

Introduction: Until extended half-life (EHL) factor IX (FIX) concentrates became available in Japan in 2010, patients with hemophilia B received intravenous FIX replacement therapy with standard half-life (SHL) FIX concentrates. Purpose: To investigate the amount of factor dispensed and the associated medical expenditures for the treatment of hemophilia B in the real-world clinical setting in Japan. Methods: This retrospective study comprised patients with hemophilia B (N=197) who had filled prescriptions for FIX concentrates reported in Japan's Medical Data Vision database from 2015 to 2019. Patients were included if they had 2 or more prescriptions for the same FIX concentrates within the first 6 months of the study period and the interval between prescriptions was at least 2 weeks. Results: Since 2015, there was a decrease in the proportion of patients using SHL FIX concentrates and a corresponding increase in international units of dispensed EHL FIX concentrates. Median annualized dispensed dosages (IU/kg body weight) of EHL FIX concentrates were lower than for SHL concentrates for outpatient use only. Annual total health care expenditures per patient and annual expenditures for prescribed FIX concentrates increased each year during the study period. Following a switch from an SHL to an EHL concentrate, the median amount of prescribed FIX concentrate decreased slightly, although median total health care expenditures and FIX concentrate expenditures increased. Conclusion: In the real-world setting in Japan, medical expenditures and the proportion of patients prescribed EHL FIX concentrates for the treatment of hemophilia B have increased. [ABSTRACT FROM AUTHOR]

Published
2023
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14. Examining patient and professional perspectives in the UK for gene therapy in haemophilia

Author

Woollacott, Ione, Morgan, George, Chowdary, Pratima, O'Hara, Jamie, Franks, Bethany, van Overbeeke, Eline, Dunn, Nicola, Michelsen, Sissel, Huys, Isabelle, Martin, Antony, Cawson, Matthew, Brownrigg, Jack, Winburn, Ian, Thomson, Jim, Woollacott, Ione, Morgan, George, Chowdary, Pratima, O'Hara, Jamie, Franks, Bethany, van Overbeeke, Eline, Dunn, Nicola, Michelsen, Sissel, Huys, Isabelle, Martin, Antony, Cawson, Matthew, Brownrigg, Jack, Winburn, Ian, and Thomson, Jim

Abstract

Introduction: With the development of gene therapy for people with haemophilia (PWH), it is important to understand how people impacted by haemophilia (PIH) and clinicians prioritise haemophilia treatment attributes to support informed treatment decisions. Objective: To examine the treatment attribute preferences of PIH and clinical experts in the United Kingdom (UK) and to develop a profile of gene therapy characteristics fit for use in future discrete choice experiments (DCEs). Methods: Semi-structured interviews were conducted with PIH (n = 14) and clinical experts (n = 6) who ranked pre-defined treatment attributes by importance. Framework analysis was conducted to identify key themes and treatment attributes; points were allocated based on the rankings. Synthesis of results by a multidisciplinary group informed development of a profile of gene therapy characteristics for use in future research. Results: Key themes identified by PIH and clinical experts included patient relevant features and the importance of ‘informed decision making'. The six top-ranked treatment attributes were ‘effect on factor level’ (79 points), ‘uncertainty regarding long-term risks’ (57 points), ‘impact on daily life’ (41 points), ‘frequency of monitoring’ (33 points), ‘impact on ability to participate in physical activity’ (29 points), and ‘uncertainty regarding long-term benefits’ (28 points). The final treatment characteristics were categorised as therapeutic option, treatment effectiveness, safety concerns, impact on self-management and quality of life (role limitations). Conclusion: We identified several gene therapy characteristics important to PIH and clinicians in the UK. These characteristics will be used in a future DCE to further investigate patient preferences for gene therapy., IMI-PREFER

Published
2022
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15. Examining patient and professional perspectives in the UK for gene therapy in haemophilia

Author

Woollacott, Ione, primary, Morgan, George, additional, Chowdary, Pratima, additional, O'Hara, Jamie, additional, Franks, Bethany, additional, van Overbeeke, Eline, additional, Dunn, Nicola, additional, Michelsen, Sissel, additional, Huys, Isabelle, additional, Martin, Antony, additional, Cawson, Matthew, additional, Brownrigg, Jack, additional, Winburn, Ian, additional, and Thomson, Jim, additional

Published
2022
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16. How to assess, detect, and manage joint involvement in the era of transformational therapies: Role of point‐of‐care ultrasound.

Author

Minno, Matteo Nicola Dario Di, Martinoli, Carlo, Pasta, Gianluigi, la Corte‐Rodriguez, Hortensia De, Samy, Islam, Stephensen, David, Timmer, Merel A., and Winburn, Ian

Subjects
HEMOPHILIA treatment, ULTRASONIC imaging, JOINTS (Anatomy), POINT-of-care testing, ANKLE
Abstract

Background: Patients with haemophilia experience recurring hemarthroses, mainly involving knees, elbows and ankles, which lead to haemophilic arthropathy, the major chronic complication of haemophilia. With new approaches to haemophilia treatment leading to fewer joint bleeds and, in some cases, no bleeding events, assessing whether current outcome assessment tools provide adequate sensitivity and specificity for management and care of patients with haemophilia is needed. Methods: An overview of current imaging tools for monitoring joint health, novel osteochondral damage and synovial proliferation biomarkers, and the relationship between assessments for functionality and imaging modalities is provided. Usefulness and sensitivity of point‐of‐care ultrasound (POCUS) to complement other assessments and use of ultrasound to monitor haemophilic arthropathy are also examined. Results: This review provides rationale for haemophilia teams to move beyond traditional outcomes in joint imaging, as well as guidance and evidence on assessment of joint health for potential new treatment modalities, such as gene therapy. The role of POCUS in the existing paradigm for haemophilia care and management along with the use of ultrasound as a complement to other outcome assessment tools are also discussed. Finally, the clinical effects of subclinical bleeding on joint function are described, to motivate screening for synovial proliferation. Conclusion: POCUS can facilitate the early detection of joint damage and can monitor disease progression while providing insights into the efficacy of treatment regimens, and should be considered as an essential assessment tool for managing the care of patients with haemophilia. [ABSTRACT FROM AUTHOR]

Published
2023
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17. Follow-up of More Than 5 Years in a Cohort of Patients with Hemophilia B Treated with Fidanacogene Elaparvovec Adeno-Associated Virus Gene Therapy

Author

Samelson-Jones, Ben J., primary, Sullivan, Spencer K., additional, Rasko, John E.J., additional, Giermasz, Adam, additional, George, Lindsey A., additional, Ducore, Jonathan M., additional, Teitel, Jerome M., additional, McGuinn, Catherine E., additional, O'Brien, Amanda, additional, Winburn, Ian, additional, Smith, Lynne M, additional, Chhabra, Amit, additional, and Rupon, Jeremy, additional

Published
2021
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22. A plain language summary of AAV-based gene therapy: challenges and potential solutions for people with antibodies against AAV

Author

Moroz, Tara, Schulz, Martin, Levy, Daniel I., Bashirians, George, Winburn, Ian, Mahn, Matthias, Somanathan, Suryanarayan, Cheng, Seng H., Trantham, Shandra J., and Byrne, Barry J.

Abstract

What is this article about?This is a summary of a review article about gene therapy. Review articles summarize many previously published scientific articles. The review was about how a common viruscould change how one type of gene therapy works. It also discussed the importance of antibody testing for people who may receive gene therapy. The original review was published in Molecular Therapyin 2023.Gene therapy is a type of medical treatment. The goal of a specific gene therapy is to treat a specific genetic condition. A genetic condition is a health problem caused by a variation (change or mutation) in a geneor genes. Genes are instructions that tell the body how to grow and work.Some genetic conditions can cause rare diseases. A rare disease affects only a very small percentage of people. Gene therapy may help people with some rare diseases because it aims to correct the root cause of the condition instead of just treating symptoms.Gene therapies work by getting into the cellsin a person's body. Many gene therapies are modeled after viruses because viruses are good at getting into the body.One of the viruses used in gene therapy is called adeno-associated virus(AAVfor short). This naturally occurring virus infects many people. People can get infected without realizing it because AAV doesn't typically make a person sick.After being infected with a virus, people make antibodiesto fight off that virus. These antibodies can prevent another infection. Antibodies that fight off natural AAV may prevent gene therapy based on AAV from working as it should.A common reason why people may not be able to receive gene therapy is because they have antibodies against AAV. This means that some people with some genetic conditions or rare diseases may not be able to benefit from gene therapy. Researchers are trying to understand how antibodies against AAV can prevent gene therapy from working. They are also studying what side effectsthey may cause. This research is important for people with a genetic condition or rare disease that may be treated with gene therapy.What conclusions did we draw?People who have a certain level of antibodies against AAV might not be able to take part in gene therapy clinical trials. They might not be able to receive AAV-based gene therapies that have been approved for commercial use by a regulatory agency such as the FDA (Food and Drug Administration) or EMA (European Medicines Agency).For most gene therapies, people need to be tested for antibodies against AAV.There are two different types of tests that measure if someone has antibodies against AAV.Researchers are looking at ways to lower the effects of antibodies against AAV. This research could allow more people to have gene therapy in the future.

Published
2024
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25. World Federation of Hemophilia Gene Therapy Registry.

Author

Konkle, Barbara A., Coffin, Donna, Pierce, Glenn F., Clark, Cary, George, Lindsey, Iorio, Alfonso, Mahlangu, Johnny, Naccache, Mayss, O'Mahony, Brian, Peyvandi, Flora, Pipe, Steve, Quartel, Adrian, Sawyer, Eileen K., Skinner, Mark W., Tortella, Bartholomew, Watson, Crystal, and Winburn, Ian

Subjects
GENE therapy, INTERNATIONAL organization, HEMOPHILIA, MEDICAL personnel, RECOMBINANT DNA
Published
2020
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27. How to assess, detect, and manage joint involvement in the era of transformational therapies: Role of point-of-care ultrasound

Author

Matteo Nicola Dario Di Minno, Carlo Martinoli, Gianluigi Pasta, Hortensia De la Corte‐Rodriguez, Islam Samy, David Stephensen, Merel A. Timmer, Ian Winburn, DI MINNO, Matteo, Martinoli, Carlo, Pasta, Gianluigi, De la Corte‐Rodriguez, Hortensia, Samy, Islam, Stephensen, David, Timmer, Merel A., and Winburn, Ian

Subjects
joint disease, ultrasound, haemarthrosi, haemophilia A, Hematology, General Medicine, haemorrhage, Genetics (clinical)
Abstract

Background: Patients with haemophilia experience recurring hemarthroses, mainly involving knees, elbows and ankles, which lead to haemophilic arthropathy, the major chronic complication of haemophilia. With new approaches to haemophilia treatment leading to fewer joint bleeds and, in some cases, no bleeding events, assessing whether current outcome assessment tools provide adequate sensitivity and specificity for management and care of patients with haemophilia is needed. Methods: An overview of current imaging tools for monitoring joint health, novel osteochondral damage and synovial proliferation biomarkers, and the relationship between assessments for functionality and imaging modalities is provided. Usefulness and sensitivity of point-of-care ultrasound (POCUS) to complement other assessments and use of ultrasound to monitor haemophilic arthropathy are also examined. Results: This review provides rationale for haemophilia teams to move beyond traditional outcomes in joint imaging, as well as guidance and evidence on assessment of joint health for potential new treatment modalities, such as gene therapy. The role of POCUS in the existing paradigm for haemophilia care and management along with the use of ultrasound as a complement to other outcome assessment tools are also discussed. Finally, the clinical effects of subclinical bleeding on joint function are described, to motivate screening for synovial proliferation. Conclusion: POCUS can facilitate the early detection of joint damage and can monitor disease progression while providing insights into the efficacy of treatment regimens, and should be considered as an essential assessment tool for managing the care of patients with haemophilia.

Published
2022

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