Episodios

  • Lived Experience Experts: Taking Their Rightful Place at the Research Table

    Lived Experience Experts: Taking Their Rightful Place at the Research Table
    Jun 27 2024

    In this episode of the Global Hemophilia Report, we delve into the crucial role of Lived Experience Experts (LEEs) in hemophilia research. Featuring a distinguished panel, the discussion covers the evolution, importance, and future of LEE involvement in research. The episode emphasizes the pivotal contributions of LEEs in making research more relevant and impactful, driven by personal and community experiences.

    Contributors:

    Len Valentino MD

    Samantha Carlson MSW, LMSW

    Randy Curtis

    Ray Stanhope

    Kyle Davis MD

    Senior Advisor: Donna DiMichele, MD

    Special Episode Advisor: Maria Santaella PhD(c), MSN, RN-BC, CPHON

    Hosted & Written by: Patrick James Lynch

    Featured Advertiser:

    Sanofi

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    Show Notes:

    Presenting Sponsor: Sanofi

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    50 m
  • Caregivers and Novel Therapies - Alleviating or Replacing Concerns?

    Caregivers and Novel Therapies - Alleviating or Replacing Concerns?
    May 30 2024

    Our panel of subject matter and lived experience experts engage in a data-driven discussion about caregiver burden in hemophilia, historically, and how the rapidly evolving treatment landscapes impacts that burden.



    Contributors:

    Michelle Witkop, DNP, FNP-BC

    Kate Khair, PhD

    Beatriz Caceres, MD

    Kasha Lumsden, BSN, RN, RAC-CT

    Yasmin Pavri

    Senior Advisor: Donna DiMichele, MD

    Special Episode Advisor: Michelle Witkop, DNP, FNP-BC

    Hosted & Written by: Patrick James Lynch

    Featured Advertiser:

    Sanofi

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    Show Notes:

    Presenting Sponsor: Sanofi

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    40 m
  • World Hemophilia Day

    World Hemophilia Day
    Apr 26 2024

    On this episode of the Global Hemophilia Report, host Patrick James Lynch is on location in Madrid, Spain for the WFH 2024 World Congress. Patrick talks about the history of World Hemophilia Day and talks with some of the attendees at the congress.

    Contributors:

    Hazri Aris

    Johnny Mahlangu

    Rebecca, Hematology Resident

    Senior Advisor: Donna DiMichele, MD

    Hosted & Written by: Patrick James Lynch

    Featured Advertiser:

    Sanofi

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    Show Notes:

    Presenting Sponsor: Sanofi

    Subscribe to the Global Hemophilia Report

    Connect with the Global Hemophilia Report

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    26 m
  • Translating Patient-Reported Clinical Outcomes into Patient-Informed Clinical Research Outcomes

    Translating Patient-Reported Clinical Outcomes into Patient-Informed Clinical Research Outcomes
    Mar 28 2024

    In this episode, delve into the transformative journey of patient-reported outcomes measures (PROMs) within hemophilia care, exploring their evolution from broad assessments of health-related quality of life to nuanced evaluations of physical ability, functionality, and psychological impact. Learn about pioneering instruments like the Hemo-TEM and the PROBE questionnaire, designed to capture the multifaceted burdens of hemophilia treatment and the patient experience. However, as the landscape of PROMs continues to evolve, discover lingering debates around their sufficiency compared to standardized measures like PROMIS, and the ongoing quest to ensure their responsiveness to change in evaluating novel therapeutics and treatment paradigms. Join us as we unravel the complexities of translating patient-reported clinical outcomes into patient-informed clinical research outcomes.

    Contributors:

    Mark Skinner, JD

    Brian O'Mahony

    Bradley Rayner

    Dawn Rotellini

    Mosi Williams, LCSW, MSW, PsyD

    Senior Advisor: Donna DiMichele, MD

    Hosted & Written by: Patrick James Lynch

    Featured Advertiser:

    Sanofi

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    Show Notes:

    Presenting Sponsor: Sanofi

    Subscribe to the Global Hemophilia Report

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    51 m
  • Understanding Factor VIII & IX - In Coagulation and Beyond - How Much Are We Still Missing?

    Understanding Factor VIII & IX - In Coagulation and Beyond - How Much Are We Still Missing?
    Mar 15 2024

    The topic of discussion for this episode leans more toward the scientific side but has huge clinical relevance. For today, we will be asking our expert panel to lend their experience and expertise to the topic of FVIII and FIX, which we will be discussing from several related perspectives.

    Contributors:

    Radek Kaczmarek, PhD, MSc; University of Indiana, USA

    Peter Lenting, PhD; INSERM, Paris, France

    Ben Samuelson -Jones, M.D., PhD; CHOP Research Institute, USA

    Courtney Thornburg, M.D., MS; Rady Children’s Hospital, UCSD, USA

    Senior Advisor: Donna DiMichele, MD

    Hosted & Written by: Patrick James Lynch

    Featured Advertiser:

    Sanofi

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    Show Notes:

    Presenting Sponsor: Sanofi

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    49 m
  • Hemophilia Gene Therapy Redux – The End of the Beginning?

    Hemophilia Gene Therapy Redux – The End of the Beginning?
    Jan 25 2024
    Gene therapy for hemophilia A and B now exists as a licensed, prescribable treatment option for patients in certain countries; however, many questions and challenges remain. Senior advisor Dr. Donna DiMichele and patient-host Patrick James Lynch speak to global KOLs about the current state of hemophilia A and B gene therapies, both commercially and investigationally.   Contributors: Lindsey A. George, MD Margareth Ozelo, MD Steven Pipe, MD   Senior Advisor: Donna DiMichele, MD   Hosted & Written by: Patrick James Lynch   Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report   Notes and References:   Nathwani: Hematology Am Soc Hematol Educ Program . 2022 Dec 9;2022(1):569-578.   The current state of this exciting and rapidly evolving field, as well as the challenges that need to be overcome for the widespread adaptation of this new treatment paradigm, is the subject of this review.   Pipe: N Engl J Med. 2023 Feb 23;388(8):706-718   The annualized bleeding rate decreased from 4.19 (95% confidence interval [CI], 3.22 to 5.45) during the lead-in period to 1.51 (95% CI, 0.81 to 2.82) during months 7 through 18 after treatment, for a rate ratio of 0.36 (95% Wald CI, 0.20 to 0.64; P<0.001), demonstrating noninferiority and superiority of etranacogene dezaparvovec as compared with factor IX prophylaxis. Factor IX activity had increased from baseline by a least-squares mean of 36.2 percentage points (95% CI, 31.4 to 41.0) at 6 months and 34.3 percentage points (95% CI, 29.5 to 39.1) at 18 months after treatment, and usage of factor IX concentrate decreased by a mean of 248,825 IU per year per participant in the post-treatment period (P<0.001 for all three comparisons). Benefits and safety were observed in participants with predose AAV5 neutralizing antibody titers of less than 700. No treatment-related serious adverse events occurred.   Long-Term Effects of Hemophilia B Gene Therapy. Makris M.N Engl J Med. 2023 May 18;388(20):1918.    Long-Term Effects of Hemophilia B Gene Therapy. Reply. Pipe SW, Monahan PE.N Engl J Med. 2023 May 18;388(20):1918-1919   Mahlangu: N Engl J Med. 2023 Feb 23;388(8):694-705   Results: At week 104, a total of 132 participants, including 112 with data that were prospectively collected at baseline, remained in the study. The mean annualized treated bleeding rate decreased by 84.5% from baseline (P<0.001) among the participants. From week 76 onward, the trajectory of the transgene-derived factor VIII activity showed first-order elimination kinetics; the model-estimated typical half-life of the transgene-derived factor VIII production system was 123 weeks (95% confidence interval, 84 to 232). The risk of joint bleeding was estimated among the trial participants; at a transgene-derived factor VIII level of 5 IU per deciliter measured with chromogenic assay, we expected that participants would have 1.0 episode of joint bleeding per year. At 2 years post infusion, no new safety signals had emerged and no new serious adverse events related to treatment had occurred   Miesbach : Dtsch Arztebl Int. 2022 Dec 27;119(51-52):887-894.   Data from non-randomized phase 1 to phase 3 trials reveal an adequate expression of factors VIII and IX in patients with mostly severe hemophilia A or B. Even though they were no longer receiving prophylactic treatment, most patients experienced a considerable reduction, by 53% to 96%, in the number of bleedings compared to previous therapy. Persistently elevated factor levels have been described for up to six years in hemophilia A and up to eight years in hemophilia B. The most common side effect of gene therapy is an inflammatory response with elevated alanine aminotransferase levels (17% to 89%, depending on the study), which may be associated with a reduced clotting factor level and requires treatment with transient immunosuppression. Gene therapy for hemophilia holds out the prospect of freedom from hemorrhage without the need for regular treatment with drugs. The various steps that need to be carried out in gene therapy should be coordinated in a graded and partly overlapping integrated care model (a so-called hub-and-spoke model). Electronic platforms should be used for data acquisition and transmission.   Samuelson Jones & George: Annu Rev Med. 2023 Jan 27:74:231-247.   Importantly, despite repeated proof-of-concept success in current hemophilia gene therapy, stable, durable FVIII or FIX expression able to ameliorate bleeding in all patients is an unrealized hope. This defines the development goals of the next generation of gene-based therapies for hemophilia.   *Valentino et al: J Thromb Haemost. 2023 Sep;21(9):2354-2361   In 2022, the National Hemophilia Foundation submitted a citizen petition (docket number FDA-2022-P-1444-0001) to the FDA requesting that a risk evaluation and mitigation strategy (REMS) be required as a condition of approval for both valoctocogene roxaparvovec and etranacogene dezaparvovec. Other ...
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    1 h y 1 m
  • PTSD & Hemophilia

    PTSD & Hemophilia
    Dec 28 2023

    Join us as we shed light on the often-overlooked intersection of Post Traumatic Stress Disorder (PTSD) and Hemophilia, discussing innovative treatments, personal stories, and the journey towards understanding and managing these complex disorders.

     

    Contributors:

    Stephen W. Porges, PhD

    Debbie de la Riva

    Amanda Stahl, MSW, LICSW

    Justin Levesque

     

    Senior Advisor: Donna DiMichele, MD

     

    Hosted by: Patrick James Lynch

    Written by: Simantini Karve, PhD

     

    Show Notes:

    Presenting Sponsor: Sanofi

     

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    55 m
  • The US National Research Blueprint (NRB)

    The US National Research Blueprint (NRB)
    Nov 30 2023

    The US National Research Blueprint (NRB). Thriving in the Face of an Inheritable Bleeding Disorder Begins with Community-Inspired Research. 

     

    Contributors:

    Len Valentino, MD

    Keri Norris, MD

    Maria Santaella, MD

    Michael Recht, MD

    Sammie Valadez



    Senior Advisor: Donna DiMichele, MD

     

    Hosted by: Patrick James Lynch

    Written by: Simantini Karve, PhD

     

    Show Notes:

    Presenting Sponsor: Sanofi

     

    Subscribe to the Global Hemophilia Report

     

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    Global Hemophilia Report on LinkedIn

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    1 h