30 episodes

Led by science, curiosity, and storytelling, the Global Hemophilia Report from BloodStream Media is an entertaining monthly journey through the investigations and science driving hemophilia research around the world.

Produced by: Believe Limited & BloodStream Media
Hosted by: Patrick James Lynch
Senior Advisor: Dr. Donna DiMichele
Featured Advertiser: Sanofi Genzyme

#GHRpod #hemophilia #clinicalresearch #bloodstreammedia

Global Hemophilia Report BloodStream Media

    • Health & Fitness
    • 5.0 • 10 Ratings

Led by science, curiosity, and storytelling, the Global Hemophilia Report from BloodStream Media is an entertaining monthly journey through the investigations and science driving hemophilia research around the world.

Produced by: Believe Limited & BloodStream Media
Hosted by: Patrick James Lynch
Senior Advisor: Dr. Donna DiMichele
Featured Advertiser: Sanofi Genzyme

#GHRpod #hemophilia #clinicalresearch #bloodstreammedia

    Caregivers and Novel Therapies - Alleviating or Replacing Concerns?

    Caregivers and Novel Therapies - Alleviating or Replacing Concerns?

    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
     
    Subscribe to the Global Hemophilia Report
     
    Show Notes:
    Presenting Sponsor: Sanofi
     
    Subscribe to the Global Hemophilia Report
     
    Connect with the Global Hemophilia Report
    Global Hemophilia Report on LinkedIn
    Global Hemophilia Report on Twitter
    Global Hemophilia Report on Facebook
     
    Connect with BloodStream Media:
    BloodStreamMedia.com
    BloodStream on Facebook 
    BloodStream on Twitter 
     

    • 40 min
    World Hemophilia Day

    World Hemophilia Day

    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
     
    Subscribe to the Global Hemophilia Report
    Show Notes:
    Presenting Sponsor: Sanofi
     
    Subscribe to the Global Hemophilia Report
     
    Connect with the Global Hemophilia Report
    Global Hemophilia Report on LinkedIn
    Global Hemophilia Report on Twitter
    Global Hemophilia Report on Facebook
     
    Connect with BloodStream Media:
    BloodStreamMedia.com
    BloodStream on Facebook 
    BloodStream on Twitter

    • 25 min
    Translating Patient-Reported Clinical Outcomes into Patient-Informed Clinical Research Outcomes

    Translating Patient-Reported Clinical Outcomes into Patient-Informed Clinical Research Outcomes

    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
     
    Subscribe to the Global Hemophilia Report
     
    Show Notes:
    Presenting Sponsor: Sanofi
     
    Subscribe to the Global Hemophilia Report
     
    Connect with the Global Hemophilia Report
    Global Hemophilia Report on LinkedIn
    Global Hemophilia Report on Twitter
    Global Hemophilia Report on Facebook
     
    Connect with BloodStream Media:
    BloodStreamMedia.com
    BloodStream on Facebook 
    BloodStream on Twitter 
     

    • 50 min
    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?

    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
     
    Subscribe to the Global Hemophilia Report
     
    Show Notes:
    Presenting Sponsor: Sanofi
     
    Subscribe to the Global Hemophilia Report
     
    Connect with the Global Hemophilia Report
    Global Hemophilia Report on LinkedIn
    Global Hemophilia Report on Twitter
    Global Hemophilia Report on Facebook
     
    Connect with BloodStream Media:
    BloodStreamMedia.com
    BloodStream on Facebook 
    BloodStream on Twitter

    • 48 min
    Hemophilia Gene Therapy Redux – The End of the Beginning?

    Hemophilia Gene Therapy Redux – The End of the Beginning?

    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 
    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 
    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 organizations including the World Federation of Hemophilia, Hemophilia Federation of America, and the European Haemophilia Consortium joined this effort submitting separate but supportive statements to the FDA. However, the FDA reasoned that the requirements for the REMS for etranacogene dezaparvovec

    • 1 hr
    PTSD & Hemophilia

    PTSD & Hemophilia

    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
     
    Subscribe to the Global Hemophilia Report
     
    Connect with the Global Hemophilia Report
    Global Hemophilia Report on LinkedIn
    Global Hemophilia Report on Twitter
    Global Hemophilia Report on Facebook
     

    • 54 min

Customer Reviews

5.0 out of 5
10 Ratings

10 Ratings

ShuaBragg ,

Outstandingly Comprehensive

This is the most outstandingly comprehensive hemophilia podcast out there. It’s science based, and can at times be a bit dense, but the host does a really fantastic job of simplifying the harder to understand elements of the research being done, and I feel up to date with parts of the hemophilia world I didn’t even know about!

Hopscotch03 ,

Amazing content, so much history.

Really impressive podcast. As someone with hemophilia, I appreciate knowing a show like this exists.Ii learned a lot about novel therapies, joint health, and women with hemophilia. Thank you!

CandidHams4000 ,

Love it!

Great inside-baseball coverage of the state of bleeding disorders. Glad I stumbled on this.

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