Global Hemophilia Report BloodStream Media

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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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
 
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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
 
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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
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Global Hemophilia Report on Twitter
Global Hemophilia Report on Facebook
 
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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

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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