Global Hemophilia Report

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   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   

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   

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

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

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 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 were not met, and it has received marketing authorization from the FDA without the requirement for the REMS. While this may be the case for people living with hemophilia B, for whom the benefit to risk equation for gene therapy is generally favorable [[58]], it may not be the same for people with hemophilia A when considering gene therapy. Standard and extended half-life products for the prevention of bleeding for people living with either hemophilia A and B may provide very good outcomes but at a substantial burden of treatment and also fail to prevent all bleeding.   Another approach is a restrictive introduction, such as a conditional marketing authorization in which a medicine fulfills an unmet medical need but there is still a need for comprehensive safety, efficacy, and quality data generation after approval [[62]]. Such approvals are granted for 1 year but can be renewed annually. Compliance with specific obligations with defined timelines is a necessary prerequisite for continued authorization [   [63]]. This mechanism was utilized in the case of onasemnogene abeparvovec, an AAV9 gene therapy for spinal muscular atrophy [[64]].   Here, we recommend that the following steps be taken by the hemophilia community to ensure the safety and optimal outcomes for PwH who choose to receive a gene therapy product:   1.Training and education must be provided for physicians and HCPs on gene therapy and the management of PwH who receive a gene therapy product 2.Training and education on shared decision making must be provided for physicians and HCPs who will evaluate, administer, and follow PwH who are candidates to receive a gene therapy product [ 3.Facilities administering valoctocogene roxaparvovec and etranacogene dezaparvovec must receive adequate training and instruction on all aspects of gene therapy [ 4.Valoctocogene roxaparvovec and etranacogene dezaparvovec must only be administered at or in conjunction with a hemophilia treatment center with knowledge and expertise in evaluating, administering, and managing PwH who have received investigational gene therapy products [ 5.Individuals receiving valoctocogene roxaparvovec and etranacogene dezaparvovec must be enrolled in the global gene therapy registry initiated by the World Federation of Hemophilia in order to collect robust data, including adverse events of special interest [ 6.Educational support should be developed in a transparent and unbiased way to facilitate learning by PwH so that they may participate in shared decision making  7.Formal collaboration between the relevant national hemophilia patient organizations and the centers administering gene therapy on the provision of education and information should be ensured so that the PwH is ready to fully participate in a shared and informed decision-making process. 8.Work to ensure that postmarket studies, registries, and future registrational studies take into consideration principles of health equity in their design.   *Baas: J Thromb Haemost. 2023 Mar;21(3):413-420. (Ethics)   Based on our analysis of the literature, we identified 3 ethical themes. The theme Living up to expectations describes the existing hopes for gene therapy and the unlikelihood of the currently approved product becoming a permanent cure. In the theme Psychosocial impacts, we discuss the fear that gene therapy will impact the identity of people with hemophilia and their need for psychosocial support. The theme Costs and access discusses the expected cost-effectiveness of gene therapy and its implications on accessibility worldwide. We conclude that it may be necessary to change the narratives surrounding gene therapy, from describing it as a cure to describing it as one of the many treatments that temporarily relieve symptoms and that there is a need to reevaluate the desirability of gene therapy for hemophilia, given the availability of other treatments. *Kumar: J Thromb Haemost. 2024 Jan;22(1):23-34    Of note, multiple preclinical studies using platelet-specific lentiviral gene delivery to hematopoietic stem cells in hemophilia have demonstrated promising results with therapeutic levels of neo-protein that rescue the hemorrhagic bleeding phenotype and induce antigen-specific immune tolerance. Further studies using ovalbumin as a surrogate protein for platelet gene therapy have shown robust antigen-specific immune tolerance induced via peripheral clonal deletions of antigen-specific CD4- and CD8-T effector cells and induction of antigen-specific regulatory T (Treg) cells. This review discusses platelet-targeted gene therapy, focusing on immune tolerance induction.   Shah: Curr Med Res Opin. 2023 Feb;39(2):227-237   Bayesian and Frequentist linear mixed

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  

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   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   

In this episode, we address the critical intersections of LGBTQ+ health and hemophilia care. Delving into the historical links with the HIV crisis, we scrutinize the disparities in care access, the mental health implications, and the unique challenges faced during transition to independent care. We discuss the intricacies of prophylactic drug regimens, vaccines, and the hemophilic bleeding risk related to LGBTQ+ sexual health. With insights into gender-affirming care, reproductive counseling, and knowledge gaps, we provide a comprehensive perspective tailored for physicians and clinicians aiming for holistic patient care.   Contributors: Nathan Connell, MD Nancy Sokkary, MD Robert Sidonio, MD Greig Blamey, Physiotherapist Dakota J. Rosenfelt, PharmD, RPh, MBA    Senior Advisor: Donna DiMichele, MD Hosted by: Patrick James Lynch Written by: Simantini Karve, PhD  

On this episode, we will address defining sexual health and its importance to hemophilia care, best practices and standards for addressing sexual health, implementing a practice of addressing sexual health into a comprehensive care model, envisioning progress in care models for addressing sexual health in both global and personalized ways and more.    Contributors: Greig Blamey, Physiotherapist Maureen Baldwin, MD William McKeown, MD   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   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter   

Persons with mild hemophilia have been shown to experience more bleeding - related morbidity than previously recognized while achieving lifespans that parallel the general population. Consequently, persons living with mild hemophilia are more likely to experience the comorbidities associated with aging, often complicating their management in their later years.    Contributors: Prof Karin (C. J.) Fijnvandraat MD PhD Maria Elisa Mancuso, MD PhD Michael Recht, MD PhD MBA Vaughn Ripley   Senior Advisor: Donna DiMichele, MD   Hosted by: Patrick James Lynch Written by: Simantini Karve, PhD  

Telehealth options are assumed to provide added benefit to providers and patients alike, but how much do we really know and understand about telemedicine? Listen in to the latest episode of Global Hemophilia Report as our contributors share how telemedicine has potentially benefited and detracted from hemophilia care across different populations as well as what research must still be done on the rise of telehealth in hemophilia care   Contributors: Sativa Rangarajan, Associate Professor of Clinical Haematology  Michelle Witkop, DNP, FNP-BC  Dr. Roshni Kulkarni, Professor & Former Director Michigan State University Centers For Bleeding And Clotting Disorders Annette von Drygalski, MD, PharmD   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   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter   

The social determinants of health (SDH) are the non-medical factors that influence health outcomes. They are the conditions in which people are born, grow, work, live, and age, and the wider set of forces and systems shaping the conditions of daily life. These forces and systems include economic policies and systems, development agendas, social norms, social policies and political systems.   Contributors: Keri Norris, PhD, JM, MPH, MCHES Karina Lopez, MPH Adrian Palau-Tejeda Marissa Melton, MPH Chris Bombardier Mosi Williams   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   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter   

Historically, camps for kids with hemophilia have played a specific and valuable role, enabling a young person with hemophilia to experience all the benefits of a traditional, summer camp experience. On this episode, we dig into the camp experience with some of the leading voices within the space.  Contributors: Tracey Gaslin PhD, CPNP, FNP-BC, CRNI, RN-BC Michael Wang, MD Pat Torrey Sarah Shinkman Chris Bombardier   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   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter   

Historically, people with hemophilia and especially children, have lived with considerable limitations when it comes to engagement in sports and athletics, thereby missing out on the numerous biopsychosocial and general health. However, both advancements in hemophilia care/treatment and advancements in understanding the benefits of engaging in sports have significantly changed the risk/benefit equation– but exactly, how?   Contributors: Marilyn Manco Johnson, M.D. Olav Versloot, PhD Paul Mclaughlin Chris Bombardier   Senior Advisor: Donna DiMichele, MD   Hosted by: Patrick James Lynch   Show Notes: Presenting Sponsor: Sanofi   Subscribe to the Global Hemophilia Report   Watch the film Bombardier Blood Listen to The Final Summit podcast   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   

On this episode we’re in conversation with research leaders to learn more about the current state of hemophilia research, the barriers and opportunities we're facing today, and what the future of hemophilia research might look like.     Contributors: Flora Peyvandi, MD, PhD Keith Hoots, MD Len Valentino, MD Michelle Witkop, DNP, FNP-BC Glenn Pierce, MD, PhD   Senior Advisor: Donna DiMichele, MD   Hosted by: Patrick James Lynch   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   

There has been a remarkable increase in the life expectancy of persons living with hemophilia (PLWH) over the past century, largely due to access to safe factor replacement products.  However, the course of progress has been uneven, and rocky, for many. This is part two of our series on Aging with Hemophilia. Contributors: William McKeown, MD Randall Curtis Gerry Dolan, MD, MBChB, FRCP, FRCPath Barbara Konkle, MD Sara L. Schwartz, PhD  Tam Perry, PhD   Senior Advisor: Donna DiMichele, MD   Hosted by: Patrick James Lynch   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   

There has been a remarkable increase in the life expectancy of persons living with hemophilia (PLWH) over the past century, largely due to access to safe factor replacement products.  However, the course of progress has been uneven, and rocky, for many.   Contributors: William McKeown, MD Randall Curtis Gerry Dolan, MD, MBChB, FRCP, FRCPath Barbara Konkle, MD   Senior Advisor: Donna DiMichele, MD   Hosted by: Patrick James Lynch   Listen to Blood of the Paladin Podcast   Show Notes: Presenting Sponsor: Sanofi   Subscribe to the Global Hemophilia Report Listen to BloodStream’s Journey to Joint Health episode.   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 

Renowned hematologist, researcher, and senior advisor to the Global Hemophilia Report Dr. Donna DiMichele shares some of her Season 1 takeaways and thoughts about the future of hemophilia research. Senior Advisor: Donna DiMichele, MD   Hosted by: Patrick James Lynch   Show Notes: Presenting Sponsor: Sanofi   Subscribe to the Global Hemophilia Report Listen to BloodStream’s Journey to Joint Health episode.   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 

On this episode, we continue our discussion of chronic pain in hemophilia. We’ll discuss exercise, medication and investigations.   Contributors: Nathalie Anne Roussel, PhD, MSc, PT Paul Mc Laughlin,  BSc, MSc, MMACP   Senior Advisor: Donna DiMichele, MD   Episode Advisors (also contributors): Michelle Witkop, DNP, FNP-BC Tyler Buckner, MD   Hosted by: Patrick James Lynch   Show Notes: Presenting Sponsor: Sanofi   Subscribe to the Global Hemophilia Report Listen to BloodStream’s Journey to Joint Health episode.   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 

According to the Centers for Disease Control, pain is one of the most common reasons people seek medical care. However, for persons affected by hemophilia A and B, the experience of pain has provided the subtext for many issues related to health and wellbeing across the lifespan, and irrespective of gender, age, and, potentially, hemophilia severity levels. Although less-well characterized in children, pain is already embedded in the life experience of a PWH, and a substantial contributor to poor health-related quality of life in adolescence and young adulthood, further increasing in negative impact throughout adult life. In this episode, we explore pain’s prevalence, mechanisms, and more.    Contributors: Nathalie Anne Roussel, PhD, MSc, PT Paul Mc Laughlin,  BSc, MSc, MMACP Michelle Rice    Senior Advisor: Donna DiMichele, MD   Episode Advisors (also contributors): Michelle Witkop, DNP, FNP-BC Tyler Buckner, MD   Hosted by: Patrick James Lynch   Show Notes: Presenting Sponsor: Sanofi Subscribe to the Global Hemophilia Report   Listen to BloodStream’s Pain Podcast   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