Page 15 - HFA Dateline 2021 Q2 Summer

The panel was comprised of HFA and thrombosis, Department of webinars presented by HFA,
Director of Public Health and Pediatrics, Children’s Healthcare of representatives from four
Outcomes Projects Mabel Crescioni, Atlanta. pharmaceutical manufacturers—
DrPH, JD, LLM; National Hemophilia CSL Behring, Genentech, HEMA
Foundation’s head of research, “What to Expect When Participating Biologics and Sanofi Genzyme—
Michelle Witkop, DNP; and Plasma in Clinical Trials” was presented by hosted sessions that provided
Protein Therapeutics Association’s Julie Smith, CCRP, who was able community members with updates
senior vice president of public to provide the perspective of an and information about available
affairs, Julie Birkofer. HTC clinical research manager. treatment options. In keeping with
Smith co-manages a team of six their mission of connecting patients,
Iglewski also moderated “Shared clinical research coordinators at HFA also hosted rap sessions, led
Decision-Making: Making the Most of the University of Colorado Denver by an HTC nurse, that were open to
the Patient-Provider Relationship.” Hemophilia and Thrombosis Center. community members and caregivers
Longtime advocates and active as an opportunity to meet with other
members of the community Will David Clark, a retired chemical community members and discuss
Hubbert and Cazandra MacDonald engineer with more than 35 years their experiences. Rap sessions were
joined the panel to provide of experience in the biotechnology, grouped by diagnosis of hemophilia
patient perspectives. The provider plasma and tissue industries, hosted A or B, von Willebrand disease,
perspective was brought by Chris the “Emerging Therapies: The Latest inhibitor or other rare bleeding
Guelcher, a hematology/oncology in Clinical Trials” session. Patients disorder.
learned about the ever-changing
landscape of bleeding When asked why this event was made
disorders treatment, a priority for the HFA team, Wilkes
basics of clinical trial said, “It was 17 years ago when I was
progression and items choosing a treatment option for my
of discussion for newborn son with severe hemophilia
patients/caregivers A. The overwhelming feeling remains
to consider with their vivid. It was a confusing and scary
health care provider process, despite having wonderful
when thinking about support from the staff at our local
participation in a trial. hemophilia treatment center. I was
a mom, unsure of how to even
Acronyms abound in spell hemophilia at that point, just
the bleeding disorders wanting to make the right call for
world—how do you my baby’s health. But, I didn’t know
make sense of FVIII, what questions to ask, what specifics
FIX, vWF, QoL, HCV, to consider or even what our options
MASAC, ISTH, EHC, were.”
AAV and the plethora
of others? To help Recordings of each session and
community members biographies about each speaker are
learn what these letters available on HFA’s website at www.
mean and represent, hemophiliafed.org/dateline-live.
HFA’s director of policy Subscriptions to the quarterly print
and principal legal counsel, Miriam
clinic nurse at Children’s National in magazine are free; sign up today at
Washington, DC, and Robert Sidonio, Goldstein, JD, along with Wilkes, www.hemophiliafed.org. To request
MD, associate professor of pediatrics hosted an interactive session called a copy of the 2021 Product Guide &
at Emory University School of “Bleeding Disorders Alphabet Soup: Emerging Therapies Special Issue,
Medicine, clinical director of the What Do These Letters Mean?” please send your name and address
hemophilia treatment center (HTC) In addition to the educational to info@hemophilafed.org.
and associate director of hemostasis

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