Sylvia von Mackensen, PhD
Highlights From the 65th ASH Annual Meeting and Exposition

Health-related Quality of Life in Adults With Hemophilia B After Receiving Gene Therapy With Fidanacogene Elaparvovec

Presented by: Sylvia von Mackensen, PhD

Sylvia von Mackensen, PhD1, Jonathan M. Ducore, MD, MPH2, Lindsey A. George, MD3,4, Adam Giermasz, MD, PhD5, Catherine McGuinn, MD6, John E. J. Rasko, MBBS, PhD7, Ben J. Samelson-Jones, MD, PhD4,8, Spencer K. Sullivan, MD3,4, Jerome M. Teitel, MD9, Amit Chhabra, MBBS, MPH10, Annie F. Fang, MD, PhD10, Amanda O'Brien11, Frank Plonski, MA, RN, BS11, Jeremy Rupon, MD, PhD11, Lynne Smith, MBA11, and Ian Winburn12

1 Department of Medical Psychology, University Medical Centre Hamburg-Eppendorf, Hamburg-Eppendorf, DEU
2 Hemophilia Treatment Center, University of California Davis, Sacramento, CA
3 The Children's Hospital of Philadelphia, Philadelphia, PA
4 Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA
5 University of California, Davis, Sacramento, CA
6 Pediatric Hematology Oncology Division, Weill Cornell Medical College, New York, NY
7 Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital, Sydney, NSW, AUS
8 Division of Hematology, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA
9 St. Michael's Hospital, Toronto, ON, CAN
10 Pfizer Inc., New York, NY
11 Pfizer Inc., Collegeville, PA
12 Pfizer Inc., Tadworth, GBR

Data Points

This graphic represents the results from the Health-related Quality of Life (HRQoL) questionnaire taken by patients with hemophilia B between weeks 52 and 156 following administration of fidanacogene elaparvovec.

This graphic represents the results from the Health-related Quality of Life (HRQoL) questionnaire taken by patients with hemophilia B, between weeks 52 and 156, following administration of fidanacogene elaparvovec. From pre-vector infusion to week 156, there was a clinically meaningful decrease in the following domains:

  • “Physical Health” domain score decreased by 11.9 (15.6)
  • “Sport and Leisure” domain score decreased by 29.4 (25.0)
  • Total score decreased by 15.2 (10.3)

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Supported through educational grants from BioMarin, CSL Behring, Pfizer Inc., and Spark Therapeutics

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