PB1250 - Quality of Life in Females and Males with Mild Congenital Hemophilia A: A Longitudinal Study from the PicnicHealth Database of PROMIS®-29 v2.0 Scores

Tuesday, June 27, 2023

18:30 – 19:30 ET

Room: Exhibition

Co-Author(s)

Angela C. Weyand, MD (she/her/hers)

Associate Professor
University of Michigan Medical School
Ann Arbor, Michigan, United States

Co-Author(s)

FN

Francis Nissen

Senior Data Scientist
F. Hoffmann-La Roche Ltd
Basel, Basel-Stadt, Switzerland
SR

Spyros Roumpanis

Senior Data Scientist
F. Hoffmann-La Roche Ltd
Basel, Basel-Stadt, Switzerland
KM

Katya Moreno

Therapeutic Area Head Hematology and Global Product Development / Medical Affairs
F. Hoffmann-La Roche Ltd
Basel, Basel-Stadt, Switzerland

Presenting Author(s)

Dakota Rosenfelt, PharmD, RPh, MBAc (he/him/his)

Senior Medical Science Liaison
Genentech, Inc.
San Francisco, California, United States

Co-Author(s)

GH

Gillian Hanson

Director – Data & Informatics
PicnicHealth
San Francisco, California, United States
EC

Emily Cibelli

Data Scientist
PicnicHealth
San Francisco, California, United States
KP

Krisha Patel

RWD Epidemiologist
PicnicHealth
San Francisco, California, United States
JS

Jessica Scrimshire

Data Sciences Industrial Placement Intern
F. Hoffmann-La Roche Ltd
Welwyn Garden City, England, United Kingdom

Background: Congenital hemophilia A (HA) is a hereditary X-linked bleeding disorder caused by deficiency in factor VIII. Males with HA (MwHA) make up the majority of the HA population; however, females are also affected (FwHA). Data on the quality of life (QoL) of females with mild HA are needed to gain further understanding of this population.

Aims: To assess QoL in females and males with mild HA.

Methods: The PicnicHealth platform is a real-world, patient-centric, longitudinal database, collating demographics and disease characteristics for people with HA. Participants enrolled in PicnicHealth since 2019 report details of bleeds, treatment, physical/social activity, pain, and QoL via an online platform. This analysis was funded by F. Hoffmann-La Roche; informed consent and ethics approval were obtained. The Patient-Reported Outcomes Measurement Information System (PROMIS®-29 v2.0; National Institutes of Health) is a concise, disease-agnostic QoL measure that captures comprehensive data across multiple health domains. Raw PROMIS®-29 v2.0 scores are rescaled into a standardized T-score metric, with a population mean of 50 and a standard deviation (SD) of 10.

Results: At data cut-off (May-31-2022), 45 people with mild HA had been recruited for the patient-reported survey: 17 FwHA and 28 MwHA (Table 1). Mean (SD) T-score metric for ‘Physical Function’ and ‘Ability to Participate in Social Roles and Activities’ was 48.0 (8.2) and 52.5 (8.6), respectively, for MwHA, and 41.7 (8.0) and 46.7 (9.6), respectively, for FwHA, with higher scores indicating better QoL (Figure 1). There was no notable difference in ‘Anxiety’, ‘Depression’, ‘Fatigue’, ‘Sleep Disturbance’, ‘Pain Interference’, or ‘Pain Intensity’ between males and females. Descriptive analyses were performed due to small cohort sizes.

Conclusion(s): PROMIS®-29 scores were similar for males and females with mild HA. Additional research with larger sample sizes is required to corroborate these preliminary findings and allow comparison between males and females.