PB1241 - ATHN 11: Observational Study of Long-Term Outcomes of Liver Transplantation

Tuesday, June 27, 2023

18:30 – 19:30 ET

Room: Exhibition

Presenting Author(s)

Margaret V. Ragni, MD, MPH (she/her/hers)

Professor of Medicine and Clinical and Translational Science
Department of Medicine, University of Pittsburgh, Pittsburgh, PA, USA
Pittsburgh, Pennsylvania, United States

Co-Author(s)

BH

Brian Hu

Statistician
ATHN
Rochester, New York, United States
JC

Jessica Callis

Assistant Project Manager
American Thrombosis Hemostasis Network
Rochester, New York, United States
MM

Matthew Manuel

Statistician
ATHN
Rochester, New York, United States
JS

Jarren Santos

Statistics
ATHN
Rochester, New York, United States
KF

Kenneth Friedman

Laboratory Director
Versiti Blood Research Institute
Waukesha, Wisconsin, United States
Peter Kouides, MD

Physician - Hematology/Oncology
Mary M. Gooley Hemophilia Centre, Rochester, NY, USA
Rochester, New York, United States
PK

Philip Kuriakose

Assistant professor
Wayne State University Medical School, Henry Ford Health System
Detroit, Michigan, United States
AL

Andrew D. Leavitt

Professor and Medical Director
Adult Hemophilia Treatment Center, University of California San Francisco, San Francisco, CA, USA
San Francisco, California, United States
ML

Ming Lim

Assistant Professor and Director
University Utah
Salt Lake City, Utah, United States
NM

Nicoletta Machin

Assistant Professor
University Pittsburgh
Pittsburgh, Pennsylvania, United States
Michael Recht, MD, PhD, MBA (he/him/his)

Chief Medical and Scientific Officer
National Hemophilia Foundation
New York, New York, United States

Background: As gene therapy (GT) is becoming a reality for hemophilia A and B (HA, HB), little is known about long-term phenotypic cures. Few data assess real-life impact of long-term GT factor correction. The analogy to liver transplantation (OLTX) is clear. As the United Network for Organ Sharing (UNOS) database lacked coding for hemophilia, we identified OLTX+ and OLTX- in ATHN-affiliated HTCs to compare post-OLTX FVIII, FIX on patient-reported outcomes (PRO) and quality of life (QoL).

Aims: In the ATHN 11 multicenter observational study, Haem-A-QoL and PROMIS-29 were compared, OLTX+ vs OLTX-.

Methods: Deidentified data from medical records were entered into ATHN electronic forms. OLTX- were matched to OLTX+ by age, race, HA, HB type and severity. Participants completed QoL tools. Demographic data were analyzed by descriptive statistics, and PRO and QoL were compared by OLTX status, using student’s t-test and univariate regression models. Significance was set at 0.05.

Results: Of 71 HA or HB patients cared for at 11 HTCs, 20 OLTX+ and 51 OLTX- were identified. There was no difference between OLTX+ vs OLTX-, respectively, in median age, 59 vs 57 yr; race, 18(90.0%) vs 47(92.1%) Caucasian; type hemophilia 15(75.0%) vs 43(84.3%) HA; or severity, 39(76.0%) vs 45(88.0%) with severe disease; or with target joint disease, 11 (55.0%) vs 25(49.0%). OLTX status was strongly associated with presence of a genetic mutation, p< 0.001, but not with three Haem-A-Qol scores (physical health, feelings, future health) or with the PROMIS score (physical function). QoL scores were most strongly correlated with history of target joint disease, followed by hemophilia severity and type, p< 0.05.

Conclusion(s): Hemophilic target joint disease is more strongly correlated with QoL and PRO than disease severity, suggesting the irreversible impact of arthropathy on QoL and PRO. Further analysis will assess sustained factor levels and minimally important differences (MID).