ATHN 11: Observational Study of Long-Term Outcomes of Liver Transplantation
Recommended Citation
Ragni MV, Callis J, Daoud N, Hu B, Manuel M, Santos J, Schwartz J, Friedman KD, Kouides P, Kuriakose P, Leavitt AD, Lim MY, Machin N, Recht M, Chrisentery-Singleton T. ATHN 11: Observational Study of Long-Term Outcomes of Liver Transplantation. Res Pract Thromb Haemost 2023; 7.
Document Type
Conference Proceeding
Publication Date
12-18-2023
Publication Title
Res Pract Thromb Haemost
Abstract
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).
Volume
7
