Observational cohort study of long-term outcomes of liver transplantation in haemophilia

Abstract

Introduction: Gene therapy is now a reality for individuals with haemophilia, yet little is known regarding the quality-of-life impact of factor correction. As few data exist, and recognizing the analogy to liver transplantation (OLTX), we identified OLTX+ and OLTX- men in the ATHNdataset to compare post-OLTX factor VIII and IX on quality of life (QoL) by Haem-A-QoL and PROMIS-29. Methods: OLTX- were matched to OLTX+ by age, race, and haemophilia type and severity. Deidentified demographic data, including post-transplant factor levels, genotype and target joint disease were analysed by descriptive statistics. Haem-A-Qol and PROMIS-29 were compared in OLTX+ and OLTX- by student's t-test and univariate regression models. Results: Of 86 people with haemophilia A (HA) or haemophilia B (HB) cared for at 10 haemophilia treatment centers (HTCs), 21 (24.4%) OLTX+ and 65 (75.6%) OLTX- were identified. OLTX+ and OLTX- had a similar frequency of target joint disease (p =.806), HA genotypes, null versus non-null (p =.696), and HIV infection (p =.316). At a median 9.2 years post-OLTX, median FVIII,.63 IU/mL [IQR 0.52–0.97] and FIX,.91 IU/mL [IQR.63–1.32], Haem-A-QoL, PROMIS-29, and HOT scores were comparable. Severe HA/HB had lower post-OLTX ‘dealing with haemophilia’ scores (p =.022) and higher ‘sports and leisure’ (p =.010) and ‘view of yourself’ scores (p =.024) than OLTX+ non-severe participants. Non-caucasian OLTX+ had significantly lower scores in sports and leisure (p =.042), future expectations (p =.021) and total score (p =.010). Conclusion: Nine years after OLTX, QoL is comparable to OLTX–, but significantly better in OLTX+ with severe than non-severe disease and in caucasians than non-caucasians.