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

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.