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Observational cohort study of long-term outcomes of liver transplantation in haemophilia.
Ragni, Margaret V; Callis, Jessica; Daoud, Nabil; Hu, Brian; Manuel, Matthew; Santos, Jarren; Schwartz, Jonathan; Friedman, Kenneth D; Kouides, Peter; Kuriakose, Philip; Leavitt, Andrew D; Lim, Ming Y; Machin, Nicoletta; Recht, Michael; Chrisentery-Singleton, Tammuella.
Afiliación
  • Ragni MV; Department of Medicine, Division of Hematology/Oncology, University of Pittsburgh Medical Center, and Hemophilia Center of Western PA, Pittsburgh, Pennsylvania, USA.
  • Callis J; American Thrombosis Hemostasis Network, Rochester, New York, USA.
  • Daoud N; American Thrombosis Hemostasis Network, Rochester, New York, USA.
  • Hu B; American Thrombosis Hemostasis Network, Rochester, New York, USA.
  • Manuel M; American Thrombosis Hemostasis Network, Rochester, New York, USA.
  • Santos J; American Thrombosis Hemostasis Network, Rochester, New York, USA.
  • Schwartz J; American Thrombosis Hemostasis Network, Rochester, New York, USA.
  • Friedman KD; Comprehensive Center for Bleeding Disorders, Milwaukee, Wisconsin, USA.
  • Kouides P; Mary M. Gooley Hemophilia Center, Rochester, New York, USA.
  • Kuriakose P; Henry Ford Health System Bleeding and Thrombosis Center, Detroit, Michigan, USA.
  • Leavitt AD; San Francisco Hemophilia Treatment Center, University of California, San Francisco, California, USA.
  • Lim MY; Utah Center for Bleeding and Clotting Disorders, Salt Lake City, Utah, USA.
  • Machin N; Department of Medicine, Division of Hematology/Oncology, University of Pittsburgh Medical Center, and Hemophilia Center of Western PA, Pittsburgh, Pennsylvania, USA.
  • Recht M; American Thrombosis Hemostasis Network, Rochester, New York, USA.
  • Chrisentery-Singleton T; Yale University Medical Center, New Haven, Connecticut, USA.
Haemophilia ; 30(1): 87-97, 2024 Jan.
Article en En | MEDLINE | ID: mdl-38111071
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.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Infecciones por VIH / Hemofilia B / Trasplante de Hígado / Hemofilia A / Artropatías Límite: Humans / Male Idioma: En Revista: Haemophilia Asunto de la revista: HEMATOLOGIA Año: 2024 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Reino Unido
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Infecciones por VIH / Hemofilia B / Trasplante de Hígado / Hemofilia A / Artropatías Límite: Humans / Male Idioma: En Revista: Haemophilia Asunto de la revista: HEMATOLOGIA Año: 2024 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Reino Unido