Haemophilia : the official journal of the World Federation of Hemophilia
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Comparative Study Clinical Trial
An exploratory comparison of single intra-articular injection of platelet-rich plasma vs hyaluronic acid in treatment of haemophilic arthropathy of the knee.
Intra-articular platelet-rich plasma (PRP) injection therapy has been extensively applied in clinical practice to treat musculoskeletal disorders such as osteoarthritis, but the treatment for haemophilic arthropathy is rarely reported. ⋯ Our study demonstrates that, in comparison with five weekly injections of HA, a single PRP injection resulted in better improvement in pain relief and knee joint function, and greater reduction in synovial hyperaemia for up to 6 months. Our results suggest that PRP may be practical and effective for haemophilic knee arthropathy, and further investigation is warranted.
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The haemorrhagic phenotype in patients with von Willebrand disease (VWD) is heterogeneous, and assays of von Willebrand factor ristocetin cofactor activity (VWF:RCo) do not always reflect clinical severity, especially in those individuals classed as type 1 VWD. Recent studies have shown that whole blood ristocetin-induced platelet agglutination (WB-RIPA) using an easy-to-use analyzer, Multiplate® platelet impedance technique, could be informative as a diagnostic test in VWD, although inconsistencies were evident in patients with the type 1 disorder, possibly associated with clinical symptoms. ⋯ WB-RIPA did not reflect clinical severity in type 1 VWD patients.
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In this first in a series of round table meetings, the 1st World Federation of Hemophilia Gene Therapy Round Table was convened to initiate a global dialogue on the expected challenges and opportunities that a disruptive therapy, such as gene therapy, will bring to the haemophilia community. Perspectives from key stakeholder groups, including healthcare professionals, regulators, payors, people with hemophilia and pharmaceutical industry representatives, were sought in the identification of the key issues we expect to face. ⋯ These were followed by small group work. This manuscript outlines the key issues identified and the path forward.
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In sub-Saharan African countries, research on haemophilia is limited. Since 2015, a partnership has been established through the World Federation of Hemophilia (WFH)'s twinning programme between the haemophilia treatment centre (HTC) of the Centre Hospitalier universitaire of Yopougon in Abidjan, Côte d'Ivoire, and the Cliniques universitaires Saint-Luc of Brussels, Belgium. ⋯ This study highlights the value of simple, feasible and inexpensive tools to collect data in the Ivorian haemophilia population and provides the basis for developing and implementing locally appropriate strategies to improve screening, diagnosis, preventive care, treatment and education. It demonstrated the WFH twinning programme benefits for haemophilia care in the developing world.
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It is unclear which outcome indicators should be used to measure the success of haemophilia transition programs, and what are key elements of a haemophilia transition program to ensure success. ⋯ Results will contribute towards the development of a haemophilia transition outcome instrument and provide guidance for future studies of the effectiveness of transition programs.