International Society on Thrombosis and Haemostasis Clinical Practice Guideline for Treatment of Congenital Haemophilia-A Critical Appraisal

• Autoři: ALBISETTI Manuela; ARDILA Jesus; ASTERMARK Jan; BLATNÝ Jan; CARCAO Manuel; CHOWDARY Pratima; CONNELL Nathan T; CRATO Miguel; DARGAUD Yesim; D'OIRON Roseline; DUNN Amy L; ESCOBAR Miguel A; ETTINGSHAUSEN Carmen Escuriola; FISCHER Kathelijn; GOUIDER Emna; HARROCHE Annie; HERMANS Cedric; JIMENEZ-YUSTE Victor; KACZMAREK Radoslaw; KENET Gili; KHOO Liane; KLAMROTH Robert; LANGER Florian; LILLICRAP David; MAHLANGU Johnny; MALE Christoph; MANCUSO Maria Elisa; MATSUSHITA Tadashi; MEUNIER Sandrine; MIESBACH Wolfgang; NOLAN Beatrice; OLDENBURG Johannes; O'MAHONY Brian; OZELO Margareth; PIERCE Glenn F; RAMOS Gloria; RECHT Michael; ROMERO-LUX Olivia; ROTELLINI Dawn; SANTORO Rita C; SINGLETON Tammuella C; SKINNER Mark W; SRIVASTAVA Alok; SUSEN Sophie; TALKS Kate; TRAN Huyen; VALENTINO Leonard A; WINDYGA Jerzy; YANG Renchi
• Rok publikování: 2025
• Druh: Článek v odborném periodiku
• Časopis / Zdroj: Haemophilia
• Fakulta / Pracoviště MU: Lékařská fakulta
• Citace: ALBISETTI, Manuela, Jesus ARDILA, Jan ASTERMARK, Jan BLATNÝ, Manuel CARCAO, Pratima CHOWDARY, Nathan T CONNELL, Miguel CRATO, Yesim DARGAUD, Roseline D'OIRON, Amy L DUNN, Miguel A ESCOBAR, Carmen Escuriola ETTINGSHAUSEN, Kathelijn FISCHER, Emna GOUIDER, Annie HARROCHE, Cedric HERMANS, Victor JIMENEZ-YUSTE, Radoslaw KACZMAREK, Gili KENET, Liane KHOO, Robert KLAMROTH, Florian LANGER, David LILLICRAP, Johnny MAHLANGU, Christoph MALE, Maria Elisa MANCUSO, Tadashi MATSUSHITA, Sandrine MEUNIER, Wolfgang MIESBACH, Beatrice NOLAN, Johannes OLDENBURG, Brian O'MAHONY, Margareth OZELO, Glenn F PIERCE, Gloria RAMOS, Michael RECHT, Olivia ROMERO-LUX, Dawn ROTELLINI, Rita C SANTORO, Tammuella C SINGLETON, Mark W SKINNER, Alok SRIVASTAVA, Sophie SUSEN, Kate TALKS, Huyen TRAN, Leonard A VALENTINO, Jerzy WINDYGA a Renchi YANG. International Society on Thrombosis and Haemostasis Clinical Practice Guideline for Treatment of Congenital Haemophilia-A Critical Appraisal. Haemophilia. HOBOKEN: Blackwell Science, 2025, 10 s. ISSN 1351-8216. Dostupné z: https://dx.doi.org/10.1111/hae.15135.
• www: https://onlinelibrary.wiley.com/doi/10.1111/hae.15135
• Doi: http://dx.doi.org/10.1111/hae.15135
• Klíčová slova: evidence-based medicine; GRADE; guideline; haemophilia; ISTH; WFH
• Popis: IntroductionEvidence-based clinical practice guidelines drive optimal patient care and facilitate access to high-quality treatment. Creating guidelines for rare diseases such as haemophilia, where evidence does not often come from randomized controlled trials but from non-randomized and well-designed observational studies and real-world data, is challenging. The methodology used for assessing available evidence should consider this critical fact. In formulating guidelines, it is essential to include treatment goals and patient preference.AimThis paper comprehensively critiques, against this background, the recommendations of the ISTH clinical practice guidelines for the treatment of haemophilia.MethodsEach recommendation was critically reviewed against available evidence as well as existing guidelines and commented upon for its scientific validity, impact on clinical practice and access to care globally. The validity of the way in which the GRADE methodology was applied to existing evidence was also assessed.ResultsThe critique provided shows that these recommendations have major limitations: they did not state treatment goals and contradict existing guidelines; opportunities for providing access to innovation were missed when the therapeutic benefits of the products approved in the last decades were not included. A major reason for this is the inappropriate adoption of the GRADE methodology without adaptations and without considering treatment goals and patient-relevant outcomes.ConclusionThese recommendations may mislead healthcare professionals, payers and governments and therefore cannot serve the patient community well. They setback the advances made in haemophilia care because they overlook important available evidence and do not guide clinical practice to contemporary standards.