Contrasting Approaches in the Implementation of GRADE Methodology in Guidelines for Haemophilia and Von Willebrand Disease
(2024) In Haemophilia- Abstract
Introduction: The 2024 ISTH clinical practice guideline (CPG) for treatment of congenital haemophilia, the NBDF-McMaster Guideline on Care Models for Haemophilia Management, and ASH ISTH NBDF WFH guidelines on the diagnosis and management of VWD all utilised GRADE methodology. Aim: Discuss missed opportunities and the methodological approach of the ISTH Guideline in contrast to how GRADE was previously applied in rare diseases. Methods: Critically analyse the methodology of each guideline along with best practices in the use of GRADE. Where applicable, the WFH Guidelines for the Management of Haemophilia were analysed. Results: Important differentiating features in applying GRADE were identified. Where a strong evidence base is lacking,... (More)
Introduction: The 2024 ISTH clinical practice guideline (CPG) for treatment of congenital haemophilia, the NBDF-McMaster Guideline on Care Models for Haemophilia Management, and ASH ISTH NBDF WFH guidelines on the diagnosis and management of VWD all utilised GRADE methodology. Aim: Discuss missed opportunities and the methodological approach of the ISTH Guideline in contrast to how GRADE was previously applied in rare diseases. Methods: Critically analyse the methodology of each guideline along with best practices in the use of GRADE. Where applicable, the WFH Guidelines for the Management of Haemophilia were analysed. Results: Important differentiating features in applying GRADE were identified. Where a strong evidence base is lacking, data other than those from randomized controlled trials, which may not always be justified, need to be considered, including incorporation of outcomes important to people living with the disease. Justification and stakeholder input to prioritize questions requiring a new guideline, panel composition with necessary patient participation and content expertise were also found to be significant differentiating features. Conclusion: The puristic approach taken in the ISTH Guideline development process, without consideration of accepted adaptations to GRADE implementation, created a missed opportunity for progressing haemophilia care, leading to guideline recommendations that have been widely deemed invalid and obsolete by expert healthcare professionals and by those living with the condition, the very people who are expected to implement or bear the impact of the recommendations. Lessons learnt from this comparative analysis should guide future guideline development and encourage collaboration to further advance haemophilia.
(Less)
- author
- author collaboration
- organization
- publishing date
- 2024
- type
- Contribution to journal
- publication status
- epub
- subject
- keywords
- GRADE, guideline, haemophilia, ISTH, rare disease, von Willebrand disease
- in
- Haemophilia
- publisher
- John Wiley & Sons Inc.
- external identifiers
-
- pmid:39648854
- scopus:85211629642
- ISSN
- 1351-8216
- DOI
- 10.1111/hae.15136
- language
- English
- LU publication?
- yes
- id
- 9e683301-ccf5-4cf6-9112-eb92a54e02f5
- date added to LUP
- 2025-01-31 14:07:30
- date last changed
- 2025-07-05 02:50:37
@article{9e683301-ccf5-4cf6-9112-eb92a54e02f5,
abstract = {{<p>Introduction: The 2024 ISTH clinical practice guideline (CPG) for treatment of congenital haemophilia, the NBDF-McMaster Guideline on Care Models for Haemophilia Management, and ASH ISTH NBDF WFH guidelines on the diagnosis and management of VWD all utilised GRADE methodology. Aim: Discuss missed opportunities and the methodological approach of the ISTH Guideline in contrast to how GRADE was previously applied in rare diseases. Methods: Critically analyse the methodology of each guideline along with best practices in the use of GRADE. Where applicable, the WFH Guidelines for the Management of Haemophilia were analysed. Results: Important differentiating features in applying GRADE were identified. Where a strong evidence base is lacking, data other than those from randomized controlled trials, which may not always be justified, need to be considered, including incorporation of outcomes important to people living with the disease. Justification and stakeholder input to prioritize questions requiring a new guideline, panel composition with necessary patient participation and content expertise were also found to be significant differentiating features. Conclusion: The puristic approach taken in the ISTH Guideline development process, without consideration of accepted adaptations to GRADE implementation, created a missed opportunity for progressing haemophilia care, leading to guideline recommendations that have been widely deemed invalid and obsolete by expert healthcare professionals and by those living with the condition, the very people who are expected to implement or bear the impact of the recommendations. Lessons learnt from this comparative analysis should guide future guideline development and encourage collaboration to further advance haemophilia.</p>}},
author = {{Skinner, Mark W. and Albisetti, Manuela and Ardila, Jesús and Astermark, Jan and Blatny, Jan and Carcao, Manuel and Chowdary, Pratima and Connell, Nathan T. and Crato, Miguel and Dargaud, Yesim and d'Oiron, Roseline and Dunn, Amy L. and Escobar, Miguel A. and Escuriola-Ettingshausen, Carmen and Gouider, Emna and Harroche, Annie and Hermans, Cedric and Jimenez-Yuste, Victor and Kaczmarek, Radoslaw and Kenet, Gili and Khoo, Liane and Klamroth, Robert and Langer, Florian and Lillicrap, David and Mahlangu, Johnny and Male, Christoph and Matsushita, Tadashi and Meunier, Sandrine and Miesbach, Wolfgang and Nolan, Beatrice and Oldenburg, Johannes and O'Mahony, Brian and Ozelo, Margareth and Pierce, Glenn F. and Ramos, Gloria and Recht, Michael and Romero-Lux, Olivia and Rotellini, Dawn and Santoro, Rita C. and Singleton, Tammuella C. and Srivastava, Alok and Susen, Sophie and Talks, Kate and Tran, Huyen and Valentino, Leonard A. and Windyga, Jerzy and Yang, Renchi and Mancuso, Maria Elisa}},
issn = {{1351-8216}},
keywords = {{GRADE; guideline; haemophilia; ISTH; rare disease; von Willebrand disease}},
language = {{eng}},
publisher = {{John Wiley & Sons Inc.}},
series = {{Haemophilia}},
title = {{Contrasting Approaches in the Implementation of GRADE Methodology in Guidelines for Haemophilia and Von Willebrand Disease}},
url = {{http://dx.doi.org/10.1111/hae.15136}},
doi = {{10.1111/hae.15136}},
year = {{2024}},
}