Contrasting Approaches in the Implementation of GRADE
Methodology in Guidelines for Haemophilia and Von Willebrand
Disease

J 2026

Contrasting Approaches in the Implementation of GRADE Methodology in Guidelines for Haemophilia and Von Willebrand Disease

SKINNER, Mark W; Manuela ALBISETTI; Jesus ARDILA; Jan ASTERMARK; Jan BLATNÝ et al.

Základní údaje

Originální název

Contrasting Approaches in the Implementation of GRADE Methodology in Guidelines for Haemophilia and Von Willebrand Disease

Autoři

SKINNER, Mark W; Manuela ALBISETTI; 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; 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; 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; Alok SRIVASTAVA; Sophie SUSEN; Kate TALKS; Huyen TRAN; Leonard A VALENTINO; Jerzy WINDYGA; Renchi YANG a Maria Elisa MANCUSO

Vydání

Haemophilia, HOBOKEN, Blackwell Science, 2026, 1351-8216

Další údaje

Jazyk

angličtina

Typ výsledku

Článek v odborném periodiku

Obor

30205 Hematology

Stát vydavatele

Spojené státy

Utajení

není předmětem státního či obchodního tajemství

Odkazy

URL

Impakt faktor

Impact factor: 3.000 v roce 2024

Označené pro přenos do RIV

Organizační jednotka

Lékařská fakulta

Klíčová slova anglicky

GRADE; guideline; haemophilia; ISTH; rare disease; von Willebrand disease

Štítky

14110321

Příznaky

Mezinárodní význam, Recenzováno

Změněno: 16. 2. 2026 12:24, Mgr. Tereza Miškechová

Anotace

V originále

IntroductionThe 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.AimDiscuss missed opportunities and the methodological approach of the ISTH Guideline in contrast to how GRADE was previously applied in rare diseases.MethodsCritically 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.ResultsImportant 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.ConclusionThe 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.

Citovat

@article{2478508,
   author = {Skinner, Mark W and Albisetti, Manuela and Ardila, Jesus and Astermark, Jan and Blatný, 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 EscuriolaandEttingshausen, Carmen and Gouider, Emna and Harroche, Annie and Hermans, Cedric and JimenezandYuste, 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 RomeroandLux, 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},
   article_location = {HOBOKEN},
   doi = {https://doi.org/10.1111/hae.15136},
   keywords = {GRADE; guideline; haemophilia; ISTH; rare disease; von Willebrand disease},
   language = {eng},
   issn = {1351-8216},
   journal = {Haemophilia},
   title = {Contrasting Approaches in the Implementation of GRADE Methodology in Guidelines for Haemophilia and Von Willebrand Disease},
   url = {https://onlinelibrary.wiley.com/doi/10.1111/hae.15136},
   year = {2026}
}

TY  - JOUR
ID  - 2478508
AU  - Skinner, Mark W - 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 - Escuriola-Ettingshausen, Carmen - 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 - 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 - Srivastava, Alok - Susen, Sophie - Talks, Kate - Tran, Huyen - Valentino, Leonard A - Windyga, Jerzy - Yang, Renchi - Mancuso, Maria Elisa
PY  - 2026
TI  - Contrasting Approaches in the Implementation of GRADE Methodology in Guidelines for Haemophilia and Von Willebrand Disease
JF  - Haemophilia
PB  - Blackwell Science
SN  - 13518216
KW  - GRADE
KW  - guideline
KW  - haemophilia
KW  - ISTH
KW  - rare disease
KW  - von Willebrand disease
UR  - https://onlinelibrary.wiley.com/doi/10.1111/hae.15136
N2  - IntroductionThe 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.AimDiscuss missed opportunities and the methodological approach of the ISTH Guideline in contrast to how GRADE was previously applied in rare diseases.MethodsCritically 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.ResultsImportant 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.ConclusionThe 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.
ER  -

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