Diagnosis and management of pyruvate kinase deficiency: international expert guidelines

Result code in IS VaVaI

<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00098892%3A_____%2F24%3A10158625" target="_blank" >RIV/00098892:_____/24:10158625 - isvavai.cz</a>

Alternative codes found

RIV/61989592:15110/24:73625892

Result on the web

<a href="https://www.sciencedirect.com/science/article/abs/pii/S2352302623003770" target="_blank" >https://www.sciencedirect.com/science/article/abs/pii/S2352302623003770</a>

DOI - Digital Object Identifier

<a href="http://dx.doi.org/10.1016/S2352-3026(23)00377-0" target="_blank" >10.1016/S2352-3026(23)00377-0</a>

Result language

angličtina

Original language name

Diagnosis and management of pyruvate kinase deficiency: international expert guidelines

Original language description

Pyruvate kinase (PK) deficiency is the most common cause of chronic congenital non-spherocytic haemolytic anaemia worldwide, with an estimated prevalence of one in 100 000 to one in 300 000 people. PK deficiency results in chronic haemolytic anaemia, with wide ranging and serious consequences affecting health, quality of life, and mortality. The goal of the International Guidelines for the Diagnosis and Management of Pyruvate Kinase Deficiency was to develop evidence-based guidelines for the clinical care of patients with PK deficiency. These clinical guidelines were developed by use of GRADE methodology and the AGREE II framework. Experts were invited after consideration of area of expertise, scholarly contributions in PK deficiency, and country of practice for global representation. The expert panel included 29 expert physicians (including adult and paediatric haematologists and other subspecialists), geneticists, laboratory specialists, nurses, a guidelines methodologist, patients with PK deficiency, and caregivers from ten countries. Five key topic areas were identified, the panel prioritised key questions, and a systematic literature search was done to generate evidence summaries that were used in the development of draft recommendations. The expert panel then met in person to finalise and vote on recommendations according to a structured consensus procedure. Agreement of greater than or equal to 67% among the expert panel was required for inclusion of a recommendation in the final guideline. The expert panel agreed on 31 total recommendations across five key topics: diagnosis and genetics, monitoring and management of chronic complications, standard management of anaemia, targeted and advanced therapies, and special populations. These new guidelines should facilitate best practices and evidence-based PK deficiency care into clinical practice.

Czech name

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Czech description

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Type

J_imp - Article in a specialist periodical, which is included in the Web of Science database

CEP classification

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OECD FORD branch

30205 - Hematology

Project

—

Continuities

I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace

Publication year

2024

Confidentiality

S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

Name of the periodical

Lancet Haematology

ISSN

2352-3026

e-ISSN

—

Volume of the periodical

11

Issue of the periodical within the volume

3

Country of publishing house

GB - UNITED KINGDOM

Number of pages

12

Pages from-to

"e228"-"e239"

UT code for WoS article

001202131100001

EID of the result in the Scopus database

2-s2.0-85183945694