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Genetic and clinical characteristics of patients with Shwachman Diamond syndrome with special consideration of treatment with granulocyte-colony stimulating factor

The result's identifiers

  • Result code in IS VaVaI

    <a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00064203%3A_____%2F25%3A10496713" target="_blank" >RIV/00064203:_____/25:10496713 - isvavai.cz</a>

  • Alternative codes found

    RIV/00216208:11130/25:10496713

  • Result on the web

    <a href="https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=8tldGBvjLW" target="_blank" >https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=8tldGBvjLW</a>

  • DOI - Digital Object Identifier

    <a href="http://dx.doi.org/10.3324/haematol.2024.286119" target="_blank" >10.3324/haematol.2024.286119</a>

Alternative languages

  • Result language

    angličtina

  • Original language name

    Genetic and clinical characteristics of patients with Shwachman Diamond syndrome with special consideration of treatment with granulocyte-colony stimulating factor

  • Original language description

    Shwachman Diamond syndrome (SDS) is a rare disorder characterized by exocrinepancreatic insufficiency, bone marrow failure (BMF), congenital abnormalities including theskeleton and a varying degree of neurodevelopmental delay). Patients have apredisposition for myeloid malignancies (myelodysplastic syndrome (MDS) and acutemyeloid leukemia (AML)).Our current study has shown that despite of genetic homogeneity, there arevariable clinical and hematological phenotypes in SDS patients. There is an increasing risk ofbone marrow failure with age. G-CSF therapy does not increase the risk of developingMDS/leukemia and is therefore safe to use in SDS patients with severe neutropenia.

  • Czech name

  • Czech description

Classification

  • Type

    J<sub>imp</sub> - Article in a specialist periodical, which is included in the Web of Science database

  • CEP classification

  • OECD FORD branch

    30205 - Hematology

Result continuities

  • Project

  • Continuities

    I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace

Others

  • Publication year

    2025

  • Confidentiality

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

Data specific for result type

  • Name of the periodical

    Haematologica

  • ISSN

    0390-6078

  • e-ISSN

    1592-8721

  • Volume of the periodical

    110

  • Issue of the periodical within the volume

    9

  • Country of publishing house

    IT - ITALY

  • Number of pages

    5

  • Pages from-to

    2171-2175

  • UT code for WoS article

    001573786100028

  • EID of the result in the Scopus database

    2-s2.0-105014651855