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    Criteria for evaluating response and outcome in clinical trials for children with juvenile myelomonocytic leukemia.

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    Authors
    Niemeyer, Charlotte M
    Loh, Mignon L
    Cseh, Annamaria
    Cooper, Todd
    Dvorak, Christopher C
    Chan, Rebecca
    Xicoy, Blanca
    Germing, Ulrich
    Kojima, Seiji
    Manabe, Atsushi
    Dworzak, Michael
    De Moerloose, Barbara
    Starý, Jan
    Smith, Owen P
    Masetti, Riccardo
    Catala, Albert
    Bergstraesser, Eva
    Ussowicz, Marek
    Fabri, Oskana
    Baruchel, André
    Cavé, Hélène
    Zwaan, Michel
    Locatelli, Franco
    Hasle, Henrik
    van den Heuvel-Eibrink, Marry M
    Flotho, Christian
    Yoshimi, Ayami
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    Affiliation
    Our Ladys Hospital Crumin, Dublin
    Issue Date
    2015-01
    Keywords
    LEUKAEMIA
    CHILD HEALTH
    MeSH
    Child
    Clinical Trials as Topic
    Combined Modality Therapy
    Humans
    Leukemia, Myelomonocytic, Juvenile
    Neoplasm Recurrence, Local
    Practice Guidelines as Topic
    Prognosis
    Survival Rate
    
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    Show full item record
    Citation
    Criteria for evaluating response and outcome in clinical trials for children with juvenile myelomonocytic leukemia. 2015, 100 (1):17-22 Haematologica
    Publisher
    Haematologica
    Journal
    Haematologica
    URI
    http://hdl.handle.net/10147/579898
    DOI
    10.3324/haematol.2014.109892
    PubMed ID
    25552679
    Abstract
    Juvenile myelomonocytic leukemia is a rare myeloproliferative disease in young children. While hematopoietic stem cell transplantation remains the only curative therapeutic option for most patients, children with juvenile myelomonocytic leukemia increasingly receive novel agents in phase I-II clinical trials as pre-transplant therapy or therapy for relapse after transplantation. However, response criteria or definitions of outcome for standardized evaluation of treatment effect in patients with juvenile myelomonocytic leukemia are currently lacking. Here we propose criteria to evaluate the response to the non-transplant therapy and definitions of remission status after hematopoietic stem cell transplantation. For the evaluation of non-transplant therapy, we defined 6 clinical variables (white blood cell count, platelet count, hematopoietic precursors and blasts in peripheral blood, bone marrow blast percentage, spleen size and extramedullary disease) and 3 genetic variables (cytogenetic, molecular and chimerism response) which serve to describe the heterogeneous picture of response to therapy in each individual case. It is hoped that these criteria will facilitate the comparison of results between clinical trials in juvenile myelomonocytic leukemia.
    Item Type
    Article
    Language
    en
    ISSN
    1592-8721
    ae974a485f413a2113503eed53cd6c53
    10.3324/haematol.2014.109892
    Scopus Count
    Collections
    Children's Health Ireland (CHI) at Crumlin

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