Criteria for evaluating response and outcome in clinical trials for children with juvenile myelomonocytic leukemia.
Authors
Niemeyer, Charlotte MLoh, 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
Affiliation
Our Ladys Hospital Crumin, DublinIssue Date
2015-01Keywords
LEUKAEMIACHILD HEALTH
MeSH
ChildClinical Trials as Topic
Combined Modality Therapy
Humans
Leukemia, Myelomonocytic, Juvenile
Neoplasm Recurrence, Local
Practice Guidelines as Topic
Prognosis
Survival Rate
Metadata
Show full item recordCitation
Criteria for evaluating response and outcome in clinical trials for children with juvenile myelomonocytic leukemia. 2015, 100 (1):17-22 HaematologicaPublisher
HaematologicaJournal
HaematologicaDOI
10.3324/haematol.2014.109892PubMed ID
25552679Abstract
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
ArticleLanguage
enISSN
1592-8721ae974a485f413a2113503eed53cd6c53
10.3324/haematol.2014.109892
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