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

Hdl Handle:
http://hdl.handle.net/10147/579898
Title:
Criteria for evaluating response and outcome in clinical trials for children with juvenile myelomonocytic leukemia.
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
Affiliation:
Our Ladys Hospital Crumin, Dublin
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
Issue Date:
Jan-2015
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
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
ISSN:
1592-8721

Full metadata record

DC FieldValue Language
dc.contributor.authorNiemeyer, Charlotte Men
dc.contributor.authorLoh, Mignon Len
dc.contributor.authorCseh, Annamariaen
dc.contributor.authorCooper, Todden
dc.contributor.authorDvorak, Christopher Cen
dc.contributor.authorChan, Rebeccaen
dc.contributor.authorXicoy, Blancaen
dc.contributor.authorGerming, Ulrichen
dc.contributor.authorKojima, Seijien
dc.contributor.authorManabe, Atsushien
dc.contributor.authorDworzak, Michaelen
dc.contributor.authorDe Moerloose, Barbaraen
dc.contributor.authorStarý, Janen
dc.contributor.authorSmith, Owen Pen
dc.contributor.authorMasetti, Riccardoen
dc.contributor.authorCatala, Alberten
dc.contributor.authorBergstraesser, Evaen
dc.contributor.authorUssowicz, Mareken
dc.contributor.authorFabri, Oskanaen
dc.contributor.authorBaruchel, Andréen
dc.contributor.authorCavé, Hélèneen
dc.contributor.authorZwaan, Michelen
dc.contributor.authorLocatelli, Francoen
dc.contributor.authorHasle, Henriken
dc.contributor.authorvan den Heuvel-Eibrink, Marry Men
dc.contributor.authorFlotho, Christianen
dc.contributor.authorYoshimi, Ayamien
dc.date.accessioned2015-10-19T15:00:41Zen
dc.date.available2015-10-19T15:00:41Zen
dc.date.issued2015-01en
dc.identifier.citationCriteria for evaluating response and outcome in clinical trials for children with juvenile myelomonocytic leukemia. 2015, 100 (1):17-22 Haematologicaen
dc.identifier.issn1592-8721en
dc.identifier.pmid25552679en
dc.identifier.doi10.3324/haematol.2014.109892en
dc.identifier.urihttp://hdl.handle.net/10147/579898en
dc.description.abstractJuvenile 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.en
dc.language.isoenen
dc.publisherHaematologicaen
dc.rightsArchived with thanks to Haematologicaen
dc.subjectLEUKAEMIAen
dc.subjectCHILD HEALTHen
dc.subject.meshChilden
dc.subject.meshClinical Trials as Topicen
dc.subject.meshCombined Modality Therapyen
dc.subject.meshHumansen
dc.subject.meshLeukemia, Myelomonocytic, Juvenileen
dc.subject.meshNeoplasm Recurrence, Localen
dc.subject.meshPractice Guidelines as Topicen
dc.subject.meshPrognosisen
dc.subject.meshSurvival Rateen
dc.titleCriteria for evaluating response and outcome in clinical trials for children with juvenile myelomonocytic leukemia.en
dc.typeArticleen
dc.contributor.departmentOur Ladys Hospital Crumin, Dublinen
dc.identifier.journalHaematologicaen
dc.description.fundingNo fundingen
dc.description.provinceLeinsteren
dc.description.peer-reviewpeer-reviewen

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