Thumbnail Image
Publication

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

Niemeyer, Charlotte M
; 
Loh, Mignon L
; 
Cseh, Annamaria
; 
Cooper, Todd
; 
Dvorak, Christopher C
; 
Chan, Rebecca
; 
Xicoy, Blanca
; 
Germing, Ulrich
; 
Kojima, Seiji
; 
Manabe, Atsushi
... show 10 more
Author
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
Advisors
Editors
Other Contributors
Departments
Our Ladys Hospital Crumin, Dublin
Date
2015-01
Date Submitted
Keywords
LEUKAEMIA
CHILD HEALTH
Other Subjects
Subject Mesh
Child
Clinical Trials as Topic
Combined Modality Therapy
Humans
Leukemia, Myelomonocytic, Juvenile
Neoplasm Recurrence, Local
Practice Guidelines as Topic
Prognosis
Survival Rate
Planned Date
Start Date
Collaborators
Principal Investigators
Files
Thumbnail Image
Main Article
Adobe PDF, 181.53 KB
Alternative Titles
Publisher
Haematologica
URI
https://hdl.handle.net/10147/579898
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.
Language
en
Citation
Criteria for evaluating response and outcome in clinical trials for children with juvenile myelomonocytic leukemia. 2015, 100 (1):17-22 Haematologica
ISSN
1592-8721
eISSN
ISBN
DOI
10.3324/haematol.2014.109892
PMID
25552679
PMCID
Sponsorships
Funding Sources
Funding Amounts
Grant Identifiers
Methodology
Duration
Ethical Approval