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

(2015) HAEMATOLOGICA. 100(1). p.17-22
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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.
Keywords
DISEASE, STEM-CELL TRANSPLANTATION, AZACITIDINE, PTPN11, MUTATIONS, JMML, RETROSPECTIVE ANALYSIS, MYELODYSPLASTIC SYNDROMES, INTERNATIONAL WORKING GROUP, CHRONIC MYELOGENOUS LEUKEMIA

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Citation

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Chicago
Niemeyer, Charlotte M, Mignon L Loh, Annamaria Cseh, Todd Cooper, Christopher C Dvorak, Rebecca Chan, Blanca Xicoy, et al. 2015. “Criteria for Evaluating Response and Outcome in Clinical Trials for Children with Juvenile Myelomonocytic Leukemia.” Haematologica 100 (1): 17–22.
APA
Niemeyer, C. M., Loh, M. L., Cseh, A., Cooper, T., Dvorak, C. C., Chan, R., Xicoy, B., et al. (2015). Criteria for evaluating response and outcome in clinical trials for children with juvenile myelomonocytic leukemia. HAEMATOLOGICA, 100(1), 17–22.
Vancouver
1.
Niemeyer CM, Loh ML, Cseh A, Cooper T, Dvorak CC, Chan R, et al. Criteria for evaluating response and outcome in clinical trials for children with juvenile myelomonocytic leukemia. HAEMATOLOGICA. 2015;100(1):17–22.
MLA
Niemeyer, Charlotte M, Mignon L Loh, Annamaria Cseh, et al. “Criteria for Evaluating Response and Outcome in Clinical Trials for Children with Juvenile Myelomonocytic Leukemia.” HAEMATOLOGICA 100.1 (2015): 17–22. Print.
@article{5839638,
  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.},
  author       = {Niemeyer, Charlotte M and Loh, Mignon L and Cseh, Annamaria and Cooper, Todd and Dvorak, Christopher C and Chan, Rebecca and Xicoy, Blanca and Germing, Ulrich and Kojima, Seiji and Manabe, Atsushi and Dworzak, Michael and De Moerloose, Barbara and Star{\'y}, Jan and Smith, Owen P and Masetti, Riccardo and Catala, Albert and Bergstraesser, Eva and Ussowicz, Marek and Fabri, Oskana and Baruchel, Andr{\'e} and Cav{\'e}, H{\'e}l{\`e}ne and Zwaan, Michel and Locatelli, Franco and Hasle, Henrik and Van Den Heuvel-Eibrink, Marry M and Flotho, Christian and Yoshimi, Ayami},
  issn         = {0390-6078},
  journal      = {HAEMATOLOGICA},
  language     = {eng},
  number       = {1},
  pages        = {17--22},
  title        = {Criteria for evaluating response and outcome in clinical trials for children with juvenile myelomonocytic leukemia},
  url          = {http://dx.doi.org/10.3324/haematol.2014.109892},
  volume       = {100},
  year         = {2015},
}

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