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

CM. Niemeyer, ML. Loh, A. Cseh, T. Cooper, CC. Dvorak, R. Chan, B. Xicoy, U. Germing, S. Kojima, A. Manabe, M. Dworzak, B. De Moerloose, J. Starý, OP. Smith, R. Masetti, A. Catala, E. Bergstraesser, M. Ussowicz, O. Fabri, A. Baruchel, H. Cavé, M....

. 2015 ; 100 (1) : 17-22.

Jazyk angličtina Země Itálie

Typ dokumentu časopisecké články, Research Support, N.I.H., Extramural, práce podpořená grantem

Perzistentní odkaz   https://www.medvik.cz/link/bmc16000412

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.

Aflac Cancer and Blood Disorders Center Children's Healthcare of Atlanta Emory University Atlanta GA USA

Department of Genetics Hôpital Robert Debré and Paris Diderot University Paris France

Department of Hematology and Oncology University Children's Hospital Zurich Switzerland

Department of Hematology and Transfusiology Comenius University Bratislava Slovakia

Department of Hematology Hospital Germans Trias i Pujol and Institut Català d'Oncologia José Carreras Leukemia Research Institute Badalona Spain

Department of Hematology Hospital Sant Joan de Déu Barcelona Spain

Department of Hematology Oncology and Clinical Immunology Heinrich Heine University Düsseldorf Germany

Department of Pediatric Hemato Oncology Ghent University Hospital Belgium

Department of Pediatric Hematology and Oncology Bambino Gesù Children's Hospital Rome University of Pavia Italy

Department of Pediatric Hematology and Oncology Charles University and University Hospital Motol Czech Pediatric Hematology Working Group Prague Czech Republic

Department of Pediatric Hematology of Robert Debré Hospital and Paris Diderot University Paris France

Department of Pediatric Oncology and Hematology University of Bologna Italy

Department of Pediatric Oncology Hematology and BMT Wroclaw Medical University Poland

Department of Pediatrics Aarhus University Hospital Skejby Aarhus Denmark

Department of Pediatrics and Adolescent Medicine Division of Pediatric Hematology and Oncology University of Freiburg Germany

Department of Pediatrics and the Helen Diller Comprehensive Cancer Center University of California San Francisco CA USA

Department of Pediatrics Nagoya University Graduate School of Medicine Japan

Department of Pediatrics St Luke's International Hospital Tokyo Japan

Department of Pediatrics The Herman B Wells Center for Pediatric Research Indiana University School of Medicine Indianapolis IN USA

Division of Pediatric Allergy Immunology and Bone Marrow Transplant Benioff Children's Hospital University of California San Francisco CA USA

ErasmusMC Sophia Children's Hospital Erasmus Medical Center Rotterdam and Dutch Childhood Oncology Group The Hague The Netherlands

ErasmusMC Sophia Children's Hospital Erasmus Medical Center Rotterdam and the Netherlands and ITCC

Pediatric Oncology and Hematology Our Lady's Children's Hospital Crumlin Dublin Ireland

St Anna Children's Hospital and Children's Cancer Research Institute Department of Pediatrics Medical University of Vienna Austria

Citace poskytuje Crossref.org

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$a Criteria for evaluating response and outcome in clinical trials for children with juvenile myelomonocytic leukemia / $c CM. Niemeyer, ML. Loh, A. Cseh, T. Cooper, CC. Dvorak, R. Chan, B. Xicoy, U. Germing, S. Kojima, A. Manabe, M. Dworzak, B. De Moerloose, J. Starý, OP. Smith, R. Masetti, A. Catala, E. Bergstraesser, M. Ussowicz, O. Fabri, A. Baruchel, H. Cavé, M. Zwaan, F. Locatelli, H. Hasle, MM. van den Heuvel-Eibrink, C. Flotho, A. Yoshimi,
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