Definitions, methodological, and statistical issues for phase 3
clinical trials in chronic myeloid leukemia: a proposal by the
European LeukemiaNet

J 2012

Definitions, methodological, and statistical issues for phase 3 clinical trials in chronic myeloid leukemia: a proposal by the European LeukemiaNet

GUILHOT, Joëlle; Michele BACCARANI; Richard E. CLARK; Francisco CERVANTES; François GUILHOT et al.

Základní údaje

Originální název

Definitions, methodological, and statistical issues for phase 3 clinical trials in chronic myeloid leukemia: a proposal by the European LeukemiaNet

Autoři

Vydání

Blood, Washington DC, USA, American Society of Hematology, 2012, 0006-4971

Další údaje

Jazyk

angličtina

Typ výsledku

Článek v odborném periodiku

Obor

30200 3.2 Clinical medicine

Stát vydavatele

Spojené státy

Utajení

není předmětem státního či obchodního tajemství

Impakt faktor

Impact factor: 9.060

Označené pro přenos do RIV

Ano

Kód RIV

RIV/00216224:14110/12:00059930

Organizační jednotka

Lékařská fakulta

DOI

https://doi.org/10.1182/blood-2011-10-383711

UT WoS

000307398700010

Klíčová slova anglicky

TYROSINE KINASE INHIBITORS; CHRONIC GRANULOCYTIC-LEUKEMIA; PROGRESSION-FREE SURVIVAL; INTERFERON-ALPHA; COMPETING RISKS; PROGNOSTIC DISCRIMINATION; CYTOGENETIC RESPONSE; MOLECULAR RESPONSE; RANDOMIZED-TRIALS; STI571 IRIS

Příznaky

Mezinárodní význam, Recenzováno

Změněno: 10. 2. 2013 10:22, Ing. Mgr. Věra Pospíšilíková

Anotace

V originále

The treatment policy of chronic myeloid leukemia (CML), particularly with tyrosine kinase inhibitors (TKIs), has been influenced by several recent studies that were well designed and rapidly performed, but their interpretation is of some concern because different endpoints and methodologies were used. In order to understand and compare the results of the previous and future studies and to translate their conclusion into clinical practice, there is a need for common definitions and methods for analyses of CML studies. A panel of experts was appointed by the European LeukemiaNet with the aim of developing a set of definitions and recommendations to be used in design, analyses and reporting of phase III clinical trials in this disease. This paper summarizes the consensus of the panel on events and major endpoints of interest in CML. It also focuses on specific issues concerning the intention to treat principle, and longitudinal data analyses in the context of long term follow-up. The panel proposes that future clinical trials follow these recommendations.

Citovat

GUILHOT, Joëlle; Michele BACCARANI; Richard E. CLARK; Francisco CERVANTES; François GUILHOT; Andreas HOCHHAUS; Sergei KULIKOV; Jiří MAYER; Andreas L. PETZER; Gianantonio ROSTI; Philippe ROUSSELOT; Giuseppe SAGLIO; Susanne SAUSSELE; Bengt SIMONSSON; Juan-Luis STEEGMANN; Andrey ZARITSKEY a Rüdiger HEHLMANN. Definitions, methodological, and statistical issues for phase 3 clinical trials in chronic myeloid leukemia: a proposal by the European LeukemiaNet. Blood. Washington DC, USA: American Society of Hematology, 2012, roč. 119, č. 25, s. 5963-5971. ISSN 0006-4971. Dostupné z: https://doi.org/10.1182/blood-2011-10-383711.

@article{982445,
   author = {Guilhot, Joëlle and Baccarani, Michele and Clark, Richard E. and Cervantes, Francisco and Guilhot, François and Hochhaus, Andreas and Kulikov, Sergei and Mayer, Jiří and Petzer, Andreas L. and Rosti, Gianantonio and Rousselot, Philippe and Saglio, Giuseppe and Saussele, Susanne and Simonsson, Bengt and Steegmann, JuanandLuis and Zaritskey, Andrey and Hehlmann, Rüdiger},
   article_location = {Washington DC, USA},
   article_number = {25},
   doi = {https://doi.org/10.1182/blood-2011-10-383711},
   keywords = {TYROSINE KINASE INHIBITORS; CHRONIC GRANULOCYTIC-LEUKEMIA; PROGRESSION-FREE SURVIVAL; INTERFERON-ALPHA; COMPETING RISKS; PROGNOSTIC DISCRIMINATION; CYTOGENETIC RESPONSE; MOLECULAR RESPONSE; RANDOMIZED-TRIALS; STI571 IRIS},
   language = {eng},
   issn = {0006-4971},
   journal = {Blood},
   title = {Definitions, methodological, and statistical issues for phase 3 clinical trials in chronic myeloid leukemia: a proposal by the European LeukemiaNet},
   volume = {119},
   year = {2012}
}

TY  - JOUR
ID  - 982445
AU  - Guilhot, Joëlle - Baccarani, Michele - Clark, Richard E. - Cervantes, Francisco - Guilhot, François - Hochhaus, Andreas - Kulikov, Sergei - Mayer, Jiří - Petzer, Andreas L. - Rosti, Gianantonio - Rousselot, Philippe - Saglio, Giuseppe - Saussele, Susanne - Simonsson, Bengt - Steegmann, Juan-Luis - Zaritskey, Andrey - Hehlmann, Rüdiger
PY  - 2012
TI  - Definitions, methodological, and statistical issues for phase 3 clinical trials in chronic myeloid leukemia: a proposal by the European LeukemiaNet
JF  - Blood
VL  - 119
IS  - 25
SP  - 5963-5971
EP  - 5963-5971
PB  - American Society of Hematology
SN  - 00064971
KW  - TYROSINE KINASE INHIBITORS
KW  - CHRONIC GRANULOCYTIC-LEUKEMIA
KW  - PROGRESSION-FREE SURVIVAL
KW  - INTERFERON-ALPHA
KW  - COMPETING RISKS
KW  - PROGNOSTIC DISCRIMINATION
KW  - CYTOGENETIC RESPONSE
KW  - MOLECULAR RESPONSE
KW  - RANDOMIZED-TRIALS
KW  - STI571 IRIS
N2  - The treatment policy of chronic myeloid leukemia (CML), particularly with tyrosine kinase inhibitors (TKIs), has been influenced by several recent studies that were well designed and rapidly performed, but their interpretation is of some concern because different endpoints and methodologies were used. In order to understand and compare the results of the previous and future studies and to translate their conclusion into clinical practice, there is a need for common definitions and methods for analyses of CML studies. A panel of experts was appointed by the European LeukemiaNet with the aim of developing a set of definitions and recommendations to be used in design, analyses and reporting of phase III clinical trials in this disease. This paper summarizes the consensus of the panel on events and major endpoints of interest in CML. It also focuses on specific issues concerning the intention to treat principle, and longitudinal data analyses in the context of long term follow-up. The panel proposes that future clinical trials follow these recommendations.
ER  -

GUILHOT, Jo$\backslash$''elle; Michele BACCARANI; Richard E. CLARK; Francisco CERVANTES; Fran$\backslash$c cois GUILHOT; Andreas HOCHHAUS; Sergei KULIKOV; Jiří MAYER; Andreas L. PETZER; Gianantonio ROSTI; Philippe ROUSSELOT; Giuseppe SAGLIO; Susanne SAUSSELE; Bengt SIMONSSON; Juan-Luis STEEGMANN; Andrey ZARITSKEY a Rüdiger HEHLMANN. Definitions, methodological, and statistical issues for phase 3 clinical trials in chronic myeloid leukemia: a proposal by the European LeukemiaNet. \textit{Blood}. Washington DC, USA: American Society of Hematology, 2012, roč.~119, č.~25, s.~5963-5971. ISSN~0006-4971. Dostupné z: https://doi.org/10.1182/blood-2011-10-383711.

Přehled o publikaci