Clinical trial NCT01252589: Patient Reported Outcomes in Chronic Myeloid Leukemia
Official title: Patient Reported Outcomes in Chronic Myeloid Leukemia. GIMEMA QoL - CML0310.
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Chronic Myeloid Leukemia
Keywords: Chronic Myeloid Leukemia
Quality of Life
Symptoms
Patient-Reported outcomes
Questionnaire
Lead sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto
Sponsor class: OTHER
First posted: 2010-12-03
Enrollment: 480
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: CROSS_SECTIONAL
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Adult patients (18 years of age or older) with confirmed diagnosis of Philadelphia chromosome positive CML undergoing all possible treatments available.

Brief summary: Although the impact of disease and treatment related burden on health-related quality of life (HRQOL) in patients with solid tumours has been well studied, with several clinical trials that included HRQOL as an endpoint, the general understanding in patients with Chronic Myeloid Leukaemia (CML) is lacking in comparison.

The literature shows that patients' perspective is unique and should always be measured with methodologically sound instruments that are devised for this purpose.

The main scope of this project is develop to an international validated questionnaire for the purpose of HRQOL assessment; such a tool will then be used to provide important data, from the patients' perspective, to make more informed treatment decisions.

Detailed description: The development process will follow the international guidelines published by the EORTC Quality of Life Group (QLG). This process will follow three main phases.

Phase 1 of will deal with the review of the literature, identification of an item list and interviews with experts in the treatment of CML and CML patients (undergoing all possible treatments).

In Phase II the identified issues from Phase I will be worded and translated into possible items for the provisional questionnaire using existing EORTC questionnaires and the EORTC QLG Item Bank.

In the Phase III the provisional CML questionnaire will be pre-tested by to a larger sample of CML patients (not the same patients involved in previous phases) and individual structured interviews will be conducted as well. These latter will identify questions that might be irrelevant or whether there will be additional issues not previously included.

Overall up to 480 patients will be enrolled in this study (up to 180 in phase I and up to 300 in phase III).

Eligibility criteria: Inclusion Criteria:

* Adult patients (18 years of age or older)
* Confirmed diagnosis of Philadelphia chromosome positive CML
* Informed consent provided
* Patients enrolled in investigational treatment trials are eligible
* Ability to speak and read language of the Questionnaire

Exclusion Criteria:

* Freedom from overt cognitive impairment or major psychiatric conditions that may confound HRQOL evaluation

Primary outcomes:
measure: Development of an EORTC questionnaire to assess HRQOL of patients with CML.
timeFrame: By the end of the study.
Secondary outcomes:
measure: Comparison between physicians' perception of relevance of HRQOL issues with that of patients.
timeFrame: By the end of the study.
measure: Development of an EORTC CML symptom checklist.
timeFrame: By the end of the study.
measure: HRQOL in CML patients undergoing 2nd line treatment with TKIs.
timeFrame: By the end of the study.
measure: Decision making process for choosing between different 2nd line treatments with TKIs.
timeFrame: By the end of the study.
Other outcomes:
Locations:
city: Boston
country: United States
facility: Dana-Farber Cancer Institute Harvard University
state: Massachusetts
city: Innsbruck
country: Austria
facility: Innsbruck University Hospital
city: Ghent
country: Belgium
facility: University of Ghent
city: Poitiers
country: France
facility: Centre Hospitalier Universitaire of Poitiers
city: Heidelberg
country: Germany
facility: University of Heidelberg
city: Athens
country: Greece
facility: University of Athens
city: Baghdad
country: Iraq
facility: University of Baghdad
city: Bologna
country: Italy
facility: Policlinico S. Orsola - Malpighi, Università di Bologna
city: Cagliari
country: Italy
facility: Ematologia Ospedale "Binaghi", Cagliari
city: Meldola
country: Italy
facility: Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori- IRST
city: Ravenna
country: Italy
facility: Ospedale S.Maria delle Croci
city: Roma
country: Italy
facility: Ematologia - Sapienza Università di Roma
city: Tilburg
country: Netherlands
facility: Tilburg University
city: Taipei
country: Taiwan
facility: National Taiwan University
Study officials:
affiliation: GIMEMA DATA CENTER, ROME, ITALY
name: Fabio Efficace, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Efficace F, Baccarani M, Breccia M, Saussele S, Abel G, Caocci G, Guilhot F, Cocks K, Naeem A, Sprangers M, Oerlemans S, Chie W, Castagnetti F, Bombaci F, Sharf G, Cardoni A, Noens L, Pallua S, Salvucci M, Nicolatou-Galitis O, Rosti G, Mandelli F. International development of an EORTC questionnaire for assessing health-related quality of life in chronic myeloid leukemia patients: the EORTC QLQ-CML24. Qual Life Res. 2014 Apr;23(3):825-36. doi: 10.1007/s11136-013-0523-5. Epub 2013 Sep 13.