ALBERT

Description: Abstract 3132 Background The treatment of chronic myeloid leukemia (CML) has changed dramatically over the last decade with the advent of targeted therapies. Health related quality of life (HRQOL) is now an important treatment otucome for this population but there is lack of internationally validated HRQOL measures for CML patients. Aims The main objective of this paper is to present results of the cross cultural initial development of an EORTC HRQOL questionnaire for patients with CML. Methods The development is following the EORTC Quality of Life Group (QLG) module development guidelines (consisting of 4 phases) as well as the Food and Drug Administration (FDA) international recommendations for developing patient-reported outcome instruments. According to phases I and II of EORTC QLG module development, an extensive literature search on potentially relevant HRQOL issues for CML patients was conducted. In addition, patient and CML expert evaluations were performed on an international basis. For the purpose of this project, and in an effort to further strength content validity of the questionnaire from the very beginning, a CML Patient advocacy group was also involved. Results More than 600 CML related articles were scrutinized and an initial list of 74 issues, including potentially relevant HRQOL aspects for CML patients, was drafted. Overall 236 CML patients provided their input on the initial list of items. 137 CML patients were interviewed in the following countries: (Italy: 67; Germany: 45; Taiwan: 10; Iraq: 8 and Greece: 7) and 99 patients provided their input through a national patient advocacy website. The data stemming from this latter group were used for supportive analyses only. In addition 59 experts (mainly physicians) were also interviewed on possible relevance of issues. At the time of interviews patients were receiving the following treatments: Imatinib (58%), Nilotinib (25%), Dasatinib (13%). Fifty-two percent were in treatment for more than 5 years ensuring that our sample included a reasonable percentage of long-term cancer survivors. Based on analysis of mean scores, prevalence ratio, range and priority ratings given by patients and experts the initial list of 74 issues was reduced to 30 items grouped in the following scales: symptoms, psychosocial aspects, adherence to therapy, infertility, body image and satisfaction with care. Conclusions A list of 30 items has been derived based on this initial development process (phase I and II according to the EORTC QLG guidelines) and this is now being further tested in a larger CML sample. Eventually, we expect this EORTC QoL-CML questionnaire to be used as a key outcome measure in future clinical trials of novel therapies for CML patients. Disclosures: No relevant conflicts of interest to declare.

Description: Abstract 4236 BACKGROUND: Tyrosine kinase inhibitors (TKIs) have revolutionized treatment of chronic myeloid leukemia (CML) patients and quality of life (QoL) data can be of crucial importance in the current CML arena to make more informed treatment decisions. To date, a number of biomedical and laboratory data have been accumulated on clinical efficacy and toxicity of various TKIs; however, there is paucity of data on the impact of TKIs on patient outcomes. AIMS: The main objective of this systematic review is thus to quantify and to summarize all studies that have included QoL, or any other type of patient-reported outcomes (PROs) in patients with CML treated with TKIs. METHODS: A systematic review was performed, following the Cochrane methodology on all studies conducted in CML that have assessed QoL or any other type of PRO (e.g., symptom burden). The search was conducted on all full length manuscripts published up to November 2012. Candidate articles were identified mainly by PubMed, the Cochrane Library, PsycINFO and PsyArticles. Criteria for selection of studies were as follow. Types of participants: Patients diagnosed with CML, regardless of patients' age and the stage of the disease. Types of intervention: All treatments with TKIs, either used alone or in combination with other drugs. Types of outcome measures examined: Any studies including PROs were considered. Studies addressing adherence to therapy were not included. Types of studies: All type of studies were considered regardless of the design (e.g., prospective or cross-sectional study). No restriction in the number of enrolled patients or type of analysis (e.g. qualitative or quantitative) was applied. Two reviewers independently evaluated all candidate abstracts retrieved from electronic databases based on the above selection criteria. Extracted data from full length manuscripts were crosschecked and discrepancies resolved by consensus. The reviewers abstracted a number of basic features of the studies, including the type of treatment, the measures used to assess QoL and clinical characteristics of patients enrolled. Also, a summary of main PROs findings was provided. RESULTS: Six studies, enrolling overall 2171 CML patients, were identified up to November 2012. None of these studies were published before 2003. Out of six studies, two were conducted on a national level and four recruited patients in an international setting. Four studies reported QoL data of patients treated with imatinib, one on bosutinib and the other one included patients receiving various TKIs. QoL of patients younger than 60 years, who are in treatment with long-term imatinib therapy is greatly impaired when compared with that of their peers in the general population. This study suggests that although the less toxic profile of TKIs therapies is unquestionable, still much has to be done to further improve patient's QoL and reduce symptom burden. Also, another study suggested that fatigue is the main factor influencing QoL regardless of the type of TKIs. Some data also indicates that physicians might underestimate the importance of symptoms. Another study, conducted in patients treated with bosutinib, showed that QoL profile of patients who have failed first line Imatinib therapy, due to either resistance or intolerance, is not different. Second line therapy with bosutinb provide clinically meaningful QoL improvements in imatinib-intolerant patients (but not in imatinib-resistant patients). Remarkably, no study was identified measuring QoL, or any other type of PROs, in patients treated with dasatinib or nilotinib. Two studies reported a gender effect, showing that male tend to report better QoL outcomes than female patients. CONCLUSIONS: This systematic review revealed the paucity of evidence base data in this area. However, QoL assessments in these CML studies emphasize the unique information provided by the patient's perspective on the burden of the disease and treatment. Investigators are encouraged to include PROs in future CML studies to obtain additional meaningful data to make more informed treatment decisions. Disclosures: Efficace: Novartis: Research Funding; Bristol Myers Squibb: Honoraria.