LEONIDAS: Quality of Life Study in Chronic Myeloid Leukemia Patients
- Conditions
- Chronic Myeloid Leukemia
- Interventions
- Other: QoL Survey Booklet
- Registration Number
- NCT02164903
- Brief Summary
The broad goal of this study is to investigate if differences exist (and in which areas and of what magnitude) in QoL and symptoms of patients with CML being treated with first line therapy with dasatinib versus those receiving first line therapy with imatinib. Also, an additional objective is to characterize medication-taking behavior associated with imatinib or dasatinib.
- Detailed Description
The development of molecular targeted therapies (i.e., oral tyrosine kinase inhibitors \[TKIs\]) to treat chronic myeloid leukemia (CML) is one of the great triumphs of modern oncology and one of the spearheads of personalized medicine. Since their introduction in 2001, the number of people living with CML has doubled, a trend that is expected to continue.
This study (i.e., LEONIDAS) is set up to generate evidence-based data and produce information that will advance scientific knowledge, clinical practice and health services management to facilitate clinical decision-making in CML.
In an effort to further promote patient-centered care for CML a patient advocacy organization, that is the: CML Advocates Network, is involved in this project with key representatives who will be in the advisory board team. The CML Advocates Network, hosted by the patient-run, non-profit Leukemia Patient Advocates Foundation is a worldwide network of 82 non-profit organizations from 63 countries supporting patients with CML and their relatives.
Rationale and Significance Some ten years ago, the treatment of CML was relatively straightforward as all patients received imatinib as first-line treatment and for those who failed imatinib, the only available proven alternative was allogeneic stem cell transplantation. Nowadays, with three effective drugs (i.e., imatinib, nilotinib and dasatinib), that can be used frontline in newly diagnosed chronic phase (CP) CML patients, treatment decision-making for individual patients in daily clinical practice has thus rapidly grown in complexity. Moreover, a new tyrosine kinase inhibitors (TKI), bosutinib, has been recently approved for as second-line treatment and undergoing clinical trials are assessing its efficacy as frontline strategy in CML.
To further complicate the scenario, is the fact that despite nilotinib and dasatinib have been shown to have higher rates of cytogenetic and molecular responses (compared to imatinib), none of these three drugs is dramatically better than the others. To illustrate, only slightly differences exist in terms of progression-free survival for nilotinib and no differences for overall survival at 24 and/or 36 months amongst imatinib, dasatinib and nilotinib.
Nevertheless, physician-reported toxicity data suggests these drugs have different toxicity profiles, with imatinib inducing a higher proportion of low-grade side effects than second generation tyrosine kinase inhibitors (TKI) (i.e. nilotinib and dasatinib).
While a wealth of biomedical and laboratory data exists on clinical efficacy of these three drugs, the impact of these from the patients' perspective, in terms of Quality of Life (QoL) and symptom burden, has been poorly investigated. No data exists on QoL of patients treated with nilotinib or dasatinib and it is not known if these drugs provide better QoL outcomes over imatinib when used as first line therapy. Such information would be critical in the current CML arena to make more informed treatment decisions. This is a significant gap in our knowledge with respect to the modern management of CML, also because, as long-term continuous exposure to the tyrosine kinase inhibitors (TKI) is necessary, even low grade side effects can heavily impact on patients' QoL.
Thus, the objective of this study is to investigate if differences exist (and in which areas and of what magnitude) in QoL and symptoms of patients with CML being treated with first line therapy with dasatinib versus those receiving first line therapy with imatinib, as well as characterizing medication-taking behavior associated with imatinib or dasatinib.
Recruitment & Eligibility
- Status
- UNKNOWN
- Sex
- All
- Target Recruitment
- 323
- 18 years or older at the time of study entry;
- Diagnosis of Philadelphia chromosome positive and/or BCR-ABL positive CML confirmed by cytogenetic and/or molecular analysis;
- At least in CCyR (as documented by chromosome banding analysis of marrow cell metaphases) or in MMR (≤0.1% BCR-ABL IS)
- CP-CML Patients in first line treatment with either dasatinib or imatinib for no more than 3 years;
- Written informed consent.
- Major cognitive deficits or psychiatric problems hampering a self-reported evaluation.
- Not speaking and reading the language of the participating country.
- Having received any CML treatment prior to therapy with imatinib or dasatinib for more than three months.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Dasatinib QoL Survey Booklet Patients in first line treatment with dasatinib for no more than 3 years. Imatinib QoL Survey Booklet Patients in first line treatment with imatinib for no more than 3 years.
- Primary Outcome Measures
Name Time Method Severity of the impact of therapy on daily life in patients with CP-CML being treated with first line therapy with dasatinib and those receiving first line therapy with imatinib. 24 months. (outcome measure: European Organization for Research and EORTC QLQ-CML24 "Impact on daily life" scale)
- Secondary Outcome Measures
Name Time Method Relationship between patient's perception of disease-/treatment-related information received , QoL profile and adherence to therapy. 24 months. (defined according to the EORTC-QLQ-INFO25)
Proportion of patients considered as low, medium and high adheres by type of therapy. 24 months. (outcome measure: Morisky-Medication Adherence Scale-MMAS)
Degree of agreement in the assessment of symptom severity, for a set of core CML treatment related symptoms , between patients and their treating physicians. 24 months. (according to the EORTC-QLQ-CML24)
Differences in Patient-reported outcomes between imatinib- and dasatinib- treated CP- CML patients in the following QoL domains: Fatigue, Physical, Social and Role Functioning and Impact on worry and mood and Symptom burden. 24 months. (outcome measure: EORTC QLQ-C30 and EORTC QLQ-CML24)
Trial Locations
- Locations (49)
U.O. Ematologia 1 - Centro Trapianti di Midollo - Ospedale Maggiore Milano
🇮🇹Milano, Italy
Dipartimento di Med. Clinica e Sperimentale- Area di Ematologia - Facoltà di Medicina e Chirurgia - Università degli Studi di Napoli "Federico II" -
🇮🇹Napoli, Italy
Dipartimento di Scienze Cliniche e Biologiche - Ospedale S. Luigi Gonzaga
🇮🇹Orbassano, Italy
Clinica di Ematologia - Policlinico Umberto I- Università degli Studi "Sapienza"
🇮🇹Roma, Italy
XIX Divisione di Ematologia con trapianto - A.O.R.N. "A. Cardarelli"
🇮🇹Napoli, Italy
Divisione di Ematologia con trapianto di midollo - A.O.U. Policlinico "Paolo Giaccone"
🇮🇹Palermo, Italy
Ematologia - A.O. Sant'Andrea
🇮🇹Roma, Italy
S.C.D.O. Ematologia II - A.O.U.S. San Giovanni Battista di Torino (Molinette)
🇮🇹Torino, Italy
U.O.C. Ematologia e Trapianto cellule staminali - Pad Cesalpino- A.O. San Camillo Forlanini
🇮🇹Roma, Italy
U.O. Ematologia - Ospedale S. Gennaro
🇮🇹Napoli, Italy
Department of Hematology, Hospital Universitario de la Princesa
🇪🇸Madrid, Spain
Ematologia ed Immunologia Clinica - Università degli studi di Padova
🇮🇹Padova, Italy
Rimini Ospedale "Infermi"
🇮🇹Rimini, Italy
Ematologia - AOU Sassari
🇮🇹Sassari, Italy
U.O.C. Ematologia - A.O. SS Annunziata - P.O.S.G. Moscati
🇮🇹Taranto, Italy
Onco-Ematologia - Ospedale S. Andrea- Vercelli
🇮🇹Vercelli, Italy
U.O di Ematologia d. U. - Azienda Ospedaliera Universitaria Integrata Verona
🇮🇹Verona, Italy
U.O. di Ematologia - Casa Sollievo della Sofferenza
🇮🇹San Giovanni Rotondo, Italy
Clinica Ematologica ed Unità di Terapie Cellulari Carlo Melzi - A.O. Universitaria
🇮🇹Udine, Italy
Universität Heidelberg - III. Medizinische Klinik - Universitätsmedizin Mannheim
🇩🇪Mannheim, Germany
Universitat Heidelberg
🇩🇪Mannhein, Germany
S. C. di Ematologia - Azienda Ospedaliera - SS. Antonio e Biagio e Cesare Arrigo
🇮🇹Alessandria, Italy
Clinica di Ematologia - Azienda Ospedaliera Regionale di Torrette
🇮🇹Ancona, Italy
U.O. Ematologia con trapianto - Azienda Ospedaliero-Universitaria Policlinico di Bari
🇮🇹Bari, Italy
Istituto di Ematologia "L. e A. Seragnoli" - Università degli Studi di Bologna - Policlinico S. Orsola - Malpighi
🇮🇹Bologna, Italy
USD - Centro Trapianti Midollo Osseo Adulti - Cattedra di Ematologia - Azienda Spedali Civili - Brescia
🇮🇹Brescia, Italy
U.O. Ematologia Brindisi- Ospedale A. Perrino ASL BR
🇮🇹Brindisi, Italy
U.O. Ematologia CTMO "Businco"
🇮🇹Cagliary, Italy
CTMO-Ematologia-Ospedale "Binaghi"
🇮🇹Cagliari, Italy
Divisione clinicizzata di Ematologia - Dipartimento di Scienze Mediche - Osp. Ferrarotto
🇮🇹Catania, Italy
Azienda Ospedaliera - Arcispedale S. Anna Sezione di Ematologia e Fisiopatologia delle Emostasi
🇮🇹Ferrara, Italy
Divisione di Ematologia - Policlinico Careggi
🇮🇹Firenze, Italy
Clinica Ematologica - Dipartimento di Medicina Interna - IRCCS San Martino - IST
🇮🇹Genova, Italy
Divisione di Ematologia - Azienda Ospedaliera Ospedali Riuniti "Papardo Piemonte"
🇮🇹Messina, Italy
Divisione Ematologia - Dipartimento di Medicina Interna -Policlinico Universitario di Messina
🇮🇹Messina, Italy
U.O. Ematologia - Azienda USLL12 Veneziana - Ospedale dell'Angelo e Ospedale civile S. Giovanni e Paolo
🇮🇹Mestre - Venezia, Italy
Divisione di Ematologia - Dip. Di Medicina Clinica e Sperimentale & BRMA - Università Piemonte Orientale "Amedeo Avogato"
🇮🇹Novara, Italy
U.O.C. Ematologia - A.O. Ospedali Riuniti "Villa Sofia-Cervello"
🇮🇹Palermo, Italy
Ematologia e CTMO - A.O.U. Università degli Studi di Parma
🇮🇹Parma, Italy
Unità Operativa Ematologia e Centro Trapianti - Dipartimento di Oncologia ed Ematologia - AUSL Ospedale di Piacenza
🇮🇹Piacenza, Italy
U.O. Ematologia - A.O.U. Pisana
🇮🇹Pisa, Italy
U.O. Ematologia - A.O. San Carlo
🇮🇹Potenza, Italy
Divisione di Ematologia - Ospedale S.Eugenio
🇮🇹Roma, Italy
U.O. di Ematologia - Ente Ospedaliero San Giovanni Addolorata
🇮🇹Roma, Italy
Divisione di Ematologia - A.O. Ospedali Riuniti di Reggio Calabria "Bianchi-Melacrino-Morelli"
🇮🇹Reggio Calabria, Italy
Department of Onco-Hematology - IRCCS; Centro di Riferimento Oncologico della Basilicata
🇮🇹Rionero in Vulture, Italy
Ematologia Policlinico - Università degli Studi di Roma Tor Vergata (PTV)
🇮🇹Roma, Italy
S.C. di Oncoematologia - A.O. "S. Maria"
🇮🇹Terni, Italy
Department of Hematology and Oncology, Hôpital Mignot, Université Versailles Saint-Quentin-en-Yvelines
🇫🇷Le Chesnay, France