QoL-CML0713: LEONIDAS: Quality of Life Study in Chronic Myeloid Leukemia Patients

Sponsor

Gruppo Italiano Malattie EMatologiche dell'Adulto (Other)

Overall Status

Unknown status

CT.gov ID

NCT02164903

Collaborator

European Leukemia Net (Other), CML Advocates Network (Other)

323

Enrollment

Locations

48.7

Anticipated Duration (Months)

6.6

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Chronic Myeloid Leukemia	Other: QoL Survey Booklet

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.

Study Design

Study Type:

Observational

Actual Enrollment :

323 participants

Observational Model:

Case-Control

Time Perspective:

Cross-Sectional

Official Title:

Mid to Long-term Quality of Life Effects Of imatiNIb Versus DASatinib in Chronic Myeloid Leukemia Patients (LEONIDAS)

Actual Study Start Date :

Sep 11, 2014

Actual Primary Completion Date :

Jan 25, 2017

Anticipated Study Completion Date :

Oct 1, 2018

Arms and Interventions

Arm	Intervention/Treatment
Imatinib Patients in first line treatment with imatinib for no more than 3 years.	Other: QoL Survey Booklet
Dasatinib Patients in first line treatment with dasatinib for no more than 3 years.	Other: QoL Survey Booklet

Arm

Intervention/Treatment

Imatinib

Patients in first line treatment with imatinib for no more than 3 years.

Other: QoL Survey Booklet

Dasatinib

Patients in first line treatment with dasatinib for no more than 3 years.

Other: QoL Survey Booklet

Outcome Measures

Primary Outcome Measures

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

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)
Proportion of patients considered as low, medium and high adheres by type of therapy. [24 months.]
(outcome measure: Morisky-Medication Adherence Scale-MMAS)
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)
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)

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

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.

Exclusion Criteria:

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.

Contacts and Locations

Locations

	Site	City	Country
1	Department of Hematology and Oncology, Hôpital Mignot, Université Versailles Saint-Quentin-en-Yvelines	Le Chesnay	France
2	Universität Heidelberg - III. Medizinische Klinik - Universitätsmedizin Mannheim	Mannheim	Germany
3	Universitat Heidelberg	Mannhein	Germany
4	S. C. di Ematologia - Azienda Ospedaliera - SS. Antonio e Biagio e Cesare Arrigo	Alessandria	Italy
5	Clinica di Ematologia - Azienda Ospedaliera Regionale di Torrette	Ancona	Italy
6	U.O. Ematologia con trapianto - Azienda Ospedaliero-Universitaria Policlinico di Bari	Bari	Italy
7	Istituto di Ematologia "L. e A. Seragnoli" - Università degli Studi di Bologna - Policlinico S. Orsola - Malpighi	Bologna	Italy
8	USD - Centro Trapianti Midollo Osseo Adulti - Cattedra di Ematologia - Azienda Spedali Civili - Brescia	Brescia	Italy
9	U.O. Ematologia Brindisi- Ospedale A. Perrino ASL BR	Brindisi	Italy
10	CTMO-Ematologia-Ospedale "Binaghi"	Cagliari	Italy
11	U.O. Ematologia CTMO "Businco"	Cagliary	Italy
12	Divisione clinicizzata di Ematologia - Dipartimento di Scienze Mediche - Osp. Ferrarotto	Catania	Italy
13	Azienda Ospedaliera - Arcispedale S. Anna Sezione di Ematologia e Fisiopatologia delle Emostasi	Ferrara	Italy
14	Divisione di Ematologia - Policlinico Careggi	Firenze	Italy
15	Clinica Ematologica - Dipartimento di Medicina Interna - IRCCS San Martino - IST	Genova	Italy
16	Divisione di Ematologia - Azienda Ospedaliera Ospedali Riuniti "Papardo Piemonte"	Messina	Italy
17	Divisione Ematologia - Dipartimento di Medicina Interna -Policlinico Universitario di Messina	Messina	Italy
18	U.O. Ematologia - Azienda USLL12 Veneziana - Ospedale dell'Angelo e Ospedale civile S. Giovanni e Paolo	Mestre - Venezia	Italy
19	U.O. Ematologia 1 - Centro Trapianti di Midollo - Ospedale Maggiore Milano	Milano	Italy
20	Dipartimento di Med. Clinica e Sperimentale- Area di Ematologia - Facoltà di Medicina e Chirurgia - Università degli Studi di Napoli "Federico II" -	Napoli	Italy
21	U.O. Ematologia - Ospedale S. Gennaro	Napoli	Italy
22	XIX Divisione di Ematologia con trapianto - A.O.R.N. "A. Cardarelli"	Napoli	Italy
23	Divisione di Ematologia - Dip. Di Medicina Clinica e Sperimentale & BRMA - Università Piemonte Orientale "Amedeo Avogato"	Novara	Italy
24	Dipartimento di Scienze Cliniche e Biologiche - Ospedale S. Luigi Gonzaga	Orbassano	Italy
25	Ematologia ed Immunologia Clinica - Università degli studi di Padova	Padova	Italy
26	Divisione di Ematologia con trapianto di midollo - A.O.U. Policlinico "Paolo Giaccone"	Palermo	Italy
27	U.O.C. Ematologia - A.O. Ospedali Riuniti "Villa Sofia-Cervello"	Palermo	Italy
28	Ematologia e CTMO - A.O.U. Università degli Studi di Parma	Parma	Italy
29	Unità Operativa Ematologia e Centro Trapianti - Dipartimento di Oncologia ed Ematologia - AUSL Ospedale di Piacenza	Piacenza	Italy
30	U.O. Ematologia - A.O.U. Pisana	Pisa	Italy
31	U.O. Ematologia - A.O. San Carlo	Potenza	Italy
32	Divisione di Ematologia - A.O. Ospedali Riuniti di Reggio Calabria "Bianchi-Melacrino-Morelli"	Reggio Calabria	Italy
33	Rimini Ospedale "Infermi"	Rimini	Italy
34	Department of Onco-Hematology - IRCCS; Centro di Riferimento Oncologico della Basilicata	Rionero in Vulture	Italy
35	Clinica di Ematologia - Policlinico Umberto I- Università degli Studi "Sapienza"	Roma	Italy
36	Divisione di Ematologia - Ospedale S.Eugenio	Roma	Italy
37	Ematologia - A.O. Sant'Andrea	Roma	Italy
38	Ematologia Policlinico - Università degli Studi di Roma Tor Vergata (PTV)	Roma	Italy
39	U.O. di Ematologia - Ente Ospedaliero San Giovanni Addolorata	Roma	Italy
40	U.O.C. Ematologia e Trapianto cellule staminali - Pad Cesalpino- A.O. San Camillo Forlanini	Roma	Italy
41	U.O. di Ematologia - Casa Sollievo della Sofferenza	San Giovanni Rotondo	Italy
42	Ematologia - AOU Sassari	Sassari	Italy
43	U.O.C. Ematologia - A.O. SS Annunziata - P.O.S.G. Moscati	Taranto	Italy
44	S.C. di Oncoematologia - A.O. "S. Maria"	Terni	Italy
45	S.C.D.O. Ematologia II - A.O.U.S. San Giovanni Battista di Torino (Molinette)	Torino	Italy
46	Clinica Ematologica ed Unità di Terapie Cellulari Carlo Melzi - A.O. Universitaria	Udine	Italy
47	Onco-Ematologia - Ospedale S. Andrea- Vercelli	Vercelli	Italy
48	U.O di Ematologia d. U. - Azienda Ospedaliera Universitaria Integrata Verona	Verona	Italy
49	Department of Hematology, Hospital Universitario de la Princesa	Madrid	Spain