The EMPATHY Pilot Study

NCT ID: NCT04384848

Last Updated: 2024-03-04

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 94 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-11-01
• Study Completion Date: 2022-07-31

Brief Summary

The proposed study, may significantly contribute to improve healthcare delivery in patients with Chronic Myeloid Leukemia (CML) treated with modern tyrosine kinase inhibitors (TKIs) in two ways. First, it may provide novel empirical data on the positive effects of systematically monitoring of patient-reported adverse events (AEs) in routine practice for improving symptom management and adherence to therapy. Second, it will inform the development of a large international randomized controlled trial (RCT) to test whether systematic collection of patient-reported AEs, could improve clinical response to TKI therapy.

Detailed Description

The evolution in the understanding of the biology of Chronic Myeloid Leukemia (CML), that eventually translated into highly effective molecular targeted therapies, is unparalleled in cancer medicine. This knowledge led the development of a number of orally active molecule tyrosine kinase inhibitors (TKIs) that have dramatically improved clinical outcomes. In less than two decades, the 10-year survival probability increased from 20 to 53% with previous (IFN)-therapies to about 90% in the TKI era. Life expectancy of these patients now approaches that of the general population. While oral TKIs are now the standard of care for CML, it should be considered that therapy is lifelong and patients are requested to take medication on a daily basis. Importantly, there is convincing evidence that full adherence to therapy is a critical factor to obtain and maintain an optimal response to therapy. However, non-adherence is a major challenge in CML, since side effects induced by these drugs negatively impact on patient's quality of life (QoL), seriously undermine full adherence with treatment schedule and thereby often lead to sub-optimal clinical responses to drugs. From previous Preliminary Data we extrapolated the following evidences: Systematic monitoring of Patient-Reported Outcomes (PRO) in routine care has several advantages, such as improved symptom control, enhanced patient-physician communication, as well as patient satisfaction and wellbeing. Furthermore, it was found that Adverse Events (AEs) are the most frequent cause for non-adherence to CML therapy and that even experienced physicians tend to underestimate burden of TKIs of their patients. This mismatch might have major clinical implications in disease management, as physicians might not be able to early identify those patients who might be at heightened risk of poor adherence behavior. Given these findings, we hypothesized that systematic electronic monitoring of Patient-Reported AEs in CML routine practice may improve adherence to therapy, quality of life, and clinical response to therapy. This hypothesis will be addressed in the experiments of the following Specific Aims: 1) To develop an online platform for systematic monitoring of patient-reported AE assessment that is tailored to the unique demands of TKI therapy for CML. 2) To assess patient and physician acceptability and satisfaction with use of this platform in CML routine practice and evaluate its value in improving symptom management, quality of life, adherence to therapy as well as preliminary efficacy. We anticipate this study will provide unprecedented information on the value of systematically collecting patient-reported AEs information in CML routine care. If positive, our results will inform the development of large international randomized controlled trial (RCT) to investigate whether this experimental approach can improve depth and rates of clinical responses to TKI therapies in CML patients.

Dr. Fabio Efficace is also a Principal Investigator on this study. He is also a Professor in the Department of Medical Social Sciences at Northwestern University, Feinberg School of Medicine.

Conditions

Leukemia, Chronic Myeloid; Cancer

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: SUPPORTIVE_CARE
• Blinding Strategy: NONE

Study Groups

Chronic myeloid leukemia (CML) patients

CML patients undergoing first-line tyrosine kinase inhibitor (TKI) therapy

Group Type: EXPERIMENTAL

EMPATHY Pilot

Intervention Type: OTHER

The goal of this pilot is to develop and pilot a tailored monitoring intervention targeting symptomatic, Patient-reported adverse events (AEs) in chronic myeloid leukemia (CML) Patients undergoing first-line tyrosine kinase inhibitor (TKI) therapy. The tailored monitoring intervention will draw primarily from the PRO-CTCAE Item Library, with additional items drawn from the FACIT and EORTC Item libraries as necessary. After identifying the full set of AEs to be monitored, we will load the Patient assessment and report program into tablets for electronic administration in the busy clinic setting. We will then pilot a six-month intervention aimed to monitor and manage emerging AEs, coupled with assessment of intervention feasibility, Patient acceptability and satisfaction, provider acceptability and clinical management utility, adherence to TKI therapy, and HRQoL. We expect that adherence to therapy, clinical response, and HRQoL will be enhanced by such an intervention.

Interventions

EMPATHY Pilot

The goal of this pilot is to develop and pilot a tailored monitoring intervention targeting symptomatic, Patient-reported adverse events (AEs) in chronic myeloid leukemia (CML) Patients undergoing first-line tyrosine kinase inhibitor (TKI) therapy. The tailored monitoring intervention will draw primarily from the PRO-CTCAE Item Library, with additional items drawn from the FACIT and EORTC Item libraries as necessary. After identifying the full set of AEs to be monitored, we will load the Patient assessment and report program into tablets for electronic administration in the busy clinic setting. We will then pilot a six-month intervention aimed to monitor and manage emerging AEs, coupled with assessment of intervention feasibility, Patient acceptability and satisfaction, provider acceptability and clinical management utility, adherence to TKI therapy, and HRQoL. We expect that adherence to therapy, clinical response, and HRQoL will be enhanced by such an intervention.

Intervention Type: OTHER

Eligibility Criteria

Inclusion Criteria

1. Diagnosis of Philadelphia chromosome positive and/or BCR-ABL positive CML confirmed by cytogenetic and/or molecular analysis;

2. Newly diagnosed chronic phase (CP)-CML Patients planned to receive one of the following TKI approved as first line treatment: imatinib, dasatinib, nilotinib or bosutinib;

3. Adult Patients (≥18 years) at the time of study entry; Children under the age of 18 will be excluded from the study. The exclusion of children is justified by the following circumstances: a) The condition is relatively rare in children, as compared to adults; b) Issues of study preclude direct applicability of hypotheses and/or interventions to both adults and children.

4. Written informed consent.

5. Written informed consent from Patient's physician as a participant.

6. Newly diagnosed Chronic Phase (CP)-CML Patients who are within 4 weeks of first line TKI therapy (anyone of the TKI approved in the USA and Europe, that is: imatinib, dasatinib, nilotinib or bosutinib).

7. Ability to read/converse in English (Northwestern University and Augusta University Sites). Ability to read/converse in Italian (GIMEMA Centers).

Please note that Physician consent is requisite for the Patient to be enrolled as a participant in the study.

Exclusion Criteria

1. Major cognitive deficits or psychiatric problems hampering a self-reported evaluation;

2. Having received any CML treatment - other than TKI - for more than 3 months prior to receiving current TKI therapy.

1. Major cognitive deficits or psychiatric problems hampering a self-reported evaluation

2. Having received any CML treatment prior to therapy with imatinib, dasatinib, bosutinib or nilotinib for more than three months

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Cancer Institute (NCI)

NIH

Sponsor Role: collaborator

Fondazione GIMEMA

OTHER

Sponsor Role: collaborator

Augusta University

OTHER

Sponsor Role: collaborator

Northwestern University

OTHER

Sponsor Role: lead

Responsible Party

David Cella

Professor, Medical Social Sciences; Feinberg School of Medicine

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

David Cella, PhD

Role: PRINCIPAL_INVESTIGATOR

Northwestern University

Fabio Efficace, PhD

Role: PRINCIPAL_INVESTIGATOR

Fondazione GIMEMA

Locations

Augusta University, Hematology and Oncology

Augusta, Georgia, United States

Northwestern University, Robert H. Lurie Comprehensive Cancer Center Comprehensive Cancer Center (RHLCCC)

Chicago, Illinois, United States

Policlinico Sant'Orsola Malpighi - UOC Ematologia - Azienda Ospedaliero-Universitaria di Bologna

Bologna, , Italy

Ospedale "A. Businco" - Struttura Complessa di Ematologia e CTMO - Azienda Ospedaliera G. Brotzu

Cagliari, , Italy

Milano Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico - UOC Oncoematologia - Padiglione Marcora

Milan, , Italy

Azienda Ospedaliera Universitaria - Università degli Studi di Napoli "Federico II" - UOC Ematologia

Napoli, , Italy

Azienda Ospedaliera Universitaria Maggiore della Carità di Novara - SCDU Ematologia

Novara, , Italy

AUSL Reggio Emilia - Arcispedale S. Maria Nuova, IRCSS - SC Ematologia

Reggio Emilia, , Italy

Azienda Ospedaliera Universitaria Policlinico Umberto I - Università degli Studi "Sapienza" - UOC Ematologia

Roma, , Italy

Ospedale Sant'Eugenio

Roma, , Italy

The GIMEMA Foundation (Italian Group for Adult Hematologic Diseases)

Rome, , Italy

Azienda Ospedaliero-Universitario Città della Salute e della Scienza di Torino - Ospedale S. Giovanni Battista Molinette - SC Ematologia

Torino, , Italy

Ospedale Mauriziano Umberto I - Torino - SCDU Ematologia

Torino, , Italy

ASUI di Udine - Presidio Ospedaliero "Santa Maria della Misericordia" - Clinica Ematologica

Udine, , Italy

Azienda Ospedaliera Universitario Integrata di Verona, Policlinico G.B. Rossi - UOC Ematologia

Verona, , Italy

USL 6 - Ospedale San Bortolo - Vicenza

Vicenza, , Italy

Countries

United States; Italy

References

Druker BJ, Sawyers CL, Kantarjian H, Resta DJ, Reese SF, Ford JM, Capdeville R, Talpaz M. Activity of a specific inhibitor of the BCR-ABL tyrosine kinase in the blast crisis of chronic myeloid leukemia and acute lymphoblastic leukemia with the Philadelphia chromosome. N Engl J Med. 2001 Apr 5;344(14):1038-42. doi: 10.1056/NEJM200104053441402.

Reference Type: BACKGROUND

PMID: 11287973 (View on PubMed)

Druker BJ, Talpaz M, Resta DJ, Peng B, Buchdunger E, Ford JM, Lydon NB, Kantarjian H, Capdeville R, Ohno-Jones S, Sawyers CL. Efficacy and safety of a specific inhibitor of the BCR-ABL tyrosine kinase in chronic myeloid leukemia. N Engl J Med. 2001 Apr 5;344(14):1031-7. doi: 10.1056/NEJM200104053441401.

Reference Type: BACKGROUND

PMID: 11287972 (View on PubMed)

Rosti G, Castagnetti F, Gugliotta G, Baccarani M. Tyrosine kinase inhibitors in chronic myeloid leukaemia: which, when, for whom? Nat Rev Clin Oncol. 2017 Mar;14(3):141-154. doi: 10.1038/nrclinonc.2016.139. Epub 2016 Oct 18.

Reference Type: BACKGROUND

PMID: 27752053 (View on PubMed)

Cella D, Nowinski CJ, Frankfurt O. The impact of symptom burden on patient quality of life in chronic myeloid leukemia. Oncology. 2014;87(3):133-47. doi: 10.1159/000362816. Epub 2014 Jul 8.

Reference Type: BACKGROUND

PMID: 25012261 (View on PubMed)

Bower H, Bjorkholm M, Dickman PW, Hoglund M, Lambert PC, Andersson TM. Life Expectancy of Patients With Chronic Myeloid Leukemia Approaches the Life Expectancy of the General Population. J Clin Oncol. 2016 Aug 20;34(24):2851-7. doi: 10.1200/JCO.2015.66.2866. Epub 2016 Jun 20.

Reference Type: BACKGROUND

PMID: 27325849 (View on PubMed)

Efficace F, Baccarani M, Breccia M, Cottone F, Alimena G, Deliliers GL, Barate C, Specchia G, Di Lorenzo R, Luciano L, Turri D, Martino B, Stagno F, Dabusti M, Bergamaschi M, Leoni P, Simula MP, Levato L, Fava C, Veneri D, Sica S, Rambaldi A, Rosti G, Vignetti M, Mandelli F. Chronic fatigue is the most important factor limiting health-related quality of life of chronic myeloid leukemia patients treated with imatinib. Leukemia. 2013 Jul;27(7):1511-9. doi: 10.1038/leu.2013.51. Epub 2013 Feb 18.

Reference Type: BACKGROUND

PMID: 23417029 (View on PubMed)

Guerin A, Chen L, Ionescu-Ittu R, Marynchenko M, Nitulescu R, Hiscock R, Keir C, Wu EQ. Impact of low-grade adverse events on health-related quality of life in adult patients receiving imatinib or nilotinib for newly diagnosed Philadelphia chromosome positive chronic myelogenous leukemia in chronic phase. Curr Med Res Opin. 2014 Nov;30(11):2317-28. doi: 10.1185/03007995.2014.944973. Epub 2014 Aug 5.

Reference Type: BACKGROUND

PMID: 25025755 (View on PubMed)

Williams LA, Garcia Gonzalez AG, Ault P, Mendoza TR, Sailors ML, Williams JL, Huang F, Nazha A, Kantarjian HM, Cleeland CS, Cortes JE. Measuring the symptom burden associated with the treatment of chronic myeloid leukemia. Blood. 2013 Aug 1;122(5):641-7. doi: 10.1182/blood-2013-01-477687. Epub 2013 Jun 18.

Reference Type: BACKGROUND

PMID: 23777764 (View on PubMed)

Noens L, Hensen M, Kucmin-Bemelmans I, Lofgren C, Gilloteau I, Vrijens B. Measurement of adherence to BCR-ABL inhibitor therapy in chronic myeloid leukemia: current situation and future challenges. Haematologica. 2014 Mar;99(3):437-47. doi: 10.3324/haematol.2012.082511.

Reference Type: BACKGROUND

PMID: 24598855 (View on PubMed)

Marin D, Bazeos A, Mahon FX, Eliasson L, Milojkovic D, Bua M, Apperley JF, Szydlo R, Desai R, Kozlowski K, Paliompeis C, Latham V, Foroni L, Molimard M, Reid A, Rezvani K, de Lavallade H, Guallar C, Goldman J, Khorashad JS. Adherence is the critical factor for achieving molecular responses in patients with chronic myeloid leukemia who achieve complete cytogenetic responses on imatinib. J Clin Oncol. 2010 May 10;28(14):2381-8. doi: 10.1200/JCO.2009.26.3087. Epub 2010 Apr 12.

Reference Type: BACKGROUND

PMID: 20385986 (View on PubMed)

Noens L, van Lierde MA, De Bock R, Verhoef G, Zachee P, Berneman Z, Martiat P, Mineur P, Van Eygen K, MacDonald K, De Geest S, Albrecht T, Abraham I. Prevalence, determinants, and outcomes of nonadherence to imatinib therapy in patients with chronic myeloid leukemia: the ADAGIO study. Blood. 2009 May 28;113(22):5401-11. doi: 10.1182/blood-2008-12-196543. Epub 2009 Apr 6.

Reference Type: BACKGROUND

PMID: 19349618 (View on PubMed)

Ibrahim AR, Eliasson L, Apperley JF, Milojkovic D, Bua M, Szydlo R, Mahon FX, Kozlowski K, Paliompeis C, Foroni L, Khorashad JS, Bazeos A, Molimard M, Reid A, Rezvani K, Gerrard G, Goldman J, Marin D. Poor adherence is the main reason for loss of CCyR and imatinib failure for chronic myeloid leukemia patients on long-term therapy. Blood. 2011 Apr 7;117(14):3733-6. doi: 10.1182/blood-2010-10-309807. Epub 2011 Feb 23.

Reference Type: BACKGROUND

PMID: 21346253 (View on PubMed)

Efficace F, Cannella L. The value of quality of life assessment in chronic myeloid leukemia patients receiving tyrosine kinase inhibitors. Hematology Am Soc Hematol Educ Program. 2016 Dec 2;2016(1):170-179. doi: 10.1182/asheducation-2016.1.170.

Reference Type: BACKGROUND

PMID: 27913477 (View on PubMed)

Experts in Chronic Myeloid Leukemia. The price of drugs for chronic myeloid leukemia (CML) is a reflection of the unsustainable prices of cancer drugs: from the perspective of a large group of CML experts. Blood. 2013 May 30;121(22):4439-42. doi: 10.1182/blood-2013-03-490003. Epub 2013 Apr 25.

Reference Type: BACKGROUND

PMID: 23620577 (View on PubMed)

Kotronoulas G, Kearney N, Maguire R, Harrow A, Di Domenico D, Croy S, MacGillivray S. What is the value of the routine use of patient-reported outcome measures toward improvement of patient outcomes, processes of care, and health service outcomes in cancer care? A systematic review of controlled trials. J Clin Oncol. 2014 May 10;32(14):1480-501. doi: 10.1200/JCO.2013.53.5948. Epub 2014 Apr 7.

Reference Type: BACKGROUND

PMID: 24711559 (View on PubMed)

Velikova G, Booth L, Smith AB, Brown PM, Lynch P, Brown JM, Selby PJ. Measuring quality of life in routine oncology practice improves communication and patient well-being: a randomized controlled trial. J Clin Oncol. 2004 Feb 15;22(4):714-24. doi: 10.1200/JCO.2004.06.078.

Reference Type: BACKGROUND

PMID: 14966096 (View on PubMed)

Efficace F, Gaidano G, Lo-Coco F. Patient-reported outcomes in hematology: is it time to focus more on them in clinical trials and hematology practice? Blood. 2017 Aug 17;130(7):859-866. doi: 10.1182/blood-2017-03-737403. Epub 2017 Jul 10.

Reference Type: BACKGROUND

PMID: 28694324 (View on PubMed)

Efficace F, Baccarani M, Rosti G, Cottone F, Castagnetti F, Breccia M, Alimena G, Iurlo A, Rossi AR, Pardini S, Gherlinzoni F, Salvucci M, Tiribelli M, Vignetti M, Mandelli F. Investigating factors associated with adherence behaviour in patients with chronic myeloid leukemia: an observational patient-centered outcome study. Br J Cancer. 2012 Sep 4;107(6):904-9. doi: 10.1038/bjc.2012.348. Epub 2012 Aug 7.

Reference Type: BACKGROUND

PMID: 22871884 (View on PubMed)

Dueck AC, Mendoza TR, Mitchell SA, Reeve BB, Castro KM, Rogak LJ, Atkinson TM, Bennett AV, Denicoff AM, O'Mara AM, Li Y, Clauser SB, Bryant DM, Bearden JD 3rd, Gillis TA, Harness JK, Siegel RD, Paul DB, Cleeland CS, Schrag D, Sloan JA, Abernethy AP, Bruner DW, Minasian LM, Basch E; National Cancer Institute PRO-CTCAE Study Group. Validity and Reliability of the US National Cancer Institute's Patient-Reported Outcomes Version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE). JAMA Oncol. 2015 Nov;1(8):1051-9. doi: 10.1001/jamaoncol.2015.2639.

Reference Type: BACKGROUND

PMID: 26270597 (View on PubMed)

Basch E, Deal AM, Kris MG, Scher HI, Hudis CA, Sabbatini P, Rogak L, Bennett AV, Dueck AC, Atkinson TM, Chou JF, Dulko D, Sit L, Barz A, Novotny P, Fruscione M, Sloan JA, Schrag D. Symptom Monitoring With Patient-Reported Outcomes During Routine Cancer Treatment: A Randomized Controlled Trial. J Clin Oncol. 2016 Feb 20;34(6):557-65. doi: 10.1200/JCO.2015.63.0830. Epub 2015 Dec 7.

Reference Type: BACKGROUND

PMID: 26644527 (View on PubMed)

Osterberg L, Blaschke T. Adherence to medication. N Engl J Med. 2005 Aug 4;353(5):487-97. doi: 10.1056/NEJMra050100. No abstract available.

Reference Type: BACKGROUND

PMID: 16079372 (View on PubMed)

Weidle PJ, Wamai N, Solberg P, Liechty C, Sendagala S, Were W, Mermin J, Buchacz K, Behumbiize P, Ransom RL, Bunnell R. Adherence to antiretroviral therapy in a home-based AIDS care programme in rural Uganda. Lancet. 2006 Nov 4;368(9547):1587-94. doi: 10.1016/S0140-6736(06)69118-6.

Reference Type: BACKGROUND

PMID: 17084759 (View on PubMed)

Kripalani S, Risser J, Gatti ME, Jacobson TA. Development and evaluation of the Adherence to Refills and Medications Scale (ARMS) among low-literacy patients with chronic disease. Value Health. 2009 Jan-Feb;12(1):118-23. doi: 10.1111/j.1524-4733.2008.00400.x.

Reference Type: BACKGROUND

PMID: 19911444 (View on PubMed)

Cella DF, Tulsky DS, Gray G, Sarafian B, Linn E, Bonomi A, Silberman M, Yellen SB, Winicour P, Brannon J, et al. The Functional Assessment of Cancer Therapy scale: development and validation of the general measure. J Clin Oncol. 1993 Mar;11(3):570-9. doi: 10.1200/JCO.1993.11.3.570.

Reference Type: BACKGROUND

PMID: 8445433 (View on PubMed)

Phillips KM, Pinilla-Ibarz J, Sotomayor E, Lee MR, Jim HS, Small BJ, Sokol L, Lancet J, Tinsley S, Sweet K, Komrokji R, Jacobsen PB. Quality of life outcomes in patients with chronic myeloid leukemia treated with tyrosine kinase inhibitors: a controlled comparison. Support Care Cancer. 2013 Apr;21(4):1097-103. doi: 10.1007/s00520-012-1630-5. Epub 2012 Nov 20.

Reference Type: BACKGROUND

PMID: 23179489 (View on PubMed)

Yellen SB, Cella DF, Webster K, Blendowski C, Kaplan E. Measuring fatigue and other anemia-related symptoms with the Functional Assessment of Cancer Therapy (FACT) measurement system. J Pain Symptom Manage. 1997 Feb;13(2):63-74. doi: 10.1016/s0885-3924(96)00274-6.

Reference Type: BACKGROUND

PMID: 9095563 (View on PubMed)

Basch E, Iasonos A, Barz A, Culkin A, Kris MG, Artz D, Fearn P, Speakman J, Farquhar R, Scher HI, McCabe M, Schrag D. Long-term toxicity monitoring via electronic patient-reported outcomes in patients receiving chemotherapy. J Clin Oncol. 2007 Dec 1;25(34):5374-80. doi: 10.1200/JCO.2007.11.2243.

Reference Type: BACKGROUND

PMID: 18048818 (View on PubMed)

Baccarani M, Deininger MW, Rosti G, Hochhaus A, Soverini S, Apperley JF, Cervantes F, Clark RE, Cortes JE, Guilhot F, Hjorth-Hansen H, Hughes TP, Kantarjian HM, Kim DW, Larson RA, Lipton JH, Mahon FX, Martinelli G, Mayer J, Muller MC, Niederwieser D, Pane F, Radich JP, Rousselot P, Saglio G, Saussele S, Schiffer C, Silver R, Simonsson B, Steegmann JL, Goldman JM, Hehlmann R. European LeukemiaNet recommendations for the management of chronic myeloid leukemia: 2013. Blood. 2013 Aug 8;122(6):872-84. doi: 10.1182/blood-2013-05-501569. Epub 2013 Jun 26.

Reference Type: BACKGROUND

PMID: 23803709 (View on PubMed)

Charlson ME, Pompei P, Ales KL, MacKenzie CR. A new method of classifying prognostic comorbidity in longitudinal studies: development and validation. J Chronic Dis. 1987;40(5):373-83. doi: 10.1016/0021-9681(87)90171-8.

Reference Type: BACKGROUND

PMID: 3558716 (View on PubMed)

O'Brien S, Berman E, Borghaei H, Deangelo DJ, Devetten MP, Devine S, Erba HP, Gotlib J, Jagasia M, Moore JO, Mughal T, Pinilla-Ibarz J, Radich JP, Shah Md NP, Shami PJ, Smith BD, Snyder DS, Tallman MS, Talpaz M, Wetzler M; National Comprehensive Cancer Network. NCCN clinical practice guidelines in oncology: chronic myelogenous leukemia. J Natl Compr Canc Netw. 2009 Oct;7(9):984-1023. doi: 10.6004/jnccn.2009.0065. No abstract available.

Reference Type: BACKGROUND

PMID: 19878641 (View on PubMed)

Sokal JE, Cox EB, Baccarani M, Tura S, Gomez GA, Robertson JE, Tso CY, Braun TJ, Clarkson BD, Cervantes F, et al. Prognostic discrimination in "good-risk" chronic granulocytic leukemia. Blood. 1984 Apr;63(4):789-99.

Reference Type: BACKGROUND

PMID: 6584184 (View on PubMed)

Alonso J, Bartlett SJ, Rose M, Aaronson NK, Chaplin JE, Efficace F, Leplege A, Lu A, Tulsky DS, Raat H, Ravens-Sieberer U, Revicki D, Terwee CB, Valderas JM, Cella D, Forrest CB; PROMIS International Group. The case for an international patient-reported outcomes measurement information system (PROMIS(R)) initiative. Health Qual Life Outcomes. 2013 Dec 20;11:210. doi: 10.1186/1477-7525-11-210.

Reference Type: BACKGROUND

PMID: 24359143 (View on PubMed)

Cottone F, Deliu N, Collins GS, Anota A, Bonnetain F, Van Steen K, Cella D, Efficace F. Modeling strategies to improve parameter estimates in prognostic factors analyses with patient-reported outcomes in oncology. Qual Life Res. 2019 May;28(5):1315-1325. doi: 10.1007/s11136-018-02097-2. Epub 2019 Jan 18.

Reference Type: BACKGROUND

PMID: 30659449 (View on PubMed)

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.

Reference Type: BACKGROUND

PMID: 24026634 (View on PubMed)

Efficace F, Cottone F, Yanez B, Kota V, Castagnetti F, Caocci G, Bonifacio M, Patriarca A, Capodanno I, Miggiano MC, Tiribelli M, Breccia M, Luciano L, Giai V, Iurlo A, Abruzzese E, Fava C, Dinner S, Altman JK, Rosti G, Cortes J, Vignetti M, Cella D. Patient-reported symptom monitoring and adherence to therapy in patients with newly diagnosed chronic myeloid leukemia. Cancer. 2024 Jan;130(2):287-299. doi: 10.1002/cncr.35021. Epub 2023 Oct 6.

Reference Type: DERIVED

PMID: 37801052 (View on PubMed)

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Related Links

https://wizard.facit.org

FACIT Searchable Library and Custom Form Developer (Build-a-PRO)

https://www.eortc.be/itemlibrary/

EORTC Quality of Life Group Item Library

Other Identifiers

1R21CA230367-01A1

Identifier Type: NIH

Identifier Source: org_study_id

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