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Clinical Trial
. 2024 Jan;130(2):287-299.
doi: 10.1002/cncr.35021. Epub 2023 Oct 6.

Patient-reported symptom monitoring and adherence to therapy in patients with newly diagnosed chronic myeloid leukemia

Fabio Efficace  1   2 , Francesco Cottone  1 , Betina Yanez  2 , Vamsi Kota  3 , Fausto Castagnetti  4   5 , Giovanni Caocci  6 , Massimiliano Bonifacio  7 , Andrea Patriarca  8 , Isabella Capodanno  9 , Maria Cristina Miggiano  10 , Mario Tiribelli  11 , Massimo Breccia  12 , Luigia Luciano  13 , Valentina Giai  14 , Alessandra Iurlo  15 , Elisabetta Abruzzese  16 , Carmen Fava  17 , Shira Dinner  18 , Jessica K Altman  18 , Gianantonio Rosti  19 , Jorge Cortes  3 , Marco Vignetti  1 , David Cella  2   18
Affiliations
Clinical Trial

Patient-reported symptom monitoring and adherence to therapy in patients with newly diagnosed chronic myeloid leukemia

Fabio Efficace et al. Cancer. 2024 Jan.

Abstract

Background: The authors assessed the clinical utility of patient-reported symptom monitoring in the setting of newly diagnosed chronic myeloid leukemia (CML). The primary objective was to evaluate adherence to therapy.

Methods: The authors conducted an international prospective study that included patients with newly diagnosed, chronic-phase CML. Before clinical consultation, patients were provided a tablet computer to self-rate their symptoms, and the results were available in real time to each physician during the patient's visit. Adherence was assessed by pill count and with a validated self-reported questionnaire. The proportions of optimal responders at 3 and 6 months were assessed according to the European LeukemiaNet criteria.

Results: Between July 2020 and August 2021, 94 patients with a median age of 57 years were enrolled. Pill count adherence analysis indicated that 86 of 93 evaluable patients (92.5%) took at least 90% of prescribed tyrosine kinase inhibitor therapy during the 6-month observation period. The online platform was well accepted by patients and physicians. An optimal response was achieved by 69 of 79 patients (87.3%) at 3 months and by 61 of 81 patients (75.3%) at 6 months.

Conclusions: Patient-reported symptom monitoring from the beginning of therapy in patients with CML may be critical to improve adherence to therapy and early molecular response rates (ClinicalTrials.gov identifier NCT04384848).

Keywords: adherence; chronic myeloid leukemia; digital health; molecular response; quality of life; symptoms.

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Conflict of interest statement

CONFLICT OF INTEREST STATEMENT

Fabio Efficace reports personal fees from AbbVie, Incyte Corporation, Janssen Pharmaceuticals, Inc., Novartis, and Syros outside the submitted work. Vamsi Kota reports personal fees from Kite Pharma, Inc., Novartis, and Pfizer, Inc., outside the submitted work. Fausto Castagnetti reports personal fees from Bristol-Myers Squibb, Incyte Corporation, Novartis, and Pfizer, Inc., outside the submitted work. Giovanni Caocci reports speakers’ fees from Bristol-Myers Squibb, Novartis, and Pfizer, Inc., outside the submitted work. Massimiliano Bonifacio reports personal fees from AbbVie, Bristol-Myers Squibb, Incyte Corporation, Novartis, and Pfizer, Inc., outside the submitted work. Andrea Patriarca reports personal fees from Novartis, Pfizer, Inc., Sanofi and Genzyme US Companies, and Sobi, Inc.; and travel support from Alexion Pharmaceuticals outside the submitted work. Isabella Capodanno reports speakers’ fees from Incyte Corporation, Novartis, and Pfizer, Inc., outside the submitted work. Mario Tiribelli reports support from Bristol-Myers Squibb and Incyte Corporation and personal fees from Novartis outside the submitted work. Massimo Breccia reports fees for expert witness testimony from Incyte Corporation, Novartis Pharma, and Pfizer, Inc., outside the submitted work. Luigia Luciano reports speakers’ fees from Bristol-Myers Squibb, Incyte Corporation, Novartis, and Pfizer, Inc., outside the submitted work. Valentina Giai reports honoraria from Novartis and fees for expert witness testimony from Alexion Pharmaceuticals outside the submitted work. Alessandra Iurlo reports speakers’ fees from Bristol-Myers Squibb, Incyte Corporation, Novartis, and Pfizer, Inc., outside the submitted work. Elisabetta Abruzzese reports personal fees from Bristol-Myers Squibb, Incyte Corporation, Novartis, and Pfizer, Inc., outside the submitted work. Carmen Fava reports grants from Pfizer, Inc.; and honoraria from Incyte Corporation and Novartis outside the submitted work. Shira Dinner reports personal fees from Kita Pharma, Inc., Novartis, Pfizer, Inc., and Rigel Pharmaceuticals, Inc.; and other support from “Great Debates and Updates in Hematologic Malignancies” outside the submitted work. Jessica K. Altman reports personal fees from AbbVie, American Society of Hematology, Astellas Pharma, BlueBird Bio, Bristol-Myers Squibb, Curio, Daiichi-Sankyo, Gilead Sciences, HMP Education, Incyte Corporation, Kura, Kymera, MDEducation, NCI, PeerView, Rigel Pharmaceuticals, Inc., Stemline Therapeutics, Inc., and Syros; service on a Data and Safety Monitoring Board for GlycoMimetics; and travel support from Biosight and Insights in Hematology outside the submitted work. She also serves on the NCCN CML guidelines and she is the vice-chair for the NCCN AML guidelines. Gianantonio Rosti reports speakers’ fees from Bristol-Myers Squibb, Incyte Corporation, Novartis, Pfizer, Inc., outside the submitted work. Jorge Cortes reports institutional research support from Kartos Pharmaceuticals, Novartis, Pfizer, Inc., Sun Pharmaceutical Industries, Inc., and Takeda Oncology; and personal fees from AbbVie, Biopath Holdings, Bristol-Myers Squibb, Kartos Pharmaceuticals, Novartis, Pfizer, Inc., Rigel Pharmaceuticals, Inc., and Takeda Oncology outside the submitted work. Marco Vignetti reports honoraria from AbbVie, Amgen, AstraZeneca, Dephaforum Srl., Incyte Corporation, and Novartis; and personal fees from Amgen outside the submitted work. David Cella is the President of FACIT.org; he reports research funding from Boehringer-Ingelheim, Bristol-Myers Squibb, Clovis, Fulcrum, and Pfizer, Inc.; and personal fees from consultancies with BMS, Astellas, Bristol-Myers Squibb, Ipsen, Immunogen, Novartis, Pfizer, Inc., and Sanofi outside the submitted work.

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