How could patient reported outcomes improve patient management in chronic myeloid leukemia?

Alanazi²,

et al. 2017

Bangladesh J Med Sci

Introduction: Philadelphia chromosome is a cytogenetic marker for chronic myeloid leukemia (CML). The main aims of this study were to assess the positive responses, side-effects and survival of CML patients treated with imatinib mesylate. Methods: All recently diagnosed CML patients who were treated with imatinib were recruited to this study. We investigated hematological and cytogenetic parameters by CBC, FISH and RT-PCR individually. Results: Of the 10 cases, 7 (70%) were males and 3 (30%) were female. Four (40%) of the cases were analyzed retrospectively and 8 cases (80%) exhibited general exhaustion (75%), fever (80%), and splenomegaly (80%). Indications of bleeding and rashes were rarely seen at presentation. The majority of the patients had a generally low risk profile (70%), 30% had intermediate risk; with no subjects exhibiting high risk CML, 9 subjects (90%) were in remission.

Section: Preparation Of Mononuclear Cells For Culture and Cfu-gm Cultursupporting

confidence: 81%

Imatinib reduces proliferation of leukemic cells in vitro

Alanazi²,

et al. 2017

Bangladesh J Med Sci

“…Given the need for lifelong therapy, HRQoL from the patient perspective has become a key component of clinical decision-making [25], in addition to response to individual therapies. Although evaluation of HRQoL through PRO assessments in clinical trials for CML is limited [7], studies of bosutinib have routinely collected PRO data to assess the symptom burden and functional health status of patients [8][9][10][11]. In the phase 3 BFORE trial of bosutinib versus imatinib in patients with newly diagnosed CP CML, similar degrees of improvement or preservation of HRQoL were seen in both treatment arms after 12 months [11].…”

Section: Discussionmentioning

confidence: 99%

“…To date, prospective assessment of patient-reported HRQoL in CML trials of most first-and second-generation TKIs has been scarce [7]. However, patient-reported outcome (PRO) data from clinical studies of bosutinib have indicated that patients with CML experienced stable or, in some cases, improved HRQoL during treatment compared with pretreatment status [8][9][10][11].…”

Section: Introductionmentioning

confidence: 99%

Relationship between molecular response and quality of life with bosutinib or imatinib for chronic myeloid leukemia

et al. 2020

Patients with newly diagnosed chronic phase chronic myeloid leukemia (CP CML) can be effectively treated with tyrosine kinase inhibitors (TKIs) and achieve a lifespan similar to the general population. The success of TKIs, however, requires long-term and sometimes lifelong treatment; thus, patient-assessed health-related quality of life (HRQoL) has become an increasingly important parameter for treatment selection. Bosutinib is a TKI approved for CP CML in newly diagnosed adults and in those resistant or intolerant to prior therapy. In the Bosutinib Trial in First-Line Chronic Myelogenous Leukemia Treatment (BFORE), bosutinib demonstrated a significantly higher major molecular response rate compared with imatinib, with maintenance of HRQoL (measured by the Functional Assessment of Cancer Therapy-Leukemia (FACT-Leu) questionnaire), after 12 months of first-line treatment. We examined relationships between molecular response (MR) and HRQoL. MR values were represented by a log-reduction scale (MRLR; a continuous variable). A repeated-measures longitudinal model was used to estimate the relationships between MRLR as a predictor and each FACT-Leu domain as an outcome. Effect sizes were calculated to determine strength of effects and allow comparisons across domains. The majority of FACT-Leu domains (with the exception of social well-being and physical well-being) demonstrated a significant relationship with MRLR (p < 0.05). Our results showed variable impact of clinical improvement on different dimensions of HRQoL. For patients who achieved MR5, emotional well-being and leukemia-specific domains showed the greatest improvement, with medium differences in effect sizes, whereas social well-being and physical well-being had the weakest relationship with MR.

“…Patients reporting their own outcomes have improved control of symptoms, medication adherence, and patient satisfaction. Furthermore, PROs provide distinct prognostic information, exceeding clinical measurements [ 54 - 56 ].…”

Section: Discussionmentioning

confidence: 99%

The Development of a Web-Based, Patient-Centered Intervention for Patients With Chronic Myeloid Leukemia (CMyLife): Design Thinking Development Approach

Ector¹,

Westerweel²,

Hermens³

et al. 2020

J Med Internet Res

Background With the global rise in chronic health conditions, health care is transforming, and patient empowerment is being emphasized to improve treatment outcomes and reduce health care costs. Patient-centered innovations are needed. We focused on patients with chronic myeloid leukemia (CML), a chronic disease with a generally good long-term prognosis because of the advent of tyrosine kinase inhibitors. However, both medication adherence by patients and guideline adherence by physicians are suboptimal, unnecessarily jeopardizing treatment outcomes. Objective The aim of this study was to develop a patient-centered innovation for patients with CML using a design thinking methodology. Methods The 5 phases of design thinking (ie, empathize, define, ideate, prototype, and test) were completed, and each phase started with the patient. Stakeholders and end users were identified and interviewed, and observations in the care system were made. Using tools in human-centered design, problems were defined and various prototypes of solutions were generated. These were evaluated by patients and stakeholders and then further refined. Results The patients desired (1) insights into their own disease; (2) insights into the symptoms experienced, both in terms of knowledge and comprehension; and (3) improvements in the organization of care delivery. A web-based platform, CMyLife, was developed and pilot-tested. It has multiple features, all targeting parts of the bigger solution, including a website with reliable information and a forum, a guideline app, personal medical records with logs of symptoms and laboratory results (including a molecular marker and linked to the guideline app), tailored feedback based on the patients’ symptoms and/or results, screen-to-screen consulting, delivery of medication, and the collection of blood samples at home. Conclusions The multifeatured innovation, CMyLife, was developed in a multidisciplinary way and with active patient participation. The aim of developing CMyLife was to give patients the tools to monitor their results, interpret these results, and act on them. With this tool, they are provided with the know-how to consider their results in relation to their personal care process. Whether CMyLife achieves its goal and the evaluation of the added value will be the focus of future studies. CML could become the first malignancy for which patients are able to monitor and manage their disease by themselves.