Attitudes and Perceptions of Patients (pts) with Chronic Myeloid Leukemia in Chronic Phase (CML-CP) Toward Treatment-Free Remission (TFR)
Authors: Boquimpani Carla Maria , Szczudlo Tomasz , Mendelson Estella , Benjamin Katy , Masszi Tamás 
Published: October 2019

Background: With effective tyrosine kinase inhibitor (TKI) therapy, CML-CP disease burden can be reduced to minimal levels, and pts with CML-CP can have a life expectancy similar to that of the general population. Current guidelines recommend that pts continue TKI therapy indefinitely; however, in clinical trials (eg, the Stop Imatinib trials), some pts with deep molecular responses were able to suspend therapy and remain in TFR. This qualitative study assessed pt perspectives on CML treatment and TFR.

Methods: Adults with CML-CP were recruited via third-party panels and interviewed by telephone (≈ 45 min) by trained staff from Oxford Outcomes using a standardized semistructured interview guide and open-ended questions. Some questions were not asked in all interviews; reported data are based on pts with responses for the indicated topic. Key themes and perceptions about TFR and potential impacts on health-related quality of life were identified by thematic analysis. Basic demographic information was also collected.

Results: Of 40 participants, 68% were female and 68% were < 60 y old. Mean ± SD time since CML diagnosis was 5.2 y ± 4.6 y. Current CML treatment was imatinib (53%), dasatinib (25%), nilotinib (15%), or ponatinib (3%); 33% of pts were receiving second- or later-line TKI therapy; others were not receiving therapy due to a physician-supervised medication holiday (3%) or pregnancy (3%). Frequency of blood work (hematology/chemistry and/or molecular monitoring) for CML was every 3 mo for 60% of pts; 23% and 18% of pts had more frequent or less frequent blood work, respectively. Approximately half of pts (55%) had been told they achieved complete molecular response (CMR). Most pts (85%) did not experience/expect any positive physical impacts of CMR, but 68% said it would provide peace of mind that their CML was not progressing. Pts reported a variety of negative impacts of CML treatment, including financial burden (53%), limited ability to perform normal activities (social activities [25%], hobbies/physical activities [18%], work productivity [15%], and/or housework [10%]), and concern about long-term effects on their physical well-being (23%); 35% of pts reported low or no impact of CML treatment on their daily lives. Most pts (75%) reported having medication side effects, most commonly fatigue (60%), bone/joint pain (28%), nausea (18%), and active bowels/gastrointestinal issues (15%). Most pts (77%) said they had some understanding of TFR, and 58% were cautiously positive about attempting TFR. If their physician recommended it, 77% of pts ≥ 60 y old and 52% of pts < 60 y old said they would consider attempting TFR. The most frequently expressed expected positive impacts of TFR were relief of medication side effects (75%), reduced financial burden (58%), convenience (43%), positive emotional impact (43%), and increased activity level (30%). The most frequently expressed concerns about TFR included fear of resistance to therapy upon relapse (90%), low chance of successfully maintaining TFR (45%), emotional response to relapse and re-initiation of therapy (35%), desire for more frequent disease monitoring (33%), and fear of severe side effects upon re-initiation of therapy (33%). Some pts (28%) said their families may not want them to risk their health by attempting TFR. Among pts < 60 y old, 15% expressed concerns about the well-being of dependent children if they were to attempt TFR and relapse. Pts expressed a desire for more long-term data evaluating the safety of TFR in clinical trials. Pts reported a willingness to attempt TFR if there was at least a 10% chance of sustaining TFR for 2 y (range, 10%-100%), and the shortest duration of TFR they found acceptable ranged from “any amount of time” to 7 y.

Conclusions: In this qualitative study, pts perceived many potential positive impacts of TFR, with relief of medication side effects being the most frequently expressed. Although TFR clinical trials have shown high rates of response to re-initiation of TKI therapy in pts with molecular relapse, a perceived risk of developing resistance to therapy was the most notable pt concern about TFR, and pts felt more clinical data are needed. Pt responses also revealed the importance of considering family when discussing TFR. With effective education and in the context of a controlled clinical trial, TFR may be an appropriate goal associated with meaningful pt benefits. More research into the pt perspective on TFR is needed.

Disclosures

Boquimpani: Bristol Myers Squibb: Speakers Bureau; Novartis: Speakers Bureau. Off Label Use: Current CML treatment recommendations indicate that BCR-ABL TKI therapy should be administered indefinitely, and treatment-free remission (TFR) is an investigational approach that falls outside of current BCR-ABL TKI labels. However, this concept is not new, and promising preliminary results from several clinical trials of TFR have been reported. This abstract does not include any clinical data, but focuses on patient preferences and perceptions of TFR. . Szczudlo:Novartis: Employment, Equity Ownership. Mendelson:Novartis: Employment, Equity Ownership. Benjamin:Novartis Pharmaceuticals Corporation: Consultancy. Masszi:Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees.