Treatmentfree remission for CML Patients Results slide pack

Treatment-free remission for CML Patients Results slide pack (v 4. 1) of the worldwide TFR Survey of the CML Advocates Network Any use must reference to the original source of the TFR Survey at https: //www. cmladvocates. net/tfr-cml-patients

Introduction to Treatment Free Remission Stopping CML treatment has become a more widely spread phenomenon in regular clinical practice However much is still unknown about the experiences of those considering and undertaking TFR does not mean the end of CML for many patients, but rather a different phase that needs be dealt with in a unique way • According to current first-line studies, about 65 -75% of CML patients will, over years, achieve a durable deep molecular response (minimum MR 4) with current TKI treatments • Of those then stopping treatment, about half will remain in durable therapy-free remission. • The technical as well as the emotional impact of stopping • The prevention and management of TKI withdrawal symptoms • Emotional and physical impact of disease reoccurrence • Impact of an ongoing commitment to monitoring • Experience shows that both doctors and patients do not get the information and support they need to meet that challenge

Project Objectives The overall objective of the project was to provide a set of information packs and documents to its member organisations, more specifically: 1 To provide patients with all information they need when: a. Considering stopping treatment b. Having stopped treatment c. Having failed therapy-free remission 2 To avoid patients’ confusions by using consistent terminology on stopping CML treatment 3 To inform doctors about patients concerns, considerations and needs when: a. Considering stopping treatment b. Having stopped treatment c. Having failed therapy-free remission

Response Rates Countries with > 20 respondents Countries with < 20 respondents Total of 1016 respondents Covering 69 countries Methodology: • All online – Recruited by patient associations via online forums and other methods • Available in eleven languages: Arabic, Danish, English, Finnish, French, German, Hebrew, Italian, Japanese, Russian and Spanish • Fieldwork – 14 th March 2018 – 1 st August 2018 Publications: • Treatment-free remission in chronic myeloid leukemia: the patient perspective and areas of unmet needs. Sharf, Marin, Bradley, Pemberton-Whiteley, Bombaci, Christensen, Gouimi, Deekes, Daban, Geissler, in: Leukemia (2020), doi: 10. 1038/s 41375 -020 -0867 -0 • Oral presentation at ESH 2020. Posters presented at EHA 2020, ASH 2019. Full slide deck of results available at https: //www. cmladvocates. net/tfr-cml-patients

Responses by Phase The questionnaire was split into five sections, with respondents only answering the questions that related to their experiences of stopping treatment. • About you – answered by all respondents to get background information about them and their CML treatment • Phase I – Considerations around stopping treatment: what considerations and discussions took place while deciding whether to stop treatment • Phase II – Probation period: experiences during the first 6 months of stopping treatment • Phase IIIA – Restarting treatment: experiences if CML reoccurred • Phase IIIB – Long term remission: experiences of being in long term treatment free remission

Scoring methodology • For all questions (with the exception of those asked in the form of “tick all that apply”) the percentage responses are calculated after excluding those respondents that did not answer that particular question • The base size for questions which have been asked in the form of “tick all that apply” is determined by the number of respondents eligible to respond. As such the missing count for a tick all that apply response option represents any eligible respondents who have chosen not to select that particular option. • All percentages are rounded to the nearest whole number. When added together, the percentages for all answers to a particular question may not total 100% because of this rounding • On some questions, scores have been recalculated to exclude non-specific responses, (such as don’t know/can’t remember) or responses indicating that the question was not applicable to the participant's circumstances. These are indicated in the footnotes

Demographics (All respondents) 55% Female 45% Male Q 1 / base = all respondents (n=1003) - Are you male or female? Q 2 / base = all respondents (n=1004) - In what year were you born? Q 4 / base = all respondents (n=1007) - What is the highest degree or level of school you have completed? Age Groups

Where respondents are treated* *Respondents could select all answer options that applied (tick all that apply) Q 5 / base = all respondents (n=1016) - Where are you usually treated for your CML?

Where respondents are treated* – by CML Advocate group region *Respondents could select all answer options that applied (tick all that apply) Q 5/ base = all respondents - Where did you get support and find information about stopping treatment? All regions (n=1016) Western Europe (n= 395) Asia and Pacific (n= 233) North America (n= 109) Latin America (n= 56) Middle East and Africa (n=92) Central and Eastern Europe & West Asia (n=23) Not Classified (n= 108)

Length of time living with CML (All respondents) Q 6 / base = all respondents (n=991) - How long have you been living with CML?

CML medications taken since diagnosis* (All respondents) *Respondents could select all answer options that applied (tick all that apply) Q 7 / base = all respondents (n=1016) - Which CML medication have you taken since your diagnosis?

Which respondents have already stopped treatment (All respondents) Q 8 / base = all respondents (n=1011) - Have you already stopped treatment?

Respondents who have already stopped treatment Q 8 / base = all respondents who have stopped treatment (n=518) - Have you already stopped treatment?

Current CML medication (Respondents who have not stopped) Q 9 / base = all respondents (n=480) - What CML medication are you currently taking?

CML medication being taken before stopping (Respondents who stopped) Q 10 / base = all respondents (n=510) - What CML medication were you taking just before you stopped?

Phase I – Considerations around stopping treatment This section of the questionnaire was answered by all respondents Maximum base (n=1016) – All respondents

How respondents FIRST heard about the possibility of stopping treatment Q 11 / base = all respondents (n=976) - How did you first hear about the possibility of stopping treatment?

How respondents FIRST heard about the possibility of stopping treatment – by CML Advocate Group regions On average most respondents reported first hearing about TFR through a healthcare professional. However, there is variation between the Advocate Group regions. Western Europe and Latin America had the highest proportion of patients first being told about TFR by a health professional. Patients from countries without a designated Advocate Group were least likely to say the same. Q 11 / base= all respondents - How did you first hear about the possibility of stopping treatment? All regions (n=976) Western Europe (n= 385) Asia and Pacific (n= 226) North America (n= 106) Latin America (n= 49) Middle East and Africa (n=87) Central and Eastern Europe & West Asia (n=20) Not Classified (n= 103)

How respondents FIRST heard about the possibility of stopping treatment – by CML Advocate Group regions Respondents from the Middle East & Africa, and Central Eastern Europe & West Asia regions were most likely to say they first heard about TFR from a patient organisation. Nearly the same percentage of respondents from Middle East & Africa, and Central Eastern Europe & West Asia regions said they first heard about TFR from a patient organisation as from a healthcare professional. Just 6% of respondents from North America said they first heard about TFR from a patient organisation. Q 11 / base = all respondents - How did you first hear about the possibility of stopping treatment? All regions (n=976) Western Europe (n= 385) Asia and Pacific (n= 226) North America (n= 106) Latin America (n= 49) Middle East and Africa (n=87) Central and Eastern Europe & West Asia (n=20) Not Classified (n= 103)

How respondents FIRST heard about the possibility of stopping treatment – by CML Advocate Group regions Nearly a quarter of respondents from North America said they first heard about TFR through social media, this is much higher than the other regions. Q 11 / base = all respondents - How did you first hear about the possibility of stopping treatment? All regions (n=976) Western Europe (n= 385) Asia and Pacific (n= 226) North America (n= 106) Latin America (n= 49) Middle East and Africa (n=87) Central and Eastern Europe & West Asia (n=20) Not Classified (n= 103)

Topics discussed with doctor whilst making decision to try stopping treatment* *Respondents could select all answer options that applied (tick all that apply) Q 12 / base = all respondents (n=1016) - What topics did you discuss with your doctor whilst making your decision to try stopping treatment?

Topics that were still a concern following discussion with doctor* *Respondents could select all answer options that applied (tick all that apply) Q 13/ base = all respondents (n=1016) - What topics did you discuss with your doctor whilst making your decision to try stopping treatment?

Did they consider switching to 2 nd generation treatment (In order to achieve a deeper molecular response and therefore stop treatment) Q 14 / base = all respondents (n=972) - Did you consider switching to a 2 nd generation treatment for CML, like Dasatinib or Nilotinib, to achieve a deeper molecular response, allowing you to stop treatment?

Would have considered adding an additional treatment (In order to achieve a deeper molecular response and therefore stop treatment) Q 15 / base = all respondents (n=990) - Would you have considered adding an additional treatment to your current treatment, if as a result it allowed you to achieve a deeper molecular response and therefore stop treatment altogether?

Main reasons for considering stopping treatment* *Respondents could select all answer options that applied (tick all that apply) Q 16 / base = all respondents (n=1016) - What are the main reasons that made you consider stopping treatment?

Main reasons for considering stopping treatment* - by Gender *Respondents could select all answer options that applied (tick all that apply) Q 16 / base = all respondents - What are the main reasons that made you consider stopping treatment? Males (n=453) Females (n=550)

Main worries about stopping treatment* *Respondents could select all answer options that applied (tick all that apply) Q 17 / base = all respondents (n=1016) - What are the main reasons that made you worry about stopping treatment?

Main worries about stopping treatment* - by Gender *Respondents could select all answer options that applied (tick all that apply) Q 17 / base = all respondents - What are the main reasons that made you worry about stopping treatment? Males (n=453) Females (n=550)

Doctor support Q 18 / base = respondents who answered yes/no (n=812) -Did your doctor support your decision to try stopping treatment? Q 19 / base (n= 880) Excludes those who answered don’t know/can’t remember( n=102) - How much did your doctor influence your decision to try stopping treatment?

Doctor support – by CML Advocate Group regions On average most respondents reported that their doctor supported their decision to try stopping treatment but there is difference between the Advocate Group regions. Western Europe and North America report most positively, and Central Eastern Europe & West Asia and Asia & Pacific least positively. Q 18 / base = respondents who answered yes/no - Did your doctor support your decision to try stopping treatment? All regions (n=812) Western Europe (n= 354) Asia and Pacific (n= 161) North America (n= 95) Latin America (n= 35) Middle East and Africa (n=76) Central and Eastern Europe & West Asia (n=17) Not Classified (n= 74)

Where respondents received support and information about stopping treatment from* *Respondents could select all answer options that applied (tick all that apply) Q 20 / base = all respondents (n=1016) - Where did you get support and find information about stopping treatment?

Where respondents received support and information about stopping treatment from* – by CML Advocate group region *Respondents could select all answer options that applied (tick all that apply) Q 20 / base = all respondents - Where did you get support and find information about stopping treatment? All regions (n=1016) Western Europe (n= 395) Asia and Pacific (n= 233) North America (n= 109) Latin America (n= 56) Middle East and Africa (n=92) Central and Eastern Europe & West Asia (n=23) Not Classified (n= 108)

How the support and information received helped respondents* *Respondents could select all answer options that applied (tick all that apply) Q 21/ base=all respondents (n=1016) - How did this support help you?

What information about stopping treatment and TFR respondents would liked to have received *Respondents could select all answer options that applied (tick all that apply) Q 22 / base = all respondents (n=1016) - What information about stopping treatment and therapy-free remission would you have liked to have received?

Respondents proceeding to stop treatment Q 23 / base = all respondents (n=1016) - Did you proceed to stopping treatment?

Respondents proceeding to stop treatment Q 23 / base = all respondents - Did you proceed to stopping treatment? Male (n= 453) Female (n=550) Western Europe (n= 395) Asia and Pacific (n= 233) North America (n= 109) Latin America (n= 56) Middle East and Africa (n=92) Central and Eastern Europe & West Asia (n=23) Not Classified (n= 108)

Phase II – Probation Period This section of the questionnaire was answered by respondents who stopped treatment Maximum base (n=494) – All respondents who answered - Yes - Q 23 - Did you proceed to stopping treatment?

Respondents feeling well informed when stopping treatment Q 24 / base (n= 479) Excludes those who answered - No, but I didn't want to have all the information (n=11) - When you stopped treatment did you feel that you were well informed?

Length of time on medication for CML before stopping Q 25 / base = all respondents (n=468) - How many years were you on medication for CML before stopping?

Length of time in deep molecular response* before stopping CML treatment * Defined as at least MR 4, or BCR-ABL below 0. 01%) Q 26 / base = all respondents (n=491) - How long were you in deep molecular response (at least MR 4, or BCR-ABL below 0. 01%) before you stopped CML treatment?

Impact of side effects BEFORE stopping treatment Respondents were asked to grade the impact of the listed side effects from 1 -5 1 meant the side effect mildly affected their everyday life, and 5 meant the side effect completely effected everyday life. Side effects not experienced were to be left blank. The chart below gives the mean score for each side effect Q 27 / all respondents - Please rate the extent to which you were affected by the following side effects in the weeks BEFORE stopping treatment? Fatigue (n=402) Muscle soreness or cramping (n=385) Swelling of hands, feet, abdomen, and around eyes (n=326) Feeling of malaise (n=301) Disturbed sleep (n=314) Skin pigment change (n=284) Rash or skin change (n=302) Pain (n=291) Remembering things (n=308) Numbness or tingling (n=289) Shortness of breath (n=287) Difficulty thinking clearly (n=298) Other (n=46) Feeling distressed (n=272) Nausea (n=281) Hair loss (n=292) Diarrhoea (n=307) Anxiety (n=289) Dry mouth (n=282) Feeling sad (n=268) Headaches (n=272) Eye bleeds (n=283) Menstrual cycle issues (n=214) Bruising (n=256) Lack of appetite (n=241) Vomiting (n=242)

Topics discussed with doctor during the stopping phase Q 28 / base = all respondents - During the stopping phase did you discuss the following topics with your doctor? Q 28 a / How to deal withdrawal symptoms (n=426) Q 28 b / Response levels and when/if to restart treatment (n=446) Q 28 c / How often to monitor the BCR-ABL levels (PCR test) (n=465) Q 28 d / Time taken to receive the results of the last PCR test (n=418) Q 28 e / How to deal with psychological aspects (n=380) Q 28 f/ Other (n=53)

Withdrawal symptoms (1) Q 29 / base (n= 471) Excludes those who answered Don’t know/can’t remember (n=18) - Did your doctor or another healthcare professional ask you if you were experiencing any physical withdrawal effects during the stopping of treatment?

Withdrawal symptoms (2) Q 30 a / base = all respondents (n=479) - During your stopping phase, how long did you experience withdrawal symptoms for? Q 30 b / base (n= 288) Excludes those who answered I did not experience withdrawal symptoms (n=191) - During your stopping phase, how long did you experience withdrawal symptoms for?

Withdrawal symptoms* (3) *Respondents could select all answer options that applied (tick all that apply) Q 31 What withdrawal effects did you experience when treatment was stopped? / base = respondents answering that they experienced withdrawal symptoms to Q 30 (n=288) - During your stopping phase, how long did you experience withdrawal symptoms for?

Withdrawal symptoms (4) Q 32 Did your doctor support you in managing all your physical withdrawal effects during the stopping of treatment? / base = (respondents answering that they experienced withdrawal symptoms to Q 30 During your stopping phase, how long did you experience withdrawal symptoms for? Also excludes those who answered – Don’t know / can’t remember (n=5) Total base (n=280)

Withdrawal symptoms (5) – by CML Advocate Group regions Q 32 Did your doctor support you in managing all your physical withdrawal effects during the stopping of treatment? / base = respondents answering that they experienced withdrawal symptoms to Q 30 During your stopping phase, how long did you experience withdrawal symptoms for? Also excludes those who answered – Don’t know / can’t remember (n=4) All regions (n=280) Western Europe (n=147) Asia and Pacific (n=34) North America (n= 47) Middle East and Africa (n=21) Not Classified (n=23)

Emotional impact (1) Q 33 / base = all respondents (n=478) - During your stopping phase, how often did you feel fear or anxiety?

Emotional impact (2) - By Gender A higher proportion of male respondents reported that they did not feel fearful or anxious during the stopping phase. Whilst both sexes reported feeling most anxious around testing, a higher percentage of female respondents said this was the case. Q 33 / base = all respondents - During your stopping phase, how often did you feel fear or anxiety? Males (n=188) Females (n=286)

Emotional impact(3) - By gender and age Q 33 / base = all respondents - During your stopping phase, how often did you feel fear or anxiety? Males 18 -40 (n=23) Males 41 -55 (n=56) Males 56+ (n=109) Females 18 -40 (n=63) Females 41 -55 (n=91) Females 56+ (n=132)

Psychological support whilst stopping treatment (1) Q 34 / base (n= 464) Excludes those who answered Don’t know/can’t remember (n=18) - Did your doctor ask you if you needed psychological support during the stopping of treatment? Q 35 / base (n= 472) Excludes those who answered Don’t know/can’t remember (n=9) - Did you receive psychological and/or emotional support during the stopping of treatment?

Psychological support whilst stopping treatment (2) *Respondents could select all answer options that applied (tick all that apply) Q 36 What psychological support did you receive? / base = (n=93) respondents answering ‘Yes’ to Q 35 - Did you receive psychological and/or emotional support during the stopping of treatment?

Psychological support whilst stopping treatment (3) – by Gender Male patients were more likely to say they did not feel psychological support was necessary. Female patients were more likely to say they received psychological support. Q 34 / Excludes those who answered Don’t know/can’t remember - Did your doctor ask you if you needed psychological support during the stopping of treatment? Males (n=177) Females (n=283) Q 35 / Excludes those who answered Don’t know/can’t remember - Did you receive psychological and/or emotional support during the stopping of treatment? Males (n=183) Females (n=285)

Psychological support whilst stopping treatment (4) – by Gender *Respondents could select all answer options that applied (tick all that apply) Q 36 What psychological support did you receive? / base = respondents answering ‘Yes’ to Q 35 - Did you receive psychological and/or emotional support during the stopping of treatment? Males (n=23) Females (n=70)

Reported benefits of stopping treatment Q 37 When treatment was stopped did you experience the following benefits? / base = respondents answering ‘Yes’ to Q 35 - Did you receive psychological and/or emotional support during the stopping of treatment? Q 37 a / Relief from treatment side effects (n=456) Q 37 b / Relief from the need to remember to take regular medication (n=449) Q 37 c / Positive impact on finances (n=381) Q 37 d / Positive impact on emotional well-being (n=424) Q 37 e / Positive impact on work / education (n=394) Q 37 f / Positive impact on family and social relationships (n=412) Q 37 g / Other (n=58)

Monitoring (1) Q 38 / base = all respondents (n=485) - After stopping treatment, on average, how often were you monitored by your doctor by a PCR test in the first 6 months? Q 39 / base (n= 456) Excludes those who answered - Don’t know / Can’t remember (n=23) - After stopping treatment, how long did it take (on average) between your blood tests and until you heard about the result of the PCR?

Monitoring (2) – by CML Advocate group region Q 38 / base = all respondents - After stopping treatment, on average, how often were you monitored by your doctor by a PCR test in the first 6 months? Western Europe (n=255) Asia and Pacific (n=69) North America (n= 67) Middle East and Africa (n=39) Not Classified (n=38)

Monitoring (3) – by CML Advocate group region Q 39 / base excludes those who answered - Don’t know / Can’t remember - After stopping treatment, how long did it take (on average) between your blood tests and until you heard about the result of the PCR? Western Europe (n=242) Asia and Pacific (n=61) North America (n= 65) Middle East and Africa (n=35) Not Classified (n=36)

Monitoring (4) Q 40/ base (n= 469) Excludes those who answered - Don’t know / Can’t remember (n=11) - After stopping treatment, were you satisfied with how often your CML was monitored?

Monitoring (5) – by CML Advocate group region Q 40/ base excludes those who answered - Don’t know / Can’t remember - After stopping treatment, were you satisfied with how often your CML was monitored? Western Europe (n=249) Asia and Pacific (n=66) North America (n= 64) Middle East and Africa (n=36) Not Classified (n=37)

Results of stopping treatment Q 41 / base = all respondents (n=494) - Following stopping treatment, did the disease reoccur and treatment have to restart?

Phase IIIA – Restarting treatment This section of the questionnaire was answered by respondents who stopped treatment but had to restart Maximum base (n=159) – All respondents who answered – Yes to Q 41 - Following stopping treatment, did the disease reoccur and treatment have to restart?

Length of time after stopping, that the disease reoccurred Q 42 / base = all respondents (n=155) - How long after stopping a TKI did the disease reoccur, and treatment had to restart?

Understanding of explanation that the disease had reoccurred Q 43 / base = all respondents (n=153) - When being told your disease had reoccurred, and you had to restart treatment, was this explained in a way you could understand?

Feelings upon reoccurrence of CML Q 44 / base = all respondents - To what extent do you agree or disagree with the following statements? When I was first told my disease had reoccurred I felt…. Q 44 a/ scared / anxious (n=138) Q 44 b / disappointed (n=152) Q 44 c / depressed (n=133) Q 44 d / confused (n=123) Q 44 e / relieved (n=120)

Feelings upon reoccurrence of CML – by Gender Key differences between genders on the emotions reported upon disease reoccurrence. A higher proportion of female patients strongly agreed/agreed that they felt scared/ anxious, and depressed when they were told their disease had reoccurred. Q 44 a / base = all respondents - To what extent do you agree or disagree with the following statements? When I was first told my disease had reoccurred I felt scared / anxious Males (n=45) Females (n=92) Q 44 c / base = all respondents - To what extent do you agree or disagree with the following statements? When I was first told my disease had reoccurred I felt depressed Males (n=43) Females (n=89)

Before treatment restarted were treatment options explained Q 45/ base (n=155) Excludes those who answered - Don’t know / Can’t remember (n=1) - Before your treatment restarted were your treatment options explained to you?

Information provided when treatment had to be restarted* *Respondents could select all answer options that applied (tick all that apply) Q 46 / base = all respondents (n=159) - What information was provided to you when you were told that you had to restart the treatment? *Respondents could select all answer options that applied (tick all that apply) Q 47 / base = all respondents (n=159) - Who provided you with the necessary information?

Treatment following restarting Q 48 / base = all respondents (n=153) - Did you restart on the same treatment that you had been taking before stopping? *Respondents could select all more than one answer (tick all that apply) Q 49 Which treatment did you switch to? / base = (n=46) respondents answering that they switched treatment to Q 48 - Did you restart on the same treatment that you had been taking before stopping?

Physical and emotional change after restarting treatment* *Respondents could select all answer options that applied (tick all that apply) Q 50 / base = all respondents (n=159) - In the first weeks after restarting the treatment, how did you feel physically? Q 51 / base = all respondents (n=159) - In the first weeks after restarting the treatment, how did you feel emotionally?

Physical and emotional change after restarting treatment* -by Gender A higher proportion of female patients reported feeling worse both physically and emotionally after restarting treatment then before they stopped. A higher proportion of female patients reported feeling worse both physically and emotionally after restarting treatment then during the stopping period. *Respondents could select all answer options that applied (tick all that apply) Q 50 / base = all respondents - In the first weeks after restarting the treatment, how did you feel physically? Males (n=51) Females (n=106) Q 51 / base = all respondents - In the first weeks after restarting the treatment, how did you feel emotionally? Males (n=51) Females (n=106)

Impact of side effects after RESTARTING treatment Respondents were asked to grade the impact of the listed side effects from 1 -5 1 meant the side effect mildly affected their everyday life, and 5 meant the side effect completely effected everyday life. Side effects not experienced were to be left blank. The chart below gives the mean score for each side effect Q 52 / all respondents - Please rate the extent to which you were affected by the following side effects when you RESTARTED treatment Fatigue (n=121) Disturbed sleep (n=96) Muscle soreness or cramping (n=103) Feeling distressed (n=84) Anxiety (n=91) Feeling of malaise (n=86) Feeling sad (n=86) Pain (n=79) Rash or skin change (n=82) Swelling of hands, feet, abdomen, and around eyes (n=91) Difficulty thinking clearly (n=92) Remembering things (n=83) Headaches (n=77) Nausea (n=82) Shortness of breath (n=80) Hair loss (n=76) ) Numbness or tingling (n=74) Diarrhoea (n=81) Skin pigment change (n=70) Dry mouth (n=78) Menstrual cycle issues (n=55) Lack of appetite (n=66) Eye bleeds (n=77) Bruising (n=74) Vomiting (n=63) Other (n=14)

Comparison of side effects before stopping treatment and after restarting treatment Q 53 / base = all respondents (n=152) - Overall, when you restarted, how did side effects compare to before you stopped treatment?

Disease management after restarting treatment Q 54 a / base = all respondents (n=149) - I feel my disease is under control again now I have restarted treatment Q 54 b / base = all respondents (n=117) - I feel my disease is better monitored when I have treatment

Psychological support when restarting treatment (1) Q 55 / base (n=153) Excludes those who answered Don’t know/can’t remember (n=0) - Did you receive psychological and/or emotional support when you restarted your treatment? (This can be from any source such as a health professional, family or support groups)

Psychological support when restarting treatment (2) – by Gender A higher percentage of female patients said they received psychological/emotional support , compared to male respondents. Male respondents were more likely to say that this type of support was not necessary. Q 55 / base = all respondents - Did you receive psychological and/or emotional support when you restarted your treatment? Males (n=51) Females (n=102)

Psychological support when restarting treatment (3) *Respondents could select all answer options that applied (tick all that apply) Q 56 What psychological support did you receive? / base = (n=40) respondents answering ‘Yes’ to Q 55 - Did you receive psychological and/or emotional support when you restarted your treatment?

Feelings on considering stopping treatment in the future Q 57 / base = all respondents (n=158) - Do you think you will consider stopping your treatment again in the future?

Phase IIIB This section of the questionnaire was answered by respondents who stopped treatment and are in long-term remission beyond the 6 month probation period Maximum base (n=203) = Respondents who answered – No, I am in long-term remission to Q 41 - Following stopping treatment, did the disease reoccur and treatment have to restart? + Respondents who answered – Yes, I have already stopped treatment again and it has been successful to Q 57

Major concerns about being in long-term remission* *Respondents could select all answer options that applied (tick all that apply) Q 58 / base = all respondents (n=203) - What are your major concerns about being in Treatment Free Remission?

Changes would make to stopping treatment experience* *Respondents could select all answer options that applied (tick all that apply) Q 59 / base = all respondents (n=203) - What would you change about your experience of stopping treatment?

Monitoring in long-term remission (1) Q 60/ base = all respondents (n=199) - How often are you monitored by your doctor by a PCR test? Q 61/ base = all respondents (n=199) - How do you feel about the frequency of monitoring / PCR tests whilst in Treatment Free Remission (TFR)?

Monitoring in long-term remission (2) Q 62/ base = all respondents (n=200) - Do you keep to the monitoring schedule set by your doctor?

Monitoring in long-term remission (3) Q 63/ base = all respondents (n=199) - How concerned are you with fluctuations (PCR going up and down) / changes in your PCR test results?

Monitoring in long-term remission (4) - by Gender Q 63/ base = all respondents - How concerned are you with fluctuations (PCR going up and down) / changes in your PCR test results? Males (n=97) Females (n=101)

Psychological support in long-term remission (1) Q 64/ base = all respondents (n=197) - Do you receive psychological and/or emotional support? (This can be from any source such as a health professional, family or support groups) *Respondents could select all answer options that applied (tick all that apply) Q 65 What psychological support do you receive? / base = (n=49) respondents answering ‘Yes’ to Q 64 - Do you receive psychological and/or emotional support? (This can be from any source such as a health professional, family or support groups)

Psychological support in long-term remission (2) - by Gender Males in long-term remission were much more likely to say that they did not feel psychological/emotional support was necessary for them. Females in long-term remission were more likely to say they received psychological/emotional support. Q 64/ base = all respondents - Do you receive psychological and/or emotional support? (This can be from any source such as a health professional, family or support groups) Males (n=97) Females (n=99)

Care in long-term remission Q 66/ base = all respondents (n=198) - Overall, do you feel you receive adequate care in this Treatment Free Remission phase?

Respondents’ views on how doctor could improve their experience of stopping treatment *Respondents could select all answer options that applied (tick all that apply) Q 67/ base = all respondents (n=203) - How could your doctor improve your experience of stopping treatment?

Respondents’ views on if they still feel like a CML patient Q 68/ base = all respondents (n=196) - Do you feel you are still a CML patient?

Final Advice Maximum base (n=494) – All respondents who answered - Yes - to Q 23 - Did you proceed to stopping treatment?

Respondents’ advice to patients who are considering stopping treatment* (Answered by all respondents who stopped) *Respondents could select all answer options that applied (tick all that apply) Q 69 What advice would you give to a patient that is considering stopping treatment? / base = (n=494) All respondents who answered - Yes - to Q 23 - Did you proceed to stopping treatment?

Respondents’ advice to patients who are considering stopping treatment*-by Gender *Respondents could select all answer options that applied (tick all that apply) Q 69 What advice would you give to a patient that is considering stopping treatment? / base = all respondents who answered - Yes - to Q 23 - Did you proceed to stopping treatment? Males (n=196) Females (n=293)

Respondents’ advice to patients who are considering stopping treatment* - by CML Advocate group region *Respondents could select all answer options that applied (tick all that apply) Q 69 What advice would you give to a patient that is considering stopping treatment? / base = all respondents who answered - Yes - to Q 23 - Did you proceed to stopping treatment? Western Europe (n=258) Asia and Pacific (n=70) North America (n= 67) Middle East and Africa (n=41) Not Classified (n=40)

Respondents’ advice to patients who are considering stopping treatment* - by CML Advocate group region *Respondents could select all answer options that applied (tick all that apply) Q 69 What advice would you give to a patient that is considering stopping treatment? / base = all respondents who answered - Yes - to Q 23 - Did you proceed to stopping treatment? Western Europe (n=258) Asia and Pacific (n=70) North America (n= 67) Middle East and Africa (n=41) Not Classified (n=40)

Additional analysis

Psychological Support Respondents whose disease had reoccurred were more likely to want psychological/emotional support, but had the highest proportion who said they did not receive this Q 35 / base (n= 472) Excludes those who answered Don’t know/can’t remember (n=9) - Did you receive psychological and/or emotional support during the stopping of treatment? (This can be from any source such as a health professional, family or support groups) Q 55 / base all respondents (n=153) - Did you receive psychological and/or emotional support when you restarted your treatment? (This can be from any source such as a health professional, family or support groups) Q 64/ base = all respondents (n=197) - Do you receive psychological and/or emotional support? (This can be from any source such as a health professional, family or support groups)

Psychological Support Respondents whose disease had reoccurred were more likely have counselling and support from friend or family. However they were least likely to say they got support from patient organisations or social media. Respondents in long term-remission were more likely to receive support via patient organisations or social media. *Respondents could select all answer options that applied (tick all that apply) Q 36 What psychological support did you receive? / base = (n=93) respondents answering ‘Yes’ to Q 35 - Did you receive psychological and/or emotional support during the stopping of treatment? ? (This can be from any source such as a health professional, family or support groups) Q 56 What psychological support did you receive? / base = (n=40) respondents answering ‘Yes’ to Q 55 - Did you receive psychological and/or emotional support when you restarted your treatment? (This can be from any source such as a health professional, family or support groups) Q 65 What psychological support did you receive? / base = (n=49) respondents answering ‘Yes’ to Q 64 - Do you receive psychological and/or emotional support? (This can be from any source such as a health professional, family or support groups)

Key Observations

General observations • The most common reason patients consider TFR is to attempt to reduce/eliminate the side effects of treatment (51%) • 53% of women gave this reason compared to 49% of men • 73% of patients report that their doctor supported their decision to try stopping treatment, however this varies from 85% down to 35% between different regions • 66% were monitored every month or more frequently during the probation period, but this varies between regions • 67% completely agreed they receive adequate care whilst in long term remission, however this varies between regions

Hearing about TFR for the first time • Less than half of respondents (49%) first heard about stopping treatment from a healthcare professional • Just under a quarter (21%) first heard about stopping treatment from a • patient organisation There are differences between the CML Advocate regions as to where patients report first hearing about TFR: • Over half of Western Europe (57%) and Latin America (53%) patients first heard from a healthcare professional. Compared to 40% of Central & Eastern Europe & West Asia and 41% of Middle East and Africa, or 38% of patients from countries without a designated Advocate Group • 43% of Central & Eastern Europe & West Asia and 43% of Middle East and Africa first heard from a patient organisation compared to 6% in North America and 15% in Western Europe

Psychological and Emotional Support • During the stopping phase, only 18% of respondents said they discussed how to deal with psychological aspects with their doctor • Although a high proportion of patients report feeling psychological/emotional support was not necessary, this varies by gender and point in TFR journey: • Female patients are more likely to have worries and fears, and want psychological/emotional support than male patients • 32% of female patients who stopped would advise other patients to get psychological support • 43% of female patients who stopped would advise other patients to get emotional support • Patients whose disease had reoccurred are more likely to want support than during the stopping phase, or those who are in long-term remission • There are patients across all the points in the TFR journey who want psychological/emotional support and are not receiving this

Support for Withdrawal Symptoms • 60% of respondents who had stopped treatment reported that they experienced some kind of withdrawal symptoms. However, opportunities to support patients with these are being overlooked: • Just 21% of patients discussed withdrawal symptoms with their doctor before making decision to stop treatment • 40% of respondents reported they would have like to have received information about withdrawal symptoms when making their decision • During the stopping phase only 39% of respondents discussed with • their doctor how to deal withdrawal symptoms Over half of the respondents said that they weren’t asked if they were experiencing withdrawal effects during stopping • 27% of all respondents weren’t asked if they were experiencing withdrawal effects during stopping but would have liked to been • asked Of the respondents who experienced physical withdrawal symptoms during stopping treatment; 24% said they did not have doctor support to manage them, but would have liked this

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