Abstract
Polycythemia vera (PV) is a myeloproliferative neoplasm associated with a high symptom and psychological burden, resulting in decreased quality of life (QoL). Patients with PV have an increased risk of cardiovascular (CV) complications, making regular monitoring crucial. The Landmark 2.0 survey was conducted worldwide among patients with PV and their treating physicians to identify any potential gaps in perceptions regarding PV management. Data were collected between April 2021 and April 2022 from physicians and patients across 11 countries. Overall, 133 physicians and 274 patients with PV participated in the survey. There were discrepancies between physicians and patients in reporting whether symptom assessments and basic CV assessments were conducted during routine visits (83% vs. 68% and 64% vs. 55%, respectively). Emotional assessments were not performed routinely (reported by 36% of physicians and 34% of patients). Patients attributed the highest impact on QoL to physical symptoms (67%); however, physicians were less likely to report highly prevalent symptoms such as bruising, difficulty sleeping, inactivity, and depression among the most common symptoms. While both physicians and patients aimed for symptom improvement, their treatment goals differed: physicians focused on managing hematocrit, preventing thrombotic events, and reducing spleen size, while patients focused on slowing down disease progression. Patient satisfaction with treatment was generally high but decreased in later therapy stages. Overall, these data underscore the disparity in patient–physician perceptions of PV management and treatment expectations, showing the gaps in communication and the need for greater patient education, as well as highlighting areas for potential improvement in clinical practice.
INTRODUCTION
Myeloproliferative neoplasms (MPN) are rare hematological disorders that include myelofibrosis (MF), polycythemia vera (PV), and essential thrombocythemia (ET). PV is characterized by excessive production of red blood cells and is associated with a high symptom and psychological burden that leads to reduced quality of life (QoL). 1 , 2 , 3 Signs and symptoms frequently reported in PV include fatigue, insomnia, pruritus, concentration problems, and splenomegaly. 2 , 3 Patients with PV have a high risk of developing thrombosis and cardiovascular (CV) events; up to 40% of patients with PV experience thrombotic events (TEs), which are the main cause of death from the disease. 2 , 4 , 5 , 6 The 10‐year relative survival of individuals with PV is around 73% of that expected in the general population matched for age and sex. 7 There is no known cure for PV, but available evidence shows that control of cytosis (particularly hematocrit) allows for the reduction of complications (such as vascular complications) and improves disease outcome. 2 , 3
The US and global MPN Landmark surveys aimed to understand the journey for patients with MPNs, and assess the patient experience. 8 , 9 , 10 In both surveys, most patients with PV reported that their disease symptoms reduced their QoL, and a substantial proportion experienced interference with their daily activities 8 , 10 ; a negative impact of the disease was also reported by patients with a low prognostic risk score and low symptom burden. These findings align with the results from the Living with MPN survey, which included 592 patients in the United States and noted the negative impact of MPNs on work productivity and employment status. 11 , 12 Furthermore, both surveys identified discrepancies between physicians and patients with PV regarding their top treatment goals. 9 , 10 In the US survey, disparities were also noted in physician‐ and patient‐assessed symptom burden. These differences in perception of symptom burden can lead to suboptimal treatment, which can ultimately affect patient outcomes. 9 Additionally, a large proportion of patients participating in the US survey expressed dissatisfaction with the management and communication from their physicians. 9
Landmark 2.0 is a comprehensive worldwide survey focusing on patients with MPNs (MF and PV) and their treating physicians to evaluate the level of alignment between patient and physician perceptions in the assessment, monitoring, and management of the disease. The survey aims to identify any potential gaps in the patient journey for patients with MF and PV, develop strategies to overcome these, and ultimately provide recommendations for improved patient management. The focus of the present work is specifically on patients with PV.
MATERIALS AND METHODS
Survey instrument
The Landmark 2.0 global health survey is a multi‐country, cross‐sectional online survey of patients with MF and/or PV and physicians who treated these patients to gather insights and gain a comprehensive understanding of the MF and PV patient journey in a real‐world setting. The main aim of the survey was to generate actionable recommendations to improve the management of patients with MF and PV through the identification of gaps in treatment and monitoring of patients with MF and PV.
Data were collected between April 2021 and April 2022 from eligible physicians and patients based in Australia, Brazil, Canada, China, France, Germany, Italy, Japan, Spain, Switzerland, and the UK. The physician and patient surveys were conducted independently, with no direct link established between their responses. The physician and patient surveys were conducted online and required approximately 35 and 25 min to complete, respectively. Each respondent completed a single one‐time survey, with no follow‐up or re‐contact. Eligible physicians participated in a PV‐specific survey depending on their caseload. The survey included various sections that gathered demographic information about the physicians and their consulting population of patients with PV. Additionally, data on types and frequency of disease assessment and monitoring, treatment satisfaction, burden of disease/care, communication between healthcare professionals and patients, and treatment expectations were also collected.
Patients were assigned to a survey that was specific to their PV diagnosis and were also assigned into one of the three patient groups depending on their ongoing therapy stage. The patient survey included patient demographics, types and frequency of disease assessment and monitoring, disease and treatment experience, disease burden, communication between healthcare professionals and patients, and treatment expectations.
Survey questionnaires can be found in supplementary Materials and Methods.
Study population
Eligible physicians were primary physicians who specialized in hematology, hemato‐oncology, or medical oncology, had qualified between 1989 and 2015, and had experience in managing patients with PV for a minimum of 12 months before the survey. Physicians were excluded if their caseloads were fewer than 12 cases (or 10 cases for Japan‐based physicians), or if they spent less than 1% of their time seeing patients with PV. All physicians were recruited anonymously through a fieldwork agency to complete the survey.
Eligible patients were aged 18–89 years with a confirmed diagnosis of PV. Patients were not eligible to take the survey if they were receiving a JAK inhibitor other than ruxolitinib or if they were participating in a clinical trial. Patient recruitment occurred via two approaches: in all participating countries, patients were recruited by collaborating physicians during routine consultations. Additionally, in Canada, Germany, Italy, Spain, and the UK, patient advocacy groups facilitated patient recruitment.
Patients with PV were grouped by therapy stage into three categories: patients at initiation of cytoreductive therapy (PV‐1L) who had been treated with hydroxyurea or interferon as first‐line treatment for <1 year; patients at initiation of second‐line treatment (PV‐2L) who had been receiving a second‐line treatment for <1 year; patients on ongoing management (PV‐M) receiving any line of therapy for 1 year or more.
Statistical analysis
Analyses were descriptive in nature and no formal hypothesis was tested. For numerical variables, the respondent base was reported together with the mean, standard deviation, median, and interquartile range (IQR). For categorical variables, the total number of respondents, along with the number and percentage of responses, are presented.
Ethics
The study (including the informed consent form) was approved by the central WCG IRB (reference #20211308 for the protocol and #1305368 for the study). All participants provided informed consent before completing the relevant survey.
RESULTS
Demographics and clinical characteristics
A total of 133 physicians managing patients with PV responded to the survey, of whom almost two‐thirds (60%) were specialist hematologists (Table 1). Approximately two‐thirds of responding physicians were based in a specialist center or university teaching hospital, with only 21% of physicians based in regional or community hospitals. The number of responding physicians in each country is presented in Table S1.
Table 1.
Demographics of physician respondents.
| PV physicians (N = 133) | |
|---|---|
| Primary specialty, n (%) | |
| Hematologist | 80 (60) |
| Hemato‐oncologist | 50 (38) |
| Medical oncologist | 3 (2) |
| Practice setting, n (%) | |
| Specialist cancer center/MPN center | 15 (11) |
| University teaching hospital | 75 (56) |
| Regional/community hospital | 28 (21) |
| Private hospital | 12 (9) |
| Other | 3 (2) |
| Treatments prescribed, n (%)a | |
| Hydroxyurea/hydroxycarbamide | 132 (99) |
| Ruxolitinib | 117 (88) |
| Interferon | 94 (71) |
| Number of patients managed in the past 12 months, mean (SD) | 42.3 (67.2) |
| Proportion (%) of time per week spent in clinic seeing patients with PV, mean (SD) | 35.8 (32.4) |
Abbreviations: N, number of patients in a group; n, number of patients with a characteristic; PV, polycythemia vera; SD, standard deviation.
This was a multiple choice question; therefore, responses add up to more than 100%.
Overall, 274 patients with PV responded to the survey. The number of responding patients in each country is presented in Table S2. The median age of respondents with PV was 63 years (range 29–88) and 56% were female (Table 2). The majority of patients (88%) had received hydroxyurea; most patients were at the ongoing management stage and on their second line of treatment (PV‐M, 59%).
Table 2.
Overall patient demographics and treatment characteristics.
| Overall patients (N = 274) | |
|---|---|
| Median age (range), yr | 63 (29–88) |
| Male, n (%) | 121 (44%) |
| Median time since diagnosis (range), wk | 156 (4–1716) |
| Treatments ever received for PV, n (%) | |
| Hydroxyurea | 240 (88) |
| Interferon | 51 (19) |
| Ruxolitinib | 62 (23) |
| Current treatment for PV, n (%) | |
| Hydroxyurea | 184 (67) |
| Interferon | 35 (13) |
| Ruxolitinib | 55 (20) |
| PV therapy stage, n (%) | |
| PV‐1L | 86 (31) |
| PV‐2L | 26 (9) |
| PV‐M | 162 (59) |
| Patients receiving phlebotomy as treatment for PV, n (%) | 101 (37) |
| PV‐1L | 46 (53) |
| PV‐2L | 8 (31) |
| PV‐M | 47 (29) |
| Number of phlebotomies per year, median (range) | 3 (2–6) |
| PV‐1L | 3 (2–5) |
| PV‐2L | 3 (2–6) |
| PV‐M | 3 (2–6) |
| Patient‐reported current state of health, n (%) | |
| Very poor | 0 |
| Poor | 13 (5) |
| Fair | 85 (31) |
| Good | 102 (37) |
| Very good | 59 (22) |
| Excellent | 14 (5) |
| Median duration of current treatment (range), wk | 104 (1–1716) |
Abbreviations: N, number of patients in a group; n, number of patients with a characteristic; PV, polycythemia vera; PV‐1L, patients at initiation of cytoreductive therapy, treated with hydroxyurea or interferon as first‐line treatment for <1 year; PV‐2L, patients at initiation of second‐line treatment, receiving a second line of treatment for <1 year; PV‐M, patients on ongoing management, receiving any line of therapy for 1 year or more.
Overall, 101/274 patients (37%) were receiving phlebotomy as treatment for PV; the highest proportion of patients receiving phlebotomy was among those in PV‐1L (Table 2). Median (IQR) number of phlebotomies in the past 12 months for all patients with PV was 3.0 (2‒6). Nearly half of all patients (44.6%) stated that phlebotomies had minimal or no impact on their QoL.
More than a third of patients at each disease stage described their current health as fair or poor, with the highest proportion in those initiating their second‐line treatment (PV‐2L, 46%) (Table S3). The median duration of current treatment for all patients was 104 weeks (range 1–1716). The majority of patients in PV‐2L switched their first‐line treatment to receive ruxolitinib (69%).
Assessment and monitoring of PV
The majority of patients with PV (82%) expressed satisfaction with the number of visits related to their condition; however, 7% felt they had too many physician visits, and 12% felt they did not have enough visits. Patients in PV‐2L reported visiting their treating physician more regularly than patients in other therapy groups, with a mean of 7.4 physician visits in the past 6 months compared with the PV‐1L group, which had a mean of 5.7 physician visits. Overall, patients and physicians reported similar assessments conducted during routine visits, including blood tests, spleen assessments, and overall emotional assessments. However, there was a discrepancy in the reported frequency of symptom and CV assessments (Figure 1).
Figure 1.
Comparison between proportions of physicians and patients reporting assessments at routine clinical visits. CV, cardiovascular.
Most physicians (>80%) consistently reported conducting symptom assessments at routine visits in all stages of PV treatment. However, only 68% of patients reported having had their symptoms assessed, with most (68%) reporting being very or somewhat satisfied with the frequency of symptom assessment. Symptom assessment was most frequently performed by physicians in patients in PV‐1L (Figure S1A), whereas patients in PV‐2L and PV‐M expressed the highest levels of dissatisfaction with the frequency of symptom assessments (24% and 9%, respectively). The assessment of symptoms at routine visits was reported by only 59% of patients in PV‐M, compared to 83% and 81% of patients in PV‐1L and PV‐2L, respectively (Figure S1B).
Although the reported frequencies of emotional assessments and basic CV assessments at routine visits were aligned between physicians and patients, these assessments were performed relatively infrequently (proportion reported by physicians vs. patients: 36% vs. 34% and 64% vs. 55%, respectively) (Figure 1). The frequency of basic CV assessment (e.g., blood pressure and cholesterol assessments) varied by disease stage and was ever performed at routine visits by 77%, 62%, and 53% of physicians treating patients in PV‐1L, PV‐2L, and PV‐M, respectively, whereas a comprehensive CV assessment (e.g., electrocardiogram and/or echocardiogram) was ever performed by 60%, 47%, and 32% of physicians, respectively (Figure S1A).
In contrast to physicians' responses, only 55% of patients across treatment stages reported having ever received basic CV assessments during routine visits (Figure 1). Fewer patients in PV‐M received advice on reducing CV risk (45% in the last 12 months) compared to patients in PV‐1L and PV‐2L (73% and 69%, respectively, in the last 6 months).
Among physicians who ever performed emotional burden assessments, the majority (73%–87%) across all therapy groups reported that they always/often conducted these assessments (Figure S1C). In contrast, among patients ever receiving these assessments, only 50%‒55% of patients in PV‐1L and PV‐M, and 10% in PV‐2L reported always/often receiving these assessments.
Among physicians who never conducted basic (n = 21) or comprehensive (n = 40) CV assessments, 38% and 43%, respectively, considered the assessment unnecessary (Figure 2). Physicians' reasons for not performing these assessments also included insufficient time (38% and 15%, respectively) or insufficient resources (19% and 35%, respectively). Overall, 32% and 37% of all physicians believe that basic or comprehensive CV assessments, respectively, could be used more effectively in patient management.
Figure 2.
Physician‐reported reasons for not performing emotional burden, basic, and comprehensive CV assessments. CV, cardiovascular.
While the reported frequencies of assessments of skin conditions (such as skin cancers and leg ulcers) at routine visits were aligned between physicians and patients, these assessments were performed relatively infrequently (physicians vs. patients: 33% and 28%, respectively) (Figure 1).
Approximately one‐third of physicians deemed the evaluation of emotional burden unnecessary (31%) (Figure 2). Other reasons for not performing these assessments included lack of time (33%) and insufficient staff resources (25%). However, 43% of physicians across all treatment stages reported that the assessment of emotional burden might be more effectively used to optimize patient management.
Despite the disconnect in reported frequency of symptom assessment (particularly in patients in PV‐M), physicians and patients were overall aligned on the benefits of self‐monitoring of symptoms. Over 90% of physicians believed that self‐monitoring of symptoms would be somewhat/very beneficial for patients, as this would improve their ability to monitor changes in symptoms and improve patient–physician communication. In agreement with this result, 84% of patients across therapy stages believed that self‐monitoring would enable them to better explain their symptom burden to their treating clinician and improve their ability to monitor changes in symptoms.
Burden of PV disease/care
Physicians recognized the most common PV symptoms identified by patients including fatigue, itching, and abdominal discomfort, but were less likely to include the highly prevalent symptoms of bruising, difficulty sleeping, inactivity, night sweats, and depression among the top reported symptoms (Figure 3).
Figure 3.
Top symptoms reported by patients according to patients and physicians.
The highest impact on QoL was attributed to physical symptoms by most patients across therapy stages (all patients, 67%; PV‐1L, 67%; PV‐2L, 50%; PV‐M, 69%), whereas lower impact on QoL was reported for emotional well‐being (all patients, 23%; PV‐1L, 26%; PV‐2L, 27%; PV‐M, 21%) or economic status (all patients, 7%; PV‐1L, 7%; PV‐2L, 19%; PV‐M, 5%) (Figure S2). Interestingly, more physicians than patients reported a large negative impact of PV on emotional well‐being (55% vs. 33%) and economical status (39% vs. 21%) (Figure 4). Overall, most patients and physicians felt very or somewhat comfortable discussing the impact of PV on their emotional well‐being and economic status.
Figure 4.
Burden of PV on emotional and economic status and comfort with discussing. (A) Impact on emotional well‐being and mental health. (B) Impact on economic status. (C) Comfort discussing PV emotional impact with patient or physician. (D) Comfort discussing PV economic impact with patient or physician. Impact is scored from 0 to 5, where 0 represents that PV has no impact and 5 represents that PV has a significant impact. PV, polycythemia vera.
Treatment expectations and treatment satisfaction
Physicians and patients were aligned on treatment goals such as symptom improvement and better QoL (Figure 5). However, more physicians than patients reported the prevention of vascular events/TEs and maintaining hematocrit <45% among the top 3 treatment goals; slowing or delaying PV progression was reported more frequently by patients as compared to physicians (47% vs. 23%, respectively). Regarding therapy stage, physician‐perceived and patient‐reported treatment goals differed particularly in PV‐M, with patient goals more focused on delaying the progression of the disease and less on symptom and QoL improvement compared to other therapy stages (Table S4).
Figure 5.
Comparison between physician‐reported and patient‐reported treatment goals. Physicians and patients could indicate three top treatment goals from a list. PV, polycythemia vera.
Physicians considered that most of their top treatment goals could be achieved with currently available treatment options (very feasible and largely feasible: symptom improvement, 16% and 53%; prevention of vascular events/TEs, 27% and 44%; maintaining hematocrit <45%, 25%, and 56%, respectively). However, a large proportion of physicians agreed that there was room for improvement, particularly in maintaining healthy blood counts and reducing the frequency of phlebotomy. Patients generally felt that their main treatment goals were achieved, though satisfaction was lower for patients in PV‐2L, with a larger proportion reporting their treatment goals were not met.
In terms of communication on treatment goals, 90% of physicians reported discussing treatment goals with their patients; in contrast, only 65% of patients reported having discussed treatment goals with their physician (Figure S3A). This disparity was more pronounced for patients in PV‐M (Figure S3B).
There were notable differences in treatment satisfaction levels between physicians and patients, particularly regarding significant symptoms. Physicians generally reported higher levels of treatment satisfaction than patients across most significant symptoms; lowest levels of satisfaction were reported for the management of fatigue (Figure 6A). Substantial proportions of patients expressed being very or somewhat satisfied with the management of symptoms such as itching, night sweats, and abdominal discomfort. The lowest proportions of satisfied patients were recorded for the management of fatigue, depression, bruising, and inactivity (Figure 6B).
Figure 6.
Satisfaction with the management of significant symptoms. (A) Physician‐reported satisfaction. (B) Patient‐reported satisfaction.
Physician‐reported satisfaction with PV management options was higher when treating patients in PV‐1L than those in later therapy stages; however, the proportion of physicians who reported being very/somewhat dissatisfied with disease management options remained constant across therapy stages (Figure S4A). Patient satisfaction with available treatments decreased with the therapy stage: 21% of patients in the ongoing monitoring phase (PV‐M) expressed dissatisfaction with therapies they were receiving (Figure S4B).
Only half of patients reported being very or somewhat satisfied with the management of the emotional burden of PV (24% and 28%, respectively). Conversely, 60% of physicians reported satisfaction (very/somewhat) with the support their patients received for emotional burden of PV.
Physician/patient communication
Physicians and patients were aligned on the majority of information topics communicated, such as disease background, types of symptoms, types of available treatments, and their associated side effects (Figure 7). However, fewer patients reported receiving information regarding the rationale for treatment selection, strategies to help patients to cope with PV, disease prognosis, and rationale for performing specific tests. This disconnect was observed across all therapy stages (Table S5) and was in contrast to the fact that physicians and patients were aligned on the sufficiency of the information provided, with over 60% of physicians and patients satisfied with the extent of the information provided (Figure S5).
Figure 7.
Comparison between information communicated by physicians and information patients report receiving at the initiation of current treatment. PV, polycythemia vera.
DISCUSSION
The Landmark 2.0 worldwide survey aimed to gather insights from both patients with PV and their treating physicians to gain a comprehensive understanding of the PV patient journey in a real‐world setting. The results highlight gaps in patient and physician perception of PV symptoms, assessments, treatment goals, and satisfaction.
Most responding patients were at the ongoing management stage of their disease, and more than a third of patients overall described their current health as fair or poor. This is in agreement with results from the US and international MPN Landmark 1.0 surveys, which found significant symptom burden and impact in QoL associated with MPNs. 8 , 10
There were discrepancies regarding reported symptom assessment between physicians and patients (83% vs. 68%), mostly for patients in ongoing management. Despite these differences, physicians' and patients' perspectives were aligned on the importance of self‐monitoring PV symptoms. A substantial proportion of responding physicians reported not performing basic or comprehensive CV assessments as part of routine clinical practice; for both basic and comprehensive CV assessments, the proportion of physicians performing these assessments decreased in patients in more advanced therapy stages. The main reasons given by physicians for not performing these assessments were that they considered them unnecessary and lacked time and/or resources. Fewer patients reported ever receiving basic CV assessments during routine visits than physicians reported performing them; however, this could be explained by patients not fully understanding what these assessments or tests involve, or by the assessments being carried out by a general practitioner or cardiologist outside of routine visits. Most physicians reported not performing emotional burden assessments routinely, again citing a lack of time and resources and no need for these assessments as reasons not to perform them. It is possible that some of these assessments were not reimbursed by health insurance at the physicians' institution or country. Dedicated cardio‐oncology clinics are established in some centers and may provide an opportunity to assess and optimize vascular risk.
The considerable proportion of physicians reporting lack of time or resources to perform basic or comprehensive CV assessments, or emotional assessments highlights that physicians face barriers, which may impact their ability to conduct assessments that are helpful for patients. Given these barriers, self‐assessment of symptoms could help drive treatment discussions during routine visits. Recognition by physicians of the full burden of disease associated with PV is imperative for patients to receive optimal treatment; however, the observed discrepancies suggest that physicians may not fully appreciate PV‐associated signs and symptoms, which could result in patients with PV not switching to more effective treatments at the right time. It is possible that patients are not reporting their full range of symptoms at routine visits: this phenomenon has been widely studied in chronic myeloid leukemia and other diseases. 13 , 14 , 15 Patients' cultural approaches to liaising with healthcare professionals also differ across countries and may affect patient–physician communication. 16 Patient education on the relevance of reporting their symptoms may then improve communication with treating physicians regarding symptoms. Frequent use of validated patient‐reported outcome tools such as MF Symptom Assessment Form (MPN‐SAF) and European Organization for Research and Treatment of Cancer Quality‐of‐Life Questionnaire‐Core 30 (EORTC QLQ‐C30) could enhance the number and severity of symptoms captured; online versions of these tools may further increase capture and facilitate their use. 17 A new standardized tool for assessing patient‐reported symptoms may also be needed to improve the management of patients with PV.
When prompted, physical symptoms were considered by two‐thirds of patients to be the most burdensome disease factor on their QoL, followed by the impact on emotional well‐being/mental health; the same pattern was observed irrespective of therapy stage. Notably, physicians considered the impact of PV on the emotional well‐being of patients to be larger than that reported by patients themselves. Physicians also reported that the assessment of emotional burden might be more effectively used in patient management. The importance of psychological distress was highlighted in a recent study, which found high distress and low treatment adherence to be correlated, underscoring the importance of integrating psychological support into the management of PV. 18
While results show that physicians appreciated the burden of physical symptoms, many highly prevalent symptoms did not appear to be recognized by reporting physicians; this is in agreement with the results from the US MPN Landmark 1.0 survey, where physicians reported that lower proportions of patients with PV experienced symptoms at diagnosis compared to patient‐reported data. 9 Interestingly, patients reported the lowest levels of treatment satisfaction for most of these unrecognized symptoms. For bruising, it is possible that physicians did not report it as a disease‐related symptom as it may be considered an adverse event associated with the anticoagulant treatment that patients usually receive. More structured assessments allowing for the discrimination of signs and symptoms associated with the disease and those associated with the side effects of treatments used would allow to improve this gap in communication.
There is an increased risk of skin cancer associated with both hydroxyurea and ruxolitinib, which may be exacerbated in participating countries with high sun exposure and a substantial population with fair skin. 19 , 20 , 21 Hydroxyurea is associated with significant cutaneous toxicities, such as leg ulcers. 20 However, the reported frequency of skin assessments in routine visits in this survey was low. It is possible that some patients had skin assessments performed independently through a dermatologist or skin cancer clinic. Better education regarding sun protection and surveillance for cutaneous complications of therapy may be helpful.
Regarding treatment goals, symptom improvement was reported as a key goal for physicians and patients alike. However, for other treatment goals, physicians focused on maintaining markers such as hematocrit and spleen size within certain margins, whereas patients focused on more general goals such as delaying disease progression. It is possible that physicians stated their goals based on the known efficacy of existing therapies, whereas patients directly stated their personal goals. Interestingly, reduced frequency of phlebotomies was an important goal for only a minority of patients (12%) and was twice as often prioritized by physicians. Remarkably, there was also a discrepancy between physicians and patients regarding whether treatment goals were discussed during visits; this discrepancy appeared to have been driven mostly by patients in PV‐M. Considering that TEs are the leading cause of mortality in patients with PV, 2 , 4 , 5 , 6 it is somewhat surprising that the prevention of TEs was not a top treatment goal for more physicians. However, this finding aligns with a substantial number of physicians who reported not performing basic or comprehensive CV assessments, with some viewing them as unnecessary. Responding patients also highlighted delaying disease progression as a key treatment goal in the US and international MPN Landmark 1.0 surveys 9 , 10 ; however, many current treatments for PV are not efficacious at modifying disease. 22 , 23 These results suggest a gap in patient understanding of what current treatments can do for them, showing that physicians should improve their communication of treatment outcomes to manage patient expectations.
Patient‐reported treatment satisfaction was generally high but decreased at later therapy stages; physician‐reported treatment satisfaction followed a similar pattern, although the difference between therapy stages was not as noticeable. The highest level of dissatisfaction with symptom management for both physicians and patients was reported for fatigue at all treatment stages. Dissatisfaction with the management of key symptoms was higher during ongoing management (PV‐M).
Overall, physicians considered that they provided sufficient information during visits, which was mostly aligned with patient‐reported data. However, some discrepancies were observed regarding certain topics such as the rationale for treatment selection, strategies to help patients to cope with PV, and disease prognosis, on which fewer patients reported having received information. In other diseases, communication between physicians and patients has been correlated with treatment adherence 9 ; improved physician communication around the identified topics could therefore increase treatment effectiveness through improved adherence. Patient advocacy organizations could help to address some of these information gaps by focusing on these topics in their educational material.
This study has several limitations, mostly a consequence of the data being self‐reported and descriptive, meaning that no statistical comparisons can be made. Diagnosis of PV was reported by patients themselves, which may constitute a limitation; however, since the recruitment approach was focused on networks of patients who had signed up to an MPN‐specific patient advocacy group (as opposed to open social media groups), the possibility of recruiting non‐genuine respondents was mitigated. The survey was carried out online, which may have been selected for younger patients (noting that the median age of respondents was approximately a decade younger than expected) and/or patients with a relatively high level of education and/or socioeconomic status. Female patients responded more often, whereas PV is more common in males. Data on symptoms and QoL were not obtained through validated instruments such as patient‐reported outcome tools, and so may not be as reliable. Some of the discrepancies reported may be the result of patients misremembering information regarding their disease management rather than due to lack of communication or diligence on the physician's side. The surveyed physician group may not accurately represent the physicians who treated the patients surveyed because they were not always matched (e.g., recruited patients were not always recruited with their treating physicians). As a result, some of the differences observed in reported rates of assessments could be attributed to this mismatch. The results may also be biased due to the characteristics of the responding populations: the majority of responding physicians were specialist hematologists working in specialist centers or teaching hospitals, whereas PV is quite rare in the general population. Patients from those countries where patient advocacy groups supported recruitment may have been particularly engaged with their disease management and more likely to report symptoms than those who were recruited directly by their treating physician. These factors may have resulted in a survey population that may not reflect the whole population of patients with PV and their treating physicians worldwide. Furthermore, the sample size of this survey limits our ability to draw definitive conclusions, and these findings require further validation in a study with a larger cohort. With a larger sample size, stratification of data by patient factors such as age or risk profile, and by physician experience or practice setting would provide further insights into the patient and physician perception of PV.
Overall, these data demonstrate a disparity in patient–physician perception of PV management and treatment expectations, which may impact treatment satisfaction, adherence, and ultimately outcomes for patients with PV. These findings underscore important learning points for the management of patients with PV (Table 3). Considering the importance of CV risk factors in PV, structured approaches to risk assessment and optimization (e.g., cardio‐oncology clinics or provision of information to primary care physicians) could be useful. Improved physician–patient communication and wider use of patient‐reported outcome instruments, as well as new or enhanced patient reporting tools, could have a positive impact in PV treatment outcomes.
Table 3.
Key learnings from the Landmark 2.0 survey.
| Gap | Potential approach to address | |
|---|---|---|
| 1. | Lack of basic or comprehensive CV assessments |
|
| 2. | Absence of regular assessment of the emotional burden of PV |
|
| 3. | Insufficient patient knowledge of symptoms leading to under reporting |
|
| 4. | Deficiency in physician recognition of the burden of physical symptoms |
|
| 5. | Gaps in patient knowledge of treatment goals |
|
Abbreviations: CV, cardiovascular; EORTC QLQ‐C30, European Organization for Research and Treatment of Cancer Quality‐of‐Life Questionnaire‐Core 30; MPN‐SAF, Myeloproliferative Neoplasm Symptom Assessment Form; PV, polycythemia vera.
AUTHOR CONTRIBUTIONS
All authors contributed to data interpretation and read and approved the final manuscript for submission. Laura Mirams, Abigail McMillan, and Gavin Harper participated in designing and coordinating the study and drafting the manuscript. Catalin Bobirca, Laura Mirams, Abigail McMillan, and Gavin Harper participated in analyzing data.
CONFLICT OF INTEREST STATEMENT
C. N. H. has received research funding (institutional) from Celgene, Constellation, and Novartis; has participated in advisory roles with AOP Orphan Pharmaceuticals, Celgene, CTI BioPharma Corp, Galecto, Gilead Sciences, Incyte, Keros, Novartis, Promedior, Roche, and Sierra Oncology; and has participated in speakers' bureau from AbbVie, AOP Orphan Pharmaceuticals, Bristol Myers Squibb, Celgene, CTI BioPharma Corporation, Gilead Sciences, Incyte, Janssen, Novartis, and Shire outside the submitted work. D. M. R. has consulted for Keros, and the institution received compensation; has received honoraria from Novartis personally and to the institution; has received compensation from Novartis to attend a meeting; and has received compensation to the institution for participation in advisory board meetings for Novartis, Menarini, and Takeda. L. F. has received research funding from Celgene, Incyte, PharmaEssentia, Constellation GSK, and CTI Biopharma; has received honoraria from Novartis and GSK; and has participated in advisory board meetings for GSK, Medison, Paladin labs, Incyte, Novartis, and Celgene. F. H. H. has participated in advisory roles for Novartis, AOP Orphan, Bristol Myers Squibb, GSK, Merck, AbbVie, and Kartos; and has received research funding from Novartis, Bristol Myers Squibb, and CTI. G. A. P. has had a consulting or advisory role for Novartis, AOP Orphan Pharmaceuticals, AbbVie, AstraZeneca, Celgene/Bristol Myers Squibb, GlaxoSmithKline, and Morphosys; has participated in speakers bureau for Novartis, AOP Orphan Pharmaceuticals, Celgene/Bristol Myers Squibb, and AbbVie; and has received travel/expense fees from Takeda, Novartis, BeiGene, AbbVie, and Johnson & Johnson. M. B. has received honoraria from AbbVie, AOP, Bristol Myers ‐Squibb, GSK, Incyte, Jazz, Novartis, and Pfizer. N. K. has received consulting fees from TORII and JT and is a board member of PharmaEssentia Japan KK. J. M. L. has received honoraria from Janssen Oncology, Novartis, Gilead, Roche, Astellas, Sanofi, and BMS; and has received research funding (institutional) from Bristol Myers Squibb/Celgene. A. R. has received research grants (institutional) from Novartis, CSL Behring, and AG Alexion; has received consulting fees from Novartis; and has received honoraria/participated in advisory board meetings for Novartis, Orpha Swiss GmbH, SOBI, and BMS. C. B. is a Novartis employee. L. M., A. M., and G. H. are employees of Adelphi Real World, who received funding from Novartis to conduct the study. J. J. K. has received honoraria from Novartis; has participated on a data safety monitoring board or advisory board meeting for AbbVie, AOP Orphan, Bristol Myers Squibb, Incyte, and Novartis. L. M. F, L. B., Z. X., M. K., K. K., B. X. C., and C. P. declare no conflicts of interest.
FUNDING
This study was funded by Novartis Pharmaceuticals Corporation.
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ACKNOWLEDGMENTS
The authors would like to thank the patients and physicians who took part in the survey, as well as the patient advocacy groups who facilitated patient recruitment: Canadian MPN Research Foundation (Canada), MPN Netzwerk (Germany), MPN España (Spain), AIPAMM (Italy), and MPN Voice (UK). Editorial assistance was provided by Vanesa Martinez Lopez, PhD, of Novartis Ireland Ltd, and was supported by Novartis Pharmaceuticals Corporation in accordance with Good Publication Practice (GPP22) guidelines (https://www.ismpp.org/gpp-2022).
DATA AVAILABILITY STATEMENT
Novartis is committed to sharing access to patient‐level data and supporting clinical documents from eligible surveys with qualified external researchers upon request. These requests are reviewed and approved by an independent review panel based on scientific merit. All data provided are anonymized to respect the privacy of patients who have participated in the survey consistent with applicable laws and regulations. For additional information, please visit www.clinicalstudydatarequest.com.
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Associated Data
This section collects any data citations, data availability statements, or supplementary materials included in this article.
Supplementary Materials
Supporting information.
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Data Availability Statement
Novartis is committed to sharing access to patient‐level data and supporting clinical documents from eligible surveys with qualified external researchers upon request. These requests are reviewed and approved by an independent review panel based on scientific merit. All data provided are anonymized to respect the privacy of patients who have participated in the survey consistent with applicable laws and regulations. For additional information, please visit www.clinicalstudydatarequest.com.