Myeloproliferative neoplasm questionnaire: assessing patient disease knowledge in the modern digital information era

Abstract

There is growing internet and social media use among patients with rare blood cancers, notably myeloproliferative neoplasms (MPNs). A 38-item online questionnaire was developed to assess patients’ (n=983) disease understanding and use of online resources regarding MPN. Many responders (74%) reported unawareness of additional mutations beyond their primary molecular marker(s); 32% were unsure of their prognostic risk stratification. Additionally, 89% reported using online resources (Facebook (61%); Google/Google+ (42%); YouTube (34%); blogs (26%); Twitter (5%)) to seek information about MPN. Despite this, results showed many gaps in patients’ basic disease knowledge. Our findings suggest an important difference in social media habits between physicians and patients: physicians are rapidly adopting Twitter as their preferred medium for sharing medical knowledge; however, patients often prefer other social mediums. Educational campaigns should be designed in more personalized ways, aiming to fit a variety of online platforms to maximize reach and impact for patients with MPN.

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Introduction

Myeloproliferative neoplasms (MPN) are a group of chronic myeloid disorders consisting of three classical Philadelphia-chromosome-negative (or BCR-ABL-1-negative) MPNs including polycythemia vera (PV), essential thrombocythemia (ET), and primary myelofibrosis (PMF)¹. Patients with these MPNs are often found to have the presence of either one of the three driver mutations (in Janus kinase 2 (JAK2), calreticulin (CALR), or myeloproliferative leukemia virus oncogene (MPL)^2–4). These MPNs are considered to be rare hematologic neoplasms with heterogeneous clinical presentations, usually with terminal myeloid cell expansion in peripheral blood¹. Notable complications include hemorrhage, thromboses, and clonal evolution with transformation to acute myeloid leukemia (AML)^4,5. Therapy in PV and ET is often low-dose aspirin plus phlebotomy for thrombosis prevention; hydroxyurea and interferon-alfa are used for cytoreduction in high-risk patients. For patients with PMF, stem cell transplantation remains the only curative approach and pharmacologic JAK-inhibition is the only approved targeted therapy strategy widely available at this time^3,6.

As a field, it is vital to continue to highlight together, among all healthcare stakeholders, importance of focusing on patients with rare diseases and rare cancers. An emerging area of focus is the growing use of the internet and social media sites among patients with rare blood cancers. The internet enables patients with rare cancers to have access to information, increase understanding of their disease, and garner support from a community. All of these benefits can improve the lives of patients from the comfort of their own environment, without being restricted to a physical clinic space.

A common use of internet resources among patients and physicians is the use of social media websites. Healthcare providers have rapidly adopted the use of Twitter for information gathering and content creation, specifically with creation of disease-specific hashtag (#) medical communities. In the field of rare hematologic malignancies, the use of #BPDCN (blastic plasmacytoid dendritic cell neoplasm)^7,8 and #MPNSM (myeloproliferative neoplasms on social media)^9,10 has led to rapid dissemination of accessible information and garnering a community of experts to discuss conversations relevant to this rare cancer fields. It is exciting to see the growth of information sharing among physicians and patients on social media, especially in rare blood cancers. Though these Twitter communities have been paramount for connection and information sharing among hematology/oncology professionals, little is known about how patients prefer and frequent the use of Twitter for their own disease-specific education.

Administration of internet-based patient reported outcomes has previously demonstrated high participation rates and regular collection of questionnaires that report on symptom assessment and quality of life¹¹. Little is known about MPN patients’ understanding of their own disease, however, and their use of online and social media resources to gain more information. Addressing this important gap could help us to facilitate improved access to accurate informational resources about rare, complex cancers, such as MPN. Our primary aim of this study was to evaluate knowledge and awareness of MPN in a sample of MPN patents in the Patient Power online information and support community.

Materials and Methods

This research was approved by the University of Texas MD Anderson Cancer Center Institutional Review Board. We developed a 38-item online questionnaire that assessed MPN knowledge and awareness, demographic and clinical characteristics, and use of online resources among patients with self-reported MPN diagnosis. The study population included patients who participated in the Patient Power online community and who self-identified with MPN diagnosis. Responder information was kept completely confidential and no patient-identifying information was obtained. It is estimated that N=4,314 subscribers (no charge/free to users) self-identify as patients or caregivers with MPNs. Patient Power distributed the study questionnaire to its subscribers using an online survey platform, with an invitation to complete it if one self-identified as having an MPN diagnosis. Feasibility of administering the questionnaire was determined in a pilot sample of n=20 responders (design phase), which was subsequently allowed to continue to enroll more responders through an expansion phase.

Assessment of self-reported patient knowledge of disease was derived from responders’ answers to the following questions asked in the survey:

1. What type of MPN do you have?

2. Do you know any of your genetic/molecular markers?

3. Besides JAK2, CALR, MPL, or triple negative (meaning negative for all 3 mutations), do you know if you have any additional molecular mutations?

4. At diagnosis, did your doctor describe your level of disease risk?

5. Do you know your chromosomes/baseline karyotype? (Examples include: diploid, +8, −7/7q-, I [17q], −5/5q-, 12p-, inv[3], 11q23)

Results

Between March to November 2019, patients (n=983) completed the questionnaire. Age range of patients were 20 to 90 years old (mean 59.9 years), 74% of responders were female, and 93% were Caucasian. The majority (64%) of patients reside in the United States of America (USA); 36% of non-USA respondents lived in the United Kingdom, Canada, Australia, and several countries in Europe, South America, Asia, and Africa. 52% reported receiving their care at a major cancer center. Patient demographics, comorbidities and presenting symptoms at time of MPN diagnosis are reported in Table 1.

Table 1.

Demographics and Characteristics of Respondents

Variable	Total n=983 (%)	Variable	Total n=983 (%)
Age (years) mean  (min, max)	59.9 (20, 90)	Age of diagnosis
Gender		21 – 30 years	45 (5)
Female	727 (74)	31 – 40 years	115 (12)
Male	256 (26)	41 – 50 years	206 (21)
Highest level of education achieved		51 – 60 years	277 (29)
Some high school, no diploma	17 (2)	61 – 70 years	254 (26)
High school graduate, diploma, or equivalent	98 (10)	71 – 80 years	58 (6)
Some college credit, no degree	175 (18)	Over 80 years	7 (1)
Trade/technical/vocational training	51 (5)	Risk level described by physician for MPN
Associate degree	58 (6)	High risk	202 (21)
Bachelor’s degree	243 (25)	Intermediate risk	195 (20)
Master’s degree	196 (20)	Low risk	250 (26)
Professional degree	63 (6)	Doctor did not describe risk level / unsure	315 (33)
Doctorate degree	58 (6)	Co-existing cardiovascular risk factors
Other / No response	23 (2)	Diabetes mellitus	42 (5)
Country of Residence		Hypertension	283 (30)
United States of America	634 (64)	Venous thromboembolism	190 (20)
Other	349 (36)	Previous myocardial infarction	32 (3)
Race/Ethnicity		Previous cerebrovascular accident	103 (11)
Caucasian	909 (93)	Other	142 (15)
African-American	9 (1)	Tobacco smoking	48 (5)
Asian	14 (1)	History of other malignancy
Latino/of Hispanic Origin	17 (2)	Solid cancer	144 (15)
Indian	3 (0)	Hematologic cancer	31 (3)
Other	30 (3)	Event leading to diagnosis
Location for majority of disease treatment		Abnormal result from routine blood test	701 (74)
A major cancer center	508 (52)	Abdominal pain / discomfort	101 (11)
A local doctor’s office	286 (29)	Bone pain	81 (9)
Other	189 (19)	Venous thromboembolism	53 (6)
Type of diagnosis		Fatigue	265 (28)
Polycythemia Vera	395 (41)	Early satiety	56 (6)
Essential Thrombocythemia	319 (33)	Headache	185 (20)
Myelofibrosis	210 (22)	Pruritis	135 (14)
Other / Multiple	29 (3)	Inability to concentrate	95 (10)
Do not know / unsure	9 (1)	Major bleeding event	31 (3)
Genetic / Molecular Markers		Night sweats	128 (14)
Janus Kinase 2 (JAK2)	716 (74)	Stroke	51 (5)
Calretinin (CALR)	118 (12)	Unexplained fevers	27 (3)
Myeloproliferative leukemia virus oncogene (MPL)	26 (3)	Unintentional weight loss	47 (5)
Triple Negative	47 (5)
Do not know / unsure	73 (8)

At time of MPN diagnosis, 55% were between age 51–70 years; 70% of patients had a diagnostic bone marrow biopsy in addition to diagnostic blood tests. MPN subtypes were: PV (41%), ET (33%), MF (22%), other/write-in/don’t know (4%). Molecular subtypes included: JAK2 (74%), CALR (12%), MPL (3%), triple-negative (5%) (Table 1). A high percentage (74%) reported not being aware of additional mutations (ASXL1, TET2, TP53, SRSF2, EZH2, DNMT3a, IDH1 or IDH2, etc.), and 96% of patients reported that they did not know their chromosomes or baseline karyotype. Prognostic stratification included high risk (21%), intermediate risk (20%), low risk (26%); 32% said their physician did not provide their risk stratification/didn’t know (Table 1). In terms of family history, 13% reported one or more affected family members with MPN, and 25% reported one or more family members with other blood cancers/disorders (including lymphoma, leukemia, myeloma) other than MPNs. Patients reported a personal history of diagnosis of other hematologic cancer (3%) or various solid malignancies (15%).

Patients reported that they were managed with a variety of therapies (Table 2). Of patients surveyed, 64% reported an improvement in symptoms with therapy, most commonly attributed to hydroxyurea (53%), low-dose aspirin (32%), phlebotomy (32%), and ruxolitinib (28%) (Table 2). Out of 234 responders to a specific question regarding stem cell transplantation, 6% of responders underwent transplant, and it is still a consideration in 18%. Of the 10% of patients who had participated in a clinical trial for their MPN, the three most common ways responders learned about clinical trials were from their physician, from a conference/meeting/organized MPN event, or from an online platform/social media.

Table 2.

Treatment and subjective responses

Variable	Total n=variable responses (%)
Treatment
Anagrelide	167 of 716 (23)
Hydroxyurea	636 of 877 (73)
Hypomethylator agent (i.e. decitabine or azacytidine)	16 of 678 (2)
Interferon	94 of 683 (14)
Ruxolitinib	239 of 742 (32)
Low-dose aspirin	795 of 899 (88)
Pegylated Interferon	157 of 717 (22)
Phlebotomy	441 of 788 (56)
Stem Cell Transplantation	19 of 671 (3)
Corticosteroids	56 of 681 (8)
Thalidomide, lenalidomide, or other “IMiD” drug	11 of 671 (1)
Clinical trial therapy / combination therapies	67 of 688 (10)
After starting treatment for MPN, did symptoms improve?	(n=942 responders)
Yes	599 (64)
No	211 (22)
Did not have symptoms / did not have treatment	132 (14)
Which treatments improved symptoms?
Anagrelide	26 (4)
Hydroxyurea	316 (53)
Hypomethylator agent (i.e. decitabine or azacytidine)	4 (1)
Interferon	23 (4)
Ruxolitinib	166 (28)
Low-dose aspirin	193 (32)
Pegylated Interferon	72 (12)
Phlebotomy	192 (32)
Stem Cell Transplantation	13 (2)
Corticosteroids	15 (3)
Thalidomide, lenalidomide, or other “IMiD” drug	5 (1)
Clinical trial therapy / combination therapies	23 (4)
Other	50 (8)

The survey group frequently engaged in online research, as 89% reported that using internet/online resources allowed them to look up information about MPN therapies prior to or in between doctor visits. Timing for use of online resources by MPN patients included day of doctor visits (1%), week before doctor visits (3%), and ongoing use of online resources (95%) in the overwhelming majority of responders. Responders indicated that online support resources allowed them to look up information about MPN or therapies (86%), see laboratory data (43%), connect with other people with a shared condition (72%), prepare for visits and make care decisions (57%), find out about clinical trials (28%), and improves patient-provider communication (44%) (Figure 1). Among the most commonly used online mediums were: Facebook (61%), Google/Google+ (42%), YouTube (34%), blogs (26%), Twitter (5%) (Figure 2). 48% of patients reported interest in their local doctor’s office facilitating more of MPN patient care through online/internet-based activity (i.e., communication with provider, looking up own medical records, etc.). Responders indicated that interaction with others online specifically about MPN has benefited them in several ways through improved information sharing, connection, sense of community, access to experts/research, and improved quality of life (Figure 3).

Figure 1. Impact of online support resources on overall MPN care.

Patients responses to the question regarding how online support resources have impacted overall care for their myeloproliferative neoplasm (MPN).

Figure 2. Patient use of social media networks to research cancer care.

Patients responses to the question regarding which social media networks they have used to research their own cancer care.

Figure 3. How interacting with others online specifically about MPN has benefitted patients.

Patients responses to the question regarding perceptions and benefit from interacting with others online specifically about their myeloproliferative neoplasm (MPN).

When asked if survey responders would be willing to participate in a de-identified MPN patient registry/central database for clinical research, 93% responded “yes.”

Discussion

An increased awareness of patients’ baseline knowledge of disease and treatment preferences may help with symptom management and improved quality of life. The chronic myeloid leukemia (CML) literature has shown that treatment non-adherence is common in CML patients, and demonstrated a strong correlation between adherence and improved response to treatment^12,13. A subsequent worldwide patient survey of patterns of behavior and motivation found that support from hematologists providing better information regarding disease, therapy, and side effect management is paramount to improve patient adherence¹⁴. Moreover, a survey of patients with CML was able to accurately characterize patient perspectives reporting treatment satisfaction and difficulties, adverse medication effects, and other important factors related to adherence and quality of life¹⁵. Interestingly, patients and healthcare professionals agreed that the most important issues impacting quality of life are fatigue, swelling, muscle cramps, social support for patients with disease, and uncertainty about future health conditions¹⁶. These studies are important and help maintain an open line of communication between patients and healthcare providers regarding patient knowledge, experience, and satisfaction with their disease and its management.

Surveys of patients with MPN have reported a decreased health related quality of life, despite adhering to a healthier lifestyle than the general population^17–19. Recently, an international survey of patients with MPN found that 90% of patients with MPN experienced symptoms that reduced quality of life, most often fatigue¹⁸. Several surveys have revealed a shared aspect of patients reporting a similar degree of high symptom burden among all MPN subtypes, with fatigue being the most common and the most burdensome^17,18,20. Similarly, in our study, fatigue was the most common presenting symptom, along with headache, abdominal pain, pruritis, and night sweats. Interestingly, recent studies showed the impact of depression and abnormal body mass index (both overweight and underweight) confounding the symptom of fatigue in patients with MPN^21,22. Both of these are important factors to consider as potential modifiable etiologies for the symptom of fatigue in these patients.

More than two thirds of our patients with symptoms did report an improvement with certain treatments they received. It is interesting to note which specific therapies patients felt improved their symptoms, most commonly hydroxyurea, low-dose aspirin, phlebotomy, and ruxolitinib (Table 2). In prior studies, patient perception of treatment success was derived from blood counts and feedback from their physicians, suggesting a baseline patient understanding of the disease. However, there was often a discrepancy between physician and patient treatment goals, resulting in a need for improved communication and agreement in goals¹⁸. Moreover, a recent study demonstrated discordance between control in blood counts and degree of symptom burden in patients with PV²³. This issue is especially prudent in the era of the COVID-19 pandemic, which has shifted clinical care for many patients and increased the use of telemedicine^24–26.

Integrative medicine strategies such as massage, aerobic activity, yoga, breathing techniques, strength training, and support groups have been employed for patients to help reduce MPN symptom burden, fatigue, and depression^27,28. There were varying responses to each modality in terms of reduced fatigue, depression, and improved quality of life; suggesting areas for future research. It is important for physicians to be aware of the various factors that impact their patients’ symptom burden, and all of the potential resources they can recommend in addition to disease-treatment to help improve patient quality of life and symptoms.

In terms of response, it was interesting to note that female patients comprised nearly three quarters of responders. In other recent surveys of MPN patients, female responders also represented a majority (56–63%)^17,18,20. Women may be more likely to be recruited to surveys via Facebook²⁹, and previous academic studies using web-based surveys have shown more frequent responses in those with older age, higher education level, female gender, and overall higher health status^17,29,30. Another finding is that ET, the most common subtype of MPN, only represented one-third of our responders. However, a relatively similar percentage have been represented in recent surveys of MPN patients in which patients with ET made up 28–43% of responders^17,18,20.

While our MPN patient sample cohort reported actively using online resources to seek information about their disease and treatment, results showed many gaps in basic knowledge about MPN. Based on this information, innovative proposals can be put forward to augment the patient experience and understanding of their MPN with more online educational tools, handouts/information packets in physician offices, improved approaches to educate physicians and their patients about basics of MPN diagnosis, staging, and basic and advanced molecular mutational assessments. Interestingly, some patients suggested and requested that doctors’ offices would provide referrals to online social media support groups for patients, and provide links and references for the most up-to-date research on MPN. Additionally, our findings suggest an important difference in online and social media habits of physicians compared to patients with regards to medical information and dissemination: physicians and investigators are rapidly adopting Twitter as their preferred medium for sharing medical knowledge³¹; however, patients may prefer other mediums such as Facebook, Google, or YouTube (Figure 2). This finding suggests that MPN educational campaigns should be designed in more personalized ways, in order to aim to fit a variety of online platforms to maximize reach and impact for patients with MPN.

Social media has been a great advent for disseminating general advice to a public audience and for sharing experiences among patients and healthcare providers. However, it cannot replace arguably the most important factor for specific individualized medical recommendations, which must come from the patient-doctor relationship. This mutual trust-based connection between physician and patient is paramount for promoting healing, recovery, and adherence to treatment^32,33. Further, while there are many positive aspects of readily accessing the internet, there is often a plethora of unscientific, outdated, or biased information that may mislead and misinform patients³⁴. Despite the ever-changing technological landscape and amongst the era of the worldwide novel coronavirus pandemic which has changed the way doctors and patients interact and collaborate, empathy, respect, and honest communication remain vital to the patient-doctor relationship^35,36. A personalized relationship between doctor and patient will always be of utmost importance in educating and caring for patients, even in the digital era.

Limitations to our study include the descriptive, self-reporting nature of patient responses for analysis. Further, online administration of this survey perhaps may have excluded a certain subset of MPN patients who may not have financial means or educational level to access and understand an online survey format. Overall, this study identified knowledge gaps in the MPN-patient community, and areas for improvement by physicians to personalize patient education regarding basics of MPN diagnosis, staging, and basic and advanced molecular mutational assessments. Future studies are needed to evaluate the impact of physicians offering specific online resources and social media referrals to patients on improvement in patient-physician communication, and MPN-patient knowledge and awareness of their disease and its management.

Data availability statement:

The data that support the findings of this study are available from the corresponding author, NP, upon reasonable request.