Newly Diagnosed Patients with Polycythemia Vera and Essential Thrombocythemia Have an Impaired Quality of Life Compared to Age-Matched Control Individuals

Research Article

Ann Hematol Oncol. 2019; 6(7): 1256.

Newly Diagnosed Patients with Polycythemia Vera and Essential Thrombocythemia Have an Impaired Quality of Life Compared to Age-Matched Control Individuals

Pettersson H¹, Anderson L², Abelsson J¹, Samuelsson J³, Johansson P¹, Duncombe A4, Mesa R5, McMullin MF6 and Andréasson B¹*

¹Hematology Section, Specialist Medicine, NU Hospital Group, UK

²Centre for Public Health, Queen’s University Belfast, UK

³Department of Internal Medicine, Stockholm South Hospital, UK

4Department of Hematology, University Hospitals Southampton NHS Foundation Trust, UK

5UT Health San Antonio Cancer Center, San Antonio, USA

6Centre for Cancer Research and Cell Biology, Queen’s University Belfast, UK

*Corresponding author: Björn Andreasson, Specialist Medicine, NU Hospital Group, Uddevalla sjukhus, Fjällvägen 9, 45180 Uddevalla, UK

Received: April 08, 2019; Accepted: July 02, 2019;Published: July 09, 2019


Purpose: The Myeloproliferative Neoplasms (MPN) are rare disorders with a prevalent and often severe symptom burden. The MPN- Symptom Assessment Form (SAF) is a well-documented and validated tool for measuring symptom burden in these patients. In this study we compared , symptom burden and overall Quality of Life (QoL) measured by the MPN-SAF, in 164 patients with newly diagnosed Polycythemia Vera (PV) or Essential Thrombocythemia (ET), 119 patients with elevated hematocrit and/or platelet count (non-PV/ET) without evidence of MPN and 495 healthy, age-matched control subjects.

Methods: The MPN-SAF is a 27-item questionnaire specifically tailored to MPN patients was sent to the patients and healthy control subjects, and returned by mail. MPN-SAF Total Symptom Score (TSS) and Brief Fatigue Inventory (BFI) were calculated from the MPN-SAF questionnaire. The scores of the three groups were compared to each other using the Mann-Whitney U Test.

Results: The control group had significantly lower means for all 27 items, measured by MPN-SAF, including TSS compared to the newly diagnosed PV/ ET patients, as well as compared to the non-PV/ET group. Surprisingly, the non- PV/ET group had a more severe symptom burden than the newly diagnosed PV and ET patients with higher scores for all items except weight loss.

Conclusions: This is to date the largest set of data on how age-matched healthy subjects score in MPN-SAF, for possible reference in future studies. Newly diagnosed PV and ET patients have significantly impaired QoL compared to the general population. Also, MPN-SAF does not distinguish MPN patients from patients with elevated blood parameters not due to MPN, but these patients should be further reviewed due to their substantial symptom burden.

Keywords: Polycythemia vera; Thrombocythemia; Quality of life


MPNs: Myeloproliferative Neoplasms; PV: Polycythemia Vera; ET: Essential Thrombocythemia; MF: Myelofibrosis; QoL: Quality of Life; SAF: Symptom Assessment Form; Hct= Hematocrit; TSS: Total Symptom Score; BFI: Brief Fatigue Inventory


The Philadelphia chromosome negative Myeloproliferative Neoplasms (MPNs) are rare clonal hematologic malignancies including Polycythemia Vera (PV), Essential Thrombocythemia (ET) and Myelofibrosis (MF) [1]. These entities are heterogeneous and have differences as to morbidity and prognosis, but also share features such as risk for vascular complications and transformation into acute leukemia [2-6]. The MPNs also share a prevalent and often severe symptom burden [7-9].

In recent years, several studies with focus on symptom burden and the Quality of Life (QoL) of MPN patients have been published. It is evident that MPN patients have impaired QoL and several instruments have been used to capture this [8,10-12]. The widely used instruments in cancer, such as the European Organization of Research and Treatment of Cancer Quality of Life Questionnaire Core model (EORTC QLQ-C30) and Functional Assessment of Cancer Therapy-Anemia (FACT-AN), do not fully reflect the specific symptoms which affect QoL in MPN patients [13,14]. To adequately profile the symptoms of MPN, the MPN-Symptom Assessment Form (SAF) was developed, including 27 items. This tool has been clinically validated and has been translated to several languages, to be used in international studies. International studies using MPN-SAF have illustrated an impaired QoL and substantial symptom burden in MPN patients both at diagnosis and during the course of the disease [14]. The symptom burden seems to be more severe in patients with MF over time [14], whereas PV patients were found to worst impairment of QoL at the time of MPN diagnosis [12].

Very few comparisons between QoL in patients with MPN and the general population have been done. To our knowledge, the only study presenting data including a control group is the one by Anderson et al, [11]. Herein, the MPN-SAF scores from 106 MPN patients from the UK and 1446 from the US were compared to the scores from 124 healthy control subjects. In all 27 MPN-SAF items the MPN patients scored significantly higher compared to the controls, i.e. had an increased symptom burden.

The aim of this study was to compare QoL, using MPN-SAF, in patients with newly diagnosed PV or ET with patients referred to a hematologic clinic due to elevated Hematocrit (Hct) and/or platelet counts who had no evidence of MPN and an age adjusted group of healthy individuals.


Patients and control subjects

The control individuals were recruited among relatives of patients and hospital staff. The only inclusion criteria were age =55 years and that they considered themselves healthy. The control group comprised 401 subjects from Sweden and 94 subjects from Great Britain, 291 were female and 204 were male. The British group of healthy individuals is a subgroup of a cohort earlier described by Anderson et al, [11], i.e. those with age =55 years.

The 119 patients with elevated Hct and/or elevated platelet counts, referred to as non-PV/ET, were recruited from three Swedish centers; Stockholm South Hospital, Uddevalla Hospital and Sahlgrenska University Hospital. At the time of referral the patient had an Hct over 48% in women and over 50% in men and/or a platelet count exceeding 450 x109/L. To be included in the study the patients with elevated Hct had to have a normal serum erythropoietin and absence of JAK2 V617F mutation. The patients with thrombocytosis had to have absence of JAK2 V617F mutation and a bone marrow biopsy without MPN features when no evident cause of platelet elevation found.

The control groups and the patients with high blood values were contacted per written letter with study information, consent form and MPN-SAF questionnaires. Age and gender were recorded.

The majority of patients with newly diagnosed ET (n=80) and PV (n=73) from Sweden were recruited from six hematology clinics, and are described in detail in a previously published study of Abelsson et al, [12]. In addition, eleven ET or PV patients were included from Uddevalla Hospital. All included patients had their diagnosis established according to the 2008 World Health Organization (WHO) Guidelines [15] and completed the survey as soon as the diagnosis was established.


The MPN-SAF is a 27-item questionnaire specifically tailored to MPN patients. This instrument has been validated in 2012 and been translated to many languages. Each item was answered with a linear analog scale of 0-10, where 0 corresponded to absence of symptom and 10 to the worst possible. A high score in MPN-SAF would reflect a poor quality of life [13,14].

MPN-SAF Total Symptom Score (TSS) is a shorter form of the questionnaire which includes 10 items: fatigue, concentration problems, early satiety, inactivity, night sweats, itching, bone pain, abdominal discomfort, weight loss and fever [14].

Brief Fatigue Inventory (BFI) is a 9-item score included in MPNSAF [16]. BFI is an average score of 9 items reflecting fatigue and how it affects related activity [8].

Ethical consideration

The study was approved by the Office for Research Ethics Committee, Northern Ireland and by the Regional Ethical Board in Gothenburg, Sweden. All procedures performed in studies involving human participants were in accordance with the 1964 Helsinki declaration and its later amendments or comparable ethical standards. All patients and control subject gave their informed consent.


The mean scores and the standard deviation were calculated with standard statistical methods. The scores of the three groups were compared to each other using the Mann-Whitney U Test. A 95% confidence interval was chosen.


The group of healthy controls included 401 Swedish and 94 British individuals with a mean age of 66.6 years. The group of 119 patients with non-PV/ET had a mean age of 61 years, which did not differ significantly when compared to the controls (p=0.158). On the other hand, the group of 164 newly diagnosed PV and ET patients was significantly older than both controls and the non-PV/ET patient group, mean age of 70.1 years, (p <0.001, for both).

The score for every MPN-SAF item were similar and the means did not differ significantly in the healthy controls from Sweden compared to the British, except the item addressing sexual problems where the Swedish controls had a mean score of 2.0 compared to 1.1 for the British subjects (p<0.001).

The group of 164 newly diagnosed PV and ET patients had significantly higher means in all MPN-SAF items, p =0.005, compared to the healthy controls, including BFI and TSS. Thus, the mean score was at least double in the PV/ET group in all MPN-SAF items except BFI, insomnia, sexual problems, cough, night sweats, bone pain and overall QoL. Detailed data are shown in (Table I) and illustrated in (Figure 1).

Because of the significant difference in mean age between the healthy controls and the PV/ET patients we also compared the MPNSAF scores for the control subjects with age >62 years. This group comprised 332 subjects and had a mean age comparable to the patients, 70.3±6.0 years. When this age-matched group was compared to the patients with newly diagnosed MPN all the highly significant differences (p =0.005) remained, except for the items addressing early satiety and sexual problems (p =0.025 and 0.053, respectively).

The MPN-SAF scores from the 119 non-PV/ET patients were compared with the scores from the healthy control group. In all items the control group had mean scores that were highly significantly lower (p<0.001, in all items). In all items in the questionnaire the non-PV/ET patients had at least a double mean value compared to the means of the controls, except for the questions about insomnia and sexual problems.

In spite of the fact that the group of patients with newly diagnosed PV/ET had a significantly higher mean age their mean MPN-SAF scores were lower for all items, except weight loss, when compared to the means for the non-PV/ET patients. Statistical significance was reached for BFI, early satiety, abdominal pain and discomfort, inactivity, numbness, night sweats, overall QoL and TSS. All mean scores and significance levels are shown in Table 1 and illustrated in (Figure 2).