Physician and Patient Sources

Data for patients with MDS were provided by 264 physicians (France 48, Germany 49, Italy 50, Spain 52, UK 33, US 32) who provided information on 1445 patients. Overall, approximately half of specialists were hematologists or hematologist-oncologists, although the physicians in Germany, UK and the US were predominantly hematologist-oncologists (Table S1). Of the physicians who actively referred LR patients, 26% stated they did so to cardiologists, while other physicians commonly referred patients to hematologists (25%), oncologists (23%), family practitioners (22%) or stem cell transplant specialists (21%). Of those physicians who actively referred HR patients to other specialties, 64% frequently referred HR patients to a transplant specialist, ranging from 56% in Germany to 73% in Italy (Table S1).

Demographic CharacteristicsPhysician-Reported Patient Data

The characteristics of patients with MDS stratified into LR-MDS or HR-MDS and by geographic region are shown in Table S1, respectively. Overall, patients had a mean (SD) age of 72.8 (9.1) years, slightly lower in the US (69.1 (10.4) years); 59% were male, 93% were white and 5% Black, with a greater proportion of Black patients in the US (28%). Most patients had normal BMI (mean 25.8 [13.0]), which was slightly higher in Germany (27.2 [26.2]) and the US (26.4 [4.3]). Three quarters of patients were retired, with more in France (92%) and fewer in the US (44%) where one quarter of patients were in full- or part-time employment (Table S2). According to physicians, most patients (59% overall) needed a non-professional caregiver (Table 3), predominantly their partner or spouse (75%), but often an offspring (32%) (Table 1). The physician-reported frequency of use of a professional caregiver was generally low (7%), apart from patients in France (25%), especially for patients with HR-MDS (30%).

Table 1 Demographic characteristics of patients with MDS (physician-reported patient data)Caregiver Data

Caregiver-reported data were matched with patient data. The characteristics of caregivers are shown by disease severity in Table 2 and by geographic region in Table S3. Overall, caregivers were generally younger than the patients (mean [SD] 65 [9.7] years), 20% were in full- or part-time employment, and 85% were living with the patient, mainly their partner or spouse (83%).

Table 2 Demographic characteristics of caregivers matched with patients with MDSPhysician-Reported Clinical Characteristics

The baseline clinical characteristics of the full sample of 1445 patients with MDS stratified by disease severity and geographic region are shown in Tables 3 and S4, respectively. The majority of patients (92%) were diagnosed with primary or de novo MDS, and the stratification into low- or high-risk MDS was consistent with the IPSS-R score, with LR-MDS patients having a very low (8%) or low (43%) score compared with intermediate, high or very high scores in patients with HR-MDS (8%, 34% and 7%, respectively, Fig. 2 and Table S4). World Health Organization classification of disease stage showed that single lineage dysplasia was most commonly associated with LR-MDS and excess blasts with HR-MDS, which was consistent across all regions (Table S4). Patients with LR-MDS had consistently been diagnosed for longer than those with HR-MDS (mean [SD] 670 [766] and 345 [431] days, respectively). Overall, ECOG performance status scores indicated that approximately one half of patients (50%) were restricted in physically strenuous activity but were ambulatory and able to carry out light work (ECOG 1), while 28% were capable of all self-care but unable to carry out work activities (ECOG 2). These debilities were consistent, irrespective of whether the patient had been diagnosed with LR-MDS or HR-MDS, with similar distribution across all regions (Table S4).

Table 3 Baseline clinical characteristics of patients with MDS (physician-reported patient data)Fig. 2

IPSS-R status at the time of data collection. HR, higher risk; IPSS-R, International Prognostic Scoring System; LR, lower risk; MDS, myelodysplastic syndromes

Treatments

Most patients were receiving some form of supportive therapy (74%) at the time of data collection, with a variety of therapies being prescribed (Table 3). Erythropoietins were reported to be the most frequent overall but less frequently in HR-MDS, followed by iron chelation agents and granulocyte colony-stimulating factors. Overall, 44% of patients with MDS had received at least one line of anti-AML treatment (chemotherapy, hypomethylating agent, immunotherapy or targeted therapy) at the time of data collection (LR 20%, HR 70%), with some patients receiving as many as five lines; patients with HR-MDS were consistently more frequently treated than those with LR-MDS (Table S5).

Transfusion History

Approximately half (42%) of patients had received at least three blood transfusions (mean [SD] 4.1 [5.8]) during the 12 months prior to data collection (Table 4), most in the UK (51%) and least in the US (19%), where the mean (SD) number of transfusions in the prior 12 months was lower than in European countries (1.9 [1.8]), consistent with the generally shorter time since diagnosis (Table S6). Across all regions other than France, patients with HR-MDS had received more transfusions in the last 12 months than those with LR-MDS (Table S6). Of the 233 patients on a transfusion schedule, their involvement in the scheduling of this was recorded from the physician perspective. Irrespective of their risk status or region, in most cases (76%) there was some involvement in the schedule (Table 4), although somewhat less frequently in France and Germany (55% and 62%, respectively, Table S6). Caregivers had less involvement in the treatment decisions overall (62%) compared to the patients themselves (Tables 4 and S6).

Table 4 Transfusion history with involvement of patients and caregivers (physician-reported patient data)Requirement for Care

Over half of patients with MDS were reported by physicians to require assistance with transportation (60%), housekeeping (53%) and shopping/meal preparation (51%), while approximately one third required help with managing medications (34%) and finances (30%) (Table 5). Other care needs included assistance with personal hygiene, ambulation, dressing, feeding and self-efficacy. Caregivers reported the most troublesome activities requiring assistance to be transportation (25%), meal preparation/cooking (22%) and remembering/making appointments (19%) (Fig. 3). Caregivers reported a mean (SD) of 42 (47.4) and 49.3 (37.2) hours per week for patients with LR-MDS and HR-MDS, respectively, for care being provided across all sources (Fig. 4, Table 5). There was a slight discrepancy between the number of hours spent by caregivers among physician, patient and caregiver estimates, especially in France, with patients reporting much less care requirement (Fig. 5 and S8).

Table 5 Caregiver needs by patients with MDS and associated burdenFig. 3

Top ten most frequent symptoms reported by patients and physicians at the time of data collection. HR higher risk; LR lower risk; MDS myelodysplastic syndromes; GI gastrointestinal

Fig. 4

Caregiver-reported most troublesome activity requiring caregiver support. HR higher risk; LR lower risk; MDS myelodysplastic syndromes

Fig. 5

Caregiver hours reported by patients, caregivers and physicians (hours per week). HR higher risk; LR lower risk; MDS myelodysplastic syndromes

Patient Symptoms

Of 324 patients who completed a self-completion questionnaire and reported on their symptoms, a large majority of patients reported experiencing anemia (79%) or feeling tired and fatigued (68%), often more frequently by patients with HR-MDS than those with LR-MDS; these symptoms were generally considered to be the most burdensome symptoms of their disease (Table S7). Patient data were matched with data reported by their physician, and although the reporting of symptoms was fairly consistent, patients more frequently reported subjective symptoms such as excessive bruising (30%) and unusually pale skin (25%) compared with physicians (14% and 14%, respectively, Fig. 3).

Patient- and Caregiver-Reported Outcomes

Of 329 patients who self-reported on their quality of life, the current health status of patients with LR-MDS (n = 173) was recorded to be higher than for patients with HR-MDS (n = 153), with overall utility scores on the EQ-5D-5L of 0.86 and 0.83, respectively (Fig. 6 and Table S9). Patients with LR-MDS also reported higher scores for global health on the EORTC QLQ-C30 (indicating a better HRQoL) as well as for all other subdomains: physical, role, emotional, cognitive and social functioning (Fig. 7 and Table S9). Patients with HR-MDS had a worse score for fatigue symptoms than those with LR-MDS (36.5 and 43.2, respectively).

Fig. 6

Patient-reported EQ5D-5L utility and VAS scores at the time of data collection. EQ5D EuroQol 5 Dimension; HR higher risk; LR lower risk; MDS myelodysplastic syndromes; VAS visual analog scale

Fig. 7

Patient-reported EORTC QLQ-C30 scores at the time of data collection. EORTC QLQ-C30 European Organization for the Research and Treatment of Cancer Quality of Life Questionnaire; HR higher risk; LR lower risk; MDS myelodysplastic syndromes. Note that sample size fluctuates between each PRO value

Patients with HR-MDS also had greater impairment in activities of daily living across all geographic regions than those with LR-MDS as recorded on the WPAI (Table S9), while patients with HR-MDS scored higher (indicating greater burden) in both the anxiety and depression scores of the HADS (Fig. 8), with patients in France reporting the lowest anxiety and depression scores and Germany the highest (Table S9).

Fig. 8

Patient-reported HADS scores at the time of data collection. HADS Hospital Anxiety and Depression Scale, HR higher risk; LR lower risk; MDS, myelodysplastic syndromes

A total of 106 caregivers who reported on their own health status had lower scores on the EQ-5D-5L VAS of 60.1 in those caring for patients with HR-MDS compared with 62.9 for those with patients with LR-MDS (Fig. 9). Scores on the ZBI also indicated a higher burden for carers of HR-MDS patients than for those with LR-MDS patients (26.4 and 24.5, respectively) (Table 2).

Fig. 9

Caregiver-reported outcomes at the time of data collection (EQ5D-5L and ZBI). HR higher risk; LR lower risk; MDS myelodysplastic syndromes; EQ5D EuroQol 5 Dimension; VAS visual analog scale; ZBI, Zarit Burden Interview. Note that sample size fluctuates between each PRO value