Myelodysplastic Neoplasms Patients' and Caregivers' Unmet Needs and Their Impact on Their Quality of Life and Symptoms

Background and aims. Myelodysplastic neoplasms (MDS) prevail in the elderly and are generally incurable. Thus, maintaining/improving quality of life (QoL) is one of the main goals of treatment. There are many factors related to MDS and unmet needs that may impact QoL of patients and their caregivers. The aim of this study was therefore to explore the impact of MDS on patients and caregivers, their preferences and unmet needs in Italy. Methods. The survey (online and paper versions) was promoted by the Associazione Italiana Pazienti Sindromi Mielodisplastica (AIPaSiM) advocacy group. Sixty items relevant to patients and 20 relevant to family caregivers were identified by hematologists and patients. The patient-reported outcome measures (PROMs), QOL-E and HM-PRO, were included in the survey. Lower scores in QOL-E and higher scores in HM-PRO represent worse outcomes. Results. From June 2022 to May 2023, 259 patients of median age 73 (range 27-98) years, 56% female and 71 family caregivers (80% females and 94% family members) completed the survey. MDS duration was over 5 years in 28% of cases. Seventy percent of patients were receiving treatment: 64% for lower risk and 36% for higher risk MDS. Only 15% of patients felt that treatment impacted negatively on everyday life. Seventy-seven percent of patients were retired. MDS affected work life of 20% of patients, leading to changes in job type or working hours or to retirement/activity stop. Distance from the treating center was far for 20% and 48% needed a caregiver to accompany them to hospital. For 44%, travelling to hospital was claimed distressful in terms of physical and economic burden and impacted on family members and work. Comparing PROMs of these patients to the remaining 56%, distress was associated with significantly worse median HM-PRO physical (PB, 50 vs 21, p<0.001), emotional (EB, 54 vs 32, p<0.001), social (SB, 33 vs 0, p=0.001), eating and drinking habits (ED, 34 vs 19, p<0.001), Part A (39 vs 15, p<0.001) and Part B symptom score (SS, 20 vs 9, p<0.001) and QOL-E physical (Ph, 50 vs 62, p<0.001), functional (Fun, 22 vs 89, p<0.001), social (Soc, 25 vs 75, p<0.001), fatigue (Fat, 64 vs 81, p<0.001), general (Gen, 47 vs 72, p=0.003), MDS specific (MDSS, 44 vs 74, p<0.001) and treatment-outcome index scores (TOI, 38 vs 70, p<0.001). Forty-two percent of patients received regular red blood cell transfusions. The increasing frequency of transfusions (<2/4 units/month, 34%); 2-4 units/month, 53%; >4 units, 13%) was associated with worse HM-PB (p=0.042), SB (p=0.038) and part A scores (p=0.029) and QOL-E Soc (p=0.012), Fat (p= 0.034), MDSS (p=0.002) and Gen scores (p=0.049). Only 2% received transfusions at home; however, 38% would prefer being transfused at home, 77% of which experienced distress reaching the hospital, even though only 20% were far from the center of care. Such patients, in fact, had worse PROMs. Waiting time in hospital was unacceptable for 40% of patients and was associated with worse HM-PRO PB (p=0.014), EB (p<0.001), SB (p=0.025), Part A (p<0.001) and SS scores (p=0.002), and QOL-E Fun (p=0.004), Soc (p=0.012), Fat (p=0.001), MDSS (p=0.001), Gen (p=0.003) and TOI scores (p=0.003). Telemedicine communication was preferred in 54% of cases, particularly if reaching hospital was distressful (62% yes vs 45% no, p=0.035) Other unmet needs were comprehension of diagnosis for 15% and treatment options for 21%, and access to new drugs for 35%. Thirty-six percent of patients had a family caregiver of median age 56 (28-83) years and 16% of patients felt to be a burden for their family. The caregiver's work was affected in 34% leading to a job change in 29% of them. On a scale 0-10 on the impact on work-life, family caregivers scored median 4 (interquartile range 2-6). Conclusions This Italian study has highlighted the issues related to patients with MDS and their caregivers. More time should be dedicated to the communication of the diagnosis, prognosis and treatment options. Furthermore, for both patients and caregivers, there is an economic burden and distress in attending and waiting in hospital. For patients, transfusion frequency is correlated with worse QoL. These data highlight the need of treatments that reduce transfusion burden, hospital visits and allow patients to be treated at home. Identifying patients' and caregivers' unmet needs and their impact on QoL and symptoms can provide optimized patient care and guide hematologists and governance choices.