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Background Adherence to long-term chelation therapy in transfusion-dependent patients is critical to prevent iron overload-related complications. Once-daily deferasirox dispersible tablets (DT) have proven long-term efficacy and safety in patients ≥2 years old with chronic transfusional iron overload. However, barriers to optimal adherence remain, including palatability, preparation time, and requirements for fasting state. A new film-coated tablet (FCT) formulation was developed, swallowed once daily (whole/crushed) with/without a light meal. Methods The open-label, Phase II ECLIPSE study evaluated patient-reported outcomes (PROs) in transfusion-dependent thalassemia or lower-risk myelodysplastic syndromes patients randomized 1:1 to receive deferasirox DT or FCT over 24 weeks as a secondary outcome of the study. Three PRO questionnaires were developed to evaluate both deferasirox formulations: 1) Modified Satisfaction with Iron Chelation Therapy Questionnaire; 2) Palatability Questionnaire; 3) Gastrointestinal (GI) Symptom Diary. Results One hundred seventy three patients were enrolled; 87 received the FCT and 86 the DT formulation. FCT recipients consistently reported better adherence (easier to take medication, less bothered by time to prepare medication and waiting time before eating), greater satisfaction/preference (general satisfaction and with administration of medicine), and fewer concerns (less worry about not swallowing enough medication, fewer limitations in daily activities, less concern about side effects). FCT recipients reported no taste or aftertaste and could swallow all their medicine with an acceptable amount of liquid. GI summary scores were low for both formulations. Conclusions These findings suggest a preference in favor of the deferasirox FCT formulation regardless of underlying disease or age group. Better patient satisfaction and adherence to chelation therapy may reduce iron overload-related complications. Trial registration ClinicalTrials.gov identifier: NCT02125877; registered April 26, 2014.

Myelodysplastic syndromes (MDS) are clonal stem cell disorders managed by risk stratification: lower-risk disease receives erythropoiesis-stimulating agents; higher-risk disease receives azacitidine. Red blood cell (RBC) transfusions manage symptomatic anaemia and improve quality of life (QoL) but carry risks of iron overload and alloimmunisation. No standardised transfusion strategy exists, requiring systematic evidence synthesis comparing liberal versus restrictive haemoglobin (Hb) thresholds for their effects on quality of life and transfusion-related complications. We performed a systematic review evaluating liberal versus restrictive RBC transfusion thresholds in adults with MDS not undergoing curative treatment such as stem cell transplantation. Primary outcome was health-related QoL measured by validated instruments. Secondary outcomes included mortality, transfusion reaction, iron overload, RBC utilisation and rise in ferritin level. Of 4,295 records screened, 212 articles underwent full-text review, with three RCTs meeting inclusion criteria. Liberal transfusion strategies were associated with improved QoL compared to restrictive approaches, with pooled standardised mean difference (Hedges g) of 0.54 (95% CI 0.06–1.02; p  = 0.33; I²=9.7%). Subgroup analysis of EQ‑5D outcomes across studies revealed minimal but statistically significant difference between strategies (pooled mean difference 0.084; 95% CI: 0.033 to 0.134). Mortality (hazard ratio 0.913; 95% CI 0.167–4.98) and transfusion reactions (risk difference − 0.01; 95% CI-0.10 to 0.09) did not differ. Notably, liberal thresholds required average of 4 additional RBC units per patient (95% CI 1.43–6.79), raising concerns about iron overload (rise in ferritin; mean difference 868 µg/L; 95% CI 482–1255). Limited evidence suggests that liberal transfusion strategies in MDS may improve short-term QoL but at the cost of increased transfusion burden, with no clear impact on survival or safety. A patient-centred, multidisciplinary approach remains essential when tailoring transfusion thresholds. The study protocol has been registered in PROSPERO (CRD420251085221).  https://www.crd.york.ac.uk/PROSPERO/view/CRD420251085221 .

Decitabine, including its new oral formulation (decitabine-cedazuridine, DEC-C), is commonly used in AML and MDS, particularly in older or unfit patients. While its clinical efficacy and tolerability are well documented, evidence regarding patient-reported outcomes (PROs) and health-related quality of life (HRQoL) remains limited. We conducted a review of the available literature on PROs in patients with AML and MDS treated with decitabine, with the aim of evaluating its impact on HRQoL, symptom burden, and patient preferences. A systematic literature search of PubMed up to October 2024 identified studies evaluating HRQoL or PROs in adult AML and/or MDS patients receiving decitabine, regardless of study design. Ten studies met the inclusion critera. Decitabine-based regimens were associated with preservation of HRQoL compared with intensive chemotherapy and improvements in fatigue and physical functioning versus best supportive care. In patients with AML, baseline HRQoL scores were found to be predictive of survival outcomes. Surveys consistently indicated strong patient preference for oral DEC-C due to reduced treatment burden and greater convenience, though longitudinal data remain limited. In conclusion, currently available HRQoL evidence for decitabine provides meaningful insight to guide further research. Findings from patient surveys and the availability of decitabine in both intravenous and oral formulations emphasize new treatment aspects that can be effectively captured through PROs. Their systematic integration may help uncover critical issues such as symptom burden, adherence, and patient priorities, ultimately fostering more patient-centered care.

In myelodysplastic syndromes (MDS), health-related quality of life (HRQoL) represents a relevant patient-reported outcome, which is essential in individualized therapy planning. Prospective data on HRQoL in lower-risk MDS remain rare. We assessed HRQOL by EQ-5D questionnaire at initial diagnosis in 1690 consecutive IPSS-Low/Int-1 MDS patients from the European LeukemiaNet Registry. Impairments were compared with age- and sex-matched EuroQol Group norms. A significant proportion of MDS patients reported moderate/severe problems in the dimensions pain/discomfort (49.5%), mobility (41.0%), anxiety/depression (37.9%), and usual activities (36.1%). Limitations in mobility, self-care, usual activities, pain/discomfort, and EQ-VAS were significantly more frequent in the old, in females, and in those with high co-morbidity burden, low haemoglobin levels, or red blood cells transfusion need (p < 0.001). In comparison to age- and sex-matched peers, the proportion of problems in usual activities and anxiety/depression was significantly higher in MDS patients (p < 0.001). MDS-related restrictions in the dimension mobility were most prominent in males, and in older people (p < 0.001); in anxiety/depression in females and in younger people (p < 0.001); and in EQ-VAS in women and in persons older than 75 years (p < 0.05). Patients newly diagnosed with IPSS lower-risk MDS experience a pronounced reduction in HRQoL and a clustering of restrictions in distinct dimensions of HRQoL as compared with reference populations.

Social media platforms offer valuable insights into patients' experience, revealing organic conversations that reflect their immediate concerns and needs. Through active listening to lived experiences, we can identify unmet needs and discover the real-world challenges that patients and caregivers face. The aim of our study is to develop a reusable framework to collect and analyze evolving social media data, capturing insights into the experiences of individuals with myelodysplastic syndromes (MDS) and higher-risk MDS and their caregivers. The findings can inform the development of appropriate patient support interventions. We conducted a structured Google search of English-language websites relevant to MDS from January 1, 2008, to December 31, 2022, using validated URLs and keywords. Data were sourced from MDS-specific platforms to ensure clinical relevance. Contextual embeddings (rather than simple keyword matching) were applied to detect semantically meaningful mentions of \"MDS.\" Scraping algorithms collected, cleaned, and standardized the data. Posts were classified as originating from patients or caregivers using decision-tree tagging based on contextual summaries. Users were categorized as HR-MDS based on explicit mentions of \"high-risk\" or by referencing criteria aligned with National Comprehensive Cancer Network guidelines (eg, blast count, transplant, chemotherapy use). Each post was analyzed for major themes and sentiment using a supervised machine learning classifier, while latent topics were identified through a semisupervised model. We analyzed ~5.5 million words from 42,000 posts across 5500 threads by ~4000 users from the United States, United Kingdom, and Canada. Of the 1249 HR-MDS users identified, 587 (47%) were patients and 662 (53%) were caregivers. Dominant sentiments among HR-MDS users included concern (n=974, 78%), anxiety (n=749, 60%), frustration (n=724, 58%), fear (n=724, 58%), and confusion (n=612, 49%). Concern was the top sentiment among caregivers (n=390, 59%), while anxiety led among patients (n=323, 55%). Key topics included blood counts (n=674, 54%), disease burden (n=537, 43%), quality of life (n=450, 36%), treatment options (n=387, 31%), and disease progression (n=387, 31%). Anxiety was frequently tied to health (n=600, 48%), treatment (n=325, 26%), and the diagnostic process (n=250, 20%). Fear stemmed from complications (n=237, 19%) and progression (n=240, 19%). Confusion about diagnosis and disease understanding was reported by 300 (24%). Information-seeking behaviors revealed user interest in treatment interventions (n=238, 19%) and ongoing research (n=212, 17%). The application of sophisticated natural language processing techniques demonstrates promise in effectively identifying the emerging complex themes and sentiments experienced by HR-MDS users, thereby highlighting the unmet needs, barriers, and facilitators associated with the disease.

Background/Objectives: Myelodysplastic syndromes (MDS) are clonal hematopoietic disorders characterized by ineffective hematopoiesis and a risk of progression to acute myeloid leukemia (AML). Cognitive impairments, including deficits in memory, attention, and executive function, are frequently reported in MDS patients. These impairments are linked to systemic inflammation, neurotoxic treatment effects, and the psychological burden of chronic disease. This review synthesizes existing evidence on cognitive dysfunction in MDS, highlighting knowledge gaps and opportunities for future research. Methods: A scoping review was conducted following PRISMA-ScR guidelines. MEDLINE and Scopus databases were searched for studies examining cognition in MDS patients, using terms like “myelodysplastic syndromes” and “cognition.” Inclusion criteria were original, English-language studies reporting cognitive outcomes in MDS. Reviews, animal studies, and abstracts were excluded. Data on study design, patient characteristics, cognitive tests, and outcomes were extracted and descriptively analyzed. Results: The review included 25 studies involving 2390 patients with hematologic malignancies, 493 of whom had MDS. Key findings identified cognitive deficits primarily in attention, executive function, and memory. Systemic inflammation and treatment-related neurotoxicity were significant contributors, with older age compounding these effects. Longitudinal studies demonstrated persistent cognitive challenges post-treatment, though the severity varied by patient demographics and therapeutic regimens. Conclusions: Cognitive impairments in MDS are multifactorial and significantly impact patients’ quality of life. Current research highlights the need for routine cognitive assessments and targeted interventions. Future studies should focus on longitudinal designs to explore specific cognitive domains and develop therapeutic strategies to mitigate cognitive decline.

Older patients with acute myeloid leukemia (AML) and advanced myelodysplastic syndrome (aMDS) must decide between receiving intensive induction chemotherapy (IC) or nonintensive chemotherapy/best supportive care (NIC). Little information exists about what factors influence treatment decisions and what quality of life (QOL) is associated with treatment choices. We prospectively examined 43 patients 60 years or older who were interviewed at diagnosis and periodically over 1 year. IC choice was associated with younger age (66 vs 76 years, P =0.01) and AML diagnosis, but not with performance status, comorbidities, or QOL. In total, 63% of all patients reported not being offered other treatment options despite physician documentation of alternatives. Patient and physician estimates of cure differed significantly: 74% of patients estimated their chance of cure to be 50% or greater, yet for 89% of patients physician estimates of cure were 10% or less. IC patients experienced decreased QOL at 2 weeks, but rebounded to baseline and to NIC levels by 6 weeks. Initial QOL is not associated with treatment choice in older AML and aMDS patients. Regardless of treatment choice, patients report not being offered treatment options and overestimate their chances of cure. In IC patients, QOL decreases during hospitalization but rebounds after discharge.

The primary objective of this study was to evaluate the health-related quality of life (HRQOL) in lower-risk, transfusion-dependent patients with myelodysplastic syndromes (MDS) treated with deferasirox. A secondary objective was to investigate the relationship between HRQOL, serum ferritin levels and transfusion dependency. This was a prospective multicentre study enrolling 159 patients, of whom 152 received at least one dose of deferasirox. HRQOL was assessed with the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30) at baseline and then at 3, 6, 9 and 12 months. Primary analysis was performed estimating mean HRQOL scores over time by a linear mixed model on selected scales. The median age of treated patients was 72 years (range 24-87 years). No statistically significant changes over time were found in mean scores for global health status/quality of life (p=0.564), physical functioning (p=0.409) and fatigue (p=0.471) scales. Also, no significant changes were found for constipation (p=0.292), diarrhoea (p=0.815) and nausea and vomiting (p=0.643). Serum ferritin levels were not associated with HRQOL outcomes. A higher patient-reported baseline pain severity was an independent predictive factor of an earlier achievement of transfusion independence with a HR of 1.032 (99% CI 1.004 to 1.060; p=0.003). HRQOL of transfusion-dependent patients with MDS receiving deferasirox therapy remains stable over time. HRQOL assessment might also provide important predictive information on treatment outcomes. NCT00469560.

To explore the use of routine telephone follow-up as a supportive care strategy for patients with hematologic malignancies from the patients' perspectives. A qualitative design based on a series of open-ended interviews and one focus group. Leukaemia Foundation of Queensland, Australia. 50 participants recruited from the Leukaemia Foundation of Queensland database that represented a sample of major diagnostic groups, age, gender, and geographic location of patients with hematologic malignancies. Interviews and focus groups were open-ended and were recorded, transcribed verbatim, coded, and thematically analyzed to form the basis of the findings. The majority of participants in the study saw a perceived benefit in regular telephone follow-up as a supportive care service. Benefits included the positive aspects of individualized attention, potential support created for those not open to conventional types of support, and the positive effects of allowing support organizations to keep track of patient progress. However, some participants did not want to receive regular telephone follow-up support because of a desire to move on and access to other support through friends, family, or healthcare professionals. The results from the current study affirm previous research. Attitudes toward a telephone follow-up support service vary from patient to patient. Research demonstrates that patients with cancer responded with attitudes that range from favorable to unfavorable toward the benefits and usefulness of telephone support. Oncology nurses provide supportive care for patients with hematologic malignancies, and they serve as a key professional group to provide follow-up telephone supportive care. Based on the findings from the current article, oncology nurses who provide supportive care should be aware of differing attitudes among patients with hematologic malignancies toward telephone follow-up support. Nurses participating in support initiatives should recognize the notion of patient receptivity and how it affects telephone support strategies.