Myelodysplastic syndromes (MDS) are stem cell malignancies with diverse clinical presentations and variable prognoses. Patient-reported outcomes (PROs) may be invaluable in treatment decisions, particularly given modest survival benefit of new MDS therapies. We present here psychosocial assessment data, using a National Institutes of Health (NIH) developed instrument for rare conditions, on participants enrolled in the Comprehensive Molecular and Clinical Evaluation of Pediatric and Adult MDS (NCT05350748) at NCI.

At reporting, 40 subjects with suspected MDS per eligibility requirement were enrolled with n=27 completing both the NIH developed psychosocial assessment and the European Organization for Research and Treatment of Cancer (EORTC) QLQ-C30. Diagnoses after full evaluation included n=6 found to have no malignancy, 2 CHIP/CCUS, 5 non-MDS/AML hematologic malignancy, 12 MDS, and 2 AML cases. Average age was 63.4 years (15 male, 12 female). Races were White (n=20), Black or African American (n=4), Asian (n=1), and unknown (n=2); ethnicity included 22 Not Hispanic or Latino, 4 Hispanic or Latino, and 1 unknown. Eight (29.6%) were currently on treatment including DNMTi, erythropoietin, venetoclax, and luspatercept.

At study visit, 7.4% participants reported health was “excellent”, 22.2% “very good”, and 48.1% “good”. The majority (77.7%) were experiencing infrequent pain yet 40.7% reported this pain interfered with their life and 52.5% endorsed MDS impacting their quality of life “moderately” to “a lot”. History of mental health visits was reported by 59.3% for anxiety, depression, and other health related issues (PTSD, erectile dysfunction) and 44.4% reported prescription treatment history for anxiety, 37% for depression, and 11.1% for attention. Over the past year, the following symptoms were reported to cause “moderate amount” to “a lot” of problems, fatigue (55.6%), weakness (33.3%), shortness of breath (29.6%), bleeding (7.4%), skin issues (14.8%), bruising (22.2%), frequent infections (3.7%), anxiety (29.6%), and depression (3.7%). In an open-ended question, participants reported the hardest part of living with their disease was the fatigue limiting social activities and anxiety pertaining to uncertainties of living with a potentially fatal disease.

The majority of participants (85.2%) found information provided pertaining to MDS helpful to understand health care needs, including being more aware of possible disease-related future health care. To why participants enrolled on this clinical study, the majority reported wanting to learn more about the disease (96.3%), wanting to help others and contribute to scientific research (88.9%), being followed closely for “peace of mind” (66.6%) and to be connected with scientists (88.9%); while only 14.8% reported enrolling for financial related reasons.

Participants reported other services that would be helpful including education about MDS (88.9%), financial services (77.8%), fatigue interventions (59.3%), nutritional guidance (55.6%), integrated complimentary services (e.g., acupuncture) (55.6%), counseling for self (40.7%), for family (25.9%), meet others with MDS (40.7%), anxiety reduction interventions (29.6%), support group for self or family (22.2%), spiritual support (14.8%), and support for fertility issues (7.4%).

Participants also completed the EORTC QLQ-C30 where, unexpectantly, overall health status of the cohort was 67.9 +/- 23.4, consistent US normative data of individuals 60-69 years (66.3 +/- 21) (Rogge et al. 2024 Eur J Cancer) with no difference in overall health status when separating participants based on final diagnosis. Importantly, measures across the two instruments scored consistently.

These data address the importance of assessing psychosocial needs for people living with MDS and related conditions due to emotional, psychological, physical and social challenges that accompany diagnosis. Despite reporting their health as “good”, participants reported significant anxiety due to uncertainty, fatigue and other serious symptoms. These data also provide valuable feedback on participation in clinical trials and a host of unmet needs in health care delivery. This information can help health care professionals offer tailored support and planning of future intervention trials to improve clinical outcomes, as well as to help patients cope more effectively with their condition.

This content is only available as a PDF.
2025
Sign in via your Institution