PROs Used in Low-Risk MDS Found to Have Strong Face, Content Validity

A recent study evaluated the content validity of 2 patient-reported outcome instruments used in lower-risk myelodysplastic syndrome (MDS).

Writing in the Journal of Patient-Reported Outcomes, the authors noted that MDS be underestimated in the United States and is projected to grow; one estimate, from 2015, said that prevalence may range between 60,000 to 170,000, and incidence among patients aged 65 years and above at 75 to 162 per 100,000 patients.

MDS, as stratified by the International Prognostic Scoring System (IPSS), falls into 1 of the 2 risk categories: lower-risk (IPSS: low and intermediate-1) or higher-risk (IPSS: intermediate-2 and high).

Lower-risk MDS tends to progress slowly compared with higher-risk MDS. It is marked by macrocytic anemia, which is treated with erythropoiesis-stimulating agents (ESAs), but the response duration is limited. Cause of death in lower-risk MDS is typically related to bone marrow failure rather than progression to acute myeloid leukemia.

Patients have a worse health-related quality of life (HRQoL), particularly stemming from anemia with red blood cell (RBC) transfusion dependency and subsequent iron overload. Symptoms include fatigue, lethargy, headache, dizziness, shortness of breath, pale skin, and heart palpitations.

Patient-reported outcome (PROs) tools are increasingly used in hematologic malignancies to capture HRQoL data, although one challenge is ensuring that they have content validity for the patient population to ensure that they are robust and comprehensible.

“PRO data are an important complement to assess how patients feel and function when used in conjunction with other study endpoints like survival, transfusion independence or hematologic improvement,” the authors noted.

The current study evaluated 2 PRO questionnaires for content validity in low-risk MDS: the Quality of Life in Myelodysplasia Scale (QUALMS) and the Functional Assessment of Cancer Therapy-Anemia (FACT-An). The QUALMS, with 38 questions, is MDS-specific and assesses HRQoL. The FACT-An is a 47-item cancer-specific PRO instrument that evaluates the effect of anemia on HRQoL.

The researchers interviewed 16 adult patients with low-risk MDS who were RBC transfusion dependent. Their median age was 67.5 years (range: 51–91) and 50% were female. Nine (56.2%) participants had intermediate-1-risk MDS and 10 (62.5%) were relapsed or refractory to erythropoiesis-stimulating agent treatment.

The semi-structured interviews had 2 parts. In the first part, patients were asked open-ended questions about their experiences with MDS (ie, the concept elicitation section).

In the second part, patients answered the 2 survey instruments with the interviewer using what the authors called a “think aloud” approach (ie, the cognitive domain section). The patients shared their thoughts as they answered each question, which gave the interviewer a chance to assess the understanding of each question. In addition, patients were asked about “their interpretation and understanding of instructions and item wording, the relevance of concepts, the appropriateness of the response options and recall period, and whether any concepts were missing.”

The most common and bothersome symptoms were fatigue/tiredness (100.0%), shortness of breath (87.5%), weakness (81.2%), and low energy (75.0%).

Out of 7 domains of HRQoL, the most often-cited impacted areas included activities of daily living (n = 16, 100.0%), physical functioning (n = 15, 93.8%), emotional wellbeing (n = 13, 81.3%), social functioning (n = 12, 75.0%), sleep disturbance (n = 9, 56.3%), and impact on work (n = 9, 56.3%).

Patients understood both tools and found them relevant and easy to use.

“Both QUALMS and FACT-An demonstrated a strong face and content validity in patients with lower-risk MDS, suggesting that these instruments are appropriate for assessing HRQoL in this population,” the authors concluded.

Reference

Trudeau J, He J, Rose E, Panter C, Randhawa S, Gater A. Content validity of patient-reported outcomes for use in lower-risk myelodysplastic syndromes. J Patient Rep Outcomes. 2020;12(4)69. doi: 10.1186/s41687-020-00235-4