Quality of Life in Low-Risk MDS: An Undervalued Endpoint

Abstract

The opinion I put forward in this paper is that attention must be paid to health-related quality of life as a study endpoint in lower-risk MDS patients. At the moment therapeutic options are limited in this population. New treatments are predominantly available in clinical studies. In announcing trial publications and during manuscript introductions, quality of life is widely valued as a treatment goal. However, data on health-related quality of life during phase III studies are not published in the original publications, thereby undermining the importance of quality of life as a study endpoint. What seems to be forgotten is that quality of life comprises more than a study endpoint. It is the highest good of lower-risk MDS patients and should also be acknowledged as a safety aspect. Current phase II trials with new medications do not collect data on health-related quality of life, a practice that I consider unethical. In this opinion I demonstrate the current attitude towards health-related quality of life in lower risk MDS patients among leading journals and trial sponsors with several recent examples. I also argue that health-related quality of life should be the main treatment goal in this population. In the event that we shift our focus towards health-related quality of life as the main treatment goal, new treatments could come to a field where gains in overall survival have been marginal over the years.

MDS-Related Anemia Is Associated with Impaired Quality of Life but Improvement Is Not Always Achieved by Increased Hemoglobin Level

“…Despite accepting the idea that the relief of symptoms and improving QoL should be major goals of treatment, at least in lower-risk MDS [ 4 , 5 , 13 ], little has been conducted in terms of studying the topic or defining it as a major endpoint in clinical trials [ 9 , 16 , 30 , 31 ].…”

Disease Perception Is Correlated with Health-Related Quality of Life in Patients Suffering from Myelodysplastic Syndromes: Results of the Belgian Be-QUALMS Study

“…This is not only necessary to ameliorate the daily life of MDS patients, restrictions in HRQoL may also prelude a less favorable clinical outcome [4]. An objective HRQoL assessment can also provide a response criterion to treatment and clears the path to implement HRQOL as a safety aspect as we argued earlier [5]. The growing interest in HRQoL has led to the development and propagation of disease specific PRO-tools that have become standard content in interventional trials with new medications.…”