Disease Burden and Treatment Impact Associated with Myelodysplastic Syndromes: Preliminary Findings From a Web-Based Survey.

Research has led to a better clinical understanding of myelodysplastic syndromes (MDS), but little is known about its burden from the patient perspective. In order to gain insight into the effects of MDS and its treatments on the lives of patients with MDS, patients with MDS self-administered a patient-reported outcomes (PRO) questionnaire.

No single comprehensive disease-specific questionnaire currently exists to assess health-related quality of life (HRQoL) in MDS patients. Based on a review of the literature and an understanding of the important issues that MDS patients face, we developed a PRO questionnaire which included: HRQoL items from existing questionnaires; health care resource utilization (HCRU) items including how the MDS diagnosis was made; the use of prior and current treatments; complications associated with treatment; and the impact of symptoms such as thrombocytopenia on usual activities. All adult patients with MDS are eligible to complete the questionnaire one time. Questionnaires are available at patient forums or alternatively patients can complete the survey online via the MDS Foundation website. The questionnaire, includes widely established measures such as the EuroQol (EQ)-5D, the FACT-Th [containing the Functional Assessment of Cancer Therapy (FACT)-General plus the 18 additional items for thrombocytopenia], and some additional questions developed specifically for this study and/or questionnaires currently being used in clinical studies. Data are currently available for the first 82 respondents, and data collection will continue until a total of approximately 200 MDS patients complete the survey.

EQ-5D scoring (Index and VAS scores) and FACT (total and thrombocytopenia scale) scores are based on published scoring algorithms. HRQoL comparisons were made across patients based on current hemoglobin levels (< 8 grams/dL versus ≥ 8 grams/dL) and current platelet count values (< 30 × 10⁹/L versus ≥ 30 × 10⁹/L).

The mean time since MDS diagnosis was 4.1 ± 4.3 years, while the most common diagnosis type was RARS (21%) followed by MDS associated with del (5q) or 5q- (17%). Forty seven percent had low risk, 36% intermediate risk 1, 11% intermediate risk 2 and 6% reported high risk IPSS scores. Mean age was 64.7 ± 11.2 years (range: 29.7 to 82.8) and a slight majority (54%) was male. Twenty six percent worked for pay, while 30% reported they were no longer able to work for pay or had to work part time instead of full time due to their MDS. Although sample sizes were small in the low platelet and low hemoglobin groups as reflected in the table below scores, the EQ-5D and FACT reflected better HRQoL in the higher hemoglobin and higher platelet value groups.

Results from this study provide valuable preliminary findings on the burden associated with MDS. Once data collection is complete, additional analyses will focus on the influence of current treatment, time since diagnosis, age and gender on disease burden. This PRO instrument is intended to serve as a basis for a quality of life instrument specific to the myelodysplastic syndromes currently under development by the MDS Foundation.

No relevant conflicts of interest to declare.