Abstract Topic: 35. Quality of life and palliative care
Background: It is well known that quality of life is compromised in myelodysplastic syndromes (MDS). The impact of severe thrombocytopenia and its treatment in such patients has not yet been reported. EQOL-MDS is an international, multicenter, randomized, single-blind, placebo-controlled trial to evaluate safety and efficacy of eltrombopag for the treatment of severe thrombocytopenia of lower risk MDS. Primary endpoints (platelet response in the first 24 weeks, safety, duration of platelet response) have been reached and previously published, favouring eltrombopag with a response rate of 42% versus 11% for placebo.
Aims: The aim is to evaluate, as a secondary endpoint, the changes in patient-reported outcome measures (PROMs) at 24 weeks.
Methods: Main inclusion criteria are age ≥ 18 years with morphological diagnosis of low or intermediate-1 international prognostic scoring system MDS with stable platelet count <30 Gi/L. 169 participants (104 males; median age 72 [IQR 65 -79] years) were randomly assigned (2:1) to receive eltrombopag or matching placebo at an initial dose of 50 mg once daily, titrated in 50 mg increments every 2 weeks up to a maximum of 300 mg to achieve and maintain a complete platelet response, defined as a platelet count ≥100 Gi/L without bleeding. PROMs included EORTC QLQ-C30 and the MDS-specific instrument, QOL-E, assessed at baseline and at 24 weeks.
Results: For those achieving a platelet response, there were proportionally more cases with stability or clinically meaningful and significant improvement in PROMS with respect to non-responders who had a significantly higher proportion of worsening (Table).
Summary/Conclusion: This is the first assessment of PROMs in patients with lower risk MDS receiving treatment for severe thrombocytopenia in a randomized trial. Changes in platelet counts were associated with clinically meaningful improvements in quality of life and symptoms which confers further value to treatment with eltrombopag beyond increasing platelet counts.
Table
| QOL-E Domain changes at 24 weeks | Responders (%) | Non-responders (%) | p-value | |
|---|---|---|---|---|
| Functional | Worsened | 15.0 | 42.4 | 0.023 |
| Stable | 50.0 | 39.4 | ||
| Improved | 35.0 | 18.2 | ||
| Physical | Worsened | 43.5 | 45.7 | 0.732 |
| Stable | 21.7 | 25.7 | ||
| Improved | 34.8 | 28.6 | ||
| Social | Worsened | 16.0 | 33.3 | 0.178 |
| Stable | 60.0 | 48.5 | ||
| Improved | 24.0 | 18.2 | ||
| Fatigue | Worsened | 8.7 | 45.7 | < 0.0001 |
| Stable | 47.8 | 37.1 | ||
| Improved | 43.5 | 17.1 | ||
| MDS-Specific | Worsened | 6.3 | 50.0 | 0.004 |
| Stable | 68.8 | 34.6 | ||
| Improved | 24.9 | 15.4 | ||
| General | Worsened | 9.1 | 40.0 | 0.012 |
| Stable | 54.5 | 50.0 | ||
| Improved | 36.4 | 10.0 | ||
| All | Worsened | 11.1 | 43.8 | 0.018 |
| Stable | 44.5 | 43.8 | ||
| Improved | 44.4 | 12.4 | ||
| Treatment-outcome index | Worsened | 8.3 | 60.9 | 0.007 |
| Stable | 58.3 | 17.4 | ||
| Improved | 33.4 | 21.7 | ||
| EORTC QLQ-C30 Domain changes at 24 weeks | Responders (%) | Non-responders (%) | p-value | |
| Global Health Status | Worsened | 11.1 | 37.8 | 0.010 |
| Stable | 40.7 | 35.2 | ||
| Improved | 48.2 | 27.0 | ||
| Physical | Worsened | 11.5 | 41.7 | 0.028 |
| Stable | 57.7 | 36.1 | ||
| Improved | 30.8 | 22.2 | ||
| Role function | Worsened | 8.0 | 36.1 | 0.260 |
| Stable | 60.0 | 27.8 | ||
| Improved | 32.0 | 36.1 | ||
| Emotional | Worsened | 14.8 | 32.4 | 0.136 |
| Stable | 59.3 | 48.6 | ||
| Improved | 25.9 | 19.0 | ||
| Cognitive | Worsened | 33.3 | 27.0 | 0.948 |
| Stable | 37.0 | 48.7 | ||
| Improved | 29.7 | 24.3 | ||
| Social | Worsened | 29.6 | 45.9 | 0.594 |
| Stable | 59.3 | 32.5 | ||
| Improved | 11.1 | 21.6 | ||
| Fatigue | Worsened | 19.2 | 25.7 | 0.832 |
| Stable | 57.7 | 48.6 | ||
| Improved | 23.1 | 25.7 | ||
Keywords: Myelodysplastic syndrome, Clinical trial, Thrombocytopenia, Quality of life