Table 3 Summary of QLQ-QLQ-MY20 results from 15 RCTs.
Study (Year) | RCT name and treatment comparison | N | QLQ-MY20 Subscale | Statistically significant mean difference? | Statistically significant TTD differences? | Evaluation of PRO results alongside clinical results in Abstract/Conclusion/Discussion | Further analyses of the QLQ-MY20 data |
|---|---|---|---|---|---|---|---|
Verelst S. et al. [33] | HOVON49 MPT vs MP | 284 | DS | X | NA | “In conclusion, this prospective study shows that the higher frequency of adverse effects associated with MPT does not translate into a negative effect on the HRQoL as reported by patients and that MPT holds a better patient perspective. This can be interpreted as a favorable result because the current standard treatment of MPT for elderly patients with myeloma is not only known to have an improved clinical outcome but also no reduction in HRQoL.” | Association of clinical response and toxicity with QLQ-C30 and MY24 scores. |
SE | X | NA | |||||
FP | X | NA | |||||
Dimopoulos M.A. et al. [46]* | Vantage 088 VOR + BTZ vs placebo | – | – | – | – | – | – |
Dimopoulos M.A. et al. [34] | MM-015 MP vs MPR vs MPR-R (MP vs MPR-R primary HRQoL aim) | 459 | DS | X (Statistically significant symptom relief in both arms) | NA | “evidence of a favorable effect of MPR-R over MP in terms of Physical Functioning, and a clear trend in improvements in all other HRQoL domains tested, including Global QoL, Fatigue, Pain, and Disease Symptoms. The statistically non-significant change in Side Effects of Treatment scores from baseline in the MPR-R group and its comparability with scores in the MP group highlights the favorable tolerability profile of the MPR-R regimen, both during induction and maintenance.” | – |
SE | X | NA | |||||
Weisel K. et al. [37] | MM-003 POM + LoDEX vs HiDEX | 433 | DS | NA | X | “These results report a real patient benefit: HRQoL is improved while patients receive therapy, and deteriorations in HRQoL are delayed in patients receiving POM þ LoDEX versus HiDEX.” | Proportion of patients improved/stable/worsened. Baseline scores versus best score prior to progression vs scores at progression. |
SE | NA | ✓ | |||||
Delforge M. et al. [36] | FIRST Rd vs MPT | 1623 | DS | X (Statistically significant symptom relief in both arms) | NA | “Continuous lenalidomide and low-dose dexamethasone delays disease progression versus melphalan, prednisone, thalidomide and has been associated with a clinically meaningful improvement in health-related quality-of-life. These results further establish continuous lenalidomide and low-dose dexamethasone as a new standard of care for initial therapy of myeloma by demonstrating superior health-related quality-of-life during treatment, compared with melphalan, prednisone, halidomide.” | Effect of age on treatment group differences with respect to QLQ-C30 and QLQ-MY20 scores. Deterioration of HRQoL with progression. |
SE | ✓ | NA | |||||
Stewart A.K. et al. [38] | ASPIRE KRd vs Rd | 792 | DS | x | x | “Results from the ASPIRE study confirm that the clinical benefits of the KRd triplet regimen, compared with the Rd doublet regimen, are associated with significant improvements in GHS/QoL, and there was no evidence of a detrimental impact from the triplet regimen on other aspects of HR-QoL.” | Proportion of patients improved/stable/worsened HRQoL by clinical response |
SE | x | x | |||||
Ludwig H. et al. [48] | ENDEAVOR Kd vs Vd | 929 | DS | x | x | “The delay in time to deterioration was significantly longer for Kd56 versus Vd for global HR-QoL, physical, nausea/vomiting, and side effects. The doubling of progression-free survival in the ENDEAVOR trial is associated with a prolonged period of time before deterioration of HR-QoL in the Kd56 versus Vd group; this is particularly relevant given that patients’ HR-QoL steadily degrades as the disease progresses and patients relapse and develop resistance to therapy” | Proportion improved |
SE | ✓ | ✓ | |||||
Richardson P. G. et al. 40] | PANORAMA-1 PAN + BTZ + DEX vs PBO + BTZ + DEX | 147 | DS | NA | NA | “The EORTC QLQ Myeloma module (EORTC QLQ-MY20) demonstrated initial improvements and subsequent stabilization of disease symptom scores in both arms and initial worsening and subsequent improvement of side effects of treatment scores, with the initial worsening more pronounced and recovery less pronounced with PAN + BTZ + DEX. Overall, these PRO findings support the addition of PAN to the BTZ + DEX regimen as an efficacious treatment option, with limited symptomatology and impact on patients’ QoL.” | Descriptive analysis only |
SE | NA | NA | |||||
FP | NA | NA | |||||
BI | NA | NA | |||||
Royle K. L. et al. [41] | Myeloma IX CVAD vs CTD + ASCT (intensive) Or CTD vs MP (non-intensive) | 1819 | DS | NA | NA | “The results of this study showed that improvements in clinical outcomes were not at the detriment of patient reported HR-QoL. The findings are reassuring in the context of continuing development of sequential treatment for induction, consolidation and maintenance. However, such large-scale studies, this being the largest to date, are a major undertaking and very unlikely to be given priority in future studies. Indeed, it could be that more sensitive QoL instruments, or potentially instruments focussed on specific domains, for example neurological, will be required to identify clinically relevant differences between treatment combinations.” | – |
SE | NA | NA | |||||
FP | NA | NA | |||||
BI | NA | NA | |||||
Cella D. et al. [42] | ELOQUENT-2 Eld vs Ld | 646 | DS | x | NA | “These findings show that previously reported improvements in progression-free survival and response rate with elotuzumab are achieved without detriment to HRQoL, which is maintained over time…Treatment responders showed more HRQoL and pain benefit than non-responders, supporting the clinical relevance of PROs in MM care.” | - |
SE | x | NA | |||||
Leleu X. et al. [39] | TOURMALINE-MM1 IRd vs placebo-Rd | 772 | DS | X (Reduced in both arms) | NA | “Findings from this double-blind study demonstrate that addition of ixazomib to Rd significantly improved efficacy while HRQoL was maintained, reflecting the limited additional toxicity seen with IRd versus placebo-Rd, and support the feasibility of long-term IRd administration.” | Proportion of patients improved/stable/worsened Changes in HRQol by depth of response |
SE | X (Increased in both arms) | NA | |||||
FP | ✓ (Increased in both arms, statistically significant greater improvements in IRd vs placebo-Rd at later timepoints) | NA | |||||
BI | x | NA | |||||
Nielsen L. K. et al. [24] | Danish Myeloma Study Group CLAIM study Clarthromycin + VCD vs placebo + VCD | 55 | DS | x (Clinically relevant differences) | NA | “adding clarithromycin to the VCD regimen in patients with myeloma resulted in impaired HRQoL during the VCD induction phase continuing up to two months after HDT… The PRO data in the CLAIM study played a key role in explaining the causality link between the observed complications and the possible interaction between clarithromycin and bortezomib” | – |
SE | x (Clinically relevant differences) | NA | |||||
FP | x | NA | |||||
BI | ✓ (Clinical and statistical relevant differences) | NA | |||||
Ahmedzai S.H. et al. [43] | Myeloma X sASCT vs NTC | 171 | DS | x | NA | “The small and diminishing differences in Global health status and Side effects of treatment need to be considered alongside the results of Myeloma X, which showed a significant benefit of sASCT on OS. The benefits of sASCT should be considered alongside the relatively short-term negative effects on QoL and pain when making patient treatment decisions and further support the use of sASCT.” | Association between baseline scales and time to progression clinical outcome |
SE | ✓ | NA | |||||
FP | x | NA | |||||
BI | x | NA | |||||
Moreau P. et al. [44] | ARROW Once-weekly Kd70mg/m2 vs twice-weekly Kd27mg/m2 | 469 | DS | x | ✓ | “Collectively, the primary A.R.R.O.W. safety and efficacy data and the current PRO analysis reinforce that once-weekly Kd70 mg/m2 dose is superior and convenient while delivering more favorable HRQOL than the commonly used Kd27 mg/m2 dose. Thus, once-weekly Kd70 mg/m2 should be considered an important alternative to twice-weekly Kd27 mg/m2 in clinical practice.” | Proportion of patients improved/stable/worsened |
SE | x | x | |||||
FP | x | x | |||||
Nielsen L.K. et al. | HOVON-87/NMSG18 MPT-T or MPR-R | 596 | DS | x | NA | “treatment with MPT-T and MPR-R improved HRQoL in elderly patients with NDMM and in general is clinically meaningful to the patients during maintenance therapy only. This supports the current paradigm of continuous treatment, not only improving survival, but also maintaining, and even improving, specific subscales of HRQoL.” | Proportion of patients improved/deteriorated Peripheral neuropathy analysed at the item level and showed statistically significant worsening during maintenance. All MY20 subscales except body image showed statistically significant changes over time within arms. |
SE | ✓ | NA | |||||
FP | x | NA | |||||
BI | ✓ (maintenance only) | NA |
