Page 195 - Haematologica - Vol. 105 n. 6 - June 2020

HRQoL with thalidomide and lenalidomide
Figure 1. Consort diagram. Consort diagram of the number of patients participating in the health-related quality of life (HRQoL) study, the number of answered ques- tionnaires and the number of patients off protocol and reason for treatment discontinuation.
Percentage of patients reaching a clinically meaningful change in HRQoL
In both arms, clinically relevant improvement in global QoL was more prominent than deterioration (Figure 2). During MPT induction therapy, improvement occurred in 48% of patients versus deterioration in 32%. With MPR induction improvement was reported in 52% of patients versus in 28% deterioration. After 1 year of thalidomide maintenance, 54% of patients improved versus 32% dete- riorated. For lenalidomide maintenance these figures were 61% versus 19%, respectively. The results for all other sub- scales are presented in Figure 2. Clinically relevant deteri- oration in peripheral neuropathy was significantly more frequently reported in the patients treated with MPT-T than in the patients treated with MPR-R, both after induc- tion (55% vs. 27%; P<0.001) and after maintenance (63% vs. 31%; P=0.003). A significantly higher percentage of patients treated with MPR reported clinically relevant worsening of diarrhea, compared to MPT, however after induction only (31% vs. 9%; P<0.001).
Changes in HRQoL within each treatment arm during induction and maintenance
In both arms, a significant improvement in HRQoL over time was observed for the majority of scales, irrespective
of the received treatment. Global QoL, role and emotional functioning, fatigue, pain, and future perspective improved clinically relevant in both arms. In addition, patients who were treated with thalidomide reported a clinically relevant improvement in social functioning, insomnia and appetite loss, while physical functioning improved in patients who were treated with lenalidomide (see Online Supplementary Table S2 for significant changes over time; see Figure 3 and Online Supplementary Figure S2 for clinical meaningful changes within arms). Overall, these HRQoL changes corresponded to medium clinical effects, except for pain reduction, which corresponded to a large clinical effect.24 In both arms, pain reduction was observed, irrespective of the number of bone lesions (0 vs. 1-2 vs. ≥3) (Online Supplementary Figure S3). In general, clin- ically meaningful improvement occurred from T3 and T4 onwards only (i.e. after six and 12 months of maintenance therapy). In contrast, global QoL, future perspective and pain improved already during induction therapy and was sustained throughout the whole treatment (Figure 3 and Online Supplementary Figure S2). Patients treated with MPT-T reported a statistically significant, but not clinically meaningful increase (small effect according to Cocks24) in constipation and side effects of treatment (P=0.003 and P<0.001 respectively, see Figure 3, Online Supplementary
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