Improvement of quality of life in patients with steroid-refractory chronic graft-versus-host disease treated with the mTOR inhibitor everolimus.

Compromised quality of life (QoL) is a frequent consequence of treatment-refractory chronic graft-versus-host disease (cGvHD). Here, we report on the assessment of QoL in a subgroup of 22 patients with a median age of 54 (17-70) yr receiving an everolimus-based salvage therapy at a single center for their steroid-refractory cGvHD. Five patients suffered from mild, 13 from moderate, and four from severe cGvHD according to NIH consensus criteria when everolimus was introduced. Median treatment duration was 390 d ranging from 86 to 814 d. We performed actual and retrospective assessments of QoL (EuroQol EQ-5D questionnaire) and degree of bother experienced by cGvHD symptoms (Lee cGvHD Symptom Scale). Seventeen of 22 patients showed an improved QoL according to the EQ-5D visual analog scale (37.5% vs. 70.0%; p < 0.001), and a decline in the median Lee cGvHD Symptom Scale was noted in 20 of 22 patients (28 vs. 17; p < 0.001). Furthermore, an improvement was noted in each of the five dimensions of the EQ-5D descriptive system. These data even when limited by their retrospective nature suggest that beyond physical responses everolimus may have contributed to the rebuilding of patients' QoL.