title: Improvement of quality of life in patients with steroid-refractory chronic graft-versus-host disease treated with the mTOR inhibitor everolimus.
abstract: 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.
authors.affiliation: Allogeneic Stem Cell Transplantation Section, Department of Internal Medicine II, Würzburg University Medical Center, Würzburg, Germany.
authors.forename: Mathias
authors.initials: M
authors.lastname: Lutz
authors.forename: Markus
authors.initials: M
authors.lastname: Kapp
authors.forename: Hermann
authors.initials: H
authors.lastname: Einsele
authors.forename: Götz Ulrich
authors.initials: GU
authors.lastname: Grigoleit
authors.forename: Stephan
authors.initials: S
authors.lastname: Mielke
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D002908:Chronic Disease; D000068338:Everolimus; D005260:Female; D005500:Follow-Up Studies; D005938:Glucocorticoids / Q000627:therapeutic use*; D006086:Graft vs Host Disease / Q000188:drug therapy* / Q000209:etiology / Q000523:psychology; D019337:Hematologic Neoplasms / Q000628:therapy; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D008297:Male; D008875:Middle Aged; D011183:Postoperative Complications; D011379:Prognosis; D011788:Quality of Life / Q000523:psychology*; D012189:Retrospective Studies; D012307:Risk Factors; D016879:Salvage Therapy; D012720:Severity of Illness Index; D020123:Sirolimus / Q000031:analogs & derivatives* / Q000627:therapeutic use; D011795:Surveys and Questionnaires; D058570:TOR Serine-Threonine Kinases / Q000037:antagonists & inhibitors*; D014184:Transplantation, Homologous; D055815:Young Adult
chemical_list: D005938:Glucocorticoids; D007166:Immunosuppressive Agents; D000068338:Everolimus; C546842:MTOR protein, human; D058570:TOR Serine-Threonine Kinases; D020123:Sirolimus
keywords: allogeneic hematopoietic stem cell transplantation; chronic graft-versus-host disease; everolimus; quality of life; retrospective analysis