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

Clin Transplant. 2014 Dec;28(12):1410-5. doi: 10.1111/ctr.12472. Epub 2014 Nov 3.

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.

Keywords: allogeneic hematopoietic stem cell transplantation; chronic graft-versus-host disease; everolimus; quality of life; retrospective analysis.

Publication types

  • Clinical Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Chronic Disease
  • Everolimus
  • Female
  • Follow-Up Studies
  • Glucocorticoids / therapeutic use*
  • Graft vs Host Disease / drug therapy*
  • Graft vs Host Disease / etiology
  • Graft vs Host Disease / psychology
  • Hematologic Neoplasms / therapy
  • Hematopoietic Stem Cell Transplantation
  • Humans
  • Immunosuppressive Agents / therapeutic use
  • Male
  • Middle Aged
  • Postoperative Complications
  • Prognosis
  • Quality of Life / psychology*
  • Retrospective Studies
  • Risk Factors
  • Salvage Therapy
  • Severity of Illness Index
  • Sirolimus / analogs & derivatives*
  • Sirolimus / therapeutic use
  • Surveys and Questionnaires
  • TOR Serine-Threonine Kinases / antagonists & inhibitors*
  • Transplantation, Homologous
  • Young Adult

Substances

  • Glucocorticoids
  • Immunosuppressive Agents
  • Everolimus
  • MTOR protein, human
  • TOR Serine-Threonine Kinases
  • Sirolimus