Health-related quality of life in pediatric patients after allogeneic SCT: development of the PedsQL Stem Cell Transplant module and results of a pilot study

Abstract

With increased survival after pediatric allogeneic hematopoietic SCT health-related quality of life (HRQL) has emerged as an essential health outcome. The impact of transplant and chronic GVHD (cGVHD)-associated morbidity remains a major obstacle. In 2005, the National Institutes of Health (NIH) Consensus Conference on Criteria for Clinical Trials in cGVHD recommended HRQL tools as an independent measure of the impact of disease burden. The NIH recommendations did not provide a cGVHD-specific tool for HRQOL measures in children. This report focuses on the development of an SCT-specific instrument to assess HRQL in children and adolescents. For the assessment of generic HRQL we chose the PedsQL (Pediatric Quality of Life Inventory) Generic Cores Scales, which have been used in a large number of healthy, acutely ill and chronically ill children and adolescents. To capture SCT-and, specifically, cGVHD-related problems, we developed the PedsQL Stem Cell Transplant module by reviewing the literature, taking over some items/scales of other PedsQL modules, interviewing patients, parents and members of the health-care team, and applying the PedsQL measurement methods. The final PedsQL Stem Cell Transplant module consists of the HRQL domains: pain and hurt, fatigue/sleeping problems/weakness, nausea, worry/anxiety about disease/treatment, nutritional problems, neurocognitive problems, communication about disease/treatment, loneliness, physical functioning and additional somatic complaints (pruritus, skin inflammation, oral problems, eyes or breathing) including patients' and parents' assessment. It was tested in 35 pediatric patients, who were referred to our SCT Outpatient Clinic about 100 days post SCT. Both the generic PedsQL and the SCT-specific scales showed high internal consistency, with Cronbach alpha levels of ⩾ 0.70 in almost all scales. Most problems were detected within the HRQL domains of physical functioning and pain. The summary scores of the generic PedsQL and the PedsQL Stem Cell Transplant module showed high correlations (r = 0.89 in patients' and r = 0.81 in parents' assessments). Moreover, both tools discriminated between patients with and without cGVHD. The PedsQL Stem Cell Transplant module is practical for use and suitable across a broad age range (2-18 years) both in patients with and without cGVHD. However, it is still a pilot instrument and needs further development and testing in a larger patient population.

Psychometric properties of the Activities Scale for Kids-performance after allogeneic hematopoietic stem cell transplantation in adolescents and children

“…Consistent with the finding of another study by Christakou et al [28] we observed that the lack of a pain scale within the ASKp is worth mentioning as many cGVHD patients especially with joint and fascia involvement and/or steroid side effects have pain along with their symptoms of cGVHD. We have previously published results from a prospective study on health-related quality of life after pediatric HSCT that most problems were detected within the domains of physical functioning and pain [12].…”

“…Chronic GVHD is an extremely complex multisystemic chronic disorder with heterogeneous pathogenesis [6] and clinical manifestations resembling a variety of autoimmune diseases with involvement of many tissues and organs in adults, adolescent and pediatric patients [7,8]. Furthermore, cGVHD is associated with decreased survival, prolonged duration of immunosuppression [9], adverse physical functioning, disability [10] and decreased QOL [11,12].…”

Measuring Therapeutic Response in Chronic Graft-versus-Host Disease. National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease: IV. The 2014 Response Criteria Working Group Report

“…Instead, the Working Group recommends exploration of the PedsQL based on results in other chronic illnesses and hematopoietic cell transplantation. 93–95 …”

Safety and Feasibility of Rehabilitation Interventions in Children Undergoing Hematopoietic Stem Cell Transplant With Thrombocytopenia

“…5–14 Children undergoing HSCT require prolonged protective isolation given their vulnerability to infections and many of them are very sedentary while hospitalized and confined to their room. Prolonged immobility negatively affects multiple organ systems, including musculoskeletal, 15–17 cardiovascular, 17,18 respiratory, 17,19 integumentary 17 and endocrine systems.…”