Robert J Klaassen, Shabbir Alibhai, Melanie Kirby Allen, Katherine Moreau, Manuela Merelles Pulcini, Melissa Forgie, Victor Blanchette, Rena Buckstein, Isaac Odame, Ian Quirt, Karen Yee, Durhane Wong Rieger and Nancy L Young
Background: Patients with thalassemia major require red cell transfusions for survival and have to deal with iron overload and chelation. Chelation is burdensome, traditionally involving nightly prolonged subcutaneous infusion therapy. We developed a disease-specific tool for these patients (TranQol) to measure their unique quality of life issues.
Methods: Pediatric and adult thalassemia health care professionals and quality of life methodology experts generated 69 potential items. 74 further questions were generated through interviews with patients (pediatric and adult) and parents.
Results: 120 participants contributed: 16 healthcare workers, 31 children and 30 adults with thalassemia and 43 parents. Duplicate and infrequent questions were discarded leaving 58 items. Three self-reported questionnaires (child, parent and adult) and one child proxy-report for parents were developed. Questionnaire length ranged from 29 (child’s) to 39 (parent’s). Questions were grouped into four domains: physical health, emotional health, family functioning, and school and career functioning. A fifth category on sexual activity included only one item. Cognitive debriefing was done by interviewing additional children, parents, and adults. As a result, three items were added, one was deleted and 16 were modified.
Conclusion: The TranQol is a new disease-specific quality of life measure for thalassemia major patients developed using rigorous methodology.