title: Prospective assessment of health-related quality of life in pediatric patients with beta-thalassemia following hematopoietic stem cell transplantation.
abstract: Although hematopoietic stem cell transplantation (HSCT) has been widely used to treat pediatric patients with beta-thalassemia major, evidence showing whether this treatment improves health-related quality of life (HRQoL) is lacking. We used child-self and parent-proxy reports to prospectively evaluate HRQoL in 28 children with beta-thalassemia from Middle Eastern countries who underwent allogeneic HSCT in Italy. The PedsQL 4.0 Generic Core Scales were administered to patients and their parents 1 month before and 3, 6, and 18 months after transplantation. Two-year overall survival, thalassemia-free survival, mortality, and rejection were 89.3%, 78.6%, 10.9% and 14.3%, respectively. The cumulative incidence of acute and chronic graft-versus-host disease (GVHD) was 36% and 18%, respectively. Physical functioning declined significantly from baseline to 3 months after HSCT (median PedsQL score, 81.3 vs 62.5; P = .02), but then increased significantly up to 18 months after HSCT (median score, 93.7; P = .04). Agreement between child-self and parent-proxy ratings was high. Chronic GVHD was the most significant factor associated with lower HRQoL scores over time (P = .02). The child-self and parent-proxy reports showed improved HRQoL in the children with beta-thalassemia after HSCT. Overall, our study provides preliminary evidence-based data to further support clinical decision making in this area.
authors.affiliation: Bone Marrow Transplant Center, R. Binaghi Hospital, Cagliari, Italy. gcaocci@alice.it
authors.forename: Giovanni
authors.initials: G
authors.lastname: Caocci
authors.forename: Fabio
authors.initials: F
authors.lastname: Efficace
authors.forename: Francesca
authors.initials: F
authors.lastname: Ciotti
authors.forename: Maria Grazia
authors.initials: MG
authors.lastname: Roncarolo
authors.forename: Adriana
authors.initials: A
authors.lastname: Vacca
authors.forename: Eugenia
authors.initials: E
authors.lastname: Piras
authors.forename: Roberto
authors.initials: R
authors.lastname: Littera
authors.forename: Raji Suleiman
authors.initials: RS
authors.lastname: Dawood Markous
authors.forename: Gary Stephen
authors.initials: GS
authors.lastname: Collins
authors.forename: Fabio
authors.initials: F
authors.lastname: Ciceri
authors.forename: Franco
authors.initials: F
authors.lastname: Mandelli
authors.forename: Sarah
authors.initials: S
authors.lastname: Marktel
authors.forename: Giorgio
authors.initials: G
authors.lastname: La Nasa
mesh_terms: D000203:Activities of Daily Living; D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D005260:Female; D006086:Graft vs Host Disease / Q000401:mortality / Q000473:pathology / Q000523:psychology*; D006304:Health Status; D018380:Hematopoietic Stem Cell Transplantation / Q000401:mortality / Q000523:psychology*; D006801:Humans; D015994:Incidence; D007558:Italy; D008297:Male; D008877:Middle East; D010290:Parents; D010372:Pediatrics; D011446:Prospective Studies; D011788:Quality of Life / Q000523:psychology*; D018434:Sickness Impact Profile; D011795:Surveys and Questionnaires; D016019:Survival Analysis; D017086:beta-Thalassemia / Q000401:mortality / Q000473:pathology / Q000523:psychology* / Q000628:therapy