Prospective assessment of health-related quality of life in pediatric patients with beta-thalassemia following hematopoietic stem cell transplantation.

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.

Caocci G ，Efficace F ，Ciotti F ，Roncarolo MG ，Vacca A ，Piras E ，Littera R ，Dawood Markous RS ，Collins GS ，Ciceri F ，Mandelli F ，Marktel S ，La Nasa G ... -  《-》

Quality of life assessment in hematopoietic stem cell transplantation performed on thalassemia major patients.

Uygun V ，Tayfun F ，Akcan M ，Karasu GT ，Kupesiz A ，Hazar V ，Yeşilipek A ... -  《-》

Measuring health-related quality of life in children with ESRD: performance of the generic and ESRD-specific instrument of the Pediatric Quality of Life Inventory (PedsQL).

Goldstein SL ，Graham N ，Warady BA ，Seikaly M ，McDonald R ，Burwinkle TM ，Limbers CA ，Varni JW ... -  《-》

Stem cell transplant for children with sickle cell anemia: parent and patient interest.

We describe adolescents' and parents' interest in hematopoietic stem cell transplant (HSCT) as a cure for sickle cell disease (SCD) and factors associated with increased interest. We administered a 40 question survey to assess the interest in HSCT in parents and adolescents with HBSS or HBSβ(0) thalassemia. The survey tool assessed factors that may influence interest in HSCT including demographic data, disease severity, views on prognosis, and health-related quality of life (HRQOL). All participants were given a handout on the risks and benefits of an HSCT before completing the survey. One hundred twenty-nine parents and 59 adolescents completed the survey. Forty-five percent of parents (54 of 119) would likely have their child undergo HSCT, and 35% of adolescents (19 of 55) would likely undergo HSCT if it was recommended by their hematologist. Parents of adolescents, as well as adolescent patients with better HRQOL, were more interested in HSCT. Prior exchange transfusion was associated with increased interest in HSCT (62% [23 of 37] versus 38% [29 of 76]; P = .02). The majority of parents believe their child's SCD will get better (66%; [80 of 122]), will not likely prevent their child from achieving life goals (83%; [100 of 121]), and will not shorten their child's lifespan (86%; [102 of 119]). There is strong parent and adolescent interest in HSCT as a cure for SCD. It is concerning that few parents and adolescents believe SCD will negatively impact their prognosis. Education on the potential long-term sequelae of SCD is needed when considering the role for HSCT.

Roth M ，Krystal J ，Manwani D ，Driscoll C ，Ricafort R ... -  《-》

Impaired health-related quality of life in children and adolescents with chronic conditions: a comparative analysis of 10 disease clusters and 33 disease categories/severities utilizing the PedsQL 4.0 Generic Core Scales.

Varni JW ，Limbers CA ，Burwinkle TM 《Health and Quality of Life Outcomes》