Mulas Olga, Efficace Fabio, Orofino Maria Grazia, Piroddi Antonio, Piras Eugenia, Vacca Adriana, Barella Susanna, Costa Alessandro, Giesinger Johannes M, La Nasa Giorgio, Caocci Giovanni
Hematology Unit, Businco Hospital, Department of Medical Sciences and Public Health, University of Cagliari, 09124 Cagliari, Italy.
Health Outcomes Research Unit, Italian Group for Adult Hematologic Diseases (GIMEMA) Data Center, 00161 Rome, Italy.
J Clin Med. 2023 Sep 19;12(18):6047. doi: 10.3390/jcm12186047.
Matched hematopoietic stem cell transplantation (HSCT) is a feasible and curative treatment in pediatric patients with beta thalassemia major (β-TM). However, little data are available regarding patients and their parents' health-related quality of life (HRQoL) after the procedure. As such, we investigated the HRQoL of pediatric patients with β-TM after HSCT compared to that of patients treated with blood transfusions and iron chelation. The health-related quality of life of 43 β-TM pediatric patients and 43 parents were evaluated using the Pediatric Quality of Life Inventory (PedsQL). A total of 25 patients underwent HSCT: 15 from a sibling and 10 from an HLA-matched donor. The median follow-up time from HSCT was 5 years (range 1-13 years). The mean ages at the survey were 10.1 years (range 5-15) and 9.6 years (range 5-15) for transfused and transplanted patients, respectively. A significant reduction in HRQoL was reported in the group of transfused patients compared with that of patients transplanted in the following PedsQL domains: children's and parents' physical functions, Δ = -15.4, 0.009 and Δ = -11.3, 0.002, respectively; children's and parents' emotional functioning, Δ = -15.2, 0.026 and Δ = -15.2, 0.045, respectively; child's and parents' school functioning, Δ = -25, 0.005 and Δ = -22.5, 0.011, respectively; total child and parents scores, Δ = -14.5, 0.004 and Δ = -13.2, 0.005, respectively. The results of a multivariable analysis showed that the HSCT procedure was significantly associated with a higher total child PedsQL score (adjusted mean difference = 15.3, 0.001) and a higher total parent PedsQL score (adjusted mean difference = 14.1, 0.006). We found no significant difference in the HRQoL measured after sibling or unrelated human leukocyte antigen (HLA)-matched HSCT. Finally, a significant positive correlation across all the PedsQL domains was found between the scores reported by the children and those reported by their parents. In conclusion, our study shows that HSCT in pediatric patients with β-TM is associated with a good overall HRQoL profile. This information further supports physicians when counseling patients and their parents before the HSCT procedure.
匹配的造血干细胞移植（HSCT）对于重型β地中海贫血（β-TM）患儿是一种可行的治愈性治疗方法。然而，关于该手术后患儿及其父母的健康相关生活质量（HRQoL）的数据却很少。因此，我们调查了HSCT术后β-TM患儿的HRQoL，并与接受输血和铁螯合治疗的患儿进行了比较。使用儿童生活质量量表（PedsQL）对43例β-TM患儿及其43位父母的健康相关生活质量进行了评估。共有25例患儿接受了HSCT：15例来自同胞供者，10例来自人类白细胞抗原（HLA）匹配的供者。HSCT后的中位随访时间为5年（范围1 - 13年）。输血组和移植组患儿在调查时的平均年龄分别为10.1岁（范围5 - 15岁）和9.6岁（范围5 - 15岁）。在PedsQL的以下领域中，与移植组患儿相比，输血组患儿的HRQoL显著降低：儿童和父母的身体功能，差异分别为Δ = -15.4，P = 0.009和Δ = -11.3，P = 0.002；儿童和父母的情感功能，差异分别为Δ = -15.2，P = 0.026和Δ = -15.2，P = 0.045；儿童和父母的学校功能，差异分别为Δ = -25，P = 0.005和Δ = -22.5，P = 0.011；儿童和父母的总分，差异分别为Δ = -14.5，P = 0.004和Δ = -13.2，P = 0.005。多变量分析结果显示，HSCT手术与儿童PedsQL总分显著升高（调整后平均差异 = 15.3，P = 0.001）以及父母PedsQL总分显著升高（调整后平均差异 = 14.1，P = 0.006）相关。我们发现同胞供者或无关HLA匹配的HSCT术后所测量的HRQoL没有显著差异。最后，在所有PedsQL领域中，儿童报告的分数与父母报告的分数之间存在显著的正相关。总之，我们的研究表明，β-TM患儿进行HSCT与良好的总体HRQoL状况相关。这些信息在HSCT手术前为医生向患儿及其父母提供咨询时提供了进一步的支持。
