Department of Pediatrics, Faculty of Medicine Ramathibodi Hospital, Mahidol University, Bangkok, Thailand.
Ramathibodi Comprehensive Cancer Center, Faculty of Medicine Ramathibodi Hospital, Mahidol University, Bangkok, Thailand.
Int J Hematol. 2021 Apr;113(4):556-565. doi: 10.1007/s12185-020-03055-w. Epub 2021 Jan 1.
Data on chronic graft-versus-host disease (cGVHD) in patients with thalassemia after hematopoietic stem cell transplantation (HSCT) have not been specifically explored. The present study aimed to determine the incidence and clinical manifestations of cGVHD in children and adolescents with thalassemia who underwent HSCT and to compare healthcare utilization and medical cost between patients with and without cGVHD. We retrospectively analyzed the presentations, treatments, and outcomes of historical cGVHD (Seattle criteria), post-transplant admissions and direct medical cost for HSCT patients (n = 66). We used the 2014 NIH consensus criteria to reclassify the diagnosis of cGVHD (NIH cGVHD). Among 28 historical cGVHD patients, 13 (46.4%) fulfilled the NIH criteria. Reasons why the NIH criteria were unmet were reclassification as late acute GVHD and presence of distinctive signs without confirmatory tests. At 2 years after HSCT, the cumulative incidence of NIH cGVHD was 21.67% (95% CI, 12.31-32.74%). Lung cGVHD was associated with inferior survival with a hazard ratio of 13.6 (95% CI, 1.42-131.48). Patients with historical cGVHD had significantly increased frequency of inpatient admissions and medical cost. In conclusion, cGVHD was common in children with thalassemia receiving HSCT. Patients with cGVHD required prolonged immunosuppressive treatment and incurred high medical expenses.
关于接受造血干细胞移植 (HSCT) 后的地中海贫血患者慢性移植物抗宿主病 (cGVHD) 的数据尚未专门探讨。本研究旨在确定接受 HSCT 的地中海贫血儿童和青少年中 cGVHD 的发生率和临床表现，并比较有和无 cGVHD 患者的医疗保健利用和医疗费用。我们回顾性分析了 66 例 HSCT 患者的历史 cGVHD（西雅图标准）、移植后入院和直接医疗费用的表现、治疗和结局。我们使用 2014 年 NIH 共识标准重新分类 cGVHD 的诊断（NIH cGVHD）。在 28 例历史 cGVHD 患者中，13 例（46.4%）符合 NIH 标准。不符合 NIH 标准的原因是重新分类为晚期急性移植物抗宿主病和存在特征性体征但无确认性检查。在 HSCT 后 2 年，NIH cGVHD 的累积发生率为 21.67%（95%CI，12.31-32.74%）。肺 cGVHD 与生存率降低相关，风险比为 13.6（95%CI，1.42-131.48）。有历史 cGVHD 的患者住院次数和医疗费用明显增加。总之，接受 HSCT 的地中海贫血患者中 cGVHD 很常见。患有 cGVHD 的患者需要长期免疫抑制治疗，并产生高额医疗费用。
