Servicio de Hematología y Oncología Pediátrica, Hospital Universitario La Paz, Madrid, Spain.
Facultad de Medicina, Universidad Autónoma de Madrid, Madrid, Spain.
An Pediatr (Engl Ed). 2024 Apr;100(4):259-267. doi: 10.1016/j.anpede.2024.03.011. Epub 2024 Mar 28.
Total body irradiation (TBI) is part of the myeloablative conditioning for hematopoietic stem cell transplantation (HSCT) in malignant hematologic disorders. This therapy has recently shown improved survival in acute lymphoblastic leukemia (ALL) compared to chemotherapy-based regimens. However, side effects are a significant limitation, especially in the pediatric population.
We retrospectively analyzed the survival of patients with ALL who underwent an HSCT at a tertiary hospital between 1996 and 2009 (N = 69 HSCT in 57 patients). We differentiated a cohort that received TBI (N = 44) from another that did not (N = 25). Subsequently, we interviewed the survivors from the TBI group with a minimum of 10 years of follow-up (N = 18), asking about the presence of side effects.
The overall survival (OS) at 2 and 5 years was 79.1% and 65.2% respectively for the TBI group and 66.2% and 55.8% for the non-TBI group, although this difference was not significant (P=.31). The event-free survival (EFS) at 2 and 5 years was 77.3% and 63.6% respectively for the TBI group and 56% and 32% for the non-TBI group (P=.02). The probability of relapse (PR) at 2 years for those who received TBI was 10% compared to 28.6% for those who did not receive TBI (P=.005). Survivors who received TBI developed secondary neoplasms (39%), dyslipidemia (67%), cognitive impairments affecting memory (44%), recurrent respiratory infections (39%), thyroid abnormalities (45%), premature ovarian failure (89%), cataracts (22%), and psychological problems (44%). However, the quality of life, as self-assessed by the patients, was considered good for 83% of the participants..
Patients who received TBI had significantly higher EFS and lower PR. However, adverse effects are frequent and significant, although they do not subjectively affect quality of life.
全身照射（TBI）是恶性血液病造血干细胞移植（HSCT）中骨髓清除性预处理的一部分。与基于化疗的方案相比，这种治疗方法最近在急性淋巴细胞白血病（ALL）中显示出了提高生存率的效果。然而，副作用是一个显著的限制，尤其是在儿科人群中。
我们回顾性分析了 1996 年至 2009 年期间在一家三级医院接受 HSCT 的 ALL 患者的生存情况（57 例患者中有 69 例 HSCT）。我们将接受 TBI 的患者分为一组（N=44），将未接受 TBI 的患者分为另一组（N=25）。随后，我们对 TBI 组中至少随访 10 年的幸存者（N=18）进行了采访，询问他们是否存在副作用。
TBI 组的总生存率（OS）在 2 年和 5 年时分别为 79.1%和 65.2%，而非 TBI 组分别为 66.2%和 55.8%，但差异无统计学意义（P=.31）。TBI 组的无事件生存率（EFS）在 2 年和 5 年时分别为 77.3%和 63.6%，而非 TBI 组分别为 56%和 32%（P=.02）。接受 TBI 的患者在 2 年内复发的概率为 10%，而未接受 TBI 的患者为 28.6%（P=.005）。接受 TBI 的幸存者发生了继发性肿瘤（39%）、血脂异常（67%）、影响记忆的认知障碍（44%）、复发性呼吸道感染（39%）、甲状腺异常（45%）、卵巢早衰（89%）、白内障（22%）和心理问题（44%）。然而，患者自我评估的生活质量被认为良好的占 83%。
接受 TBI 的患者 EFS 显著提高，PR 显著降低。然而，不良影响是常见且严重的，尽管它们不会主观上影响生活质量。
