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儿童异基因造血干细胞移植后自身免疫性溶血性贫血的特征和结局。

Characteristics and outcomes of autoimmune hemolytic anemia after pediatric allogeneic stem cell transplant.

机构信息

Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, Massachusetts, USA.

Department of Pediatrics, Boston Children's Hospital, Boston, Massachusetts, USA.

出版信息

Pediatr Blood Cancer. 2022 Jan;69(1):e29410. doi: 10.1002/pbc.29410. Epub 2021 Oct 28.

Abstract

BACKGROUND

Autoimmune hemolytic anemia (AIHA) after allogeneic hematopoietic stem cell transplant (HSCT) is a rare but complex and serious complication. Detailed descriptions of cases and management strategies are needed due to lack of prospective trials.

OBJECTIVES

Describe the incidence, clinical characteristics, and management of AIHA after HSCT in a pediatric cohort.

METHODS

This is a retrospective cohort study of 33 pediatric patients with AIHA after HSCT at an academic tertiary care center from 2003 to 2019.

RESULTS

The overall incidence of AIHA after allogeneic HSCT was 3.8% (33/868). AIHA was significantly more common after transplant for nonmalignant versus malignant diagnoses (7.0% [26/370] vs. 1.4% [7/498], p < .0001). AIHA developed at a median of 4.7 months (range 1.0-29.7) after transplant. Sixteen of 33 patients (48.5%) required new AIHA-directed pharmacologic therapy; 17 (51.5%) were managed on their current immunosuppression and supportive care. Patients managed without additional therapy were significantly older, more likely to have a malignant diagnosis, and tended to develop AIHA at an earlier time point after transplant. Patients received a median of two red blood cell transfusions within the first 2 weeks of diagnosis and a median of one AIHA-directed medication (range one to four), most commonly corticosteroids and rituximab.

CONCLUSIONS

AIHA after HSCT is rare but occurs more commonly in patients transplanted for nonmalignant diagnoses. While some pediatric patients who develop AIHA after transplant can be managed on current immunosuppression and supportive care, many require AIHA-directed therapy including second-line medications.

摘要

背景

异基因造血干细胞移植(HSCT)后自身免疫性溶血性贫血(AIHA)是一种罕见但复杂且严重的并发症。由于缺乏前瞻性试验,需要详细描述病例和管理策略。

目的

描述儿科患者 HSCT 后 AIHA 的发生率、临床特征和管理方法。

方法

这是一项对 2003 年至 2019 年在学术三级护理中心接受 HSCT 的 33 例 AIHA 儿科患者的回顾性队列研究。

结果

异基因 HSCT 后 AIHA 的总体发生率为 3.8%(33/868)。与恶性诊断相比,非恶性诊断后 AIHA 的发生率明显更高(7.0%[26/370]比 1.4%[7/498],p<0.0001)。AIHA 发生在移植后中位数 4.7 个月(范围 1.0-29.7)。33 例患者中有 16 例(48.5%)需要新的 AIHA 靶向药物治疗;17 例(51.5%)在当前免疫抑制和支持治疗的基础上进行管理。未接受额外治疗的患者年龄明显较大,更有可能患有恶性诊断,并且在移植后更早的时间点发生 AIHA。诊断后前 2 周内,患者平均接受 2 次红细胞输注,平均接受 1 种(范围 1-4 种)AIHA 靶向药物,最常用的是皮质类固醇和利妥昔单抗。

结论

HSCT 后 AIHA 虽罕见,但在接受非恶性诊断的患者中更为常见。虽然一些移植后发生 AIHA 的儿科患者可以在当前免疫抑制和支持治疗的基础上进行管理,但许多患者需要 AIHA 靶向治疗,包括二线药物。

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