高危急性早幼粒细胞白血病ATRA/ATO治疗期间继发性颅内高压：一例病例报告及实用管理框架
Secondary Intracranial Hypertension During ATRA/ATO Therapy in High-Risk Acute Promyelocytic Leukemia: A Case Report and Practical Management Framework.
文献信息
| PMID | 42930178 |
|---|---|
| 原文 | 在 PubMed 查看原文 ↗ |
| 发表日期 | 2026 |
| 作者 | Luca Roeh |
| 作者单位 | Department of Hematology, Oncology and Tumor Immunology, Charité Campus Mitte, Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, 10117 Berlin, Germany. |
| 期刊 | Current oncology (Toronto, Ont.) |
| SCI 分区 | Q2 |
| IF | 3.6 |
| 研究类型 | 临床研究 · 临床 |
| 所属专科 | 耳科 |
中文摘要
全反式维甲酸（ATRA）联合三氧化二砷（ATO）是急性早幼粒细胞白血病（APL）的标准治疗，包括高危疾病。继发性颅内高压（SIH），历史上称为假性脑瘤（PTC），是维甲酸暴露已知但少见且可能限制治疗的并发症，主要报道于儿童和年轻成人患者，且最常发生于诱导期。我们报告一名31岁男性高危微颗粒型APL患者，在首个ATRA/ATO巩固周期中诊断出SIH。临床过程包括进行性头痛、眼痛、搏动性耳鸣、聚焦困难、视力下降和复视。眼科评估显示双侧视乳头水肿伴明显的视乳头周围视网膜神经纤维层增厚，尽管中心视力保留。磁共振成像显示双侧视神经鞘扩张、视神经头突出、轻度垂体变扁和双侧横窦狭窄。腰椎穿刺显示开放压力为32 cm H2O，脑脊液其他方面无异常；移除约30 mL后症状立即改善。管理包括暂时中断ATRA、治疗性腰椎穿刺、乙酰唑胺以及个体化ATRA再挑战和剂量滴定。反复尝试剂量递增再现了症状，ATRA最终维持在最大耐受剂量30 mg/m2/天，同时继续全剂量ATO。首个巩固周期后达到分子学缓解，并在18个月随访时持续维持。本病例强调了在整个治疗过程中识别ATRA相关SIH的重要性，并说明了一种在标准剂量治疗受神经毒性限制时保留ATRA暴露的实用策略。然而，不能从单个病例推断减量ATRA的肿瘤学等效性。
英文摘要
All-trans retinoic acid (ATRA) combined with arsenic trioxide (ATO) is the standard of care for acute promyelocytic leukemia (APL), including high-risk disease. Secondary intracranial hypertension (SIH), historically termed pseudotumor cerebri (PTC), is a recognized but uncommon and potentially therapy-limiting complication of retinoid exposure, reported predominantly in pediatric and young-adult patients and most frequently during induction. We report a 31-year-old man with high-risk microgranular APL in whom SIH was diagnosed during the first ATRA/ATO consolidation cycle. The clinical course included progressive headache, ocular pain, pulsatile tinnitus, focusing difficulties, visual deterioration, and diplopia. Ophthalmological assessment demonstrated bilateral papilledema with marked peripapillary retinal nerve fiber layer thickening despite preserved central visual acuity. Magnetic resonance imaging showed bilateral optic nerve sheath distension, prominent optic nerve heads, mild pituitary flattening, and bilateral transverse sinus stenosis. Lumbar puncture demonstrated an opening pressure of 32 cm H2O with otherwise unremarkable cerebrospinal fluid; removal of approximately 30 mL resulted in immediate symptomatic improvement. Management consisted of temporary ATRA interruption, therapeutic lumbar punctures, acetazolamide, and individualized ATRA rechallenge and dose titration. Repeated attempts at dose escalation reproduced symptoms, and ATRA was ultimately maintained at a maximally tolerated dose of 30 mg/m2/day while full-dose ATO was continued. Molecular remission was achieved after the first consolidation cycle and remains sustained at 18-month follow-up. This case highlights the importance of recognizing ATRA-associated SIH throughout the treatment course and illustrates a pragmatic strategy for preserving ATRA exposure when standard-dose treatment is limited by neurological toxicity. However, the oncological equivalence of reduced-dose ATRA cannot be inferred from a single case.