Burkert Sanne, Peters Lynn, Bloehdorn Johannes, Grüner Beate
Department of Internal Medicine III, Division for Infectious Diseases, Ulm University Hospital, 89081 Ulm, Germany.
Pathogens. 2022 Mar 9;11(3):333. doi: 10.3390/pathogens11030333.
Benzimidazoles are the only approved drugs for the treatment of inoperable human alveolar echinococcosis but may be limited due to intolerance or, rarely, ineffectiveness. A medical second-line or salvage therapy is not available, though it is urgently needed. We report long-term follow-up data from 14 patients who underwent salvage therapy with repurposed drugs with cumulatively 53.25 patient-years. Treatment response was evaluated by both clinical outcome and image studies, preferably PET/CT. Eleven patients received amphotericin B, and 70% of evaluable cases showed some positive treatment response, but side effects often limited therapy. Five patients received nitazoxanide, of which two showed clear progression but one achieved a lasting stable disease. One patient was treated with mefloquine combination therapy in advanced disease, and overall, a positive treatment response could not be assessed. Furthermore, we report on one patient receiving pembrolizumab for a concomitant malignancy, which did not result in a reduction of echinococcal manifestation. In summary, current options of salvage therapy can sometimes induce persistent disease control, although with potentially significant side effects and high treatment costs, and mortality remains high. No clear recommendation for a salvage therapy can be given; treatment remains highly experimental, and non-pharmaceutical interventions have to be considered.
苯并咪唑类药物是唯一被批准用于治疗无法手术的人类肺泡型棘球蚴病的药物，但可能因不耐受或极少数情况下无效而受到限制。目前尚无二线或挽救治疗方案，尽管这一需求极为迫切。我们报告了14例接受药物重新利用进行挽救治疗患者的长期随访数据，累计随访时间达53.25患者年。通过临床结果和影像学检查（最好是PET/CT）评估治疗反应。11例患者接受了两性霉素B治疗，70%的可评估病例显示出一定的积极治疗反应，但副作用常常限制了治疗。5例患者接受了硝唑尼特治疗，其中2例病情明显进展，但1例病情持续稳定。1例晚期疾病患者接受了甲氟喹联合治疗，总体而言，无法评估其治疗反应是否积极。此外，我们报告了1例因合并恶性肿瘤接受帕博利珠单抗治疗的患者，该治疗并未使棘球蚴病表现减轻。总之，目前的挽救治疗方案有时可实现疾病的持续控制，尽管可能伴有显著副作用和高昂治疗成本，且死亡率仍然很高。无法给出明确的挽救治疗推荐；治疗仍处于高度试验阶段，必须考虑非药物干预措施。
