Salvia S A, Amore M A, Papendieck C M
Phlebology and Lymphology Unit, Cardiovascular Surgery Division, Central Military Hospital, Buenos Aires, Argentina.
Lymphology Unit, University Hospital, Favaloro Foundation, Buenos Aires, Argentina.
Lymphology. 2021;54(2):106-111.
Microcystic lymphatic malformations as described in the international literature form a subgroup of low-flow congenital vascular malformations (VM) resulting from irregular embryological development. Microcystic lesions normally manifest as an accumulation of lymph- and blood-filled vesicles that, when externalized, cause skin maceration with consequent pain and potential infection resulting in the impairment of the patient's quality of life. There is no consensus on a standardized algorithm nor clear guidelines for successful treatment of this type of lymphatic malformation, and treatment options employed often result in ambivalent and transient outcomes with a high rate of recurrence. The topical formulation of tacrolimus is a well-known FDAapproved anti-T cell agent that was recently identified as a potent activator of ALK1, which is involved in several processes and functions including angiogenesis. We investigated if topical administration of tacrolimus may be an effective therapy for directly targeting cutaneous microcystic lymphatic malformations as a complement to systemic treatment. The study enrolled four patients with cutaneous microcystic lymphatic malformations: three male (ages: 13,15,18) and one female (age: 30). Two of the patients presented lesions on their backs, one patient on the left hand and one on the left lower limb. All four patients received treatment with topical tacrolimus 0.1% twice a day for 10 weeks on a previously selected area for application. Weekly clinical follow-ups were conducted along with close physician-patient contact. All patients displayed a satisfactory response after treatment. Lymphorrhea and bleeding were stopped in all cases and the esthetic aspect of lesions improved in two patients. To date, all patients presented no clinically significant changes to the size or extension of the lesion. Topical tacrolimus treatment is a promising and reasonable option for microcystic lymphatic malformations. Our results encourage further exploration in larger populations with the consideration that it is a safe and effective alternative or complementary therapy to systemic treatment.
国际文献中描述的微囊性淋巴管畸形是由胚胎发育不规则导致的低流量先天性血管畸形（VM）的一个亚组。微囊性病变通常表现为充满淋巴液和血液的小泡积聚，这些小泡外露时会导致皮肤浸渍，进而引起疼痛和潜在感染，导致患者生活质量受损。对于这类淋巴管畸形的成功治疗，目前尚无关于标准化算法的共识，也没有明确的指导方针，所采用的治疗方案往往导致矛盾和短暂的结果，复发率很高。他克莫司的局部制剂是一种著名的经美国食品药品监督管理局（FDA）批准的抗T细胞药物，最近被确定为ALK1的强效激活剂，ALK1参与包括血管生成在内的多个过程和功能。我们研究了局部应用他克莫司是否可能作为全身治疗的补充，成为直接针对皮肤微囊性淋巴管畸形的有效疗法。该研究招募了4名患有皮肤微囊性淋巴管畸形的患者：3名男性（年龄分别为13岁、15岁、18岁）和1名女性（年龄为30岁）。其中2名患者的病变位于背部，1名患者在左手，1名在左下肢。所有4名患者在预先选定的应用区域每天两次外用0.1%他克莫司，持续10周。每周进行临床随访，并保持医生与患者的密切联系。所有患者治疗后均显示出满意的反应。所有病例的淋巴漏和出血均停止，2例患者病变的美观方面得到改善。迄今为止，所有患者病变的大小或范围均未出现临床上的显著变化。局部应用他克莫司治疗是微囊性淋巴管畸形一种有前景且合理的选择。我们的结果鼓励在更大规模人群中进一步探索，因为考虑到它是全身治疗的一种安全有效的替代或补充疗法。
