Blood and Tissue Bank (BST), Barcelona, Spain.
Transfusional Medicine Research Group, Vall d'Hebron Research Institute (VHIR), Barcelona, Spain.
Blood Transfus. 2021 Jul;19(4):347-356. doi: 10.2450/2020.0130-20. Epub 2020 Oct 9.
Current treatments for several corneal lesions show limited efficacy. Here we report the clinical evaluation of the efficacy of a novel eye drop preparation produced in a public cord blood (CB) bank.
In a multicentre, retrospective, consecutive case study we evaluated 33 patients (46 eyes) unresponsive to conventional treatments who required urgent intervention. The patients were given allogeneic eye drops obtained from cord blood platelet lysate (CBED) to treat severe ocular surface lesions under a compassionate use protocol. The CBED were prepared from CB units donated for haematopoietic stem cell transplantation that did not contain the minimum stem cell dose required for this use. Patients were grouped by acute conditions (neurotrophic ulcers: group I; other corneal ulcers: group II; corneal burns: group III), and chronic conditions (ocular graft-versus-host disease: group IV; severe dry eye syndrome: group V). The patients received one or two drops of the product to the affected eye four to six times per day for 19 days. A further 19-day cycle of treatment could be repeated according to the initial clinical response.
Patients received a median of 19 CBED vials (interquartile range 19-57, range 19-442) to complete the therapy. Group I-II-III patients showed full and partial ulcer recovery in 25 (78%) and six (19%) eyes respectively. One eye (3%) did not respond to treatment. For groups IV-V improvement was reported for 12 (85%) eyes and lesions worsened on treatment in both eyes (15%) of one patient. No severe adverse events were directly attributed to CBED.
Promptly available CBED resulted in a well-tolerated allogeneic treatment that showed evidence of efficacy in this cohort of patients. These positive results support further studies on CBED from platelet lysate as a novel product of CB banks. A prospective clinical trial in neurotrophic keratitis (NCT03084861) is ongoing to confirm these preliminary data.
目前，几种角膜病变的治疗方法疗效有限。在这里，我们报告了一种新型滴眼液制剂的临床疗效评估，该制剂由公共脐带血（CB）库生产。
在一项多中心、回顾性、连续病例研究中，我们评估了 33 名（46 只眼）对常规治疗无反应、需要紧急干预的患者。根据同情使用方案，这些患者使用来自脐带血血小板裂解液（CBED）的同种异体滴眼液治疗严重的眼表病变。CBED 是从用于造血干细胞移植的 CB 单位中制备的，这些单位不包含用于该用途的最低干细胞剂量。根据急性情况（神经营养性溃疡：I 组；其他角膜溃疡：II 组；角膜烧伤：III 组）和慢性情况（眼移植物抗宿主病：IV 组；严重干燥性眼综合征：V 组）对患者进行分组。患者每天向受影响的眼睛滴 1 到 2 滴产品，每天 4 到 6 次，共 19 天。根据初始临床反应，可以重复进行为期 19 天的治疗周期。
患者接受中位数为 19 瓶 CBED（四分位间距 19-57，范围 19-442）以完成治疗。I 组-II 组-III 组患者分别有 25 只（78%）和 6 只（19%）眼睛的溃疡完全和部分愈合。1 只眼睛（3%）对治疗无反应。对于 IV 组-V 组，12 只（85%）眼睛的病变得到改善，1 名患者的 2 只眼睛（15%）的病变在治疗过程中恶化。没有直接归因于 CBED 的严重不良事件。
迅速获得的 CBED 产生了一种耐受性良好的同种异体治疗方法，在该患者队列中显示出疗效的证据。这些积极的结果支持进一步研究 CB 血小板裂解液作为 CB 库的新型产品。一项关于神经营养性角膜炎的前瞻性临床试验（NCT03084861）正在进行中，以确认这些初步数据。
