Long-term safety and efficacy of autologous platelet lysate drops for treatment of ocular GvHD
Long-term safety and efficacy of autologous platelet lysate drops for treatment of ocular GvHD
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DOI：
10.1038/bmt.2016.221
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发表时间：
2017-01-01
影响因子：
4.8
通讯作者：
Antoniazzi, E.
中科院分区：
文献类型：
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作者：
Pezzotta, S.;Del Fante, C.;Antoniazzi, E.
目前的眼部GvHD（oGvHD）治疗是次优的。我们研究了局部常规治疗难治性oGvHD干眼综合征（DES）评分2-3的患者长期连续使用自体血小板裂解物（PL）滴剂治疗的安全性和有效性。每隔6个月进行眼科评价。使用青光眼症状量表（GSS）评估症状。当患者在6个月访视时显示国立卫生研究院眼部评分较基线降低至少一个等级时，将其定义为“应答者”。31例患者入选，16例（51%）完成了36个月的随访（范围6.5-72.7）。6个月时，所有患者均被归类为应答者：中位GSS症状评分从70降至41（36个月时为33），中位GSS功能评分从68降至46（36个月时为33）（均P < 0.001）。6个月后，中位泪膜破裂时间从3秒改善至6秒，并随时间推移而保持。所有体征在6个月和36个月时均改善（临床和统计学显著性）。未发生严重不良事件。PL滴剂的长期治疗对oGvHD是安全有效的，并且可以是从oGvHD的初始阶段开始的有效治疗选择，以防止永久性眼部损伤并改善生活质量。
Current ocular GvHD (oGvHD) treatments are suboptimal. We investigated the safety and efficacy of long-term continuous treatment with autologous platelet lysate (PL) drops in patients with oGvHD Dry Eye Syndrome (DES) score 2-3 refractory to topical conventional therapy. Ophthalmic evaluation was performed at 6 month intervals. Symptoms were assessed using the Glaucoma Symptom Scale (GSS). Patients were defined 'responders' when showing a reduction at least one grade on National Institutes of Health Eye Score from baseline at the 6 month visit. Thirty-one patients were included, and 16 (51%) completed 36 months of follow-up (range 6.5-72.7). At 6 months all patients were classified as responders: median GSS symptom score decreased from 70 to 41 (33 at 36 months), median GSS function score reduced from 68 to 46 (33 at 36 months) (all P < 0.001). Median Tear Break Up Time improved from 3 to 6 s after 6 months and was maintained over time. All signs improved at 6 and 36 months (clinical and statistical significance). No severe adverse events occurred. Long-term treatment with PL drops is secure and effective for oGvHD and can be an efficient therapy option from initial stages of oGvHD to prevent permanent ocular impairment and improving quality of life.