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Clinical trials of sickle cell disease (SCD) typically focus on healthcare practices for painy vascular acuity (vaso-occlusive crises, VOC).
SCD patients with painful VOC is caused by sickle cells blocking blood vessels and cutting off the blood supply to the corresponding organ tissue, which can last for several days.
pain VOC can be treated with painkillers or may relieve itself, but serious complications such as infection, stroke, etc. may also occur.
Unfortunately, there are no objective, quantifiable pain biomarkers present, pain is not unique to VOCs, plus different patient visit rates are also different, VOC patients' prognosis has not been completely clear.
Pittman et al. conducted a non-interventional, longitudinal, six-month study aimed at developing tools to identify whether VOCs occur in patients with or without SCD.
SCD patients who had studied the design wore activity recorders to record sleep and activity.
SCD patients use electronic patient reporting prognostic (ePRO) tools to document pain, medication, fatigue, and daily function.
self-reporting (VOC day) when experiencing VOC pain.
biomarkers (non-VOCs) are collected every 3 weeks.
blood collection after self-reporting VOC.
the VOC events reported by patients and their duration, the study recruited 37 SCD patients, 35 of whom completed the study.
reported 114 VOC events and 346 VOC days, of which 62.3% and 78.3% were self-treated at home.
ePRO and activity recorders record indicators of pain, function, fatigue, activity, and sleep; Changes in biomarkers at
VOC were significantly different from non-VOC baseline values collected on VOC day, including leukocyte-plate plate aggregation, microflow-controlled blood cell adhesion, leukocyte mesokine-6, C-reactive protein, leukocyte mesotyn-10, tumor necrosis factor-α and clotting enzyme-anticoagulantase.
In summary, the study showed that in SCD-related clinical trials, patient-reported VOC days were used as a potential endpoint to accurately monitor out-of-hospital pain;