ABSTRACT
BACKGROUND: Immune thrombocytopenia (ITP) is an autoimmune disease associated with isolated thrombocytopenia, which is caused by an imbalance between platelet production and platelet destruction. Petechial and mucous membrane hemorrhages are characteristic of ITP, but life-threatening bleeding rarely occurs. Depending on the bleeding symptoms, ITP can be treated with glucocorticoids (GC), intravenous immunoglobulins (IVIG), or in severe cases, platelet transfusions. Mild bleeding does not necessarily require therapy. Using the German Surveillance Unit for rare Pediatric Diseases (ESPED) we conducted a prospective survey on ITP patients in all German Children's Hospitals between September 2018 and August 2019. We collected data on ITP, including the clinical course, therapy implementation recommendations (according to the Association of German Scientific Medical Societies guidelines), outcome, and influence of treatment regimens depending on the treating physician´s experience with ITP patients. RESULTS: Of the 287 recorded cases of children with ITP, 268 questionnaires were sent to the authors. Two hundred seventeen of the questionnaires fulfilled the inclusion criteria. ITP affected boys and girls similarly, and the median age of manifestation was 3.5 years. The main reasons for hospitalization were thrombocytopenia, bleeding signs, hematomas, and/or petechiae. Bleeding scores were ≤ 3 in 96% of children, which corresponded to a low-to-moderately low risk of bleeding. No life-threatening bleeding was documented. The most common therapies were IVIG (n = 59), GC (n = 33), or a combination of these (n = 17). Blood products (i.e., red blood cells, platelet concentrate, and fresh frozen plasma) were given to 13 patients. Compared to the established guidelines, 67 patients were over-treated, and 2 patients were under-treated. CONCLUSIONS: Adherence to German ITP treatment guidelines is currently limited. To improve patient safety and medical care, better medical training and dissemination of the guidelines are required in line with targeted analyses of patients with serious bleeding events to identify potential risk constellations.
ABSTRACT
OBJECTIVES: To report validation data for the Pupillographic Sleepiness Test (PST) in children and adolescents, evaluate its applicability for diagnosing excessive daytime sleepiness and its relationship to sleepiness-associated outcomes. METHODS: A cross-sectional diagnostic test accuracy study was performed. Patients underwent three PST at 9 a.m. (T1), 11 a.m. (T2) and 1 p.m. (T3) plus the Multiple Sleep Latency Test (MSLT) on a single day. Additionally, two neurocognitive tests were performed and three questionnaires about quality of life, sleep-related self-efficacy and behavioural aspects completed. Gender-stratified z-values of the natural logarithm of the Pupillary Unrest Index (z-lnPUI) were correlated to Sleep Latency (SL) and Mean Sleep Latency (MSL) and to variables of neurocognitive tests and questionnaires using Spearman's rank correlation. Cut-off values were determined by receiver operating characteristic (ROC) analysis. RESULTS: 47 patients were recruited (median 10.6 years, range 6-18). Correlation between z-lnPUI and SL was rT1 = -0.373 (p = 0.011); rT2 = -0.320 (p = 0.028) and rT3 = -0.336 (p = 0.022). Correlation between z-lnPUI and MSL was rT1 = -0.338 (p = 0.020); rT2 = -0.202 (p = 0.173); rT3 = -0.117 (p = 0.433). ROC analysis showed an area under the curve of 90.7% and PUI cut-off values of 12.6 mm/min (boys) and 11.6 mm/min (girls). There were moderate correlations between z-lnPUIT1 and reaction time and omission errors in neurocognitive tests (r = 0.394, p = 0.007 and 0.391, p = 0.008). CONCLUSIONS: We found satisfactory correlations between PST and MSLT results. The z-lnPUIT1 was related to MSL and objective measures of attention ability. Given this accuracy, the PST may be used as a screening tool for evaluating daytime sleepiness in children and adolescents. Corresponding gender-related reference values are now available.
