Quantification of intramuscular fat in patients with late-onset Pompe disease by conventional magnetic resonance imaging for the long-term follow-up of enzyme replacement therapy

OBJECTIVE: The objective of this study was to evaluate a quantitative method based on conventional T1-weighted magnetic resonance (MR) imaging to assess fatty muscular degeneration in patients with late-onset Pompe disease and to compare it with semi-quantitative visual evaluation (the Mercuri score). In addition, a long-term retrospective data analysis was performed to evaluate treatment response to enzyme replacement therapy with alglucosidase alfa. METHODS: MR images of the lumbar spine were acquired in 41 patients diagnosed with late-onset Pompe disease from 2006 through 2015. Two independent readers retrospectively evaluated fatty degeneration of the psoas and paraspinal muscles by applying the Mercuri score. Quantitative semi-automated muscle and fat tissue separation was performed, and inter-observer agreement and correlations with clinical parameters were assessed. Follow-up examinations were performed in 13 patients treated with alglucosidase alfa after a median of 39 months; in 7/13 patients, an additional follow-up examination was completed after a median of 63 months. RESULTS: Inter-observer agreement was high. Measurements derived from the quantitative method correlated well with Medical Research Council scores of muscle strength, with moderate correlations found for the 6-minute walk test, the 4-step stair climb test, and spirometry in the supine position. A significant increase in the MR-derived fat fraction of the psoas muscle was found between baseline and follow-up 1 (P = 0.016), as was a significant decrease in the performance on the 6-minute walk test (P = 0.006) and 4-step stair climb test (P = 0.034), as well as plasma creatine kinase (P = 0.016). No statistically significant difference in clinical or MR-derived parameters was found between follow-up 1 and follow-up 2. CONCLUSIONS: Quantification of fatty muscle degeneration using the semi-automated method can provide a more detailed overview of disease progression than semi-quantitative Mercuri scoring. MR-derived data correlated with clinical symptoms and patient exercise capacity. After an initial worsening, the fat fraction of the psoas muscle and performance on the 6-minute walk test stayed constant during long-term follow-up under enzyme replacement therapy

Validation of the SNACOR clinical scoring system after transarterial chemoembolisation in patients with hepatocellular carcinoma

Background Transarterial chemoembolisation is the standard of care for intermediate stage (BCLC B) hepatocellular carcinoma, but it is challenging to decide when to repeat or stop treatment. Here we performed the first external validation of the SNACOR (tumour Size and Number, baseline Alpha-fetoprotein, Child-Pugh and Objective radiological Response) risk prediction model. Methods A total of 1030 patients with hepatocellular carcinoma underwent transarterial chemoembolisation at our tertiary referral centre from January 2000 to December 2016. We determined the following variables that were needed to calculate the SNACOR at baseline: tumour size and number, alpha-fetoprotein level, Child-Pugh class, and objective radiological response after the first transarterial chemoembolisation. Overall survival, time-dependent area under receiver-operating characteristic curves, Harrell’s C-index, and the integrated Brier score were calculated to assess predictive ability. Finally, multivariate analysis was performed to identify independent predictors of survival. Results The study included 268 patients. Low, intermediate, and high SNACOR scores predicted a median survival of 31.5, 19.9, and 9.2 months, respectively. The areas under the receiver-operating characteristic curve for overall survival were 0.641, 0.633, and 0.609 at 1, 3, and 6 years, respectively. Harrell’s C-index was 0.59, and the integrated Brier Score was 0.175. Independent predictors of survival included tumour size (P < 0.001), baseline alpha-fetoprotein level (P < 0.001) and Child-Pugh class (P < 0.004). Objective radiological response (P = 0.821) and tumour number (P = 0.127) were not additional independent predictors of survival. Conclusions The SNACOR risk prediction model can be used to identify patients with a dismal prognosis after the first transarterial chemoembolisation who are unlikely to benefit from further transarterial chemoembolisation. However, Harrell’s C-index showed only moderate performance. Accordingly, this risk prediction model can only serve as one of several components used to make the decision about whether to repeat treatment

Planning and Development of Social Services for Persons with Disabilities

Soziale Dienste zur Unterstützung von Menschen mit Behinderungen haben sich in den letzten Jahren dynamisch entwickelt und unterliegen auch aktuell einem erheblichen Veränderungsdruck. Die Forschungsarbeiten, die in diesem Band versammelt sind, haben die Entwicklung hin zu einer inklusionsorientierten Unterstützung in zahlreichen Projekten auf unterschiedlichen Ebenen aktiv begleitet.Social services to support persons with disabilities have developed dynamically in recent years and are currently subject to considerable pressure to change. The research work collected in this volume has actively accompanied the development towards inclusion-oriented support in numerous projects at different levels

Digitale Teilhabe

Das Themenheft Digitale Teilhabe beschäftigt sich zentral mit den Potentialen der Nutzung der neuen Informations- und Kommunikationstechnologien durch benachteiligte Menschen. Im Leitartikel wird der Versuch unternommen, mögliche theoretische Anknüpfungspunkte und Forschungsfragen für weitere Studien- und Forschungsarbeiten in dem noch jungen Themenfeld der Digitalen Teilhabe zu identifizieren. Hierzu wird zunächst das zugrunde liegende Verständnis von Behinderung/Benachteiligung diskutiert und inklusive (Medien-)Bildung als Teil der Persönlichkeitsbildung skizziert. In verschiedenen Diskursen bzw. Disziplinen werden dann theoretische Anknüpfungspunkte für weitere Forschungsarbeiten benannt. Die Idee für das Themenheft ist im Rahmen des Projekts "Begleitforschung im PIKSL-Labor" des Zentrums für Planung und Evaluation Sozialer Dienste der Uni Siegen (ZPE) entstanden. Das PIKSL-Projekt zielt darauf ab, Menschen mit Behinderungen moderne Kommunikationstechnologien zugänglich zu machen, um ihnen Teilhabemöglichkeiten zu erleichtern und zugleich die personale Abhängigkeit von professioneller Unterstützung zu reduzieren. Der inter- und transdisziplinäre Ansatz von PIKSL wird durch die Vielfalt der Artikel in dem Heft deutlich: Digitale Teilhabe wird nicht alleine aus (medien-)pädagogischer bzw. sozialwissenschaftlicher Perspektive betrachtet. Die Besonderheit liegt in der Kooperation unterschiedlicher Disziplinen wie Soziale Arbeit, Kunst und Webdesign

Reproducibility and comparison of oxygen-enhanced T-1 quantification in COPD and asthma patients

T1 maps have been shown to yield useful diagnostic information on lung function in patients with chronic obstructive pulmonary disease (COPD) and asthma, both for native T1 and Delta T1, the relative reduction while breathing pure oxygen. As parameter quantification is particularly interesting for longitudinal studies, the purpose of this work was both to examine the reproducibility of lung T1 mapping and to compare T1 found in COPD and asthma patients using IRSnapShotFLASH embedded in a full MRI protocol. 12 asthma and 12 COPD patients (site 1) and further 15 COPD patients (site 2) were examined on two consecutive days. In each patient, T1 maps were acquired in 8 single breath-hold slices, breathing first room air, then pure oxygen. Maps were partitioned into 12 regions each to calculate average values. In asthma patients, the average T-1,T-RA = 1206ms (room air) was reduced to T-1,T-O2 = 1141ms under oxygen conditions (Delta T1 = 5.3%, p < 5.10(-4)), while in COPD patients both native T-1,T-RA = 1125ms was significantly shorter (p < 10(-3)) and the relative reduction to T-1,T-O2 = 1081ms on average Delta T1 = 4.2%(p < 10(-5)). On the second day, with T-1,T-RA = 1186ms in asthma and T-1,T-RA = 1097ms in COPD, observed values were slightly shorter on average in all patient groups. Delta T1 reduction was the least repeatable parameter and varied from day to day by up to 23% in individual asthma and 30% in COPD patients. While for both patient groups T1 was below the values reported for healthy subjects, the T1 and Delta T1 found in asthmatics lies between that of the COPD group and reported values for healthy subjects, suggesting a higher blood volume fraction and better ventilation. However, it could be demonstrated that lung T1 quantification is subject to notable inter-examination variability, which here can be attributed both to remaining contrast agent from the previous day and the increased dependency of lung T1 on perfusion and thus current lung state

Comparison of 1.0 M gadobutrol and 0.5 M gadopentate dimeglumine-enhanced MRI in 471 patients with known or suspected renal lesions: Results of a multicenter, single-blind, interindividual, randomized clinical phase III trial

The purpose of this phase III clinical trial was to compare two different extracellular contrast agents, 1.0 M gadobutrol and 0.5 M gadopentate dimeglumine, for magnetic resonance imaging (MRI) in patients with known or suspected focal renal lesions. Using a multicenter, single-blind, interindividual, randomized study design, both contrast agents were compared in a total of 471 patients regarding their diagnostic accuracy, sensitivity, and specificity to correctly classify focal lesions of the kidney. To test for noninferiority the diagnostic accuracy rates for both contrast agents were compared with CT results based on a blinded reading. The average diagnostic accuracy across the three blinded readers ('average reader') was 83.7% for gadobutrol and 87.3% for gadopentate dimeglumine. The increase in accuracy from precontrast to combined precontrast and postcontrast MRI was 8.0% for gadobutrol and 6.9% for gadopentate dimeglumine. Sensitivity of the average reader was 85.2% for gadobutrol and 88.7% for gadopentate dimeglumine. Specificity of the average reader was 82.1% for gadobutrol and 86.1% for gadopentate dimeglumine. In conclusion, this study documents evidence for the noninferiority of a single i.v. bolus injection of 1.0 M gadobutrol compared with 0.5 M gadopentate dimeglumine in the diagnostic assessment of renal lesions with CE-MRI