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The Association of Placental Grading with Perinatal Outcomes: A Systematic Review and Meta-Analysis
Get PDFObjective: Premature placental calcification (PPC) has been implicated in adverse
perinatal outcomes, yet its clinical significance remains controversial. This meta-analysis
aimed to quantitatively synthesize current data on the association between PPC, defined
as grade 3 placental calcification before 36+6 weeks of gestation and adverse perinatal
outcomes. Data Sources: A systematic search was conducted in MEDLINE, Scopus and
The Cochrane Library from inception until 11 March 2025, to identify eligible studies.
Study Eligibility Criteria: Observational studies including singleton pregnancies with PPC
diagnosed via ultrasonography between 28+0 and 36+6 weeks of gestation and comparing
them with pregnancies with Grannum grade 0, 1, or 2 placentas were considered eligible.
Methods: Study quality was assessed using the Newcastle−Ottawa Scale, and the risk of
bias was evaluated with the Quality In Prognosis Studies tool. The primary outcomes were
small-for-gestational-age (SGA) neonates and preeclampsia. Heterogeneity was assessed
using Cochran’s Q test and the I2
statistic. Meta-analyses were conducted using a randomeffects model, with outcomes reported as relative risk (RR) or mean difference (MD) with
95% confidence intervals (CIs). Results: In total, nine cohort studies were included. PPC
was associated with an increased risk of SGA (RR, 1.99; 95% CI, 1.46−2.70), preeclampsia
(RR, 5.27; 95% CI, 2.24−12.40), fetal growth restriction (RR, 2.31; 95% CI, 1.30−4.09),
preterm delivery (RR, 2.11; 95% CI, 1.00−4.45), suspected fetal hypoxia (RR, 1.71; 95% CI,
1.13–2.56), low 5 min Apgar score (RR, 2.28; 95% CI, 1.50−3.44) and neonatal intensive
care unit admission (RR, 1.80; 95% CI, 1.02−3.18). No significant associations were found
with fetal or neonatal death (RR, 2.75; 95% CI, 0.87−8.71), cesarean delivery (RR, 1.26;
95% CI, 0.90−1.78), gestational diabetes mellitus (RR, 1.17; 95% CI, 0.81−1.70), neonatal
resuscitation (RR, 1.04; 95% CI, 0.92−1.16), birthweight (MD, −187.46 g; 95% CI, −413.14 to
+38.21), or gestational age at birth (MD, −0.62 weeks; 95% CI, −1.36 to +0.11). A sensitivity
analysis excluding high-risk-of-bias studies yielded consistent results. Conclusions: PPC
is associated with several adverse perinatal outcomes, including SGA and preeclampsia.
While the clinical significance of placental grading has remained limited in recent years,
this study has shown that PPC may serve as an early indicator of placental insufficiency,
warranting enhanced fetal surveillance and risk assessment in affected pregnancies. Further
research is needed to refine its prognostic utility and integration into obstetric practice
Expanding the myxobacterial biosynthetic potential using advanced analytical and chemical methods
No full textMyxobacteria are a rich source of structurally diverse and bioactive natural products. This thesis explores the expansion of the myxobacterial metabolome through mutasynthetic studies of the potent antibiotic myxopyronin A, employing a myxobacterial heterologous producer. These efforts led to the generation of non-natural derivatives, including a tri fluorinated congener of myxopyronin A that exhibited superior pharmacokinetic properties while maintaining bioactivity. Additionally, the previously unknown biosynthetic origin of a class of chlorinated natural products was elucidated in the model organism Myxococcus xanthus DK1622. Using mo lecular biology methods and organic chemistry, the study identified the involvement of a halogenase encoded genetically distant from the precursr’s biosynthetic gene cluster (BGC), providing new insights into the biosynthetic processes of one of the most extensively stud ied myxobacterium. Throughout this work, state-of-the-art analytical chemistry techniques were applied, with a focus on high-resolution mass spectrometry (HRMS), ultra-high-performance liquid chromatography (UHPLC), ion mobility spectrometry, and MS² spectrometry. Notably, a comprehensive evaluation of novel analytical methodologies was conducted, including the recently developed matrix-assisted laser desorption ionization with post-ionization (MALDI-2) technique, applied in an untargeted metabolomics workflow following a One Strain Many Compounds (OSMAC) approach.Myxobakterien sind eine reiche Quelle strukturell vielfältiger und bioaktiver Naturstoffe. Diese Dissertation untersucht die Erweiterung des myxobakteriellen Metaboloms durch mutasynthetische Studien des potenten Antibiotikums Myxopyronin A unter Verwendung eines myxobakteriellen heterologen Produzenten, mit dem Resultat der Erzeugung nicht natürlicher Derivate, darunter eines trifluorierten Derivates, welches verbesserte pharma kokinetische Eigenschaften unter Erhalt der Bioaktivität aufwies. Darüber hinaus wurde der bislang unbekannte biosynthetische Ursprung einer Klasse chlo rierter Naturstoffe im intensiv untersuchten Modellorganismus Myxococcus xanthus DK1622 aufgeklärt. Mithilfe molekularbiologischer Methoden und organischer Chemie konnte die Beteiligung einer Halogenase identifiziert werden, die genetisch weit entfernt vom Biosynthese-Gencluster (BGC) des Vorläufermoleküls kodiert ist. Im Verlauf dieser Arbeit kamen hochmoderne Techniken der analytischen Chemie zum Einsatz, mit einem Schwerpunkt auf hochauflösender Massenspektrometrie (HRMS), Ult rahochleistungsflüssigkeitschromatographie (UHPLC), Ionenmobilitätsspektrometrie und MS²-Spektrometrie. Besonders hervorzuheben ist die umfassende Evaluierung neuer ana lytischer Methoden, darunter die kürzlich entwickelte Matrix-unterstützte Laser-Desorpti ons-Ionisation mit Nachionisierung (MALDI-2), die in einem ungerichteten Metabolom Workflow nach dem One Strain Many Compounds (OSMAC)-Ansatz angewandt wurde
Red blood cell shape stability & deformability In single-cell capillary flow: Effects of confinement, age and membrane rigidity
No full textThe thesis studies red blood cell (RBC) morphology and deformability under microfluidic conditions to reveal their behavior in the microcirculation. Using novel experiments, we analyze how RBC properties, channel geometry, and flow interact. Rectangular channels with height near the RBC diameter and confinement ratios above 0.9 favor distinct croissant and slipper shapes, correlating shear rate with cell elongation. Less confined channels yield varied unstable shapes. We also examine effects of RBC age and membrane rigidity by fractionating cells and altering membranes chemically, finding a reduced formation of stable asymmetric slippers at high flow in aged or stiffened cells. Older cells more often form symmetric croissants, a pattern suppressed by membrane stiffening. Finally, we introduce a deformation index (DI) from compression-expansion tests to quantify deformability and study influences of density and rigidity. Our results enhance understanding of RBC dynamics in confined flows, aiding biomicrofluidic design and hematological diagnostics, and advancing insight into microcirculatory function and disease.Diese Arbeit untersucht die Morphologie und Verformbarkeit roter Blutkörperchen (Erythrozyten) unter mikrofluidischen Bedingungen, um deren Verhalten im Mikrozirkulationssystem zu verstehen. Mit neuartigen Experimenten analysieren wir das Zusammenspiel von Zell-Eigenschaften, Kanalgeometrie und Strömungsdynamik. Rechteckige Kanäle mit einer Höhe nahe dem Zell-Durchmesser und einer Konfektionsrate über 0,9 begünstigen ausgeprägte croissant- und slipperförmige Formen, wobei eine starke Korrelation zwischen Scherrate und Zellverlängerung besteht. Geringere Konfinierung führt zu vielfältigen instabilen Formen. Außerdem werden Alter und Membransteifigkeit der Zellen durch Fraktionierung und chemische Modifikation untersucht, wobei ¨altere oder versteifte Zellen weniger stabile asymmetrische slipper formen. Ältere Zellen zeigen verstärkt symmetrische croissants, was durch Membranversteifung unterdrückt wird. Abschließend wird mit einem neu eingeführten Deformationsindex (DI) die Verformbarkeit durch Kompressions- und Expansionsversuche quantifiziert. Die Resultate verbessern das Verst¨andnis der Erythrozyten-Dynamik in engen Strömungen und unterstützen die Entwicklung mikrofluidischer Geräte sowie hämatologischer Diagnostik
Beidseitige Papillenschwellung und visuelle Symptome als Präsentation eines Astrozytoms WHO Grad 4: ein interdisziplinärer Diagnoseweg
Get PDFReversible tuning of membrane sterol levels by cyclodextrin in a dialysis setting
No full textLarge unilamellar vesicles are popular membrane models for studying the impact of lipids and bilayer properties on the structure and function of transmembrane proteins. However, the functional reconstitution of transmembrane proteins in liposomes can be challenging, especially if the hydrophobic thickness of the protein does not match the thickness of the lipid bilayer. Such hydrophobic mismatch causes protein aggregation and low yields during the reconstitution procedure, which are exacerbated in sterol-rich membranes featuring low membrane compressibility. Here, we explore new approaches to reversibly tune the sterol content of (proteo)liposomes with methyl-β-cyclodextrin (mβCD) in a dialysis setting. Maintaining (proteo)liposomes in a confined compartment minimizes loss of material during cholesterol transfer and facilitates efficient removal of mβCD. We monitor the sterol concentration in the membrane with help of the solvatochromic probe C-Laurdan, which reports on lipid packing. Using Förster resonance energy transfer, we show that cholesterol delivery to proteoliposomes induces the oligomerization of a membrane property sensor, whereas a subsequent removal of cholesterol demonstrates full reversibility. We propose that tuning membrane compressibility by mβCD-meditated cholesterol delivery and removal in a dialysis setup provides a new handle to study the impact of sterols and membrane compressibility on membrane protein structure, function, and dynamics
Impact of modifications on the characteristics of salivary pellicle on dental hard tissue: a scoping review
No full textObjectives
The salivary pellicle regulates interfacial events on dental surfaces and is a promising target for modification to improve dental health. This scoping review systematically examines substances that modify the pellicle, evaluating their potential and identifying knowledge gaps.
Data
This review followed the PRISMA-ScR guidelines. Studies investigating how substances applied during pellicle formation on enamel or dentin affect its structure, composition, or protective function were included.
Sources
A systematic search of Medline was conducted up to March 2024 using predefined terms. Only English-language research articles were included. No hand-searching was performed.
Study selection
A total of 864 records were identified. After title and abstract screening, 110 articles were assessed in full, resulting in 85 eligible studies for data charting. These studies investigated human saliva-derived pellicles on enamel or dentin, comparing their composition, structure, or protective properties with and without modification. Most studies, conducted equally in vitro and in situ, focused on fluorides and metals (n = 28), peptides and proteins (n = 21), and polyphenols (n = 18), while other substances were less frequently examined (n = 17). Modifications affected the pellicle’s ultrastructure, altered its proteome, or enhanced its protective role against erosion, abrasion, and bacterial adherence.
Conclusions
Despite numerous studies and distinct substance classes, findings remain inconclusive and must be interpreted within the context of individual study designs.
Clinical significance
Modifying the pellicle can improve its protective function, presenting a promising approach for preventive dentistry. However, the long-term effects of these substances within the dynamic oral environment remain unclear. This review underscores the need for further research to close existing knowledge gaps and confirm clinical relevance
Discovery of a novel microtubule-associated protein that plays a multifaceted role in primary cilia and midbodies
No full textThe primary cilium is a microtubule-based organelle that serves as a signaling hub in nearly all
vertebrate cells. Through changes in the ciliary proteome, it regulates downstream signaling
processes important for cell-cell communication, development and tissue homeostasis. Defects
in its structure or signaling function can lead to diseases, called ciliopathies. The cilium
formation itself is linked to the cell cycle as it is templated by the mother centriole.
Additionally, cilium formation requires a controlled coordination of microtubules (MTs), the
actin cytoskeleton and vesicle trafficking. Emerging links of microtubule associated proteins
(MAPs) contributing to the structural and functional integrity of cilia were drawn. Many ciliary
MAPs do not exclusively localize to primary cilia but also other MT-based structures, such as
centrioles. Besides the mother centriole as ciliary templating structure, also midbodies were
linked to cilium formation. Given the broad disease spectrum of ciliopathies related to ciliary
proteins, studying MAPs is crucial, as they influence not only cilia but also other cellular MT
networks, centrioles and spindle structures.
In this study I characterized a novel ciliary protein, FCAP33, which was identified through a
proteomic screen of primary cilia in murine inner medullary collecting duct cells (IMCD3)
cells. Microscopy revealed the localization of FCAP33 to primary cilia, basal bodies,
centrioles, midbodies and the mitotic spindle. This suggested a role in MT-based structures
beyond primary cilia. Further imaging-based approaches demonstrated its ability to bind to
preformed MTs indicating its role as a MAP rather than being a MT inner binding protein. The
microtubule association was also confirmed to the ciliary axoneme, where it potentially fulfills
a stabilizing role. Additionally, FCAP33 appeared as a static component with limited mobility
within the primary cilium in live cell photobleaching experiments. Functional studies revealed
that overexpressed full-length FCAP33 promotes ciliary elongation whereas the
overexpression of the N-terminal part of FCAP33 had a dominant negative effect, leading to
shorter cilia and lower ciliation rates. Immunofluorescence and pull-down assays of full-length
and short variants indicate that FCAP33 forms redox-sensitive oligomers via its N-terminus,
which also plays a role in oligomerization and MT binding whereas the C-terminus might be
involved in regulatory functions. This hypothesis can be supported by in silico and structural
analysis of the protein. Beyond a ciliary localization FCAP33 also localizes to midbodies
including the stem body region and the midbody remnant.
This study introduces FCAP33 as a novel ciliary MAP with multifaceted roles in MT-based
organelles by potentially regulating ciliogenesis and MT stability.Das primäre Zilium ist ein auf Mikrotubuli basiertes Zellorganell, das in fast allen
Wirbetlierzellen als Signalvermittlungszentrum gilt. Durch Veränderungen des ziliären
Proteoms reguliert es nachgeschaltete Signalprozesse, die für Kommunikation, Entwicklung
und Gewebehomöostase wichtig sind. Defekte in seiner Struktur oder Signalfunktion können
zu Krankheiten führen, die als Ziliopathien bezeichnet werden. Die Zilienbildung selbst ist mit
dem Zellzyklus verknüpft, da sie von der Mutterzentriole gesteuert wird. Darüber hinaus
erfordert die Zilienbildung eine kontrollierte Koordination von Mikrotubuli (MT), des
Aktinzytoskeletts und des Vesikeltransports. Es wurden neue Verbindungen zwischen
Mikrotubuli assoziierten Proteinen (MAPs) und Zilien hergestellt, die zur strukturellen und
funktionellen Integrität von Zilien beitragen. Viele ziliare MAPs sind nicht ausschließlich in
primären Zilien lokalisiert, sondern auch in anderen MT-basierten Strukturen, wie z.B.
Zentriolen. Neben der Mutterzentriole als ziliäre Grundstruktur wurden auch Midbodies mit
der Zilienbildung in Verbindung gebracht. Angesichts des breiten Krankheitsspektrums von
Ziliopathien, die mit ziliären Proteinen zusammenhängen, ist die Untersuchung von MAPs von
entscheidender Bedeutung, da sie nicht nur Zilien, sondern auch andere zelluläre MTNetzwerke,
Zentriolen und Spindelstrukturen beeinflussen. In dieser Studie charakterisierte ich
ein neuartiges Zilienprotein FCAP33, das durch ein proteomisches Screening von primären
Zilien in mIMCD3-Zellen identifiziert wurde. Mikroskopie zeigte die Lokalisierung von
FCAP33 in primären Zilien, ziliären Basalkörpern, Zentriolen, Midbodies und der mitotischen
Spindel. Dies deutet über die primären Zilien hinaus auf eine Rolle in MT-basierten Strukturen
hin. Weitere bildgebende Verfahren zeigten die Fähigkeit von FCAP33, an bereits gebildete
MTs zu binden, was auf seine Rolle als MAP und nicht als MT-Innenbindungsprotein hinweist.
Die Mikrotubuli-Assoziation wurde auch im Axonem der Zilien bestätigt, wo es
möglicherweise eine stabilisierende Rolle spielt. Darüber hinaus ist FCAP33 eine eher statische
Komponente, die in Live-Cell-Photobleaching Experimenten nicht mobil im Zilium
transportiert wird. Funktionelle Studien ergaben, dass überexprimiertes FCAP33 in voller
Länge die ziliare Elongation fördert, während die Überexpression des N-terminalen Teils von
FCAP33 einen dominant negativen Effekt hatte, der zu kürzeren Zilien und niedrigeren
Ziliationsraten führte. Immunfluoreszenz und Pull-Down Versuche von Volllängen- und
Kurzvarianten deuten darauf hin, dass FCAP33 redox-empfindliche Oligomere bildet, durch
den N-terminus, der ebenso eine Rolle bei der Oligomerisierung und MT-Bindung spielt,
während der C-terminus an regulatorischen Funktionen beteiligt sein könnte. Diese Hypothese kann durch in silico und strukturelle Analysen des Proteins gestützt werden. In dieser Studie
wird FCAP33 als ein neuartiges ziliäres MAP vorgestellt, mit vielfältigen Funktionen in MTbasierten
Organellen, durch die mögliche Regulation der Ziliogenese und der MT-Stabilität
Nicht-interventionelle Kohortenstudie zur Versorgung von Patienten mit nicht-organischen Schlafstörungen, die homöopathisch behandelt werden, im Vergleich zu Patienten, die eine medikamentöse Therapie mit einem chemisch-synthetischen Arzneimittel erhalten
No full textHintergrund
Diese Dissertation befasst sich mit der strukturierten Analyse der Behandlung von nichtorganischen
Schlafstörungen im hausärztlichen Setting. Dazu wurde der Versorgungsalltag von
Privat- und Kassenärzten, welche konventionell / schulmedizinisch therapieren, dokumentiert und
dem Versorgungsalltag von Privat- oder Kassenärzten mit der Zusatzqualifikation Homöopathie
gegenübergestellt.
Methodik
Die Untersuchung fand im Rahmen einer offenen, zweiarmigen, prospektiven, multizentrischen,
nicht-interventionellen Kohortenstudie gemäß §4 Absatz 23 Satz 2 des Arzneimittelgesetzes
(AMG) unter Alltagsbedingungen statt. Insgesamt wurden 88 Erwachsene mit
behandlungsbedürftiger Insomnie in die Studie eingeschlossen und über einen Zeitraum von 12
Wochen begleitet. Die Patienten wurden nach Diagnosestellung einer nicht-organischen Insomnie
in Orientierung an die Definition nach der S3-Leitlinie „Nicht erholsamer Schlaf / Schlafstörung,
Kapitel „Insomnie bei Erwachsenen“ entweder durch Hausärzte mit der Zusatzqualifikation
Homöopathie homöopathisch behandelt, oder durch konventionelle Hausärzte schulmedizinisch.
Untersucht wurden insgesamt 3 Forschungsfragestellungen, welche als Endpunkte dienten.
Die erste Forschungsfragestellung befasste sich mit der Frage, inwieweit sich die Patienten der
konventionellen Therapiegruppe (KON) und der Homöopathiegruppe (HOM) bezüglich des
Schweregrades ihrer Schlafstörung sowie weiterer soziodemographischer Merkmale an Baseline
voneinander unterscheiden. Dazu wurden unter anderem eine ausführliche Anamnese sowie
Fragebögen wie der Pittsburgh Sleep Quality Index (PSQI) sowie der Measure Yourself Medical
Outcome Profile-Score (MYMOP-Score) analysiert.
Die zweite Fragestellung befasste sich damit, inwieweit sich die Patienten der Gruppen KON und
HOM bezüglich der Schlafstörungsparameter nach 4, 8 und 12 Wochen Behandlung
unterscheiden. Hierbei gingen ebenfalls die Fragebögen zu PSQI und MYMOP-Score in
Kombination mit Schlaftagebüchern der Patienten in die Auswertung ein.
Die dritte und letzte Forschungsfrage untersuchte, inwieweit sich die Patienten der Gruppen KON
und HOM bezüglich ihrer Behandlungszufriedenheit nach 12 Wochen Behandlung unterscheiden. Zur Beantwortung dieser Frage wurde der Integrative Medicine Patient Satisfaction Scale (IMPSS)
zur Analyse eingesetzt.
Ergebnisse
Die Ergebnisse zeigten, dass beide Therapieformen mit einer signifikanten Verbesserung der
subjektiven Schlafqualität und des allgemeinen Beschwerdebildes einhergingen. Der PSQI-Score
sank in beiden Gruppen deutlich, ohne dass ein signifikanter Unterschied zwischen den Gruppen
beobachtet wurde. Auch der MYMOP-Score verbesserte sich in beiden Gruppen signifikant, mit
tendenziell stärkeren Effekten in der HOM-Gruppe. Die Analyse der IMPSS zeigte eine signifikant
höhere Patientenzufriedenheit in der HOM-Gruppe.
Während die konventionelle Therapie eine geringere Variabilität in der verordneten Medikation
zeigte, zeichnete sich die homöopathische Therapie durch häufige Arzneimittelwechsel aus, was
am ehesten Ausdruck des individualisierten Therapieansatzes war. Die Compliance war in beiden
Gruppen hoch. UAW traten häufiger in der KON-Gruppe auf, waren aber durchweg nicht
schwerwiegend und ohne Folgeschäden.
Zusätzlich wurde eine detaillierte Analyse sowohl verschiedener Einflussfaktoren auf als auch der
Auswirkungen von Schlafstörung durchgeführt. Dabei zeigten sich unter anderem häufige
psychosoziale Belastungen wie Sorgen im familiären oder beruflichen Kontext, Tagesmüdigkeit
und eingeschränkte Leistungsfähigkeit. Diese Faktoren besserten sich im Verlauf, blieben jedoch
bei einem Teil der Patienten auch nach 12 Wochen bestehen. Die Analyse der Beschwerden
hinsichtlich Einschlaf- und Durchschlafstörungen ergab, dass beide Gruppen zu Beginn deutliche
Einschränkungen aufwiesen, wobei sich beide Parameter im Verlauf der Behandlung in beiden
Gruppen verbesserten
Schlussfolgerung
Die Ergebnisse sprechen dafür, dass unter Alltagsbedingungen sowohl konventionelle als auch
homöopathische Behandlungsansätze zu einer subjektiv wahrgenommenen Besserung von
Schlafstörungen führen können. Weitere Studien, insbesondere mit einer größeren Fallzahl sind
notwendig, um weitere sowie detailliertere Aussagen zu beiden Therapieformen zu treffen.Background
This dissertation addresses the structured analysis of the treatment of non-organic sleep disorders
in general practice. The routine care provided by private and statutory health insurance physicians
practicing conventional medicine was documented and compared with the treatment provided by
physicians with an additional qualification in homeopathy.
Methods
The study was conducted as an open-label, two-arm, prospective, multicenter, non-interventional
cohort study in accordance with §4, paragraph 23, sentence 2 of the German Medicines Act
(AMG), under real-life conditions. A total of 88 adults with clinically relevant insomnia were enrolled
and observed over a 12-week period. Following the diagnosis of non-organic insomnia—based on
the criteria of the German S3 guideline “Non-restorative sleep / sleep disorders”, chapter “Insomnia
in adults”—patients were treated either conventionally or homeopathically, depending on whether
the treating physician held the additional qualification in homeopathy.
Three research questions were investigated, each serving as a study endpoint.
The first question examined whether the patients in the conventional treatment group (KON) and
the homeopathy group (HOM) differed at baseline with respect to the severity of their sleep
disorder and other sociodemographic characteristics. To this end, detailed anamneses and
questionnaires such as the Pittsburgh Sleep Quality Index (PSQI) and the Measure Yourself
Medical Outcome Profile (MYMOP) were analyzed.
The second question focused on whether sleep disorder parameters differed between the KON
and HOM groups after 4, 8, and 12 weeks of treatment. The PSQI and MYMOP scores, in
combination with patient sleep diaries, were used for evaluation.
The third question addressed differences in patient satisfaction between the KON and HOM groups
after 12 weeks of treatment. The Integrative Medicine Patient Satisfaction Scale (IMPSS) was used
for this analysis. Results
Both treatment approaches were associated with significant improvements in subjective sleep
quality and overall symptom burden. The PSQI score decreased significantly in both groups,
without a statistically significant difference between them. The MYMOP score also improved in
both groups, with slightly stronger effects observed in the HOM group. The IMPSS analysis
revealed significantly higher patient satisfaction in the HOM group.
While the conventional group showed less variation in prescribed medications, the homeopathy
group frequently switched remedies—reflecting the individualized nature of homeopathic therapy.
Treatment adherence was high in both groups. Adverse drug reactions occurred more frequently in
the KON group but were mild and without long-term consequences.
A detailed analysis was also conducted of both the various influencing factors on and the
consequences of sleep disorders. Psychosocial stressors, such as concerns in the family or
workplace, daytime fatigue, and reduced performance capacity, were common among participants
and showed partial improvement over the course of the study. The evaluation of sleep-onset and
sleep-maintenance difficulties revealed considerable impairments in both groups at baseline, with
improvements observed across both groups over time.
Conclusion
The findings suggest that both conventional and homeopathic treatment approaches can lead to
subjectively perceived improvements in sleep disturbances under routine care conditions. Further
studies—particularly with larger sample sizes—are needed to allow for more detailed and
differentiated conclusions regarding both therapeutic models
Modeling cytoskeletal and cell dynamics
No full textHere we give an overview of recent theoretical and experimental work on modeling the mechanics and dynamics of the cytoskeleton. The cytoskeleton is a multicomponent, complex and active material that is essential to cell mechanics and dynamics. We focus on one of the main components of this material, namely actin filaments. We discuss these filaments and their interactions with other proteins within the cytoskeleton. To fully understand the cytoskeleton, it is important to consider both theoretical and experimental work in calculo, in silico, in vitro, in vivo, and in situ. We review the current state of knowledge and look forward to further work to come on aspects not yet understood
Agreement Between Child Self- and Parent Proxy-Reports of Health-Related Quality of Life in Spinal Muscular Atrophy: Preliminary Insights from a Nationwide Patient Registry in Germany
No full textObjective
The objective of this pilot study was to examine the agreement between child self- and parent proxy-assessment of health-related quality of life (HRQoL) in spinal muscular atrophy (SMA) in the era of disease-modifying therapy.
Methods
Children with SMA and one of their parents were recruited via the German national TREAT-NMD SMA patient registry. HRQoL was measured using the Pediatric Quality of Life Inventory 3.0 Neuromuscular Module (PedsQL 3.0 NMM), KIDSCREEN-27, and the Health Utilities Index (HUI). Agreement between child self- and parent proxy-ratings of ordinal measures was estimated using Cohen’s κ, and for continuous measures using intraclass correlation coefficients (ICCs) from one-way random-effects models.
Results
The final sample comprised 17 children with SMA (mean age: 9.88 years, SD: 4.33 years, range: 5–16 years; 59% female) and one of their parents. All but two patients (88%) were receiving disease-modifying therapy (nusinersen or risdiplam). The ICC for the total PedsQL 3.0 NMM score was estimated at 0.85 (95% CI: 0.64–0.94, p < 0.001) (indicative of excellent agreement). The corresponding estimate for the KIDSCREEN total score was 0.27 (95% CI: 0.00–0.75, p = 0.197) (poor/fair agreement) and the global HUI utility 0.98 (95% CI: 0.93 to 0.9952, p < 0.001) (excellent agreement). The lowest levels of concordance were found for school and family life, as well as mental well-being, as opposed to physical functioning and disability.
Conclusion
We show that the agreement between child self- and parent proxy-reports of HRQoL in SMA varies markedly across HRQoL measures and examined domains, ranging from poor/fair to excellent. Compared with previous research, agreement for the PedsQL 3.0 NMM was markedly higher in our contemporary cohort of patients treated with novel therapies. These preliminary findings will be helpful in informing the design of future research of HRQoL in SMA