bluuebunny/crossref_metadata_2025 huggingface dataset:
Quality of life among cancer inpatients 80 years and older: a systematic review
Authors: Drageset Jorunn , Sandvik Reidun Karin , Eide Leslie Sofia Pareja , Austrheim Gunhild , Fox Mary , Beisland Elisabeth Grov
Published: March 2021

Objective

The aim of this systematic review was to summarize and assess the literature on quality of life (QoL) among cancer patients 80 years and older admitted to hospitals and what QoL instruments have been used.

Methods

We searched systematically in Medline, Embase and Cinahl. Eligibility criteria included studies with any design measuring QoL among cancer patients 80 years and older hospitalized for treatment (surgery, chemotherapy or radiation therapy). Exclusion criteria: studies not available in English, French, German or Spanish. We screened the titles and abstracts according to a predefined set of inclusion criteria. All the included studies were assessed according to the Critical Appraisal Skills Programme checklists, and the Preferred Reporting Items for Systematic Reviews and Meta-Analyses Statement checklist was used to ensure rigor in conducting and reporting. This systematic review was registered in PROSPERO (CRD42017058290).

Results

We included 17 studies with 2005 participants with various cancer diagnoses and Classification of Malignant Tumors stages (TNM). The included studies used a range of different QoL instruments and had different aims and outcomes. Both cancer-specific and generic instruments were used. Only one of the 17 studies used an age-specific instrument. All the studies included patients 80 years and older in their cohort, but none specifically analyzed QoL outcomes in this particular subgroup. Based on findings in the age-heterogeneous population (age range 20–100 years), QoL seems to be correlated with the type of diagnosed carcinoma, length of stay, depression and severe symptom burden.

Conclusion

We were unable to find any research directly exploring QoL and its determinants among cancer patients 80 years and older since none of the included studies presented specific analysis of data in this particular age subgroup. This finding represents a major gap in the knowledge base in this patient group. Based on this finding, we strongly recommend future studies that include this increasingly important and challenging patient group to use valid age- and diagnosis-specific QoL instruments.
Quality of life in primary sclerosing cholangitis: a systematic review
Authors: Marcus Elena , Stone Paddy , Krooupa Anna-Maria , Thorburn Douglas , Vivat Bella
Published: March 2021

Background

Primary sclerosing cholangitis (PSC) is a rare bile duct and liver disease which can considerably impact quality of life (QoL). As part of a project developing a measure of QoL for people with PSC, we conducted a systematic review with four review questions. The first of these questions overlaps with a recently published systematic review, so this paper reports on the last three of our initial four questions: (A) How does QoL in PSC compare with other groups?, (B) Which attributes/factors are associated with impaired QoL in PSC?, (C) Which interventions are effective in improving QoL in people with PSC?.

Methods

We systematically searched five databases from inception to 1 November 2020 and assessed the methodological quality of included studies using standard checklists.

Results

We identified 28 studies: 17 for (A), ten for (B), and nine for (C). Limited evidence was found for all review questions, with few studies included in each comparison, and small sample sizes. The limited evidence available indicated poorer QoL for people with PSC compared with healthy controls, but findings were mixed for comparisons with the general population. QoL outcomes in PSC were comparable to other chronic conditions. Itch, pain, jaundice, severity of inflammatory bowel disease, liver cirrhosis, and large-duct PSC were all associated with impaired QoL. No associations were found between QoL and PSC severity measured with surrogate markers of disease progression or one of three prognostic scoring systems. No interventions were found to improve QoL outcomes.

Conclusion

The limited findings from included studies suggest that markers of disease progression used in clinical trials may not reflect the experiences of people with PSC. This highlights the importance for clinical research studies to assess QoL alongside clinical and laboratory-based outcomes. A valid and responsive PSC-specific measure of QoL, to adequately capture all issues of importance to people with PSC, would therefore be helpful for clinical research studies.
MBTPS2, a membrane bound protease, underlying several distinct skin and bone disorders
Authors: Caengprasath Natarin , Theerapanon Thanakorn , Porntaveetus Thantrira , Shotelersuk Vorasuk
Published: March 2021

TheMBTPS2gene on the X-chromosome encodes the membrane-bound transcription factor protease, site-2 (MBTPS2) or site-2 protease (S2P) which cleaves and activates several signaling and regulatory proteins from the membrane. The MBTPS2 is critical for a myriad of cellular processes, ranging from the regulation of cholesterol homeostasis to unfolded protein responses. While its functional role has become much clearer in the recent years, how mutations in theMBTPS2gene lead to several human disorders with different phenotypes including Ichthyosis Follicularis, Atrichia and Photophobia syndrome (IFAP) with or without BRESHECK syndrome, Keratosis Follicularis Spinulosa Decalvans (KFSD), Olmsted syndrome, and Osteogenesis Imperfecta type XIX remains obscure. This review presents the biological role of MBTPS2 in development, summarizes its mutations and implicated disorders, and discusses outstanding unanswered questions.
Disease activity index is associated with subclinical atherosclerosis in childhood-onset systemic lupus erythematosus
Authors: Medeiros Priscila B. S. , Salomão Roberta G. , Teixeira Sara R. , Rassi Diane M. , Rodrigues Luciana , Aragon Davi C. , Fassini Priscila G. , Ued Fábio V. , Tostes Rita C. , Monteiro Jacqueline P. , Ferriani Virgínia P. L. , de Carvalho Luciana M.
Published: March 2021

Background

Systemic lupus erythematosus (SLE) is an independent risk factor for cardiovascular events. The present study determined the prevalence of subclinical atherosclerosis in childhood-onset SLE using the carotid intima-media thickness (CIMT) measurement and investigated associations between traditional and nontraditional risk factors for atherosclerosis, such as medications, SLE Disease Activity Index - SLEDAI-2 K and SLICC-ACR damage index and CIMT.

Methods

Cross-sectional prospective study between 2017 and 2018. CIMT was assessed by ultrasonography. Data were collected by chart review, nutritional evaluation and laboratory tests and analyzed by Fisher, Wilcoxon-Mann-Whitney tests, multiple linear and log binomial regression.

Results

Twenty-eight patients (mean age 13.9 years, SD 3) were enrolled. The prevalence of subclinical atherosclerosis was 32% (95% CI 14.8, 49.4). The mean CIMT was 0.43 ± 0.035 mm. The most common traditional risk factors observed were dyslipidemia (82.1%), uncontrolled hypertension (14.2%), obesity (14.3%), and poor diet (78.6%). Uncontrolled hypertension (p = 0.04), proteinuria (p = 0.02), estimated glomerular filtration rate < 75 ml /min/1.73 m2 (p = 0.02) and SLEDAI-2 K > 5 (P = 0.04) were associated with subclinical atherosclerosis. SLEDAI-2 K > 5 maintained association with CIMT after adjusting for control variables.

Conclusion

Subclinical atherosclerosis is frequently observed in cSLE, mainly in patients with moderate to severe disease activity.
Joint contractures responsive to immunosuppressive therapy in a girl with childhood‐onset systemic sclerosis double‐seropositive for rare anti‐nucleolar autoantibodies: a case report
Authors: Tanaka Riki , Tani Yumi , Kaburaki Yoichiro , Kinoshita Manao , Kawaguchi Yasushi , Okazaki Yuka , Kuwana Masataka , Harigai Masayoshi , Nagata Satoru , Miyamae Takako
Published: March 2021

Background

Systemic sclerosis (SSc; scleroderma) is an autoimmune connective tissue disease that affects the skin and subcutaneous tissue, in addition to the internal organs of the whole body. Onset in childhood is uncommon; however, both patients with childhood-onset and adult-onset SSc are positive for anti-nuclear antibodies (ANAs).Detection of SSc-related anti-nuclear antibodies is often useful for predicting clinical features, disease course, and outcomes.

Case presentation

A 5-year-old Japanese female manifested gradually progressive abnormal gait disturbance, regression of motor development, Raynaud’s phenomenon, and the shiny appearance of the skin of the face and extremities at age 2. On admission, she presented a mask-like appearance, loss of wrinkles and skin folds, puffy fingers, moderate diffuse scleroderma (18/51 of the modified Rodnan total skin thickness score), and contracture in the ankle and proximal interphalangeal joints. Grossly visible capillary hemorrhage on nail fold and severe abnormal capillaroscopy findings including bleeding, giant loop and disappearance of capillaryconsistent with the late phase in SSc. A skin biopsy showed fibrous thickening of the dermis, entrapment of an eccrine sweat glands, and thickened fiber. Chest high-resolution computed tomographic scanning demonstrated patchy areas of ill-defined air-space opacity and consolidation predominantly involving the posterior basilar aspects of the lower lobes presenting withinterstitial lung disease. Positive ANA (1:160 nucleolar and homogeneous nuclear staining by indirect fluorescent antibody technique) and double-seropositive for anti-Th/To and anti-PM-Scl antibodies were identified. She was diagnosed with diffuse cutaneous SSc based on the Pediatric Rheumatology European Society/American College of Rheumatology/European League Against Rheumatism Provisional Classification Criteria for Juvenile Systemic Sclerosis and was successfully treated with immunosuppressive agents, including methylprednisolone pulses and intravenous cyclophosphamide.

Conclusions

We experienced the first case of juvenile SSc with anti-PM-Scl and anti-Th/To antibodies. ILD was identified as a typical feature of patients with these autoantibodies; however, diffuse cutaneous SSc and joint contraction were uncharacteristically associated. The case showed unexpected clinical findings though the existence of SSc-related autoantibodies aids in determining possible organ involvement and to estimate the children’s outcome.
Incidence and predictors of early loss to follow up among patients initiated on protease inhibitor-based second-line antiretroviral therapy in southwestern Uganda
Authors: Nuwagira Edwin , Lumori Boniface A. E. , Muhindo Rose , Kanyesigye Michael , Amir Abdallah , Muyindike Winnie , Muzoora Conrad
Published: March 2021

Background

Good adherence to antiretroviral therapy (ART) and retention in care are essential for the effectiveness of an HIV care program. With the current increase in numbers of people living with HIV taking second-line ART in sub-Saharan Africa, there is a need to establish their treatment outcomes and the rate of loss to follow up. In this study, we determined the incidence and predictors of loss to follow up among patients taking second-line ART at an experienced HIV treatment center in southwestern Uganda.

Methods

This was a retrospective review of an electronic database at Mbarara Regional Referral Hospital HIV clinic in southwestern Uganda. Second-line ART included at least two of the nucleoside reverse transcriptase inhibitors and a boosted protease inhibitor. Loss to follow-up was defined as failure to return to the health facility for care or treatment refill for 180 days or more from the previous visit. After excluding children less than 15 years, we pooled data that included socio-demographic, clinical, and laboratory data for patients who started second-line ART between 2002 and 2017. Multiple imputation was done for variables with missing data. Variables that had a p < 0.05 in unadjusted bivariate analyses were included in a multivariate binomial regression model using a stepwise backward selection procedure to describe the factors that independently predicted loss to follow-up.

Results

Between 2002 and 2017, 1121 patients had been initiated on second-line ART. We included data from 924 participants and of these, 518 (56.1%) were female, the mean age (SD) was 38.4 (± 10.5) years, and 433 (52.4%) had a CD4 count less than 100 cells/µl at the start of second-line ART. The incidence of loss to follow-up was 26.7 per 100 person-years. Male gender (Adjusted risk ratio (ARR) = 1.8, 95% CI 1.5–2.0) p < 0.001 and anemia ARR 1.4, 95% CI 1.1–1.6) p < 0.001 were strongly associated with loss to follow up.

Conclusions

There is a high incidence of loss to follow up among patients taking protease-inhibitor based second-line ART at a tertiary HIV center in southwestern Uganda. There is a need to routinely measure hemoglobin during clinic reviews, and establish mechanisms to retain males initiated on second-line ART in care. The association of anemia and loss to follow up needs to be investigated.
The effect of maternal depressive symptoms on infant feeding practices in rural Ethiopia: community based birth cohort study
Authors: Woldetensay Yitbarek Kidane , Belachew Tefera , Ghosh Shibani , Kantelhardt Eva Johanna , Biesalski Hans Konrad , Scherbaum Veronika
Published: March 2021

Background

Maternal depression and other psychosocial factors have been shown to have adverse consequences on infant feeding practices. This study explored the longitudinal relationship of maternal depressive symptoms and other selected psychosocial factors with infant feeding practices (IFPs) in rural Ethiopia using summary IFP index.

Methods

This study uses existing data from the ENGINE birth cohort study, conducted from March 2014 to March 2016 in three districts in the southwest of Ethiopia. A total of 4680 pregnant women were recruited and data were collected once during pregnancy (twice for those in the first trimester), at birth, and then every 3 months until the child was 12 months old. A standardized questionnaire was used to collect data on IFPs, maternal depressive symptoms, household food insecurity, intimate partner violence (IPV), maternal social support, active social participation, and other sociodemographic variables. A composite measure of IFP index was computed using 14 WHO recommended infant and young child feeding (IYCF) practice indicators. High IFP index indicated best practice. Prenatal and postnatal maternal depressive symptoms were assessed using the patient health questionnaire (PHQ-9). Linear multilevel mixed effects model was fitted to assess longitudinal relationship of IFPs with maternal depression and other psychosocial factors.

Results

Reports of higher postnatal depressive symptoms (ß = − 1.03, P = 0.001) and IPV (ß = − 0.21, P = 0.001) were associated with lower scores on the IFP index. Whereas, reports of better maternal social support (ß = 0.11, P = 0.002) and active social participation (ß = 0.55, P < 0.001) were associated with higher scores on the IFP index. Contrary to expectations, moderate household food insecurity (ß = 0.84, P = 0.003), severe household food insecurity (ß = 1.03, P = 0.01) and infant morbidity episodes (ß = 0.63, P = 0.013) were associated with higher scores on the IFP index.

Conclusions

Overall, a multitude of factors are related to IFPs and hence coordinated, multi-sectoral and multi-stakeholder interventions including maternal depressive symptoms screening and management are needed to improve infant feeding practices.
The effectiveness of generic emails versus a remote knowledge broker to integrate mood management into a smoking cessation programme in team-based primary care: a cluster randomised trial
Authors: Minian Nadia , Ahad Sheleza , Ivanova Anna , Veldhuizen Scott , Zawertailo Laurie , Ravindran Arun , de Oliveira Claire , Baliunas Dolly , Mulder Carol , Bolbocean Corneliu , Selby Peter
Published: March 2021

Background

Knowledge brokering is a knowledge translation approach that has been gaining popularity in Canada although the effectiveness is unknown. This study evaluated the effectiveness of generalised, exclusively email-based prompts versus a personalised remote knowledge broker for delivering evidence-based mood management interventions within an existing smoking cessation programme in primary care settings.

Methods

The study design is a cluster randomised controlled trial of 123 Ontario Family Health Teams participating in the Smoking Treatment for Ontario Patients programme. They were randomly allocated 1:1 for healthcare providers to receive either: a remote knowledge broker offering tailored support via phone and email (group A), or a generalised monthly email focused on tobacco and depression treatment (group B), to encourage the implementation of an evidence-based mood management intervention to smokers presenting depressive symptoms. The primary outcome was participants’ acceptance of a self-help mood management resource. The secondary outcome was smoking abstinence at 6-month follow-up, measured by self-report of smoking abstinence for at least 7 previous days. The tertiary outcome was the costs of delivering each intervention arm, which, together with the effectiveness outcomes, were used to undertake a cost minimisation analysis.

Results

Between February 2018 and January 2019, 7175 smokers were screened for depression and 2765 (39%) reported current/past depression. Among those who reported current/past depression, 29% (437/1486) and 27% (345/1277) of patients accepted the mood management resource in group A and group B, respectively. The adjusted generalised estimating equations showed that there was no significant difference between the two treatment groups in patients’ odds of accepting the mood management resource or in the patients’ odds of smoking abstinence at follow-up. The cost minimisation analysis showed that the email strategy was the least costly option.

Conclusions

Most participants did not accept the resource regardless of remote knowledge broker strategy. In contexts with an existing KT infrastructure, decision-makers should consider an email strategy when making changes to a programme given its lower cost compared with other strategies. More research is required to improve remote knowledge broker strategies.

Trial registration

ClinicalTrials.gov, NCT03130998. Registered April 18, 2017, (Archived on WebCite at www.webcitation.org/6ylyS6RTe)
Current status and developments of German curriculum-based residency training programmes in radiation oncology
Authors: Büttner Marcel , Cordes Nils , Gauer Tobias , Habermehl Daniel , Klautke Gunther , Micke Oliver , Mäurer Matthias , Sokoll Jan , Troost Esther Gera Cornelia , Christiansen Hans , Niyazi Maximilian
Published: March 2021

Purpose

The current status of German residency training in the field of radiation oncology is provided and compared to programmes in other countries. In particular, we present the DEGRO-Academy within the international context.

Methods

Certified courses from 2018 and 2019 were systematically assigned to the DEGRO-Curriculum, retrospectively for 2018 and prospectively for 2019. In addition, questionnaires of course evaluations were provided, answered by course participants and collected centrally.

Results

Our data reveal a clear increase in curriculum coverage by certified courses from 57.6% in 2018 to 77.5% in 2019. The analyses enable potential improvements in German curriculum-based education. Specific topics of the DEGRO-Curriculum are still underrepresented, while others decreased in representation between 2018 and 2019. It was found that several topics in the DEGRO-Curriculum require more attention because of a low DEGRO-curriculum coverage. Evaluation results of certified courses improved significantly with a median grade of 1.62 in 2018 to 1.47 in 2019 (p = 0.0319).

Conclusion

The increase of curriculum coverage and the simultaneous improvement of course evaluations are promising with respect to educational standards in Germany. Additionally, the early integration of radiation oncology into medical education is a prerequisite for resident training because of rising demands on quality control and increasing patient numbers. This intensified focus is a requirement for continued high standards and quality of curriculum-based education in radiation oncology both in Germany and other countries.
Application of modified closed biopsy in rabbit model of VX2-transplanted bone tumor
Authors: Bai Lei Peng , Lv Jia Xing , Kong Ling Wei , Cao Hai Ying , Jin Yu
Published: March 2021

Background

This study was aimed to explore the application value of modified closed biopsy technique in puncture biopsy of rabbit VX2 transplanted bone tumor model.

Methods

VX2 tumor was transplanted into the bilateral tibia of 30 rabbits through the tibial plateau to make the model of VX2 transplanted bone tumor. Seven days after modeling, the proximal tibia biopsy was performed under the guidance of X-ray, and the biopsy specimen was examined pathologically. The left leg was biopsied with modified closed biopsy technique (experimental group), and the right leg was biopsied with hollow needle (control group). After 14 days of modeling, all rabbits were killed after X-ray examination around the puncture hole, and the soft tissue around the puncture hole was taken for pathological examination, and the expression levels of PCNA and CD34 in the tissue extract were detected by enzyme-linked immunosorbent assay (ELISA).

Results

By the end of the experiment, a total of 3 rabbits died, and finally, 27 rabbits were included in the study. Tumor cells were detected in all the intramedullary specimens obtained by puncture biopsy. On the 14th day after modeling, X-ray showed that the occurrence rate of periosteal reaction and extraosseous high-density shadow around the puncture hole was 14.81% (4/27) in the experimental group and 40.74% (11/27) in the control group. The difference was statistically significant (P<0.05). The pathological results of soft tissue around the puncture hole showed that the tumor cell metastasis rate was 29.63% (8/27) in the experimental group and 100% (27/27) in the control group, and the difference was statistically significant (P<0.05). The expression levels of PCNA and CD34 in the experimental group were lower than those in the control group (P < 0.05).

Conclusion

Both the modified closed biopsy technique and needle aspiration biopsy can provide sufficient biopsy tissue for the diagnosis of VX2-transplanted bone tumor in rabbits. At the same time, the improved closed biopsy technique has a certain application value in preventing local metastasis of tumor cells along the puncture channel.
Impact of standardized computed tomographic angiography for minimally invasive mitral and tricuspid valve surgery
Authors: Immohr Moritz B. , Sugimura Yukiharu , Kröpil Patric , Aubin Hug , Minol Jan-Philipp , Albert Alexander , Boeken Udo , Lichtenberg Artur , Akhyari Payam
Published: March 2021

Background

Femoral cannulation for extracorporeal circulation (ECC) is a standard procedure for minimally invasive cardiac surgery (MICS) of the atrio-ventricular valves. Vascular pathologies may cause serious complications. Preoperative computed tomography-angiography (CT-A) of the aorta, axillary and iliac arteries was implemented at our department.

Methods

Between July 2017 and December 2018 all MICS were retrospectively reviewed (n = 143), and divided into 3 groups.

Results

In patients without CT (n = 45, 31.5%) ECC was applied via femoral arteries (91.1% right, 8.9% left). Vascular related complications (dissection, stroke, coronary and visceral ischemia, related in-hospital death) occurred in 3 patients (6.7%). In patients with non-contrast CT (n = 35, 24.5%) only femoral cannulation was applied (94.3% right) with complications in 4 patients (11.4%). CT-angiography (n = 63, 44.1%) identified 12 patients (19.0%) with vulnerable plaques, 7 patients (11.1%) with kinking of iliac vessels, 41 patients (65.1%) with multiple calcified plaques and 5 patients (7.9%) with small femoral artery diameter (d ≤ 6 mm). In 7 patients (11.1%) pathologic findings led to alternative cannulation via right axillary artery, additional 4 patients (6.3%) were cannulated via left femoral artery. Only 2 patients (3.2%) suffered from complications.

Conclusions

CT-A identifies vascular pathologies otherwise undetectable in routine preoperative preparation. A standardized imaging protocol may help to customize the operative strategy.
Comparison of flexible, open with semi-rigid, closed annuloplasty-rings for mitral valve repair
Authors: Cetinkaya Ayse , Waheed Maryam , Bramlage Karin , Liakopoulos Oliver Johannes , Zeriouh Mohamed , Hein Stefan , Bramlage Peter , Schönburg Markus , Choi Yeong-Hoon , Richter Manfred
Published: March 2021

Background

Mitral regurgitation is a frequent valvular disease, with an increasing prevalence. We analysed the long-term outcomes of mitral valve repair procedures conducted over the last 10 years in our clinic using almost exclusively two different annuloplasty ring types.

Methods

A single-centre, retrospective analysis of mitral valve surgeries conducted between January 2005 and December 2015 for patients undergoing first-line mitral valve repair with either open (Cosgrove) or closed (CE Physio / Physio II) annuloplasty (OA or CA, respectively) rings.

Results

In total, 1120 patient documentations were available of which 528 underwent OA and 592 patients CA. The median age of patients was 64.0 years and 41.1% were female. The majority of these patients underwent the procedure because of degenerative valve disease. Rates of successful repair were about 90%, 72 h procedural mortality was 0.6% and the rate of re-intervention was 0.6% within the first 30 days. Functional (mitral regurgitation, left ventricular ejection fraction, left ventricular end-diastolic and systolic diameter and New York Heart Association class) as well as hard outcomes were comparable. 77.7 and 74.4% of patients were alive at the 10-year follow-up in the OA and CA groups, respectively. Upon multivariable adjustment, the hazard ratio was 0.926 (95% CI: 0.642–1.3135; p = 0.681).

Conclusions

The functional outcome and survival rates up to 10 years after mitral valve repair were comparable using open and closed annuloplasty rings. Whether this means these rings are interchangeable or a carefully selection of the best-for-the-patient devices will be subject of future investigations.
Surgical treatment for brachiocephalic artery aneurysm with Takayasu arteritis using isolated cerebral perfusion: a case study
Authors: Sugiyama Kayo , Watanuki Hirotaka , Futamura Yasuhiro , Okada Masaho , Makino Satoshi , Matsuyama Katsuhiko
Published: March 2021

Background

Takayasu arteritis (TA) is a chronic inflammatory disease that induces stenosis, occlusion, or aneurysmal degeneration of the aorta and its major branches. Though rarely reported, proximal aneurysmal lesions from the aortic root to the arch are more common in Asian populations than in Western populations. In the surgical treatment of TA, anastomotic aneurysm can be problematic. Furthermore, atherosclerotic complications should be considered in surgical treatment for elderly TA patients.

Case presentation

Here, we report a case of brachiocephalic artery (BCA) aneurysm with TA for which surgical treatment was successful. Though it was solely a lesion of the brachiocephalic artery, after considering the patient’s clinical background and the features of TA, we chose a partial arch replacement. Further, for avoidance of anastomotic aneurysm, both distal and proximal anastomosis were reinforced with Teflon felt strips. Preoperative computed tomography detected severe atherosclerotic changes in the arch vessels. The patient underwent partial arch replacement using isolated cerebral perfusion (ICP) for brain protection and recovered without any neurological deficits.

Conclusions

In avoidance with anastomotic aneurysm, reinforcement of the anastomosis was introduced. ICP was effective for brain protection in case with severe atherosclerotic changes.
Energy devices safety and impact on video-assisted thoracoscopic lung lobectomy postoperative course: monopolar electrocautery versus ultrasonic dissector
Authors: Cattoni Maria , Rotolo Nicola , Nardecchia Elisa , De Maio Silvia , Dominioni Lorenzo , Imperatori Andrea
Published: March 2021

Background

This study aims to compare safety and impact of monopolar electrocautery and ultrasonic dissector (Harmonic ACE Plus®) on postoperative short-term outcomes after video-assisted thoracoscopic (VATS) lobectomy and lymphadenectomy for lung cancer.

Methods

We analyzed the prospectively collected data of 140 consecutive patients [59% male; median age: 71(IQR:62–76) years] undergoing VATS lobectomy and lymphadenectomy in our institution between October 2016 and November 2019. Patients were divided in two groups based on device used: monopolar electric hook in 79 cases (Group A); ultrasonic dissector in 61(Group B). Energy instrument-related intraoperative accidents, hemothorax/chylothorax incidence, total pleural effusion volume at 48 postoperative hours and chest tube duration were compared between groups. Multivariable analysis was performed to test energy device as possible independent risk factor either for increased pleural effusion volume or for prolonged chest tube duration.

Results

No intraoperative accidents due to energy device occurred. No hemothorax was recorded. Postoperative chylothorax incidence was slightly higher in Group A (2.5% vs 0%; p-value = 0.21). Total pleural effusion volume at 48 h was significantly higher in Group B: 400 (285–500) vs 255 (150–459) ml (p-value = 0.005). Chest tube duration was similar in the two groups: 5 (3–9) vs 5 (3–8) days (p-value = 0.77). At multivariable analysis the energy device used was not associated with increased pleural effusion volume (p-value = 0.43) nor with prolonged chest tube duration (p-value = 0.28).

Conclusions

Monopolar electrocautery and Harmonic ACE Plus® were safe and had a similar impact on short-term outcomes after VATS lobectomy and lymphadenectomy, suggesting that energy devices choice could be left to surgeon’s preference.
D-galactose supplementation in individuals with PMM2-CDG: results of a multicenter, open label, prospective pilot clinical trial
Authors: Witters Peter , Andersson Hans , Jaeken Jaak , Tseng Laura , van Karnebeek Clara D. M. , Lefeber Dirk J. , Cassiman David , Morava Eva
Published: March 2021

PMM2-CDG is the most prevalent congenital disorder of glycosylation (CDG) with only symptomatic therapy. Some CDG have been successfully treated with D-galactose. We performed an open-label pilot trial with D-galactose in 9 PMM2-CDG patients. Overall, there was no significant improvement but some milder patients did show positive clinical changes; also there was a trend toward improved glycosylation. Larger placebo-controlled studies are required to determine whether D-galactose could be used as supportive treatment in PMM2-CDG patients.

Trial registration ClinicalTrials.gov Identifier: NCT02955264. Registered 4 November 2016, https://clinicaltrials.gov/ct2/show/NCT02955264
Assessing rare diseases prevalence using literature quantification
Authors: Shourick Jason , Wack Maxime , Jannot Anne-Sophie
Published: March 2021

Introduction

Estimating the prevalence of diseases is crucial for the organization of healthcare. The amount of literature on a rare pathology could help differentiate between rare and very rare diseases. The objective of this work was to evaluate to what extent the number of publications can be used to predict the prevalence of a given pathology.

Methods

We queried Orphanet for the global prevalence class for all conditions for which it was available. For these pathologies, we cross-referenced the Orphanet, MeSH, and OMIM vocabularies to assess the number of publication available on Pubmed using three different query strategies (one proposed in the literature, and two built specifically for this study). We first studied the association of the number of publications obtained by each of these query strategies with the prevalence class, then their predictive ability.

Results

Class prevalence was available for 3128 conditions, 2970 had a prevalence class < 1/1,000,000, 41 of 1–9/1,000,000, 84 of 1–9/100,000, and 33 of 1–9/10,000. We show a significant association and excellent predictive performance of the number of publication, with an AUC over 94% for the best query strategy.

Conclusion

Our study highlights the link and the excellent predictive performance of the number of publications on the prevalence of rare diseases provided by Orphanet.
Clinical, humanistic, and economic burden of severe hemophilia B in the United States: Results from the CHESS US and CHESS US+ population surveys
Authors: Burke Tom , Asghar Sohaib , O’Hara Jamie , Sawyer Eileen K. , Li Nanxin
Published: March 2021

Background

Hemophilia B is a rare congenital bleeding disorder that has a significant negative impact on patients’ functionality and health-related quality of life. The standard of care for severe hemophilia B in the United States is prophylactic factor IX replacement therapy, which incurs substantial costs for this lifelong condition. Accurate estimates of the burden of hemophilia B are important for population health management and policy decisions, but have only recently accounted for current management strategies. The ‘Cost of Severe Hemophilia across the US: a Socioeconomic Survey’ (CHESS US) is a cross-sectional database of medical record abstractions and physician-reported information, completed by hematologists and care providers. CHESS US+ is a complementary database of completed questionnaires from patients with hemophilia. Together, CHESS US and CHESS US+ provide contemporary, comprehensive information on the burden of severe hemophilia from the provider and patient perspectives. We used the CHESS US and CHESS US+ data to analyze the clinical, humanistic, and economic burden of hemophilia B for patients treated with factor IX prophylaxis between 2017 and 2019 in the US.

Results

We conducted analysis to assess clinical burden and direct medical costs from 44 patient records in CHESS US, and of direct non-medical costs, indirect costs, and humanistic burden (using the EQ-5D-5L) from 57 patients in CHESS US+. The mean annual bleed rate was 1.73 (standard deviation, 1.39); approximately 9% of patients experienced a bleed-related hospitalization during the 12-month study period. Nearly all patients (85%) reported chronic pain, and the mean EQ-5D-5L utility value was 0.76 (0.24). The mean annual direct medical cost was $614,886, driven by factor IX treatment (mean annual cost, $611,971). Subgroup analyses showed mean annual costs of $397,491 and $788,491 for standard and extended half-life factor IX treatment, respectively. The mean annual non-medical direct costs and indirect costs of hemophilia B were $2,371 and $6,931.

Conclusions

This analysis of patient records and patient-reported outcomes from CHESS US and CHESS US+ provides updated information on the considerable clinical, humanistic, and economic burden of hemophilia B in the US. Substantial unmet needs remain to improve patient care with sustainable population health strategies.
About classical molecular genetics, cytogenetic and molecular cytogenetic data not considered by Genome Reference Consortium and thus not included in genome browsers like UCSC, Ensembl or NCBI
Authors: Liehr Thomas
Published: March 2021

Background

The Genome Reference Consortium (GRC) has according to its own statement the “mission to improve the human reference genome assembly, correcting errors and adding sequence to ensure it provides the best representation of the human genome to meet basic and clinical research needs”. Data from GRC is included in genome browsers like UCSC (University of California, Santa Cruz), Ensembl or NCBI (National Center for Biotechnology Information) and are thereby bases for scientific and diagnostically working human genetic community.

Method

Here long standing knowledge deriving from classical molecular genetic, cytogenetic and molecular cytogenetic data, not being considered yet by GRC was revisited.

Results

There were three major points identified: (1) GRC missed to including three chromosomal subbands, each, for 1q32.1, 2p21, 5q13.2, 6p22.3 and 6q21, which were defined by International System for Human Cytogenetic Nomenclature (ISCN) already back in 1980s; instead GRC included additional 6 subbands not ever recognized by ISCN. (2) GRC defined 34 chromosomal subbands of 0.1 to 0.9 Mb in size, while it is general agreement of cytogeneticists that it unlikely to detect chromosomal aberrations below 1–2 Mb in size by GTG-banding. And (3): still all sequences used in molecular cytogenetic routine diagnostics to detect heterochromatic and/ or pericentromeric satellite DNA sequences within the human genome are not included yet into human reference genome. For those sequences, localization and approximate sizes have been determined in the 1970s to 1990, and if included at least ~ 100 Mb of the human genome sequence could be added to the genome browsers.

Conclusion

Overall, taking into account the here mentioned points and correcting and including the data will definitely provide to the still not being completely finished mapping of the human genome.
Introducing fairness in Norwegian air ambulance base location planning
Authors: Jagtenberg Caroline J. , Vollebergh Maaike A. J. , Uleberg Oddvar , Røislien Jo
Published: March 2021

Background

A primary task of the Norwegian helicopter emergency medical services (HEMS) is to provide advanced medical care to the critical ill and injured outside of hospitals. Where HEMS bases are located, directly influences who in the population can be reached within a given response time threshold and who cannot. When studying the locations of bases, the focus is often on efficiency, that is, maximizing the total number of people that can be reached within a given set time. This approach is known to benefit people living in densely populated areas, such as cities, over people living in remote areas. The most efficient solution is thus typically not necessarily a fair one. This study aims to incorporate fairness in finding optimal air ambulance base locations.

Methods

We solve multiple advanced mathematical optimization models to determine optimal helicopter base locations, with different optimization criteria related to the level of aversion to inequality, including the utilitarian, Bernoulli-Nash and iso-elastic social welfare functions. This is the first study to use the latter social welfare function for HEMS.

Results

Focusing on efficiency, a utilitarian objective function focuses on covering the larger cities in Norway, leaving parts of Norway largely uncovered. Including fairness by rather using an iso-elastic social welfare function in the optimization avoids leaving whole areas uncovered and in particular increases service levels in the north of Norway.

Conclusions

Including fairness in determining optimal HEMS base locations has great impact on population coverage, in particular when the number of base locations is not enough to give full coverage of the country. As results differ depending on the mathematical objective, the work shows the importance of not only looking for optimal solutions, but also raising the essential question of ‘optimal with respect to what’.
Recruitment patterns in a large international randomized controlled trial of perioperative care in cancer patients
Authors: Gazendam Aaron , Bozzo Anthony , Schneider Patricia , Giglio Victoria , Wilson David , Ghert Michelle
Published: March 2021

Introduction

The Prophylactic Antibiotic Regimens in Tumor Surgery (PARITY) randomized controlled trial (RCT) was the first study to prospectively enroll and randomize orthopedic oncology patients in multiple centers internationally. The objective of this study was to describe recruitment patterns, to examine the differences in enrollment across different PARITY sites, and to identify variables associated with differing levels of recruitment.

Methods

Data from this study was obtained from the PARITY trial Methods Center and records of correspondence between the Methods Center and recruiting sites. We performed descriptive statistics to report the recruitment patterns over time. We compared recruitment, time to set up, and time to enroll the first patient between North American and international sites, private and public healthcare models, and the presence or absence of research personnel. Two-tailed non-paired t tests were performed to test average monthly recruitment rates between groups.

Results

A total of 602 patients from 36 North American and 12 international sites were recruited from 2013 to 2019. North American sites were able to become fully enrollment-ready at an average of 19.5 months and international sites at an average of 27 months. Once enrolling, international sites were able to enroll 0.59 patients per/month whereas North American sites averaged a monthly recruitment rate of 0.2 patients/month once enrolling. Sites with research personnel reached enrollment-ready status at an average of 19.3 months and sites without research support at an average of 30.3 months. Once enrolling, the recruitment rate was 0.28 patients/month and 0.2 patients per month for sites with and without research support, respectively. Publicly funded sites had a monthly enrollment of 0.4 patients/month whereas privately funded sites had a monthly enrollment rate of 0.17 patients/month.

Conclusions

As a collaborative group, the PARITY investigators increased the pace of recruitment throughout the trial, likely by increasing the number of active sites. The longer time to start-up at international sites may be due to the complex governing regulations of pharmaceutical trials. Nevertheless, international sites should be considered essential as they recruited significantly more patients per month once active. The absence of research support personnel may lead to delays in the time to start-up. The results of the current study will provide guidance for choosing which sites to recruit for participation in future collaborative clinical trials in orthopedic oncology and other surgical specialties.

Trial registration

ClinicalTrials.gov NCT01479283. Prospectively registered on November 24, 2011
Systematic review of outcome domains and instruments used in designs of clinical trials for interventions that seek to restore bilateral and binaural hearing in adults with unilateral severe to profound sensorineural hearing loss (‘single-sided deafness’)
Authors: Katiri Roulla , Hall Deborah A. , Killan Catherine F. , Smith Sandra , Prayuenyong Pattarawadee , Kitterick Pádraig T.
Published: March 2021

Background

This systematic review aimed to identify, compare and contrast outcome domains and outcome instruments reported in studies investigating interventions that seek to restore bilateral (two-sided) and/or binaural (both ears) hearing in adults with single-sided deafness (SSD). Findings can inform the development of evidence-based guidance to facilitate design decisions for confirmatory trials.

Methods

Records were identified by searching MEDLINE, EMBASE, PubMed, CINAHL, ClinicalTrials.gov, ISRCTN, CENTRAL, WHO ICTRP and the NIHR UK clinical trials gateway. The search included records published from 1946 to March 2020. Included studies were those as follows: (a) recruiting adults aged 18 years or older diagnosed with SSD of average threshold severity worse than 70 dB HL in the worse-hearing ear and normal (or near-normal) hearing in the better-hearing ear, (b) evaluating interventions to restore bilateral and/or binaural hearing and (c) enrolling those adults in a controlled trial, before-and-after study or cross-over study. Studies that fell just short of the participant eligibility criteria were included in a separate sensitivity analysis.

Results

Ninety-six studies were included (72 full inclusion, 24 sensitivity analysis). For fully included studies, 37 exclusively evaluated interventions to re-establish bilateral hearing and 29 exclusively evaluated interventions to restore binaural hearing. Overall, 520 outcome domains were identified (350 primary and 170 secondary). Speech-related outcome domains were the most common (74% of studies), followed by spatial-related domains (60% of studies). A total of 344 unique outcome instruments were reported. Speech-related outcome domains were measured by 73 different instruments and spatial-related domains by 43 different instruments. There was considerable variability in duration of follow-up, ranging from acute (baseline) testing to 10 years after the intervention. The sensitivity analysis identified no additional outcome domains.

Conclusions

This review identified large variability in the reporting of outcome domains and instruments in studies evaluating the therapeutic benefits and harms of SSD interventions. Reports frequently omitted information on what domains the study intended to assess, and on what instruments were used to measure which domains.

Trial registration

The systematic review protocol is registered on PROSPERO (International Prospective Register of Systematic Reviews): Registration Number CRD42018084274. Registered on 13 March 2018, last revised on 7th of May 2019.
Action observation treatment-based exoskeleton (AOT-EXO) for upper extremity after stroke: study protocol for a randomized controlled trial
Authors: Chen Zejian , Xia Nan , He Chang , Gu Minghui , Xu Jiang , Han Xiaohua , Huang Xiaolin
Published: March 2021

Background

Stroke produces multiple symptoms, including sensory, motor, cognitive and psychological dysfunctions, among which motor deficit is the most common and is widely recognized as a major contributor to long-term functional disability. Robot-assisted training is effective in promoting upper extremity muscle strength and motor impairment recovery after stroke. Additionally, action observation treatment can enhance the effects of physical and occupational therapy by increasing neural activation. The AOT-EXO trial aims to investigate whether action observation treatment coupled with robot-assisted training could enhance motor circuit activation and improve upper extremity motor outcomes.

Methods

The AOT-EXO trial is a multicentre, prospective, three-group randomized controlled trial (RCT). We will screen and enrol 132 eligible patients in the trial implemented in the Department of Rehabilitation Medicine of Tongji Hospital, Optical Valley Branch of Tongji Hospital and Hubei Province Hospital of Integrated Chinese & Western Medicine in Wuhan, China. Prior to study participation, written informed consent will be obtained from eligible patients in accordance with the Declaration of Helsinki. The enrolled stroke patients will be randomized to three groups: the CT group (conventional therapy); EXO group (exoskeleton therapy) and AOT-EXO group (action observation treatment-based exoskeleton therapy). The patients will undergo blinded assessments at baseline, post-intervention (after 4 weeks) and follow-up (after 12 weeks). The primary outcome will be the Fugl-Meyer Assessment for Upper Extremity (FMA-UE). Secondary outcomes will include the Action Research Arm Test (ARAT), modified Barthel Index (MBI), kinematic metrics assessed by inertial measurement unit (IMU), resting motor threshold (rMT), motor evoked potentials (MEP), functional magnetic resonance imaging (fMRI) and safety outcomes.

Discussion

This trial will provide evidence regarding the feasibility and efficacy of the action observation treatment-based exoskeleton (AOT-EXO) for post-stroke upper extremity rehabilitation and elucidate the potential underlying kinematic and neurological mechanisms.

Trial registration

Chinese Clinical Trial Registry ChiCTR1900026656. Registered on 17 October 2019.