Needs of amyloidosis patients and their care providers: design & first results of the MY-NEED research and care program.

Background: Amyloidosis represents a rare yet heterogeneous multi-system disorder associated with a grave prognosis and an enormous psycho-emotional strain on patients, relatives, and caregivers. We here present the overall study design and first results of MY-NEED, a research program aiming to systematically assess the needs of patients suffering from amyloidosis, their relatives and health care professionals (HCPs), and develop an amyloidosis-specific care approach.

Methods: MY-NEED uses a mixed-methods approach including focus groups (step 1), a questionnaire-based broad evaluation within the local amyloidosis patient collective (step 2), and the development of a needs-adapted care concept (step 3).

Pre-assessment of patients with suspected axial spondyloarthritis combining student-led clinics and telemedicine: a qualitative study.

Objective: Patients referred to rheumatologists are currently facing months of inefficient waiting time due to the increasing demand and rising workforce shortage. We piloted a pre-assessment of patients with suspected axial spondyloarthritis (axSpA) combining student-led clinics and telemedicine (symptom assessment, symptom monitoring and at-home capillary self-sampling) to improve access to rheumatology care. The aim of this study was to explore (1) current challenges accessing axSpA care and (2) patients' first-hand experiences.

Neurological outcome in long-chain hydroxy fatty acid oxidation disorders.

Objective: This study aims to elucidate the long-term benefit of newborn screening (NBS) for individuals with long-chain 3-hydroxy-acyl-CoA dehydrogenase (LCHAD) and mitochondrial trifunctional protein (MTP) deficiency, inherited metabolic diseases included in NBS programs worldwide.

Methods: German national multicenter study of individuals with confirmed LCHAD/MTP deficiency identified by NBS between 1999 and 2020 or selective metabolic screening. Analyses focused on NBS results, confirmatory diagnostics, and long-term clinical outcomes.

Real-world analysis of teclistamab in 123 RRMM patients from Germany.

Teclistamab, a B-cell maturation antigen (BCMA) × CD3 directed bispecific antibody, has shown high response rates and durable remissions in the MAJESTEC-1 trial in patients with relapsed and refractory multiple myeloma (RRMM). We retrospectively assessed efficacy and tolerability in 123 patients treated at 18 different German centers to determine whether outcome is comparable in the real-world setting. Most patients had triple-class (93%) or penta-drug (60%) refractory disease, 37% of patients had received BCMA-directed pretreatment including idecabtagene vicleucel (ide-cel) CAR-T cell therapy (21/123, 17.

Sarcoidosis-Associated Pulmonary Hypertension.

Pulmonary hypertension is a life-threatening complication of advanced sarcoidosis. Many mechanisms can cause an elevation of pulmonary pressure in sarcoidosis, leading to precapillary or postcapillary pulmonary hypertension. Sarcoidosis-associated pulmonary hypertension contributes to severe exertional dyspnea, reduced exercise capacity, and notably compromised the survival.

The ratio of cytochrome c oxidase subunit 4 isoform 4I1 and 4I2 mRNA is changed in permanent atrial fibrillation.

Aims: The conditions of hypoxia are suggested to induce permanent atrial fibrillation (AF). The regulation of COX4I2 and COX4I1 depends on oxygen availability in tissues. A role of COX4I2 in the myocardium of AF patients is supposed for pathogenesis of AF and subsequent alterations in the electron transfer chain (ETC) under hypoxia.

From the microscopic to the macroscopic: clinical-radiological-pathological correlation in pulmonary hypertension.

https://bit.ly/3RtiFVK

Clinical-radiological-pathological correlation in pulmonary arterial hypertension.

Pulmonary hypertension (PH) is defined by the presence of a mean pulmonary arterial pressure >20 mmHg. Current guidelines describe five groups of PH with shared pathophysiological and clinical features. In this paper, the first of a series covering all five PH classification groups, the clinical, radiological and pathological features of pulmonary arterial hypertension (PAH) will be reviewed.

A German-Wide Systematic Study on Mobilization and Collection of Hematopoietic Stem Cells in Poor Mobilizer Patients with Multiple Myeloma prior to Autologous Stem Cell Transplantation.

Introduction: In patients with a clinical indication for autologous hematopoietic stem cell transplantation (ASCT), sufficient mobilization of CD34 precursor cells into peripheral blood is essential to ensure adequate hematopoietic stem cell (HSC) collection prior to intensive therapy. However, with standard granulocyte-colony stimulating factor (G-CSF)-based mobilization schemes, an important minority of patients fail to mobilize sufficient (e.g.

ChatGPT Versus Consultants: Blinded Evaluation on Answering Otorhinolaryngology Case-Based Questions.

Background: Large language models (LLMs), such as ChatGPT (Open AI), are increasingly used in medicine and supplement standard search engines as information sources. This leads to more "consultations" of LLMs about personal medical symptoms.

Objective: This study aims to evaluate ChatGPT's performance in answering clinical case-based questions in otorhinolaryngology (ORL) in comparison to ORL consultants' answers.

Automated Machine Learning Analysis of Patients With Chronic Skin Disease Using a Medical Smartphone App: Retrospective Study.

Background: Rapid digitalization in health care has led to the adoption of digital technologies; however, limited trust in internet-based health decisions and the need for technical personnel hinder the use of smartphones and machine learning applications. To address this, automated machine learning (AutoML) is a promising tool that can empower health care professionals to enhance the effectiveness of mobile health apps.

Objective: We used AutoML to analyze data from clinical studies involving patients with chronic hand and/or foot eczema or psoriasis vulgaris who used a smartphone monitoring app.

Supporting the Bereaved in the COVID-19 Era: A Scoping Review of Interventions.

People whose family member(s) friend(s) have died from COVID-19 or other causes have been deeply affected by the physical and social restrictions imposed during the pandemic. These limitations have affected end-of-life care and support for the bereaved. The purpose of this review is to identify: the published studies of evaluated programs about interventions for people who have experienced bereavement during the COVID-19 pandemic, and to develop recommendations for researchers and policy makers.

Digital Approaches for Myositis.

Purpose Of Review: This article serves as a comprehensive review, focusing on digital approaches utilized in the diagnosis, monitoring, and treatment of patients with idiopathic inflammatory myopathies (IIM). The authors critically assess the literature published in the last three years, evaluating the advancements and progress achieved in this specific domain.

Recent Findings: Remarkable strides have been made in the realm of digital diagnostic support, particularly in image analysis and clinical prediction models, showing promise in aiding the diagnosis of IIM.

Cancer patients' expectations of advance care planning: A typological content analysis of qualitative interviews.

Background: Advance care planning (ACP) can help to elicit cancer patients' preferences in a discussion process to promote person-centred medical decision-making. Expectations are known to be highly relevant determinants of decisional processes. So far, however, little is known about cancer patients' expectations of ACP that lead to acceptance or refusal of the programme.

Mobilization and Hematopoietic Stem Cell Collection in Poor Mobilizing Patients with Lymphoma: Final Results of the German OPTIMOB Study.

Introduction: Successful mobilization and collection of peripheral hematopoietic stem cells (HSCs) are necessary for lymphoma patients eligible for myeloablative chemotherapy with subsequent autologous stem cell transplantation (ASCT). Albeit G-CSF alone or combined with chemotherapy is well-established methods for HSC mobilization, up to 40% of the patients fail to mobilize (poor mobilizer, PM). Plerixafor (PLX) is commonly used in PM patients resulting in increased migration of HSCs into peripheral blood and thus improves the collection outcome.

Rate of Complications after Hip Fractures Caused by Prolonged Time-to-Surgery Depends on the Patient's Individual Type of Fracture and Its Treatment.

Introduction: Hip fractures are common injuries in the elderly and are usually treated with timely surgery. While severe postoperative complications are reported for up to 10% of patients, many studies identified predictive factors for the occurrence of complications postoperatively. A controversially discussed factor is "time-to-surgery".

B-Mode Ultrasound and Contrast-Enhanced Ultrasound for the Detection of Splenic Involvement in Hodgkin Lymphoma: A Retrospective Analysis of 112 Patients.

Purpose: To assess splenic involvement using B-mode ultrasound (US) and contrast-enhanced ultrasound (CEUS) compared with standard imaging with contrast-enhanced computerized tomography (CT) / 18-fluorodeoxyglucose positron-emission tomography (PET-CT) in patients with Hodgkin lymphoma.

Materials And Methods: Imaging data from 112 patients from 12/2003 to 10/2022 with histologically confirmed Hodgkin lymphoma during staging or relapse were analyzed for splenic lymphoma involvement. In all patients, standard imaging (CT/PET-CT), along with B-mode US and CEUS examinations, was performed.

Implementation and evaluation of a palliative care training unit for EMS providers.

Background: The prevalence of children with life-limiting conditions (LLCs) is rising. It is characteristic for these children to require 24/7 care. In emergencies, families must decide to call the emergency medical service (EMS) or a palliative care team (PCT)-if available.

Isatuximab, Carfilzomib, Lenalidomide, and Dexamethasone for the Treatment of High-Risk Newly Diagnosed Multiple Myeloma.

Purpose: The GMMG-CONCEPT trial investigated isatuximab, carfilzomib, lenalidomide, and dexamethasone (Isa-KRd) in transplant-eligible (TE) and transplant-noneligible (TNE) patients with newly diagnosed multiple myeloma (NDMM) with exclusively high-risk disease for whom prospective trials are limited, aiming to induce minimal residual disease (MRD) negativity.

Methods: This academic, investigator-initiated, multicenter, phase II trial enrolled patients with high-risk NDMM (HRNDMM) defined by mandatory International Staging System stage II/III combined with del17p, t(4;14), t(14;16), or more than three 1q21 copies as high-risk cytogenetic aberrations (HRCAs). Patients received Isa-KRd induction/consolidation and Isa-KR maintenance.