Treating to target in multiple sclerosis: Do we know how to measure whether we hit it?

Background And Purpose: The rapidly evolving landscape of effective treatment options in multiple sclerosis has led to a shift of treatment objectives towards a treat-to-target approach aiming to suppress disease activity below the level of detectability early during the disease. To enable treat-to-target, a thorough reappraisal of available outcome measures with respect to their ability in this regard is required.

Methods: To that end, we conducted a comprehensive systematic literature review of more than 1000 studies using PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analyses) 2020 methodology focusing on underlying evidence as well as utility and implementability in clinical practice.

Outcomes of melphalan 140 mg/m followed by autologous stem cell transplantation in multiple myeloma patients with co-morbidities: Single-centre experience.

High-dose melphalan followed by stem cell rescue is the standard consolidative therapy for transplant-eligible patients with multiple myeloma (MM) in the United Kingdom. A melphalan dose of 200 mg/m (Mel200) is considered the "gold standard" for autologous stem cell transplant (ASCT) conditioning for fit patients ≤70 years old; however, with a peak diagnosis incidence at 80-89 years old in the UK dose adjustments will be inevitable to limit toxicities. In this single-centre UK-based retrospective analysis, data was collected from patients with plasma cell dyscrasias who underwent a first reduced-intensity, Mel140, ASCT from 2006 to 2019, a total of 81 patients.

White matter variations in congenital adrenal hyperplasia: possible implications for glucocorticoid treatment.

Congenital adrenal hyperplasia has been reported to manifest with white matter aberrations. However, many previous studies included only small samples, restricted their analyses to females, lacked a control group and/or did not correct for brain size. Here, we examined the largest sample to date, comprising 53 male and female participants with congenital adrenal hyperplasia, who were matched with 53 male and female controls in terms of sex, age, education, and verbal intelligence.

Effects of Congenital Adrenal Hyperplasia (CAH) and Biological Sex on Brain Size.

Congenital Adrenal Hyperplasia (CAH) has been reported to involve structural alterations in some brain regions. However, it remains to be established whether there is also an impact on the size of the brain as a whole. Here, we compiled the largest CAH sample to date (n = 53), matched pair-wise to a control group (n = 53) on sex, age, and verbal intelligence.

Intraoperative margin assessment during radical prostatectomy: is microscopy frozen in time or ready for digital defrost?

Intraoperative frozen section (IFS) is used with the intention to improve functional and oncological outcomes for patients undergoing radical prostatectomy (RP). High resource requirements of IFS techniques such as NeuroSAFE may preclude widespread adoption, even if there are benefits to patients. Recent advances in fresh-tissue microscopic digital imaging technologies may offer an attractive alternative, and there is a growing body of evidence regarding these technologies.

Primary central nervous system lymphomas: EHA-ESMO Clinical Practice Guideline for diagnosis, treatment and follow-up.

This EHA-ESMO Clinical Practice Guideline provides key recommendations for managing primary DLBCL of the CNS.The guideline covers clinical, imaging and pathological diagnosis, staging and risk assessment, treatment and follow-up.Algorithms for first-line and salvage treatments are provided.

Cortical gyrification in women and men and the (missing) link to prenatal androgens.

Previous studies have reported sex differences in cortical gyrification. Since most cortical folding is principally defined in utero, sex chromosomes as well as gonadal hormones are likely to influence sex-specific aspects of local gyrification. Classic congenital adrenal hyperplasia (CAH) causes high levels of androgens during gestation in females, whereas levels in males are largely within the typical male range.

Prevention of complications in endourological management of stones: What are the basic measures needed before, during, and after interventions?

Objective: This narrative review aims to describe measures to minimise the risk of complications during percutaneous nephrolithotomy (PCNL), ureteroscopy, and retrograde intrarenal surgery.

Methods: A literature search was conducted from the PubMed/PMC database for papers published within the last 10 years (January 2012 to December 2022). Search terms included "ureteroscopy", "retrograde intrarenal surgery", "PCNL", "percutaneous nephrolithotomy", "complications", "sepsis", "infection", "bleed", "haemorrhage", and "hemorrhage".

International Alliance of Urolithiasis (IAU) guideline on staghorn calculi management.

Background: The stone burden based management strategy reported in the guidelines published by different associations is well known for a long time. Staghorn calculi, representing the largest burden and most complex stones, is one of the most challenging cases to practicing urologists in clinical practice. The International Alliance of Urolithiasis (IAU) has released a series of guidelines on the management of urolithiasis.

Treosulfan compared to busulfan in allogeneic haematopoietic stem cell transplantation for myelofibrosis: a registry-based study from the Chronic Malignancies Working Party of the EBMT.

We aimed to compare outcomes following treosulfan (TREO) or busulfan (BU) conditioning in a large cohort of myelofibrosis (MF) patients from the EBMT registry. A total of 530 patients were included; 73 received TREO and 457 BU (BU ≤ 6.4 mg/kg in 134, considered RIC, BU > 6.

Worldwide trends of practice and intervention in paediatric endourology: comparison of European versus Non-European responses.

Introduction: The area of paediatric endourology is unique and is recognised to be challenging, and it requires a certain level of focused training and expertise. Our aim was to conduct a worldwide survey in order to gain an overview regarding the current practice patterns for minimally invasive treatments of paediatric upper urinary tract stone patients.

Material And Methods: The survey was distributed between December 2021 and April 2022 through urology sections and societies in United Kingdom, Latin America and Asia.

Emergency laparotomy for abdominal catastrophes secondary to lymphoma: A systematic review.

Intestinal lymphomas can rarely present as abdominal catastrophes with perforation or small bowel obstruction. There is little data regarding their optimal surgical management and associated outcomes. We aimed to systematically review relevant published literature to assess the presentation, diagnosis, optimal surgical approach and associated post-operative outcomes.

Outcomes of CMML patients undergoing allo-HCT are significantly worse compared to MDS-a study of the CMWP of the EBMT.

Although CMML since long has been separated from MDS, many studies continue to evaluate the outcomes of both diseases after hematopoietic cell transplantation (allo-HCT) together. Data evaluating outcomes of a large CMML cohort after allo-HCT compared to MDS are limited. We aim to compare outcomes of CMML to MDS patients who underwent allo-HCT between 2010 and 2018.

Tamoxifen for the treatment of myeloproliferative neoplasms: A Phase II clinical trial and exploratory analysis.

Current therapies for myeloproliferative neoplasms (MPNs) improve symptoms but have limited effect on tumor size. In preclinical studies, tamoxifen restored normal apoptosis in mutated hematopoietic stem/progenitor cells (HSPCs). TAMARIN Phase-II, multicenter, single-arm clinical trial assessed tamoxifen's safety and activity in patients with stable MPNs, no prior thrombotic events and mutated JAK2, CALR or CALR peripheral blood allele burden ≥20% (EudraCT 2015-005497-38).