A novel conditioning regimen with pre-transplantation immunosuppression reduces the complication rates in hematopoietic stem cell transplantation in transfusion-dependent β-thalassemia.

Background: Allo-HSCT is a curative therapy for patients with transfusion-dependent thalassemia (TDT). The high incidence of transplant-related complications is becoming an obstacle to safe and effective unrelated donor (URD) transplantation.

Methods: In this retrospective study, we reported the survival outcomes and complications of transplantation in thalassemia patients using a novel regimen consisting of pre-transplantation immunosuppression (PTIS) and modified myeloablative conditioning based on intravenous busulfan, cyclophosphamide, fludarabine, and rabbit anti-human thymocyte immunoglobulin.

Clinical efficacy of efgartigimod combined with intravenous methylprednisolone in the acute phase of neuromyelitis optica spectrum disorders.

Background: Neuromyelitis Optica Spectrum Disorders (NMOSD) comprise a group of autoimmune-mediated, inflammatory, demyelinating central nervous system diseases caused by aquaporin-4 (AQP4) IgG autoantibodies. Efgartigimod is a human IgG Fc fragment that reduces antibody titers by targeting the neonatal Fc receptor (FcRn). This study documents the efficacy of efgartigimod combined with intravenous methylprednisolone (IVMP) in the acute phase of NMOSD.

Atypical Kawasaki disease with giant coronary artery aneurysms in a 2-month-old boy: a case report.

Background: Kawasaki disease is an acute inflammatory disorder primarily affecting medium-sized blood vessels, especially in infants and young children under 5 years old, characterized by inflammation of the arterial walls, including coronary arteries. While predominantly affecting young children, rarely Kawasaki disease is seen in adults. Reporting this case is crucial due to the patient's very young age and the disease's unusual presentation following vaccination, contributing to the limited literature on Kawasaki disease in very young infants.

Myasthenia gravis: understanding treatment patterns and direct medical costs in the Czech Republic.

Background: Myasthenia gravis (MG) is a rare autoimmune disorder with significant clinical implications, including life-threatening myasthenic crises and exacerbations. Understanding real-world treatment patterns, especially associated direct medical costs, is essential for the effective management of healthcare delivery.

Methods: We conducted a descriptive cohort study using health administrative claims data from the Czech Republic covering more than 1,500 prevalent MG patients.

Assessing hand motor function in chronic immune-mediated neuropathies: a proof-of-concept study using a data glove.

Background: Chronic immune-mediated neuropathies are clinically heterogeneous and require regular, objective, and multidimensional monitoring to individualize treatment. However, established outcome measures are insufficient regarding measurement quality criteria (e.g.

Case report: Chronic inflammatory demyelinating polyradiculoneuropathy with severe central nervous system demyelination: a clinicopathological study.

Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is an immune-mediated disease that mainly affects the peripheral nerves and nerve roots and typically presents with distal dominant motor and sensory disturbances as clinical symptoms. Central nervous system (CNS) demyelination with inflammation occurs infrequently in patients with CIDP. Here, we present a unique autopsy report of CIDP causing severe demyelination along the entire spinal cord.

Severe Transverse Myelitis Following Ankle Surgery Potentially Unmasking Late-Onset Systemic Lupus Erythematosus.

A 70-year-old female underwent open reduction and internal fixation (ORIF) of her right ankle following a mechanical trip and fell two weeks before hospital admission. Two weeks following surgery, the patient experienced sudden-onset bilateral anterior thigh paresthesias and burning mid-back pain. Over the ensuing two days, the patient developed bilateral lower extremity weakness, bilateral lower extremity numbness, and urinary retention with constipation, which led to hospital presentation.

Guillain-Barré syndrome.

Guillain-Barré syndrome (GBS) is a rare immune-mediated polyradiculoneuropathy. Patients typically develop rapidly progressive weakness and sensory deficits that can result in complete paralysis requiring mechanical ventilation. GBS is usually a monophasic disease in which an aberrant immune response to an infection or other trigger damages the peripheral nerves.

Cost-Effective and Sustainable Drug Use in Hospitals: A Systematic and Practice-Based Approach.

Background And Objective: Rising healthcare costs challenge the financial sustainability of healthcare systems. Interventional pharmacoeconomics has emerged as a vital discipline to improve the cost-effective and sustainable use of drugs in clinical practice. However, current efforts are often fragmented, highlighting the need for an integrated hospital-wide approach.

Early initiation of intravenous cyclophosphamide and one-year outcome in super-refractory cryptogenic-new onset refractory status epilepticus.

To explore the potential efficacy of early initiation of intravenous cyclophosphamide (IVCPA), we reviewed consecutive four cases of super-refractory cryptogenic-new onset refractory status epilepticus (C-NORSE) between 2015 and 2023. We compared functional outcomes at 3 months and 1 year after the onset between patients who received IVCPA within 20 days (early-treated) and those who received it later (late-treated). All patients (median age: 43 years) had a prodromal fever.

Early Antiviral Use and Supplemental Oxygen Decrease the Risk of Secondary Bacterial Infections: A Multicentre Nested Case-Control Study.

Objective: To evaluate the treatment strategies that dictate the host susceptibility to secondary bacterial infections during coronavirus disease 2019 (COVID-19).

Design: Setting and Participants: This nested case-control study was conducted in three general hospitals in China between December 1, 2022, to March 1, 2023. A total of 456 confirmed COVID-19 patients matched 1:2 (152 cases and 304 controls) based on age, sex, disease severity and age-adjusted Charlson Comorbidity Index (aCCI) using Propensity-Score Matching (PSM) were included.

Use of CRP/lymphocyte ratio as a predictor of treatment selection and mortality in COVID-19 patients in the intensive care unit.

This study primarily aimed to examine the significance of the C-reactive protein to lymphocyte ratio (CLR), a key marker of inflammation, in relation to the disease progression and management of COVID-19 patients admitted to the intensive care unit (ICU). A total of 464 patients aged 18 years or older, diagnosed with COVID-19 and admitted to the ICU between April 1, 2021, and February 1, 2022, were included in the study. Sociodemographic, laboratory, radiological, and clinical data were collected for each patient.

A Case of Refractory Kawasaki Disease With Yersinia pseudotuberculosis Infection Successfully Treated With Cefotaxime Following Immunosuppressive Therapy.

Kawasaki disease (KD) is a vasculitis mainly affecting children under five, with symptoms such as persistent fever, rash, red lips, strawberry tongue, conjunctivitis, and swollen hands and feet. Diagnosis is based on a fever lasting over five days plus at least four of these symptoms. Treatment includes intravenous immunoglobulin (IVIG) and aspirin to reduce complications, especially coronary artery issues.

SARS-CoV-2 infection-induced immune thrombocytopenia in a patient with orthotopic heart transplantation: A case report and literature review.

Unlabelled: Immune thrombocytopenia (ITP) is an acquired autoimmune disorder characterized by low platelet counts with increased risk of bleeding. In particular, ITP induced by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection has been increasingly reported. Since immunosuppressive regimens in organ transplantation are often the primary cause of increased risk of infection, ITP following solid organ transplantation has occasionally been observed.

Co-existence of autoimmune polyglandular syndrome type 3b and undifferentiated connective tissue disease with subacute combined degeneration of spinal cord in children: a case report and literature review.

Background: The clinical manifestations of subacute combined degeneration of spinal cord (SCD) in children are complex and vary greatly. Notably, some SCD patients may be complicated with autoimmune diseases, leading to high early misdiagnosis and missed diagnosis rates.

Case Presentation: In this study, a case involving an adolescent female with repetitive severe anemia, multiple joint swelling and pain in the left limbs, and paralysis of the bilateral lower limbs with serum vitamin B12 deficiency, polyglandular involvement, and various positive auto-antibodies (anti‑intrinsic factor antibody, anti‑parietal cell antibody, thyroid peroxidase antibody, thyroid globulin antibody and perinuclear anti‑neutrophil cytoplasmic antibody) is reported.

Evaluation of infants administered prophylactic intravenous immunoglobulin following exposure to measles.

Introduction: Intravenous immunoglobulin (IVIG) is an alternative for post-exposure prophylaxis if a vaccine is contraindicated and intramuscular immunoglobulin is unavailable. We retrospectively examined the effect of IVIG administration time on measles development in measles-contact infants younger than 6 months of age.

Methodology: Contact tracing of measles cases was performed by the Istanbul Public Health Directorate (IPHD) between August 24, 2012, and June 16, 2013.

Role of Pyroptosis in IVIG-Resistant Kawasaki Disease and the Establishment of a New Predictive Model.

Background: Intravenous immunoglobulin (IVIG) resistance may be an increased risk of coronary artery lesions which is the serious complication of Kawasaki disease (KD). Early and accurate identification of IVIG-resistant patients has an important clinical value.

Objective: To establish a new predicting model by detecting the pyroptosis markers with other clinical indicators.

Safety and Tolerability of Home Infusions in Down Syndrome Regression Disorder.

Purpose: Down syndrome regression disorder (DSRD) is a rare neuropsychiatric condition affecting otherwise healthy individuals with Down syndrome. Multiple studies on DSRD have revealed that immunotherapy with intravenous immunoglobulin (IVIg) is both safe and effective, although site of infusion has never been studied. This study sought to evaluate the safety and tolerability of IVIg in individuals with DSRD receiving home-based infusions.

The Use of Novel Therapies in the Management of Haemolytic Disease of the Fetus and Newborn (HDFN): Scientific Impact Paper No. 75.

Haemolytic disease of the fetus and newborn (HDFN) is a rare condition that causes a baby to develop anaemia while growing inside the woman; or after birth. Left untreated, this may lead to stillbirth or neonatal death. HDFN is caused when the pregnant woman's antibodies cross the placenta, enter the baby's circulation, and attach to proteins called antigens (inherited from the father) on the baby's haemoglobin containing red blood cells, and cause them to break apart, causing fetal anaemia.

Case report: Satralizumab as an adjunctive therapy for AQP-4 antibody and MOG antibody dual-negative optic neuritis in a third-trimester pregnancy case.

The treatment of demyelinating optic neuritis (DON) in pregnant patients is challenging, especially when there is poor or no response to intravenous methylprednisolone pulse (IVMP) therapy or adjunctive treatments such as intravenous immunoglobulin (IVIG) therapy. We herein report a case of a 28-year-old pregnant woman who experienced sequential severe vision loss in both eyes. She presented to a local hospital with the main complaint of sudden, painless vision loss in the left eye and was diagnosed with DON in the left eye.

Intravenous immunoglobulin therapy: usage patterns and response to treatment in Qatar over ten years.

Background: IVIg is a blood-derived antibody product initially designed as a replacement therapy in inborn errors of immunity (IEIs). However, over the last 50 years, IVIg has been used to treat a growing range of autoimmune, autoinflammatory, and secondary immunodeficiency disorders. The US FDA has licensed IVIg for use in the treatment of nine clinical indications; although, IVIg global usage extends to off-label indications with variable treatment responses.

Pain determinants and quality of life in Guillain-Barre syndrome: a prospective cohort study.

Background: Pain is a serious manifestation in both the acute and chronic stages of Guillain-Barre syndrome (GBS). We evaluated the frequency, characteristics and associated factors of pain and its impact on quality of life (QoL) among patients with GBS.

Methods: We enrolled 644 patients with GBS from prospective cohort studies in Bangladesh conducted between 2010 and 2024.