Cryoglobulinemia

Researchers are establishing a prospective group of patients with plasma cell disorders PCDs to better understand these conditions and their treatments. This study focuses on various PCDs including multiple myeloma, amyloidosis, and other related disorders. It aims to explore how minimal residual disease MRD status relates to patient prognosis and investigate the role of the tumor microenvironment TME in the diseases development and progression. Patients with confirmed PCDs who are hospitalized and willing to participate will be followed over time. Biological samples such as peripheral blood, bone marrow aspirate, and urine will be collected before and after treatment to support future research. The study does not involve any intervention and participants will be regularly assessed at baseline, then at 1, 3, 6, and 12 months after treatment, followed by yearly visits. During the study, clinical and laboratory data along with biological samples will be gathered to analyze disease features and treatment responses. Follow-up visits will monitor patient progress and outcomes for up to ten years. The main outcome is to maintain a comprehensive cohort and understand the relationship between MRD and prognosis in PCD patients, while also supporting further studies on tumor clone evolution and disease mechanisms.

This research aims to better understand cutaneous vasculitis by studying skin lesions in patients with various types of idiopathic vasculitis. It involves a multi-center approach with both primary vasculitis care providers and dermatologists working together to select suitable patients and collect lesion samples for analysis. The study focuses on evaluating the histopathology and gene expression profiles transcriptome of these skin lesions. Participants with active vasculitis skin lesions will undergo a punch biopsy to collect tissue samples. These samples will be examined using standardized methods to assess the histopathology and transcriptomic characteristics of the cutaneous vasculitis. The study does not involve any experimental treatments but gathers detailed clinical data and biopsy specimens for analysis. Participants will be evaluated by specialists, who will collect clinical information and perform skin biopsies at affected sites. The study will review clinical data linked to biopsy samples over one year. Researchers will monitor the samples to better characterize the disease at the tissue level. The total participation time varies, with follow-up focused on the evaluation of biopsy results and clinical data.

Researchers are conducting an observational study to collect blood samples from healthy adults aged 18 to 90 years. The goal is to support research on vascular and immune health by establishing normal biological parameters and validating technical assays. This study focuses on healthy individuals without vascular or immune diseases to provide baseline data for future research. Participants will undergo a telehealth or telephone visit to review their medical history. They will then visit the clinical center where about 10 tablespoons of blood will be drawn from a vein in the arm or hand. The blood samples may be used for various tests including complete blood counts, blood typing, genetic studies, and stored for future research. Participants may return for repeat blood collections and medical history reviews for up to 10 years. During the study, participants will have their health assessed through blood tests and medical history reviews at initial and follow-up visits. Researchers will measure vascular biomarkers and conduct mechanistic investigations to improve understanding of vascular inflammation and thrombosis. Participants receive 50 for each blood collection visit, and their involvement may last up to 10 years with periodic visits as they choose.

Researchers are collecting information from patients with rare kidney diseases to support research and improve care. This National Registry of Rare Kidney Diseases RaDaR aims to develop clinical guidelines, audit treatments and outcomes, and help develop future therapies by gathering comprehensive data. Rare kidney diseases often have genetic causes and affect patients from childhood into adulthood, but their rarity makes research and treatment development challenging. The registry gathers clinical data and biological samples from various rare kidney disease groups, each focusing on conditions like Alport Syndrome, APRT Deficiency, Polycystic Kidney Disease, and many others. It connects patients and clinicians and allows patients to contribute information about their quality of life. This infrastructure enables identification of patient groups for clinical trials, biomarker development, and genotype-phenotype studies. Participants provide clinical and disease-specific information over time, which supports epidemiological and translational research. The registry facilitates patient recruitment for studies, improves patient and clinician education, and provides access to current knowledge about rare kidney diseases. The registry is ongoing and primarily includes UK patients but also accepts international participants consented through UK NHS hospitals.

Researchers are studying amyotrophic lateral sclerosis ALS patients who have anti-NRIP autoantibodies in their plasma. This trial aims to evaluate the use of plasmapheresis as a treatment to lower these autoantibody levels, which are linked to faster motor decline and higher mortality. The study is a single group design enrolling 20 ALS patients with these antibodies to assess the treatments potential effects and safety. Participants will receive three plasmapheresis courses spaced three months apart to reduce plasma anti-NRIP autoantibody concentrations. After the treatment phase, the study will follow patients for an additional six months to monitor longer-term outcomes. The intervention involves using plasmapheresis devices to remove the harmful autoantibodies from the blood. Throughout the study, participants will undergo serial examinations and assessments to track changes in disease progression and severity. Outcome measures include the ALS Functional Rating Scale-Revised ALSFRS-R at various timepoints up to 15 months, muscle strength tests, walking distance, quality of life scores, respiratory capacity, muscle action potentials, motor unit estimates, and neurofilament levels. Safety is monitored by recording any side effects related to plasmapheresis. The total study duration per participant covers the treatment period plus follow-up assessments.

Researchers are creating a registry of patients with plasma cell disorders PCDs, such as multiple myeloma, to collect detailed assessments over time. The study aims to understand patient care patterns and satisfaction with these assessments, and to support future research in this patient group. An optional blood sample may be collected to study a genetic aging marker called p16INK4a. Participants complete a comprehensive assessment at the start and at regular intervals afterward. This assessment covers their ability to live independently, other medical conditions, cognitive and psychological health, social support, medications, and nutrition. Researchers also review patients medical records to gather more information. During the study, patients are asked to complete questionnaires and assessments repeatedly to track changes over time. The primary outcomes include building the registry and completing these assessments over ten years. Secondary outcomes focus on patient response rates to the assessment and their satisfaction. The study is observational and involves no experimental treatments, with participation lasting up to ten years.

This research aims to understand the experiences of women with vasculitis who become pregnant. It focuses on assessing the severity of their vasculitis, their pregnancy-related experiences, and the outcomes of their pregnancies. The study is observational and seeks to gather detailed information through surveys over time. Women aged 18 to 50 years who have a diagnosis of vasculitis and are currently pregnant will be invited to participate. They will complete online questionnaires at several points at study entry, during the second trimester, the third trimester, and after giving birth. These surveys take about 20 minutes each and are available in multiple languages including Spanish, Portuguese, Italian, and Turkish. Participants will provide information through these surveys, which will be securely stored and later analyzed by the study team. The main outcome measured is the unique pregnancy characteristics among women with vasculitis over a four-year period. This research involves no in-person visits and allows participants to contribute from home through their online responses.