Clinical Research Directory

BLOOD-dose: A Platform Trial Evaluating Dose Optimization in Hematological Diseases.

BLOOD-dose is a multicentre, adaptive, randomized, multidomain platform trial designed to optimize treatment dosing strategies in adult patients with haematological diseases. The BLOOD-dose core protocol outlines the overall clinical trial design that applies to all included interventions, while domain-specific appendices (DSA) detail the unique characteristics of each domain and specify domain-specific interventions. New domains will be incorporated over time to address distinct dose-optimization research questions across different haematological conditions and interventions.

This Study Aims to Clarify the Prevalence and Characteristics of Neuropathy, Along With Associated Paraclinical Findings in Patients With Waldenström's Macroglobulinemia (WM) in a Cohort of WM Patients to Optimize the Diagnostic Process

Neuropathy severely reduces patients' quality of life due to sensory loss, chronic neuropathic pain, and loss of mobility of arms and legs. Given the diverse origins of neuropathy, it is critical to identify its specific causes, particularly when effective treatments are available. Neuropathy is a frequent morbidity in Waldenström's macroglobulinemia (WM), a specific type of lymphoma caused by infiltration of clonal lymphoplasmocytic B cells in the bone marrow with the presence of IgM paraprotein. WM associated neuropathy is largely undescribed. The few existing studies are mostly retrospective indicating the neuropathy has a heterogenic pathophysiology and diverse clinical appearance from mild sensory neuropathy to aggressive with loss of ambulation and development of chronic neuropathic pain within weeks to months. With treatment of WM the speed of the disease progression including the related neuropathy can be halted. Few studies and clinical experience indicate that the nerve damage induced by WM might remit if treatment is initiated early in the course of the disease. Thus, there is need for timely interventions to reduce chronic disabilities. However, even for an experienced neurologist, it can be difficult to identify whether the neuropathy is caused by WM or other causes where treatment is not indicated. This project aims to investigate the prevalence and underlying mechanisms of neuropathy in patients with WM to help speed up the diagnostic process and thus help slow down the irreversible nerve damage that these patients experience.

Early Assessment of Lymphoma Treatment Response Using Phased Variant Analysis With Next-Generation Sequencing

Lymphoma is a prevalent lymphoid malignancy globally and in Taiwan. Large B-cell lymphoma (LBCL) is the most common subtype of aggressive B-cell lymphoma. LBCL's aggressive nature manifests through extranodal involvement, severe symptoms, and relative refractoriness to therapies, leading to a 5-year overall survival rate of 60-70% across developed countries and poorer outcomes in high-risk patients with primary refractory disease. Chemoimmunotherapy remains the primary treatment for LBCL, requiring comprehensive assessment through clinical and imaging examinations, biomarkers, and molecular testing. Currently, computed tomography (CT) and positron emission tomography (PET) scans are the standard modalities for treatment response evaluation, though their radioactive nature calls for the development of safer alternatives. Circulating tumor DNA (ctDNA) analysis has emerged as a promising field, providing insights into tumor molecular characteristics, clinical status, and treatment response by analyzing DNA fragments released from tumor cells into the bloodstream. Dynamic monitoring of ctDNA during treatment can effectively gauge therapeutic efficacy-decreasing ctDNA concentrations suggest successful treatment, while increasing levels may indicate treatment failure or tumor recurrence. The detection of ctDNA has been much improved through advances in next-generation sequencing (NGS) technologies, particularly taking advantage of analyzing phased variants, consecutive gene mutations on the same chromosome, enhances the sensitivity and specificity.