Gene Therapy for Chronic Granulomatous Disease in Korea
Chronic Granulomatous DiseaseThe purpose of this study is to evaluate the safety and efficacy of administration of autologous hematopoietic stem cells transduced with MT-gp91 retroviral vector for patients with X-linked chronic granulomatous disease.
Plerixafor/G-CSF as Additional Agents for Conditioning Before HSCT in CGD Patients
Chronic Granulomatous DiseaseTreatment Study to assess of safety and efficiency of conditioning with Plerixafor and G-CSF as additional agents for prevention of graft failure after transplantation in patients with chronic granulomatous disease
Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation...
Chronic Granulomatous DiseaseIn this study the investigators plan to use optimal busulfan dose through pharmacokinetic study in stem cell transplantation of CGD patients.
Granuloma Annulare Treated With Rifampin, Ofloxacin, and Minocycline Combination Therapy
Granuloma AnnulareGranuloma annulare is a benign, usually self-limiting, cutaneous dermatosis, which typically presents as asymptomatic, flesh-colored or red papules, frequently arranged in an annular pattern on the distal extremities. Although localized granuloma annulare is most commonly observed, a generalized or disseminated form can occur. The etiology of granuloma annulare is unknown, however, multiple inciting factors have been proposed. The purpose of the investigators is to review the outcomes of treatment of granuloma annulare (GA) resistant to standard regimens that resolved after a 3-months treatment course of monthly rifampin, ofloxacin, and minocycline (ROM) therapy, successfully used to treat paucibacillary leprosy, a disease with clinical and histopathologic similarity to GA.
Learning and Behavior Problems in Children With Chronic Granulomatous Disease and Related Disorders...
Chediak Higashi SyndromeChronic Granulomatous Disease2 moreThis study will try to determine what causes learning, behavioral and emotional problems in children with chronic granulomatous disease (GCD) and other phagocyte disorders. (Phagocytes are a type of white blood cell.) Children with these disorders have frequent severe infections that require hospitalization, sometimes for long periods of time. Many of them also have problems with school, learning, behavior, anxiety and depression. This study will explore whether these latter problems are a direct result of the illness itself or are a consequence of frequent, long hospitalizations, or are due to other factors. Test findings in these children will be compared with those of children with cystic fibrosis-another disease that causes frequent infections requiring prolonged hospitalization. Patients age 2 or older with GCD or other phagocytic disorders or cystic fibrosis may be eligible for this study. Participants (or a parent or guardian) will complete questionnaires including personal information such as age, gender and marital status, a family medical history, and information on their illness. Patients will be given various psychological and intelligence tests, and they and their parents or guardians will be interviewed by a child psychiatrist. The tests and interviews take a total of about 5 hours and are given in two or three separate sessions. The tests may reveal problems such as learning disorders, attention-deficit hyperactivity disorder, anxiety, or depression. If any of these problems are identified, appropriate referrals will be made for specialized services, such as special school placement, tutoring, or counseling.
Role of Interferon-gamma 1-b (IFN-γ) on Cells of the Innate Immune System: Functional, Biochemical...
Chronic Granulomatous DiseaseThe overall goal of the study is to investigate the functional, biochemical, and gene expression effects of Interferon-gamma 1-b (IFN-γ) on the neutrophils of patients with Chronic Granulomatous Disease (CGD). The investigators hypothesize that the clinical effects demonstrated in patients with CGD treated with IFN-γ (decreased number and severity of infections) are the result of biochemical processes and upregulation of specific genes, which lead to enhanced functionality of this immune cell population.
Simple Hygiene Versus Alcohol Based Usual Care for Umbilical Cord
InfectionSkin Diseases2 moreThe purpose of this study is to determinate whether in newborns just cleaning the umbilical stump when changing diapers (with water and soap if necessary)is effective in preventing local cord infections and not delaying cord separation as just as cord care alcohol, usually applied in our hospital and suggested to parents at dismission.
Gene Therapy for Chronic Granulomatous Disease
Granulomatous DiseaseChronicThe aim of the study is to evaluate the side effects and risks after infusion of retroviral gene corrected autologous CD34+ cells of the peripheral blood of chemotherapy conditioned (busulphan)patients with chronic granulomatous disease (CGD). Also gene corrected and functional active granulocytes in the peripheral blood and the engraftment in the bone marrow of the patients will be monitored an documented.
Evaluation of 41 Cases of Pediatric Granuloma Annulare
Granuloma AnnulareINTRODUCTION: Granuloma annulare is a non-infectious, granulomatous disease that can affect children and adults. Although there are many studies examining adult patients, there are limited studies examining pediatric granuloma annulare cases. It is aimed to examine the demographic, clinical, and pathological features of pediatric granuloma annulare cases in this study. MATERIALS and METHODS: This study was performed retrospectively in a single-center, tertiary dermatology hospital. Forty-one patients, under the age of 18 who were admitted to the dermatology outpatient clinic between 2008-2021 and diagnosed with granuloma annulare were scanned from the hospital database and patient photograph archive. Demographic characteristics, and clinical and histopathological features were evaluated.
Imaging of Active Granulomas With [18F]FDG and Selected Inflammatory PET Tracers in Pulmonary Sarcoidosis...
Pulmonary SarcoidosisThe goal of the current study is to evaluate the potential of the selected PET tracers to detect sarcoidotic lesions in lungs. The tracers are already in clinical use for the detection of certain inflammatory processes or malignant tumors, and their targets present similarities with molecular mechanisms of sarcoidosis.