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- Background/Rationale: Epidemiological data about Primary Immunodeficiency Disorders (PIDD) in Kuwait is needed to better understand peculiarities and to compare them with other regions and ethnicity. - Study hypothesis: PIDD is relatively common in Kuwait compared to populations from different geographic areas. The distribution of PIDD in Kuwait is different from other geographic areas with more severe forms being more frequent. - Brief inclusion and exclusion criteria of study participants: PIDD patients presented at different clinics/hospital in Kuwait. Patients with secondary immunodeficiencies (drug induced, virus induced, and immunodeficiency associated with metabolic disorders... ect), will be excluded - Estimated sample size of the study: All patients who were registered in KNPIDR since 2004 will be included in the study along with the new patients who will be recruited during the study period. - Primary objectives: - Determine the prevalence and frequency of different PIDD in Kuwait - Identify clinical presentation patterns for PIDD in Kuwait - Identify natural history of PIDD in Kuwait - Help to asses epidemiology of PIDD in Kuwait - Determine particularities about PIDD affecting the population in Kuwait - Determine the health impact of PIDD in Kuwait - Development of strategies to improve the care and the quality of life of patients with PIDD
To assess the safety, efficacy, and pharmacokinetics of IGIV-SN in pediatric subjects with primary immunodeficiency humoral diseases (PHID)
The pathophysiology of primary immunodeficiencies (PID), which encompass a broad range of different diseases with susceptibility to infection and/or a deregulated inflammatory response, is poorly understood. Available treatments are often not specific for a distinct target and might be associated with side effects. To elucidate pathophysiology of different PIDs, stool, urine, blood, tissue biopsies and/or bone marrow will be collected and analysed for anti-microbial activity and inflammatory response. In a second step, targeted treatment for different PIDs might be developed preclinically and ex vivo according to underlying pathophysiology.
Analysis of DNA samples of patients with molecularly undetermined PID by whole exome/genome sequencing. Transcriptome analysis of patients with molecularly undetermined PID.
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially matched family donor. The study will assess whether immune cells, called T cells, from the family donor, that are specially grown in the laboratory and given back to the patient along with the stem cell transplant can help the immune system recover faster after transplant. As a safety measure these T cells have been programmed with a self-destruct switch so that they can be destroyed if they start to react against tissues (graft versus host disease).
Related donor Epstein-Barr Virus (EBV) specific cytotoxic T cells (CTLs) manufactured with the Miltenyi CliniMACS Prodigy Cytokine Capture System will be administered in children, adolescents and young adults with refractory EBV infection post Allogeneic Hematopoietic Stem Cell Transplantation (AlloHSCT) or with primary immunodeficiencies (PID).
CMV cytotoxic T cells (CTLs) manufactured with the Miltenyi CliniMACS Prodigy Cytokine Capture System will be administered in children, adolescents and young adults (CAYA) with refractory cytomegalovirus (CMV) infection post Allogeneic Hematopoietic Stem Cell Transplantation (AlloHSCT) or with primary immunodeficiencies (PID).
Related donor Adenovirus (ADV) specific cytotoxic T cells (CTLs) manufactured with the Miltenyi CliniMACS Prodigy Cytokine Capture System will be administered intravenously in in children, adolescents and young adults with refractory ADV infection post Allogeneic Hematopoietic Stem Cell Transplantation (AlloHSCT) or with primary immunodeficiencies (PID).
This aim of this research project is to determine if low to moderate level exercise can have an impact on stress, fatigue, and quality of life for individuals diagnosed with a primary immunodeficiency disease. This 8-week study will compare participants engaging in a semi-customized, home exercise program (exercise intervention group) to participants performing normal activities (non-exercise control group). This study will track stress, fatigue, and quality of life in individuals with a diagnosis of primary immunodeficiency disease, using standardized questionnaires, journals, and interviews.
The investigators wish to characterize alterations in the architecture and function of immune cells in patients with a primary immunodeficiency that also develop inflammatory bowel disease. In addition, The investigators' goal is to characterize the microbiome of these patients, in order to determine whether specific microbial alterations are related to inflammation.