Phagocytic cell dysfunction

This study looked at whether the HPV vaccine works in girls aged 9 to 17 who are taking medicines that weaken their immune system, such as after an organ transplant or for diseases like lupus. The goal was to see if their bodies produce enough antibodies to protect against HPV. T…

This study tested the drug tofacitinib (Xeljanz) in 10 adults with chronic granulomatous disease (CGD) who had ongoing inflammation in the gut, skin, or lungs. Participants took the pill daily for 3 months to see if it was safe and could help control symptoms. The goal was to fin…

This phase 2 trial tested the drug samotolisib in 18 children and young adults with advanced solid tumors, non-Hodgkin lymphoma, or histiocytic disorders that had spread, returned, or stopped responding to treatment. Only patients whose tumors had specific mutations in the TSC or…

This study tested a drug called tazemetostat in 20 children whose cancers had come back or weren't responding to treatment. The children had specific gene changes (EZH2, SMARCB1, or SMARCA4) in their tumors. The goal was to see if the drug could shrink or slow the growth of these…

This study tested a drug called palbociclib in 23 children and young adults (ages 1–21) whose cancers had come back or didn't respond to treatment. The drug works by blocking proteins that help cancer cells grow. The goal was to see if it could shrink tumors or slow the disease i…

This study tested whether a liquid diet called Neocate Splash could change gut bacteria and ease stomach symptoms in people with chronic granulomatous disease (CGD) and inflammatory bowel disease (IBD). Three participants followed the diet for up to 4 weeks, with hospital stays a…

This study looked at 137 people with Chronic Granulomatous Disease (CGD), a rare immune disorder. It compared those who received a stem cell transplant with those who got standard care. The goal was to see which patients benefit most from transplant and what type of transplant wo…

This study tested a document called Voicing My CHOiCES, designed to help young adults (ages 18-39) with serious illnesses like cancer or HIV discuss their care preferences. Participants filled out questionnaires about anxiety, social support, and communication, then reviewed the …

This completed study looked at people with genetic conditions that affect how the lungs clear mucus, such as cystic fibrosis and primary ciliary dyskinesia. Researchers examined 87 participants, including healthy volunteers, to understand why these patients get repeated lung infe…

This study collected blood, saliva, stool, urine, and skin samples from 19 people with unknown or poorly understood immune system problems. The goal was to learn how different parts of the immune system work together. No treatments or drugs were tested. Participants were followed…