Wiskott-Aldrich syndrome (WAS) is a rare primary immune deficiency (PID). Because PIDs are so rare, medical centers have banded together into the Rare Disease Clinical Research Network-Primary Immune Deficiency Treatment Consortium. Memorial Sloan Kettering Cancer Center is part of this research group, which is conducting this study.
In one part of this study, researchers want to learn more about WAS by analyzing patients who received treatment for WAS (with either a bone marrow transplant or gene therapy) at least two years before enrolling in the study. The other part of this study will enroll patients who are newly diagnosed. These patients will be followed as they continue onward to get their treatment. Researchers will collect medical information and samples at routine medical visits to Memorial Sloan Kettering. Patients and their parents will also be asked to complete questionnaires about their quality of life.
The results of this study will help doctors better understand WAS and how to diagnose and treat it most effectively.