Understanding a rare blood disorder that can lead to cancer

This is a long-term research study for people with familial platelet disorder with associated myeloid malignancy (FPDMM), a rare inherited condition caused by changes in the RUNX1 gene. Researchers will collect blood samples, genetic information, and health updates from participants over several years to understand how the condition develops, predict which patients may develop blood cancer, and identify new ways to help treat it.

FPDMM is a rare condition that increases the risk of developing blood cancer, but doctors don't yet know which patients will develop cancer or when it might happen. This study aims to find biological signs (biomarkers) that can predict disease progression and help doctors provide better care and earlier treatment.