On behalf of the PIDTC 6906 Project Working Team and in collaboration with the IEWP, we are collecting data to assess the outcomes of patients with STAT1 Gain of Function of any age who have undergone hematopoietic cell transplantation since 2010, including survivors and non-survivors with any length of follow-up. Data collection will be performed via an electronic CRF (Castor software).
This project serves to update the report written by Jen Leiding and Troy Torgerson in 2018. Given that these patients will also likely be eligible for PIDTC Protocol 6906, we encourage you to save the data gathered for this sub-project so as to not duplicate efforts for entry of the patient onto 6906 if eligible.
Inclusion criteria are STAT1 GOF patients of any age post-HSCT (survivors and non-survivors alike). STAT1 mutations should be previously described as GOF or confer GOF in an in vitro model.
Data will be collected via eCRF. The CRF can be completed on a rolling basis (information is saved as completed) and will be open until February 28, 2023.