Hematopoietic stem cell transplantation in 29 patients hemizygous for hypomorphic IKBKG/NEMO mutations
Miot C., Imai K., Imai C., Mancini AJ., Kucuk ZY., Kawai T., Nishikomori R., Ito E., Pellier I., Dupuis Girod S., Rosain J., Sasaki S., Chandrakasan S., Pachlopnik Schmid J., Okano T., Colin E., Olaya-Vargas A., Yamazaki-Nakashimada M., Qasim W., Espinosa Padilla S., Jones A., Krol A., Cole N., Jolles S., Bleesing J., Vraetz T., Gennery AR., Abinun M., Güngör T., Costa-Carvalho B., Condino-Neto A., Veys P., Holland SM., Uzel G., Moshous D., Neven B., Blanche S., Ehl S., Döffinger R., Patel SY., Puel A., Bustamante J., Gelfand EW., Casanova J-L., Orange JS., Picard C.
Key Points Global survival rate was 74% at a median follow-up after HSCT of 57 months. Preexisting mycobacterial infection and colitis were associated with poor HSCT outcome.
