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.

DOI

10.1182/blood-2017-03-771600

Type

Journal article

Publisher

American Society of Hematology

Publication Date

2017-09-21T00:00:00+00:00

Volume

130

Pages

1456 - 1467

Total pages

11

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