Open access
Disseminated Nontuberculous Mycobacterial Disease in NEMO and Iκbα Defects.
Chen Wang
A. Rivera
David E. Kleiner
C. Ma
M. V. Vander Lugt
J. LiPuma
Alyssa R. Letourneau
Janine R. Daub
Anahita Agharahimi
Annalie J. Harris
Lavenda Chirombo Kluczynski
Lindsey B. Rosen
Howard M. Lederman
M. E. Younger
Elias T. Zambidis
Maura Manion
Lu-Xin Pei
I. Sereti
Morgan N. Similuk
Danielle Fink
D. L. Long Priel
J. Gallin
D. Kuhns
A. Zelazny
G. Uzel
Amy P. Hsu
Christa S. Zerbe
Alexandra F. Freeman
Steven M. Holland
Medicine
Abstract
Disseminated nontuberculous mycobacterial disease is a severe complication of NEMO (IKBKG) and IκBα (NFKBIA) defects. It may present in adulthood, and diagnosis was often delayed. Despite intensive multidrug therapy and adjunctive IFN-γ, treatment responses were frequently inadequate. Hematopoietic cell transplantation may offer immune reconstitution but carries risks.