Patient characteristics and treatments.
hypogammaglobulinemia, WHIM
WARTS, HYPOGAMMAGLOBULINEMIA
We enrolled 20 patients with WHIM at the NIH Clinical Center (NIH-CC) from October 14, 2014, through November 6, 2017 (Thirteen patients had all 4 acronymic WHIM phenotypes. Two patients lacked only hypogammaglobulinemia, 2 lacked only warts, and 2 lacked both warts and hypogammaglobulinemia (At enrollment, 8 of the 15 patients with a history of hypogammaglobulinemia were receiving supplemental immunoglobulin, and 2 patients were receiving prophylactic oral antibiotics; these treatments were continued (The protocol began with a 0.5- to 4-month prerandomization screening phase (The screening phase concluded with randomization, followed by a 2-day washout of G-CSF to determine baseline blood cell counts, and then by a quadruple-masked 28-month crossover treatment period composed of two 12-month treatment phases, each preceded by a 2-month dose-finding drug equilibration phase (During the equilibration phase, no patients required adjustment of the G-CSF starting dose (Initial drug doses during the treatment phase were the same as the final drug doses during the preceding equilibration phase for each patient and were maintained within, slightly above, or slightly below the prespecified target ranges (