Phase I trial of low-dose interleukin 2 therapy in patients with Wiskott-Aldrich syndrome

Publication date: Available online 14 February 2017
Source:Clinical Immunology
Author(s): Soma Jyonouchi, Brenda Gwafila, Lavesh A. Gwalani, Maria Ahmad, Chistopher Moertel, Cecil Holbert, Ji Young Kim, Nathan Kobrinsky, Sumita Roy-Ghanta, Jordan S. Orange
BackgroundLow dose IL-2 can restore the function of T and NK cells from Wiskott-Aldrich (WAS) patients. However, the safety of in vivo IL-2 in WAS is unknown.ObjectivesA phase-I study to assess safety of low dose IL-2 in WAS.MethodsPatients received 5 daily subcutaneous IL-2 injections, every 2months, for three courses. A "3+3" dose escalation method was used.Results6 patients received the 0.5millionunits/m²/day dose without serious adverse events. However, 2 of 3 patients receiving the 1millionunits/m²/day dose developed thrombocytopenia requiring platelet transfusions. A statistically significant platelet increase occurred in patients receiving the 0.5millionunits/m²/day dose. A trend toward higher T, B and NK cell numbers and higher T regulatory cell percentages was observed.ConclusionWe have identified a safe IL-2 dose for WAS patients. Additional trials are indicated to study the efficacy of this immunostimulant as a therapy for WAS.



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