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Updated: Mar 7, 2026
Intralymphatic Immunotherapy and Vaccination in Mice
Published on: February 2, 2014
Phase I trial of low-dose interleukin 2 therapy in patients with Wiskott-Aldrich syndrome
Soma Jyonouchi1, Brenda Gwafila2, Lavesh A Gwalani3
1Division of Allergy and Immunology, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA, United States.
Background:
Low 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.
Objectives:
A phase-I study to assess safety of low dose IL-2 in WAS.
Methods:
Patients received 5 daily subcutaneous IL-2 injections, every 2months, for three courses. A "3+3" dose escalation method was used.
Results:
6 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.
Conclusion:
We 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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