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

Soma Jyonouchi, Brenda Gwafila, Lavesh A. Gwalani, Maria Ahmad, Chistopher Moertel, Cecil Holbert, Ji Young Kim, Nathan Kobrinsky, Sumita Roy-Ghanta, Jordan S. Orange

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Abstract

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 2 months, for three courses. A “3 + 3” dose escalation method was used. Results 6 patients received the 0.5 million units/m2/day dose without serious adverse events. However, 2 of 3 patients receiving the 1 million units/m2/day dose developed thrombocytopenia requiring platelet transfusions. A statistically significant platelet increase occurred in patients receiving the 0.5 million units/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.

Original languageEnglish (US)
Pages (from-to)47-53
Number of pages7
JournalClinical Immunology
Volume179
DOIs
StatePublished - Jun 1 2017

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Keywords

  • Low dose IL-2
  • Wiskott-Aldrich syndrome
  • X-linked thrombocytopenia

Cite this

Jyonouchi, S., Gwafila, B., Gwalani, L. A., Ahmad, M., Moertel, C., Holbert, C., Kim, J. Y., Kobrinsky, N., Roy-Ghanta, S., & Orange, J. S. (2017). Phase I trial of low-dose interleukin 2 therapy in patients with Wiskott-Aldrich syndrome. Clinical Immunology, 179, 47-53. https://doi.org/10.1016/j.clim.2017.02.001