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

Research output: Contribution to journalArticlepeer-review

27 Scopus citations


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
StatePublished - Jun 1 2017

Bibliographical note

Publisher Copyright:
© 2017 Elsevier Inc.


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


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