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

23 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

Funding Information:
This project was supported by Food and Drug Administration (grant number R01FD004091) and from the National Center for Research Resources (grant number UL1RR024134). The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Center for Research Resources or the National Institutes of Health. This project was supported in part by the Institute for Translational Medicine and Therapeutics (ITMAT) Transdisciplinary Program in Translational Medicine and Therapeutics (grant number UL1RR024134). This project also received support from the Children's Hospital of Philadelphia Clinical and Translational Science Award Research Center: NIH/NCATS (National Center for Advancing Translational Sciences), Grant UL1TR000003 (NCATS), The Jeffrey Modell Foundation and Texas Children's Hospital.

Publisher Copyright:
© 2017 Elsevier Inc.


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


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