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 journalArticle

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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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Wiskott-Aldrich Syndrome
Interleukin-2
Natural Killer Cells
Therapeutics
T-Lymphocytes
Safety
Immunologic Adjuvants
Platelet Transfusion
Regulatory T-Lymphocytes
Thrombocytopenia
B-Lymphocytes
Blood Platelets
Cell Count
Injections

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., ... 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

Phase I trial of low-dose interleukin 2 therapy in patients with Wiskott-Aldrich syndrome. / Jyonouchi, Soma; Gwafila, Brenda; Gwalani, Lavesh A.; Ahmad, Maria; Moertel, Chistopher; Holbert, Cecil; Kim, Ji Young; Kobrinsky, Nathan; Roy-Ghanta, Sumita; Orange, Jordan S.

In: Clinical Immunology, Vol. 179, 01.06.2017, p. 47-53.

Research output: Contribution to journalArticle

Jyonouchi, S, Gwafila, B, Gwalani, LA, Ahmad, M, Moertel, C, Holbert, C, Kim, JY, Kobrinsky, N, Roy-Ghanta, S & Orange, JS 2017, 'Phase I trial of low-dose interleukin 2 therapy in patients with Wiskott-Aldrich syndrome', Clinical Immunology, vol. 179, pp. 47-53. https://doi.org/10.1016/j.clim.2017.02.001
Jyonouchi, Soma ; Gwafila, Brenda ; Gwalani, Lavesh A. ; Ahmad, Maria ; Moertel, Chistopher ; Holbert, Cecil ; Kim, Ji Young ; Kobrinsky, Nathan ; Roy-Ghanta, Sumita ; Orange, Jordan S. / Phase I trial of low-dose interleukin 2 therapy in patients with Wiskott-Aldrich syndrome. In: Clinical Immunology. 2017 ; Vol. 179. pp. 47-53.
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