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 language | English (US) |
---|---|
Pages (from-to) | 47-53 |
Number of pages | 7 |
Journal | Clinical Immunology |
Volume | 179 |
DOIs | |
State | Published - Jun 1 2017 |
Bibliographical note
Publisher Copyright:© 2017 Elsevier Inc.
Keywords
- Low dose IL-2
- Wiskott-Aldrich syndrome
- X-linked thrombocytopenia